Source – BridgeBio
BridgeBio is preparing to seek FDA approval for its acoramidis drug to treat transthyretin amyloidosis (ATTR) cardiomyopathy, following positive results from a Phase III trial reported recently.
The biotech’s shares surged approximately 75% after announcing that acoramidis outperformed the placebo in the ATTRibute-CM trial, showing a statistically significant improvement in the primary endpoint. This endpoint is a combination of factors such as all-cause mortality, cardiovascular-related hospitalization, changes in a biomarker called NT-proBNP, and changes in a 6-minute walk test.
Acoramidis demonstrated a “Win Ratio” 1.8 times better than placebo on the primary endpoint, indicating substantial efficacy. Additionally, patients taking the drug had a 25% lower likelihood of dying during the treatment phase compared to the control group. The frequency of cardiovascular-related hospitalizations also saw a significant relative risk reduction of 50%, leading to “consistent and clinically meaningful benefits” of this orally-active transthyretin (TTR) stabilizer drug.
This outcome represents a significant turnaround from a previous readout in late 2021 when acoramidis performed worse than placebo on the 6-minute walk test at 12 months, causing a decline in BridgeBio’s shares.
The only drawback in the dataset was the drug’s failure to improve all-cause mortality on its own, although the trial was not designed to provide a conclusive result for each individual component of the Win Ratio.
ATTR is a condition characterized by the formation of amyloid fibril deposits in tissues and organs, particularly affecting the heart in patients with cardiomyopathy.
BridgeBio aims to submit acoramidis for FDA approval before the end of the year and for European approval in 2024, with expectations of blockbuster sales if it reaches the market.
If approved, acoramidis will compete with Pfizer’s Vyndaqel/Vyndamax (tafamidis), which operates in a similar way and obtained FDA approval for ATTR cardiomyopathy in 2019, generating approximately $2.5 billion in global sales last year.
Currently, Vyndaqel is the only FDA-approved therapy for ATTR cardiomyopathy, although there are several drugs available for another form of the disease called ATTR polyneuropathy, including Alnylam’s Ambattur (vutrisiran) and Onpattro (patisiran), as well as Ionis’ Tegsedi (inotersen).
Alnylam is also developing Onpattro for ATTR cardiomyopathy, making it a potential competitor to BridgeBio, and has already filed for FDA approval for this indication, with a decision expected in October. Meanwhile, BridgeBio is also conducting late-stage trials for acoramidis in ATTR polyneuropathy.
