iHub News
5月前
BioMarin, Ascendis slide as BridgeBio shakes up achondroplasia landscapeFebruary 12, 2026 10:25 AM
IH Market News
Shares of BioMarin Pharmaceutical (NASDAQ:BMRN) and Ascendis Pharma (NASDAQ:ASND) fell roughly 6% after rival BridgeBio Pharma (NASDAQ:BBIO) reported encouraging Phase 3 results for its experimental achondroplasia therapy.BridgeBio said its oral drug infigratinib achieved the primary endpoint in the late-stage PROPEL 3 trial, delivering superior annualized height velocity compared with placebo. The company cited a mean treatment difference of +2.10 cm per year, and noted the therapy also produced the first statistically significant improvement in body proportionality versus placebo in a randomized achondroplasia study.The update is viewed as a potential competitive challenge to BioMarin’s Voxzogo, currently the only treatment for achondroplasia approved by the U.S. Food and Drug Administration.Ascendis, meanwhile, is awaiting a regulatory decision on its once-weekly injectable therapy for achondroplasia, with potential FDA approval expected by February 28. Its shares also retreated following the BridgeBio announcement.Investors appear to be weighing the potential advantages of BridgeBio’s oral formulation, which showed strong efficacy and no serious adverse events linked to the study drug. The company plans to file regulatory submissions in the second half of 2026.Achondroplasia is a rare genetic disorder that affects bone growth, occurring in roughly one in 15,000 to 40,000 births. An estimated 55,000 people across the U.S. and European Union are living with the condition.BioMarin Pharmaceutical stock priceAscendis Pharma stock priceBridgeBio Pharma stock price
Original: BioMarin, Ascendis slide as BridgeBio shakes up achondroplasia landscape
iHub News
5月前
BridgeBio rallies on strong Phase 3 data for achondroplasia pillFebruary 12, 2026 10:22 AM
IH Market News
BridgeBio Pharma (NASDAQ:BBIO) shares climbed about 9% on Thursday after the biotech group unveiled positive Phase 3 topline results for its oral drug infigratinib in achondroplasia, marking what it said were the first statistically significant gains in body proportionality seen in the condition.The late-stage PROPEL 3 study met its primary endpoint, showing that patients treated with infigratinib achieved superior improvements in annualized height velocity (AHV) versus placebo at 52 weeks. The company reported a mean treatment difference of +2.10 cm per year, with a least squares mean of +1.74 cm per year.In a pre-specified exploratory subgroup analysis of children under eight — who accounted for more than half of trial participants — the therapy also delivered the first statistically significant improvement in body proportionality compared with placebo in achondroplasia. The least squares mean treatment difference was -0.05.Safety data appeared favorable. The company said the treatment was well tolerated, with no drug-related serious adverse events or discontinuations. Three cases (4%) of hyperphosphatemia were recorded, all mild and temporary, and none required dose reductions or treatment interruption.BridgeBio said it intends to file both a New Drug Application (NDA) in the U.S. and a Marketing Authorization Application (MAA) in Europe in the second half of 2026. Oral infigratinib is currently the only therapy in development for achondroplasia that has received Breakthrough Therapy Designation from the FDA.Buoyed by the results, the company also plans to speed up development of the drug for hypochondroplasia, with enrollment already underway for the observational run-in phase of a separate Phase 3 trial.Achondroplasia is a rare genetic disorder driven by overactivity of the FGFR3 pathway, leading to impaired bone growth and disproportionate short stature, particularly affecting limb length and overall body proportions.BridgeBio Pharma stock price
Original: BridgeBio rallies on strong Phase 3 data for achondroplasia pill
02opida
4年前
PALO ALTO, Calif., May 13, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (NASDAQ:BBIO) (BridgeBio),?a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced today that it has entered into a definitive agreement with an undisclosed purchaser to sell its PRV for $110 million.
