Acoramidis

PALO ALTO, Calif., Nov. 18, 2024 (GLOBE NEWSWIRE) — BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, presented positive initial outcomes from the ATTRibute-CM open-label extension (OLE) study of acoramidis in ATTR-CM at the American Heart Association (AHA) Scientific Sessions. ATTRibute-CM was designed to evaluate the efficacy and safety of acoramidis, an investigational, near-complete, orally-administered, small molecule stabilizer of TTR. The preliminary results from this ongoing OLE study were also simultaneously published in Circulation . The OLE study involves 389 participants who completed the 30-month ATTRibute-CM Phase 3 study. “Results from the ATTRibute-CM OLE continue to showcase the potential of acoramidis, with ongoing data across the study suggesting that early intervention with this stabilizer leads to early separation from placebo, with sustained benefit for patients with ATTR-CM,” said Daniel Judge, M.D., professor of medicine and cardiology at the Medical University of South Carolina. “The prescribing community is eager to have another important treatment option given the remaining high unmet need for ATTR-CM patients.” Key initial results from the OLE study, presented by Dr. Judge at AHA, show that continuous treatment with acoramidis led to: The OLE data build on previously reported results from ATTRibute-CM in which acoramidis demonstrated clinically important treatment effects on mortality, CVH, and quality of life, further supporting that greater transthyretin (TTR) stabilization can improve clinical outcomes for patients. This included a 50% reduction in the cumulative frequency of CVH relative to placebo at Month 30. “We are pleased to share the initial results from the ongoing open-label extension study of ATTRibute-CM, which showcase the sustained benefits of acoramidis treatment for patients with ATTR-CM,” said Jonathan Fox, M.D., Ph.D., chief medical officer of BridgeBio Cardiorenal. “Coupled with acoramidis’ earliest time to separation of any known ATTR-CM treatment on clinical outcomes at 3 months, these analyses continue to support acoramidis as a meaningful first line option.” In addition to the featured science oral presentation at AHA, BridgeBio also shared three moderated posters: These findings reinforce the importance of an effective therapy that reduces CVH and improves survival in patients with ATTR-CM. Based on the positive results from ATTRibute-CM, BridgeBio submitted a New Drug Application to the U.S. Food and Drug Administration, which has been accepted with a PDUFA action date of November 29, 2024, and a Marketing Authorization Application to the European Medicines Agency, with a decision expected in 2025. BridgeBio has granted exclusive rights to Bayer to commercialize acoramidis for ATTR-CM in Europe. About BridgeBio Pharma, Inc. BridgeBio Pharma (BridgeBio) is a new type of biopharmaceutical company founded to discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases. 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, Twitter and Facebook. BridgeBio Forward Looking Statements This press release contains forward-looking statements. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as “anticipates,” “believes,” “continues,” “estimates,” “expects,” “hopes,” “intends,” “may,” “plans,” “projects,” “remains,” “seeks,” “should,” “will,” and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Exchange Act. These forward-looking statements, including statements relating to the impact of acoramidis on clinical outcomes, including the reduction of ACM and CVH, TTR stabilization and the benefits of early intervention; potential benefits of acoramidis; the statements related to the planned actions and decisions of the U.S. Food and Drug Administration and the European Medicines Agency regarding our New Drug Application and Marketing Authorization Application submissions for acoramidis for the treatment of ATTR-CM; and the potential outcomes and expected timing of regulatory reviews by the U.S. Food and Drug Administration and the European Medicines Agency, and the corresponding statistically significant benefits on clinical event outcomes; and the clinical, therapeutic and market potential of our clinical development program and timeline for acoramidis reflect our current views about our plans, intentions, expectations and strategies, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations and strategies as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a number of risks, uncertainties and assumptions, including, but not limited to, initial and ongoing data from our preclinical studies and clinical trials not being indicative of final data, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, the U.S. Food and Drug Administration or such other regulatory agencies not agreeing with our regulatory approval