Moorestown, New Jersey, United States
biotecHOPE™ 2023
biotecHOPE™ 2023
Crinecerfont
APHEXDA™ (motixafortide) in Combination with Filgrastim (G-CSF)
AFM13 IN COMBINATION WITH ALLONK®
Neurocrine Biosciences Announces Positive Top-Line Data from Phase 3 Study of Crinecerfont in Adults for the Treatment of Congenital Adrenal Hyperplasia (CAH)
09/12/23
CAHtalyst™ Met Primary Endpoint Demonstrating a Statistically Significant Decrease from Baseline Daily Glucocorticoid Dose with Androgen Control
Key Secondary Endpoint Achieved Statistically Significant Decrease in Androstenedione at Week 4 versus Placebo
Key Secondary Endpoint Demonstrated a Statistically Significant Number of Patients on Crinecerfont Achieved a Reduction to a Physiologic Glucocorticoid Dose versus Placebo
Crinecerfont Was Generally Well-Tolerated
SAN DIEGO, Sept. 12, 2023 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced positive top-line data from the Phase 3 CAHtalyst™ Adult Study evaluating the efficacy, safety, and tolerability of crinecerfont in adults with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21-OHD).
The Phase 3 study met its primary endpoint at Week 24, demonstrating that treatment with crinecerfont resulted in a statistically significant percent reduction in daily glucocorticoid (GC) dose versus placebo while maintaining androgen control (p-value <0.0001).
The study also met important key secondary endpoints, with a statistically significant decrease in androstenedione at Week 4 versus placebo (p-value <0.0001). At Week 24, approximately 63% of patients on crinecerfont achieved a reduction to a physiologic GC dose versus approximately 18% on placebo (p-value <0.0001).
Crinecerfont was generally well tolerated. The most common adverse events during the double-blind, placebo-controlled period of the trial were fatigue, headache, and coronavirus infection. There were few serious adverse events, with none assessed as related to crinecerfont.
"I am gratified to see the extremely positive and clinically meaningful results from this study, the largest ever interventional trial conducted in this rare disease. It required a global effort, and the top-line results confirm our confidence in crinecerfont as a potential first-in-class medication and first-ever non-glucocorticoid treatment option for patients living with CAH," said Richard Auchus, M.D., Ph.D., Principal Investigator, Professor of Pharmacology and Internal Medicine, Division of Metabolism, Endocrinology, and Diabetes at the University of Michigan. "It has been 60 years since we've seen a significant treatment advance for patients with CAH, and the data from this study suggest that crinecerfont might improve their outcomes and quality of life."
"CAH patients suffer from a number of debilitating symptoms and have had suboptimal treatment options with existing standard of care for their whole lives. These data, along with data from the open label treatment period, will allow us to proceed with our regulatory submissions to the FDA in 2024 and European Medicines Agency afterwards," said Kevin Gorman, Ph.D., Chief Executive Officer, Neurocrine Biosciences.
"CAH is a difficult disorder to live with for patients and their caregivers, taking a huge toll physically and mentally," said Eiry Roberts, M.D., Chief Medical Officer at Neurocrine Biosciences. "For physicians, the current treatment paradigm is problematic, relying on glucocorticoids for a dual purpose: not only to address the underlying cortisol deficiency but typically at supraphysiologic doses to treat androgen excess resulting in well-known complications over the long-term. The CAHtalyst Phase 3 Adult data bring us one step closer to a new approach to treating CAH with a therapy that has demonstrated the ability to substantially reduce glucocorticoid doses while maintaining or improving androgen control."
Additional information regarding the results from the Phase 3 CAHtalyst study will be discussed at the Morgan Stanley 21st Annual Global Healthcare Conference at 10:50 a.m. Eastern Time on September 12 in New York. The live presentation will be webcast and may be accessed on the Company's website under Investors at www.neurocrine.com.
A replay of the presentation will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month. Additional data from the Phase 3 CAHtalyst study will be provided in a peer-reviewed medical journal or at a medical conference at a future date.
Data from the Phase 3 CAHtalyst Pediatric Study will be available, as planned, in early Q4 2023.
About Classic Congenital Adrenal Hyperplasia
Congenital adrenal hyperplasia (CAH) refers to a group of genetic conditions that result in an enzyme deficiency that alters the production of adrenal hormones which are essential for life. Approximately 95% of CAH cases are caused by a mutation that leads to deficiency of the enzyme 21-hydroxylase (21-OHD). In classic CAH, severe deficiency of this enzyme leads to an inability of the adrenal glands to produce cortisol and, in approximately 75% of cases, aldosterone. If left untreated, classic CAH can result in salt wasting, dehydration, and even death.
There are currently no non-glucocorticoid treatments approved by the U.S. Food and Drug Administration (FDA) for classic CAH. Glucocorticoids (GCs), the current standard of care, are used not only to correct the endogenous cortisol deficiency but typically used at greater than physiologic (supraphysiologic) doses to try to suppress the high levels of corticotropin-releasing factor (CRF) and adrenocorticotropic hormone (ACTH) that result in androgen excess. However, glucocorticoid treatment at supraphysiologic doses has been associated with serious and significant complications of steroid excess, including metabolic issues such as weight gain and diabetes, cardiovascular disease, and osteoporosis. Additionally, long-term treatment with supraphysiologic GC doses may have psychological and cognitive impact such as changes in mood and memory. Androgen excess has been associated with abnormal bone growth and development in pediatric patients, female health problems such as acne, excess hair growth and menstrual irregularities, testicular rest tumors in males, and fertility issues in both sexes. To learn more about CAH, click here.
About Crinecerfont
Crinecerfont is an investigational, oral, selective corticotropin-releasing factor type 1 receptor (CRF1) antagonist being developed to reduce and control excess adrenal androgens through a steroid-independent mechanism for the treatment of congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency (21-OHD). Antagonism of CRF type 1 receptors in the pituitary has been shown to decrease adrenocorticotropic hormone (ACTH) levels, which in turn decreases the production of adrenal androgens and potentially the symptoms associated with classic CAH. Our data demonstrates that lowering androgen levels enables lower, more physiologic dosing of glucocorticoids and thus could potentially reduce the complications associated with exposure to greater than normal glucocorticoid doses in patients with classic CAH.
To learn more about crinecerfont, click here.
About the CAHtalyst™ Phase 3 Study in Adults
The CAHtalyst™ Phase 3 global registrational study was designed to evaluate the safety, efficacy, and tolerability of crinecerfont in adults (18 years of age and older) with classic congenital adrenal hyperplasia (CAH) due to 21-OHD. The study enrolled 182 female and male patients with CAH and consisted of a 24-week randomized, double-blind, placebo-controlled period followed by one-year of open-label crinecerfont treatment and optional open-label extension. The study started in December 2020, and the open-label treatment portion is still ongoing.
For more information about the CAHtalyst Phase 3 study in adults, please visit Clinical TrialsAdult.gov.
For more information about the CAHtalyst Pediatric Phase 3 study, please visit ClinicalTrialsPediatric.gov.
About Neurocrine Biosciences
Neurocrine Biosciences is a leading neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs, but few options. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, neuroendocrine and neuropsychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, Parkinson's disease, endometriosis* and uterine fibroids*, as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders, because you deserve brave science. For more information, visit neurocrine.com, and follow the company on LinkedIn, Twitter, and Facebook.
(*in collaboration with AbbVie)
NEUROCRINE, CAHtalyst, and the Neurocrine logo are registered trademarks of Neurocrine Biosciences, Inc.
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SOURCE Neurocrine Biosciences, Inc.
Neurocrine gains after Phase 3 win for genetic disorder drug
Sep. 12, 2023 11:57 AM ET
Neurocrine Biosciences, Inc. (NBIX)
By: Dulan Lokuwithana, SA News Editor
- Neurocrine Biosciences (NASDAQ:NBIX) traded higher Tuesday after announcing that its Phase 3 CAHtalyst trial for crinecerfont met its primary endpoint in adults with classic congenital adrenal hyperplasia (CAH), a group of genetic disorders.
- CAHtalyst was a global registrational study designed to evaluate the corticotropin-releasing factor type 1 receptor (CRF1) antagonist in adults with CAH due to 21-hydroxylase deficiency (21-OHD).
- Glucocorticoids are considered standard care in CAH despite potential implications such as androgen excess, weight gain, and diabetes at high doses.
- https://seekingalpha.com/news/4011036-neurocrine-stock-gains-after-phase-3-win-crinecerfont
- Neurocrine Biosciences, Inc. (NBIX)
- https://www.neurocrine.com/
- https://www.neurocrine.com/pipeline/
- https://seekingalpha.com/symbol/NBIX
AFM13 IN COMBINATION WITH ALLONK®
APHEXDA™ (motixafortide) in Combination with Filgrastim (G-CSF)
AFM13 IN COMBINATION WITH ALLONK®
AFFIMED RECEIVES FDA FAST TRACK DESIGNATION FOR AFM13 IN COMBINATION WITH ALLONK® FOR THE TREATMENT OF PATIENTS WITH RELAPSED OR REFRACTORY HODGKIN LYMPHOMA
Press Release - September 12, 2023
- AFM13 in combination with AlloNK® (also known as AB-101) will be investigated in the LuminICE-203 open-label, multi-center, multi-cohort, phase 2 study evaluating the efficacy and safety of the treatment in patients with relapsed/refractory Hodgkin lymphoma
- The FDA’s fast track designation is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need
Mannheim, Germany, September 12, 2023 – Affimed N.V. (Nasdaq: AFMD) (“Affimed”, or the “Company”), a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer, today announced that the FDA has granted fast track designation to the combination of its innate cell engager (ICE®) AFM13 with AlloNK® for the potential treatment of relapsed/refractory (r/r) Hodgkin lymphoma (HL). The combination treatment is entering Phase 2 development and will be investigated in Affimed’s LuminICE-203 study (NCT05883449), which received IND-clearance earlier this year; the study also includes an exploratory cohort of CD30-positive peripheral T-cell lymphoma patients.
“Our clinical data of AFM13 in combination with allogeneic NK cells has shown outstanding efficacy and a well-managed safety profile in late-stage, muti-refractory, patients with r/r Hodgkin and Non-Hodgkin lymphoma,” said Dr. Wolfgang Fischer, Chief Operating Officer at Affimed. “The FDA fast track designation is a testament to the powerful potential our combination approach may deliver for these patients in high need, and we remain committed to working closely with the FDA to expedite development of this important therapy.”
Fast Track is a process designed to facilitate the development, and expedite the review, of new drugs that are intended to treat or prevent serious conditions and have the potential to address an unmet medical need. The FDA’s decision is based on available data showing the potential of the AFM13 and AlloNK® combination therapy to overcome current limitations in the treatment of r/r HL. With the Fast Track Designation, the therapeutic development of the combination can benefit from more frequent engagement with the FDA, which will support the collection of appropriate data needed to accelerate its development.
LuminICE-203 builds on the clinical findings from the phase 1/2 AFM13-104 trial (NCT04074746), in which investigators assessed AFM13 in combination with cord blood-derived natural killer cells in heavily pretreated patients with CD30-positive Hodgkin lymphoma and non-Hodgkin lymphoma. Data presented to date from this trial have shown outstanding clinical results in late-stage, multi-refractory, patients with a 94% overall response rate (ORR), a 71% complete response (CR) rate and a well-managed safety profile at the recommended phase 2 dose (RP2D); specifically in the 31 r/r HL patients treated, the ORR and CR were 97% and 77% respectively (see press release here).
About FDA Fast Track Designation
Fast track is a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need. Its purpose is to get important new drugs to patients earlier. Fast Track addresses a broad range of serious conditions. With Fast Track Designation, a new therapy is eligible for some or all of the following:
- More frequent meetings with FDA to discuss the drug’s development plan and ensure collection of appropriate data needed to support drug approval
- More frequent written communication from FDA about such things as the design of the proposed clinical trials and use of biomarkers
- Eligibility for Accelerated Approval and Priority Review, if relevant criteria are met
- Rolling Review, which means that a drug company can submit completed sections of its Biologic License Application (BLA) or New Drug Application (NDA) for review by FDA, rather than waiting until every section of the NDA is completed before the entire application can be reviewed. BLA or NDA review usually does not begin until the drug company has submitted the entire application to the FDA.
Please refer to Fast Track | FDA for further information.
About AFM13
AFM13 is a first-in-class innate cell engager (ICE®) that uniquely activates the innate immune system to destroy CD30-positive hematologic tumors. AFM13 induces specific and selective killing of CD30-positive tumor cells, leveraging the power of the innate immune system by engaging and activating natural killer (NK) cells and macrophages. AFM13 is Affimed’s most advanced ICE® clinical program and was evaluated as monotherapy in a phase 2B trial in patients with relapsed/refractory peripheral T cell lymphoma (REDIRECT). Additional details can be found at www.clinicaltrials.gov (NCT04101331). The study achieved an ORR of 32.4% demonstrating anti-tumor activity with a DOR of 2.3 months and a well-managed safety profile. AFM13 is a tetravalent bispecific innate cell engager designed to act as a bridge between the innate immune cells and the tumor creating the necessary proximity for the innate immune cells to specifically destroy the tumor cells. About Affimed N.V. Affimed (Nasdaq: AFMD) is a clinical-stage immuno-oncology company committed to giving patients back their innate ability to fight cancer by actualizing the untapped potential of the innate immune system. The Company’s proprietary ROCK® platform enables a tumor-targeted approach to recognize and kill a range of hematologic and solid tumors, enabling a broad pipeline of wholly-owned and partnered single agent and combination therapy programs. The ROCK® platform predictably generates customized innate cell engager (ICE®) molecules, which use patients’ immune cells to destroy tumor cells. This innovative approach enabled Affimed to become the first company with a clinical-stage ICE®. Headquartered in Mannheim, Germany, with offices in New York, NY, Affimed is led by an experienced team of biotechnology and pharmaceutical leaders united by a bold vision to stop cancer from ever derailing patients’ lives. For more about the Company’s people, pipeline and partners, please visit: www.affimed.com
About AlloNK®
AlloNK® (also known as AB-101) is a non-genetically modified, cord blood-derived, allogeneic, cryopreserved, ADCC-enhancing NK cell therapy candidate for use in combination with monoclonal antibodies or innate-cell engagers in the out-patient setting. Artiva is investigating AlloNK® in a Phase 1/2 multicenter clinical trial (ClinicalTrials.gov Identifier: NCT04673617 ) to assess the safety and clinical activity of AlloNK® alone and in combination with the anti-CD20 monoclonal antibody, rituximab, in patients with relapsed or refractory B-cell-non-Hodgkin lymphoma (B-NHL). Artiva is also investigating the safety and clinical activity of AlloNK® in combination with rituximab in patients with lupus nephritis. Artiva selects cord blood units with the high affinity variant of the CD16 receptor and a KIR-B haplotype for enhanced product activity. Using the company’s cell therapy manufacturing platform, Artiva can generate thousands of doses of AlloNK® from a single umbilical cord blood unit while retaining the high and consistent expression of CD16 and other activating NK receptors, without the need for engineering. AlloNK® is being administered in the outpatient setting over multiple doses and multiple cycles.
About Artiva Biotherapeutics
Artiva’s mission is to deliver highly effective, off-the-shelf, allogeneic NK cell-based therapies that are safe and accessible to patients. Artiva has taken a Manufacturing-First approach to create a highly scaled process integrating cell expansion, activation, and engineering technology developed by Artiva’s strategic partner, GC Cell Corporation, a member of the GC family of companies, a leading healthcare company in Korea. Artiva’s pipeline includes AlloNK®, an ADCC enhancer NK-cell therapy candidate for use in combination with monoclonal antibodies or innate-cell engagers. Artiva’s pipeline also includes AB-201, an anti-HER2 CAR-NK cell therapy candidate for the treatment of HER2-overexpressing tumors, such as breast, gastric, and bladder cancers, and for which an IND has been allowed by FDA, and a pipeline of CAR-NK candidates. Artiva is headquartered in San Diego. For more information, visit www.artivabio.com.
Affimed gains on FDA fast track tag for lymphoma therapy
Sep. 12, 2023 7:20 AM ET
By: Dulan Lokuwithana, SA News Editor
- German biotech Affimed N.V. (NASDAQ:AFMD) added ~10% pre-market Tuesday after announcing that the U.S. Food and Drug Administration (FDA) issued its fast-track designation for an experimental therapy for Hodgkin lymphoma.
- The treatment is composed of Affimed’s (AFMD) innate cell engager (ICE) AFM13 and Artiva Biotherapeutics’ (RTVA) NK cell therapy candidate AlloNK. The fast track designation covers its use in relapsed/refractory (r/r) Hodgkin lymphoma (HL).
- A Phase 2 trial, known as LuminICE-203, is expected to test the combo in patients with r/r HL and those with CD30-positive peripheral T-cell lymphoma.
- https://seekingalpha.com/news/4010889-affimed-stock-gains-fda-fast-track-tag-lymphoma-drug
- Affimed N.V. (AFMD), RTVA
- https://www.affimed.com/
- https://www.affimed.com/pipeline/
APHEXDA™ (motixafortide) in Combination with Filgrastim (G-CSF)
APHEXDA™ (motixafortide) in Combination with Filgrastim (G-CSF)
APHEXDA™ (motixafortide) in Combination with Filgrastim (G-CSF)
BioLineRx Announces FDA Approval of APHEXDA™ (motixafortide) in Combination with Filgrastim (G-CSF) to Mobilize Hematopoietic Stem Cells for Collection and Subsequent Autologous Transplantation in Patients with Multiple Myeloma
September 11, 2023
- APHEXDA is the first innovation in stem cell mobilization for multiple myeloma to be approved in the U.S. in a decade -
- One dosage of APHEXDA plus filgrastim enabled a majority of patients to achieve the collection goal of ≥ 6 million hematopoietic stem cells among a contemporary population of multiple myeloma patients -
- Management to hold conference call on Tuesday, September 12, 2023 at 8:00 a.m. U.S. EDT -
TEL AVIV, Israel, Sept. 11, 2023 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ/TASE: BLRX), a commercial stage biopharmaceutical company focused on certain cancers and rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved APHEXDA™ (motixafortide) in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma. APHEXDA is administered by injection, for subcutaneous use.
Experience the full interactive Multichannel News Release here: https://www.multivu.com/players/English/9174951-biolinerx-fda-approval-aphexda/
Multiple myeloma is the second most-common hematologic malignancy. Autologous stem cell transplantation (ASCT) is part of the standard of care treatment paradigm for multiple myeloma and delivers prolonged survival for patients with this cancer type.1 The success of ASCT depends on adequate mobilization of stem cells during the treatment process. The American Society for Transplantation and Cellular Therapy (ASTCT) guidelines recommend a collection target of 3-5 x 106 CD34+ cells/kg.2 Additionally, collection of a sufficient number of stem cells to perform two transplantations is recommended.2-5 Historically, depending on induction regimens and mobilization strategies, up to 47% of patients have had challenges collecting target numbers of hematopoietic stem cells for ASCT after one apheresis session.6-7
"Greater numbers of patients with multiple myeloma are candidates for autologous stem cell therapy; however, achieving target collection goals can be difficult in some patients given modern barriers, including the treatment of older patients and use of contemporary induction regimens," said John DiPersio, MD, PhD, primary investigator for the GENESIS trial and
Professor of Medicine, Pathology and Immunology and Director of the Center for Gene and Cellular Immunotherapy at Washington University School of Medicine in St. Louis. "Innovation in this area of medicine has been needed, and today's approval of APHEXDA addresses the demand for new therapies that can meet today's challenges by delivering more reliability in stem cell mobilization, versus filgrastim alone, with fewer days of apheresis sessions and fewer doses of filgrastim for people living with this cancer."
The FDA approval of APHEXDA is based on results from the 2-part, Phase 3 GENESIS trial, a randomized, double-blind, placebo-controlled study evaluating the safety and efficacy of APHEXDA (motixafortide) plus filgrastim, compared to placebo plus filgrastim, for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients. Part 1 was a single center, lead-in, open-label study involving 12 patients treated with motixafortide plus filgrastim designed to ascertain the dose. Part 2 involved 122 patients who were randomized 2:1 in a double-blind, placebo-controlled, multicenter study.8
The assessment of CD34+ cells was performed by central and local laboratories. Central laboratory assessments were used for the efficacy results. Local laboratory results were used for clinical treatment decisions. APHEXDA plus filgrastim enabled 67.5% of patients to achieve the stem cell collection goal of ≥ 6 × 106 CD34+ cells/kg within two apheresis sessions, versus 9.5% for the placebo plus filgrastim regimen, as measured by central laboratory.9 Additionally, 92.5% of patients reached the stem cell collection goal in up to two apheresis sessions in the APHEXDA arm and 21.4% in the placebo arm, as measured by local laboratories.9 Local laboratory data were used for a sensitivity analysis. The data are descriptive and were not statistically powered nor prespecified. The information should be cautiously interpreted.
In GENESIS, the safety was evaluated in 92 patients with multiple myeloma who received APHEXDA 1.25 mg/kg subcutaneously plus filgrastim, and 42 patients who received placebo plus filgrastim. Serious adverse reactions occurred in 5.4% of patients receiving APHEXDA plus filgrastim. These reactions included vomiting, injection site reaction, hypersensitivity reaction, injection site cellulitis, hypokalemia and hypoxia. The most common adverse reactions occurring in GENESIS (incidence >20%) were injection site reactions (pain, erythema and pruritus), pruritus, flushing, and back pain.9
"Given the strong efficacy data shown in the GENESIS trial, which included patients who are representative of the current multiple myeloma patient population, we believe APHEXDA will play a critical role in addressing unmet needs and introduce a new treatment paradigm for this challenging cancer," said Philip Serlin, Chief Executive Officer of BioLineRx Ltd. "The company is working relentlessly to make this important innovation in stem cell mobilization available to appropriate patients, their physicians and transplant teams."
"FDA approval of APHEXDA, the company's first approved therapeutic, is a tremendously exciting and important moment in our history and validates our drug development programs," said Ella Sorani, PhD, Chief Development Officer of BioLineRx Ltd. "We would like to thank all of the patients and families who have contributed to the research and development of APHEXDA."
Increased age, as well as exposure to lenalidomide-containing induction regimens, including 3-4 drug combination regimens, have been associated with impaired stem cell mobilization.2-3 The GENESIS study included patients considered representative of the typical multiple myeloma population undergoing ASCT, with a median age of 63 years and with ~70% of patients in both arms of the trial receiving lenalidomide-containing induction therapy.8 In this contemporary population, patients in the APHEXDA plus filgrastim arm were able to mobilize more than four times the amount of stem cells with a single dose over a 24-hour period compared with placebo plus filgrastim.8
BioLineRx expects to make APHEXDA available later this month. For further information about APHEXDA, please see the Important Safety Information below and the full Prescribing Information, and visit www.APHEXDA.com.
APHEXDA Investor Conference Call
The Company will host an investor conference call on September 12, 2023 at 8:00 a.m. EDT featuring remarks by Philip Serlin, Chief Executive Officer.
To access the conference call, please dial +1-888-281-1167 from the U.S. or +972-3-918-0685 internationally. A live webcast and a replay of the call can be accessed through the event page on the Company's website. Please allow extra time prior to the call to visit the site and download any necessary software to listen to the live broadcast. The call replay will be available approximately two hours after completion of the live conference call. A dial-in replay of the call will be available until September 14, 2023; please dial +1-888-295-2634 from the US or +972-3-925-5904 internationally.
About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that affects some white blood cells called plasma cells, which are found in the bone marrow. When damaged, these plasma cells rapidly spread and replace normal cells in the bone marrow. According to the American Cancer Society, in 2023, it is estimated that more than 35,000 people will be diagnosed with multiple myeloma, and nearly 13,000 people will die from the disease in the U.S.10 While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels, kidney problems, or infections.
About Autologous Stem Cell Transplantation
Autologous stem cell transplantation (ASCT) is part of the standard treatment paradigm for a number of blood cancers, including multiple myeloma. In the U.S., as many as 8,000 ASCTs are performed each year in patients with multiple myeloma.11 The current ASCT standard of care includes 4-6 cycles of induction therapy (an initial drug-combination regimen to position the patient for as deep a treatment response as possible). To begin the stem cell mobilization process, a patient will receive a daily dose of filgrastim (G-CSF) for four days. Daily doses of filgrastim will continue until the target collection goal is met with the addition of up to four daily doses of plerixafor as needed.12 For patients unable to mobilize sufficient numbers of cells for harvesting during this primary mobilization phase, rescue therapy may be carried out followed by an additional number of apheresis sessions as necessary.2
About the GENESIS Trial
GENESIS (NCT 03246529) is a 2-part, Phase-3, randomized, double-blind, placebo-controlled, multicenter study evaluating the safety and efficacy of APHEXDA (motixafortide) plus filgrastim (G-CSF), compared to placebo plus filgrastim, for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients. Part 1 was a single center, lead-in, open-label study involving 12 patients treated with motixafortide plus filgrastim designed to ascertain the dose. Part 2 involved 122 patients who were randomized 2:1 in a double-blind, placebo-controlled, multicenter study.8
The primary objective of the study was to evaluate if one dose of motixafortide plus filgrastim is superior to placebo plus filgrastim in the ability to mobilize ≥ 6 million CD34+ cells in up to two apheresis sessions. A key secondary objective of the study was to evaluate if one dose of motixafortide plus filgrastim is superior to placebo plus filgrastim in the ability to mobilize ≥ 6 million CD34+ cells in one apheresis session.8
The study met the primary endpoint with a high degree of statistical significance (p<0.0001). The assessment of CD34+ cells was performed by central and local laboratories. Central laboratory assessments were used for the efficacy results. Local laboratory results were used for clinical treatment decisions. APHEXDA plus filgrastim enabled 67.5% of patients to achieve the cell collection goal of ≥ 6 × 106 CD34+ cells/kg in up to two apheresis sessions with a single administration, versus 9.5% for the placebo plus filgrastim regimen, as measured by central laboratory.9 Additionally, 92.5% of patients reached the stem cell collection goal in up to two apheresis sessions in the APHEXDA arm and 21.4% in the placebo arm, as measured by local laboratories.13 Local laboratory data were used for a sensitivity analysis. The data are descriptive and were not statistically powered nor prespecified. The information should be cautiously interpreted.
The safety of APHEXDA was evaluated in 92 patients with multiple myeloma who received APHEXDA 1.25 mg/kg subcutaneously plus filgrastim and 42 patients who received placebo plus filgrastim for mobilization of hematopoietic stem cells for collection and apheresis. Serious adverse reactions occurred in 5.4% of patients receiving APHEXDA plus filgrastim. Serious adverse reactions included vomiting, injection site reaction, hypersensitivity reaction, injection site cellulitis, hypokalemia and hypoxia. The most common adverse reactions occurring in GENESIS (incidence >20%) were injection site reactions (pain, erythema, and pruritus), pruritus, flushing and back pain.9
Please see important safety information below.
About APHEXDA™
APHEXDA (motixafortide) is a CXCR4 antagonist with long receptor occupancy (greater than 72 hours) that, in combination with filgrastim (G-CSF), enables mobilization of hematopoietic stem cells to the peripheral blood for collection and subsequent autologous stem cell transplantation in patients with multiple myeloma.9
INDICATION AND IMPORTANT SAFETY INFORMATION
INDICATION
APHEXDA is indicated in combination with filgrastim (G-CSF) to mobilize hematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in patients with multiple myeloma.
https://biolinerx.com/motixafortide-in-hsc-mobilization/
Please see the accompanying full Prescribing Information.
About BioLineRx
BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a commercial stage biopharmaceutical company pursuing life-changing therapies for certain cancers and rare diseases. The company's first approved product is APHEXDA™ (motixafortide) with an indication in the U.S. for stem cell mobilization for autologous transplantation in multiple myeloma. BioLineRx is advancing a pipeline of investigational medicines for patients with sickle cell disease, pancreatic cancer, and other solid tumors. Headquartered in Israel, and with operations in the U.S., the company is driving innovative therapeutics with end-to-end expertise in development and commercialization, ensuring life-changing discoveries move beyond the bench to the bedside.
Learn more about who we are, what we do, and how we do it at www.biolinerx.com, or on Twitter and LinkedIn.
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SOURCE BioLineRx Ltd.
BioLineRx gains after winning first FDA approval
Sep. 11, 2023 7:47 AM ET
BioLineRx Ltd. (BLRX)By: Dulan Lokuwithana, SA News Editor
- Israel-based biotech BioLineRx Ltd. (NASDAQ:BLRX) added ~23% pre-market Monday after announcing that the FDA greenlighted its lead asset Aphexda (motixafortide) for patients with multiple myeloma, marking the company's first regulatory approval.
- Accordingly, the subcutaneous injection will be indicated in the U.S. with filgrastim for multiple myeloma patients to mobilize hematopoietic stem cells to the peripheral blood. The company expects to launch the product later this month.
- https://seekingalpha.com/news/4010520-biolinerx-stock-gains-first-fda-approva
- BioLineRx Ltd. (BLRX)
- https://ir.biolinerx.com/
- https://biolinerx.com/pipeline/
- https://biolinerx.com/motixafortide-in-hsc-mobilization/
PALTUSOTINE (ORAL SST2 AGONIST)
PALTUSOTINE (ORAL SST2 AGONIST)
APHEXDA™ (motixafortide) in Combination with Filgrastim (G-CSF)
CRINETICS’ ONCE-DAILY ORAL PALTUSOTINE ACHIEVED THE PRIMARY AND ALL SECONDARY ENDPOINTS IN THE PHASE 3 PATHFNDR-1 STUDY EVALUATING TREATMENT OF PATIENTS WITH ACROMEGALY
- 83% of Participants on Paltusotine Maintained IGF-1 ≤1.0 xULN vs. 4% on placebo (p<0.0001)
- Mean IGF-1 Levels Were Maintained on Paltusotine vs. an Increase on Placebo (p<0.0001) After Switching from Injected Depot Standard of Care
- Mean Acromegaly Symptom Diary Scores Were Maintained on Paltusotine vs. an Increase on Placebo (p=0.02) After Switching from Injected Depot Standard of Care
- Paltusotine Was Well-Tolerated with No Severe or Serious Adverse Events
- Management Will Host a Conference Call Monday, September 11, 2023 at 8:00 a.m. Eastern Time
SAN DIEGO, September 10, 2023 — Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that paltusotine, an oral, once-daily investigational compound, achieved positive results by meeting the primary endpoint and all secondary endpoints of the Phase 3 PATHFNDR-1 study (NCT04837040). PATHFNDR-1 was a randomized, double-blind, placebo-controlled 36-week treatment period followed by an optional open-label extension study evaluating paltusotine in participants with acromegaly switching from standard-of-care injected depot somatostatin analogs. The study enrolled participants with acromegaly who were biochemically controlled on octreotide or lanreotide depot monotherapy. PATHFNDR-1 is one of two ongoing, placebo-controlled Phase 3 studies of once-daily, oral paltusotine.
The study met statistical significance (p<0.0001) on the primary endpoint, based on the proportion of participants taking paltusotine (83%) who maintained an insulin-like growth factor 1 (IGF-1) level ≤ 1.0 times the upper limit of normal (xULN) compared to those taking placebo (4%). All secondary endpoints also met statistical significance:
“The results of PATHFNDR-1 are relevant to the patients we see every day in clinical practice who are biochemically controlled on standard-of-care injections. My colleagues and I are increasingly convinced many patients would appreciate an oral alternative which confers similar benefits without the burden and discomfort of the injections,” stated Monica R. Gadelha, M.D., Ph.D., professor of endocrinology at the Medical School of the Universidade Federal do Rio de Janeiro and a principal investigator in the PATHFNDR program. “This study demonstrated that the transition to paltusotine was done seamlessly and the results showed once-daily, oral paltusotine maintained both symptom control as well as biochemical control when switching from monthly injections.”
In PATHFNDR-1, paltusotine was well tolerated and no serious or severe adverse events were reported in participants treated with paltusotine. The frequency of participants with at least one treatment emergent adverse event (TEAE) was comparable in the paltusotine (PAL) treatment arm vs placebo (PBO) arm (80% vs. 100% respectively). The most commonly reported TEAEs in paltusotine included: arthralgia (27% PAL vs. 57% PBO), headache (20% PAL vs. 36% PBO), diarrhea (23% PAL vs. 14% PBO), abdominal pain (17% PAL vs. 11% PBO) and nausea (10% PAL vs. 7% PBO). The frequency of adverse events considered related to acromegaly was notably lower in paltusotine treated participants compared to placebo treated participants (30% vs. 86% respectively).
“We designed paltusotine to be the preferred therapeutic option for people living with acromegaly. We could not be more excited by the results from PATHFNDR-1, which further reinforce our conviction that, if approved, paltusotine could address patients’ unmet need for a simple, oral, once-daily therapy. These data showed that upon switching from injected standard of care, paltusotine provided reliable, durable control of their disease. We intend to seek regulatory approval as quickly as possible once we complete the PATHFNDR-2 study early next year,” said Scott Struthers, Ph.D., founder and chief executive officer of Crinetics. “I would like to express my deep gratitude to the study participants, clinical staff, and Crinetics’ employees around the world who contributed to the success of this high-quality clinical study and who have worked so hard to bring this potential medicine for people living with acromegaly one major step closer to fruition.”
“These robust results for paltusotine reaffirm the strength of Crinetics’ core platform for creating high quality, small molecule, oral drugs that act at G-protein coupled receptors,” added Stephen Betz, Ph.D., founder and chief scientific officer of Crinetics. “I am extremely excited to continue to explore the utility of paltusotine for the treatment of carcinoid syndrome, as well as advance the rest of our innovative pipeline of internally discovered investigational compounds for people who live with other endocrine diseases including congenital adrenal hyperplasia, Cushing’s disease, hyperparathyroidism, Graves’ disease, hyperinsulinism, diabetes, and obesity. Paltusotine is an important lead program, and we’re just getting started.”
A full analysis of the PATHFNDR-1 results is underway, which the Company expects to present at upcoming scientific conferences. PATHFNDR-2, a Phase 3 study of oral paltusotine in participants with acromegaly who are treatment-naïve or not currently receiving medical therapy, is fully enrolled and topline data are expected in the first quarter of 2024. Pending successful findings from the PATHFNDR-2 study, Crinetics plans to submit a new drug application to the U.S. Food and Drug Administration in 2024 seeking regulatory approval for all acromegaly patients who require pharmacotherapy, including newly diagnosed patients and those switching from other therapies.
The Company is also conducting an open-label Phase 2 study to evaluate paltusotine in patients with carcinoid syndrome and intends to report preliminary results later this year.
DATA REVIEW CONFERENCE CALL
Crinetics will hold a conference call and live webcast on Monday, September 11, 2023 at 8:00 a.m. Eastern Time to discuss topline results from the PATHFNDR-1 study. To participate, please dial 1-877-451-6152 (domestic) or 1-201-389-0879 (international) and refer to conference ID 13740941. To access the webcast, click here. Following the live event, a replay will be available on the of the Company’s website.
ABOUT THE PATHFNDR PROGRAM
The PATHFNDR Program consists of two Phase 3 double-blind, placebo-controlled studies. PATHFNDR-1 (NCT04837040) enrolled a total of 58 adults with acromegaly who entered with an IGF-1 level ≤ 1.0x ULN on octreotide or lanreotide depot monotherapy. They were randomized to receive once-daily, oral paltusotine for 36 weeks or placebo. PATHFNDR-2 (NCT05192382) enrolled 112 adults with acromegaly who had elevated IGF-1 levels but were medication naïve or were not being treated with pharmacotherapy (untreated patients).
The primary endpoint for both studies is the proportion of patients achieving IGF-1 ≤1.0 xULN compared to placebo. If successful, Crinetics believes these studies could support registration of paltusotine in the United States and Europe for all acromegaly patients who require pharmacotherapy, including untreated patients and those switching from standard of care.
ABOUT ACROMEGALY
Acromegaly is a serious rare disease generally caused by a pituitary adenoma, a benign tumor in the pituitary that secretes growth hormone (GH). Excess GH secretion causes excess secretion of IGF-1 from the liver. Prolonged exposure to increased levels of IGF-1 and GH leads to progressive and serious systemic complications, often resulting in bone, joint, cardiovascular, metabolic, cerebrovascular, or respiratory disease. Acromegaly symptoms include headache, joint aches, fatigue, sleep apnea, severe sweating, hyperhidrosis/oily skin, bone and cartilage overgrowth, abnormal growth of hands and feet, enlargement of heart, liver, and other organs and alteration of facial features. Uncontrolled acromegaly results in increased mortality and has a debilitating impact on daily functioning and quality of life.
Surgical removal of pituitary adenomas, if possible, is the preferred initial treatment for most acromegaly patients. Pharmacotherapy is used for patients who are not candidates for surgery, or when surgery is unsuccessful in achieving treatment goals. Approximately 50% of patients with acromegaly prove to be candidates for pharmacotherapy. Injectable depot somatostatin analogues are the most common initial pharmacologic treatment; however, these drugs require monthly depot injections with large gauge needles that are commonly associated with pain, injection site reactions, and an increased burden on the lives of patients.
ABOUT PALTUSOTINE
Paltusotine is the first oral, once-daily selectively targeted somatostatin receptor type 2 (SST2) agonist and is currently in Phase 3 investigational studies. It was designed by the Crinetics discovery team to provide an efficacious and convenient once-daily option for people living with acromegaly and neuroendocrine tumors. In Phase 2 studies and the recently completed PATHFNDR-1 Phase 3 study, paltusotine maintained IGF-1 levels in acromegaly patients who switched from monthly injectable medications to paltusotine. IGF-1 is the primary biomarker endocrinologists use to manage their acromegaly patients.