The Company received the voucher in February 2021 under a U.S. Food and Drug Administration (FDA) program intended to encourage the development of treatments for rare pediatric diseases. BridgeBio was awarded the voucher when its affiliate, Origin Biosciences Inc., received approval of NULIBRY™ (fosdenopterin) for injection as the first therapy to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A. The sale is subject to customary closing conditions and is expected to occur following the expiration of applicable U.S. antitrust clearance requirements.
02opida
4年前
PALO ALTO, Calif., May 20, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) and affiliate company Phoenix Tissue Repair, which is focused on advancing a novel systemic treatment for recessive dystrophic epidermolysis bullosa (RDEB), announced data from the Phase 2 trial of PTR-01, an intravenously-administered recombinant collagen 7 (rC7) protein replacement therapy, in patients with recessive dystrophic epidermolysis bullosa (RDEB). The data are being shared in a poster at the Society for Investigative Dermatology (SID) Annual Meeting 2022 between May 18 - 21, 2022 in Portland, Oregon.
RDEB is a rare genetic disorder caused by mutations in the gene encoding collagen type VII (C7) and is one of the most severe forms of epidermolysis bullosa, characterized by severe and painful skin blistering, as well as extreme fragility and scarring of mucous membranes throughout the body. There is currently no known cure or effective treatment available for patients suffering from this disease.
"In patients with recessive dystrophic epidermolysis bullosa (RDEB) even minor friction or trauma can cause debilitating blistering, tearing and scarring of the skin, along with severe pain and itching. Our data shows that treatment with PTR-01 led to rapid, consistent, and durable wound healing," said Sanuj K. Ravindran, M.D., executive chairman of Phoenix Tissue Repair. "We are hopeful that by addressing the root cause of this rare disease, we will be able to provide a treatment beyond daily wound care and pain management for patients in need."
02opida
4年前
PALO ALTO, Calif., May 17, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) ("BridgeBio" or the "Company"), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that members of the management team will participate in the following upcoming investor conferences:
Citi Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Panel, Virtual: Wednesday, May 18th at 2:30 pm ET
H. C. Wainwright Global Investment Conference, Virtual: Tuesday, May 24th at 7:00 am ET
UBS Global Healthcare Conference, New York, NY: Tuesday, May 24th at 11:30 am ET
J. P. Morgan West Coast Investor Day, San Francisco, CA: Tuesday, May 24th at 12:00 pm ET
To access more details on BridgeBio's presentations, please visit the "Events & Presentations" page within the Investors section of the BridgeBio website at http://investor.bridgebio.com.
crudeoil24
4年前
BridgeBio Pharma Reports Sale Of Rare Pediatric Disease Priority Review Voucher For $110M, Defers Principal Payment On Senior Debt By 2 Years
7:32 am ET May 13, 2022 (Benzinga) Print
-Entered into a definitive agreement to sell the rare pediatric disease Priority Review Voucher (PRV) it obtained in February 2021 for $110 million
-Secured a two-year extension of interest-only period on its existing senior secured credit facility
PALO ALTO, Calif., May 13, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (NASDAQ:BBIO) (BridgeBio),?a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced today that it has entered into a definitive agreement with an undisclosed purchaser to sell its PRV for $110 million.
The Company received the voucher in February 2021 under a U.S. Food and Drug Administration (FDA) program intended to encourage the development of treatments for rare pediatric diseases. BridgeBio was awarded the voucher when its affiliate, Origin Biosciences Inc., received approval of NULIBRY™ (fosdenopterin) for injection as the first therapy to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A. The sale is subject to customary closing conditions and is expected to occur following the expiration of applicable U.S. antitrust clearance requirements.
In connection with the PRV sale, BridgeBio has executed an amendment to its existing senior secured credit facility, extending the interest-only period by two years and principal repayment to November 17, 2026. The Company received consent for the PRV sale from its lenders with all proceeds retained by BridgeBio. BridgeBio retains access to up to $100 million in delayed debt draws through year end 2022, subject to certain conditions. The amendment was approved unanimously by existing lenders in the syndicate without adjusting pricing and without imposing financial covenants.