strategies, components of our filings, such as clinical trial designs, conduct and methodologies, or the sufficiency of data submitted, the continuing success of our collaborations, potential volatility in our share price, uncertainty regarding any impacts due to global health emergencies, including delays in regulatory review, manufacturing and supply chain interruptions, adverse effects on healthcare systems and disruption of the global economy, the impacts of current macroeconomic and geopolitical events, including changing conditions from hostilities in Ukraine and in Israel and the Gaza Strip, increasing rates of inflation and rising interest rates, on our business operations and expectations, as well as those risks set forth in the Risk Factors section of our most recent Annual Report on Form 10-K and our other filings with the U.S. Securities and Exchange Commission. Moreover, we operate in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of our management as of the date of this press release, and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. Except as required by applicable law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise. Source: BridgeBio Pharma, Inc. Acoramidis FDA Approval History Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

In one of the most closely-watched arenas in biotech, Intellia says its gene editing treatment for a heart muscle disease can stabilize or improve outcomes where other treatments have only been shown to slow the disease’s progression. But results are from a small, early-stage study with no comparison group and must be replicated in a larger Phase 3 trial. On Saturday, the Cambridge, MA-based company presented the first clinical outcomes from 36 patients in a Phase 1 study of its gene editing treatment nex-z for ATTR amyloidosis with cardiomyopathy, a disease in which misfolded proteins build up in the heart. On the six-minute walk test, a common tool used in heart and lung disease trials, patients who received nex-z saw a median 5-meter improvement one year after treatment. In addition, Intellia reported that 92% of the patients saw either improvement or no change in their New York Heart Association class. Intellia CEO John Leonard highlighted that the patients enrolled in the Phase 1 study saw disease stasis — or, in some cases, improvement — despite how a greater proportion of patients had more advanced disease compared to key studies of other therapies that have been shown to slow down the progression of ATTR cardiomyopathy. That includes Alnylam’s pivotal trial readout earlier this year of an RNA silencing drug for ATTR cardiomyopathy, which was dubbed by some to be the biggest biotech readout of 2024. “This is encouraging, but it does not yet rise to a level where we would say, ‘OK, there is a very clear signal that we are actually improving how patients are doing.’ That’s because of the open-label nature, the small sample size,” said Ahmad Masri, director of the Hypertrophic Cardiomyopathy Center at Oregon Health & Science University. “Nonetheless, it is very exciting,” he added. Masri was not involved with the Phase 1 study, but is an investigator on a Phase 3 study that’s currently ongoing. The Phase 1 outcomes were presented at the American Heart Association scientific meeting on Saturday and concurrently published in the New England Journal of Medicine . Intellia is now running a much larger 765-patient Phase 3 study called MAGNITUDE comparing nex-z to placebo in ATTR cardiomyopathy. “This is, in a way, the first time ever we have a treatment that can suppress TTR to this degree. Whether this extra suppression of TTR is adding value or not — that’s where the question is, and that’s what the MAGNITUDE trial hopefully can answer,” Masri said. At one year, the mean TTR reduction in the Phase 1 study was 90%. Intellia’s treatment nex-z is designed to use gene editing to inactivate the gene that encodes for a protein called transthyretin, which is behind the buildups that accumulate in the heart. Currently, Pfizer’s tafamidis, which is marketed as Vyndamax or Vyndaqel, is the only drug approved to treat ATTR cardiomyopathy and last year generated $3.3 billion in sales. But two new drugs are expected to reach the market soon — BridgeBio’s acoramidis and Alnylam’s vutrisiran. AstraZeneca and partner Ionis are also running a Phase 3 study of an experimental treatment for the heart muscle disease. Intellia also shared additional data on nex-z in the form of the disease that causes nerve damage, called ATTR polyneuropathy. In 33 patients who received a dose of 0.3 mg/kg or higher, the mean TTR reduction was 91%. Intellia likewise pointed to early trends in clinical measures that suggested the disease stabilized or improved in a small group of patients. Intellia is developing nex-z as part of a larger collaboration with Regeneron.