ABOUT CRINETICS PHARMACEUTICALS
Crinetics Pharmaceuticals is a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for endocrine diseases and endocrine-related tumors. Paltusotine, an investigational, first-in-class, oral somatostatin receptor type 2 (SST2) agonist, is in Phase 3 clinical development for acromegaly and Phase 2 clinical development for carcinoid syndrome associated with neuroendocrine tumors. Crinetics has demonstrated pharmacologic proof-of-concept in a Phase 1 clinical study for CRN04894 a first-in-class, investigational, oral ACTH antagonist, that is currently in Phase 2 clinical studies for the treatment of Cushing’s disease and congenital adrenal hyperplasia. All of the Company’s drug candidates are orally delivered, small molecule new chemical entities resulting from in-house drug discovery efforts, including additional discovery programs addressing a variety of endocrine conditions such as hyperparathyroidism, polycystic kidney disease, Graves’ disease, thyroid eye disease, hyperinsulinism, diabetes and obesity.
Source: Crinetics Pharmaceuticals, Inc.
Crinetics climbs as lead asset succeeds in Phase 3 trial for acromegaly
Sep. 11, 2023 6:53 AM ET
Crinetics Pharmaceuticals, Inc. (CRNX)
By: Dulan Lokuwithana, SA News Editor
- Crinetics Pharmaceuticals (NASDAQ:CRNX) added ~74% pre-market Monday after announcing that its lead candidate, paltusotine, achieved the primary endpoint and all secondary endpoints in a Phase 3 trial for patients with the rare disease acromegaly.
- https://seekingalpha.com/news/4010493-crinetics-stock-climbs-paltusotine-succeeds-acromegaly
- Crinetics Pharmaceuticals, Inc. (CRNX)
- https://crinetics.com/
- https://crinetics.com/pipeline/paltusotine-acromegaly-nets-carcinoid-syndrome/
INO-3107
PALTUSOTINE (ORAL SST2 AGONIST)
Zilebesiran (ALN-AGT)
INOVIO Announces U.S. FDA Breakthrough Therapy Designation Granted for INO-3107 for the Treatment of Recurrent Respiratory Papillomatosis
September 07, 2023
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• Designation based on clinical evidence indicating INO-3107 may demonstrate substantial improvement over existing therapies
• First Breakthrough Therapy designation for an INOVIO DNA medicine candidate
PLYMOUTH MEETING, Pa., Sept. 7, 2023 /PRNewswire/ -- INOVIO (NASDAQ:INO), a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation for INO-3107 as a potential treatment for patients with Recurrent Respiratory Papillomatosis (RRP). The FDA's Breakthrough Therapy designation is a process designed to expedite the development and review of drug candidates that are intended to treat a serious or life-threatening condition and for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy on a clinically significant endpoint(s).
"This is yet another important step for INO-3107 and recognition that this first-in-class DNA medicine candidate has the potential to improve the lives of patients with RRP," said INOVIO's President and Chief Executive Officer, Dr. Jacqueline Shea. "As we recently announced, we have been interacting with the FDA with the goal to launch a pivotal trial for INO-3107 in the near term. With this Breakthrough Therapy designation, we look forward to continuing to work with the agency so that we can generate the evidence needed to support approval of INO-3107 as quickly and efficiently as possible, with an ultimate aim to help RRP patients and deliver on the promise of DNA medicine."
The President of the Recurrent Respiratory Papillomatosis Foundation, Kim McClellan, said: "RRP patients will tell you that even one reduction in the number of disruptive, invasive surgeries they face would be life-changing. The potential impact of this treatment gives me great hope for the future and I'm happy to see that RRP is finally getting the attention it deserves."
INO-3107 is an investigational DNA medicine candidate designed to elicit a targeted T cell response against HPV-6 and HPV-11, the HPV types that cause RRP and other HPV-related disease. This Breakthrough Therapy designation for INO-3107 follows receipt of Orphan Drug designation from the European Commission in May 2023 and from the FDA in 2020.
INOVIO plans to initiate a pivotal trial of INO-3107 in the first quarter of 2024, subject to FDA clearance. As part of its development efforts, the company has engaged a leading Clinical Research Organization to help run the pivotal trial, as well as key opinion leaders and investigators interested in developing a new treatment option for RRP patients.
The Breakthrough Therapy designation is supported by data from INOVIO's completed Phase 1/2 open-label, multicenter trial that assessed INO-3107's safety, tolerability, immunogenicity, and efficacy in patients with HPV-6 and/or HPV-11-related RRP (NCT:04398433). Overall, 81.3% (26/32) patients in the trial had a decrease in surgical interventions in the year after INO-3107 administration compared to the prior year, including 28.1% (9/32) that required no surgical intervention during or after the dosing window. Patients in the trial had a median range of 4 surgeries (2-8) in the year prior to dosing. After dosing, there was a median decrease of 3 surgical interventions (95% confidence interval -3, -2). Patients received four doses of INO-3107 on Day 0, and Weeks 3, 6, and 9. At the outset of the study (Day 0), patients could have RRP tissue surgically removed, but any surgery performed after Day 0 during the dosing window was counted against the efficacy endpoint. INO-3107 was well-tolerated by participants in the trial.
Data from this Phase 1/2 trial has been presented at scientific and medical conferences, including the 2023 Annual Meeting of the American Broncho-Esophageal Association (ABEA) in May and at the European Laryngological Association's Annual Meeting in June. Data from the trial was also published in May in the peer-reviewed journal, The Laryngoscope, under the title "Interim Results of a Phase 1/2 Open-Label Study of INO-3107 for HPV-6 and/or HPV-11–Associated RRP." The Laryngoscope is the official journal of the Triological Society (TRIO), the American Laryngological Association (ALA), and the ABEA.
About RRP
RRP is a debilitating and rare disease caused primarily by HPV-6 and/or HPV-11. RRP is characterized by the development of small, wart-like growths, or papillomas, in the respiratory tract. While papillomas are generally benign, they can cause severe, life-threatening airway obstruction and respiratory complications. RRP can also significantly affect quality of life for patients by affecting the voice box, limiting the ability to speak effectively. Surgery to remove papillomas is the standard of care for RRP; however, the papillomas often grow back because the underlying HPV infection has not been eradicated.
The most widely cited U.S. epidemiology data published in 1995 estimated that there were 14,000 active cases and about 1.8 per 100,000 new cases in adults each year. More recent pediatric epidemiology data cites a range of 0.5 - 0.7 per 100,000 new cases in children in the U.S. each year.
About INO-3107
INO-3107 is INOVIO's clinical-stage DNA medicine product candidate being developed as a potential treatment for RRP. INO-3107 is designed to elicit a targeted T cell response against HPV-6 and HPV-11, the HPV types responsible for causing RRP among other HPV-related diseases. These targeted T cells are designed to seek out and kill infected cells, with the aim of potentially preventing or slowing the growth of new papillomas. INO-3107 received Orphan Drug designation from the European Commission in May 2023 and from the U.S. Food and Drug Administration in 2020. For more information about our HPV franchise, please visit https://ir.inovio.com/events-and-presentations/default.aspx.
About INOVIO
INOVIO is a biotechnology company focused on developing and commercializing DNA medicines to help treat and protect people from HPV-related diseases, cancer, and infectious diseases. INOVIO's DNA medicines in development are delivered using its investigational proprietary device, CELLECTRA®, to produce immune responses against targeted pathogens and cancers. For more information, visit www.inovio.com.
View original content:https://www.prnewswire.com/news-releases/inovio-announces-us-fda-breakthrough-therapy-designation-granted-for-ino-3107-for-the-treatment-of-recurrent-respiratory-papillomatosis-301920321.html
SOURCE INOVIO Pharmaceuticals, Inc.
Inovio gains FDA breakthrough status for DNA therapy
Sep. 07, 2023 9:03 AM ET
Inovio Pharmaceuticals, Inc. (INO)
By: Dulan Lokuwithana, SA News Editor4 Comments
- Inovio Pharmaceuticals (NASDAQ:INO) added ~9% pre-market Thursday after the U.S. FDA issued Breakthrough Therapy designation for its experimental DNA medicine INO-3107 as a treatment for the rare disease, recurrent respiratory papillomatosis (RRP).
- With the decision, INO-3107 becomes INO's first DNA candidate to receive FDA breakthrough status.
- Previously, the Pennsylvania-based company had received the orphan drug designation for INO-3107 from the European Commission and the FDA in 2023 and 2020, respectively.
- https://seekingalpha.com/news/4009900-inovio-gains-fda-breakthrough-status-for-dna-therapy
- Inovio Pharmaceuticals, Inc. (INO)
- https://inovio.com/
- https://inovio.com/dna-medicines-pipeline/
Zilebesiran (ALN-AGT)
PALTUSOTINE (ORAL SST2 AGONIST)
Zilebesiran (ALN-AGT)
Alnylam Reports Positive Topline Results from KARDIA-1 Phase 2 Dose-Ranging Study of Zilebesiran, an Investigational RNAi Therapeutic in Development to Treat Hypertension in Patients at High Cardiovascular Risk
Sep 07, 2023
- Zilebesiran Met Primary Endpoint Demonstrating Greater than 15 mmHg Reduction of Systolic Blood Pressure at Three Months of Treatment Compared to Placebo at Two Highest Single Doses Evaluated -
- Study Met Key Secondary Endpoints Showing Consistent and Sustained Reductions of Systolic Blood Pressure at Six Months, Supporting Quarterly or Biannual Dosing -
- Zilebesiran Demonstrated an Encouraging Safety and Tolerability Profile in Adult Patients with Mild-to-Moderate Hypertension -
- Full Study Results to be Presented at an Upcoming Scientific Conference -
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 7, 2023-- Alnylam Pharmaceuticals, Inc. Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the KARDIA-1 Phase 2 study of zilebesiran, an investigational RNAi therapeutic targeting liver-expressed angiotensinogen (AGT) in development for the treatment of hypertension, met the primary endpoint demonstrating a dose-dependent, clinically significant reduction in 24-hour mean systolic blood pressure (SBP) measured by ambulatory blood pressure monitoring (ABPM) at Month 3, achieving a placebo-subtracted reduction greater than 15 mmHg (p less than 0.0001) with both 300 mg and 600 mg doses. The study also met key secondary endpoints including significant change in 24-hour mean SBP as measured by ABPM at Month 6, as well as significant change in office SBP at Month 3 and Month 6, for all zilebesiran arms, compared to placebo. The study results indicate zilebesiran was associated with dose-dependent, potent and durable knockdown of serum AGT levels through Month 6. Zilebesiran also demonstrated an encouraging safety and tolerability profile that the company believes supports continued development. These findings of robust and tonic blood pressure control will help determine the optimal dose and regimen of zilebesiran for future studies.
“Hypertension is a growing global health crisis responsible for around 10 million deaths worldwide each year. Despite the availability of several classes of oral anti-hypertensive treatments, up to 80% of individuals globally remain uncontrolled, leaving them at an increased risk of cardiovascular, cerebrovascular and renal disease, which is further exacerbated by blood pressure variability, lack of nighttime blood pressure control and poor adherence,” said Professor George L. Bakris, M.D., Board-Certified Hypertension Specialist and Director of the American Heart Association Comprehensive Hypertension Center, University of Chicago Medicine. “As a physician, I believe these KARDIA-1 results, which demonstrate clinically significant reductions in systolic blood pressure of greater than 15 mmHg, along with the ability to achieve durable tonic blood pressure control, provide hope that we may one day have access to a novel therapy with the potential to address the significant unmet needs of patients with uncontrolled hypertension who are at high risk of future cardiovascular events.”
Zilebesiran demonstrated an encouraging safety and tolerability profile. There was one death due to cardiopulmonary arrest in a zilebesiran-treated patient that was considered unrelated to study drug. Serious adverse events were reported in 3.6% of zilebesiran-treated patients and 6.7% of placebo-treated patients. None were considered related to study drug. Adverse events occurring in 5% or more of zilebesiran-treated patients in any dose arm included COVID-19, injection site reaction (ISR), hyperkalemia, hypertension, upper respiratory tract infection, arthralgia and headache.
The KARDIA-1 Phase 2 trial is a randomized, double-blind (DB), placebo-controlled, multi-center global dose-ranging study designed to evaluate the efficacy and safety of zilebesiran as monotherapy in adults with mild-to-moderate hypertension. The study enrolled 394 adults representing a diverse patient population with untreated hypertension or who were on stable therapy with one or more anti-hypertensive medications. Any patients taking prior anti-hypertensive medications completed at least a two- to four-week wash-out before randomization. Patients were randomized to one of five treatment arms during a 12-month DB period and DB extension period: 150 mg zilebesiran subcutaneously once every six months; 300 mg zilebesiran subcutaneously once every six months; 300 mg zilebesiran subcutaneously once every three months; 600 mg zilebesiran subcutaneously once every six months; or placebo. Patients who received placebo were randomized to one of the four initial zilebesiran dose regimens beginning at Month 6.
The primary endpoint is the change from baseline in SBP at Month 3, assessed by 24-hour ABPM. Key secondary and exploratory endpoints in this study include additional measures of blood pressure reduction at six months, time-adjusted change in blood pressure, and change in daytime average and night-time average blood pressure.
“We are thrilled that the KARDIA-1 Phase 2 results show zilebesiran’s ability to achieve sustained blood pressure reductions of greater than 15 mmHg, as well as long-term efficacy out to six months with infrequent dosing. We believe these results further validate the differentiated profile we observed in Phase 1. Moreover, they reinforce the potential for zilebesiran to be a transformative therapy to reduce cardiovascular risk in patients with hypertension and to offer new possibilities in a field of medicine that has seen limited innovation in nearly 20 years,” said Simon Fox, Ph.D., Vice President, Zilebesiran Program Lead at Alnylam. “We look forward to sharing the full KARDIA-1 results at an upcoming scientific conference and to reporting topline results from our KARDIA-2 Phase 2 study of zilebesiran in combination with one of three standard classes of anti-hypertensive medications in patients with mild-to-moderate hypertension in early 2024. It is a very exciting time for Alnylam, as these results build on the momentum from the recent strategic agreement to co-develop and co-commercialize zilebesiran with our collaboration partner, Roche, to potentially transform the landscape for patients with cardiovascular diseases.”
The KARDIA-2 Phase 2 study of zilebesiran used in combination with one of three standard classes of anti-hypertensive medications completed enrollment in June 2023. Topline results are expected in early 2024.
About Zilebesiran
Zilebesiran is an investigational, subcutaneously administered RNAi therapeutic targeting angiotensinogen (AGT) in development for the treatment of hypertension in high unmet need populations. AGT is the most upstream precursor in the Renin-Angiotensin-Aldosterone System (RAAS), a cascade which has a demonstrated role in blood pressure (BP) regulation and its inhibition has well-established anti-hypertensive effects. Zilebesiran inhibits the synthesis of AGT in the liver, potentially leading to durable reductions in AGT protein and ultimately, in the vasoconstrictor angiotensin (Ang) II. Zilebesiran utilizes Alnylam's Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables infrequent subcutaneous dosing with increased selectivity and the potential to achieve tonic blood pressure control demonstrating consistent and durable blood pressure reduction throughout a 24-hour period, sustained up to six months after a single dose of zilebesiran. The safety and efficacy of zilebesiran have not been established or evaluated by the FDA, EMA or any other health authority. Zilebesiran is being co-developed and co-commercialized by Alnylam and Roche.
About Hypertension
Uncontrolled hypertension is the chronic elevation of blood pressure (BP), defined by the 2017 ACC/AHA guidelines as ≥130 mmHg systolic blood pressure (SBP) and ≥80 mmHg diastolic blood pressure (DBP). More than one billion people worldwide live with hypertension.i Approximately one in three adults are living with hypertension worldwide, with up to 80% of individuals remaining uncontrolled despite the availability of several classes of oral anti-hypertensive treatments. Despite the availability of anti-hypertensive medications, there remains a significant unmet medical need, especially given the poor rates of adherence to existing daily oral medications, resulting in inconsistent BP control and an increased risk for stroke, heart attack and premature death.ii In particular, there are a number of high unmet need settings where novel approaches to hypertension warrant additional development focus, including patients with poor medication adherence and in patients with high cardiovascular risk.
About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
About Alnylam Pharmaceuticals
Alnylam Pharmaceuticals (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), AMVUTTRA® (vutrisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), and Leqvio® (inclisiran), which is being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on X (formerly Twitter) at @Alnylam, or on LinkedIn, Facebook, or Instagram.
View source version on businesswire.com: https://www.businesswire.com/news/home/20230907817269/en/
Alnylam Pharmaceuticals, Inc.
Source: Alnylam Pharmaceuticals, Inc.
Alnylam succeeds in Phase 2 trial for blood pressure drug
Sep. 07, 2023 8:25 AM ET
Alnylam Pharmaceuticals, Inc. (ALNY), RHHBY, RHHBF
By: Dulan Lokuwithana, SA News Editor
- Alnylam Pharmaceuticals (NASDAQ:ALNY) announced Thursday that its experimental RNAi therapeutic, zilebesiran, reached the primary endpoint in a Phase 2 study for adults with mild-to-moderate hypertension.
- Roche (OTCQX:RHHBY) (OTCQX:RHHBF) has partnered with Alnylam to develop and commercialize the subcutaneously delivered therapy.
- Citing topline data from the KARDIA-1 trial at month three, Alnylam (ALNY) said that zilebesiran led to a decline in 24-hour mean systolic blood pressure (SBP) with a dose-dependent, clinically significant effect in achieving its primary goal.
- https://seekingalpha.com/news/4009869-alnylam-succeeds-phase-2-trial-blood-pressure-drug?mailingid=32631948&messageid=2900&serial=32631948.4015&utm_campaign=rta-stock-news&utm_content=link-1&utm_medium=email&utm_source=seeking_alpha&utm_term=32631948.4015
- Alnylam Pharmaceuticals, Inc. (ALNY)
- https://www.alnylam.com/sites/default/files/pdfs/ALN-AGT-Fact-Sheet.pdf
- https://www.alnylam.com/
VLA15
BRII-179 (VBI-2601) in Combination With PEG-IFNα
BRII-179 (VBI-2601) in Combination With PEG-IFNα
Valneva and Pfizer Report Positive Pediatric and Adolescent Phase 2 Booster Results for Lyme Disease Vaccine Candidate
September 7, 2023
- Strong immune response shown in both children and adolescents one month after booster dose (month 19) in VLA15-221 study
- Previously observed high anamnestic antibody response in adults confirmed
- VLA15 well-tolerated in all age groups following booster dose
Saint-Herblain (France) & New York, September 7, 2023 – Valneva SE (Nasdaq: VALN; Euronext Paris: VLA) and Pfizer Inc. (NYSE: PFE) announced today positive pediatric and adolescent immunogenicity and safety data for their Lyme disease vaccine candidate, VLA15, when given as a booster. These results from the VLA15-221 Phase 2 study showed a strong anamnestic antibody response for all serotypes in pediatric (5 to 11 years of age) and adolescent participants (12 to 17 years of age), as well as in adults (18 to 65 years of age), one month after administration of a booster dose (month 19).
Depending on the primary schedule they received (month 0-2-6 or month 0-6), participants seroconverted after the booster dose, yielding seroconversion1 rates (SCRs) of 95.3% and 94.6% for all outer surface protein A (OspA) serotypes in all age groups, respectively. Additionally, OspA antibody titers were significantly higher one month after the booster dose compared to one month after the primary schedule with 3.3- to 3.7-fold increases (Geometric Mean Fold Rises) in adults, 2.0- to 2.7- fold increases in adolescents and 2.3- to 2.5-fold increases in children for all serotypes.
Juan Carlos Jaramillo M.D., Chief Medical Officer of Valneva, said, “We are pleased with these data which validate the use of a booster dose in all age groups. Lyme disease continues to spread, representing an important unmet medical need that impacts the lives of many people in the Northern Hemisphere. With each new set of positive data, we come one step closer to potentially bringing this vaccine to both adults and children living in areas where Lyme disease is endemic.”
The Phase 2 booster results emphasize the vaccine candidate’s potential to provide immunity against Lyme disease in pediatric and adolescent populations. Geometric Mean Titers (GMTs) one month following the booster dose were similarly high for children and adolescents.
The safety and tolerability profile of VLA15 after a booster dose was consistent with previous studies as the vaccine candidate was well-tolerated in all age groups regardless of the primary vaccination schedule. No vaccine-related serious adverse events (SAEs) and no safety concerns were observed by an independent Data Safety Monitoring Board (DSMB).
“Protection against Lyme disease is important for anyone who lives or spends time outdoors in areas where Lyme disease is endemic. This data from the VLA15-221 study is vital to improve our understanding of how vaccination may help to protect both adults and children from this potentially devastating disease,” said Annaliesa Anderson, Ph.D., Senior Vice President and Head Vaccine Research and Development at Pfizer. “We are encouraged by the positive Phase 2 results for VLA15, and, in partnership with Valneva, look forward to continuing to study the vaccine candidate in ongoing Phase 3 clinical trials.”
These results follow six-month antibody persistence data in children and adults reported for the VLA15-221 study in December 20222 and positive immunogenicity and safety data reported in April 20223.
In August 2022, Pfizer and Valneva initiated the currently ongoing Phase 3 clinical study, Vaccine Against Lyme for Outdoor Recreationists (VALOR) (NCT05477524), to investigate the efficacy, safety and immunogenicity of VLA15 in participants five years of age and older in highly endemic regions in the United States (U.S.) and Europe4. A second Phase 3 study (VLA15-1012), aiming to provide further evidence on the safety profile of VLA15 in the pediatric population, is also ongoing.
Pfizer aims to submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) and Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) in 2026, subject to positive Phase 3 data.
About VLA15
There are currently no approved human vaccines for Lyme disease, and VLA15 is the most advanced Lyme disease vaccine candidate currently in clinical development, with a Phase 3 study in progress. This investigational multivalent protein subunit vaccine uses an established mechanism of action for a Lyme disease vaccine that targets the outer surface protein A (OspA) of Borrelia burgdorferi, the bacteria that cause Lyme disease. OspA is a surface protein expressed by the bacteria when present in a tick. Blocking OspA inhibits the bacterium’s ability to leave the tick and infect humans. The vaccine covers the six most common OspA serotypes expressed by the Borrelia burgdorferi sensu lato species that are prevalent in North America and Europe. VLA15 has demonstrated a strong immune response and satisfactory safety profile in pre-clinical and clinical studies so far. Valneva and Pfizer entered into a collaboration agreement in April 2020 to co-develop VLA15, with updates to the terms within this agreement made in June 2022.5,6 The program was granted Fast Track designation by the U.S. FDA in July 2017.7
About Clinical Study VLA15-221
VLA15-221 is a randomized, observer-blind, placebo-controlled Phase 2 study. It is the first clinical study with VLA15 which enrolled a pediatric population (5-17 years old).
585 healthy participants received VLA15 in two immunization schedules (month 0-2-6 [N=190] or month 0-6 [N=187]) or three doses of placebo (month 0-2-6 [N=208]). Vaccine recipients received VLA15 at a dose of 180 µg, which was selected based on data generated in the two previous Phase 2 studies. The main safety and immunogenicity readout was performed one month after the primary vaccination series. All eligible subjects received a booster dose of VLA15 or placebo at month 18 (booster phase) and will be followed for three additional years to monitor antibody persistence. In addition, all eligible subjects will be asked to receive an additional booster dose of VLA15 or placebo at month 30, in order to generate additional data and assess the need for periodic booster doses.
VLA15 is tested as an alum-adjuvanted formulation and administered intramuscularly. The study is being conducted at U.S. sites located in areas where Lyme disease is endemic and has enrolled both volunteers with a prior infection with Borrelia burgdorferi as well as Borrelia burgdorferi-naïve volunteers.
About Lyme Disease
Lyme disease is a systemic infection caused by Borrelia burgdorferi bacteria transmitted to humans by the bite of an infected Ixodes ticks.8 It is considered the most common vector-borne illness in the Northern Hemisphere.9 While the true incidence of Lyme disease is unknown, it is estimated to annually affect approximately 476,000 people in the U.S. and 129,000 people in Europe.10,11 Early symptoms of Lyme disease (such as a gradually expanding erythematous rash called Erythema migrans or more nonspecific symptoms like fatigue, fever, headache, mild stiff neck, arthralgia or myalgia) are often overlooked or misinterpreted. Left untreated, the disease can disseminate and cause more serious complications affecting the skin, joints (arthritis), the heart (carditis) or the nervous system.10 The medical need for vaccination against Lyme disease is steadily increasing as the geographic footprint of the disease widens.12
About Pfizer: Breakthroughs That Change Patients’ Lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. In addition, to learn more, please visit us on www.Pfizer.com and follow us on Twitter at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
About Valneva SE
We are a specialty vaccine company focused on the development, manufacturing and commercialization of prophylactic vaccines for infectious diseases. We take a highly specialized and targeted approach to vaccine development by focusing on vaccine solutions addressing unmet medical needs to ensure we can make a difference to peoples’ lives. We apply our deep understanding of vaccine science, including our expertise across multiple vaccine modalities, and our established vaccine development capabilities, to develop vaccines against diseases which are not yet vaccine-preventable, or for which there are limited effective treatment options. Today, we are leveraging our expertise and capabilities to rapidly advance a broad range of vaccines into and through the clinic, including candidates against the chikungunya virus and Lyme disease.
Pfizer, Valneva post Phase 2 booster data for Lyme disease vaccine
Sep. 07, 2023 6:56 AM ET
By: Dulan Lokuwithana, SA News Editor
Pfizer (PFE) and its French partner Valneva SE (NASDAQ:VALN) announced Thursday that the companies’ Lyme disease vaccine candidate, VLA15, caused a robust immune response in children and adolescents as a booster shot.
Citing results from their 585-subject VLA15-221 trial, the companies said that the antibody levels against outer surface protein A (OspA) indicated a 2.0–2.7-fold increase one month after VLA15 as a booster in adolescents compared to one month after the primary schedule.
https://seekingalpha.com/news/4009828-pfizer-valneva-post--booster-data-lyme-disease-vaccine
BRII-179 (VBI-2601) in Combination With PEG-IFNα
BRII-179 (VBI-2601) in Combination With PEG-IFNα
BRII-179 (VBI-2601) in Combination With PEG-IFNα
VBI Vaccines Partner Brii Biosciences Announces Topline Interim Results of Phase 2 Study Evaluating BRII-179 (VBI-2601) in Combination With PEG-IFNα for the Treatment of Chronic Hepatitis B
September 6, 2023
- BRII-179 (VBI-2601) as an add-on therapy to standard of care PEG-IFNα increases HBsAg loss rate at the end of treatment and 12 weeks follow up
- Significantly increased seroconversion rate is strongly associated with BRII-179 (VBI-2601) treatment and correlates with the increased rate of HBsAg loss
- Safety findings are similar to those of PEG-IFNα and BRII-179 (VBI-2601) as previously reported
CAMBRIDGE, Mass. –
VBI Vaccines Inc. (Nasdaq: VBIV) (“VBI” or the “Company”) today announced that its strategic hepatitis B (HBV) partner, Brii Biosciences (Brii Bio) (Stock code: 2137.HK), announced topline cohort-level unblinded Week 36 data from interim analysis of a randomized, placebo-controlled and double-blinded Phase 2 study of BRII-179 (VBI-2601), a first-in-class Pre-S1/Pre-S2/S therapeutic vaccine, in combination treatment with pegylated interferon-alpha (PEG-IFNα) in chronic hepatitis B virus (HBV) patients compared with PEG-IFNα only treatment. VBI and Brii Bio reported in previous studies that BRII-179 (VBI-2601) induced broad antibody and T-cell responses against Pre-S1, Pre-S2, and S epitopes in HBV patients. Brii Bio’s detailed press release can be found here: https://www.briibio.com/en/media/press-release/20230906/.
“We share Brii Bio’s excitement about this data and congratulate them on their continued ability to move quickly and execute clinical programs as they seek to deliver higher hepatitis B functional cure rates,” said Jeff Baxter, President and CEO of VBI. “Brii Bio’s ongoing commitment, investments, and partnerships in this field continue to reinforce the strategic potential of our partnership and we look forward to seeing further data from this study and future studies of this first-in-class immunotherapeutic candidate.”
In July 2023, VBI and Brii Bio announced an expansion of their HBV partnership to include an exclusive global license to develop and commercialize BRII-179 (VBI-2601), and an exclusive license to develop and commercialize PreHevbri®, VBI’s prophylactic 3-antigen adult HBV vaccine, in the Asia Pacific region, excluding Japan.
About BRII-179 (VBI-2601) + PEG-IFNα Combination Study
The Phase 2 study is a multicenter, randomized, double-blind, placebo-controlled, parallel study designed to evaluate the safety and efficacy of BRII-179 (VBI-2601) as an add-on therapy to PEG-IFNα and NrtI therapy for the treatment of chronic HBV infection. This study enrolled adult HBV patients in mainland China who had received 24 to 28 doses of PEG-IFNα per treatment guideline, at least 12 months of NrtI therapy, and met a pre-defined criteria for partial response. Subjects were randomized 1:1 to receive BRII-179 (VBI-2601) or placebo every three weeks for a total of 7 doses over 18 weeks while continuing PEG-IFNα treatment for 48 weeks. Subjects who met Nrtl discontinuation criteria would stop Nrtl treatment and would be followed up for additional 48 weeks.
About BRII-179 (VBI-2601)
BRII-179 (VBI-2601) is a novel recombinant, protein-based HBV immunotherapeutic candidate that builds upon the 3-antigen conformation of VBI’s prophylactic 3-antigen HBV vaccine candidate and is designed to target enhanced and broad B-cell and T-cell immunity. BRII-179 (VBI-2601) has been licensed to Brii Biosciences (Brii Bio) under an exclusive global development and licensing agreement. BRII-179 (VBI-2601) is currently being investigated in two Phase 2 clinical trials in combination with BRII-835 (VIR-2218) and PEG-IFNα as part of a potential functional cure regimen for the treatment of chronic HBV infection.
About PreHevbri®
PreHevbri is the only 3-antigen hepatitis B vaccine, comprised of the three hepatitis B surface antigens of the hepatitis B virus – Pre-S1, Pre-S2, and S. It is approved for use in the United States, European Union/European Economic Area, United Kingdom, Canada, and Israel. The brand names for this vaccine are: PreHevbrio® (US/Canada), PreHevbri® (EU/EEA/UK), and Sci-B-Vac® (Israel).
Please visit www.PreHevbrio.com for U.S. Important Safety Information for PreHevbrio® [Hepatitis B Vaccine (Recombinant)], or please see U.S. Full Prescribing Information.
U.S. Indication
PreHevbrio is indicated for prevention of infection caused by all known subtypes of hepatitis B virus. PreHevbrio is approved for use in adults 18 years of age and older.
Please see Full Prescribing Information.
About VBI Vaccines Inc.
VBI Vaccines Inc. (“VBI”) is a biopharmaceutical company driven by immunology in the pursuit of powerful prevention and treatment of disease. Through its innovative approach to virus-like particles (“VLPs”), including a proprietary enveloped VLP (“eVLP”) platform technology, VBI develops vaccine candidates that mimic the natural presentation of viruses, designed to elicit the innate power of the human immune system. VBI is committed to targeting and overcoming significant infectious diseases, including hepatitis B, coronaviruses, and cytomegalovirus (CMV), as well as aggressive cancers including glioblastoma (GBM). VBI is headquartered in Cambridge, Massachusetts, with research operations in Ottawa, Canada, and a research and manufacturing site in Rehovot, Israel.
Website Home: http://www.vbivaccines.com/
News and Resources: http://www.vbivaccines.com/news-and-resources/
Investors: http://www.vbivaccines.com/investors/
View source version on businesswire.com: https://www.businesswire.com/news/home/20230906245918/en/
VBI Vaccines gains after Phase 2 data for Hep B candidate
Sep. 06, 2023 7:33 AM ET
VBI Vaccines Inc. (VBIV), BRIBF
By: Dulan Lokuwithana, SA News Editor
VBI Vaccines (NASDAQ:VBIV) traded ~13% higher pre-market Wednesday after its partner, Hong Kong-listed Brii Biosciences Limited (OTCPK:BRIBF), announced mid-stage data for BRII-179, an experimental immunotherapy targeted at the hepatitis B virus (HBV).
https://seekingalpha.com/news/4009419-vbi-vaccines-stock-gains-after-data-hep-b-candidate
VBI Vaccines Inc. (VBIV), BRIBF
MGD024
BRII-179 (VBI-2601) in Combination With PEG-IFNα
MGD024
Sep 5, 2023
MacroGenics Announces Achievement of $15 Million Milestone Related to Gilead’s Nomination of a Bispecific Research Program
ROCKVILLE, MD, Sept. 05, 2023 (GLOBE NEWSWIRE) -- MacroGenics, Inc. (NASDAQ: MGNX), a biopharmaceutical company focused on developing, manufacturing and commercializing innovative antibody-based therapeutics for the treatment of cancer, today announced that its partner, Gilead Sciences, Inc. (NASDAQ: GILD), nominated the first of two research programs, leveraging MacroGenics’ DART® and TRIDENT® platforms for generating bispecific antibodies. This nomination grants Gilead an exclusive option, upon achievement of a pre-defined preclinical milestone, to license worldwide rights to the research program.
Under the October 2022 agreement, MacroGenics will receive $15 million related to the nomination of a bispecific research program to be conducted by MacroGenics and funded by Gilead. Pursuant to this agreement, which covers MGD024, an investigational, bispecific antibody that binds CD123 and CD3 using MacroGenics’ DART platform, and up to two additional bispecific research programs, MacroGenics remains eligible to receive up to $1.7 billion in target nomination, option fees, and development, regulatory and commercial milestones. MacroGenics will also be eligible to receive tiered, double-digit royalties on worldwide net sales of MGD024 and a flat royalty on worldwide net sales of products under the two research programs.
About MacroGenics, Inc.
MacroGenics (the Company) is a biopharmaceutical company focused on developing, manufacturing and commercializing innovative monoclonal antibody-based therapeutics for the treatment of cancer. The Company generates its pipeline of product candidates primarily from its proprietary suite of next-generation antibody-based technology platforms, which have applicability across broad therapeutic domains. The combination of MacroGenics' technology platforms and protein engineering expertise has allowed the Company to generate promising product candidates and enter into several strategic collaborations with global pharmaceutical and biotechnology companies. For more information, please see the Company's website at www.macrogenics.com. MacroGenics, the MacroGenics logo, MARGENZA and DART are trademarks or registered trademarks of MacroGenics, Inc.
Source: MacroGenics, Inc.
MacroGenics achieves $15M milestone related to Gilead's nomination of bispecific research program
Sep. 05, 2023 4:46 PM ET
By: Jaskiran Singh, SA News Editor
- MacroGenics (NASDAQ:MGNX) Tuesday announced the achievement of a pre-defined preclinical milestone, enabling MacroGenics to receive $15 million related to the nomination of a bispecific research program.
- https://seekingalpha.com/news/4009286-macrogenics-achieves-15m-milestone-related-to-gileads-nomination-of-bispecific-research-program
- MacroGenics, Inc. (MGNX)
- https://macrogenics.com/pipeline/mgd024-cd123-x-cd3/
- https://macrogenics.com/
KB408
CAMZYOS® (mavacamten)
MGD024
Krystal Biotech Announces Orphan Drug Designation Granted to KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
September 5, 2023
- KB408 IND filed on August 15, 2023
PITTSBURGH, Sept. 05, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, announced today that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD).
AATD is caused by mutations in the SERPINA1 gene that lead to decreased levels and/or decreased functionality of alpha-1 antitrypsin protein. The protein is primarily produced in the liver and secreted into the bloodstream, where it acts as a circulating serine protease inhibitor whose principal substrate is neutrophil elastase in the lungs. Over time, the deficiency can lead to progressive enzymatic destruction of the lung tissue, ultimately causing life-threatening pulmonary impairment and severe respiratory insufficiency. In severe cases, current disease management includes intravenous augmentation therapy which requires weekly infusions, the clinical benefit of which remains to be established.
KB408 is an inhaled (nebulized) formulation of the Company’s novel replication-defective, non-integrating HSV-1-based vector designed to deliver two copies of the SERPINA1 transgene, that encodes for human alpha-1 antitrypsin protein, for the treatment of AATD.
"This important designation is a milestone in the advancement of KB408, and this decision by the FDA underscores the need for potential new treatment options for patients with AATD," said Suma Krishnan, President, Research & Development, Krystal Biotech, Inc. "We are encouraged by the profile of KB408 in preclinical studies to date and look forward to dosing patients once we receive clearance from the FDA."
About Orphan Drug Designation
Orphan Drug Designation is granted by the FDA to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the U.S. Orphan drug status provides benefits to drug developers, including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of post-approval marketing exclusivity.
About Krystal Biotech, Inc.
Krystal Biotech, Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEK™ is the Company’s first commercial product, the first-ever redosable gene therapy, and the only medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and Twitter.
Source: Krystal Biotech, Inc.
Krystal Biotech gets FDA orphan drug designation for AATD drug candidate
Sep. 05, 2023 11:40 AM ET
By: Val Brickates Kennedy, SA News Editor
Krystal Biotech (NASDAQ:KRYS) said it has received FDA orphan drug designation for its drug candidate KB408 in the treatment of alpha-1 antitrypsin deficiency, or AATD.
CAMZYOS® (mavacamten)
CAMZYOS® (mavacamten)
CAMZYOS® (mavacamten)
Long-Term Follow-Up Data from Two Phase 3 Studies of CAMZYOS® (mavacamten) Demonstrate Consistent and Durable Response in Patients with Symptomatic Obstructive Hypertrophic Cardiomyopathy (HCM)
08/28/2023
CATEGORY:
Results from VALOR-HCM LTE (56 weeks) demonstrated that with longer follow-up, CAMZYOS continued to reduce eligibility for invasive SRT at 56 weeks
EXPLORER-LTE data (cumulative analysis up to 120 weeks) showed sustained improvements in LVOT obstruction, symptoms, and NT-proBNP levels in patients with symptomatic obstructive HCM, with no new safety signals observed
CAMZYOS was recently approved in the European Union, following its approval in the U.S. and other markets worldwide, and is the first and only cardiac myosin inhibitor approved to treat adult patients with symptomatic obstructive HCM
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced new long-term follow-up results from two Phase 3 studies evaluating CAMZYOS® (mavacamten), a first-in-class cardiac myosin inhibitor, in adult patients with symptomatic obstructive hypertrophic cardiomyopathy (HCM). Results from the 56-week analysis of the VALOR-HCM long-term extension (LTE) study were presented as a late-breaking oral presentation, with simultaneous publication in JAMA Cardiology, and results from the cumulative 120-week analysis of the EXPLORER cohort of the MAVA-LTE study were presented as an oral presentation at the European Society of Cardiology (ESC) Congress 2023.
