Cardiomyopathy, Restrictive

ATTR-CM is a fatal condition that leads to restrictive cardiomyopathy and heart failure. Credit: PanuShot/Shutterstock. The UK Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorisation for BridgeBio Pharma’s acoramidis under the name Beyonttra, for treating wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Acoramidis is an orally administered selective small molecule that stabilises transthyretin (TTR) by more than 90%. This authorisation was supported by the outcomes from the pivotal ATTRibute-CM Phase III trial, which demonstrated cardiovascular benefits. In the trial, the therapy’s safety and efficacy were assessed in 632 symptomatic ATTR-CM subjects, and they were given either the therapy or a placebo for a 30-month period. The primary clinical endpoints were met at month 30, showing a decrease in cardiovascular-related hospitalisations, preservation of functional capacity, improved survival and quality of life for subjects. The therapy received the US Food and Drug Administration’s (FDA) approval under the name Attruby in November 2024, and as Beyonttra by the European Commission (EC) in February 2025. GlobalData Strategic Intelligence US Tariffs are shifting - will you react or anticipate? Don’t let policy changes catch you off guard. Stay proactive with real-time data and expert analysis. By GlobalData Learn more about Strategic Intelligence It was also approved in Japan by the Ministry of Health, Labour, and Welfare in March 2025. In the UK, Bayer will manage all commercial activities for the therapy. In March 2024, the company and Bayer entered a partnership granting the latter exclusive rights to commercialise the therapy in Europe. According to the licencing agreement, BridgeBio will obtain tiered royalties starting in the low-thirties percent on UK sales after commercialisation efforts commence. ATTR-CM is a fatal condition that causes heart failure due to restrictive cardiomyopathy. BridgeBio Cardiorenal chief medical officer and president Jonathan Fox stated: “We are proud to add another approval for acoramidis and thrilled that patients in the UK will now have access to Beyonttra since they are in great need of new disease-modifying treatments for their condition. “We look forward to extending our collaboration with our European partner, Bayer, to serve ATTR-CM patients across the UK and the rest of Europe, and will continue to work towards reaching patients in as many regions as possible around the world.”

- The UK approval is based on positive results from the Phase 3 ATTRibute-CM study, in which acoramidis demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date - In as few as 3 months, the time to first event (all-cause mortality (ACM) or cardiovascular-related hospitalization (CVH)) durably separated relative to placebo- A 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30- A 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30 - Acoramidis is the first and only approved ATTR-CM treatment in the U.S., EU, UK and Japan that all have a label specifying near-complete stabilization (≥90%) - Relative increases in serum TTR concentrations resulting from greater TTR stability have been associated with reduced risk of all-cause and cardiovascular mortality in the general population in recent literature1 - BridgeBio will receive royalties in a tiered structure beginning in the low-thirties percent on sales of Beyonttra in the UK PALO ALTO, Calif., April 28, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced the Medicines and Healthcare products Regulatory Agency has granted marketing authorization in the United Kingdom (UK) for acoramidis, under the brand name BEYONTTRA®, for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). Acoramidis is a selective small molecule, orally administered near-complete (≥90%) transthyretin (TTR) stabilizer. ATTR-CM is a progressive fatal disease that presents as an infiltrative, restrictive cardiomyopathy resulting in heart failure. Bayer will be responsible for all commercial activity for acoramidis in the UK. “ATTR-CM is a progressive and debilitating disease that poses significant challenges not only for patients but also for the healthcare systems. The condition profoundly impacts patients’ quality of life. Symptoms attributable to amyloidosis are usually nonspecific, varied and associated with low awareness, frequently resulting in delayed or completely missed diagnosis,2 which may lead to delayed treatment and a worse prognosis. In the absence of intervention, ATTR-CM causes progressive heart failure leading to increased hospitalizations and escalating healthcare costs and is ultimately fatal,3-5” said