“The sale of this voucher will help us advance our pipeline of drug development programs targeting genetic diseases and cancers,” said Brian Stephenson, Ph.D., CFA, BridgeBio’s Chief Financial Officer. “We believe this deal, coupled with our amended loan agreement, offers us the opportunity to read out more data within the duration of our debt and advance meaningful medicines to patients in need in the years to come.”
© 2022 Benzinga.com. Benzinga does not provide investment advice. All rights reserved.
02opida
4年前
PALO ALTO and SOLANA BEACH, Calif., March 08, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company that focuses on genetic diseases and cancers, and Sentynl Therapeutics, Inc. (Sentynl), a U.S.-based biopharmaceutical company focused on bringing innovative therapies to patients living with rare diseases owned by Zydus Lifesciences Ltd. (formerly known as Cadila Healthcare Ltd.), today announced the execution of an asset purchase agreement (the Agreement) for the sale of BridgeBio's NULIBRY(TM) (Fosdenopterin) for Injection. NULIBRY is approved by the U.S. Food and Drug Administration (FDA) to reduce the risk of mortality in patients with molybdenum cofactor deficiency (MoCD) Type A, an ultra-rare, life-threatening pediatric genetic disorder. The closing of the asset purchase is subject to customary closing conditions.
"Sentynl's focus on meaningful treatments for serious rare diseases is further enhanced by the acquisition of Fosdenopterin. We will leverage our existing platform of ultra-rare pediatric disease initiatives to facilitate early diagnosis and treatment by enhancing awareness, newborn screening, genetic testing and patient support across multiple products and rare diseases. By partnering with BridgeBio, we hope to reach even more patients born with MoCD Type A as quickly as possible with the hope of reducing the risk of mortality and progression of this devastating disease," said Matt Heck, CEO of Sentynl.
Under the Agreement, Sentynl will acquire global rights to NULIBRY and will be responsible for the ongoing development and commercialization of NULIBRY in the United States and developing, manufacturing and commercializing Fosdenopterin globally. BridgeBio will share development responsibilities for Fosdenopterin through approval of the marketing authorization application already under accelerated assessment with the European Medicines Agency and through approval of its regulatory submission with the Israeli Ministry of Health. Sentynl will provide cash payments upon the achievement of certain regulatory milestones. BridgeBio will be eligible to receive commercial milestone payments as well as tiered royalties on adjusted net sales of NULIBRY.
"Sentynl is an ideal partner given its expertise in the rare pediatric neurodevelopment space and its relationships with physicians who diagnose and treat children with MoCD Type A," said Neil Kumar, Ph.D., founder and CEO of BridgeBio. "Focused execution means reducing the scope of our internal activity. We will continue to advance high-quality programs in our pipeline while expanding our reach to patients in need of options."
About Mol
02opida
4年前
PALO ALTO, Calif., Jan. 27, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced that the first patient has been dosed in ADventure, its Phase 1/2 clinical trial of BBP-631, an investigational adeno-associated virus (AAV) 5 gene therapy for the treatment of classic congenital adrenal hyperplasia (CAH). CAH is one of the most prevalent genetic diseases, with more than 75,000 cases estimated in the United States and European Union.
“Dosing the first patient in our CAH trial is a landmark milestone and we are grateful for the support from the medical and patient communities who helped us reach this moment. For more than 50 years people living with CAH have had the same limited standard of care – lifelong daily steroid replacement treatment. Our investigational gene therapy offers patients a potential single-dose intervention designed to restore their body’s hormone and steroid balance by making their own cortisol and aldosterone,” said Eric David, M.D., J.D., CEO at BridgeBio Gene Therapy. “This is the second gene therapy trial we have initiated in less than four months, and we are excited to advance this trial and our other gene therapy programs in the hope of improving patients’ lives.”
“Adults, children and families affected by CAH experience the daily burden of the disease and often, unfortunately, the side effects and morbidities associated with the current treatment regimens. As an endocrinologist, it’s incredibly exciting to reimagine a new approach to treating this disease,” added Adam Shaywitz, M.D., Ph.D., chief medical officer at BridgeBio Gene Therapy. Adrenas Therapeutics, the affiliate company of BridgeBio focused on developing BBP-631 for CAH, is part of BridgeBio Gene Therapy’s portfolio.