“The new long-term data presented at ESC were consistent with the primary analyses from each study, further underscoring the benefit our first-in-class therapy can provide to patients with symptomatic obstructive HCM,” said Amy Sehnert, M.D., Vice President, Head of Cardiomyopathy and Heart Failure Clinical Development, Bristol Myers Squibb. “These positive data reinforce the clinically meaningful significance of these two Phase 3 trials that led to the approval of CAMZYOS in the United States, the European Union and other countries around the globe. We are excited for the ongoing exploration of the potential of CAMZYOS and remain dedicated to providing support for obstructive HCM patients worldwide.”
Key findings from the 56-week analysis of VALOR-HCM LTE include:
- Treatment with CAMZYOS demonstrated sustained improvements across key study endpoints in both the original CAMZYOS group over 56 weeks and those transitioned to the placebo cross-over group over 40 weeks.
- At Week 56, 5 of 56 patients (8.9%) in the original CAMZYOS group and 10 of 52 patients (19.2%) in the placebo cross-over group at Week 40 decided to proceed with septal reduction therapy (SRT) or were SRT-eligible.
- CAMZYOS demonstrated sustained reduction in peak resting LVOT gradient (-34.0 mmHg for the original CAMZYOS group [95% CI -43.5 to -24.5] and -33.2 mmHg for the placebo cross-over group [95% CI -41.9 to -24.5]).
- Proportion with NYHA class improvement of ≥1 class was observed in 93% of patients in the original CAMZYOS group at Week 56 and 73% of patients from the placebo cross-over group at Week 40.
- On the patient-reported 23-item Kansas City Cardiomyopathy Questionnaire Clinical Summary Score (KCCQ-23 CSS)*, average scores of symptom frequency, symptom burden and physical limitation continued to improve with a 14.1 point increase for the original CAMZYOS group (95% CI 9.9 to 18.3) and 11.7 point increase for the placebo crossover group (95% CI 6.9 to 16.4).
- CAMZYOS was also associated with sustained reduction across biomarkers of cardiac wall stress and myocardial injury including reduction in N-terminal pro brain natriuretic peptide (NT-proBNP), with -376 ng/L for the original CAMZYOS group (95% CI -723 to -225) and -423 ng/L for the placebo cross-over group (95% CI -624 to -252) and reduction in cardiac troponin I with -7.0 ng/L for the original CAMZYOS group (95% CI -10 to -2.3) and -6.2 ng/L for the placebo cross-over group (95% CI -11.5 to -3.3).
- No new safety signals were observed, and safety and efficacy were consistent across both patient groups.
*The KCCQ‑23 CSS is derived from the Total Symptom Score (TSS) and the Physical Limitations (PL) score of the KCCQ‑23. The CSS ranges from 0 to 100 with higher scores representing better health status.
“The 56-week late-breaking analysis of VALOR-HCM LTE builds upon previous findings and demonstrates the consistent impact of this oral treatment for severely symptomatic obstructive HCM patients by showing that nearly 9 out of 10 patients treated with this drug have continued in this long-term extension trial without SRT at either 40 or 56 weeks of treatment,” said Milind Desai, M.D., MBA, director, center for hypertrophic cardiomyopathy and vice chair of education in the Heart, Vascular & Thoracic Institute at Cleveland Clinic. “These findings are important for our continued understanding of this treatment and encouraging for patients hoping for non-surgical options.”
Key findings from the cumulative 120-week analysis of EXPLORER-LTE include:
- No new safety signals were observed.
- Overall, 75.9% of patients improved by ≥1 NYHA class from baseline at the start of the LTE study to Week 120.
- Of the 14 patients who were in NYHA class I, 12 remained in NYHA class I at the latest available assessment.
- Treatment with CAMZYOS was associated with sustained improvements from baseline at the start of the LTE study in echocardiographic parameters, including E/e’ average and NT-proBNP.
- Mean LVEF remained within the normal range at all study visits.
- Since the previous interim analysis in August 2021, one new patient experienced a transient reduction in LVEF <50% resulting in temporary treatment interruption.
“The presentation of data − the largest and longest analysis of patients on CAMZYOS to-date − illustrates the promise of this game-changing treatment for patients with symptomatic obstructive HCM,” said Pablo García-Pavia, M.D., Ph.D., head of the Inherited Cardiac Diseases and Heart Failure Unit at the Department of Cardiology of Hospital Universitario Puerta de Hierro and professor at the Spanish Cardiovascular Research Institute (CNIC) in Madrid, Spain. “Studies like EXPLORER-LTE are important for understanding longer-term results that assess key cardiac measures and support the use of CAMZYOS in patients living with this chronic condition.”
About the VALOR-HCM and LTE Trial
VALOR-HCM (NCT04349072) was a randomized, double-blind, placebo-controlled, multicenter Phase 3 study of patients with symptomatic, obstructive HCM (NYHA class II-IV) who met guideline criteria for septal reduction therapy (SRT; LVOT gradient of ≥50 mmHg and NYHA class III-IV, or class II with exertional syncope or near syncope) and had been referred or under active consideration (within the past 12 months) for an invasive procedure. Patients were required to have LVOT peak gradient ≥50 mmHg at rest or with provocation, and LVEF ≥60%. The study enrolled 112 patients (mean age of 60 years; 51% men; 93% ≥ NYHA class III) randomized on a 1:1 basis to receive mavacamten or placebo. At baseline, 95% of patients were on background therapies of a beta blocker, calcium channel blocker, disopyramide or a combination. The primary endpoint was a composite of the proportion of patients who decided to proceed with SRT prior to or at Week 16 or who remained SRT-guideline eligible (LVOT gradient of ≥50 mmHg and NYHA class III-IV, or class II with exertion induced syncope or near syncope) at Week 16. Key secondary endpoints included the change from baseline on post-exercise LVOT gradient, NYHA class, Kansas City Cardiomyopathy Questionnaire (KCCQ-23) Clinical Summary Score and cardiac biomarkers (NT-proBNP and Cardiac Troponin I) at Week 16.
The group initially randomized to CAMZYOS continued the drug for 32 weeks, and the placebo group crossed over to dose-blinded CAMZYOS from Week 16 to Week 32. From the 112 randomized patients with obstructive HCM, 108 (mean age, 60.3 years; 50% men; 94% in NYHA class III/IV) qualified for Week 32 evaluation (56 in the original CAMZYOS group and 52 in the placebo cross-over group) and continued once-daily CAMZYOS until Week 128. During this LTE period, CAMZYOS dose remained blinded.
About the EXPLORER-HCM and the MAVA-LTE Trials
The EXPLORER-HCM Phase 3 trial (NCT03470545) was a double-blind, randomized, placebo-controlled, parallel group trial that enrolled a total of 251 adult patients with symptomatic (NYHA class II or III) obstructive hypertrophic cardiomyopathy. All participants had measurable left ventricular ejection fraction (LVEF) ≥55% and at least one peak LVOT gradient ≥50 mmHg (at rest or with provocation at diagnosis); in addition, Valsalva LVOT gradient ≥30 mmHg at baseline was required at screening. Ninety-two percent of patients were on background therapies of a beta blocker or calcium channel blocker. The primary endpoint was a composite functional endpoint, assessed at 30 weeks, and was defined as the proportion of patients who achieved either improvement of mixed venous oxygen tension (pVO2) by ≥1.5 mL/kg/min plus improvement in NYHA class by at least 1 or improvement of pVO2 by ≥3.0 mL/kg/min plus no worsening in NYHA class. Key secondary endpoints include impact on exercise gradient LVOT, pVO2, NYHA class and Kansas City Cardiomyopathy Questionnaire (KCCQ) and Hypertrophic Cardiomyopathy Symptom Questionnaire (HCMSQ) at Week 30.
EXPLORER-LTE is a cohort of the MAVA-LTE study (NCT03723655), an ongoing, dose-blinded, 5-year study of CAMZYOS in patients with symptomatic obstructive HCM who completed the EXPLORER-HCM trial. All participants in the EXPLORER-LTE cohort started on 5 mg of CAMZYOS daily and dose adjustments were made at Weeks 4, 8 and 12 based on site-read echocardiography measures of Valsalva LVOT gradient and LVEF only. Dose adjustment was also possible at Week 24 following site-read echocardiography assessment of post-exercise LVOT gradient. Subsequent to Week 24, dose adjustment was possible if site-read Valsalva LVOT gradient was >30 mmHg and LVEF ≥50%.
About CAMZYOS (mavacamten)
CAMZYOS® (mavacamten) is the first and only cardiac myosin inhibitor approved in the U.S., indicated for the treatment of adults with symptomatic New York Heart Association (NYHA) class II-III obstructive hypertrophic cardiomyopathy (HCM) to improve functional capacity and symptoms, and in the European Union, indicated for the treatment of symptomatic (NYHA, class II-III) obstructive HCM in adult patients. It has also received regulatory approvals in Australia, Brazil, Canada, Great Britain, Macau, Singapore, South Korea and Switzerland. CAMZYOS is an allosteric and reversible inhibitor selective for cardiac myosin. CAMZYOS modulates the number of myosin heads that can enter “on actin” (power-generating) states, thus reducing the probability of force-producing (systolic) and residual (diastolic) cross-bridge formation. Excess myosin actin cross-bridge formation and dysregulation of the super-relaxed state are mechanistic hallmarks of HCM. CAMZYOS shifts the overall myosin population towards an energy-sparing, recruitable, super-relaxed state. In HCM patients, myosin inhibition with CAMZYOS reduces dynamic left ventricular outflow tract (LVOT) obstruction and improves cardiac filling pressures.
Further information is available at www.CAMZYOSREMS.com or by telephone at 1-833-628-7367.
Please see U.S. Full Prescribing Information, including Boxed WARNING and Medication Guide.
About Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Source: Bristol Myers Squibb
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https://seekingalpha.com/symbol/BMY
BMY Dividend History
https://www.nasdaq.com/market-activity/stocks/bmy/dividend-history
Bristol's Camzyos shows long-term benefit for heart condition
Aug. 28, 2023 2:05 PM ET
Bristol-Myers Squibb Company (BMY)
By: Jonathan Block, SA News Editor
New phase 3, long-term data shows that Bristol-Myers Squibb's (NYSE:BMY) Camzyos (mavacamten) led to a reduced need for invasive cardiac therapies and a sustained reduction in an indicator of cardiovascular failure.
Bristol (BMY) presented results for two trials, VALOR-HCM LTE (56 weeks follow-up) and EXPLORER-LTE (up to 120 weeks).
https://seekingalpha.com/news/4006799-bristol-camzyos-long-term-benefit-heart-condition
Acoramidis
CAMZYOS® (mavacamten)
CAMZYOS® (mavacamten)
bridgebio presents detailed positive results from phase 3 attribute-cm study of acoramidis for patients with transthyretin amyloid cardiomyopathy (attr-cm) at european society of cardiology congress 2023
08.27.2023 at 6:15 AM EDT
- The primary endpoint was met (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001); this primary endpoint result consistently favored acoramidis treatment across key subgroups, including across both variant and wild-type ATTR patients as well as across New York Heart Association (NYHA) Class I, II, and III patients. In particular, in contrast to results observed in prior studies of TTR stabilizers, consistency against cardiovascular-related hospitalizations (CVH) was observed across all prespecified subgroups at 30 months
- Absolute values observed across all-cause mortality (ACM), cardiovascular mortality (CVM) and CVH showed that over 30 months, patients survived more and were hospitalized less than has been seen in prior controlled studies of ATTR-CM to the company’s knowledge
- The 81% survival rate on acoramidis approaches the survival rate in the age-matched US database (~85%)
- The 0.29 mean annual CVH rate on acoramidis approaches the annual hospitalization rate observed in the broader US Medicare population (~0.26)
- Assessment of measures of disease progression in the trial suggest that on acoramidis, 45% of subjects experienced an improvement from baseline in N-terminal prohormone of brain natriuretic peptide (NT-proBNP) versus 9% on placebo, and 40% of subjects experienced an improvement from baseline on 6-minute walk distance (6MWD) versus 24% on placebo; to the company’s knowledge, the proportions of treated patients improving on these measures over 30 months are higher than have been observed in prior controlled studies in ATTR-CM
- Acoramidis achieved near-complete stabilization of transthyretin (TTR) in both wild-type and variant ATTR patients; serum TTR was promptly and consistently elevated throughout the study
- In an exploratory post-hoc analysis of the relationship between on-treatment serum TTR levels and on-treatment measures of CVH, NT-proBNP, and Kansas City Cardiomyopathy Questionnaire (KCCQ), there was an association between the mean on-treatment TTR level and each of these three variables, consistent with the premise that ever-higher degrees of stabilization lead to ever-better outcomes for patients
- As was previously reported, in a comparative exploratory post hoc analysis enabled by tafamidis drop-in, albeit at low patient numbers, acoramidis showed a 42% greater increase in serum TTR levels relative to placebo + tafamidis
- Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified
- Company intends to file a New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) by end of 2023
PALO ALTO, Calif., Aug. 27, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced the presentation of detailed positive results from its Phase 3 ATTRibute-CM study of acoramidis for patients with ATTR-CM by Julian Gillmore, MBBS, M.D., Ph.D., FRCP, FRCPath, in a Hot Line session at the European Society of Cardiology Congress 2023. In July, BridgeBio announced positive topline results from ATTRibute-CM, which was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent small molecule stabilizer of transthyretin (TTR). BridgeBio will host an investor call on August 28, 2023, at 8:30 am ET to discuss these results.
“The results of the ATTRibute-CM study of acoramidis in ATTR cardiomyopathy stand out for me as a clinician, not only for the highly significant outcome on the primary endpoint but for the consistency of treatment benefit across components of the primary and key secondary endpoints,” said Dr. Gillmore. “These results contribute to my anticipation that acoramidis will provide patients and their providers with an attractive new treatment option that will surely be welcomed across the community.”
A highly statistically significant result, demonstrated by a Win Ratio of 1.8 (p<0.0001), was observed on the primary endpoint (a hierarchical analysis prioritizing in order: ACM, then frequency of CVH, then change from baseline in NT-proBNP, then change from baseline in 6MWD). This result was consistent across both variant and wild-type ATTR-CM patients, as well as across NYHA Class I, II and III patients.
Other key results from the study at Month 30 included:
- An 81% survival rate on acoramidis (versus a 74% survival rate on placebo) for an absolute risk reduction of 6.4% and a relative risk reduction of 25%, despite a ~50% higher rate of tafamidis use on the placebo arm relative to the treatment arm
- The on-treatment 81% survival rate, the highest observed rate in a controlled, prospective study of ATTR-CM patients to the Company’s knowledge, approaches the survival rate in the age-matched US database (~85%)
- Of note, overall 79% of deaths in the study were cardiovascular (CV) in nature, and the results for CV-related mortality were consistent with what was observed on ACM
- The treatment group experienced a 14.9% CV-related mortality rate versus a 21.3% CV-related mortality rate in the placebo group, for a relative risk reduction of 30% on CV-related mortality
- A highly statistically significant relative risk reduction of 50% (p<0.0001) on frequency of CVH
- The 0.29 mean annual CVH frequency on acoramidis approaches the annual hospitalization rate observed in the broader US Medicare population (~0.26)
- A highly statistically significant treatment effect (p<0.0001 in each case) at 30 months on change from baseline in each of NT-proBNP, KCCQ, and 6MWD
- A higher proportion of individuals experienced a reduction (improvement from baseline) in NT-proBNP on acoramidis (45%) relative to placebo (9%)
- A higher proportion of individuals experienced an increase (improvement from baseline) in 6MWD on acoramidis (40%) relative to placebo (22%)
- To the company’s knowledge, these on-treatment proportions of improving patients are higher than have been observed in prior controlled studies of ATTR-CM
- As measured by ex vivo assays of TTR stabilization, acoramidis achieved a greater degree of TTR stabilization as compared to clinically relevant concentrations of tafamidis, independent of TTR genotype
- Serum TTR was promptly and consistently elevated throughout the study in patients receiving acoramidis
- In an exploratory post-hoc analysis of the relationship between on-treatment serum TTR levels and on-treatment measures of CVH, NT-proBNP, and KCCQ, there appears to be an association between the mean on-treatment TTR level and each of these three variables
- In a comparative exploratory post hoc analysis enabled by tafamidis drop-in, albeit at low patient numbers, acoramidis showed a 42% greater increase in serum TTR levels relative to placebo + tafamidis
- Acoramidis was generally well-tolerated with no safety findings of potential clinical concern
“The consistently positive results of ATTRibute-CM, from the primary endpoint and its components to secondary endpoints of mortality, morbidity, physical function and quality of life, further substantiate our hypothesis that highly potent TTR stabilization has the potential to deliver differential benefits to patients,” said Jonathan Fox, M.D., Ph.D., President, and Chief Medical Officer of BridgeBio Cardiorenal. “In particular, we observed absolute survival and hospitalization rates approaching those of similarly aged populations not afflicted with ATTR-CM. That 45% of completers displayed improvement from baseline in NT-proBNP, and 40% walked farther in 6 minutes at study end compared to baseline, are to me remarkable observations in a cohort with a disease commonly described as relentlessly progressive. The prompt and sustained observed increase in serum TTR, an in vivo reflection of increased stabilization, serves to further strengthen the relationship between measures of acoramidis’ ability to avidly bind and stabilize TTR and the read-through to robust clinical benefits for patients with ATTR-CM. We look forward to engaging with health authorities in our continuing efforts to secure acoramidis’ registration that will lead to our bringing acoramidis to ATTR-CM patients as quickly as possible.”
The Company intends to submit its NDA to the US FDA before the end of 2023, with regulatory filings in additional markets to follow in 2024. Acoramidis has intellectual property protection out to at least 2039.
Webcast Information
BridgeBio will host an investor call and simultaneous webcast to discuss the additional data presented at ESC 2023 for the ATTRibute-CM Phase 3 trial on Monday, August 28 at 8:30 am ET. A link to the webcast may be accessed from the event calendar page of BridgeBio’s website at https://investor.bridgebio.com/. A replay of the conference call and webcast will be archived on the Company’s website and will be available for at least 30 days following the event.
About BridgeBio Pharma, Inc.
BridgeBio Pharma Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers, and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.
BridgeBio heart drug meets primary endpoint in late-stage trial
Aug. 28, 2023 12:16 AM ET
By: Jonathan Block, SA News Editor
- Acoramidis, in development by BridgeBio (NASDAQ:BBIO) for transthyretin amyloid cardiomyopathy (ATTR-CM), met its primary goal in a phase 3 trial.
- https://seekingalpha.com/news/4006589-bridgebio-heart-drug-meets-primary-endpoint-late-stage-trial
- BridgeBio Pharma, Inc. (BBIO)
- https://bridgebio.com/what-is-attr/
- https://bridgebio.com/
biotecHOPE™ 2023
WELIREG® (belzutifan)
Repotrectinib (TPX-0005, BMS-986472)
WELIREG® (belzutifan)
Merck Announces WELIREG® (belzutifan) Phase 3 LITESPARK-005 Trial Met Primary Endpoint of Progression-Free Survival in Certain Previously Treated Patients With Advanced Renal Cell Carcinoma
August 18, 2023 6:45 am ET
First positive Phase 3 results for WELIREG from LITESPARK-005 showed statistically significant improvements in PFS versus everolimus in these patients
RAHWAY, N.J.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside of the United States and Canada, today announced topline results from LITESPARK-005, the first positive Phase 3 trial investigating WELIREG, Merck’s oral hypoxia-inducible factor-2 alpha (HIF-2α) inhibitor. LITESPARK-005 is evaluating WELIREG for the treatment of adult patients with advanced renal cell carcinoma (RCC) that has progressed following PD-1/L1 checkpoint inhibitor and vascular endothelial growth factor-tyrosine kinase inhibitor (VEGF-TKI) therapies. In the trial, WELIREG showed a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to everolimus, based on a pre-specified interim analysis conducted by an independent Data Monitoring Committee. A statistically significant improvement in the trial’s key secondary endpoint of objective response rate (ORR) was also demonstrated. A trend toward improvement in overall survival (OS), a dual primary endpoint, was observed; however, this result did not reach statistical significance. OS will be tested at a subsequent analysis. The safety profile of WELIREG in this trial was consistent with that observed in previously reported studies. Results will be presented at an upcoming medical meeting and shared with regulatory authorities.
“Patients with advanced RCC face low survival rates, and for those whose cancer progresses following PD-1/L1 and VEGF-TKI therapies, there is a need for new treatment options that can reduce their risk of disease progression or death,” said Dr. Marjorie Green, senior vice president and head of late-stage oncology, global clinical development, Merck Research Laboratories. “This is the first Phase 3 trial to show positive results in advanced RCC following these therapies and the first new mechanism to demonstrate potential in advanced RCC in recent years. We look forward to discussing these results with health authorities.”
WELIREG was the first HIF-2α inhibitor therapy approved in the U.S. and is currently approved in the U.S., Great Britain, Canada and several other countries for the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated RCC, central nervous system hemangioblastomas, or pancreatic neuroendocrine tumors, not requiring immediate surgery, based on ORR and duration of response (DOR) data from the Phase 2 LITESPARK-004 trial. Additional applications are currently under regulatory agency review worldwide based on LITESPARK-004.
LITESPARK-005 is part of a comprehensive development program for WELIREG comprised of four Phase 3 trials in RCC, including LITESPARK-011 and LITESPARK-012, evaluating WELIREG in the second-line and treatment-naïve advanced disease settings, and LITESPARK-022, evaluating WELIREG in the adjuvant setting.
About LITESPARK-005
LITESPARK-005 is a randomized, open-label Phase 3 trial (ClinicalTrials.gov, NCT04195750) evaluating WELIREG compared to everolimus for the treatment of patients with advanced RCC that has progressed after prior treatment with PD-1/L1 and VEGF-TKI therapies, in sequence or in combination. The dual primary endpoints are PFS and OS. Secondary endpoints include ORR, DOR and safety and tolerability. The trial enrolled 746 patients who were randomized to receive WELIREG (120 mg orally once daily) or everolimus (10 mg orally once daily).
About renal cell carcinoma
Renal cell carcinoma is by far the most common type of kidney cancer; about nine out of 10 kidney cancer diagnoses are RCCs. Renal cell carcinoma is about twice as common in men than in women. Most cases of RCC are discovered incidentally during imaging tests for other abdominal diseases. In the U.S., approximately 15% of patients with kidney cancer are diagnosed at an advanced stage.
About WELIREG® (belzutifan) 40 mg tablets, for oral use
Indication in the U.S.
WELIREG (belzutifan) is indicated for the treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumors (pNET), not requiring immediate surgery.
Merck’s focus on cancer
Our goal is to translate breakthrough science into innovative oncology medicines to help people with cancer worldwide. At Merck, the potential to bring new hope to people with cancer drives our purpose and supporting accessibility to our cancer medicines is our commitment. As part of our focus on cancer, Merck is committed to exploring the potential of immuno-oncology with one of the largest development programs in the industry across more than 30 tumor types. We also continue to strengthen our portfolio through strategic acquisitions and are prioritizing the development of several promising oncology candidates with the potential to improve the treatment of advanced cancers. For more information about our oncology clinical trials, visit www.merck.com/clinicaltrials.
About Merck
At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on Twitter, Facebook, Instagram, YouTube and LinkedIn.
Please see Prescribing Information, including information for the Boxed Warning about embryo-fetal toxicity, for WELIREG (belzutifan) at https://www.merck.com/product/usa/pi_circulars/w/welireg/welireg_pi.pdf and Medication Guide for WELIREG at https://www.merck.com/product/usa/pi_circulars/w/welireg/welireg_mg.pdf .
Source: Merck & Co., Inc.
Merck succeeds in Phase 3 trial for kidney cancer drug
Aug. 18, 2023 7:20 AM ET
By: Dulan Lokuwithana, SA News Editor
Merck (NYSE:MRK) announced Friday that its Phase 3 LITESPARK-005 trial reached the primary endpoint of progression-free survival in adults with advanced renal cell carcinoma who received its FDA-approved rare disease drug Welireg.
Welireg is an oral therapy indicated in the U.S. for adults with von Hippel-Lindau disease, a rare multi-system disorder characterized by non-cancerous tumor growth in various parts of the body.
Citing topline data from LITESPARK-005, the New Jersey-based pharma giant said Welireg indicated an improvement in progression-free survival (PFS) with statistically significant and clinically meaningful effects against the comparator.
https://seekingalpha.com/news/4004283-merck-wins-phase-3-trial-kidney-cancer-drug-welireg
ODRONEXTAMAB
Repotrectinib (TPX-0005, BMS-986472)
WELIREG® (belzutifan)
August 17, 2023 at 8:30 AM EDT
ODRONEXTAMAB RECEIVES EMA FILING ACCEPTANCE FOR TREATMENT OF RELAPSED/REFRACTORY FOLLICULAR LYMPHOMA AND DIFFUSE LARGE B-CELL LYMPHOMA
TARRYTOWN, N.Y., Aug. 17, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced that the European Medicines Agency (EMA) has accepted for review the Marketing Authorization Application (MAA) for odronextamab to treat adult patients with relapsed/refractory (R/R) follicular lymphoma (FL) or R/R diffuse large B-cell lymphoma (DLBCL), who have progressed after at least two prior systemic therapies. The EMA previously granted odronextamab Orphan Drug Designation for FL and DLBCL. Odronextamab is an investigational CD20xCD3 bispecific antibody designed to bridge CD20 on cancer cells with CD3-expressing T cells to facilitate local T-cell activation and cancer-cell killing.
FL and DLBCL are the two most common subtypes of B-cell non-Hodgkin lymphoma (B-NHL). FL is a slow-growing subtype, with patients often relapsing within five years. DLBCL is an aggressive subtype, with up to 50% of high-risk patients experiencing progression after first-line treatment (e.g., relapsing or refractory to treatment). As these blood cancers progress, they become increasingly hard to treat, especially in the third-line setting and beyond, leaving patients with few treatment options.
The MAA is supported by data from a Phase 1 and pivotal Phase 2 trial (ELM-1 and ELM-2) investigating odronextamab in FL and DLBCL, which were last presented at the 64th American Society of Hematology Annual Meeting.
Regeneron is conducting a broad Phase 3 development program to further investigate odronextamab in earlier lines of therapy and additional B-NHLs, representing one of the largest clinical programs in lymphoma. Odronextamab is currently under clinical development, and its safety and efficacy have not been fully evaluated by any regulatory authority.
About the Odronextamab Trials
ELM-2 is an ongoing, open-label, multicenter Phase 2 trial investigating odronextamab in more than 500 patients across five independent disease-specific cohorts, including DLBCL, FL, mantle cell lymphoma, marginal zone lymphoma and other subtypes of B-NHL. The primary endpoint is objective response rate according to the Lugano Classification, and secondary endpoints include complete response, progression-free survival, overall survival, duration of response, disease control rate, safety and quality of life.
ELM-1 is an ongoing, open-label, multicenter Phase 1 trial to investigate the safety and tolerability of odronextamab in patients with CD20+ B-cell malignancies previously treated with CD20-directed antibody therapy.
About Regeneron’s Approach to Cancer Research
At Regeneron, we’re applying more than three decades of scientific innovation with the goal of developing paradigm-changing therapies for patients with cancer.
Our portfolio is built around two foundational approaches – our approved PD-1 inhibitor Libtayo and investigational bispecific antibodies – which are being evaluated both as monotherapies and in combination with emerging therapeutic modalities. Together, they provide us with unique combinatorial flexibility to develop potentially synergistic treatments for a wide range of solid tumors and blood cancers.
If you are interested in learning more about our clinical trials, please contact us (clinicaltrials@regeneron.com or 844-734-6643) or visit our clinical trials website.
About Regeneron
Regeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led for 35 years by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous FDA-approved treatments and product candidates in development, almost all of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, hematologic conditions, infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug development process through its proprietary VelociSuite® technologies, such as VelocImmune®, which uses unique genetically humanized mice to produce optimized fully human antibodies and bispecific antibodies, and through ambitious research initiatives such as the Regeneron Genetics Center®, which is conducting one of the largest genetics sequencing efforts in the world.
For additional information about Regeneron, please visit www.regeneron.com or follow Regeneron on LinkedIn.
Source: Regeneron Pharmaceuticals, Inc.
Regeneron lymphoma therapy accepted for EU review
Aug. 17, 2023 9:28 AM ET
Regeneron Pharmaceuticals, Inc. (REGN)
By: Dulan Lokuwithana, SA News Editor
- Regeneron (NASDAQ:REGN) announced Thursday that the European Medicines Agency (EMA) accepted its marketing application for the bispecific antibody odronextamab to review it as a late-line option for adults with lymphoma, a cancer of the lymphatic system.
- Regeneron’s (REGN) marketing authorization application (MAA) for odronextamab targets adults with relapsed or refractory (R/R) follicular lymphoma or R/R diffuse large B-cell lymphoma who have received at least two prior lines of therapy.
- https://seekingalpha.com/news/4003958-regeneron-granted-eu-review-lymphoma-drug
- Regeneron Pharmaceuticals, Inc. (REGN)
- https://www.regeneron.com/
Repotrectinib (TPX-0005, BMS-986472)
Repotrectinib (TPX-0005, BMS-986472)
AKEEGA™ (Niraparib and Abiraterone Acetate)
Updated Data from TRIDENT-1 Trial Show Durable Efficacy Benefits with Repotrectinib for Patients with Locally Advanced or Metastatic ROS1-Positive Non-Small Cell Lung Cancer
08/16/2023
CATEGORY:
Repotrectinib continued to demonstrate high response rates and durable responses, including robust intracranial responses, in patients with ROS1-positive locally advanced or metastatic non-small cell lung cancer who were TKI-naïve or previously treated with one TKI and no chemotherapy
Median duration of response and progression-free survival to be disclosed for the first time in the pooled Phase 1/2 population alongside updated results at the IASLC 2023 World Conference on Lung Cancer hosted by the International Association for the Study of Lung Cancer
PRINCETON, N.J.--(BUSINESS WIRE)-- Bristol Myers Squibb (NYSE: BMY) today announced updated results from the registrational TRIDENT-1 study, demonstrating that repotrectinib, a next-generation ROS1/TRK tyrosine kinase inhibitor (TKI), continued to show high response rates and durable responses in patients with ROS1-positive locally advanced or metastatic non-small cell lung cancer (NSCLC). Updated results will be featured in an oral presentation (Abstract #OA03.06) at the IASLC 2023 World Conference on Lung Cancer (#WCLC23) hosted by the International Association for the Study of Lung Cancer on September 10, 2023, from 1:02 a.m. to 1:12 a.m. EDT / 1:02 p.m. to 1:12 p.m. SGT.
Based on results from the TRIDENT-1 trial, the U.S. Food and Drug Administration accepted the New Drug Application for repotrectinib for the treatment of patients with ROS1-positive locally advanced or metastatic NSCLC and granted Priority Review; a Prescription Drug User Fee Act goal date of November 27, 2023 was assigned.
In this updated analysis, repotrectinib continued to demonstrate durable efficacy in patients with ROS1-positive NSCLC, including intracranial activity, in patients who were TKI-naïve or previously treated with one TKI and no chemotherapy. Median duration of response (DOR) and median progression-free survival (PFS) will also be disclosed for the first time in the pooled Phase 1 and Phase 2 population of patients with ROS1-positive NSCLC:
- In TKI-naïve patients (n=71) with median follow-up of 24.0 months, confirmed objective response rate (cORR) by Blinded Independent Central Review (BICR) was 79%, median DOR and PFS were 34.1 months and 35.7 months, respectively. In patients with measurable brain metastases at baseline (n=9), intracranial ORR per BICR was 89% and responses were prolonged.
- In patients who had been previously treated with one TKI and no chemotherapy (n=56) with median follow-up of 21.5 months, cORR by BICR was 38%, and median DOR and PFS were 14.8 months and 9.0 months, respectively. In this subset of patients with measurable brain metastases at baseline (n=13), intracranial ORR per BICR was 38%.
- At the recommended dose for Phase 2, the safety profile of repotrectinib was manageable and remained consistent with previous reports.
“The data from the TRIDENT-1 trial hold great significance in the field of non-small cell lung cancer research, as they add to the growing body of evidence pointing toward durable results with repotrectinib in patients who test positive for ROS1 gene fusions,” said Byoung Chul Cho, M.D., Ph.D., Yonsei Cancer Center, Yonsei University College of Medicine, DAAN Cancer Laboratory. “With these results, we are seeing durable benefit, including in the brain, in patients with ROS1-positive NSCLC that differentiates repotrectinib from existing agents and is especially impressive in the context of historic data with current ROS1 TKIs. This targeted therapy has true potential to change the treatment landscape and become a new standard of care for patients with ROS1-positive NSCLC.”
“These updated results reflect the potential of repotrectinib as a best-in-class ROS1 inhibitor. Furthermore, the data offer hope for the patients with ROS1-positive non-small cell lung cancer who still face high remaining unmet needs,” said Joseph Fiore, executive director, global program lead, repotrectinib, Bristol Myers Squibb. “Building on our heritage of transformational science with immunotherapy in the treatment of NSCLC, we are excited to advance this next-generation precision medicine, which has shown an unprecedented level of durability of responses and robust intracranial responses in patients with ROS1-positive NSCLC, so that it can hopefully help patients in their fight against cancer.”
The study remains ongoing to assess long-term outcomes and additional endpoints across patient populations with ROS1-positive locally advanced or metastatic NSCLC and NTRK-positive advanced solid tumors. Bristol Myers Squibb thanks the patients and investigators involved with the TRIDENT-1 clinical trial.
Turning Point Therapeutics is a wholly owned subsidiary of the Bristol-Myers Squibb Company. As of August 2022, Bristol Myers Squibb acquired the leading clinical stage precision oncology company and its pipeline of investigational drugs across precision oncology and advanced solid tumors, including repotrectinib.
About TRIDENT-1
TRIDENT-1 is a Phase 1/2 open-label, global, multi-center, first-in-human clinical trial evaluating the safety, tolerability, pharmacokinetics and anti-tumor activity of repotrectinib (TPX-0005, BMS-986472) in patients with advanced solid tumors, including non-small cell lung cancer (NSCLC). Phase 1 of the trial includes several primary and secondary safety and pharmacokinetics endpoints. Phase 2 of the trial has a primary endpoint of overall response rate (ORR) as assessed by Blinded Independent Central Review (BICR) using RECIST v1.1 and key secondary endpoints including duration of response (DOR), time to response (TTR), progression-free survival (PFS), overall survival (OS) and clinical benefit rate (CBR) in six distinct expansion cohorts, including tyrosine kinase inhibitor (TKI)-naïve and TKI-pretreated patients with ROS1-positive locally advanced or metastatic NSCLC and NTRK-positive advanced solid tumors.
About Lung Cancer
Lung cancer is the leading cause of cancer deaths globally. Non-small cell lung cancer (NSCLC) is one of the most common types of lung cancer, representing up to 84% of diagnoses. Survival rates vary depending on the stage and type of the cancer when diagnosed. ROS1 fusions are rare and occur in about 1-2% of patients with NSCLC. Patients with tumors that are ROS1-positive tend to be younger than the average patient with lung cancer, more often female and may have little to no smoking history. Per international treatment guidelines, ROS1 targeted agents are preferred in patients with a tumor harboring this alteration.
Bristol Myers Squibb: Creating a Better Future for People with Cancer
Bristol Myers Squibb is inspired by a single vision — transforming patients’ lives through science. The goal of the company’s cancer research is to deliver medicines that offer each patient a better, healthier life and to make cure a possibility. Building on a legacy across a broad range of cancers that have changed survival expectations for many, Bristol Myers Squibb researchers are exploring new frontiers in personalized medicine, and through innovative digital platforms, are turning data into insights that sharpen their focus. Deep scientific expertise, cutting-edge capabilities and discovery platforms enable the company to look at cancer from every angle. Cancer can have a relentless grasp on many parts of a patient’s life, and Bristol Myers Squibb is committed to taking actions to address all aspects of care, from diagnosis to survivorship. Because as a leader in cancer care, Bristol Myers Squibb is working to empower all people with cancer to have a better future.
About Repotrectinib
Repotrectinib (TPX-0005, BMS-986472) is a next-generation, potential best-in-class tyrosine kinase inhibitor (TKI) targeting ROS1- or NTRK-positive locally advanced or metastatic solid tumors, including non-small cell lung cancer (NSCLC), where there remain significant unmet medical needs for patients. Repotrectinib was designed to improve durability of response and with favorable properties to enhance intracranial activity. It is being studied in a registrational Phase 1/2 trial primarily in adults and a Phase 1/2 trial in pediatric patients.
In June 2017, repotrectinib was granted an Orphan Drug designation by the U.S. Food and Drug Administration (FDA). Since then, repotrectinib has demonstrated clinically meaningful results and was granted three Breakthrough Therapy Designations (BTDs) by the FDA for the treatment of patients with: ROS1-positive metastatic NSCLC who have not been treated with a ROS1 TKI; ROS1-positive metastatic NSCLC who have been previously treated with one ROS1 TKI and who have not received prior platinum-based chemotherapy; and advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with one or two prior tropomyosin receptor kinase (TRK) TKIs (with or without prior chemotherapy) and have no satisfactory alternative treatments.
Repotrectinib was also previously granted four fast-track designations in patients with: ROS1-positive advanced NSCLC who have been treated with disease progression following one prior line of platinum-based chemotherapy and one prior line of a ROS1 TKI; ROS1-positive advanced NSCLC who have not been treated with a ROS1 TKI; ROS1-positive advanced NSCLC who have been previously treated with one ROS1 TKI and who have not received prior platinum-based chemotherapy; and advanced solid tumors that have an NTRK gene fusion who have progressed following treatment with at least one prior line of chemotherapy and one or two prior TRK TKIs and have no satisfactory alternative treatments.
About Bristol Myers Squibb
Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, Twitter, YouTube, Facebook and Instagram.
