Acoramidis shows promise in ATTR-CM Phase 3 trial

PALO ALTO, Calif. - BridgeBio Pharma, Inc. (NASDAQ:BBIO), a $6.68 billion market cap biotech company with impressive revenue growth of over 2,000% in the last twelve months according to InvestingPro, announced significant clinical advancements for its drug acoramidis, based on a pre-specified subgroup analysis of the Phase 3 ATTRibute-CM trial. The drug demonstrated a statistically significant benefit in patients with transthyretin amyloid cardiomyopathy (ATTR-CM), both variant (ATTRv) and wild-type (ATTRwt), leading to a reduction in all-cause mortality (ACM) and first cardiovascular-related hospitalization (CVH).

In the ATTRv-CM patient subgroup, acoramidis achieved a 59% hazard reduction for the composite of ACM and CVH at Month 30 compared to placebo. This level of efficacy is notable, given the poor prognosis typically associated with this patient group, who often present with the condition at an earlier age and experience a more aggressive disease progression.

ATTR-CM patients treated with acoramidis exhibited rapid and sustained increases in serum transthyretin (TTR) levels, reflecting the drug’s ability to achieve near-complete (≥90%) stabilization of TTR. This stabilization is considered responsible for the observed clinical benefits, which included a 42% reduction in composite ACM and recurrent CVH events and a 50% reduction in the cumulative frequency of CVH events relative to placebo at Month 30.

The findings from the ATTRibute-CM trial were presented at the American College of Cardiology Annual Scientific Sessions & Expo by Dr. Margot Davis of Vancouver General Hospital, Canada. Additional data from the study highlighted improvements in New York Heart Association (NYHA) Class for patients with ATTR-CM, indicating better stabilization of heart failure symptoms and functional status.

Acoramidis, marketed as Attruby™ in the U.S., BEYONTTRA™ in Europe, and by the Japanese Pharmaceuticals and Medical Devices Agency, is the first near-complete stabilizer of TTR approved for the treatment of ATTR-CM in adults to reduce cardiovascular death and cardiovascular-related hospitalization. The drug was generally well-tolerated, with the most common side effects being mild, such as diarrhea and abdominal pain.

BridgeBio Pharma, focused on genetic diseases, emphasizes the potential of acoramidis to address the significant unmet need for patients with ATTRv-CM. More data on the benefit of acoramidis for ATTRv-CM patients is expected to be presented at future medical meetings.

This article is based on a press release statement from BridgeBio Pharma. With analyst price targets ranging from $36 to $95 per share and a consensus "Buy" rating according to InvestingPro, the market appears optimistic about the company’s prospects. Subscribers to InvestingPro can access the comprehensive Pro Research Report for BBIO, which includes detailed analysis of the company’s financial health, valuation metrics, and growth prospects among 1,400+ top stocks.

In other recent news, BridgeBio Pharma has received approval from the Japanese Ministry of Health, Labour and Welfare for its treatment, Beyonttra, targeting transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). This approval follows successful Phase 3 trials, which demonstrated significant reductions in cardiovascular-related hospitalizations and mortality. Furthermore, BridgeBio will receive a $30 million milestone payment from Alexion, AstraZeneca Rare Disease, with plans for a commercial launch in Japan in early 2025. Additionally, BridgeBio announced a proposed offering of $500 million in convertible senior notes to refinance existing debt, aiming to reduce interest expenses and increase operational flexibility.

In analyst coverage, Redburn-Atlantic initiated a Buy rating on BridgeBio with a $50 price target, citing the rapid progress of the company’s Attruby rollout. Cantor Fitzgerald also maintained an Overweight rating with a $95 price target, focusing on BridgeBio’s Medicare drug coverage dynamics. These developments underscore the company’s strategic financial maneuvers and growing product pipeline.

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