Julian Gillmore, M.D., Ph.D., University College London's Centre for Amyloidosis, UK. “The UK authorization of Beyonttra is welcome news for eligible patients living with the condition. Physicians in the UK now have another treatment option to slow the progression of symptoms and improve outcomes for patients with ATTR-CM.” The approval in the UK is based on results of the pivotal ATTRibute-CM Phase 3 study of acoramidis, which showed clear benefits on cardiovascular outcomes. ATTRibute-CM evaluated the efficacy and safety of acoramidis in 632 participants with symptomatic ATTR-CM, associated with either wild-type or variant TTR who were randomized 2:1 to receive acoramidis or placebo for 30 months. The study met its primary clinical endpoints at month 30 by significantly reducing cardiovascular-related hospitalization, improving survival, and preserving functional capacity and quality of life for patients. “We are proud to add another approval for acoramidis and thrilled that patients in the UK will now have access to BEYONTTRA since they are in great need of new disease-modifying treatments for their condition,” said Jonathan Fox, M.D., Ph.D., President and Chief Medical Officer of BridgeBio Cardiorenal. “We appreciate the time and commitment of every clinical trial participant and their families, and the dedicated support of the physicians and scientists involved in the clinical program. This important milestone would not have been possible without their commitment to the program. We look forward to extending our collaboration with our European partner, Bayer, to serve ATTR-CM patients across the UK and the rest of Europe, and will continue to work towards reaching patients in as many regions as possible around the world." Acoramidis was approved as Attruby™ by the U.S. FDA in November 2024 and was approved as BEYONTTRA by the European Commission in February 2025 and the Japanese Ministry of Health, Labour, and Welfare (MHLW) Agency in March 2025 with all labels specifying near-complete stabilization of TTR. In March 2024, BridgeBio and Bayer initiated a collaboration for acoramidis, which granted Bayer exclusive commercialization rights in Europe. Based on terms of the licensing agreement, BridgeBio will receive royalties in a tiered structure beginning in the low-thirties percent on sales of acoramidis in the UK following initiation of commercialization efforts. 1Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102.2Rintell et al. Orphanet J Rare Dis. (2021) 16:70. https://doi.org/10.1186/s13023-021-01706-7 3Rozenbaum MH, et al. Impact of delayed diagnosis and misdiagnosis for patients with transthyretin amyloid cardiomyopathy (ATTR-CM): a targeted literature review. Cardiology and therapy. 2021;10:141-59.4Mallus MT and Rizzello V. Treatment of amyloidosis: present and future. 2023;21;25(Suppl B):B99-B103.5Jain A, Zahra F. Transthyretin Amyloid Cardiomyopathy (ATTR-CM). Updated 27 April 2023. In: StatPearls [Internet]. Treasure Island (FL): StatPearls Publishing; 2024 Jan-. Available from: https://www.ncbi.nlm.nih.gov/books/NBK574531/ Last accessed: March 2025. About BEYONTTRABEYONTTRA is an orally administered near-complete (≥90%) stabilizer of transthyretin (TTR) indicated for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM). For full prescribing information, please refer to the Summary of Product Characteristics (SmPC) on the Medicines and Healthcare products Regulatory Agency website at https://products.mhra.gov.uk/. About Attruby™ (acoramidis)INDICATIONAttruby is a transthyretin stabilizer indicated for the treatment of the cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular death and cardiovascular-related hospitalization. IMPORTANT SAFETY INFORMATIONAdverse ReactionsDiarrhea (11.6% vs 7.6%) and upper abdominal pain (5.5% vs 1.4%) were reported in patients treated with Attruby versus placebo, respectively. The majority of these adverse reactions were mild and resolved without drug discontinuation. Discontinuation rates due to adverse events were similar between patients treated with Attruby versus placebo (9.3% and 8.5%, respectively). ▼: This medicine is subject to additional monitoring. This will allow quick identification of new safety information. About BridgeBio 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 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. BridgeBio intends 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 Beyonttra on clinical outcomes; and the potential benefits of Beyonttra, including its ability to reduce cardiovascular-related hospitalization, improve survival, and preserve functional capacity and quality of life, reflect BridgeBio’s