Source: Bristol Myers Squibb
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BMY Dividend History
https://www.nasdaq.com/market-activity/stocks/bmy/dividend-history
BMY Bristol-Myers Squibb Company
AKEEGA™ (Niraparib and Abiraterone Acetate)
REQORSA® Immunogene Therapy (quratusugene ozeplasmid),
AKEEGA™ (Niraparib and Abiraterone Acetate)
U.S. FDA Approves AKEEGA™ (Niraparib and Abiraterone Acetate), the First-And-Only Dual Action Tablet for the Treatment of Patients with BRCA-Positive Metastatic Castration-Resistant Prostate Cancer
Approval is based on the Phase 3 MAGNITUDE study, a prospectively designed precision medicine study including the largest population of BRCA-positive patients in combination trials to date with metastatic castration-resistant prostate cancer (mCRPC)
AKEEGA™ plus prednisone significantly improved radiographic progression-free survival compared to abiraterone acetate plus prednisone (AAP) in patients with BRCA-positive mCRPC
HORSHAM, Pa., August 11, 2023 – The Janssen Pharmaceutical Companies of Johnson & Johnson today announced that the U.S. Food & Drug Administration (FDA) has approved AKEEGA™ (niraparib and abiraterone acetate), the first-and-only dual action tablet combining a PARP inhibitor with abiraterone acetate, given with prednisone, for the treatment of adult patients with deleterious or suspected deleterious BRCA-positive mCRPC, as detected by an FDA-approved test.1
“As a physician, identifying patients with a worse prognosis is a priority, especially those whose cancers have a BRCA mutation,” said Kim Chi, M.D.*, Medical Oncologist at BC Cancer – Vancouver and principal investigator of the Phase 3 MAGNITUDE study. “We prospectively designed the MAGNITUDE study to identify the subset of patients most likely to benefit from targeted treatment with AKEEGA and to help us understand how we can potentially achieve better health outcomes for patients.”
Prostate cancer is one of the most common cancers in the U.S., with an estimated 288,300 new cases and nearly 35,000 deaths expected in 2023.2 Approximately 10 to 15 percent of patients with mCRPC have BRCA gene alterations. Patients with BRCA-positive mCRPC are more likely to have aggressive disease and may experience poor outcomes and a shorter survival time.3,4,5,6,7
“The approval of AKEEGA brings an important treatment option to patients with prostate cancer as they consider their road ahead, and it also highlights the importance of genetic testing and precision medicine for this disease,” said Shelby Moneer, MS, CHES**, Vice President of Patient Programs and Education, ZERO Prostate Cancer. “All individuals diagnosed with prostate cancer should consider genetic testing, especially those from racial and ethnic minority groups who tend to have worse cancer outcomes. This is imperative to close the racial and ethnic disparities in prostate cancer health outcomes.”
The FDA approval is based on positive results from the randomized, double-blind, placebo-controlled multi-center Phase 3 MAGNITUDE study. In BRCA-positive patients treated with the combination AKEEGA™ plus prednisone, a statistically significant 47 percent risk reduction was observed for radiographic progression-free survival (rPFS) (Hazard ratio [HR], 0.53; p=0.001). At the second interim analysis (IA2), with median follow-up at 24.8 months in the BRCA-positive subgroup, rPFS by central review demonstrated a consistent trend favoring AKEEGA™ plus prednisone, with a median rPFS of 19.5 months compared with 10.9 months for placebo and AAP (HR, 0.55 [95 percent confidence interval (CI), 0.39-0.78]). Additionally, there was an observed improvement in the secondary endpoints of time to symptomatic progression (TSP) (HR, 0.54 [95 percent CI, 0.35-0.85]) and time to initiation of cytotoxic chemotherapy (TCC) (HR, 0.56 [95 percent CI, 0.35-0.90]) for AKEEGA™ plus prednisone compared with AAP alone, supported by a trend towards improvement in overall survival (OS) (HR, 0.88 [95 percent CI, 0.58-1.34]).
The observed safety profile of the combination of AKEEGA™ plus prednisone was consistent with the known safety profile of each FDA-approved monotherapy. Of the patients in the MAGNITUDE study with a BRCA gene alteration, 41 percent who received AKEEGA™ experienced a serious adverse event (AE). The most common AEs occurring in 20 percent or more of patients who received AKEEGA™ plus prednisone versus patients who received placebo and AAP were musculoskeletal pain (44 percent vs. 42 percent, respectively), fatigue (43 percent vs. 30 percent), constipation (34 percent vs. 20 percent), hypertension (33 percent vs. 27 percent) and nausea (33 percent vs. 21 percent). Permanent discontinuation of any component of AKEEGA™ due to an adverse reaction occurred in 15 percent of patients.
“Janssen’s legacy of advancing the science of prostate cancer has contributed to the evolution of transformational treatment approaches for more than a decade,” said Kiran Patel, M.D., Vice President, Clinical Development, Solid Tumors, Janssen Research & Development, LLC. “This milestone, which marks the approval of Janssen’s third prostate cancer treatment, highlights the importance of advancing precision medicine approaches and genetic testing for the treatment of patients with BRCA-positive mCRPC.”
About AKEEGA™
AKEEGA™ (niraparib and abiraterone acetate) is the first-and-only orally administered, once daily dual action tablet (DAT) of niraparib, a highly selective poly (ADP-ribose) polymerase (PARP) inhibitor, and abiraterone acetate, an androgen biosynthesis inhibitor. The novel DAT combination therapy targets two oncogenic drivers in patients with mCRPC and HRR gene alterations. AKEEGA™ is indicated with prednisone for the treatment of adults with mCRPC and BRCA-positive mutations. The recommended starting dose is 200 mg niraparib/1,000 mg abiraterone acetate (two tablets). The 100 mg niraparib/1,000 mg abiraterone acetate dose option (two tablets) is available for dose reduction.
In April 2016, Janssen Biotech, Inc. entered a worldwide (except Japan) collaboration and license agreement with TESARO, Inc. (acquired by GlaxoSmithKline [GSK] in 2019) for exclusive rights to niraparib in prostate cancer.
About Metastatic Castration-Resistant Prostate Cancer
Metastatic castration-resistant prostate cancer characterizes cancer that no longer responds to androgen deprivation therapy and has spread to other parts of the body. The most common metastatic sites are bones, followed by lungs and liver. Prostate cancer is the second most common cancer in men worldwide, behind lung cancer. More than one million patients around the world are diagnosed with prostate cancer each year. Patients with mCRPC and HRR gene alterations, of which BRCA mutations are the most common, are more likely to have aggressive disease, poor outcomes and a shorter survival time.2,3,4,5
About MAGNITUDE
MAGNITUDE (NCT03748641) is a Phase 3, randomized, double-blind, placebo-controlled, multi-center clinical study evaluating the safety and efficacy of the combination of AKEEGA™ plus prednisone for patients with mCRPC, with or without certain HRR gene alterations, and who have not received prior therapy for mCRPC except for up to four months of AAP.
The study included patients with (HRR biomarker [BM] positive; ATM, BRCA1, BRCA2, BRIP1, CDK12, CHEK2, FANCA, HDAC2, PALB2) and without specified gene alterations (HRR BM negative), who were randomized 1:1 to receive niraparib 200 mg once daily plus AAP or placebo plus AAP.8 A total of 423 patients with HRR gene alterations were enrolled, 225 (53.2 percent) of whom had BRCA mutations.9,10,11 The primary endpoint of the MAGNITUDE trial was rPFS assessed by blinded independent central review.12 Secondary endpoints included TCC, TSP and OS. Analysis of the group of patients with BRCA alterations was alpha controlled for rPFS and prespecified for other endpoints.
Access to AKEEGA™ (niraparib and abiraterone acetate)
Once a patient and their doctor have decided that AKEEGA™ is right for the patient, Janssen can help find the resources patients may need to get started and stay on therapy. Janssen offers support navigating the payer processes to assist patients in gaining access to AKEEGA™. We can provide information on insurance coverage and potential out-of-pocket costs and identify options that may help make AKEEGA™ more affordable. If patients have commercial or private health insurance and need help paying for AKEEGA™, the Janssen CarePath Savings Program may be able to help. To learn more about access and affordability support visit JanssenCarePath.com or call 1-877-CarePath (1-877-227-3728). We also offer personalized support for patients that provides one-on-one guidance, information, and educational resources, which includes helping them explore cost support options. Patients can get connected to dedicated support from our Care Navigators by calling Janssen Compass® directly at 844-628-1234 or by visiting JanssenCompass.com and requesting an introductory call. Janssen Compass® is limited to education for patients about their Janssen therapy, its administration, and/or their disease. It is intended to supplement a patient’s understanding of their therapy and is not intended to provide medical advice, replace a treatment plan from the patient’s doctor or nurse, provide case management services, or serve as a reason to prescribe a Janssen medication.
About the Janssen Pharmaceutical Companies of Johnson & Johnson
At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.
Learn more at www.janssen.com. Follow us at @JanssenGlobal and @JanssenUS. Janssen Biotech, Inc. and Janssen Research & Development, LLC are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
JNJ Dividend History
https://www.nasdaq.com/market-activity/stocks/jnj/dividend-history
TALVEY™ (talquetamab-tgvs)
REQORSA® Immunogene Therapy (quratusugene ozeplasmid),
REQORSA® Immunogene Therapy (quratusugene ozeplasmid),
U.S. FDA Approves TALVEY™ (talquetamab-tgvs), a First-in-Class Bispecific Therapy for the Treatment of Patients with Heavily Pretreated Multiple Myeloma
Bispecific antibody targeting GPRC5D receptor showed an overall response rate of more than
70 percent with durable responses, including in patients previously treated with a bispecific antibody or CAR-T cell therapy
HORSHAM, Pa., Aug. 10, 2023 – The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the U.S. Food and Drug Administration (FDA) has granted accelerated approval of TALVEY™ (talquetamab-tgvs), a first-in-class bispecific antibody for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody.1 This indication is approved under accelerated approval based on response rate and durability of response.1 Continued approval for this indication is contingent upon verification and description of clinical benefit in confirmatory trial(s).1
TALVEY™ is a bispecific T-cell engaging antibody that binds to the CD3 receptor on the surface of T cells and G protein-coupled receptor class C group 5 member D (GPRC5D) expressed on the surface of multiple myeloma cells, non-malignant plasma cells and healthy tissue such as epithelial cells in keratinized tissues of the skin and tongue.1 TALVEY™ is approved as a weekly or biweekly subcutaneous (SC) injection after an initial step-up phase, offering physicians the flexibility to determine the optimal treatment regimen for patients.1
“The clinically meaningful efficacy and safety profile observed with talquetamab in heavily pretreated patients in this clinical trial, which included patients treated with prior BCMA-targeted bispecific or CAR-T cell therapy, has been notable,” said Ajai Chari, M.D., Director of Multiple Myeloma Program, Professor of Clinical Medicine at the University of California, San Francisco.* “Patients at this stage of disease have a poor prognosis. Talquetamab as a first-in-class therapy is a new option for patients with this difficult-to-treat blood cancer.”
The talquetamab Phase 2 MonumenTAL-1 study, which included patients who had received at least four prior lines of therapy and who were not exposed to prior T-cell redirection therapy (n=187), showed meaningful overall response rates (ORR).1 At the SC biweekly dose of 0.8 mg/kg, 73.6 percent of patients (95 percent Confidence Interval [CI], range, 63.0 to 82.4) achieved an ORR.1 With a median follow-up of nearly 6 (range, 0 to 9.5) months from first response among responders, 58 percent of patients achieved a very good partial response (VGPR) or better, including 33 percent of patients achieving a complete response (CR) or better.1 At the SC weekly dose of 0.4 mg/kg, 73.0 percent of patients (95 percent CI, range, 63.2 to 81.4) achieved an ORR.1 With a median follow-up of nearly 14 (range, 0.8 to 15.4) months from first response among responders, 57 percent of patients achieved a VGPR or better, including 35 percent of patients achieving a CR or better.1 Responses were durable with a median duration of response not reached in the 0.8 mg/kg SC biweekly dose group and 9.5 months in the 0.4 mg/kg SC weekly dose group.1 Among patients receiving the 0.8 mg/kg SC biweekly dose, an estimated 85 percent of responders maintained response for at least 9 months.1
The MonumenTAL-1 study also included 32 patients who were exposed to prior bispecific antibody or CAR-T cell therapy (94 percent B-cell maturation antigen [BCMA]-directed therapy) and had received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody, received TALVEY at the 0.4 mg/kg SC weekly dose.1 With a median duration of follow-up of 10.4 months, 72 percent of patients (95 percent CI, range, 53 to 86) achieved an ORR per an Independent Review Committee assessment, and an estimated 59 percent of responders maintained response for at least 9 months.1
The Safety Profile for TALVEY™ includes a Boxed Warning for cytokine release syndrome (CRS) and neurologic toxicity including immune effector cell-associated neurotoxicity syndrome (ICANS); Warnings and Precautions include Oral Toxicity and Weight Loss, Infections, Cytopenias, Skin Toxicity, Hepatoxicity and Embryo-fetal toxicity. The most common adverse reactions (≥20 percent) are pyrexia, CRS, dysgeusia, nail disorder, musculoskeletal pain, skin disorder, rash, fatigue, weight decreased, dry mouth, xerosis, dysphagia, upper respiratory tract infection, diarrhea, hypotension, and headache. The most common Grade 3 or 4 laboratory abnormalities (≥30 percent) are lymphocyte count decreased, neutrophil count decreased, white blood cell decreased, and hemoglobin decreased.1
"Although options for the treatment of multiple myeloma have expanded significantly in recent years, the disease remains incurable, and therefore, patients are in need of new treatment options,” said Michael Andreini, President and Chief Executive Officer, Multiple Myeloma Research Foundation.† “Today’s approval of talquetamab provides patients with a new treatment approach for relapsed or refractory disease that is a welcome addition to the myeloma community.”
“The approval of TALVEY, our fifth innovative therapy and second bispecific antibody approved for the treatment of multiple myeloma, demonstrates our commitment to expanding our portfolio of medicines to help address unmet needs for patients who continue to face challenges with this complex hematologic malignancy,” said Peter Lebowitz, M.D., Ph.D., Global Therapeutic Area Head, Oncology, Janssen Research & Development, LLC. “Our team of scientists never settles in their determination to discover and develop effective therapies. With the discovery of this new antigen, we continue to strive for research breakthroughs while remaining focused on delivering curative regimens in our commitment to eliminate cancer.”
TALVEY™ is available only through a restricted program called the TECVAYLI® and TALVEY™ Risk Evaluation and Mitigation Strategy (REMS).1 Details of the Important Safety Information are included below.
The most common non-hematologic adverse events observed in the study were oral toxicities, which occurred in 80 percent of patients, with Grade 3 occurring in 2.1 percent of patients.1 The most frequent oral toxicities were dysgeusia (49 percent), dry mouth (34 percent), dysphagia (23 percent), and ageusia (18 percent).1 In addition, 62 percent of patients experienced weight loss, including 29 percent with Grade 2 weight loss and 2.7 percent with Grade 3 weight loss.1 Serious infections occurred in 16 percent of patients, with fatal infections occurring in 1.5 percent of patients.1 Grade 3 or 4 serious infections occurred in 17 percent of patients.1 Grade 3 or 4 decreased neutrophils occurred in 35 percent of patients and decreased platelets occurred in 22 percent of patients.1 Skin reactions occurred in 62 percent of patients, with Grade 3 skin reactions in 0.3 percent.1 Permanent discontinuation of TALVEY™ due to an adverse reaction occurred in 9 percent of patients.1
About the MonumenTAL-1 Study
MonumenTAL-1 (Phase 1: NCT03399799, Phase 2: NCT04634552) is a Phase 1/2 single-arm, open-label, multicohort, multicenter dose-escalation study involving over 300 patients.2,3 Phase 1 evaluated the safety and efficacy of TALVEY™ in adults with relapsed or refractory multiple myeloma who received three or more prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 monoclonal antibody.1,2 The study excluded patients who experienced T-cell redirection therapy within 3 months, prior Grade 3 or higher CRS related to any T-cell redirection therapy, an autologous stem cell transplant within the past 12 weeks, an allogenic stem cell transplant within the past 6 months, Eastern Cooperative Oncology Group (ECOG) performance score of 3 or higher, stroke or seizure within the past 6 months, CNS involvement or clinical signs of meningeal involvement of multiple myeloma, and plasma cell leukemia, active or documented history of autoimmune disease (exception of vitiligo, resolved childhood atopic dermatitis or resolved Grave’s Disease that is euthyroid based on clinical and laboratory testing).1,2
Phase 2 of the study evaluated the efficacy of TALVEY™ in participants with relapsed or refractory multiple myeloma at the recommended Phase 2 dose(s) (RP2Ds), established at SC 0.4 mg/kg weekly and 0.8 mg/kg every two weeks, respectively.3 Efficacy was based on overall response rate (ORR) and duration of response (DOR) as assessed by an Independent Review Committee using IMWG criteria.3
TECVAYLI® and TALVEY™ REMS: TALVEY™ is available only through a restricted program under a REMS called the TECVAYLI® and TALVEY™ REMS because of the risks of CRS and neurologic toxicity, including ICANS.
Further information about the TECVAYLI® and TALVEY™ REMS program is available at www.TEC-TALREMS.com or by telephone at 1-855-810-8064.
Please read full Prescribing Information including Boxed Warning for TALVEY™.
About TALVEY™
TALVEY™ is a bispecific T-cell engaging antibody that binds to the CD3 receptor expressed on the surface of T cells and G protein-coupled receptor class C group 5 member D (GPRC5D), a novel multiple myeloma target which is highly expressed on the surface of multiple myeloma cells and non-malignant plasma cells, as well as some healthy tissues such as epithelial cells of the skin and tongue.1
TALVEY™ is currently being investigated in combination and in sequence across all lines of multiple myeloma in studies with other bispecific antibodies as well as with existing standards of care. In addition to a Phase 1/2 clinical study of TALVEY™ for the treatment of relapsed or refractory multiple myeloma, TALVEY™ is also being evaluated in combination studies (NCT04586426, NCT04108195, NCT05050097, NCT05338775) and in a randomized Phase 3 study (NCT05455320).
In May 2021 and August 2021, TALVEY™ was granted Orphan Drug Designation for the treatment of multiple myeloma by the U.S. FDA and the European Commission, respectively. TALVEY™ was also granted Breakthrough Therapy Designation from the U.S. FDA in June 2022 for the treatment of adult patients with relapsed or refractory multiple myeloma who have previously received at least four prior lines of therapy, including a proteasome inhibitor, an immunomodulatory agent, and an anti-CD38 antibody. The approval follows the FDA’s decision in February 2023 to initiate a Priority Review of the Biologics License Application (BLA) submitted in December 2022.
About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.4 In multiple myeloma, these plasma cells change, spread rapidly and replace normal cells in the bone marrow with tumors.5 Multiple myeloma is the third most common blood cancer and remains an incurable disease.6 In 2023, it is estimated that more than 35,000 people will be diagnosed with multiple myeloma in the U.S. and more than 12,000 people will die from the disease.7 People living with multiple myeloma have a 5-year relative survival rate of 59.8 percent.8 While some people diagnosed with multiple myeloma initially have no symptoms, most patients are diagnosed due to symptoms that can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels and kidney problems or infections.9,10
About the Janssen Pharmaceutical Companies of Johnson & Johnson
At Janssen, we are creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.
Learn more at www.janssen.com. Follow us at @JanssenUS and @JanssenGlobal. Janssen Biotech, Inc., and Janssen Research & Development, LLC, are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
* Dr. Chari has served as a paid consultant to Janssen; he has not been paid for any media work.
† Mr. Andreini has not been paid for any media work.
Johnson & Johnson wins FDA nod for new multiple myeloma therapy
Aug. 10, 2023 8:06 AM ET
By: Dulan Lokuwithana, SA News Editor1 Comment
- The Janssen Pharmaceutical unit of Johnson & Johnson (NYSE:JNJ) announced Thursday that the U.S. FDA granted accelerated approval for its bispecific antibody Talvey as a late-line option for adults with relapsed or refractory multiple myeloma.
- https://seekingalpha.com/news/4000875-johnson-johnson-wins-fda-nod-multiple-myeloma-therapy?mailingid=32355042&messageid=2900&serial=32355042.14041&utm_campaign=rta-stock-news&utm_content=link-1&utm_medium=email&utm_source=seeking_alpha&utm_term=32355042.14041
- Johnson & Johnson (JNJ)LGND
- https://www.jnj.com/
- https://www.janssen.com/
REQORSA® Immunogene Therapy (quratusugene ozeplasmid),
REQORSA® Immunogene Therapy (quratusugene ozeplasmid),
REQORSA® Immunogene Therapy (quratusugene ozeplasmid),
Genprex Granted FDA Orphan Drug Designation (ODD) for REQORSA® Immunogene Therapy for the Treatment of Small Cell Lung Cancer
August 10, 2023
ODD is in addition to Three FDA Fast Track Designations
Genprex expects to dose the first patient in the Acclaim-3 clinical trial in the fourth quarter of 2023
AUSTIN, Texas — (August 10, 2023) — Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to the Company’s lead drug candidate, REQORSA® Immunogene Therapy (quratusugene ozeplasmid), for the treatment of small cell lung cancer (SCLC).
In addition to ODD for the treatment of SCLC, in June 2023, the FDA granted Fast Track Designation (FTD) for REQORSA Immunogene Therapy, in combination with Genentech, Inc’s Tecentriq® in patients with extensive-stage small cell lung cancer (ES-SCLC) who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment. With ES-SCLC, the cancer has spread from one lung to the other, or to other parts of the body. Extensive-stage is the most common type of SCLC. FDA has also granted Genprex FTD for two other indications of REQORSA Immunogene Therapy, including REQORSA in combination with Tagrisso for non-small cell lung cancer (NSCLC) in patients who have progressed after Tagrisso treatment, and REQORSA in combination with Keytruda for NSCLC in patients who have progressed after Keytruda treatment.
“We are excited to receive Orphan Drug Designation from the FDA for REQORSA for patients with SCLC,” said Rodney Varner, President, Chairman and Chief Executive Officer at Genprex. “This FDA Orphan Drug Designation in combination with our recently received FDA Fast Track designation underscores the great need for better treatment options for patients with SCLC, ES-SCLC and NSCLC. We look forward to initiating the Acclaim-3 clinical trial expected in the fourth quarter of 2023 in order to bring hope of an effective new therapy to patients suffering with this life-limiting cancer.”
The FDA grants ODD status to investigational therapies being developed to treat, diagnose, or prevent a rare disease or condition affecting fewer than 200,000 people in the United States. Further, ODD provides benefits to drug developers, including assistance in the drug development process, tax credits for qualified trials, waiver of certain FDA fees, and potential for seven years of post-approval marketing exclusivity.
Genprex’s method of treating cancer is to reexpress tumor suppressor genes in cancers. Tumor suppressor genes are deleted or inactivated early in the process of cancer development. REQORSA contains a plasmid that expresses a tumor suppressor gene protein called TUSC2. Virtually 100% of small cell lung cancers have reduced or no TUSC2 protein expression, and 41% completely lack TUSC2 protein expression. Pre-clinical studies in mice suggest that re-expressing the TUSC2 protein may lead to clinical efficacy. ES-SCLC has a very poor prognosis, with a median progression free survival (PFS) of only 5.2 months. Importantly, median PFS for patients receiving Tecentriq as maintenance therapy is only 2.6 months from the start of maintenance treatment, so there is a great need for improvement in maintenance therapy.
About Acclaim-3 Clinical Trial
The Acclaim-3 clinical trial is a Phase 1/2 open-label, dose escalation and clinical response study of maintenance therapy evaluating REQORSA in combination with Tecentriq® in patients with ES-SCLC who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment.
Patients in the Acclaim-3 clinical trial will be enrolled after receiving initial treatment with 3-4 cycles of carboplatin, etoposide, and Tecentriq, and achieving complete response, partial response or stable disease. They will then receive treatment with REQORSA and Tecentriq as maintenance therapy every 21 days until disease progression.
The Phase 1 dose escalation portion of the Acclaim-3 clinical trial is expected to enroll up to 12 patients at 3-5 U.S. clinical sites to determine the Maximum Tolerated Dose (MTD). If no dose limiting toxicities occur during Phase 1, then the highest dose evaluated will be the Recommended Phase 2 Dose. The Phase 2 portion of the study will then enroll approximately 50 patients at 5-10 sites. Patients will be treated with REQORSA and Tecentriq until disease progression or unacceptable toxicity is experienced.
The primary endpoint of the Phase 2 portion of the trial is to determine the 18-week progression-free survival rate from the time of the start of maintenance therapy with REQORSA and Tecentriq in patients with ES-SCLC. Patients will also be followed for survival. A Phase 2 futility analysis will be performed after the 25th patient enrolled and treated reaches 18 weeks of follow up.
About Genprex, Inc.
Genprex is a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes. Genprex’s technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. Genprex’s oncology program utilizes its proprietary, non-viral ONCOPREX® Nanoparticle Delivery System which encapsulates the gene-expressing plasmids using lipid nanoparticles. The resultant product is administered intravenously, where it is taken up by tumor cells that then express tumor suppressor proteins that were deficient in the tumor. The Company’s lead product candidate, REQORSA® (quaratusugene ozeplasmid), is being evaluated in three clinical trials as a treatment for non-small cell lung cancer (NSCLC) and small cell lung cancer (SCLC). Each of Genprex’s three lung cancer clinical programs have received a Fast Track Designation from the Food and Drug Administration (FDA), and its SCLC program has received an FDA Orphan Drug Designation. Genprex’s diabetes gene therapy approach is comprised of a novel infusion process that uses an adeno-associated virus (AAV) vector to deliver Pdx1 and MafA genes directly to the pancreas. In models of Type 1 diabetes, GPX-002 transforms alpha cells in the pancreas into functional beta-like cells, which can produce insulin but may be distinct enough from beta cells to evade the body’s immune system. In a similar approach, GPX-003 for Type 2 diabetes, where autoimmunity is not at play, is believed to rejuvenate and replenish exhausted beta cells.
Interested investors and shareholders are encouraged to sign up for press releases and industry updates by visiting the Company Website, registering for Email Alerts and by following Genprex on Twitter, Facebook and LinkedIn.
Genprex wins FDA orphan drug designation for lead asset
Aug. 10, 2023 8:30 AM ET
By: Dulan Lokuwithana, SA News Editor
- Gene therapy developer Genprex (NASDAQ:GNPX) announced Thursday that the U.S. FDA granted Orphan Drug Designation for its lead therapeutic candidate, Reqorsa Immunogene Therapy, as a treatment for small cell lung cancer (SCLC).
- With its orphan drug designation, the FDA aims to offer financial incentives such as tax credits for clinical trial costs and seven years of marketing exclusivity for treatments targeted at rare diseases and conditions.
- https://seekingalpha.com/news/4000900-genprex-stock-gains-fda-orphan-drug-tag-lead-asset
- Genprex, Inc. (GNPX)
- https://www.genprex.com/
- https://www.genprex.com/technology/reqorsa/
- https://www.genprex.com/clinical-trials/pipeline/
ZURZUVAE™ (zuranolone)
TALVEY®▼ (talquetamab) and TECVAYLI®▼ (teclistamab)
TALVEY®▼ (talquetamab) and TECVAYLI®▼ (teclistamab)
FDA Approves ZURZUVAE™ (zuranolone), the First and Only Oral Treatment Approved for Women with Postpartum Depression, and Issues a Complete Response Letter for Major Depressive Disorder
AUGUST 4, 2023 • NEWS RELEASE
Postpartum depression (PPD) approval based on results from two Phase 3 clinical trials; in the SKYLARK Study treatment with ZURZUVAE rapidly improved symptoms of PPD at Day 15 and as early as Day 3 with sustained effect to Day 45
Mental health conditions are the leading cause of maternal mortality1 with PPD among the most common complications during and after pregnancy2
CAMBRIDGE, Mass., Aug. 04, 2023 (GLOBE NEWSWIRE) -- Biogen Inc. (Nasdaq: BIIB) and Sage Therapeutics, Inc. (Nasdaq: SAGE) announced the U.S. Food and Drug Administration (FDA) approved ZURZUVAE™ (zuranolone) 50 mg for adults with postpartum depression (PPD). ZURZUVAE is the first and only oral, once-daily, 14-day treatment that can provide rapid improvements in depressive symptoms for women with PPD. ZURZUVAE is expected to launch and be commercially available in the fourth quarter of 2023 shortly following scheduling as a controlled substance by the U.S. Drug Enforcement Administration, which is anticipated to occur within 90 days.
Additionally, the FDA issued a Complete Response Letter (CRL) for the New Drug Application (NDA) for zuranolone in the treatment of adults with major depressive disorder (MDD). The CRL stated that the application did not provide substantial evidence of effectiveness to support the approval of zuranolone for the treatment of MDD and that an additional study or studies will be needed. Biogen and Sage are reviewing the feedback and evaluating next steps.
“The approval of ZURZUVAE to treat postpartum depression is a major milestone for the hundreds of thousands of women who experience this underdiagnosed and undertreated condition,” said Christopher A. Viehbacher, President and Chief Executive Officer at Biogen. “We appreciate the support of patients, patient advocates and researchers who helped to reach this milestone. We believe that ZURZUVAE will be an important option to treat PPD and we will thoroughly review the feedback from the FDA on the use of zuranolone in MDD to determine next steps.”
“Maternal mental health has been sidelined for far too long, but today’s approval of ZURZUVAE helps to change that. Women have been waiting for an oral medicine that can specifically and rapidly improve the symptoms of PPD and we are proud to be able to deliver that,” said Barry Greene, Chief Executive Officer at Sage Therapeutics. “In regard to the CRL for MDD, we are highly disappointed for patients, particularly amid the current mental health crisis and millions of people with MDD struggling to find symptom relief. We remain committed to our mission to deliver life-changing brain health medicines.”
The approval of ZURZUVAE to treat women with PPD is based on the NEST clinical development program, which included two studies in adult women with PPD (ROBIN and SKYLARK Studies). Both studies met their primary endpoint, a significant mean reduction from baseline in the 17-item Hamilton Rating Scale for Depression (HAMD-17) total score, a common measure of depression severity, at Day 15 as compared to placebo. In the SKYLARK Study evaluating ZURZUVAE 50 mg, all key secondary endpoints were met, with significant reduction in depressive symptoms seen as early as Day 3 and sustained through Day 45. ZURZUVAE was generally well-tolerated with a consistent safety profile across both studies. The most common side effects ≥ 5% and greater than placebo in patients treated with ZURZUVAE 50 mg were somnolence, dizziness, diarrhea, fatigue and urinary tract infection. The labeling includes a boxed warning that instructs healthcare providers to advise people that ZURZUVAE causes driving impairment due to central nervous system (CNS) depressant effects. People who take ZURZUVAE should not drive a motor vehicle or engage in other potentially hazardous activities that require complete mental alertness until at least 12 hours after ZURZUVAE administration for the duration of the 14-day treatment course. Patients may not be able to assess their own degree of impairment.
“Today marks a groundbreaking day for the treatment of PPD, as with ZURZUVAE we now have an oral treatment option that can provide rapid improvements in depressive symptoms in as early as three days for women with PPD,” said Dr. Kristina Deligiannidis, a principal investigator in the ZURZUVAE clinical development program and Professor, The Feinstein Institutes for Medical Research in Manhasset, New York. “As a perinatal psychiatrist, I see the devastating impact PPD has on mothers particularly on the important mother-infant bond and long-term child development. Once available, I believe ZURZUVAE will be a meaningful option for patients in need.”
According to the Centers for Disease Control and Prevention, mental health conditions are the leading cause of maternal mortality1 with PPD among the most common complications during and after pregnancy.2 In the U.S., it is estimated approximately 1 in 8 women experience symptoms of PPD.3 Approximately half of all PPD cases may go undiagnosed without appropriate screening.4,5 Research shows only 15.8% of women with PPD symptoms receive treatment.6 PPD symptoms may persist beyond the postpartum period and can lead to prolonged maternal morbidity.7-9 Symptoms of PPD can include depressed mood, loss of interest in activities, changes in sleep patterns and appetite, decreased energy, feelings of guilt or worthlessness, trouble concentrating and in some cases thoughts of suicide.9
“Today’s approval is welcome news for the estimated 500,000 women in the United States who report experiencing symptoms of this devastating and often misunderstood illness each year,” said Wendy N. Davis, Ph.D., PMH-C, Executive Director at Postpartum Support International. “Women with PPD desperately need prompt care and additional treatment options that can provide quick relief so they can be healthy and present during this momentous time in their lives.”
Indication and Important Safety Information for ZURZUVAE
What is ZURZUVAE?
ZURZUVAE is a prescription medicine used to treat adults with postpartum depression (PPD).
It is not known if ZURZUVAE is safe and effective in children.
Please see the Full Prescribing Information, including Boxed WARNING, and Medication Guide for ZURZUVAE.
About ZURZUVAE™ (zuranolone)
ZURZUVAE is a once-daily, oral, 14-day medicine for the treatment of adults with postpartum depression (PPD). ZURZUVAE is a neuroactive steroid (NAS) GABA-A receptor positive allosteric modulator (PAM). The GABA system is the major inhibitory signaling pathway of the brain and central nervous system and contributes to regulating brain function.
About Biogen
Founded in 1978, Biogen is a leading global biotechnology company that has pioneered multiple breakthrough innovations including a broad portfolio of medicines to treat multiple sclerosis, the first approved treatment for spinal muscular atrophy, and two co-developed treatments to address a defining pathology of Alzheimer’s disease. Biogen is advancing a pipeline of potential novel therapies across neurology, neuropsychiatry, specialized immunology and rare diseases and remains acutely focused on its purpose of serving humanity through science while advancing a healthier, more sustainable and equitable world.
We routinely post information that may be important to investors on our website at www.biogen.com. Follow us on social media - Twitter, LinkedIn, Facebook, YouTube.
About Sage Therapeutics
Sage Therapeutics is a biopharmaceutical company fearlessly leading the way to create a world with better brain health. Our mission is to pioneer solutions to deliver life-changing brain health medicines, so every person can thrive. For more information, please visit www.sagerx.com.
Sage, Biogen postpartum depression pill zuranolone wins FDA approval
Aug. 04, 2023 7:53 PM ET
Sage Therapeutics, Inc. (SAGE)BIIB
By: Jonathan Block, SA News Editor10 Comments
- The US FDA has approved Sage Therapeutics' (NASDAQ:SAGE) and Biogen' (BIIB) Zurzuvae (zuranolone), the first oral treatment for postpartum depression.
- https://seekingalpha.com/news/3997942-sage-biogen-postpartum-depression-pill-zuranolone-wins-fda-approval
- Sage Therapeutics, Inc. (SAGE)BIIB
- https://www.biogen.com/
- https://www.sagerx.com/
TALVEY®▼ (talquetamab) and TECVAYLI®▼ (teclistamab)
TALVEY®▼ (talquetamab) and TECVAYLI®▼ (teclistamab)
TALVEY®▼ (talquetamab) and TECVAYLI®▼ (teclistamab)
Janssen Receives Positive CHMP Opinions for Novel Bispecific Antibodies TALVEY®▼ (talquetamab) and TECVAYLI®▼ (teclistamab) for the Treatment of Patients with Relapsed and Refractory Multiple Myeloma
Talquetamab is the first therapy targeting GPRC5D to receive a positive CHMP Opinion
Teclistamab, the first BCMA-targeting bispecific antibody to be approved in Europe, receives positive CHMP Opinion for reduced, biweekly dosing schedule
BEERSE, Belgium, 21 July 2023 –
The Janssen Pharmaceutical Companies of Johnson & Johnson announced today that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended conditional marketing authorisation (CMA) for TALVEY® (talquetamab) as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma (RRMM) who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy. Talquetamab is a subcutaneous bispecific antibody that binds G protein-coupled receptor class C group 5 member D (GPRC5D), a novel target on multiple myeloma cells, and CD3, on T-cells.1
The CHMP also recommended the approval of a Type II variation for teclistamab, providing a reduced, biweekly dosing schedule of 1.5mg/kg every other week in patients who have achieved a complete response or better for six months or longer. Teclistamab is the first bispecific antibody targeting B-cell maturation antigen (BCMA) on myeloma cells, and CD3 on T-cells to be licensed in Europe for the treatment of adult patients with RRMM who have had at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.2
Despite recent advances, multiple myeloma remains a highly heterogenous and incurable disease that is unique to every patient.3 As the disease progresses and with each successive line of treatment, responses tend to decrease and patient outcomes become progressively worse.4 An unmet need remains for more therapeutic options with different modes of action, including for patients treated with prior bispecific or CAR-T cell therapies, to better address the unique characteristics of every patient’s individual needs through different cellular targets.3
“With talquetamab, a novel bispecific antibody targeting GPRC5D, we look to build on our legacy of innovation and bring forward a vital new treatment option for patients with relapsed and refractory multiple myeloma, who have a poor prognosis,” said Edmond Chan, MBChB M.D. (Res), Senior Director EMEA Therapeutic Area Lead Haematology, Janssen-Cilag Limited. “Today’s recommendation from the CHMP marks an exciting step for patients who continue to face the challenges of this difficult-to-treat blood cancer. We look forward to working with health authorities to bring talquetamab to patients in need across the region as soon as possible, while we continue our focus on enhancing a robust multiple myeloma portfolio of therapeutics and regimens.”
The CHMP recommendation for talquetamab is based on data from the Phase 1/2 MonumenTAL-1 study (Phase 1: NCT03399799; Phase 2: NCT04634552), evaluating the safety profile and efficacy of talquetamab in patients with RRMM. The latest data from the study were recently presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting (2-6 June, Chicago) and the 2023 European Hematology Association (EHA) Hybrid Congress (8-11 June, Frankfurt).
The CHMP recommendation for teclistamab is based on data from the Phase 1/2 MajesTEC-1 study (Phase 1: NCT03145181; Phase 2: NCT04557098), evaluating the safety profile and efficacy of teclistamab in patients with RRMM. Data from the study were recently presented at the 2023 ASCO Annual Meeting.
