BridgeBio Pharma, Inc. shared positive results from the Japan Phase III trial of acoramidis in adults with transthyretin-mediated amyloid 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.