Alnylam Pharmaceuticals, Inc. News
− Echocardiographic and Cardiac Biomarker Data From the HELIOS-B Study Presented Today Support the Potential of Vutrisiran in ATTR-CM –
− Vutrisiran Significantly Improved Echocardiographic Assessments of Cardiac Structure, Systolic Function and Diastolic Function Relative to Placebo Over 30 Months –
− Vutrisiran Demonstrated Relative Stability of Cardiac Biomarkers NT-proBNP and Troponin-I Relative to Placebo Over 30 Months –
CAMBRIDGE, Mass.-- Alnylam Pharmaceuticals, Inc., an RNAi therapeutics company, announced that two new data sets from the HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis with cardiomyopathy (ATTR-CM), were presented in the Late Breaking Clinical Research Session 1 at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2024, which was held virtually. An open access recording of Alnylam presentations will be available on the HFSA website following the session.
Progression in ATTR-CM is associated with cardiac wall thickening, deterioration in systolic and diastolic function and increases in biomarkers of cardiac stress and injury, NT-pro-BNP and Troponin I.
“Consistent with demonstrated improvements in outcomes and health status, these new data show that vutrisiran attenuated measures of disease progression across multiple domains of cardiac structure and function, NT-proBNP and troponin I, in a contemporary ATTR-CM patient population,” said Pushkal Garg, M.D., Chief Medical Officer, Alnylam. “These data demonstrate that rapid knockdown of TTR leads to an early impact on cardiac biomarkers and echocardiographic parameters, indicative of a potential disease-modifying effect, and underscores the benefit of treating patients with an RNAi therapeutic earlier in the course of disease. We remain confident that, with approval, vutrisiran has the potential to become a first-line therapy for ATTR amyloidosis with cardiomyopathy and are on track to complete multiple global regulatory submissions before the end of the year.”
Analysis results
New echocardiographic data demonstrated that treatment with vutrisiran slowed disease progression in a contemporary population of patients with ATTR-CM across multiple domains of cardiac structure and diastolic and systolic function at Month 30 as compared to placebo. The magnitude of the treatment effects with vutrisiran compared to placebo were similar or greater in the monotherapy population. Significant improvements in both diastolic and systolic function were observed as early as 12 months and 18 months, respectively, in the overall population. The Month 30 results in the overall population are detailed in the table below.*