“Pending approval, this variation for teclistamab will be an important step forward for this first BCMA bispecific therapy, offering flexible, less frequent dosing depending on a patient’s response,” said Sen Zhuang, M.D., Ph.D., Vice President, Clinical Research and Development, Janssen Research & Development, LLC. “Today's positive recommendations for talquetamab and teclistamab, two novel bispecific antibodies discovered and developed at Janssen, reinforce our commitment to delivering innovative treatment options for patients with multiple myeloma.”
#ENDS#
About Talquetamab
Talquetamab is a bispecific T-cell engaging antibody that binds to CD3, on T-cells, and GPRC5D, a novel multiple myeloma target which is highly expressed on myeloma cells and hard keratinised tissues, with minimal to no expression detected on B-cells or B-cell precursors.1,5
Talquetamab, which is administered by subcutaneous injection, is currently being evaluated in several monotherapy and combination studies.6,7,8,9,10,11,12
CMA is the approval of a medicine that addresses unmet medical needs of patients based on less comprehensive data than normally required, where the available data suggest that the benefits of the medicine outweigh the risks, and the applicant can provide comprehensive clinical data in the future.13 Prior to the CHMP recommending this CMA, the EMA granted talquetamab PRIority Medicines (PRIME) designation in January 2021 and accelerated assessment in November 2022. The U.S. Food and Drug Administration (FDA) granted talquetamab Breakthrough Therapy Designation in June 2022. Janssen also received Orphan Drug Designation for talquetamab from the EMA in August 2021 and the FDA in May 2021.
About Teclistamab
Teclistamab is an off-the-shelf (or ready to use) bispecific antibody.2 Teclistamab, a subcutaneous injection, redirects T-cells through two cellular targets (BCMA and CD3) to activate the body’s immune system to fight the cancer. Teclistamab is currently being evaluated in several monotherapy and combination studies.14,15,16,17,18,19
Teclistamab received European Commission (EC) approval in August 2022. The application for CMA was reviewed by the CHMP under an accelerated timetable to enable faster patient access to this medicine.20 This was also supported through the EMA’s PRIME scheme, which provides early and enhanced scientific and regulatory support to medicines that have a particular potential to address patients’ unmet medical needs.21
For a full list of adverse events and information on dosage and administration, contraindications and other precautions when using teclistamab please refer to the Summary of Product Characteristics. In line with EMA regulations for new medicines and those given conditional approval, teclistamab is subject to additional monitoring.
About Multiple Myeloma
Multiple myeloma is an incurable blood cancer that affects a type of white blood cell called plasma cells, which are found in the bone marrow.3,22 In multiple myeloma, these malignant plasma cells change and grow out of control.22 In Europe, more than 50,900 people were diagnosed with multiple myeloma in 2020, and more than 32,400 patients died.23 While some patients with multiple myeloma initially have no symptoms, others can have common symptoms of the disease which can include bone fracture or pain, low red blood cell counts, tiredness, high calcium levels or kidney failure.24
About the Janssen Pharmaceutical Companies of Johnson & Johnson
At Janssen, we’re creating a future where disease is a thing of the past. We’re the Pharmaceutical Companies of Johnson & Johnson, working tirelessly to make that future a reality for patients everywhere by fighting sickness with science, improving access with ingenuity, and healing hopelessness with heart. We focus on areas of medicine where we can make the biggest difference: Cardiovascular, Metabolism & Retina; Immunology; Infectious Diseases & Vaccines; Neuroscience; Oncology; and Pulmonary Hypertension.
Learn more at www.janssen.com/emea. Follow us at www.linkedin.com/janssenEMEA for our latest news. Janssen Pharmaceutica NV, Janssen-Cilag Limited and Janssen Research & Development, LLC are part of the Janssen Pharmaceutical Companies of Johnson & Johnson.
J&J gets expanded approval for cardiac ablation products
Aug. 04, 2023 11:42 AM ET
By: Val Brickates Kennedy, SA News Editor
Johnson & Johnson's (NYSE:JNJ) BioSense Webster unit said that it has received FDA approval for several of its cardiac ablation products for use in workflow without fluoroscopy.
J&J said the approval covers the Thermocool Smarttouch SF catheter, Carto Vizigo Bi-Directional Guiding Sheath, Pentaray Nav Eco High Density Mapping Catheter, Decanav Mapping Catheters and Webster CS Catheter. The devices are used for cardiac ablation procedures to treat atrial fibrillation, a common type of irregular heartbeat.
UGN-102
TALVEY®▼ (talquetamab) and TECVAYLI®▼ (teclistamab)
UGN-102
UGN-102, in Development as the Potential First Non-Surgical Therapy for LG-IR-NMIBC, Met Primary Endpoints in Both Phase 3 ATLAS and ENVISION Clinical Trials
July 27, 2023 at 9:50 AM EDT
-- In ATLAS, UGN-102 Demonstrated Superiority to TURBT with a 55% Reduction of Risk for Recurrence, Progression or Death in Patients who Received UGN-102 --
-- ENVISION Showed a Complete Response Rate of 79.2% at 3-Months --
-- Side Effect Profile in ATLAS and ENVISION Consistent with Previous Clinical Trials of UGN-102 --
-- UroGen Will Review ATLAS and ENVISION Top-line Data Today at 10:00 a.m. Eastern Time --
PRINCETON, N.J.--(BUSINESS WIRE)--Jul. 27, 2023-- UroGen Pharma Ltd. (Nasdaq: URGN), a biotech company dedicated to developing and commercializing novel solutions that treat urothelial and specialty cancers, today announced positive topline data from its Phase 3 trials ATLAS and ENVISION studying UGN-102 (mitomycin) for intravesical solution in patients with low-grade, intermediate-risk non-muscle invasive bladder cancer (LG-IR-NMIBC). Both ATLAS and ENVISION trials met their primary endpoints.
In the ATLAS trial, UGN-102 met its primary endpoint of disease-free survival, reducing risk of recurrence, progression, or death by 55%. UGN-102 also showed a 64.8% complete response rate at three months for patients who only received UGN-102, compared to a 63.6% complete response rate at three months for patients who only received a TURBT.
The ENVISION trial met its primary endpoint by demonstrating that patients treated with UGN-102 had a 79.2% rate of complete response at 3-months following the initial treatment. Additional data evaluating the secondary endpoint of duration of response from ENVISION, and the submission of a New Drug Application (NDA) (assuming additional positive findings) to the U.S. Food and Drug Administration (FDA) are anticipated in 2024.
“UGN-102 has demonstrated a robust and consistent therapeutic profile across multiple clinical trials, providing a compelling picture of its potential to be a transformational product and advance the standard of care away from repetitive surgery to a minimally invasive, non-surgical option for LG-IR-NMIBC,” says Liz Barrett, President and Chief Executive Officer of UroGen. “If approved, we anticipate UGN-102 to be a significant growth driver for UroGen as the first-ever non-surgical treatment option for a disease afflicting approximately 82,000 new patients in the U.S. each year. We are on track to deliver on our previously shared guidance for JELMYTO and now find ourselves on the precipice of a new era in bladder cancer care, and that is a very exciting place to be.”
In both trials, UGN-102, a sustained release, hydrogel-based formulation that is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means, was generally well tolerated, with a side effect profile similar to that of previous clinical trials.
“While TURBT is the standard treatment for bladder cancer, the recurrent nature of LG-IR-NMIBC means that patients will undergo multiple surgeries that come with risks for this older patient population,” says Sandip Prasad, MD, M.Phil., Director of Genitourinary Surgical Oncology, Morristown Medical Center/Atlantic Health System, NJ. “Based on these compelling data, I am optimistic that UGN-102, if approved, may change the treatment paradigm for these patients who lack non-surgical options to manage the ongoing burden of this highly recurrent disease.”
The Phase 3 ATLAS clinical trial, the predecessor to ENVISION, evaluated the efficacy, durability, and safety of UGN-102 with or without TURBT vs. TURBT alone in 282 patients with LG-IR-NMIBC. ENVISION evaluated the efficacy and safety of UGN-102 as a primary chemoablative therapy in 240 patients with LG-IR-NMIBC.
UGN-102 ATLAS & ENVISION Top-line Data Review
The company is hosting a data event featuring a panel discussion with leading bladder cancer experts on Thursday, July 27, 2023 at 10:00 a.m. Eastern Time to discuss these findings and will highlight topline results from the Phase 3 ATLAS and ENVISION clinical trials.
Please register for the webinar under the Events & Presentations section of the Company’s Investor Relations site (https://investors.urogen.com/events-and-presentations).
Following the live webcast, a replay will be available on the Company's website (https://urogen.com).
About UGN-102
UGN-102 (mitomycin) for intravesical solution is an investigational drug formulation of mitomycin in Phase 3 development for the treatment of LG-IR-NMIBC. Utilizing UroGen’s proprietary RTGel® technology, a sustained release, hydrogel-based formulation, UGN-102 is designed to enable longer exposure of bladder tissue to mitomycin, thereby enabling the treatment of tumors by non-surgical means. UGN-102 is delivered to patients using a standard urinary catheter in an outpatient setting. Assuming positive findings from the ENVISION Phase 3 study, UroGen anticipates submitting a New Drug Application (NDA) for UGN-102 in 2024. If approved, UGN-102 would be the first non-surgical primary therapeutic to treat a subset of bladder cancer characterized by high recurrence rates and multiple surgeries.
About ENVISION
The Phase 3 ENVISION trial is a single-arm, multinational, multicenter study evaluating the efficacy and safety of UGN-102 (mitomycin) for intravesical solution as primary chemoablative therapy in patients with low-grade, intermediate-risk NMIBC. The Phase 3 ENVISION trial completed target enrollment with approximately 240 patients across 56 sites. Study participants received six once-weekly intravesical instillations of UGN-102. The primary endpoint evaluated the complete response rate at the 3-month assessment after the first instillation, and the key secondary endpoint will evaluate durability over time in patients who achieved a complete response at the three-month assessment. Based on discussions with the FDA, and assuming positive findings, UroGen anticipates submitting an NDA for UGN-102 in 2024. Learn more about the Phase 3 ENVISION trial at www.clinicaltrials.gov (NCT05243550)
About ATLAS
ATLAS is a global, open-label, randomized controlled Phase 3 trial designed to assess the efficacy and safety of UGN-102, with or without TURBT, vs. TURBT alone in patients diagnosed with LG-IR-NMIBC. The trial enrolled 282 patients in clinical sites in the U.S., Europe and Israel. Patients were randomized 1:1 to either UGN-102 or TURBT. Patients in the UGN-102 arm were treated with six weekly intravesical instillations of UGN-102. At the 3-month time point, patients were assessed for response. Patients who demonstrated a complete response to either UGN-102 or TURBT, were assessed for long-term follow-up for evidence of recurrence. Patients who demonstrated presence of persistent disease at 3-months, in either arm, underwent a TURBT and continued for long-term follow-up for evidence of recurrence. The primary endpoint of the study is disease-free survival. Learn more about the ATLAS trial at www.clinicaltrials.gov (NCT04688931)
About UroGen Pharma Ltd.
UroGen is a biotech company dedicated to developing and commercializing innovative solutions that treat urothelial and specialty cancers because patients deserve better options. UroGen has developed RTGel® reverse-thermal hydrogel, a proprietary sustained release, hydrogel-based platform technology that has the potential to improve therapeutic profiles of existing drugs. UroGen’s sustained release technology is designed to enable longer exposure of the urinary tract tissue to medications, making local therapy a potentially more effective treatment option. JELMYTO® (mitomycin) for pyelocalyceal solution and investigational treatment UGN-102 (mitomycin) for intravesical solution for patients with low-grade non-muscle invasive bladder cancer are designed to ablate tumors by non-surgical means. UroGen is headquartered in Princeton, NJ with operations in Israel. Visit www.urogen.com to learn more or follow us on Twitter, @UroGenPharma.
View source version on businesswire.com: https://www.businesswire.com/news/home/20230727005666/en/
Source: UroGen Pharma Ltd.
UroGen says bladder cancer therapy met main goals in Phase 3 trials
Jul. 27, 2023 10:43 AM ET
By: Dulan Lokuwithana, SA News Editor2 Comments
UroGen Pharma (NASDAQ:URGN) announced Thursday that its lead product candidate, UGN-102, reached primary endpoints in two Phase 3 trials for patients with bladder cancer.
The ATLAS and ENVISION trials were designed to evaluate UGN-102 in over 500 patients with low-grade, intermediate-risk non-muscle invasive bladder cancer.
After a brief trading halt, UroGen (URGN) surged ~41% intraday following the announcement.
https://seekingalpha.com/news/3992173-urogen-stock-gains-bladder-cancer-trials-succeed
KB707
V940 (mRNA-4157) in Combination with KEYTRUDA® (pembrolizumab)
UGN-102
Krystal Biotech Announces Pipeline Expansion into Oncology and FDA Acceptance of IND Application for Lead Oncology Candidate KB707
July 26, 2023
PITTSBURGH, July 26, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, announced today that it has expanded its R&D pipeline to oncology and that the US Food and Drug Administration (FDA) has accepted its Investigational New Drug (IND) application of its lead oncology drug candidate KB707 for the treatment of locally advanced or metastatic solid tumor malignancies. The Company will host an investor conference call and webcast, Thursday, July 27, 2023, at 8:00 am ET, to discuss the KB707 program. To join the investor conference call, please see the instructions below. The presentation for the investor conference call is attached to the Company’s Form 8-K.
“The KB707 program leverages our learnings and clinical experience in two tissue areas, the skin and the lung, and underscores the broader potential of our HSV-1 platform to deliver all types of exogenous genetic material and improve outcomes for patients with debilitating diseases,” said Krish S. Krishnan, Chairman and CEO of Krystal Biotech.
KB707 is a modified HSV-1 vector designed to deliver genes encoding both human IL-12 and IL-2 to the tumor microenvironment and promote systemic immune-mediated tumor clearance. Two formulations of KB707 are in development, a solution formulation for transcutaneous injection and an inhaled (nebulized) formulation for lung delivery.
“We believe KB707 is a unique and highly differentiated drug candidate with the potential to unlock the capabilities of cytokine-based immunotherapy,” said Suma Krishnan, President of Research & Development at Krystal Biotech. “By enabling localized and sustained cytokine expression within a treated tumor, KB707 has the potential to maximize therapeutic efficacy while avoiding the tolerability challenges of systemic cytokine treatments.”
The FDA has accepted the Company’s IND to evaluate intratumoral KB707 in patients with solid tumors accessible by transcutaneous injection, and the Company expects to initiate a Phase 1 study in the second half of 2023. The Company is planning to file an amendment to the KB707 IND in the second half of 2023 to evaluate inhaled KB707 in a clinical trial in the first half of 2024.
Interleukin-2 (IL-2) and interleukin-12 (IL-12) are secreted cytokines with complementary functions promoting cell-mediated immunity in humans. Both IL-2 and IL-12 have been shown to elicit anti-tumor immune responses in preclinical or clinical models and have been extensively studied for their potential in cancer immunotherapy. Despite promising signs of efficacy, it has proven difficult to effectively harness IL-2 and IL-12 for therapeutic benefit, as systemic administration is often poorly tolerated, and their inherently short half-lives necessitate high dose levels and extremely frequent dose intervals. KB707 leverages the Company’s modified HSV-1 vector – and its ability to efficiently deliver a durable DNA payload without active replication and minimal cytotoxicity – to drive local and sustained cytokine expression within the tumor microenvironment and maximize the therapeutic window and benefit of IL-2 and IL-12.
“There remains an urgent unmet need for new therapies in cutaneous oncology, including for patients that do not respond to current first-line options and for the many who eventually progress on available therapy,” said Jason Luke, MD, Associate Professor of Medicine in the Division of Hematology/Oncology and Director of the Cancer Immunotherapeutic Center within UPMC Hillman Cancer Center Immunology and Immunotherapy Program in Pittsburgh, PA. “As the lead investigator on multiple practice changing immunotherapy trials, I have seen first-hand the benefits that can be realized through effective immune modulation and am excited about the potential of Krystal's approach for localized, sustained cytokine delivery.”
In preclinical studies, KB707 has been shown to efficiently transduce mammalian cells in vitro leading to the secretion of bioactive IL-2 and IL-12 and can drive localized, durable cytokine expression in mouse skin after intradermal injection. Furthermore, in stringent checkpoint inhibitor refractory ‘cold’ syngeneic mouse models, HSV-1 vector based delivery of murine equivalent IL2 and IL12 elicited robust antitumor responses and survival benefits, including via intratumoral injection in single and dual flank B16F10 melanoma models, as well as via intratracheal delivery in a metastatic K7M2 osteosarcoma model, with evidence of protection from tumor rechallenge in both models suggestive of prolonged adaptive immunity.
The intratumoral KB707 Phase 1/Opal 1 study is an open-label, multi-center, monotherapy, dose escalation and expansion study, enrolling patients with locally advanced or metastatic solid tumors, who relapsed or are refractory to standard of care, with at least one measurable and injectable tumor accessible by transcutaneous route. The primary objective of the study is to evaluate safety and tolerability of KB707. Efficacy will also be assessed by multiple measures including overall response rate, progression free survival, and overall survival, and the immune effects of KB707 monotherapy will be assessed in tumor tissue, lymph nodes, and blood.
For those unable to listen to the live conference call, a replay will be available on the Investor’s section of the Company’s website at www.krystalbio.com.
About Krystal Biotech, Inc.
Krystal Biotech, Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEKTM is the Company’s first commercial product, the first-ever redosable gene therapy, and the only medicine approved by the FDA for the treatment of dystrophic epidermolysis bullosa. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory, oncology, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and Twitter.
Source: Krystal Biotech, Inc.
Krystal Biotech IND application for solid tumor candidate accepted by FDA
Jul. 26, 2023 6:10 PM ET
By: Jonathan Block, SA News Editor
- The US FDA has accepted an Investigational New Drug application submitted by Krystal Biotech (NASDAQ:KRYS) for its solid tumor malignancies candidate KB707.
- The company said the asset is a "modified HSV-1 vector designed to deliver genes encoding both human IL-12 and IL-2 to the tumor microenvironment and promote systemic immune-mediated tumor clearance."
- Krystal Biotech, Inc. (KRYS)
- https://seekingalpha.com/news/3991754-krystal-biotech-ind-application-solid-tumor-candidate-accepted-fda
- https://www.krystalbio.com/
V940 (mRNA-4157) in Combination with KEYTRUDA® (pembrolizumab)
V940 (mRNA-4157) in Combination with KEYTRUDA® (pembrolizumab)
V940 (mRNA-4157) in Combination with KEYTRUDA® (pembrolizumab)
Merck and Moderna Initiate Phase 3 Study Evaluating V940 (mRNA-4157) in Combination with KEYTRUDA® (pembrolizumab) for Adjuvant Treatment of Patients with Resected High-Risk(Stage IIB-IV) Melanoma
July 26, 2023 6:45 am ET
V940-001 is the first Phase 3 study of a planned comprehensive clinical development program being initiated following the positive primary analysis of the Phase 2b KEYNOTE-942/mRNA-4157-P201 trial reported at AACR and ASCO earlier this year
RAHWAY, N.J. & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Merck (NYSE: MRK), known as MSD outside of the United States and Canada, and Moderna, Inc. (Nasdaq: MRNA), a biotechnology company pioneering messenger RNA (mRNA) therapeutics and vaccines, today announced the initiation of the pivotal Phase 3 randomized V940-001 clinical trial evaluating V940 (mRNA-4157), an investigational individualized neoantigen therapy (INT), in combination with KEYTRUDA, Merck’s anti-PD-1 therapy, as an adjuvant treatment in patients with resected high-risk (Stage IIB-IV) melanoma. Global recruitment in V940-001 has begun, and the first patients are now enrolling in Australia.
“As we continue our efforts to advance novel treatment options for patients with high-risk Stage IIB-IV melanoma, the initiation of the V940-001 Phase 3 trial represents an important step forward in these efforts and our study of individualized neoantigen therapy,” said Dr. Marjorie Green, senior vice president and head of late-stage oncology, global clinical development, Merck Research Laboratories. “We look forward to continuing to collaborate with Moderna to evaluate this promising new approach with V940 (mRNA-4157), while also building on a standard of care laid by KEYTRUDA.”
“The initiation of the V940-001 Phase 3 trial is an exciting and important milestone for us as we work with our colleagues at Merck and the melanoma patient community to investigate how individualized neoantigen therapy may potentially transform the treatment of the most serious form of skin cancer,” said Kyle Holen, M.D., Moderna's Senior Vice President and Head of Development, Therapeutics and Oncology. “We thank the patients, investigators, and clinical trial sites across the world for helping us advance our efforts in this area.”
V940-001 is a Phase 3 global, randomized, double-blind, placebo- and active-comparator-controlled study designed to evaluate the safety and efficacy of V940 (mRNA-4157) in combination with KEYTRUDA in people with resected high-risk (Stage IIB-IV) melanoma compared to KEYTRUDA alone. The trial is slated to enroll approximately 1,089 patients at more than 165 sites in over 25 countries around the world. The primary endpoint of the study is recurrence-free survival (RFS), and secondary endpoints include distant metastasis-free survival (DMFS), overall survival (OS), and safety.
Based on data from the Phase 2b KEYNOTE-942/mRNA-4157-P201 study, the U.S. Food and Drug Administration and European Medicines Agency granted Breakthrough Therapy Designation and the Priority Medicines (PRIME) scheme, respectively, for V940 (mRNA-4157) in combination with KEYTRUDA for the adjuvant treatment of patients with high-risk melanoma. The companies presented the study’s primary endpoint, RFS, in April 2023 at the American Association for Cancer Research (AACR) Annual Meeting and presented the study’s key secondary endpoint, DMFS, in June 2023 at the American Society of Clinical Oncology (ASCO) Annual Meeting. The companies also plan to expand the development program to additional tumor types, including non-small cell lung cancer.
About V940 (mRNA-4157)
V940 (mRNA-4157) is a novel investigational messenger ribonucleic acid (mRNA)-based individualized neoantigen therapy (INT) consisting of a single synthetic mRNA coding for up to 34 neoantigens that is designed and produced based on the unique mutational signature of the DNA sequence of the patient’s tumor. Upon administration into the body, the algorithmically derived and RNA-encoded neoantigen sequences are endogenously translated and undergo natural cellular antigen processing and presentation, a key step in adaptive immunity.
Individualized neoantigen therapies are designed to train and activate the immune system so that a patient can generate an antitumor response specific to their tumor mutation signature. V940 (mRNA-4157) is designed to stimulate an immune response by generating specific T-cell responses based on the unique mutational signature of a patient’s tumor. KEYTRUDA is an immunotherapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. Based on early clinical studies and data from the Phase 2b KEYNOTE-942/mRNA-4157-P201 trial, combining V940 (mRNA-4157) with KEYTRUDA may provide an additive benefit over KEYTRUDA alone.
About V940-001 ( NCT05933577 )
V940-001 is a global, randomized, double-blind, placebo- and active-comparator-controlled Phase 3 trial evaluating 1,089 patients with resected high-risk (Stage IIB-IV) melanoma. Following complete surgical resection, participants 18 years and older will be randomized 2:1 to receive V940 (mRNA-4157) (1 mg every three weeks for up to nine doses) and KEYTRUDA (400 mg every six weeks up to nine cycles [approximately one year]) versus KEYTRUDA alone for approximately one year until disease recurrence or unacceptable toxicity, or for a total treatment duration of up to approximately 56 weeks, whichever is sooner. The primary endpoint is RFS, defined as the time from randomization to any type of disease recurrence as assessed by the investigator, or death due to any cause. The secondary endpoints are DMFS, OS, safety and quality of life.
Key eligibility criteria for the trial include: patients who have surgically resected and histologically/pathologically confirmed diagnosis of Stage IIB or IIC, III or IV cutaneous melanoma, patients who have not received any prior systemic therapy for their melanoma beyond surgical resection and no more than 13 weeks have passed between final surgical resection.
For further information, please see: https://clinicaltrials.gov/study/NCT05933577
About melanoma
Melanoma, the most serious form of skin cancer, is characterized by the uncontrolled growth of pigment-producing cells. The rates of melanoma have been rising over the past few decades, with nearly 325,000 new cases diagnosed worldwide in 2020. In the U.S., skin cancer is one of the most common types of cancer diagnosed, and melanoma accounts for a large majority of skin cancer deaths. It is estimated there will be nearly 100,000 new cases of melanoma diagnosed and almost 8,000 deaths resulting from the disease in the U.S. in 2023.
About KEYTRUDA® (pembrolizumab) injection, 100 mg
KEYTRUDA is an anti-programmed death receptor-1 (PD-1) therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD- L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.
Merck has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,600 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient's likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.
Selected KEYTRUDA® (pembrolizumab) Indications in the U.S.
Melanoma
KEYTRUDA is indicated for the treatment of patients with unresectable or metastatic melanoma.
KEYTRUDA is indicated for the adjuvant treatment of adult and pediatric (12 years and older) patients with stage IIB, IIC, or III melanoma following complete resection.
Moderna, Merck begin phase 3 trial on mRNA melanoma combo treatment
Jul. 26, 2023 9:26 AM ET
By: Jonathan Block, SA News Editor5 Comments
- Moderna (NASDAQ:MRNA) and Merck (MRK) have begun a phase 3 trial testing a combination of the former's neoantigen therapy V940 (mRNA-4157) and the latter's Keytruda (pembrolizumab) for resected high-risk melanoma.
- https://seekingalpha.com/news/3991182-moderna-merck-begin-phase-3-trial-mrna-melanoma-combo-treatment
- https://www.merck.com/
- https://www.modernatx.com/
- Moderna, Inc. (MRNA)MRK
Axatilimab (SNDX-6352)
V940 (mRNA-4157) in Combination with KEYTRUDA® (pembrolizumab)
V940 (mRNA-4157) in Combination with KEYTRUDA® (pembrolizumab)
Syndax And Incyte Announce Positive Topline Results From The Pivotal AGAVE-201 Trial Of Axatilimab In Chronic Graft-Versus-Host Disease
July 24, 2023 at 7:00 AM EDT
– Trial met its primary endpoint across all cohorts with an overall response rate (ORR) of 74% at a dose of 0.3 mg/kg administered every two weeks –
– Data highlight durable response to treatment. At the 0.3 mg/kg dose, 60% of patients who responded to axatilimab were still responding at one year –
– Results continue to support axatilimab's promising safety and efficacy profile, and reinforce its potential as a first-in-class CSF-1R monoclonal antibody in chronic graft-versus-host disease (GVHD) –
– Syndax and Incyte intend to file a Biologics License Application (BLA) by year-end 2023; full dataset to be presented at a future medical meeting –
– Syndax to host conference call today at 8:30 a.m. ET –
WALTHAM, Mass. and WILMINGTON, Del., July 24, 2023 /PRNewswire/ -- Syndax Pharmaceuticals (Nasdaq: SNDX) and Incyte (Nasdaq: INCY) today announced positive topline data from the pivotal AGAVE-201 trial of axatilimab, an anti-CSF-1R antibody, in adult and pediatric patients with chronic graft-versus-host disease (GVHD) following two or more prior lines of therapy.
The trial achieved its primary endpoint across all cohorts, with patients treated with axatilimab at doses of 0.3 mg/kg every two weeks, 1.0 mg/kg every two weeks and 3.0 mg/kg every four weeks demonstrating overall response rates (ORR) within the first six months of treatment of 74%, 67% and 50%, respectively (95% Confidence Interval [CI]: [63,83], [55,77] and [39,61], respectively). Responses were achieved across key patient subgroups, including those with prior exposure to ruxolitinib, belumosudil and/or ibrutinib.
Based on these results, and pending agreement from the U.S. Food and Drug Administration (FDA), Syndax and Incyte intend to submit a Biologics License Application (BLA) to the FDA by year-end 2023.
"Today marks an important day not only for Syndax and Incyte, but, more importantly, for patients suffering from chronic GVHD," said Michael A. Metzger, Chief Executive Officer of Syndax. "Axatilimab is the first investigational chronic GVHD treatment to target inflammation and fibrosis through the inhibition of disease associated macrophages, and the AGAVE-201 data demonstrates the potentially pronounced impact this mechanism, alone or in combination with standard of care therapies already available for the management of this disease, may have on patients suffering from chronic GVHD. These results underscore our belief that axatilimab could provide a valuable and highly differentiated therapeutic option for this devastating disease. We look forward to working with our partners at Incyte as we move axatilimab towards regulatory filing. On behalf of the entire Syndax team, I would like to thank the patients, their caregivers and the investigators who made this trial possible."
"The results from the AGAVE-201 study are extremely compelling and underscore the potential benefits axatilimab may offer appropriate patients facing the serious complications associated with chronic GVHD," said Steven Stein, M.D., Chief Medical Officer of Incyte. "At Incyte, we remain committed to advancing our research and understanding of this complex disease and will work closely with Syndax and regulatory authorities to bring this innovative medicine to chronic GVHD patients."
The AGAVE-201 pivotal study enrolled a total of 241 patients across 121 sites in 16 countries. Patients enrolled in the trial had received a median of four prior systemic therapies with 74% having previously received ruxolitinib, 23% having previously received belumosudil and 31% having previously received ibrutinib. 54% of these patients had at least four organs involved at baseline including 45% with lung involvement.
Among responders treated with 0.3 mg/kg of axatilimab, 60% of patients maintained a response at 12 months (measured from first response to new systemic therapy or death, based on the Kaplan Meier estimate). The median duration of response in this population has not been reached. Additionally, in the 0.3 mg/kg group, 55% of patients experienced a clinically meaningful improvement in symptoms, as measured by at least a seven-point decrease in the modified Lee chronic GVHD Symptom Scale score.
The most common adverse events were consistent with the on-target effects of CSF-1R inhibition and with what was previously observed with axatilimab treatment. In the overall population, adverse events in greater than 20% of patients include an increase in aspartate aminotransferase, blood creatine phosphokinase, lipase, blood lactate dehydrogenase, alanine aminotransferase and fatigue (n=239). Serious adverse events in the overall population occurred in 101 (42.3%) patients, with 37 (15.5%) patients experiencing adverse events leading to discontinuation of study treatment. In the 0.3 mg/kg dose group (n=79), fatigue was the only serious adverse event that occurred in greater than 20% of patients. Serious adverse events in the 0.3 mg/kg group occurred in 30 (38%) patients, with five (6.3%) patients experiencing adverse events leading to discontinuation of study treatment.
"Chronic GVHD is a very common complication post allogeneic hematopoietic stem cell transplant that can have profound effects on patient medical burden and quality of life. More effective treatment options for this significant complication are desperately needed," said Carrie Kitko, M.D., Medical Director of the Pediatric Stem Cell Transplant Program at the Vanderbilt-Ingram Cancer Center. "I am highly encouraged by these data which demonstrate robust responses in a heavily pre-treated patient population. These findings further support that axatilimab has the potential to provide a clinically meaningful response for patients suffering from this morbid condition."
Conference Call and Webcast
Syndax will host a conference call and webcast to discuss the results of the Phase 2 AGAVE-201 trial today, July 24, 2023 at 8:30 a.m. ET.
The live webcast may be accessed through the Events & Presentations page in the Investors section of the Company's website. Alternatively, the conference call may be accessed through the following:
Conference ID: SNDXSPEC
Domestic Dial-in Number: 800-579-2543
International Dial-in Number: 785-424-1789
Live webcast: https://www.veracast.com/webcasts/syndax/events/specconf.cfm
For those unable to participate in the conference call or webcast, a replay will be available on the Investors section of the Company's website at www.syndax.com approximately 24 hours after the conference call and will be available for 90 days following the call.
About Chronic Graft-Versus-Host Disease
Chronic graft-versus-host disease (GVHD), an immune response of the donor-derived hematopoietic cells against recipient tissues, is a serious, potentially life-threatening complication of allogeneic hematopoietic stem cell transplantation which can last for years. Chronic GVHD is estimated to develop in approximately 40% of transplant recipients, and affects approximately 14,000 patients in the U.S.1,2. Chronic GVHD typically manifests across multiple organ systems, with skin and mucosa being commonly involved, and is characterized by the development of fibrotic tissue3.
About Axatilimab
Axatilimab is an investigational monoclonal antibody that targets colony stimulating factor-1 receptor, or CSF-1R, a cell surface protein thought to control the survival and function of monocytes and macrophages. In pre-clinical models, inhibition of signaling through the CSF-1 receptor has been shown to reduce the number of disease-mediating macrophages along with their monocyte precursors, which has been shown to play a key role in the fibrotic disease process underlying diseases such as chronic GVHD and IPF. Phase 1/2 data of Axatilimab in chronic GVHD demonstrating its broad activity and tolerability was last presented at the 63rd American Society of Hematology Annual Meeting and data was published in the Journal of Clinical Oncology. Axatilimab was granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with chronic GVHD and IPF. In September 2021, Syndax and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab. Axatilimab is being developed under an exclusive worldwide license from UCB entered into between Syndax and UCB in 2016.
About AGAVE-201
The global Phase 2 AGAVE-201 dose-ranging trial evaluated the efficacy, safety, and tolerability of axatilimab in 241 adult and pediatric patients with recurrent or refractory active chronic GVHD whose disease had progressed after two prior therapies. Patients were randomized to one of three treatment groups that investigated a distinct dose of axatilimab administered at 0.3 mg/kg every two weeks, 1 mg/kg every two weeks or 3 mg/kg every four weeks. The trial's primary endpoint is the proportion of patients in each dose group who achieved an objective response as defined by 2014 NIH Consensus Criteria for chronic GVHD by cycle 7 day 1. Secondary endpoints include duration of response, percent reduction in daily steroids dose, organ specific response rates and validated quality-of-life assessments using the Modified Lee Symptom Scale.
For more information about AGAVE-201, visit https://clinicaltrials.gov/ct2/show/NCT04710576.
About Syndax Pharmaceuticals, Inc.
Syndax Pharmaceuticals is a clinical stage biopharmaceutical company developing an innovative pipeline of cancer therapies. Highlights of the Company's pipeline include revumenib, a highly selective inhibitor of the Menin–KMT2A binding interaction, and axatilimab, a monoclonal antibody that blocks the colony stimulating factor 1 (CSF-1) receptor. For more information, please visit www.syndax.com or follow the Company on Twitter and LinkedIn.
About Incyte
Incyte is a Wilmington, Delaware-based, global biopharmaceutical company focused on finding solutions for serious unmet medical needs through the discovery, development and commercialization of proprietary therapeutics. For additional information on Incyte, please visit Incyte.com and follow @Incyte.
SOURCE Syndax Pharmaceuticals, Inc.
Syndax and Incyte gain on mid-stage win for immune disorder therapy
Jul. 24, 2023 8:28 AM ET
Syndax Pharmaceuticals, Inc. (SNDX)INCY, JNJ, SNY
By: Dulan Lokuwithana, SA News Editor
- Syndax Pharmaceuticals (NASDAQ:SNDX) and Incyte (INCY) traded higher pre-market Monday after announcing that a mid-stage trial for their candidate for the immune-mediated disorder chronic graft-versus-host disease (GVHD), axatilimab, achieved its primary endpoint.
- https://seekingalpha.com/news/3990001-syndax-stock-incyte-stock-gain-on-mid-stage-win-gvhd
- Syndax Pharmaceuticals, Inc. (SNDX)INCY,
- https://syndax.com/
- https://syndax.com/pipeline/axatilimab/
biotecHOPE™ 2023
Zilebesiran (ALN-AGT)
Zilebesiran (ALN-AGT)
Zilebesiran (ALN-AGT)
Alnylam Announces Publication of Phase 1 Study Results for Zilebesiran in the New England Journal of Medicine
Jul 19, 2023
— Dose-Dependent Reductions in Serum Angiotensinogen and 24-hour Ambulatory Blood Pressure Were Sustained for Six Months After Single Doses of Zilebesiran —
— Acceptable Safety Profile Supporting Further Development —
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jul. 19, 2023-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that results from its Phase 1 study of zilebesiran, an investigational RNAi therapeutic targeting liver-expressed angiotensinogen (AGT) in development for the treatment of hypertension, were published in the New England Journal of Medicine (NEJM). The full manuscript is titled, “Zilebesiran, an RNA Interference Therapeutic Agent for Hypertension,” and will appear in the July 20, 2023 issue of NEJM. The key data reported in the publication showed that in the Phase 1 study, compared to placebo, zilebesiran was associated with dose-dependent reductions in serum AGT, achieving tonic blood pressure control with consistent and durable blood pressure reduction throughout a 24-hour period, sustained up to six months after single doses of ≥200 mg of zilebesiran. Zilebesiran also demonstrated an acceptable safety profile supporting continued clinical development; the most frequent treatment-related adverse events were mild, transient injection-site reactions.
“Hypertension is the leading cause of premature death, cardiovascular disease, and chronic kidney disease worldwide, and the global prevalence is steadily increasing in parallel with population aging and secular trends in the prevalence of risk factors including obesity, physical inactivity, and unhealthy diet. Despite the availability of effective antihypertensive treatments, nearly half of patients with hypertension fail to achieve guideline-recommended blood pressure targets, leaving them at residual risk for myocardial infarction, stroke, kidney disease progression, and mortality. For clinicians, the challenge in optimizing treatment of hypertension is frequently compounded by poor adherence to prescribed medical therapy and substantial variability in blood pressure between office visits and over the 24-hour cycle,” said Akshay Desai, M.D., the lead author of the manuscript and Director of the Cardiomyopathy and Heart Failure Program in the Advanced Heart Disease Section of the Cardiovascular Division at Brigham and Women’s Hospital. “In this context, the data we have published in NEJM are exciting, suggesting the potential role for zilebesiran to treat hypertension in a novel way via a novel, subcutaneously administered gene silencing approach to hypertension. This novel approach may provide durable, tonic blood pressure control with infrequent, office-based dosing and a favorable safety profile. Additional clinical trials will provide further insights into the potential of this approach to improve clinical outcomes in the growing population of patients with hypertension.”
“The data published in NEJM suggest the potential for zilebesiran to be an effective and highly-differentiated treatment that may help people with hypertension achieve sustained blood pressure control,” said Simon Fox, Ph.D., Vice President, Zilebesiran Program Lead at Alnylam. “To that end, we are currently evaluating the safety and efficacy of zilebesiran in our KARDIA Phase 2 clinical program either as a monotherapy (KARDIA-1) or in combination with a standard-of-care antihypertensive medication (KARDIA-2), and we look forward to reporting results from these programs in mid- and late 2023, respectively.”
