Positive Phase 3 Acoramidis Trial Outcomes in Japanese ATTR-CM Patients with Zero Mortality at 30 Months

A recent Phase III clinical trial in Japan for acoramidis, a medication developed to treat transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), has reported promising results. The study, which was an open-label, single-arm investigation, was executed by Alexion, AstraZeneca Rare Disease, and was found to align with the global ATTRibute-CM Phase III trial outcomes.

Over a 30-month period, the treatment demonstrated a favorable safety profile with no reported deaths. The study's findings are set to be presented at an upcoming medical conference and will be submitted to Japanese health authorities for regulatory review. The results are expected to support regulatory submissions in Japan.

Acoramidis, an oral, potent small molecule stabilizer of transthyretin (TTR), is designed to achieve maximum stabilization and preserve the native form of TTR. The medication was well-tolerated by patients throughout the study, with no safety concerns raised. The findings from the Japanese trial complement the positive results from BridgeBio’s global ATTRibute-CM Phase 3 trial, where acoramidis showed a significant survival rate and a reduced annualized cardiovascular hospitalization rate.

BridgeBio Pharma, a biopharmaceutical company dedicated to genetic diseases and cancers, has partnered with Eidos Therapeutics, which holds an exclusive license to develop and commercialize acoramidis in Japan. BridgeBio was established in 2015 with the aim of rapidly developing transformative medicines based on genetic advances.

The Phase III trial in Japan was a crucial step towards supporting local registration of acoramidis. The study measured various parameters, including survival, cardiovascular hospitalizations, functional improvement, and quality of life. The positive results from this trial, along with the global ATTRibute-CM Phase III trial, indicate a strong potential for acoramidis as a treatment for ATTR-CM.

BridgeBio's commitment to genetic medicine is evident through their extensive pipeline of development programs, which range from early-stage research to advanced clinical trials. The team at BridgeBio is dedicated to applying the latest in genetic medicine to provide effective treatments for patients in need.