BridgeBio touts acoramidis study win in Japan as FDA review looms
02 Feb 2024
Phase 3Clinical ResultLicense out/in
BridgeBio Pharma announced positive 30-month results from a Phase III study evaluating acoramidis in Japanese patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM)transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). The company said the findings build on outcomes from the global Phase III ATTRibute-CM trial, paving the way for a regulatory submission in Japan.
The investigational oral therapy, which is designed to stabilise transthyretin (TTR) by mimicking the structure of the naturally protective T119M variant, is seen as a potential rival to Pfizer's Vyndamax (tafamidis).
Commercial launch prep
Acoramidis was recently submitted for review at the FDA based on ATTRibute-CM data where the treatment demonstrated an 81% absolute survival rate and a mean annual frequency of 0.29 for cardiovascular-related hospitalisations (CVH).
BridgeBio says it has been fielding "a lot of interest" for acoramidis partnerships, but that its priority is to retain US rights, according to a recent note by Jefferies. The analysts predict the FDA will likely opt for a standard review, with a potential approval towards the end of November. If US regulators clear the drug, BridgeBio is set to receive a $500-million milestone payment to help support a commercial launch, based on a recent royalty agreement with Blue Owl and CPP Investments.
Jefferies also noted that BridgeBio expects about 100 to 140 sales reps to commercialise acoramidis in the US, whereas Pfizer does not have a dedicated sales team for Vyndamax in ATTR-CM, relying instead on a hybrid of contract primary care reps with an internal sales force.
No mortality over 30 months
BridgeBio said results from the single-arm Japanese study "showed consistency" to those in ATTRibute-CM with respect to survival, CVH and other measures such as six-minute walk test and quality-of-life scores at 30 months. No deaths were reported in the trial over that time.
Acoramidis was also well-tolerated, with "no safety signals of potential clinical concern," the company added. More data will be presented at a forthcoming medical meeting and submitted to Japan's health authority for review.
AstraZeneca's Alexion rare diseases unit, which conducted the trial, maintains an exclusive license with BridgeBio affiliate Eidos Therapeutics to develop and commercialise acoramidis in Japan.
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