Summary of Published Results
The study was conducted in 107* patients (zilebesiran, N=80; placebo, N=32) with mild-to-moderate hypertension. In Part A, patients were randomized 2:1 to receive single ascending subcutaneous doses of zilebesiran (10, 25, 50, 100, 200, 400, or 800 mg) or placebo. Part B of the study assessed the effects of zilebesiran (800 mg) on blood pressure under low- and high-salt diet conditions and Part E assessed the effects of zilebesiran (800 mg) coadministration with irbesartan (an angiotensin II receptor blocker). The study primary endpoint was the frequency of adverse events (AEs). AEs were reported for 58 patients receiving zilebesiran (72 percent) and 28 receiving placebo (88 percent). The most frequent treatment-related adverse events were mild, transient injection-site reactions reported in five (6 percent) patients who received zilebesiran. No events of hypotension, hyperkalemia, or worsening renal function requiring intervention were observed. Secondary and exploratory endpoints included change from baseline in serum AGT, pharmacokinetics, and change from baseline in blood pressure. In Part A, versus placebo, zilebesiran was associated with dose-dependent reductions in serum AGT that were sustained for up to six months. Single doses of zilebesiran (≥200 mg) resulted in reductions in systolic (>10 mm Hg) and diastolic (>5 mm Hg) blood pressure by Week 8, which were consistent throughout the diurnal cycle and sustained to six months. At the 800 mg dose, zilebesiran treatment resulted in mean (± standard error) systolic and diastolic reductions of 22.5 ± 5.1 mm Hg and 10.8 ± 2.7 mm Hg at Month 6, respectively. In Parts B and E of the study, blood pressure changes following zilebesiran treatment could be attenuated through high dietary salt intake and were augmented by irbesartan coadministration.
*Five patients receiving placebo in Part A of the study re-enrolled into Part E of the study and thus transitioned from placebo to zilebesiran.
About Zilebesiran
Zilebesiran is an investigational, subcutaneously administered RNAi therapeutic targeting angiotensinogen (AGT) in Phase 2 development for the treatment of hypertension in high unmet need populations. AGT is the most upstream precursor in the Renin-Angiotensin-Aldosterone System (RAAS), a cascade which has a demonstrated role in blood pressure (BP) regulation and its inhibition has well-established anti-hypertensive effects. Zilebesiran inhibits the synthesis of AGT in the liver, potentially leading to durable reductions in AGT protein and ultimately, in the vasoconstrictor angiotensin (Ang) II. Zilebesiran utilizes Alnylam's Enhanced Stabilization Chemistry Plus (ESC+) GalNAc-conjugate technology, which enables subcutaneous dosing with increased selectivity and a wide therapeutic index. The safety and efficacy of zilebesiran have not been established or evaluated by the FDA, EMA or any other health authority.
About RNAi
RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, function upstream of today’s medicines by potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.
About Alnylam Pharmaceuticals
Alnylam Pharmaceuticals (Nasdaq: ALNY) has led the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare and prevalent diseases with unmet need. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach yielding transformative medicines. Since its founding in 2002, Alnylam has led the RNAi Revolution and continues to deliver on a bold vision to turn scientific possibility into reality. Alnylam’s commercial RNAi therapeutic products are ONPATTRO® (patisiran), AMVUTTRA® (vutrisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), and Leqvio® (inclisiran), which is being developed and commercialized by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of investigational medicines, including multiple product candidates that are in late-stage development. Alnylam is executing on its “Alnylam P5x25” strategy to deliver transformative medicines in both rare and common diseases benefiting patients around the world through sustainable innovation and exceptional financial performance, resulting in a leading biotech profile. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam, on LinkedIn, on Instagram, or on Facebook at @AlnylamPharma.
View source version on businesswire.com: https://www.businesswire.com/news/home/20230719155322/en/
Alnylam Pharmaceuticals, Inc.
Source: Alnylam Pharmaceuticals, Inc.
Alnylam touts phase 1 results of RNAi hypertension asset zilebesiran
Jul. 19, 2023 5:56 PM ET
Alnylam Pharmaceuticals, Inc. (ALNY)
By: Jonathan Block, SA News Editor1 Comment
- Alnylam Pharmaceuticals (NASDAQ:ALNY) said that phase 1 data on its RNAi candidate for hypertension, zilebesiran, show it led to dose-dependent reductions in serum liver-expressed anhttps://seekingalpha.com/news/3988994-alnylam-touts-phase-1-results-rnai-hypertension-asset-zilebesirangiotensinogen ("AGT").
- Alnylam Pharmaceuticals, Inc. (ALNY)
- https://www.alnylam.com/
- https://www.alnylam.com/sites/default/files/pdfs/ALN-AGT-Fact-Sheet.pdf
XPOVIO® (selinexor)
Zilebesiran (ALN-AGT)
Zilebesiran (ALN-AGT)
Karyopharm Receives FDA Fast Track Designation for Selinexor for the Treatment of Myelofibrosis
– Regulatory Designation Includes Primary Myelofibrosis, Post-Essential Thrombocythemia Myelofibrosis and Post-Polycythemia Vera Myelofibrosis –
– Pivotal Phase 3 Study of Selinexor and Ruxolitinib in Treatment-Naïve Myelofibrosis Initiated in June 2023 –
NEWTON, Mass., July 17, 2023 /PRNewswire/ -- Karyopharm Therapeutics Inc. (Nasdaq: KPTI), a commercial-stage pharmaceutical company pioneering novel cancer therapies, today announced that the United States Food and Drug Administration (FDA) has granted Fast Track Designation to the development program of selinexor for the treatment of patients with myelofibrosis, including primary myelofibrosis, post-essential thrombocythemia myelofibrosis, and post-polycythemia vera myelofibrosis.
"Fast Track Designation for selinexor highlights its potential to address the unmet medical need in myelofibrosis, an important acknowledgement as we continue our pivotal Phase 3 study," said Reshma Rangwala, MD, PhD, Chief Medical Officer of Karyopharm. "Selinexor's unique mechanism of action, XPO1 inhibition, is a novel and potentially fundamental mechanism in myelofibrosis. We have been highly encouraged by the efficacy and safety data observed to date [in our Phase 1 study] with selinexor in combination with ruxolitinib in patients with treatment-naïve myelofibrosis and believe selinexor has the potential to shift the treatment paradigm. We look forward to continued interaction with the FDA as we advance the development of this promising treatment for patients in need."
In June 2023, Karyopharm initiated a pivotal Phase 3 clinical trial (XPORT-MF-034) (NCT04562389) to assess the efficacy and safety of once-weekly selinexor 60 mg in combination with ruxolitinib in JAKi-naïve patients with myelofibrosis. Updated data from the Phase 1 study were presented at the American Association for Cancer Research Annual Meeting 2023, American Society of Clinical Oncology 2023 and European Hematology Association 2023, which showed rapid, deep and sustained spleen responses and robust symptom improvement in patients treated with selinexor 60 mg in combination with ruxolitinib as of the April 10, 2023 cut-off date. Top-line data from the Phase 3 study is expected in 2025. The Company plans to expand its clinical development program in myelofibrosis by investigating selinexor in other JAKi-naïve settings, such as novel combinations, to benefit the greatest number of patients.
Fast Track Designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously. Features of Fast Track Designation include frequent interactions with the FDA review team, and if relevant criteria are met, eligibility for Priority Review and Rolling Review.
Further information about the Phase 3 study can be found at www.clinicaltrials.gov.
About Karyopharm Therapeutics
Karyopharm Therapeutics Inc. (Nasdaq: KPTI) is a commercial-stage pharmaceutical company pioneering novel cancer therapies. Since its founding, Karyopharm has been an industry leader in oral Selective Inhibitor of Nuclear Export (SINE) compound technology, which was developed to address a fundamental mechanism of oncogenesis: nuclear export dysregulation. Karyopharm's lead SINE compound and first-in-class, oral exportin 1 (XPO1) inhibitor, XPOVIO® (selinexor), is approved in the U.S. and marketed by the Company in three oncology indications and has received regulatory approvals in various indications in a growing number of ex-U.S. territories and countries, including Europe and the United Kingdom (as NEXPOVIO®) and China. Karyopharm has a focused pipeline targeting multiple high unmet need cancer indications, including in multiple myeloma, endometrial cancer, myelodysplastic neoplasms and myelofibrosis. For more information about our people, science and pipeline, please visit www.karyopharm.com, and follow us on Twitter at @Karyopharm and LinkedIn.
XPOVIO® and NEXPOVIO® are registered trademarks of Karyopharm Therapeutics Inc. Any other trademarks referred to in this release are the property of their respective owners.
SOURCE Karyopharm Therapeutics Inc.
Karyopharm granted Fast Track designation for selinexor for myelofibrosis
Jul. 17, 2023 5:13 PM ET
Karyopharm Therapeutics Inc. (KPTI)INCY
By: Jonathan Block, SA News Editor1 Comment
- The US FDA has awarded Fast Track designation to Karyopharm Therapeutics (NASDAQ:KPTI) selinexor for myelofibrosis.
- https://seekingalpha.com/news/3988199-karyopharm-granted-fast-track-designation-selinexor-myelofibrosis
- Karyopharm Therapeutics Inc. (KPTI)
- https://www.xpovio.com/
- https://www.karyopharm.com/
Donanemab
Zilebesiran (ALN-AGT)
Beyfortus™ (nirsevimab-alip)
Results from Lilly's Landmark Phase 3 Trial of Donanemab Presented at Alzheimer's Association Conference and Published in JAMA
July 17, 2023
Donanemab significantly slowed cognitive and functional decline for amyloid-positive early symptomatic Alzheimer's disease patients, lowering their risk of disease progression; nearly half of participants at earlier stage of disease on donanemab had no clinical progression at 1 year
Additional subpopulation analyses presented live showed that those study participants at earliest stage of disease had even greater benefit, with 60% slowing of decline compared to placebo
Furthermore, treatment effect continued to increase relative to placebo over the course of the trial, even though many participants completed their course of therapy at 6 or 12 months, supporting limited duration dosing
FDA submission completed in Q2; regulatory action expected by end of year
INDIANAPOLIS, July 17, 2023 /PRNewswire/ -- Eli Lilly and Company (NYSE: LLY) presented full results from the Phase 3 TRAILBLAZER-ALZ 2 study showing that donanemab significantly slowed cognitive and functional decline in people with early symptomatic Alzheimer's disease (AD). The data were shared at the 2023 Alzheimer's Association International Conference (AAIC) as a featured symposium and simultaneously published in the Journal of the American Medical Association (JAMA).
"The positive TRAILBLAZER-ALZ 2 data bring hope to people with Alzheimer's disease who urgently need new treatment options. This is the first Phase 3 study of a disease-modifying therapy to replicate the positive clinical results observed in a previous study," said Anne White, executive vice president of Eli Lilly and Company and president of Lilly Neuroscience. "If approved, we believe donanemab can provide clinically meaningful benefits for people with this disease and the possibility of completing their course of treatment as early as 6 months once their amyloid plaque is cleared. We must continue to remove any barriers in access to amyloid-targeting therapies and diagnostics in an already complex healthcare ecosystem for Alzheimer's disease."
Lilly previously announced that donanemab met the primary and all cognitive and functional secondary endpoints in the Phase 3 study. Submission to the U.S. FDA for traditional approval was completed last quarter with regulatory action expected by the end of the year. Submissions to other global regulators are currently underway, and the majority will be completed by year end.
The TRAILBLAZER-ALZ 2 results support Lilly's application for regulatory approval to treat people with amyloid-positive early symptomatic Alzheimer's disease (either mild cognitive impairment or mild dementia), regardless of their baseline level of tau. TRAILBLAZER-ALZ 2 enrolled participants with a broader range of cognitive scores and amyloid levels than other recent trials of amyloid plaque-targeting therapies. Participants in TRAILBLAZER-ALZ 2 were stratified by their level of tau, a predictive biomarker for disease progression, into either a low-medium tau group (sometimes referred to as intermediate tau) or a high tau group, which represented a later pathological stage of disease progression. All participants were then assessed over 18 months using scales that measure both cognition and function, including the integrated Alzheimer's Disease Rating Scale (iADRS) and the Clinical Dementia Rating-Sum of Boxes (CDR-SB).
As previously reported, among participants with low-medium levels of tau (n=1182), donanemab treatment significantly slowed decline by 35% on iADRS and 36% on CDR-SB. Among all amyloid-positive early symptomatic AD study participants (n=1736), treatment with donanemab significantly slowed decline by 22% on iADRS and 29% on CDR-SB. Additional data presented at AAIC reinforced that regardless of baseline clinical or pathological stage of disease, treatment with donanemab resulted in cognitive and functional benefits relative to placebo:
- A pre-specified subpopulation analysis of low-medium tau participants based on clinical stage showed greater benefit of donanemab in those at earlier stage of disease:
- In participants with mild cognitive impairment (n=214), donanemab slowed decline by 60% on iADRS and 46% on CDR-SB (for those with mild dementia due to AD, n=534, donanemab slowed decline by 30% on iADRS and 38% on CDR-SB, respectively).
- Similarly, a post-hoc subgroup analysis of low-medium tau participants based on age showed greater benefit of donanemab in patients under the age of 75:
- In participants under the age of 75 (n=542), donanemab slowed decline by 48% on iADRS and 45% on CDR-SB.
- In participants aged 75 or greater (n=551), donanemab slowed decline by 25% on iADRS and 29% on CDR-SB.
- Results were similar across other subgroups, including participants who carried or did not carry an ApoE4 allele.
- The overall treatment effect of donanemab continued to grow throughout the trial, with the largest differences versus placebo seen at 18 months.
"These results demonstrate that diagnosing and treating people earlier in the course of Alzheimer's disease may lead to greater clinical benefit," said Liana Apostolova, M.D., distinguished professor in Alzheimer's Disease research and professor in neurology, radiology, medical and molecular genetics at Indiana University School of Medicine, where she is associate dean for Alzheimer's disease research and directs the clinical core of the Alzheimer's Disease Center. "The delay of disease progression over the course of the trial is significant and will give people more time to do such things that are meaningful to them."
Donanemab specifically targets deposited amyloid plaque and has been shown to lead to plaque clearance in treated patients. Treatment with donanemab significantly reduced amyloid plaque levels regardless of baseline pathological stage of disease. Among all participants, treatment with donanemab reduced amyloid plaque on average by 84% at 18 months, compared with a 1% decrease for participants on placebo. Participants were able to stop taking donanemab once they achieved pre-defined criteria of amyloid plaque clearance.* Approximately half of participants met this threshold at 12 months and approximately seven of every ten participants reached it at 18 months.
In the earlier pathological stage of disease in participants with low-medium tau, treatment with donanemab resulted in 47% of participants with no progression at one year on the CDR-SB assessment, versus 29% on placebo. Those participants treated with donanemab also had a 39% lower risk of progressing to the next clinical stage of disease over the 18-month trial. This delay in progression meant that, on average, participants treated with donanemab had an additional 7.5 months before they reached the same level of cognitive and functional decline on CDR-SB compared to those on placebo.
"People living with early, symptomatic Alzheimer's disease are still working, enjoying trips, sharing quality time with family – they want to feel like themselves, for longer," said Mark Mintun, M.D., group vice president Neuroscience Research & Development at Lilly, and president of Avid Radiopharmaceuticals. "The results of this study reinforce the importance of diagnosing and treating disease sooner than we do today."
The incidence of amyloid-related imaging abnormalities (ARIA) and infusion-related reactions was consistent with the previous TRAILBLAZER-ALZ study. ARIA occurs across the class of amyloid plaque clearing antibody therapies. It is most commonly observed as temporary swelling in an area or areas of the brain (ARIA-E) or as microhemorrhages or superficial siderosis (ARIA-H), in either case detected by MRI, and these may be serious and even fatal in some cases. This risk should be managed with careful observation, monitoring with MRIs, and appropriate actions if ARIA is detected. Serious infusion-related reactions and anaphylaxis were also observed.
For full TRAILBLAZER-ALZ 2 results, see the publication in JAMA.
A live webcast of the scientific session at AAIC featuring the donanemab presentations can be viewed on the investor section of Lilly's web site.
Lilly will host an investor call on Monday, July 17, at 1:30 p.m. EDT (10:30 a.m. PDT) to discuss the company's presentations at the Alzheimer's Association International Conference.
About TRAILBLAZER-ALZ 2 Study and the TRAILBLAZER-ALZ program
TRAILBLAZER-ALZ 2 (NCT04437511) is a Phase 3, double-blind, placebo-controlled study to evaluate the safety and efficacy of donanemab in participants ages 60-85 years with early symptomatic Alzheimer's disease (MCI or mild dementia due to Alzheimer's disease) with the presence of confirmed Alzheimer's disease neuropathology. The trial enrolled 1736 participants, across 8 countries, selected based on cognitive assessments in conjunction with amyloid plaque imaging and tau staging by PET imaging.
Lilly previously announced and published in the New England Journal of Medicine (NEJM) results from the Phase 2 TRAILBLAZER-ALZ study in 2021. In addition, Lilly shared data from TRAILBLAZER-ALZ 4, the first active comparator study in early symptomatic Alzheimer's disease, at the 15th Clinical Trials on Alzheimer's Disease (CTAD) conference in 2022.
Lilly continues to study donanemab in multiple clinical trials, including TRAILBLAZER-ALZ 3, which is focused on preventing symptomatic Alzheimer's disease in participants with preclinical AD; TRAILBLAZER-ALZ 5, a registration trial for early symptomatic Alzheimer's disease currently enrolling in China; and TRAILBLAZER-ALZ 6, which is focused on expanding our understanding of ARIA through novel MRI sequences, blood-based biomarkers, and different dosing regimens of donanemab.
About Lilly
Lilly unites caring with discovery to create medicines that make life better for people around the world. We've been pioneering life-changing discoveries for nearly 150 years, and today our medicines help more than 51 million people across the globe. Harnessing the power of biotechnology, chemistry and genetic medicine, our scientists are urgently advancing new discoveries to solve some of the world's most significant health challenges, redefining diabetes care, treating obesity and curtailing its most devastating long-term effects, advancing the fight against Alzheimer's disease, providing solutions to some of the most debilitating immune system disorders, and transforming the most difficult-to-treat cancers into manageable diseases. With each step toward a healthier world, we're motivated by one thing: making life better for millions more people. That includes delivering innovative clinical trials that reflect the diversity of our world and working to ensure our medicines are accessible and affordable. To learn more, visit Lilly.com and Lilly.com/newsroom or follow us on Facebook, Instagram, Twitter and LinkedIn. P-LLY
* Participants completed their course of treatment with donanemab once they reached a pre-defined level of plaque clearance. Average baseline amyloid levels were ~100 Centiloids for participants with early symptomatic Alzheimer's disease.
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SOURCE Eli Lilly and Company
Eli Lilly full phase 3 data confirms benefit of Alzheimer's asset donanemab
Jul. 17, 2023 1:21 PM ET
Eli Lilly and Company (LLY)BIIB, ESALF, ESAIY
By: Jonathan Block, SA News Editor
Complete phase 3 data released on Eli Lilly's (NYSE:LLY) donanemab confirmed that the Alzheimer's biologic was effective in slowing cognitive decline in early stages of the disease compared to placebo.
LLY Dividend History
https://www.nasdaq.com/market-activity/stocks/lly/dividend-history
Beyfortus™ (nirsevimab-alip)
Beyfortus™ (nirsevimab-alip)
Beyfortus™ (nirsevimab-alip)
Beyfortus approved in the US for the prevention of RSV lower respiratory tract disease in infants
PUBLISHED17 July 2023
First preventive option specifically designed to protect the broad infant population through its first RSV season
Across all clinical endpoints, a single dose of Beyfortus delivered consistent and sustained efficacy against RSV disease vs placebo
AstraZeneca and Sanofi’s Beyfortus (nirsevimab) has been approved in the US for the prevention of respiratory syncytial virus (RSV) lower respiratory tract disease (LRTD) in newborns and infants born during or entering their first RSV season, and for children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season. Beyfortus will be available in the US ahead of the upcoming 2023-2024 RSV season.
The approval by the Food and Drug Administration (FDA) follows the unanimous vote by the Antimicrobial Drugs Advisory Committee (AMDAC) on the favourable benefit-risk profile of Beyfortus, and was based on the extensive clinical development programme for Beyfortus spanning three pivotal late-stage clinical trials. Across all clinical endpoints, a single dose of Beyfortus demonstrated consistent efficacy against RSV LRTD extending through five months, the duration of a typical RSV season.1-4
Beyfortus is the first preventive option approved to protect a broad infant population, including those born healthy at term, or preterm, or with specific health conditions that make them vulnerable to severe RSV disease. The single dose can be flexibly administered at the beginning of the RSV season or at birth for those born during the RSV season.
Iskra Reic, Executive Vice President, Vaccines and Immune Therapies, AstraZeneca, said: “Beyfortus represents an opportunity for a paradigm-shift in preventing serious respiratory disease due to RSV across a broad infant population in the US. The science that Beyfortus is built on demonstrates AstraZeneca’s continued leadership in addressing the needs of the most vulnerable populations and reducing the burden on healthcare systems.”
Thomas Triomphe, Executive Vice President, Vaccines, Sanofi, said: “Today’s approval marks an unprecedented moment for protecting infant health in the U.S., following an RSV season that took a record toll on infants, their families, and the U.S. healthcare system. Beyfortus is the only monoclonal antibody approved for passive immunisation to provide safe and effective protection for all infants during their first RSV season. I am proud that, by prioritising this potential game-changer, we are now about to bring Beyfortus to American families.”
RSV is the leading cause of hospitalisation for infants under the age of one in the US, averaging 16 times higher than the annual rate for influenza.5,6 Each year, an estimated 590,000 RSV disease cases in infants under one require medical care, including physician office, urgent care, emergency room visits and hospitalisations.7
Beyfortus was generally well tolerated with a favourable safety profile that was consistent across all clinical trials. The overall rates of adverse events were comparable between Beyfortus and placebo and the majority of adverse events were mild or moderate in severity. The most common adverse events were rash and injection site reactions.1-4
Beyfortus was approved in the European Union in October 2022 for the prevention of RSV LRTD in newborns and infants during their first RSV season. Regulatory applications are also currently under review in China, Japan and several other countries.
Notes
RSV
RSV is a very contagious virus that can lead to serious respiratory illness for infants, according to the Centers for Disease Control and Prevention (CDC). RSV symptoms can include runny nose, coughing, sneezing, fever, decrease in appetite, and wheezing.8 Two out of three infants are infected with RSV during their first year of life and almost all infants are infected by their second birthday.8,9 In the US, RSV is the leading cause of hospitalisation in infants under 12 months, averaging 16 times higher than the annual rate for influenza.5,6 Approximately 75% of infants hospitalised for RSV were born at term with no underlying conditions in a study conducted from 2014-2015.10 Each year in the US, an estimated 590,000 RSV disease cases in infants under one require medical care, including physician office, urgent care, emergency room visits and hospitalisations.7
Beyfortus
Beyfortus (nirsevimab) is a single dose long-acting antibody, developed and commercialised in partnership by AstraZeneca and Sanofi using AstraZeneca’s YTE technology. It is designed to protect infants born during or entering their first RSV season and for children up to 24 months of age who remain vulnerable to severe RSV disease through their second RSV season. Beyfortus, provided directly to newborns and infants as a single dose, offers rapid protection via an antibody to help prevent LRTD caused by RSV, without requiring activation of the immune system. Beyfortus administration can be timed to the start of the RSV season.11
Beyfortus has been granted regulatory designations to facilitate expedited development by several major regulatory agencies around the world. These include Breakthrough Therapy Designation and Priority Review Designation by the China Center for Drug Evaluation under the National Medical Products Administration; Breakthrough Therapy Designation from the US Food and Drug Administration; access granted to the European Medicines Agency (EMA PRIority MEdicines (PRIME) scheme; and named “a medicine for prioritized development” under the Project for Drug Selection to Promote New Drug Development in Pediatrics by the Japan Agency for Medical Research and Development (AMED).
Pivotal clinical trials
The Phase IIb (Trial 03) study was a randomised, placebo-controlled trial designed to measure the efficacy of Beyfortus against medically attended (MA) Lower Respiratory Tract Infection (LRTI) through 150 days post-dose. Healthy preterm infants of 29 to less than 35 weeks’ gestational age were randomised (2:1) to receive a single 50mg intramuscular injection of Beyfortus or placebo regardless of weight.3,12
The Beyfortus dosing regimen was determined based on further exploration of the Phase IIb data and was used in subsequent trials as a single 50 mg dose for those who weigh less than 5 kg, or a single 100 mg dose for those who weigh 5 kg or greater.3,12
The MELODY Phase III study (Trial 04) was a randomised, double-blind, placebo- controlled trial conducted across 21 countries designed to determine efficacy of Beyfortus against medically attended LRTI due to through 150 days after dosing, versus placebo, in healthy term and late preterm infants (35 weeks gestational age or greater) entering their first RSV season.1,2,12
MEDLEY (Trial 05) was a Phase II/III, randomised, double-blind, Synagis-controlled trial with the primary objective of assessing safety and tolerability for Beyfortus in preterm infants of less than 35 weeks gestational age and infants with congenital heart disease (CHD) and/or chronic lung disease of prematurity (CLD) eligible to receive Synagis.4,12 Between July 2019 and May 2021 a total of 925 infants entering their first RSV season were randomised to receive Beyfortus or Synagis. Safety was assessed by monitoring the occurrence of adverse events through 360 days post-dose. Serum levels of Beyfortus following dosing (on day 151) in this trial were comparable with those observed in the MELODY Phase III trial, indicating similar protection in this population to that in the healthy term and late preterm infants is likely. Data were published in the New England Journal of Medicine (NEJM) in March 2022.4,12
The safety profile of Beyfortus was similar to Synagis in the MEDLEY Phase II/III trial and consistent with the safety profile in healthy term and preterm infants studied in the MELODY and Phase IIb trials. While uncommon, the most reported adverse reactions were: rash 14 days post-dose, (the majority of which were mild to moderate; non-serious injection site reactions within 7 days post-dose.1,2,4,12
The results of MELODY, MEDLEY Phase II/III and the Phase IIb trials demonstrate that a single dose of nirsevimab helps protect infants during their first RSV season against RSV disease. This broad infant population includes healthy term, late preterm and preterm infants, as well as infants with specific health conditions that make them vulnerable to severe RSV disease.1-4,12
These trials formed the basis of regulatory submissions which began in 2022.
Results from the MELODY Phase III trial (Trial 04)
The primary endpoint of the MELODY Phase III trial was met, reducing the incidence of medically attended LRTI, such as bronchiolitis or pneumonia, caused by RSV by 74.9% (95% CI 50.6, 87.3; P<0.001) compared to placebo.1,2 Observed events were 1.2% in treatment arm vs 5% in placebo arm. The efficacy of Beyfortus against the secondary endpoint of hospitalisation was 60.2% (95% CI: -14.6, 86.2). Observed events were 0.6% in treatment arm vs 1.6% in placebo arm. Between July 2019 and March 2020, 1,490 infants were randomised to receive either nirsevimab or placebo at the RSV season start Initial data from the MELODY Primary Cohort were published in NEJM in March 2022 12
Results from the Phase IIb trial (Trial 03)
The primary endpoint of the Phase IIb study was met, reducing the incidence of medically attended LRTI caused by RSV by 70.1% (95% CI: 52.3, 81.2) compared to placebo. Observed events were 2.6% in treatment arm vs 9.5% in placebo arm. Between November 2016 and December 2017, 1,453 infants were randomised (Beyfortus, n=969; placebo, n=484) at the RSV season start. Research was conducted by AstraZeneca in both hemispheres, at 164 sites in 23 countries. Data were published in NEJM in July 2020.3,12
In a prespecified secondary endpoint, Beyfortus reduced medically attended RSV LRTI with hospitalisation by 78.4% (95% CI 51.9, 90.3) versus placebo. Observed events were 0.8% in treatment arm vs 4.1% in placebo arm.3,12 A post-hoc analysis of the Phase IIb study that applied the recommended 50 mg dose in a subgroup of infants weighing less than 5 kg showed the efficacy of Beyfortus against medically attended RSV LRTI and medically attended RSV LRTI with hospitalisation was 86.2% (95% CI 68.0, 94.0) and
86.5% (95% CI 53.5, 96.1), respectively.12
Sanofi Alliance
In March 2017, AstraZeneca and Sanofi announced an agreement to develop and commercialise nirsevimab. Under the terms of the agreement, AstraZeneca leads development and manufacturing activities, and Sanofi leads commercialisation activities and records revenue. The two companies share costs and profits in all territories except the US. AstraZeneca's revenue from the agreement is reported as Alliance Revenue and Collaboration Revenue in the Company’s financial statements. Following a revision to the profit-sharing arrangement relating to the development and commercialisation of nirsevimab in the US between AstraZeneca, Sanofi and Sobi, Sobi has entered into a direct relationship with Sanofi, replacing the previous participation agreement with AstraZeneca entered into in November 2018.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development, and commercialisation of prescription medicines in Oncology, Rare Diseases, and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries and its innovative medicines are used by millions of patients worldwide. Please visit astrazeneca.com and follow the Company on Twitter @AstraZeneca.
AstraZeneca's nirsevimab approved as RSV treatment for babies, infants
Jul. 17, 2023 2:03 PM ET
By: Jonathan Block, SA News Editor
- The US FDA has approved AstraZeneca's Beyfortus (nirsevimab) to prevent respiratory syncytial virus in babies and infants up to two years old.
- The monoclonal antibody is to be given as a single dose before the RSV season begins in the fall.
- AstraZeneca PLC (AZN)
- https://www.astrazeneca.com/
VYVGART Hytrulo
Beyfortus™ (nirsevimab-alip)
VYVGART Hytrulo
argenx Reports Positive Topline Data from ADHERE Study of VYVGART Hytrulo in Patients with Chronic Inflammatory Demyelinating Polyneuropathy
NewsJuly 17, 2023
argenx Reports Positive Topline Data from ADHERE Study of VYVGART Hytrulo in Patients with Chronic Inflammatory Demyelinating Polyneuropathy
Amsterdam, The Netherlands
- Study met primary endpoint (p=0.000039); VYVGART® Hytrulo demonstrated 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in risk of relapse versus placebo
- IgG autoantibodies shown to play significant role in underlying CIDP disease biology
- Favorable safety and tolerability profile consistent with previous clinical trials and confirmed safety profile of VYVGART®
- Conference call scheduled for today, July 17, 2023 at 8:30am ET (2:30pm CET)
argenx SE (Euronext & Nasdaq: ARGX), a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases, today announced positive topline results from the ADHERE study evaluating VYVGART Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) in adults with chronic inflammatory demyelinating polyneuropathy (CIDP). The study met its primary endpoint (p=0.000039), demonstrating a significantly lower risk of relapse with VYVGART Hytrulo compared to placebo. Detailed data from ADHERE will be presented at an upcoming medical meeting.
ADHERE Highlights
- Primary endpoint met (p=0.000039); VYVGART Hytrulo demonstrated 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in the risk of relapse versus placebo
- 67% of patients in open-label Stage A demonstrated evidence of clinical improvement (ECI), indicating that IgG autoantibodies play a significant role in the underlying biology of CIDP
- Safety and tolerability profile consistent with confirmed safety profile of VYVGART
- 91% (226/249) of eligible patients continued to the ADHERE-Plus open-label extension study
"CIDP is a chronic, progressive autoimmune disease that can cause substantial disability in those affected, often leading to impaired ambulation or difficulty completing normal daily tasks without help. The positive ADHERE data show that VYVGART Hytrulo may represent a new patient-forward treatment option that can prevent symptom deterioration while minimizing side effects and treatment burden,” commented Jeffrey Allen, M.D., Associate Professor, Department of Neurology, University of Minnesota. “With ADHERE, argenx has set a new standard for innovative CIDP studies that more broadly inform the neuromuscular community. The findings from the trial indicate we may have a novel weapon to combat this debilitating condition in our ongoing efforts to improve the lives of individuals affected by CIDP.”
“People living with CIDP often experience significant challenges with daily function including fatigue, numbness, tingling, pain and weakness while facing a future with limited mobility or independence. The promising ADHERE data bring hope to the CIDP community of a brighter future where they could experience more positive moments doing the things that make them most happy,” said Lisa Butler, Executive Director of the GBS-CIDP Foundation International.
“With these positive ADHERE data, we have generated strong clinical evidence that CIDP has a significant IgG-driven pathogenesis component and that VYVGART Hytrulo can meaningfully improve and stabilize disease symptoms with a favorable safety profile and a simple route of administration,” commented Luc Truyen, M.D., Ph.D., Chief Medical Officer of argenx. “We are very grateful to the patients participating in the ADHERE trial and their supporters, the investigators, our collaborators and our argenx colleagues for the success of this innovative trial. Together, we are moving one step closer to transforming the treatment of autoimmunity.”
Detailed ADHERE Results
ADHERE is the largest clinical trial of CIDP patients to date, enrolling adults who were treatment naïve (not on active treatment within the past six months) or currently on immunoglobulin therapy or corticosteroids. The trial consisted of a run-in period where current treatment was stopped followed by an open-label Stage A, after which responders to VYVGART Hytrulo advanced to a randomized, placebo- controlled Stage B.
322 patients enrolled in Stage A and received treatment with VYVGART Hytrulo
- 67% (214/322) demonstrated evidence of clinical improvement (ECI) after a run-in withdrawal period based on the Inflammatory Neuropathy Cause and Treatment (INCAT) Disability Score, the Inflammatory Rasch-built Overall Disability Scale (I-RODS) or grip strength
- 70% (214/304) demonstrated ECI excluding patients ongoing in Stage A at the time of the 88th event who did not have the full opportunity to achieve a response
- 78% (214/275) demonstrated ECI in a sensitivity analysis of patients who received at least four injections to reach the full IgG-lowering effect of VYVGART Hytrulo
- Response rates similar across all prior CIDP medication subgroups with consistent efficacy on INCAT, I-RODS and grip strength.
221 responders from Stage A entered Stage B, where the primary endpoint was the relative risk of relapse based on time to relapse on the INCAT Disability Score
- VYVGART Hytrulo significantly reduced the risk of CIDP relapse compared to placebo
- Primary endpoint was met (p=0.000039); VYVGART Hytrulo demonstrated a 61% reduction (HR: 0.39 95% CI: 0.25; 0.61) in the risk of relapse compared to placebo based on time to the first adjusted INCAT deterioration of ≥1 point
- VYVGART Hytrulo patients had a lower relapse rate compared to placebo at Week 24 (26% versus 54%) and Week 48 (34% versus 60%)
- VYVGART Hytrulo patients experienced longer time to relapse compared to those on placebo with a rapid separation of the Kaplan–Meier curves beginning at Week 4 and sustained through Week 48
- VYVGART Hytrulo patients demonstrated a clinically meaningful mean improvement of 7.7 points on I-RODS and 12.3kPa on grip strength in Stage A. This clinically meaningful benefit was maintained in Stage B by treated patients and lost in placebo patients.
- Clinical benefit observed across all efficacy scales and patient subgroups, regardless of prior therapy
VYVGART Hytrulo was well-tolerated with a safety profile that is consistent with prior clinical trials and the known profile of VYVGART. The most frequent treatment-related adverse event was injection site reactions (ISRs), which occurred in a lower percentage of patients than previous VYVGART Hytrulo trials (20% in Stage A; 10% in Stage B). All ISRs were mild to moderate and resolved over time.
About Chronic Inflammatory Demyelinating Polyneuropathy
Chronic inflammatory demyelinating polyneuropathy (CIDP) is a rare and serious autoimmune disease of the peripheral nervous system. Although confirmation of disease pathophysiology is still emerging, there is increasing evidence that IgG antibodies play a key role in the damage to the peripheral nerves. People with CIDP experience fatigue, muscle weakness and a loss of feeling in their arms and legs that can get worse over time or may come and go. These symptoms can significantly impair a person's ability to function in their daily lives. Without treatment, one-third of people living with CIDP will need a wheelchair.
About VYVGART® Hytrulo
VYVGART Hytrulo is a subcutaneous combination of efgartigimod alfa, a human IgG1 antibody fragment marketed for intravenous use as VYVGART®, and recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE® drug delivery technology to facilitate subcutaneous injection delivery of biologics. In binding to the neonatal Fc receptor (FcRn), VYVGART Hytrulo results in the reduction of circulating IgG. It is the first-and-only approved FcRn blocker administered by subcutaneous injection.
VYVGART Hytrulo is the proprietary name in the U.S. for subcutaneous efgartigimod alfa and recombinant human hyaluronidase PH20. It may be marketed under different proprietary names following approval in other regions.
See Important Safety Information below and full Prescribing Information for VYVGART Hytrulo for additional information
Important Safety Information
What is VYVGART® HYTRULO (efgartigimod alfa and hyaluronidase-qvfc)?
VYVGART HYTRULO is a prescription medicine used to treat a condition called generalized myasthenia gravis, which causes muscles to tire and weaken easily throughout the body, in adults who are positive for antibodies directed toward a protein called acetylcholine receptor (anti-AChR antibody positive).
About argenx
argenx is a global immunology company committed to improving the lives of people suffering from severe autoimmune diseases. Partnering with leading academic researchers through its Immunology Innovation Program (IIP), argenx aims to translate immunology breakthroughs into a world-class portfolio of novel antibody-based medicines. argenx developed and is commercializing the first approved neonatal Fc receptor (FcRn) blocker in the U.S., Japan, Israel, the EU, the UK and China. The Company is evaluating efgartigimod in multiple serious autoimmune diseases and advancing several earlier stage experimental medicines within its therapeutic franchises. For more information, visit www.argenx.com and follow us on LinkedIn, Twitter, and Instagram.
