- 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. — 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.
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