current views about its plans, intentions, expectations, and strategies, which are based on the information currently available to BridgeBio and on assumptions BridgeBio has made. Although BridgeBio believes that its plans, intentions, expectations, and strategies, as reflected in or suggested by these forward-looking statements, are reasonable, BridgeBio 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, the risks associated with BridgeBio’s dependence on third parties for development, manufacture, and commercialization activities related to Beyonttra; government and third-party payor actions; risks and uncertainties relating to competitive products and other changes that may limit demand for Beyonttra; the risk that regulatory authorities may require additional studies or data to support the continued commercialization of Beyonttra; the risk that drug-related adverse events may be observed during commercialization or clinical development; the risk that data and results may not meet regulatory requirements or otherwise be sufficient for further development, regulatory review, or approval; the risk of other regulatory agencies not agreeing with BridgeBio’s regulatory approval strategies, components of BridgeBio’s filings (such as clinical trial designs, conduct, and methodologies), or the sufficiency of data submitted; the continuing success of its collaborations, including compliance with applicable regulations for the purchase, distribution, storage, export, and sale of active pharmaceutical ingredients and medicinal products; 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; and increasing rates of inflation and changing interest rates on BridgeBio’s business operations and expectations. These risks, as well as those set forth in the Risk Factors section of BridgeBio’s most recent Annual Report on Form 10-K and its other filings with the U.S. Securities and Exchange Commission, should be carefully considered. Moreover, BridgeBio operates in a highly 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 BridgeBio’s 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, BridgeBio assumes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events, or otherwise. BridgeBio Media Contact:Bubba Murarka, EVP Communicationscontact@bridgebio.com(650)-789-8220 Chinmay Shukla, VP Strategic Financeir@bridgebio.com

In the Japanese Phase 3 study, 0% mortality was reported over the 30-month treatment period and acoramidis was well-tolerated The approval was based on a Japanese Phase 3 study and the global ATTRibute-CM Phase 3 trial, which demonstrated the most rapid benefit seen in any Phase 3 study of ATTR-CM to date. Key data from the ATTRibute-CM study include: In as few as 3 months, the time to first event (all-cause mortality (ACM) or cardiovascular-related hospitalization (CVH)) durably separated relative to placeboA 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30A 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30 BridgeBio will receive a $30 million milestone payment from Alexion, AstraZeneca Rare Disease, with royalties in the low double digits on net sales of Beyonttra in Japan PALO ALTO, Calif., March 27, 2025 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (“BridgeBio” or the “Company”), a new type of biopharmaceutical company focused on genetic diseases, today announced the Japanese Ministry of Health, Labour and Welfare has approved acoramidis, under the brand name Beyonttra, for the treatment of adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). Acoramidis is a selective small molecule, orally administered, near-complete (≥90%) transthyretin (TTR) stabilizer. Relative increases in serum TTR concentrations resulting from greater TTR stability have been associated with reduced risk of all-cause and cardiovascular mortality in the general population in recent literature.1 ATTR-CM is a progressive, fatal disease that presents as an infiltrative, restrictive cardiomyopathy resulting in heart failure. Alexion, AstraZeneca Rare Disease will be responsible for all commercial activity for Beyonttra in Japan. “There is significant need for new treatment options for ATTR-CM, a progressive, fatal disease, worldwide. This latest approval marks an important development for the ATTR-CM community, and we are pleased that patients living with this condition in Japan now have a new treatment option that offers early and sustained reductions in cardiovascular events,” said Dr. Jonathan Fox, BridgeBio Cardiorenal Chief Medical Officer. The approval in Japan is based on positive results from a Phase 3 open-label, single-arm study conducted in Japan by Alexion, AstraZeneca Rare Disease, and