RARE Daily

BridgeBio Shares Soar on Positive Results from Phase 3 Study of Acoramidis for Patients with ATTR-CM

July 17, 2023

Rare Daily Staff

BridgeBio Pharma’s shares soared 66 percent after the company reported positive results from ATTRibute-CM, its phase 3 study of acoramidis in transthyretin amyloid cardiomyopathy, or ATTR-CM, a buildup of faulty proteins on the heart that can cause heart failure.

The new results come after acoramidis failed to show benefit over placebo in a late-stage trial for the same indication at the end of 2021, which sent BridgeBio shares plummeting at that time.

“The outstanding results of the ATTRibute-CM study provide new hope to patients living with transthyretin amyloid cardiomyopathy, or ATTR-CM”, said Daniel Judge, professor of Medicine and Cardiology at the Medical University of South Carolina, and co-chair of the ATTRibute-CM Steering Committee. “The consistent and clinically meaningful benefits on survival, hospitalization, and additional measures of illness severity are truly remarkable.”

ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of transthyretin (TTR).

Key results from the clinical trial include a highly statistically significant improvement in the primary endpoint (a hierarchical analysis prioritizing in order: all-cause mortality, then frequency of cardiovascular-related hospitalization, then change from baseline in NT-proBNP, then change from baseline in 6-minute walk distance) demonstrated by a Win Ratio of 1.

There was an 81 percent on-treatment survival rate (versus a 74 percent survival rate on placebo), which begins to approach actuarial models of life expectancy absent ATTR-CM of 85 percent in this population. The absolute risk reduction was 6.43 percent and the relative risk reduction was 25 percent.

Results also showed a highly statistically significant relative risk reduction of 50 percent on frequency of cardiovascular-related hospitalization. The impact and marked magnitude of risk reduction was seen across all analytical methods employed.

The company consistently observed a statistically significant treatment effect at 30 months across additional measured markers of morbidity, quality of life, and function: change from baseline in N-terminal prohormone of brain natriuretic peptide (NT-proBNP); change from baseline in Kansas City Cardiomyopathy Questionnaire; and change from baseline in 6-minute walk distance.

No safety signals of potential clinical concern were identified.

“We are extremely encouraged by the robustly positive and consistent findings of the ATTRibute-CM study, which confirm our position that highly potent TTR stabilization has the potential to profoundly impact patients’ lives,” said Jonathan Fox, president and chief medical officer of BridgeBio Cardiorenal.

The company intends to submit its NDA to the U.S. Food and Drug Administration before the end of 2023, with regulatory filings in additional markets to follow in 2024.

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