Beyonttra™ (acoramidis), the First Near-complete TTR Stabilizer (≥90%), Approved in Japan to Treat ATTR-CM

Rhea-AI Summary

BridgeBio Pharma (BBIO) announced the Japanese approval of Beyonttra™ (acoramidis) for treating ATTR-CM (transthyretin-mediated amyloid cardiomyopathy) in adults. The approval stems from successful Phase 3 trials, including a Japanese study reporting 0% mortality over 30 months and a global ATTRibute-CM trial showing significant benefits.

Key clinical achievements include:

• 42% reduction in composite all-cause mortality and recurrent cardiovascular-related hospitalization events at Month 30
• 50% reduction in cumulative cardiovascular-related hospitalization events at Month 30
• Benefits observed as early as 3 months into treatment

Through its partnership with Alexion, AstraZeneca Rare Disease, BridgeBio will receive a $30 million milestone payment and low double-digit royalties on Japanese sales. Commercial launch is planned for first half 2025.

Positive

• First-in-class near-complete TTR stabilizer (≥90%) approved for ATTR-CM
• Exceptional clinical efficacy with 42% reduction in mortality and hospitalization events
• 0% mortality rate in Japanese Phase 3 trial over 30-month period
• $30M milestone payment plus ongoing royalties secured
• Fastest onset of action seen in any Phase 3 ATTR-CM study (3 months)

Negative

• Dependent on Alexion/AstraZeneca for commercialization in Japan
• to low double-digit royalties on sales

Insights

Pharmaceutical Industry Analyst

The approval of Beyonttra (acoramidis) in Japan represents a significant milestone for BridgeBio's ATTR-CM franchise. This marks the second major market approval following the US launch under the Attruby brand name, validating BridgeBio's development strategy and global expansion capabilities.

What stands out in this approval is the compelling efficacy data from both the Japanese and global clinical trials. The 0% mortality rate over 30 months in the Japanese study is remarkable for a fatal progressive disease. Additionally, the global ATTRibute-CM Phase 3 trial demonstrated rapid benefit separation from placebo at just 3 months - the fastest seen in any ATTR-CM Phase 3 study to date - with a 42% reduction in composite all-cause mortality and recurrent cardiovascular-related hospitalization events at 30 months.

The immediate financial impact includes a $30 million milestone payment plus low double-digit royalties on Japanese sales. While BridgeBio won't handle commercialization directly, the partnership with Alexion/AstraZeneca leverages their established rare disease presence in Japan, potentially accelerating market penetration.

Acoramidis's differentiation as the first near-complete (≥90%) TTR stabilizer gives it a potential competitive edge in efficacy. The well-tolerated safety profile should further support adoption. With commercialization expected in the first half of 2025, this approval establishes an important new revenue stream while validating BridgeBio's cardiorenal portfolio and strengthening their position in the growing rare disease therapeutics market.

Clinical Cardiologist

The Japanese approval of Beyonttra (acoramidis) represents a meaningful advancement for ATTR-CM patients. ATTR-CM is a progressive, infiltrative cardiomyopathy with treatment options and poor prognosis without intervention. The data supporting this approval is particularly compelling from a clinical perspective.

The complete absence of mortality during the 30-month Japanese trial is exceptional for this typically fatal condition. The global ATTRibute-CM trial data demonstrates substantial clinical benefits across critical endpoints: 42% reduction in composite all-cause mortality and recurrent cardiovascular hospitalizations, and a 50% reduction in cumulative cardiovascular hospitalizations at 30 months.

What distinguishes acoramidis mechanistically is its near-complete (≥90%) TTR stabilization. Recent literature has established that increased serum TTR concentrations from greater TTR stability correlate with reduced all-cause and cardiovascular mortality risk. This suggests acoramidis's mechanism directly addresses a key pathophysiological driver of disease progression.

The early separation of event curves at just 3 months indicates rapid therapeutic onset - crucial for a progressive disease where intervention timing significantly impacts outcomes. The favorable safety profile, with primarily mild gastrointestinal effects that resolved without discontinuation, suggests acoramidis could be well-positioned for broad clinical adoption across diverse patient populations with ATTR-CM, potentially changing the disease trajectory for affected individuals in Japan.

• 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 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

• 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 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 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 Communications
contact@bridgebio.com
(650)-789-8220

FAQ

What are the key clinical results of Beyonttra (BBIO) in treating ATTR-CM?

Beyonttra showed 42% reduction in mortality and hospitalization events, 50% reduction in hospitalization events at 30 months, and 0% mortality in Japanese trials, with benefits visible within 3 months.

What financial benefits will BBIO receive from Beyonttra's Japanese approval?

BridgeBio will receive a $30 million milestone payment and low double-digit royalties on Japanese sales from Alexion, AstraZeneca Rare Disease.

When will Beyonttra be commercially available in Japan for BBIO?

Commercial launch of Beyonttra in Japan is planned for the first half of 2025.

What makes Beyonttra unique in BBIO's ATTR-CM treatment?

Beyonttra is the first near-complete (≥90%) TTR stabilizer, showing the most rapid benefit in any Phase 3 ATTR-CM study to date.

What were the safety results for BBIO's Beyonttra in clinical trials?

Beyonttra was generally well-tolerated, with most common side effects being mild diarrhea and abdominal pain that resolved without discontinuation.