the positive results from the global ATTRibute-CM Phase 3 study. In the Japanese trial, acoramidis was generally well tolerated and 0% mortality was reported during the 30-month acoramidis treatment period. BridgeBio’s global ATTRibute-CM Phase 3 trial showed early separation at 3 months in time to first event (ACM or CVH) durably separated relative to placebo. Furthermore, within the global ATTRibute-CM trial acoramidis led to a 42% reduction in composite ACM and recurrent CVH events relative to placebo at Month 30, as well as a 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30. Alexion, AstraZeneca Rare Disease, maintains an exclusive license with BridgeBio’s affiliate, Eidos Therapeutics, Inc. to develop and commercialize acoramidis in Japan. Based on the terms of the agreement, BridgeBio will receive a $30 million milestone payment upon approval in Japan, as well as royalties in the low double digits on sales of acoramidis in Japan, with commercialization efforts planned in the first half of 2025. 1Christoffersen M et al. Transthyretin Tetramer Destabilization and Increased Mortality in the General Population. JAMA Cardiol. 2024 Dec 4:e244102. Beyonttra (acoramidis) Beyonttra is an orally administered near-complete (≥90%) stabilizer of transthyretin (TTR) indicated for the treatment of wild-type or variant transthyretin amyloid cardiomyopathy (ATTR-CM) in adults. Beyonttra was generally well-tolerated. Please refer the Japanese Prescribing Information as made available by the PMDA for Japanese specific safety labelling. In the US prescribing information, where acoramidis is approved under the brand name Attruby™, the most common side effects were mild and included diarrhea and abdominal pain that were resolved without drug discontinuation. Alexion maintains an exclusive license with BridgeBio’s affiliate, Eidos Therapeutics, Inc., to develop and commercialize Beyonttra in Japan. About BridgeBio Pharma, Inc.BridgeBio Pharma, Inc. (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 Pharma, Inc. 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. BridgeBio intends 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 Beyonttra on clinical outcomes; and the potential benefits of Beyonttra, including its efficacy and potential to reduce ACM or CVH events, reflect BridgeBio’s current views about its plans, intentions, expectations, and strategies, which are based on the information currently available to BridgeBio and on assumptions BridgeBio has made. Although BridgeBio believes that its plans, intentions, expectations, and strategies, as reflected in or suggested by these forward-looking statements, are reasonable, BridgeBio 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, the risks associated with BridgeBio’s dependence on third parties for development, manufacture, and commercialization activities related to Beyonttra; government and third-party payor actions; risks and uncertainties relating to competitive products and other changes that may limit demand for Beyonttra; the risk that regulatory authorities may require additional studies or data to support the continued commercialization of Beyonttra; the risk that drug-related adverse events may be observed during commercialization or clinical development; the risk that data and results may not meet regulatory requirements or otherwise be sufficient for further development, regulatory review, or approval; the risk of other regulatory agencies not agreeing with BridgeBio’s regulatory approval strategies, components of BridgeBio’s filings (such as clinical trial designs, conduct, and methodologies), or the sufficiency of data submitted; the continuing success of its collaborations, including compliance with applicable regulations for the purchase, distribution, storage, export, and sale of active pharmaceutical ingredients and medicinal products; 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; and increasing rates of inflation and changing interest rates on BridgeBio’s business operations and expectations. These risks, as well as those set forth in the Risk Factors section of BridgeBio’s most recent Annual Report on Form 10-K and its other filings with the U.S. Securities and Exchange Commission, should be carefully considered. Moreover, BridgeBio operates in a highly 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 BridgeBio’s 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, BridgeBio assumes no obligation to publicly update any forward-looking statements, whether as a result of new information, future events, or otherwise. BridgeBio Media Contact:Bubba Murarka, EVP Communicationscontact@bridgebio.com (650)-789-8220