Argenx/Zai Lab rise on phase 2 results for autoimmune disorder asset
Jul. 17, 2023 10:38 AM ET
By: Jonathan Block, SA News Editor
- Argenx (NASDAQ:ARGX) and Zai Lab (NASDAQ:ZLAB) said that the primary endpoint was met in a mid-stage study of Vyvgart Hytrulo (efgartigimod alfa and hyaluronidase-qvfc) in adults with chronic inflammatory demyelinating polyneuropathy (CIDP).
- Zai Lab has exclusive development and commercialization rights for Vyvgart Hytrulo in China.
- argenx SE (ARGX), ZLAB
- https://www.argenx.com/products
acoramidis
Beyfortus™ (nirsevimab-alip)
VYVGART Hytrulo
bridgebio announces consistently positive results from phase 3 attribute-cm study of acoramidis for patients with transthyretin amyloid cardiomyopathy (attr-cm)
07.17.2023 at 7:00 AM EDT
- Highly statistically significant result observed on primary endpoint with a Win Ratio of 1.8 (p<0.0001)
- 58% of ties in Finkelstein-Schoenfeld (F-S) primary analysis broken by all-cause mortality and frequency of cardiovascular-related hospitalization; statistical significance also achieved on an F-S test with those two parameters alone (p=0.0182)
- Clinically meaningful and consistent separation observed on all measures of mortality, morbidity, function, and quality of life
- On-treatment survival rate of 81% versus placebo survival rate of 74% (absolute risk reduction of 6.43%; relative risk reduction of 25%)
- Highly statistically significant relative risk reduction of 50% (p<0.0001) observed on frequency of cardiovascular-related hospitalization
- Highly statistically significant and clinically meaningful treatment benefit observed at 30 months on the secondary endpoints of NT-proBNP (p<0.0001), KCCQ (p<0.0001), and 6-minute walk distance (p<0.0001)
- In comparative exploratory post hoc analyses enabled by tafamidis drop-in, albeit at low patient numbers, acoramidis showed 42% greater increase in serum TTR levels and a 92% improvement in median NT-proBNP relative to placebo + tafamidis
- No safety signals of potential clinical concern identified
- Company intends to file a New Drug Application (NDA) with the U.S. Food and Drug Administration by end of 2023; late-breaker presentation has been accepted for annual meeting of the European Society of Cardiology
PALO ALTO, Calif., July 17, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), is a commercial-stage biopharmaceutical company focused on genetic diseases and cancers. Today, alongside the dedicated physicians and courageous patients who participated, the Company reports positive results from ATTRibute-CM, its Phase 3 study of acoramidis in transthyretin amyloid cardiomyopathy, or ATTR-CM. ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of transthyretin (TTR). BridgeBio will host an investor call on July 17, 2023 at 8:00 am ET to discuss these results.
“The outstanding results of the ATTRibute-CM study provide new hope to patients living with transthyretin amyloid cardiomyopathy, or ATTR-CM”, said Dr. Daniel Judge, Professor of Medicine and Cardiology at the Medical University of South Carolina, and Co-Chair of the ATTRibute-CM Steering Committee. “The consistent and clinically meaningful benefits on survival, hospitalization, and additional measures of illness severity are truly remarkable.”
“ATTR-CM is an increasingly recognized cause of heart failure. The results from BridgeBio’s ATTRibute-CM trial are very exciting and bring much hope to amyloidosis patients and their loved ones,” said Muriel Finkel, President of Amyloidosis Support Groups, a non-profit organization dedicated to the support of amyloidosis patients and caregivers.
Key results from the clinical trial include:
- A highly statistically significant improvement in the primary endpoint (a hierarchical analysis prioritizing in order: all-cause mortality, then frequency of cardiovascular-related hospitalization, then change from baseline in NT-proBNP, then change from baseline in 6-minute walk distance) demonstrated by a Win Ratio of 1.8 (p<0.0001).
- An 81% on-treatment survival rate (versus a 74% survival rate on placebo), which begins to approach actuarial models of life expectancy absent ATTR-CM (85% in this population as has been documented). The absolute risk reduction was 6.43% and the relative risk reduction was 25%.
- A highly statistically significant relative risk reduction of 50% (p<0.0001) on frequency of cardiovascular-related hospitalization. The impact and marked magnitude of risk reduction was seen across all analytical methods employed.
- The Company consistently observed a statistically significant treatment effect at 30 months across additional measured markers of morbidity, quality of life, and function:
- Change from baseline in N-terminal prohormone of brain natriuretic peptide (NT-proBNP) (p<0.0001)
- Change from baseline in Kansas City Cardiomyopathy Questionnaire (p<0.0001)
- Change from baseline in 6-minute walk distance (p<0.0001)
- No safety signals of potential clinical concern were identified.
A key objective in the rational drug design of acoramidis was to maximize TTR stabilization at clinically achieved blood concentrations. Several lines of evidence suggest that maximizing stabilization could lead to improved benefits for ATTR patients:
- Historical ATTR genotype/phenotype data and the disease-protective properties of trans-allelic, trans-suppressor variants relative to pathogenic variants in compound heterozygotes and the general, nonvariant population
- The outperformance of 80mg tafamidis vs 20mg tafamidis in the previously published ATTR-ACT trial
- Results from ATTR-polyneuropathy clinical trials
BridgeBio’s design strategy to maximize TTR stabilization by acoramidis was to phenocopy the hyperstabilizing molecular mechanism of the T119M trans-allelic, trans-suppressor rescue mutation. Prior preclinical and clinical studies have shown that acoramidis demonstrates approximately twice the stabilization of already-marketed stabilizers.
The allowance of tafamidis drop-in after at least 12 months in both the placebo and the acoramidis arms of the ATTRibute-CM trial provided the Company an opportunity to analyze, in an exploratory post hoc fashion and albeit at low patient numbers, differences in stabilizer performance as measured by serum TTR and NT-proBNP. The findings at 30 months were that:
- Acoramidis showed a 42% greater increase in serum TTR levels versus tafamidis
- Acoramidis showed a 92% improvement in median NT-proBNP relative to placebo + tafamidis
No comparison can be made as to the potential effectiveness or safety of acoramidis and tafamidis, given that these are exploratory analyses, and the study was not prospectively designed for a head-to-head comparison.
“Our heartfelt thanks go out to the patients, their caregivers, investigators, and study staff who have actively participated in ATTRibute-CM and continue to contribute to this pivotal research," stated Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of BridgeBio Cardiorenal. "We are extremely encouraged by the robustly positive and consistent findings of the ATTRibute-CM study, which confirm our position that highly potent TTR stabilization has the potential to profoundly impact patients’ lives. We look forward to presenting the data to health authorities to bring acoramidis to patients as expeditiously as possible."
The Company intends to submit its NDA to the US FDA before the end of 2023, with regulatory filings in additional markets to follow in 2024. This activity will occur in parallel with the prosecution of the remainder of the BridgeBio portfolio, which like acoramidis consists of medicines that target well-described diseases at their source. Acoramidis has intellectual property protection out to at least 2039.
Webcast Information
BridgeBio will host an investor call and simultaneous webcast to discuss the results from the Phase 3 ATTRibute-CM study of acoramidis in patients with ATTR-CM on July 17, 2023, at 8:00 am ET. A link to the webcast may be accessed from the event calendar page of BridgeBio’s website at https://investor.bridgebio.com/. A replay of the conference call and webcast will be archived on the Company’s website and will be available for at least 30 days following the event.
About BridgeBio Pharma, Inc.
BridgeBio Pharma, Inc. (BridgeBio) is a commercial-stage biopharmaceutical company founded to discover, create, test, and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers. BridgeBio’s pipeline of development programs ranges from early science to advanced clinical trials. BridgeBio was founded in 2015 and its team of experienced drug discoverers, developers and innovators are committed to applying advances in genetic medicine to help patients as quickly as possible. For more information visit bridgebio.com and follow us on LinkedIn and Twitter.
BridgeBio stock soars 80% on Phase 3 data, filing update for heart drug
Jul. 17, 2023 12:05 PM ET
By: Val Brickates Kennedy, SA News Editor1 Comment
Shares of BridgeBio (NASDAQ:BBIO) shot up 80% in midday trading Monday after the biotech company said it plans to file for US regulatory approval of its drug acoramidis by the end of the year after releasing positive Phase 3 data for the drug in the treatment of ATTR-CM.
BridgeBio shares opened at $26.76, recently changing hands at $32.81 at around 11:55 a.m. ET.
Earlier Monday, BridgeBio said the Phase 3 study showed an on-treatment survival rate of 81% for patients who took acoramidis versus 74% for those who took a placebo. The company also reported a "highly statistically significant" relative risk reduction of 50% for frequency of cardiovascular-related hospitalizations.
Domvanalimab
Zenocutuzumab (Zeno)
Domvanalimab
Gilead and Arcus Announce Anti-TIGIT Domvanalimab Continues to Demonstrate Consistent Improvement in Progression-Free Survival in Non-Small Cell Lung Cancer Study
June 03, 2023
DOWNLOAD PDFPDF FORMAT (OPENS IN NEW WINDOW)
– Clinically Meaningful Reduction in Risk of Progression or Death Was Observed in the Domvanalimab-Containing Study Arms Compared to Zimberelimab Monotherapy in First-Line, PD-L1-High NSCLC –
– Objective Response Rate (ORR) Improved in Both Domvanalimab-Containing Study Arms Compared to Zimberelimab Monotherapy –
– Results Will Be Presented Today During the American Society of Clinical Oncology (ASCO) Annual Meeting –
FOSTER CITY, Calif., & HAYWARD, Calif.--(BUSINESS WIRE)-- Gilead Sciences, Inc. (Nasdaq: GILD) and Arcus Biosciences, Inc. (NYSE: RCUS) today announced updated results from an interim analysis of the ARC-7 study in patients with first-line, metastatic non-small cell lung cancer (NSCLC) with PD-L1 tumor proportion score (TPS) ≥50% without epidermal growth factor receptor (EGFR) or anaplastic lymphoma kinase (ALK) mutations. ARC-7 is the first Phase 2, randomized, open-label study evaluating the combinations of Fc-silent anti-TIGIT monoclonal antibody domvanalimab plus anti-PD-1 monoclonal antibody zimberelimab (doublet) and domvanalimab plus zimberelimab and etrumadenant, an A2a/b adenosine receptor antagonist (triplet), versus zimberelimab monotherapy. These results will be presented today during the ASCO Plenary Series: Rapid Abstract Updates session by Melissa L. Johnson, M.D., Director, Lung Cancer Research, Sarah Cannon Research Institute, and Lead Investigator for the ARC-7 study. Arcus will post the updated ARC-7 results, which will include the data presented at Dr. Johnson’s session, as well as additional data, on its website on the corporate presentation page at 5:30pm PDT.
“Progression-free survival curves showed early separation of both domvanalimab-containing arms from the zimberelimab arm, which was consistently maintained, and supports the potential therapeutic benefit of inhibiting the TIGIT pathway,” said Melissa L. Johnson, M.D., Director, Lung Cancer Research, Sarah Cannon Research Institute, and Lead Investigator for the ARC-7 study. “I was also encouraged by the consistency of meaningful improvements across other outcome measures for the domvanalimab-containing arms. I look forward to continuing to work with Gilead and Arcus on the dom-zim combinations.”
At the time of data cutoff (DCO), February 7, 2023, safety and efficacy were evaluated in all patients randomized and treated (n=150). With a median follow-up time of approximately 18 months, both domvanalimab-containing study arms demonstrated sustained, clinically meaningful improvements in progression-free survival (PFS) compared to zimberelimab monotherapy, with a 33% reduction in risk of disease progression or death for the doublet and 28% for the triplet.
“At this analysis, the domvanalimab-containing study arms continued to show improved efficacy across multiple measures and both the doublet and triplet arms were generally well tolerated,” said Dimitry S.A. Nuyten, M.D., Ph.D., Chief Medical Officer of Arcus Biosciences. “These data reinforce our confidence in the domvanalimab program.”
“The ARC-7 proof-of-concept study has critically advanced our understanding of the activity of domvanalimab, the first Fc-silent anti-TIGIT monoclonal antibody in pivotal trials,” said Bill Grossman, M.D., Ph.D., Senior Vice President, Therapeutic Area Head, Gilead Oncology. “We look forward to quickly advancing our four ongoing Phase 3 registrational programs in NSCLC and upper GI cancers.”
No unexpected safety signals were observed across the three study arms at the time of DCO. The domvanalimab-containing study arms appeared to be generally well tolerated and showed an overall safety profile consistent with the known safety profiles of each individual molecule to date. Incidence of infusion-related reactions was low across all treatment arms: 4%, 4% and 12% for zimberelimab monotherapy and the domvanalimab-doublet and -triplet arms, respectively. The addition of domvanalimab to zimberelimab did not increase the incidence of infusion-related reactions, consistent with the Fc-silent design of domvanalimab.
Domvanalimab, zimberelimab and etrumadenant are investigational molecules. Neither Gilead nor Arcus has received approval from any regulatory authority for any use globally, and their safety and efficacy for the treatment of lung cancer have not been established.
About the ARC-7 Study
The ARC-7 study is the first Phase 2, multicenter, three-arm, randomized, open-label study evaluating the safety and efficacy of anti-TIGIT antibody domvanalimab plus anti-PD-1 antibody zimberelimab (doublet) versus domvanalimab plus zimberelimab and etrumadenant (triplet), an A2a/b adenosine receptor antagonist, versus zimberelimab monotherapy in 150 patients with first-line metastatic non-small cell lung cancer (NSCLC) with PD-L1 TPS ≥50% and no EGFR or ALK mutations. Patients are randomized 1:1:1 across the three study arms, and patients who progress on zimberelimab monotherapy may cross over to receive the triplet. At the time of this interim analysis, 150 patients had a median follow-up of 18.5 months. The co-primary endpoints are objective response rate and progression-free survival per Response Evaluation Criteria in Solid Tumors (RECIST 1.1). Secondary endpoints include duration of response, disease control rate, overall survival and safety. ARC-7 is a proof-of-concept study to assess the safety and efficacy of domvanalimab-containing study arms over zimberelimab monotherapy. More information about ARC-7 is available at: https://clinicaltrials.gov/ct2/show/NCT04262856.
About Domvanalimab
Domvanalimab is the first Fc-silent investigational monoclonal antibody in pivotal trials that is designed to block and bind to the T-cell immunoreceptor with Ig and ITIM domains (TIGIT), a protein receptor on immune cells that acts as a brake on the immune response. Cancer cells can exploit TIGIT to avoid detection by the immune system. By binding to TIGIT, domvanalimab is expected to free up immune activating pathways and activate immune cells to attack and kill cancer cells. Domvanalimab has demonstrated complete receptor coverage on all TIGIT-expressing peripheral leukocytes.
Domvanalimab is being evaluated in four registrational Phase 3 studies across lung and gastrointestinal cancers, including: (1) ARC-10, evaluating domvanalimab plus zimberelimab versus pembrolizumab in first-line locally advanced or metastatic PD-L1 ≥50% NSCLC; (2) PACIFIC-8, being operationalized by AstraZeneca, evaluating domvanalimab plus durvalumab in unresectable Stage 3 NSCLC; (3) STAR-121, evaluating domvanalimab plus zimberelimab and chemotherapy versus pembrolizumab plus chemotherapy in first-line PD-L1-unselected NSCLC; and (4) STAR-221, evaluating domvanalimab plus zimberelimab and chemotherapy versus nivolumab plus chemotherapy in first-line locally advanced, unresectable or metastatic gastric, esophageal and gastro-esophageal junction adenocarcinomas.
About Arcus Biosciences
Arcus Biosciences is a clinical-stage, global biopharmaceutical company developing differentiated molecules and combination medicines for people with cancer. In partnership with industry partners, patients and physicians around the world, Arcus is expediting the development of first- or best-in-class medicines against well-characterized biological targets and pathways and studying novel, biology-driven combinations that have the potential to help people with cancer live longer. Founded in 2015, the company has advanced multiple investigational medicines into clinical studies, including new combination approaches that target TIGIT, PD-1, the adenosine axis (CD73 and A2a/A2b receptors) and HIF-2a. For more information about Arcus Biosciences’ clinical and preclinical programs, please visit www.arcusbio.com.
About Gilead Sciences
Gilead Sciences, Inc. is a biopharmaceutical company that has pursued and achieved breakthroughs in medicine for more than three decades, with the goal of creating a healthier world for all people. The company is committed to advancing innovative medicines to prevent and treat life-threatening diseases, including HIV, viral hepatitis and cancer. Gilead operates in more than 35 countries worldwide, with headquarters in Foster City, California.
Source: Arcus Biosciences
Gilead raises stake in Arcus Biosciences
Jul. 06, 2023 1:15 PM ET
Arcus Biosciences, Inc. (RCUS), GILD
By: Dulan Lokuwithana, SA News Editor
Update 3:24PM EST: Adds comments from Arcus
According to a regulatory filing on Thursday, Gilead (NASDAQ:GILD) has increased its ownership in cancer-focused biotech Arcus Biosciences (NYSE:RCUS) with the purchase of ~1.0M additional RCUS shares for $19.26 apiece in late June.
The disclosure was made weeks after the duo expanded their oncology collaboration. The purchase consideration of $19.4M was spent from the available cash resources, Gilead (GILD) said in a regulatory filing.
https://seekingalpha.com/news/3986021-gilead-raises-stake-in-arcus-biosciences
PAXALISIB
Zenocutuzumab (Zeno)
Domvanalimab
KAZIA'S PAXALISIB RECEIVES FAST TRACK DESIGNATION FROM FDA FOR TREATMENT OF SOLID TUMOR BRAIN METASTASES HARBORING PI3K PATHWAY MUTATIONS IN COMBINATION WITH RADIATION THERAPY
NEWS PROVIDED BY
06 Jul, 2023, 07:00 ET
SYDNEY, July 6, 2023 /PRNewswire/ -- Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development company, is pleased to announce that its lead program, paxalisib, has been awarded Fast Track Designation (FTD) by the United States Food and Drug Administration (FDA) for the treatment of solid tumor brain metastases harboring PI3K pathway mutations in combination with radiation therapy.
The FDA's decision to grant FTD was based on promising clinical data from an interim analysis of an ongoing Phase 1 clinical trial in which patients with brain metastases from a primary tumour are receiving paxalisib in combination with radiotherapy (NCT04192981). These clinical data were presented at the 2022 Annual Conference on CNS Clinical Trials and Brain Metastases, jointly organized by the Society for Neuro-Oncology (SNO) and the American Society for Clinical Oncology (ASCO), by Dr. Jonathan Yang, lead investigator in the clinical trial. All nine evaluable patients in the trial (100%) responded to the combination of paxalisib with radiotherapy. Published benchmarks suggest a typical response rate for radiotherapy alone to be around 20-40%.
Key Points
- FTD is designed to expedite development of pharmaceutical products which demonstrate the potential to address unmet medical needs in serious or life-threatening conditions.
- FTD provides Kazia with enhanced access to FDA, including opportunities for face-to-face meetings and written consultation throughout the remaining development of paxalisib. Drugs granted FTD may also be eligible for Accelerated Approval and Priority Review, which may result in faster product approval.
- Paxalisib was previously granted FTD for glioblastoma in August 2020, giving paxalisib now two largely independent opportunities to access the benefits of this designation.
"Brain metastases are rapidly emerging as a key pillar of paxalisib's clinical development," said Dr. John Friend, Chief Executive Officer of Kazia. "We have seen a high level of interest from clinicians in the emerging data from this patient population, and it is exciting to now have that interest complemented by FDA's award of Fast Track Designation. With important data read-outs expected in adult and childhood brain cancer during CY2023, we will be working with investigators and advisors to drive forward our research in brain metastases also."
Brain Metastases
Brain metastases are a common complication of many tumours, but are particularly common in breast cancer, lung cancer, and melanoma. Brain metastases are typically highly resistant to treatment and survival rates are generally low. More than 250,000 patients are diagnosed with brain metastases each year in the United States alone.
Radiotherapy is a common treatment modality for brain metastases. Despite some efficacy, patients typically become resistant over time, and repeat courses of radiotherapy can be associated with significant neurological toxicity.
Expansion of Paxalisib Brain Metastases Study
The Phase I study (NCT04192981)is evaluating the safety and efficacy of paxalisib in combination with whole brain radiotherapy (WBRT) in patients with brain metastases and leptomeningeal metastases from any primary tumour (cancer that has spread to the brain from elsewhere in the body). The study was designed and initiated by Dr. Jonathan Yang, when he was at Memorial Sloan Kettering Cancer Center in New York, NY.
The study was designed in two stages. The first stage aimed to recruit 12 patients and was intended to establish the maximum tolerated dose (MTD) for paxalisib in conjunction with WBRT. Subject to positive results in the first stage, the study includes a second stage to assess initial efficacy signals and establish whether further development is warranted.
The results of the first stage of the study were reported at the SNO / ASCO 2022 Annual Conference on CNS Trials and Brain Metastases in August 2022. The combination was reported to be generally well-tolerated, with all nine evaluable patients showing evidence of clinical response.
Recruitment of the second stage commenced in 2H CY2022. The study is now open at two additional clinical sites: Miami Cancer Institute in Miami, FL, and the Fred Hutchinson Cancer Center in Seattle, WA, where Dr. Yang is now based.
Fast Track Designation
Introduced under the FDA Modernization Act (1997), FTD may be awarded by FDA to investigational drugs which are intended to treat a serious or life-threatening condition, and which fill an unmet medical need. FTD must be requested by the sponsor company and must be accompanied by a detailed review of both preclinical and clinical data. To be awarded FTD, drugs must generally be able to show some potential advantage over existing therapies, either in terms of safety or efficacy.
The key benefits of FTD comprise enhanced access to FDA, with regular and more frequent opportunities for consultation and discussion. In addition, drugs with FTD may be eligible for Accelerated Approval, in which a new medicine is approved based on a surrogate endpoint, and Priority Review, in which the standard 12-month review process may be reduced to eight months. Drugs with FTD may also receive a 'rolling review' of their NDA submission, in which sections are submitted for review as they become available, potentially expediting the approval process.
Next Steps
Further data from the ongoing Phase 1 study is expected in 1Q CY2024. Kazia continues to hold discussions with clinicians and advisors regarding the potential design of a registrational study in this indication.
Paxalisib is also the subject of nine other ongoing clinical trials, including the pivotal GBM AGILE study in glioblastoma, which is expected to provide final data in 2H CY2023, and a phase II study in pediatric patients with diffuse midline gliomas, which is expected to provide initial data in 3Q CY2023.
About Kazia Therapeutics Limited
Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA) is an oncology-focused drug development company, based in Sydney, Australia.
Our lead program is paxalisib, a brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway, which is being developed to treat multiple forms of brain cancer. Licensed from Genentech in late 2016, paxalisib is or has been the subject of ten clinical trials in this disease. A completed Phase II study in glioblastoma reported promising signals of clinical activity in 2021, and a pivotal study in glioblastoma, GBM AGILE, is ongoing, with final data expected in CY2023. Other clinical trials are ongoing in brain metastases, diffuse midline gliomas, and primary CNS lymphoma, with several of these having reported encouraging interim data.
Paxalisib was granted Orphan Drug Designation for glioblastoma by the FDA in February 2018, and Fast Track Designation for glioblastoma by the FDA in August 2020. In addition, paxalisib was granted Rare Pediatric Disease Designation and Orphan Drug Designation by the FDA for diffuse intrinsic pontine glioma in August 2020, and for atypical teratoid / rhabdoid tumours (AT/RT) in June 2022 and July 2022, respectively.
Kazia is also developing EVT801, a small-molecule inhibitor of VEGFR3, which was licensed from Evotec SE in April 2021. Preclinical data has shown EVT801 to be active against a broad range of tumour types and has provided compelling evidence of synergy with immuno-oncology agents. A Phase I study commenced recruitment in November 2021.
For more information, please visit www.kaziatherapeutics.com or follow us on Twitter @KaziaTx.
This document was authorized for release by John Friend, MD, Chief Executive Officer.
SOURCE Kazia Therapeutics Limited
Kazia jumps as FDA awards Fast Track tag for lead program
Jul. 06, 2023 7:29 AM ET
Kazia Therapeutics Limited (KZIA)
By: Dulan Lokuwithana, SA News Editor
Australian biotech Kazia Therapeutics (NASDAQ:KZIA) added ~29% pre-market Thursday after announcing that the FDA issued Fast Track Designation for its lead program, paxalisib, in combination with radiation therapy for certain patients with brain metastases.
https://seekingalpha.com/news/3985876-kazia-stock-jumps-fda-fast-track-tag-lead-program
Kazia Therapeutics Limited (KZIA)
https://www.kaziatherapeutics.com/site/for-researchers/paxalisib
Zenocutuzumab (Zeno)
Zenocutuzumab (Zeno)
FUJOVEE™ (Abivertinib)
Zenocutuzumab (Zeno) granted second Breakthrough Therapy Designation by the U.S. Food & Drug Administration
July 5, 2023 at 4:01 PM EDT
– Zeno granted BTD for the treatment of NRG1+ non-small cell lung cancer
UTRECHT, The Netherlands and CAMBRIDGE, Mass., July 05, 2023 (GLOBE NEWSWIRE) -- Merus N.V. (Nasdaq: MRUS), a clinical-stage oncology company developing innovative, full-length multispecific antibodies (Biclonics® and Triclonics®) for cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) for zenocutuzumab (Zeno) for the treatment of patients with advanced unresectable or metastatic NRG1 fusion (NRG1+) non-small cell lung cancer (NSCLC), following progression with prior systemic therapy. This designation for Zeno follows BTD for the treatment of patients with NRG1+ pancreatic cancer, following progression with prior systemic therapy or in patients who have no satisfactory alternative treatment options recently announced on June 30, 2023, Fast Track Designation for the treatment of patients with metastatic solid tumors harboring NRG1 gene fusions (NRG1+ cancer) that have progressed on standard of care therapy announced in January 2021 and Orphan Drug Designation for the treatment of patients with pancreatic cancer announced in July 2020.
BTD is supported by data from the ongoing phase 1/2 eNRGy trial and Early Access Program (EAP) which are assessing the safety and anti-tumor activity of Zeno monotherapy in NRG1+ cancer (Phase 1/2: NCT02912949, EAP: NCT04100694). Data from the eNRGy trial and EAP were featured as oral presentations during the 2021 and 2022 American Society of Clinical Oncology Annual Meetings (Abstract #3003, #105 respectively). As of June 1, 2023, more than 175 patients with NRG1+ cancer have been treated with Zeno monotherapy.
BTD is intended to expedite the development and review of a medicine to treat a serious or life-threatening condition, where preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on clinically significant endpoints over available therapies. BTD allows for more intensive FDA guidance on an efficient drug development program, an organizational commitment involving senior managers, and experienced review staff, as appropriate, in a collaborative, cross-disciplinary review, and eligibility for rolling review and priority review. With this BTD, Merus plans to engage in these discussions with the FDA in an expedited manner, and then provide a further update on the path and timeline to a potential Biologics License Application (BLA) submission.
Merus believes that obtaining a commercialization partnership agreement will be an essential step in bringing Zeno to patients with NRG1+ cancer, if approved.
“We are thrilled to receive the second BTD for Zeno, this time in NRG1+ lung cancer,” said Bill Lundberg, M.D., President, Chief Executive Officer of Merus. “We believe this further acknowledges Zeno’s clinically meaningful, durable response for patients with NRG1+ cancer and we aim to bring this potential first and best in class targeted agent for NRG1+ cancer to market with support from a potential commercial partner.”
Merus plans to provide a clinical update on Zeno in NRG1+ cancer at a major medical conference in 2023.
Further, Merus is evaluating Zeno in combination with androgen deprivation therapy (enzalutamide or abiraterone) in castration resistant prostate cancer (CRPC), irrespective of NRG1+ status. Merus plans to provide initial clinical data on Zeno in CRPC in the second half of 2023. Merus is also evaluating Zeno in combination with afatinib in patients with NRG1+ NSCLC.
About the eNRGy Clinical Trial
Merus is currently enrolling patients in the phase 1/2 eNRGy trial to assess the safety and anti-tumor activity of Zeno monotherapy in NRG1+ cancer. The eNRGy trial consists of three cohorts: NRG1+ pancreatic cancer; NRG1+ non-small cell lung cancer; and other NRG1+ cancer. Further details, including current trial sites, can be found at www.ClinicalTrials.gov and Merus’ trial website at www.nrg1.com or by calling 1-833-NRG-1234.
About Zeno
Zeno is an antibody-dependent cell-mediated cytotoxicity (ADCC)-enhanced Biclonics® that utilizes the Merus Dock & Block® mechanism to inhibit the neuregulin/HER3 tumor-signaling pathway in solid tumors with NRG1 fusions (NRG1+ cancer). Through its unique mechanism of binding to HER2 and potently blocking the interaction of HER3 with its ligand NRG1 or NRG1-fusion proteins, Zeno has the potential to be particularly effective against NRG1+ cancer. In preclinical studies, Zeno potently inhibits HER2/HER3 heterodimer formation thereby inhibiting oncogenic signaling pathways, leading to inhibition of tumor cell proliferation and blocking tumor cell survival.
About Merus N.V.
Merus is a clinical-stage oncology company developing innovative full-length human bispecific and trispecific antibody therapeutics, referred to as Multiclonics®. Multiclonics® are manufactured using industry standard processes and have been observed in preclinical and clinical studies to have several of the same features of conventional human monoclonal antibodies, such as long half-life and low immunogenicity. For additional information, please visit Merus’ website, Twitter and LinkedIn.
Merus' zenocutuzumab given second Breakthrough Therapy designation
Jul. 05, 2023 4:35 PM ET
By: Jonathan Block, SA News Editor1 Comment
- The US FDA has granted Breakthrough Therapy designation for the second time to Merus' (NASDAQ:MRUS) zenocutuzumab for NRG1 fusion (NRG1+) non-small cell lung cancer.
- The candidate is currently in a phase 1/2 examining the safety and anti-tumor activity of zeno monotherapy in NRG1+ cancer.
- https://seekingalpha.com/news/3985782-merus-zenocutuzumab-given-second-breakthrough-therapy-designation
- Merus N.V. (MRUS)
- https://merus.nl/
- https://merus.nl/pipeline/
FUJOVEE™ (Abivertinib)
Epcoritamab (DuoBody®-CD3xCD20)
FUJOVEE™ (Abivertinib)
Shanghai Escugen Biotechnology Co., Ltd., a partner of Levena Biopharma, a Sorrento Company, releases positive results from a first-in-human study of ESG401, a TROP2 Antibody Drug Conjugate in patients with locally advanced/metastatic solid tumors at the
June 5, 2023 at 9:00 AM EDT
CHICAGO, June 05, 2023 (GLOBE NEWSWIRE) -- Shanghai Escugen Biotechnology Co., Ltd. (“Escugen”), a partner of Levena (Suzhou) Biopharma Co., Ltd. (“Levena”), a wholly owned subsidiary of Sorrento Therapeutics, Inc. (Sorrento), today released preliminary results from a first-in-human study of ESG401, a trophoblast cell-surface antigen 2 (TROP2) antibody drug conjugate (ADC), in patients with locally advanced/metastatic solid tumors at the 2023 Annual Meeting of ASCO, the American Society of Clinical Oncology, held June 2-6 in Chicago, IL. ESG401 is an innovative ADC developed by Escugen and Levena. Escugen and Levena Biopharma jointly own the domestic and international patents of this ADC and share global rights for the product. ESG401 is composed of a humanized anti-Trop2 IgG1 monoclonal antibody (mAb) conjugated to a topoisomerase I inhibitor SN38 via a proprietary stable covalent linker with a drug antibody ratio (DAR) of 8. ESG401 has potential differentiated advantages over its competitors in terms of safety, effectiveness and process robustness. Using an innovative, highly stable and cleavable linker, this ADC demonstrated that it releases very little free toxin during circulation, which may reduce off target toxicity in a series of preclinical studies. Additionally, premature release of the mAb may compete for binding sites with the ADC to reduce its efficacy. The ADC highly enriches in tumor tissues and rapidly endocytoses, thereby effectively killing tumor cells and inhibiting tumor growth.
In the Phase I study, adult ESG401 patients with locally advanced/metastatic solid tumors refractory to or relapsed from standard treatments with measurable disease (RECIST v1.1) were eligible. ESG401 was administered by IV infusion initially in an ascending dose safety study by designated dose and regimen until unacceptable toxicity or progressive disease and followed by expansion cohorts. The Bayesian Optimal Interval (BOIN) design was used to establish the maximum tolerated dose (MTD). As of February 3, 2023, 35 heavily pretreated patients with a median age of 53 years were treated with at least one dose of ESG401 during dose escalation, 2 to 20 mg/kg administered every 3 weeks (Regimen A), or 12 to 16 mg/kg on day 1, 8, and 15 in a 4-week cycle (Regimen B). Eighty percent of the patients had an ECOG status of 1. Sixty-three percent of the patients had received at least 3 lines of prior therapy and overall the number of lines of prior therapy was a median of 4 (range 2-10).
About Escugen Biotechnology Co., Ltd.
Escugen Biotechnology Co., Ltd. is a clinical-stage biotechnology company committed to developing and commercializing innovative drugs for the treatment of cancer, autoimmune disease and other diseases with unmet medical needs. The company’s leading programs include ESG401, an anti-Trop2 antibody drug conjugate, currently in Phase Ib/II clinical trials in patients with locally advanced/metastatic solid tumors, and ESG206, an anti-BAFFR monoclonal antibody, currently in Phase I study in subjects with B-cell Lymphoid Malignancies.
About Sorrento Therapeutics, Inc.
Sorrento is a clinical and commercial stage biopharmaceutical company developing new therapies to treat cancer, pain (non-opioid treatments), autoimmune disease, and COVID-19. Sorrento's multimodal, multipronged approach to fighting cancer is made possible by its extensive immuno-oncology platforms, including key assets such as next-generation tyrosine kinase inhibitors ("TKIs"), fully human antibodies (“G-MAB™ library”), immuno-cellular therapies (“DAR-T™”), antibody-drug conjugates (“ADCs”), and oncolytic virus (“Seprehvec™”). Sorrento is also developing potential antiviral therapies and vaccines against coronaviruses, including STI-1558 and COVI-MSC™, and diagnostic test solutions, including COVIMARK™.
Sorrento's commitment to life-enhancing therapies for patients is also demonstrated by our effort to advance a TRPV1 agonist, non-opioid pain management small molecule, resiniferatoxin (“RTX”), and SP-102 (10 mg, dexamethasone sodium phosphate viscous gel) (SEMDEXA™), a novel, viscous gel formulation of a widely used corticosteroid for epidural injections to treat lumbosacral radicular pain, or sciatica, and to commercialize ZTlido® (lidocaine topical system) 1.8% for the treatment of postherpetic neuralgia (PHN). RTX has been cleared for a Phase II trial for intractable pain associated with cancer and a Phase II trial in osteoarthritis patients. Positive final results from the Phase III Pivotal Trial C.L.E.A.R. Program for SEMDEXA™, its novel, non-opioid product for the treatment of lumbosacral radicular pain (sciatica), were announced in March 2022. ZTlido® was approved by the FDA on February 28, 2018.
For more information visit www.sorrentotherapeutics.com
Sorrento sees promising phase 2 results for abivertinib for lymphoma
Jul. 05, 2023 12:02 PM ET
Sorrento Therapeutics, Inc. (SRNEQ)JNJ, ABBV
By: Jonathan Block, SA News Editor2 Comments
- New phase 2a data from China found that Sorrento Therapeutics' (OTCPK:SRNEQ) abivertinib for marginal zone lymphoma achieved an overall response rate (ORR) of ~59%.
- https://seekingalpha.com/news/3985616-sorrento-promising-phase-2-results-abivertinib-lymphoma
- Sorrento Therapeutics, Inc. (SRNEQ)
- https://sorrentotherapeutics.com/
Elafibranor (GFT505)
Epcoritamab (DuoBody®-CD3xCD20)
Epcoritamab (DuoBody®-CD3xCD20)
Ipsen and GENFIT Announce Positive Results from Phase III ELATIVE® trial of elafibranor in patients with primary biliary cholangitis, a rare cholestatic liver disease
June 30, 2023
- Trial met primary endpoint with a statistically significant higher percentage of patients achieving a clinically meaningful cholestasis response compared to placebo
- Elafibranor was well tolerated with a safety profile consistent with previous studies
- Results position elafibranor as a potentially important new treatment option, where there is still high unmet need
- Ipsen intends to submit regulatory applications for elafibranor following discussions with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA)
- GENFIT conference call (English and French) on June 30 at 8am ET / 1pm GMT / 2pm CET
Paris (France); June 30, 2023 - Ipsen (Euronext: IPN; ADR: IPSEY) and GENFIT (Nasdaq and Euronext: GNFT) today announced positive topline data from the pivotal ELATIVE® Phase III trial. In the trial the efficacy and safety of elafibranor, an investigational dual α,δ PPAR agonist, is being assessed for the treatment of patients with the rare cholestatic liver disease, primary biliary cholangitis (PBC), who have an inadequate response or intolerance to the current standard of care therapy, ursodeoxycholic acid (UDCA).
The trial met its primary composite endpoint, with 51% of patients on elafibranor 80mg achieving a cholestasis response compared with 4% on placebo (p<0.0001). Cholestasis response is defined in the trial as alkaline phosphatase (ALP) <1.67 x upper limit of normal (ULN), an ALP decrease ≥ 15 percent and total bilirubin (TB) ≤ ULN at 52 weeks. ALP and bilirubin are important predictors of disease progression. Reductions in levels of both can indicate reduced cholestatic injury and improved liver function.
The first secondary endpoint, normalization of ALP at Week 52, was also met with statistically significant improvements for investigational elafibranor compared with placebo. For the other secondary endpoint, a trend for pruritus improvement was observed with a greater decrease from baseline in the PBC Worst Itch NRS score for patients on elafibranor compared to placebo, which did not reach statistical significance. In the study, elafibranor was generally well tolerated with a safety profile consistent with that observed in previously reported studies.
