PALO ALTO, Calif. — BridgeBio Pharma, Inc. (Nasdaq: BBIO), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today shared positive results from the Japan Phase III trial of acoramidis in adults with transthyretin-mediated amyloid with cardiomyopathy (ATTR-CM), conducted by Alexion, AstraZeneca Rare Disease. Results showed consistency to those in the global BridgeBio ATTRibute-CM Phase III trial (NCT03860935), including survival, cardiovascular-related hospitalizations and other measures of improved functions (measured by six-minute walk test) and quality of life (measured by the Kansas City Cardiomyopathy Questionnaire Overall Summary Score) at 30 months. This trial in Japan was conducted to support local registration.
In this single-arm study where patients were on acoramidis treatment for 30 months, acoramidis was well-tolerated, with no safety signals of potential clinical concern identified, and no mortality was reported. The data will be presented at a forthcoming medical meeting and submitted to Japan’s health authority for regulatory review.
Alexion, AstraZeneca Rare Disease, maintains an exclusive license with BridgeBio’s affiliate, Eidos Therapeutics, Inc. to develop and commercialize acoramidis in Japan. Acoramidis is an investigational, next-generation, oral, highly potent small molecule stabilizer of transthyretin (TTR), designed to achieve maximal stabilization and preserve native TTR.
Highlights from the Phase 3 Study
– Phase 3 open-label, single-arm study conducted in Japan by BridgeBio licensing partner Alexion, AstraZeneca Rare Disease showed consistency with global ATTRibute-CM Phase III trial
– No mortality was reported over the 30 month acoramidis treatment period
– Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified
– These results support local regulatory submission in Japan
– The findings from this study build upon positive results from BridgeBio’s global ATTRibute-CM Phase 3 trial, where the primary endpoint was met (Win Ratio of 1.8) with a highly statistically significant p-value (p<0.0001) and an 81% survival rate at 30 months and a 0.29 annualized cardiovascular hospitalization rate were observed on acoramidis treatment
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.
Contact:
Vikram Bali
[email protected]
(650)-789-8220