The Phase 1/2 open-label study is designed to evaluate the safety, tolerability and pharmacodynamic activity of the company’s AAV5 gene therapy, BBP-631, in adults with classic CAH. In the initial dose-finding phase of the study, each subject will receive a single intravenous (IV) infusion. The primary outcomes of the study are safety, as well as change from baseline in endogenous cortisol levels which BBP-631 has the unique potential to restore. Change from baseline in steroid biomarkers for hydroxyprogesterone (17-OHP) levels and androstenedione (A4) levels will also be measured. Preclinical proof-of-concept data have shown the approach provides efficient and persistent delivery of functional 21-hydroxylase (21-OH) enzyme to the adrenal gland.
“We are honored to be the first site to administer gene therapy in a patient with CAH as it is a potential game-changing treatment option that targets the disease at its source,” said Kyriakie Sarafoglou, M.D., associate professor and director of the Center for Congenital Adrenal Hyperplasia at the University of Minnesota. “We are eager to see whether gene therapy can restore endogenous cortisol production, and also look forward to exploring its effect on the physiological mechanisms that regulate the hypothalamic-pituitary-adrenal axis including circadian and ultradian hormonal profiles.”
BridgeBio’s investigational AAV5 gene therapy for CAH is one of the Company’s 14 programs that are in the clinic or commercial setting for patients living with genetic diseases and cancers. Initial Phase 1/2 data readouts of the Company’s AAV5 gene therapy for CAH and the Company’s AAV9 gene therapy for Canavan disease are expected in the second half of 2022.
02opida
4年前
PALO ALTO, Calif., Jan. 13, 2022 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced a non-exclusive clinical collaboration with Amgen Inc. (Amgen) to evaluate the combination of BBP-398, a potentially best-in-class SHP2 inhibitor, with LUMAKRAS® (sotorasib), a KRASG12C inhibitor, in patients with advanced solid tumors with the KRAS G12C mutation.
The Phase 1/2 study will include a dose escalation period followed by dose expansion and optimization, and is designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary efficacy of BBP-398 in combination with LUMAKRAS. Under the terms of the non-exclusive collaboration, BridgeBio will sponsor the study and Amgen will provide a global supply of LUMAKRAS.
BBP-398 is a potent small-molecular inhibitor of SHP2 developed in collaboration with The University of Texas MD Anderson Cancer Center’s Therapeutics Discovery division. SHP2 is a protein-tyrosine phosphatase that links growth factor, cytokine and integrin signaling with the downstream RAS/ERK MAPK pathway to regulate cellular proliferation and survival. By combining SHP2 inhibition with KRASG12C inhibition in patients with the KRAS G12C mutation, there is potential that the investigational combination could prevent oncogenesis and overactive cellular proliferation.
“Overactivity of the MAPK pathway is a significant cause of many types of difficult-to-treat cancers and by combining these two agents, we aim to reduce the oncogenic potential of tumor cells,” said Frank McCormick, Ph.D., chairman of oncology at BridgeBio. “Building on our collaborations with Bristol Myers Squibb and LianBio, we are excited to be working with Amgen on this new collaboration. By harnessing the power of BBP-398 as a potentially best-in-class SHP2 inhibitor with LUMAKRAS, we are hopeful that we will be able to provide substantial relief for cancer patients in need. We will continue to pursue additional collaborations that we believe hold promise for patients.”
KRAS mutations occur in approximately 17% of malignant solid tumors. BBP-398, as a monotherapy or in combination with other targeted therapies, could potentially be a promising therapy for patients with the KRAS G12C mutation.
BridgeBio is currently advancing its Phase 1 clinical trial of its SHP2 inhibitor, BBP-398, in patients with solid tumors driven by mutations in the MAPK signaling pathway, including RAS and receptor tyrosine kinase genes. BBP-398 is part of BridgeBio’s growing precision oncology pipeline and is one of 14 programs in the broader portfolio that are being advanced in the clinic or commercial setting.
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