“These are encouraging results that suggest elafibranor could be an effective treatment to prevent progression of PBC in patients who have received UDCA. It has a good safety profile and was well- tolerated, and could provide an important new therapeutic option for long-term treatment of patients with this debilitating condition,” said Howard Mayer, Executive Vice President and Head of Research and Development for Ipsen. “PBC is a serious condition which, if not treated properly, can lead to progression of liver disease and ultimately liver failure. We are excited about the potential of this investigational treatment and Ipsen now intends to discuss these results with regulatory agencies and plans to move forward with regulatory submissions to the U.S. Food and Drug Administration and the European Medicines Agency.”
“We are pleased by these results because PBC remains a disease where significant unmet medical needs exist”, added Pascal Prigent, Chief Executive Officer of GENFIT. “This long-awaited trial outcome is therefore good news for patients and for healthcare professionals who need more options to improve the clinical management of patients with PBC. It is also a gratifying recognition of the quality of our team’s work and of GENFIT’s ability to innovate and deliver tangible results.”
PBC is a rare, progressive, autoimmune cholestatic liver disease1 in which bile ducts in the liver are gradually destroyed. The damage to bile ducts can inhibit the liver’s ability to rid the body of toxins, and can lead to scarring of liver tissue, known as cirrhosis.1 Common symptoms of PBC include fatigue and pruritus (itch), which can be severely debilitating.1 Untreated, PBC can lead to liver failure, or in some cases death. It is also a leading cause of liver transplantation. It primarily affects middle-aged women, with nine women diagnosed for every man. It is a disease where a significant proportion of patients are unable to benefit from existing therapies. The prevalence of people living with PBC is estimated to be between 23.9-39.2 per 100,000 in the U. S23and 22.27 per 100,000 in Europe.4
ELATIVE® is a multi-center, randomized, double-blind, placebo-controlled Phase III clinical trial, with an open-label long-term extension (NCT03124108). ELATIVE® is evaluating the efficacy and safety of elafibranor 80mg once daily versus placebo for the treatment of patients with PBC with an inadequate response or intolerance to UDCA, the existing first-line therapy for PBC. The trial enrolled 161 patients who were randomized 2:1 to receive elafibranor 80mg once daily or placebo. Patients with an inadequate response to UDCA would continue to receive UDCA in combination with elafibranor or placebo, while patients unable to tolerate UDCA would receive only elafibranor or placebo.
Full data from the ELATIVE® trial will be presented at a future scientific congress.
GENFIT will host a conference call on June 30, 2023 at 8:00am ET / 1:00pm GMT / 2:00pm CET in English and in French
Both the English and French conference calls will be accessible on the investor page of our website, under the events section at https://ir.genfit.com/ or by calling 888-394-8218 (toll-free U.S. and Canada), 0800 279 0425 (toll-free UK) or 0805 101 219 (France) five minutes prior to the start time (confirmation code: 6752821). A replay will be available shortly after the call.
ABOUT ELAFIBRANOR
Elafibranor is a novel, oral, once-daily, dual peroxisome activated receptor (PPAR) alpha/delta (α,δ) agonist, currently under investigation as a treatment for patients with PBC, a rare liver disease. In 2019, it was granted a Breakthrough Therapy designation by the FDA in adults with PBC who have an inadequate response to ursodeoxycholic acid (UDCA). Elafibranor has not received approval by regulatory authorities anywhere in the world.
ABOUT IPSEN
Ipsen is a global, mid-sized biopharmaceutical company focused on transformative medicines in Oncology, Rare Disease and Neuroscience. With total sales of €3.0bn in FY 2022, Ipsen sells medicines in over 100 countries. Alongside its external-innovation strategy, the Company’s research and development efforts are focused on its innovative and differentiated technological platforms located in the heart of leading biotechnological and life-science hubs: Paris-Saclay, France; Oxford, U.K.; Cambridge, U.S.; Shanghai, China. Ipsen has around 5,400 colleagues worldwide and is listed in Paris (Euronext: IPN) and in the U.S. through a Sponsored Level I American Depositary Receipt program (ADR: IPSEY). For more information, visit ipsen.com
ABOUT GENFIT
GENFIT is a late-stage biopharmaceutical company dedicated to improving the lives of patients with rare and severe liver diseases characterized by high unmet medical needs. GENFIT is a pioneer in liver disease research and development with a rich history and strong scientific heritage spanning more than two decades. Thanks to its expertise in bringing early-stage assets with high potential to late development and pre-commercialization stages, today GENFIT boasts a growing and diversified pipeline of innovative therapeutic and diagnostic solutions. Its R&D pipeline covers six therapeutic areas via seven programs which explore the potential of differentiated mechanisms of action, across a variety of development stages (pre-clinical, Phase 1, Phase 2, Phase 3). These diseases are acute on-chronic liver failure (ACLF), hepatic encephalopathy (HE), cholangiocarcinoma (CCA), urea cycle disorders (UCD), organic acidemias (OA) and primary biliary cholangitis (PBC). Beyond therapeutics, GENFIT’s pipeline also includes a diagnostic franchise focused on NASH and ACLF. GENFIT has facilities in Lille and Paris (France), Zurich (Switzerland) and Cambridge, MA (USA). GENFIT is a publicly traded company listed on the Nasdaq Global Select Market and on compartment B of Euronext’s regulated market in Paris (Nasdaq and Euronext: GNFT). In 2021, IPSEN became one of GENFIT’s largest shareholders and holds 8% of the company’s share capital. For more information, visit www.genfit.com
Attachment
Genfit soars 23% as Phase 3 trial for liver disease candidate succeeds
Jun. 30, 2023 6:58 AM ET
Genfit S.A. (GNFT), IPSEY, IPSEF
By: Dulan Lokuwithana, SA News Editor
French biotech Genfit S.A. (NASDAQ:GNFT) added ~23% pre-market Friday after the company and its partner Ipsen (OTCPK:IPSEY) (OTCPK:IPSEF) announced that a Phase 3 trial for elafibranor targeted at the rare liver disease primary biliary cholangitis (PBC) reached the primary endpoint.
https://seekingalpha.com/news/3984392-genfit-stock-soars-on-liver-disease-drug
Genfit S.A. (GNFT), IPSEY, IPSEF
https://www.genfit.com/pipeline/elafibranor/clinical-overview/
Epcoritamab (DuoBody®-CD3xCD20)
Epcoritamab (DuoBody®-CD3xCD20)
Epcoritamab (DuoBody®-CD3xCD20)
June 27, 2023
AbbVie and Genmab Announce Positive Topline Results from Phase 1/2 EPCORE™ NHL-1 Trial Evaluating Epcoritamab (DuoBody®-CD3xCD20) in Patients with Relapsed/Refractory Follicular Lymphoma (FL)
- Based on the topline results of epcoritamab from the EPCORE™ NHL-1 clinical trial, AbbVie and Genmab will engage global regulatory authorities to discuss next steps
- Additional data from the clinical trial will be presented at a future medical meeting
- Follicular lymphoma is a common form of non-Hodgkin's lymphoma and currently has limited treatment options, particularly in the relapsed/refractory setting
NORTH CHICAGO, Ill., June 27, 2023 /PRNewswire/ -- AbbVie (NYSE: ABBV) and Genmab (Nasdaq: GMAB) today announced topline results from the follicular lymphoma (FL) cohort of the Phase 1/2 EPCORE™ NHL-1 clinical trial evaluating epcoritamab (DuoBody®-CD3xCD20), an investigational T-cell engaging bispecific antibody administered subcutaneously. The study cohort includes 128 adult patients with relapsed or refractory (R/R) FL who received at least two or more lines of systemic therapy. 70.3 percent of patients were double refractory to an anti-CD20 monoclonal antibody and an alkylating agent. Epcoritamab is being co-developed by AbbVie and Genmab as part of the companies' oncology collaboration.
"We are encouraged by these topline results, which further support the clinical profile of epcoritamab as a potential therapeutic option for patients with relapsed or refractory follicular lymphoma," said Mariana Cota Stirner, M.D., Ph.D., vice president, therapeutic area head for hematology, AbbVie. "Together with our partner Genmab, these results may bring us one step closer to our goal of advancing a potential core therapy for patients with B-cell malignancies."
EPCORE™ NHL-1 is an open-label trial evaluating the safety and preliminary efficacy of epcoritamab and consists of three parts: a Phase 1 first-in-human, dose escalation part; a Phase 2a expansion part; and a Phase 2a optimization part. The topline results from this cohort showed an overall response rate (ORR) of 82 percent as confirmed by an independent review committee (IRC), which exceeded the protocol prespecified threshold for efficacy. The observed median duration of response (DOR) was not reached, and longer follow-up will be required. The median number of lines of prior therapy in this cohort was three (range: two to nine lines of therapy).
No new safety signals were observed with epcoritamab in this study at the time of this analysis. The most common treatment-emergent adverse event was cytokine release syndrome (CRS) with 66.4 percent (1.6 percent Grade 3 or higher). The optimization part of the trial is continuing to evaluate alternative step-up dosing regimens to help further mitigate the risk of CRS, preliminary data are encouraging.
Full results from the study will be submitted for presentation at a future medical meeting.
About the Phase 1/2 EPCORE™ NHL-1 trial
EPCORE™ NHL-1 is an open-label, multi-center safety and preliminary efficacy trial of epcoritamab that consists of three parts: a Phase 1 first-in-human, dose escalation part; a Phase 2a expansion part; and a Phase 2a dose optimization part. The trial was designed to evaluate subcutaneous epcoritamab in adult patients with relapsed, progressive or refractory CD20+ mature B-cell non-Hodgkin's lymphoma (NHL), including follicular lymphoma (FL). In the Phase 2a expansion part, additional patients are being enrolled to further explore the safety and efficacy of epcoritamab in three cohorts of patients with different types of relapsed or refractory (R/R) B-cell NHLs who have limited therapeutic options. The dose optimization part evaluates the potential for alternative step-up dosing regimens to help further minimize Grade 2 CRS and mitigate Grade ≥3 cytokine release syndrome. The primary endpoint of the expansion part was overall response rate as assessed by an independent review committee. Secondary efficacy endpoints included duration of response, complete response rate, duration of complete response, progression-free survival, and time to response as determined by the Lugano criteria. Overall survival, time to next therapy, and rate of minimal residual disease negativity were evaluated as secondary efficacy endpoints.
About Follicular Lymphoma
Follicular lymphoma (FL) is typically an indolent, or slow growing, form of NHL that arises from B-cell lymphocytes.1 FL is the second most common form of NHL overall, accounting for 20 to 30 percent of all NHL cases, and representing 10 to 20 percent of all lymphomas in the western world.2,3 Although FL is an indolent lymphoma, it is considered incurable with conventional therapy.4,5
About Epcoritamab
Epcoritamab is an investigational IgG1-bispecific antibody created using Genmab's proprietary DuoBody® technology and administered subcutaneously. Genmab's DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B-cells and induces T cell mediated killing of CD20+ cells.6
Epcoritamab-bysp (EPKINLY™) was recently approved in the United States (U.S.) and is indicated for the treatment of adult patients with R/R diffuse large B-cell lymphoma (DLBCL), not otherwise specified (NOS), including DLBCL arising from indolent lymphoma, and high-grade B-cell lymphoma (HGBL) after two or more lines of systemic therapy. This indication is approved under accelerated approval based on response rate and durability of response. Continued approval for this indication is contingent upon verification and description of clinical benefit in a confirmatory trial(s). Please see U.S. Important Safety Information below.
In October 2022, a Marketing Authorization Application was submitted for epcoritamab for the treatment of patients with R/R DLBCL after two or more lines of systemic therapy, which was validated by the European Medicines Agency. Additionally, in December 2022, a Japan new drug application was submitted to the Ministry of Health, Labor and Welfare of Japan for epcoritamab for the treatment of patients with R/R large B-cell lymphoma (LBCL) after two or more lines of systemic therapy. Epcoritamab is not approved in the European Union and Japan.
The companies will share commercial responsibilities in the United States and Japan, with AbbVie responsible for further global commercialization. AbbVie will continue to pursue regulatory submissions for epcoritamab across international markets excluding the United States and Japan throughout the year.
Genmab and AbbVie are continuing to evaluate the use of epcoritamab as a monotherapy, and in combination, across lines of therapy in a range of hematologic malignancies. This includes an ongoing Phase 3, open-label, randomized trial evaluating epcoritamab as a monotherapy in patients with R/R DLBCL (NCT: 04628494), an ongoing Phase 3, open-label, randomized trial evaluating epcoritamab in combination in adult participants with newly diagnosed DLBCL (NCT: 05578976), and a Phase 3, open-label clinical trial evaluating epcoritamab in combination in patients with R/R FL (NCT: 05409066). Epcoritamab is not approved to treat newly diagnosed patients with DLBCL or FL. The safety and efficacy of epcoritamab has not been established for these investigational uses. Please visit clinicaltrials.gov for more information.
EPKINLY™ (epcoritamab-bysp) U.S. IMPORTANT SAFETY INFORMATION
Please see the Full Prescribing Information and Medication Guide, including Important Warnings.
About AbbVie in Oncology
At AbbVie, we are committed to transforming standards of care for multiple blood cancers while advancing a dynamic pipeline of investigational therapies across a range of cancer types. Our dedicated and experienced team joins forces with innovative partners to accelerate the delivery of potential breakthrough medicines. We are evaluating more than 20 investigational medicines in over 300 clinical trials across some of the world's most widespread and debilitating cancers. As we work to have a remarkable impact on people's lives, we are committed to exploring solutions to help patients obtain access to our cancer medicines. For more information, please visit http://www.abbvie.com/oncology.
About AbbVie
AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology and gastroenterology, in addition to products and services across our Allergan Aesthetics portfolio. For more information about AbbVie, please visit us at www.abbvie.com. Follow @abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.
ABBV Dividend History
https://www.nasdaq.com/market-activity/stocks/abbv/dividend-history
biotecHOPE™ 2023
NGENLA (somatrogon-ghla)
NGENLA (somatrogon-ghla)
NGENLA (somatrogon-ghla)
FDA Approves Pfizer’s NGENLA™, a Long-Acting Once-Weekly Treatment for Pediatric Growth Hormone Deficiency
Wednesday, June 28, 2023 - 06:45am
New, longer-acting treatment offers option to reduce the frequency of injections for children with growth hormone deficiency from daily to once-weekly
NEW YORK & MIAMI--(BUSINESS WIRE)-- Pfizer Inc. (NYSE: PFE) and OPKO Health Inc. (NASDAQ: OPK) announced today that the U.S. Food and Drug Administration (FDA) has approved NGENLA (somatrogon-ghla), a once-weekly, human growth hormone analog indicated for treatment of pediatric patients aged three years and older who have growth failure due to inadequate secretion of endogenous growth hormone. NGENLA is expected to become available for U.S. prescribing in August 2023.
Growth hormone deficiency (GHD) is a rare disease characterized by the inadequate secretion of the growth hormone somatropin from the pituitary gland, affecting one in approximately 4,000 to 10,000 children.1,2 Without treatment, children will have persistent growth attenuation, a very short height in adulthood, and puberty may be delayed.1,2,3 Children living with GHD may also experience challenges in relation to their physical health and mental well-being.1,2,3
“For more than 30 years, Pfizer has been committed to supporting children and adults living with growth hormone deficiency, beginning with the delivery of a medicine that has long been a part of the standard of care,” said Angela Hwang, Chief Commercial Officer, President, Global Biopharmaceuticals Business, Pfizer. “We are excited to bring this next-generation treatment to patients in the United States, continuing our commitment to helping children living with this rare growth disorder reach their full potential.”
The FDA approval is supported by results from a multi-center, randomized, open-label, active-controlled Phase 3 study which evaluated the safety and efficacy of NGENLA when administered once-weekly compared to once-daily somatropin. The study met its primary endpoint of NGENLA non-inferiority compared to somatropin, as measured by annual height velocity at 12 months. NGENLA was generally well tolerated in the study and had a safety profile comparable to somatropin.
“The approval of NGENLA will be significant for children with growth hormone deficiency in the U.S. It holds potential to reduce the treatment burden that can come with daily growth hormone injections,” said Joel Steelman, M.D., Pediatric Endocrinologist, Cook Children’s Health Care System. “As a new, longer-acting option that has the ability to reduce treatment frequency from daily to weekly, NGENLA could become an important treatment option that can improve adherence for children being treated for growth hormone deficiency.”
“Throughout our collaboration with Pfizer, we have worked tirelessly toward our shared goal of helping children living with growth hormone disease and their families,” said Phillip Frost, M.D., Chairman and Chief Executive Officer, OPKO Health. “We are proud of the clinical development program that supported the FDA approval of NGENLA and are excited about its potential for these patients and their families as it becomes available in the United States.”
NGENLA is approved for the treatment of pediatric GHD in more than 40 markets including Canada, Australia, Japan, and EU Member States.
The full Prescribing Information can be found here. If it is not currently available via this link, it will be visible as soon as possible as we work to finalize the document. Please check back for the full information shortly.
About NGENLA(somatrogon-ghla) Injection
NGENLA (somatrogon-ghla) is a human growth hormone that works by replacing the lack of growth hormone in the body. NGENLA is taken by injection just below the skin, administered via a device that allows for titration based on patient need. Compared to the growth hormone GENOTROPIN® (somatropin), its action in the body lasts longer, enabling weekly injections instead of daily.
In 2014, Pfizer and OPKO entered into a worldwide agreement for the development and commercialization of NGENLA for the treatment of GHD. Under the agreement, OPKO is responsible for conducting the clinical program and Pfizer is responsible for registering and commercializing NGENLA for GHD.
About the NGENLA Clinical Program
The safety and efficacy of NGENLA (somatrogon-ghla) was demonstrated in a multi-center, randomized, open-label, active-controlled Phase 3 study (NCT 02968004). The Phase 3 study enrolled and treated 224 pediatric patients, treatment-naïve children with growth hormone deficiency who were randomized 1:1 into two arms: NGENLA (somatrogon-ghla) once-weekly at a dose of 0.66 mg/kg/day vs somatropin, once-daily at a dose of 0.034 mg/kg/day. The study met its primary endpoint of NGENLA non-inferiority compared to somatropin, measured by annual height velocity at 12 months.
About Growth Hormone Deficiency
Growth hormone deficiency is a rare disease characterized by the inadequate secretion of growth hormone from the pituitary gland and affects one in approximately 4,000 to 10,000 children.1,2 In children, this disease can be caused by genetic mutations or acquired after birth.1,4 Because the patient's pituitary gland secretes inadequate levels of somatropin, the hormone that causes growth, a child’s height may be affected and puberty may be delayed.1,2,5 Without treatment, affected children will have persistent growth attenuation and a very short height in adulthood.1,2 Children may also experience challenges in relation to physical health and mental well-being.1,2
Important NGENLA (somatrogon-ghla) Safety Information
- Growth hormone should not be used in children after the growth plates have closed.
- Growth hormone should not be used in children with some types of eye problems caused by diabetes (diabetic retinopathy).
- Growth hormone should not be used in children who have cancer or other tumors.
- Growth hormone should not be used in children who are critically ill because of some types of heart or stomach surgery, trauma, or breathing (respiratory) problems.
- Growth hormone should not be used in children with Prader-Willi syndrome who are very overweight or have breathing problems including sleep apnea.
- NGENLA should not be used by children who have had an allergic reaction to somatrogon-ghla or any of the ingredients in NGENLA. Look for prompt medical attention in case of an allergic reaction.
- Some children have developed diabetes mellitus while taking growth hormone. Dosages of diabetes medicines may need to be adjusted during treatment with NGENLA. Children should be watched carefully if NGENLA is given along with glucocorticoid therapy and/or other drugs that are processed by the body in the same way.
- In childhood cancer survivors, treatment with growth hormone may raise the likelihood of a new tumor, particularly some benign (non-cancerous) brain tumors. This likelihood may be higher in children who were treated with radiation to the brain or head. Your child’s health care provider will need to check your child for a return of cancer or a tumor.
- Children treated with growth hormone have had increased pressure in the brain. If your child has headaches, eye problems, nausea (feeling like you are going to be sick), or vomiting, contact your child’s health care provider.
- NGENLA may decrease thyroid hormone levels. Decreased thyroid hormone levels may change how well NGENLA works. Your child’s health care provider will do blood tests to check your child’s hormone levels.
- Children treated with growth hormone should be checked regularly for low serum cortisol levels and/or the need to increase the dose of the glucocorticoids they are taking.
- In children experiencing fast growth, curvature of the spine may develop or worsen. This is also called scoliosis. Children with scoliosis should be checked regularly to make sure their scoliosis does not get worse during their growth hormone therapy.
- Use a different area on the body for each injection. This can help to avoid skin problems such as lumpiness or soreness.
- Growth hormone treatment may cause serious and constant stomach (abdominal) pain. This could be a sign of pancreatitis. Tell your child’s health care provider if your child has any new stomach (abdominal) pain.
- In studies of NGENLA in children with GHD, side effects included injection site reactions such as pain, swelling, rash, itching, or bleeding. Other side effects were the common cold, headache, fever (high temperature), low red blood cells (anemia), cough, vomiting, decreased thyroid hormone levels, stomach pain, rash, or throat pain.
- A health care provider will help you with the first injection. He or she will also train you on how to inject NGENLA.
- Rx only
About GENOTROPIN(somatropin)
GENOTROPIN is a man-made, prescription treatment option. The indications GENOTROPIN is approved for vary by market. GENOTROPIN is approved for growth failure due to GHD and adult GHD, Prader-Willi Syndrome, Idiopathic Short Stature, Turner Syndrome, Small for Gestational Age (with no catch-up growth), and Chronic Renal Insufficiency. GENOTROPIN is taken by injection just below the skin and is available in a wide range of devices to fit a range of individual dosing needs. GENOTROPIN is just like the natural growth hormone that our bodies make and has an established safety profile.
Important GENOTROPIN (somatropin) Safety Information
- Somatropin should not be used for growth promotion in pediatric patients with closed epiphyses.
- Somatropin is contraindicated in patients with active proliferative or severe nonproliferative diabetic retinopathy.
- Somatropin is contraindicated in patients with active malignancy. Because growth hormone deficiency may be a sign of pituitary or other brain tumors, the presence of such tumors should be ruled out before treatment is initiated. Somatropin should not be used in patients with any evidence of progression or recurrence of an underlying intracranial tumor.
- Somatropin in pharmacologic doses should not be used to treat patients with acute critical illness due to complications from open heart surgery, abdominal surgery or multiple accidental traumas, or those patients with acute respiratory failure due to an increased mortality. The safety of continuing replacement somatropin treatment for approved uses in patients who develop these illnesses has not been established.
- Somatropin is contraindicated in patients with Prader-Willi syndrome who are severely obese or have respiratory impairment.
- GENOTROPIN is contraindicated in patients with a known hypersensitivity to somatropin or any of its excipients. Serious systemic hypersensitivity reactions including anaphylactic reactions and angioedema have been reported with postmarketing use of somatropin products. Patients and caregivers should be informed that such reactions are possible and that prompt medical attention should be sought if an allergic reaction occurs.
- New-onset Type-2 diabetes mellitus has been reported. Monitor patients with glucose intolerance closely; dosage of antihyperglycemic drug may need to be adjusted. Monitor carefully if somatropin is administered in combination with glucocorticoid therapy and/or other drugs metabolized by the CP450 pathway.
- In childhood cancer survivors, an increased risk of a second neoplasm, in particular meningiomas, has been reported in patients treated with somatropin after their first neoplasm, particularly those who were treated with cranial radiation. Children with certain rare genetic causes of short stature have an increased risk of developing malignancies. Practitioners should thoroughly consider the risks and benefits of starting somatropin in these patients and if treatment is initiated, should carefully monitor these patients for development of neoplasms. Patients should be monitored carefully for any malignant transformation of skin lesions.
- Intracranial hypertension (IH) has been reported in a small number of patients treated with somatropin. If papilledema is observed during somatropin treatment, treatment should be stopped and reassessed. Patients with Turner syndrome and Prader-Willi syndrome may be at increased risk for the development of IH.
- Undiagnosed/untreated hypothyroidism may prevent an optimal response to somatropin, in particular, the growth response in children. Patients with Turner syndrome have an inherently increased risk of developing autoimmune thyroid disease and primary hypothyroidism. In patients with growth hormone deficiency, central (secondary) hypothyroidism may first become evident or worsen during somatropin treatment. Therefore, patients treated with somatropin should have periodic thyroid function tests, and thyroid hormone replacement therapy should be initiated or appropriately adjusted when indicated.
- Patients treated with somatropin who have or are at risk for pituitary hormone deficiency(s) may be at risk for reduced serum cortisol levels and/or unmasking central hypoadrenalism and should be monitored for reduced serum cortisol levels. In addition, patients treated with glucocorticoid replacement for pre-existing hypoadrenalism may require an increase in their maintenance or stress doses following initiation of somatropin treatment and should be monitored for reduced cortisol levels and/or need for glucocorticoid dose increases.
- Progression of scoliosis can occur in patients who experience rapid growth. Patients with scoliosis should be monitored for manifestation or progression during somatropin therapy.
- Slipped capital femoral epiphyses may occur more frequently in patients with endocrine disorders (including GHD and Turner syndrome) or in patients undergoing rapid growth. Any pediatric patient with the onset of a limp or complaints of hip or knee pain during somatropin therapy should be carefully evaluated.
- Somatropin should be used during pregnancy only if clearly needed and with caution in nursing mothers because it is not known whether somatropin is excreted in human milk.
- Subcutaneous injection of somatropin at the same site repeatedly may result in tissue atrophy. This can be avoided by rotating the injection site.
- Cases of pancreatitis have been reported rarely in children and adults receiving somatropin treatment, with some evidence supporting a greater risk in children compared with adults. Published literature indicates that girls who have Turner syndrome may be at greater risk than other somatropin-treated children. Pancreatitis should be considered in any somatropin-treated patient, especially a child, who develops persistent severe abdominal pain.
- In clinical trials with GENOTROPIN in pediatric GHD patients, the following events were reported infrequently: injection site reactions, including pain or burning associated with the injection, fibrosis, nodules, rash, inflammation, pigmentation, or bleeding; lipoatrophy; headache; hematuria; hypothyroidism; and mild hyperglycemia.
- In clinical studies of 273 pediatric patients born SGA treated with GENOTROPIN, the following clinically significant events were reported: mild transient hyperglycemia; 1 patient with benign intracranial hypertension; 2 patients with central precocious puberty; 2 patients with jaw prominence; and several patients with aggravation of preexisting scoliosis, injection site reactions, and self-limited progression of pigmented nevi. Anti-hGH antibodies were not detected in any of the patients treated with GENOTROPIN.
- Deaths have been reported with the use of a growth hormone in pediatric PWS patients with severe obesity, history of upper airway obstruction or sleep apnea, and/or unidentified respiratory infection. Therefore, all patients with PWS should be evaluated and monitored for signs of upper airway obstruction, sleep apnea, and respiratory infections, and have effective weight control.
- In clinical trials with GENOTROPIN in pediatric patients with PWS, the following drug-related events were reported: edema, aggressiveness, arthralgia, benign intracranial hypertension, hair loss, headache, and myalgia.
- Somatropin may increase the occurrence of otitis media in Turner syndrome patients. In 2 clinical studies with GENOTROPIN in pediatric patients with Turner syndrome, the most frequently reported adverse events were respiratory illnesses (influenza, tonsillitis, otitis, sinusitis), joint pain, and urinary tract infection. The only treatment-related adverse event that occurred in more than 1 patient was joint pain.
- In 2 clinical studies with GENOTROPIN in pediatric patients with ISS, the most commonly encountered adverse events included upper respiratory tract infections, influenza, tonsillitis, nasopharyngitis, gastroenteritis, headaches, increased appetite, pyrexia, fracture, altered mood, and arthralgia.
- In clinical trials with GENOTROPIN in adults with GHD, the majority of side effects were symptoms of fluid retention, including peripheral swelling/edema, arthralgia, pain and stiffness of the extremities, myalgia, paresthesia, and hypoesthesia. Generally, these were transient and dose-dependent.
- In women on oral estrogen replacement, a larger dose of somatropin may be required to achieve the defined treatment goal.
- Elderly patients may be more sensitive to the action of somatropin, and therefore may be more prone to develop adverse reactions.
- The cartridges of GENOTROPIN contain m-Cresol and should not be used by patients with a known sensitivity to this preservative.
- Subcutaneous injection of somatropin at the same site repeatedly may result in tissue atrophy. This can be avoided by rotating the injection site.
- Health care providers should supervise the first injection and provide appropriate training and instruction for the proper use of all devices for GENOTROPIN.
- Rx only
For the full Prescribing Information for GENOTROPIN, please visit http://labeling.pfizer.com/ShowLabeling.aspx?id=577.
Pfizer Inc.: Breakthroughs that Change Patients’ Lives
At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety, and value in the discovery, development, and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments, and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world's premier innovative biopharmaceutical companies, we collaborate with health care providers, governments, and local communities to support and expand access to reliable, affordable health care around the world. For more than 170 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.pfizer.com. In addition, to learn more, please visit us on www.pfizer.com and follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.
About OPKO Health
OPKO is a multinational biopharmaceutical and diagnostics company that seeks to establish industry-leading positions in large, rapidly growing markets by leveraging its discovery, development, and commercialization expertise and novel and proprietary technologies. For more information, visit www.opko.com.
Source: Pfizer Inc.
Pfizer, OPKO Health win FDA nod for dwarfism therapy
Jun. 28, 2023 7:35 AM ET
By: Dulan Lokuwithana, SA News Editor1 Comment
- Pfizer (NYSE:PFE) and OPKO Health (OPK) announced Wednesday the FDA approval of Ngenla, a once-weekly human growth hormone analog for children with growth hormone deficiency, commonly known as dwarfism.
- Specifically, the FDA approval for Ngenla (somatrogon-ghla) allows its use in children aged three years and older whose growth failure is caused by insufficient production of growth hormone. The companies expect to launch Ngenla for the U.S. market in August 2023.
- https://seekingalpha.com/news/3983563-pfizer-opko-health-win-fda-nod-dwarfism-therapy
- Pfizer Inc. (PFE)OPK
- https://www.pfizer.com/
- https://www.opko.com/what-we-do/our-research/somatrogon
ACI-24.060
NGENLA (somatrogon-ghla)
NGENLA (somatrogon-ghla)
AC Immune Receives FDA Fast Track Designation For Anti-Amyloid-Beta Active Immunotherapy, ACI-24.060, To Treat Alzheimer’s Disease
Jun 27, 2023
AC Immune Receives FDA Fast Track Designation for Anti-Amyloid-beta Active Immunotherapy, ACI-24.060, to Treat Alzheimer’s Disease
- Ongoing Phase 1b/2 ABATE study enrolling well and expanding to sites in USA with Investigational New Drug (IND) clearance for ACI-24.060
- Dosed first individual with Down syndrome (DS) in the DS cohort of ABATE
- Interim safety and immunogenicity data in Alzheimer’s disease (AD) and DS cohorts expected in H2 2023
- Initial PET data on amyloid plaque reduction in AD expected H1 2024
Lausanne, Switzerland, June 27, 2023 – AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases, today announced that it has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for its wholly-owned anti-amyloid beta (Abeta) active immunotherapy (vaccine)-candidate, ACI-24.060, for treatment of Alzheimer’s disease. This follows FDA clearance of the Investigational New Drug (IND) application enabling expansion to the USA of the ongoing Phase 1b/2 ABATE study of ACI-24.060 in patients with AD and individuals with DS. Furthermore, the first individual with DS has been dosed in ABATE.
Dr. Andrea Pfeifer, CEO of AC Immune SA, commented: “We are delighted to see the quality and importance of our ACI-24.060 program reflected in the granting of Fast Track designation, which offers opportunities for expedited development and regulatory review. This regulatory progress underscores the attraction of an active immunotherapy targeting toxic species of Abeta. By inducing a polyclonal response including antibodies against both oligomeric Abeta and pyroglutamate-Abeta, ACI-24.060 targets the same toxic species as disease modifying anti-Abeta monoclonal antibodies that slowed AD progression in Phase 3 clinical trials. As ACI-24.060, created using our SupraAntigen® platform, specifically targets the most toxic forms of Abeta, we believe it may offer best-in-class efficacy with all the potential advantages in safety, administration and distribution that can be expected from a vaccine. We look forward to showing in H1 2024 the effect of ACI-24.060 on amyloid plaque reduction, a surrogate marker for disease modification.”
ACI-24.060’s Fast Track designation and IND clearance, as well as the expansion of ABATE to include individuals with DS were supported by positive initial interim safety and immunogenicity data from ABATE’s first, low dose AD cohort. Dosing in a second, higher dose AD cohort began earlier this year.
Dr. Johannes Streffer, CMO of AC Immune SA, commented: “ABATE’s expansion into the USA will allow us to accelerate the trial’s advancement and build upon the strong momentum we’ve generated in Europe. With today’s announcement, we remain firmly on track to report additional interim safety and immunogenicity data later this year, and for the crucial interim readout of Abeta-PET imaging data in AD in the first half of next year. By benchmarking the amount of Abeta plaque reduction achieved with ACI-24.060 against those achieved with FDA-approved monoclonal antibodies, we believe we can generate early evidence of our vaccine’s therapeutic potential to support its expeditious advancement towards pivotal programs in AD and DS-related AD.”
Dr. Michael Rafii, Medical Director of the Alzheimer’s Therapeutic Research Institute, Professor of Neurology at the Keck School of Medicine, and the Principal Investigator of the trial commented: “Despite representing the world’s largest population that is genetically at high risk for AD, individuals with DS are vastly underserved and underrepresented in clinical trials. I applaud AC Immune for seeking to address the urgent needs of this population and believe ACI-24.060 holds great promise as a novel therapy that can lower Abeta plaques to delay, or perhaps even prevent, the onset of clinical dementia symptoms in AD and DS-related AD. Moreover, I believe the potential safety, efficacy, and logistical advantages of a vaccine over monoclonal antibodies strongly support the development of therapeutics such as ACI-24.060 as the next generation of anti-Abeta therapies.”
About the Phase 1b/2 ABATE Study (ClinicalTrials.gov Identifier: NCT05462106)
The ABATE study is a Phase 1b/2, multicenter, adaptive, double-blind, randomized, placebo-controlled study to assess the safety, tolerability, immunogenicity, and pharmacodynamic effects of ACI-24.060 in subjects with prodromal Alzheimer’s disease and in adults with Down syndrome. All participants in the trial must have brain Abeta pathology confirmed by a positron emission tomography (PET) scan. Recent clinical studies and FDA approvals have validated Abeta as a disease modifying therapeutic target in AD and are supportive of Abeta PET imaging as a surrogate marker of efficacy. The trial begins with a dose escalation phase in AD patients, during which various doses/dosing regimens may be evaluated, and also includes individuals with DS.
About AD in Down syndrome
Individuals with Down syndrome (DS) have a third copy of all or part of chromosome 21, which contains the gene that codes for amyloid-precursor protein (APP). Overproduction of APP is believed to cause the accumulation of Abeta plaques. Virtually all individuals with DS will develop Abeta plaques and AD1, with DS-related AD sharing a similar pathophysiology and biomarkers with other forms of genetic AD. Given the more predictable onset and progression of symptoms in DS-related AD, AC Immune believes ABATE’s results will offer crucial insights into the ability of ACI-24.060 active immunotherapy to modulate neurodegeneration at its earliest stages and offer this population a much needed therapeutic option.
About ACI-24.060
ACI-24.060, derived from AC Immune’s SupraAntigen® platform, has been shown in preclinical studies to induce a strong polyclonal antibody response that matures and is maintained against both oligomeric and pyroglutamate-Abeta species, key pathological forms of Abeta believed to drive Abeta plaque formation and disease progression. ACI-24.060 is designed to enhance the formation of broad-spectrum protective antibodies with the same safety and tolerability previously demonstrated in the ACI-24 program in Phase 1 and 2 trials. This investigational candidate has the potential to efficiently inhibit plaque formation and increase plaque clearance, and thereby may reduce or prevent disease progression.
Reference
- Lott, Ira T., and Elizabeth Head. "Dementia in Down syndrome: unique insights for Alzheimer disease research." Nature Reviews Neurology 15.3 (2019): 135-147.
About AC Immune SA
AC Immune SA is a clinical-stage biopharmaceutical company that aims to become a global leader in precision medicine for neurodegenerative diseases, including Alzheimer’s disease, Parkinson’s disease, and NeuroOrphan indications driven by misfolded proteins. The Company’s two clinically validated technology platforms, SupraAntigen® and Morphomer®, fuel its broad and diversified pipeline of first- and best-in-class assets, which currently features ten therapeutic and three diagnostic candidates, five of which are currently in Phase 2 clinical trials and one of which is in Phase 3. AC Immune has a strong track record of securing strategic partnerships with leading global pharmaceutical companies including Genentech, a member of the Roche Group, Eli Lilly and Company, and others, resulting in substantial non-dilutive funding to advance its proprietary programs and >$3 billion in potential milestone payments.
SupraAntigen® is a registered trademark of AC Immune SA in the following territories: AU, EU, CH, GB, JP, RU, SG and USA. Morphomer® is a registered trademark of AC Immune SA in CN, CH, GB, JP, KR, NO and RU.
The information on our website and any other websites referenced herein is expressly not incorporated by reference into, and does not constitute a part of, this press release.
Attachment
Source: AC Immune SA
AC Immune gains 14% on FDA fast track tag for Alzheimer’s vaccine
Jun. 27, 2023 8:44 AM ET
By: Dulan Lokuwithana, SA News Editor
Swiss biotech AC Immune SA (NASDAQ:ACIU) added ~14% pre-market Tuesday after announcing that the FDA issued Fast Track designation for its anti-amyloid vaccine ACI-24.060 as a treatment for Alzheimer’s disease (AD).
The FDA offers the Fast Track designation to accelerate the development and review of drugs targeted at serious conditions with unmet medical needs. Companies with Fast Track Designations, subject to conditions, can secure the FDA’s Accelerated Approval and Priority Review, enabling patients to access those treatments sooner.
Previously, the FDA had cleared AC Immune’s (ACIU) Investigational New Drug (IND) application for ACI-24.060, allowing the company to expand its ongoing Phase 1b/2 ABATE study in the U.S. for patients with AD and Down syndrome.
https://seekingalpha.com/news/3983125-ac-immune-wins-fda-fast-track-tag-alzheimers-shot