RARE Daily

Bayer Licenses European Rights to BridgeBio’s Acoramidis in ATTR-CM

March 4, 2024

Rare Daily Staff

BridgeBio Pharma entered into a partnership with Bayer that gives the pharma giant an exclusive license to commercialize acoramidis for ATTR-CM in Europe.

In exchange, BridgeBio will receive up to $310 million in upfront and near-term milestone payments, as well as additional undisclosed sales milestones. BridgeBio will also receive royalties in a tiered structure beginning in the low-thirties percent on sales of acoramidis in Europe.

Transthyretin amyloidosis (ATTR) is an underdiagnosed and life-threatening disease with limited treatment options that can devastate the heart and nervous system. When the transthyretin (TTR) becomes unstable due to inherited variants or aging, it can accumulate as amyloid fibrils in various organs in the body, causing ATTR. TTR amyloid deposits predominantly in the heart and/or peripheral nerves cause cardiomyopathy (ATTR-CM) and/or polyneuropathy (ATTR-PN). ATTR often dramatically impairs the quality of life, functional independence and life expectancy of patients, as well as impacting caregivers due to the progressive nature of the disease. If left untreated, life expectancy from diagnosis is approximately four years.

Acoramidis is an investigational, next-generation, orally-administered, highly potent small molecule stabilizer of transthyretin. The U.S. Food and Drug Administration is expected to decide on BridgeBio’s New Drug Application for acoramidis for the treatment of ATTR-CM by the end of November.  The European Medicines Agency has accepted BridgeBio’s marketing application for acoramidis with potential EU approval in 2025.

“This partnership leverages Bayer’s established European cardiovascular infrastructure and enables us, via substantial cost savings, to focus our resources on our wholly owned geographies for acoramidis, including preparing for the U.S. launch.” said Ananth Sridhar, senior vice president of Corporate Development, BridgeBio Cardiorenal.

In July 2023, BridgeBio announced positive results from ATTRibute-CM, reporting a highly statistically significant result, demonstrated by a Win Ratio of 1.8 on the primary endpoint (a hierarchical analysis prioritizing in order: all-cause mortality, then frequency of cardiovascular hospitalization, then change from baseline in N-terminal prohormone of brain natriuretic peptide, then change from baseline in 6-minute walk distance). Acoramidis was well-tolerated, with no safety signals of potential clinical concern identified. In February 2024, BridgeBio shared positive results of a single-arm phase 3 study of acoramidis in Japanese patients with ATTR-CM, including no mortality reported in the trial at 30 months.

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