Acoramidis

- Acoramidis treatment resulted in increased serum transthyretin (TTR) levels by Day 28 that were sustained and were correlated with a reduced risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular-related hospitalization (CVH) in ATTR-CM participants through month 30 - Acoramidis treatment resulted in a significant improvement in the composite endpoint of CVM and CVH in ATTR-CM participants, with benefit evident as early as Month 3 - In ATTRibute-CM, participants with at least one CVH had a significantly higher risk of mortality, highlighting the need for ATTR-CM treatments that reduce risk of CVH - BridgeBio also shared the rationale and design of ACT-EARLY, the acoramidis ATTR amyloidosis prevention trial, which it expects to initiate later this year - BridgeBio will host an investor call on Wednesday, May 29, 2024 at 5:30 pm ET, with presentations from Mathew Maurer, M.D., of Columbia University Irving Medical Center, U.S., and Ahmad Masri, M.D., M.S., of Oregon Health & Science University, U.S. PALO ALTO, Calif., May 29, 2024 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, announced positive results of five new analyses of clinical endpoint events from its Phase 3 ATTRibute-CM study of acoramidis in ATTR-CM. These new analyses were shared in oral presentations and posters at the 2024 International Symposium of Amyloidosis (ISA). ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally administered, highly potent, small molecule stabilizer of TTR. BridgeBio will host an investor call on Wednesday, May 29th at 5:30 pm ET to discuss these results. “The data presented at ISA confirm that improved stabilization as reflected in higher serum TTR levels is directly correlated with improved clinical outcomes. Prior analyses from ATTRibute-CM demonstrated that the near-complete stabilization by acoramidis rapidly and durably increased serum TTR levels. Clinically, we now have evidence that acoramidis-mediated increase in serum TTR independently predicted statistically significant improvement in survival, and risk reduction of CVM and CVH,” said Mathew Maurer M.D. of Columbia University Irving Medical Center, U.S. Three analyses presented from ATTRibute-CM emphasized the correlation between increased serum TTR and improved clinical outcomes, including the reduced risk of both all-cause mortality and cardiovascular death as well as cardiovascular-related hospitalization: Early increase in serum transthyretin level is an independent predictor of improved survival in ATTR cardiomyopathy: Insights from acoramidis Phase 3 study ATTRibute-CM, presented by Mathew Maurer, M.D., Columbia University Irving Medical Center, U.S. Statistically significant correlation between increasing serum TTR and decreasing risk of death: for every 5mg/dL increase in serum TTR level at day 28 after treatment initiation, the risk of death through Month 30 was reduced by 30.9% (by the logistic model) and 26.1% (by the Cox proportional hazards model)Statistical modeling suggests that the acoramidis-mediated increase in serum TTR at Day 28 is an independent predictor of survival Treatment-related early increase in serum TTR is associated with lower cardiovascular mortality in ATTR-CM: Insights from ATTRibute-CM, presented by Nitasha Sarswat, M.D., University of Chicago Medicine, U.S. For each 1 mg/dL increase in serum TTR on day 28 after treatment initiation, there was a 5.5% risk reduction in cardiovascular death observed through Month 30To the company’s knowledge, this is the first prospective demonstration of the relationship between change from baseline in serum TTR and subsequent risk of cardiovascular death in ATTR-CM Acoramidis treatment-related increase in serum TTR is associated with a lower risk of cardiovascular-related hospitalization in ATTR-CM Patients: Insights from the ATTRibute-CM trial, presented by Margot Davis, M.D., Vancouver General Hospital, CA Each 1 mg/dL serum TTR increase at Day 28 after treatment initiation was associated with a 4.7% lower risk of a first cardiovascular hospitalization over 30 monthsThe analysis, the first from a prospective study of the relationship between change from baseline in TTR and subsequent risk of first CVH in ATTR, demonstrated that a greater increase in TTR is significantly associated with a lower risk of CVHThe Kaplan-Meier curves for time to first CVH were also presented; the curves separated early, showing treatment benefit at Month 3 and continuing to separate through Month 30 The results from the analysis highlighting the early reduction in cardiovascular mortality (CVM) or CVH in ATTR in the ATTRibute-CM trial were shared by Kevin M. Alexander, M.D., Stanford University School of Medicine, U.S., in an oral presentation. Key findings included: Acoramidis time-to-first event Kaplan-Meier (K-M) curves for a composite of CVM and CVH in ATTR-CM patients separated beginning at Month 3, representing an early and profound reduction on the composite endpoint of CVM and CVH in ATTR-CM patients, with a 15.2% absolute risk reduction and a 38.2% hazard reduction by Month 30 (p=0.0003). On behalf of the authors, John Whang, M.D., Chief Medical Affairs Officer of BridgeBio Cardiorenal, presented data showing a higher risk of mortality in previously hospitalized participants. Insights included: Participants with no CVH during the study had a 30-month survival rate of 86.7%, vs 60.1% in participants who had at least one CVH during the study To the company’s knowledge, this is the first time a prospective trial demonstrates that CVH portends a higher subsequent mortality in ATTR-CM patientsThis suggests that effective treatments to reduce CVH risk are critically important, and a targeted therapy for ATTR-CM that reduces CVH can improve the prognosis of patients with ATTR-CM The rationale and design of ACT-EARLY, the acoramidis TTR amyloidosis prevention trial, was also presented by Pablo Garcia-Pavia, M.D., Ph.D., Iron Gate Majadahonda University Hospital, ES. ACT-EARLY will be the first Phase 3 trial to evaluate prophylactic therapy for the prevention or delay of ATTR amyloidosis in asymptomatic pathogenic TTR variant carriers with study initiation planned in 2024. Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of BridgeBio Cardiorenal, shared the following: “The totality of acoramidis data across clinical outcomes, biomarkers, quality of life, and cardiac imaging continues to expand with the analyses shared at ISA and the data recently presented at ACC and ESC-HF. We remain encouraged by the potential benefits of targeting near-complete TTR stabilization, the resulting increases in serum TTR, and the corresponding statistically significant benefits on clinical event outcomes. We are committed to bringing acoramidis to the ATTR-CM community as quickly as possible, working toward our November 29th PDUFA date.” Additionally, BridgeBio presented six encore poster presentations on its ATTRibute-CM data, which were previously shared at the European Society of Cardiology Heart Failure (ESC-HF) Congress 2024, the American College of Cardiology (ACC) Annual Scientific Sessions & Expo 2024 and the American Heart Association (AHA) Scientific Sessions 2023. Based on the positive results from ATTRibute-CM, BridgeBio submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration, which has been accepted with a Prescription Drug User Fee Act (PDUFA) action date of November 29, 2024, and a Marketing Authorization Application (MAA) to the European Medicines Agency, with a decision expected in 2025. Webcast InformationBridgeBio will host an investor call and simultaneous webcast to discuss the recent analyses and positive data from the ATTRibute-CM Phase 3 trial and emerging real-world evidence in ATTR-CM presented at the 2024 ISA, ESC Heart Failure 2024 and the 2024 ACC Annual Scientific Sessions & Expo on Wednesday, May 29 at 5:30 pm 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 Forward Looking StatementsThis 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 survival, mortality and hospitalization rates and risks, quality of life, biomarkers and magnetic resonace imaging; potential benefits of acoramidis, including near-complete TTR stabilization, increases in serum TTR, and the corresponding statistically significant benefits on clinical event outcomes; anticipated timeline to bring acoramidis to the ATTR-CM community; 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 potential size of the target patient populations our product candidates are designed to treat not being as large as anticipated, the design and success of ongoing and planned clinical trials, future regulatory filings, approvals and/or sales, the FDA 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. BridgeBio Media Contact:Vikram Balicontact@bridgebio.com (650)-789-8220

The FDA has officially rescinded the accelerated approval granted to BridgeBio Pharma's Truseltiq (infigratinib) in 2021 for previously treated advanced cholangiocarcinoma with an FGFR2 fusion or rearrangement.The 2021 nod was contingent upon the drug's sponsor, BridgeBio affiliate QED Therapeutics, conducting post-marketing trials to verify the clinical benefit observed in the initial single-arm study that had supported the accelerated approval.However, according to an FDA notice dated May 16, QED cited difficulties in recruiting people for the required confirmatory study, which was testing Truseltiq in first-line cholangiocarcinoma. As a result, the sponsor determined it was "not commercially reasonable" to continue distributing the drug for its approved second-line indication.BridgeBio was partnered on the drug with Helsinn, which held rights to Truseltiq in the US for oncology and all other indications other than skeletal dysplasias, where BridgeBio retains full rights. That deal was potentially worth about $2.45 billion in upfront and milestone payments.Achondroplasia hopes remainThe FDA pulling its approval of Truseltiq isn't a surprise given that Helsinn had already decided back in late 2022 to permanently discontinue distribution of Truseltiq and to seek its withdrawal from the US market due to business reasons. LianBio, which has rights to the drug in China and some other Asian markets, said at the time that it intended to close the Phase III PROOF-301 trial that was testing Truseltiq in first-line cholangiocarcinoma.Meanwhile, BridgeBio is evaluating the FGFR1-3 tyrosine kinase inhibitor as a potential treatment for achondroplasia and recently granted exclusive Japanese rights to Kyowa Kirin for $100 million upfront plus unspecified milestones.  Elsewhere, the FDA is expected to make a decision on BridgeBio's TTR stabiliser acoramidis in transthyretin amyloid cardiomyopathy by November 29. Bayer recently gained exclusive rights to commercialise the therapy in Europe pending approval there. BridgeBio also recently spun out its subsidiary into a new company, which launched earlier this month with $200 million to fund some early KRAS-focused cancer programmes.