BridgeBio Pharma’s Acoramidis Phase 3 trial in ATTR-CM hits primary endpoint

BridgeBio Pharma, Inc. (Nasdaq: BBIO), a  commercial-stage biopharmaceutical company specializing in genetic diseases and cancers, announced that the Phase 3 ATTRibute-CM study of acoramidis has met its primary endpoint. Presented by Julian Gillmore at the European Society of Cardiology Congress 2023, the study confirmed the significant efficacy and safety of acoramidis for treating patients with ATTR-CM (transthyretin amyloid cardiomyopathy).

Acoramidis Proves Highly Effective in ATTR-CM Treatment

The ATTRibute-CM study showed a highly statistically significant result on its primary endpoint, with a Win Ratio of 1.8 (p<0.0001). This primary endpoint includes a hierarchical analysis that prioritizes various components such as ACM, frequency of CVH, and changes from baseline in NT-proBNP and 6MWD. Dr. Julian Gillmore stated, “These results of acoramidis in ATTR cardiomyopathy are not only highly significant for the primary endpoint but also demonstrate a consistent treatment benefit across both primary and key secondary endpoints.”

Survival Rates and Other Key Outcomes

Other key outcomes include an 81% survival rate for acoramidis-treated patients compared to a 74% survival rate for the placebo group. This promising result was consistent across variant and wild-type ATTR-CM patients, as well as NYHA Class I, II, and III patients. Dr. Jonathan Fox, President, and Chief Medical Officer of BridgeBio Cardiorenal, highlighted that these survival rates are nearing those of the age-matched US population, substantiating the efficacy of acoramidis in ATTR-CM treatment.

Statistically Significant Results Across Secondary Endpoints

Notably, the treatment group achieved a 14.9% CV-related mortality rate compared to 21.3% in the placebo group. Other secondary endpoints like NT-proBNP and 6MWD also showed a highly statistically significant treatment effect (p<0.0001 in each case). These compelling results suggest that acoramidis offers a potential breakthrough for patients afflicted with ATTR-CM.

Acoramidis: A Safe and Promising New Treatment

The study also confirmed acoramidis to be well-tolerated with no safety concerns. “Acoramidis has the potential to provide a new and attractive treatment option for ATTR-CM patients and healthcare providers,” added Dr. Gillmore.

Regulatory Filings and IP Protection

BridgeBio Pharma aims to submit its New Drug Application (NDA) to the US FDA by the end of 2023, with plans for regulatory filings in additional markets in 2024. Acoramidis boasts intellectual property protection until at least 2039, securing its position in the market for years to come.

With these positive results, acoramidis is poised to become a cornerstone in ATTR-CM treatment, offering new hope for patients and healthcare providers alike.