Alnylam Pharmaceuticals Inc. (ALNY) has announced new findings from the HELIOS-B Phase 3 study of vutrisiran, revealing that the drug significantly mitigates the progression of disease in patients with ATTR amyloidosis with cardiomyopathy. Specifically, vutrisiran has shown positive effects on various indicators of cardiac health, including NT-proBNP and troponin I levels.
The data highlights that prompt reduction of TTR can have an early beneficial impact on cardiac biomarkers and echocardiographic measures, suggestive of a potential disease-modifying effect. This underscores the advantages of early treatment with an RNAi therapeutic.
The company maintains confidence that vutrisiran, once approved, has the potential to become a first-line therapy for ATTR amyloidosis with cardiomyopathy. Alnylam is on schedule to complete several global regulatory submissions by the end of the year.
Vutrisiran is currently an investigational RNAi therapeutic designed for treating ATTR amyloidosis with cardiomyopathy (ATTR-CM).
The latest echocardiographic data demonstrated that vutrisiran treatment slows disease progression in contemporary patients with ATTR-CM across multiple cardiac structure and functional domains when compared to a placebo over a 30-month period. The treatment effects of vutrisiran, especially in a monotherapy setting, were either similar or more pronounced than placebo, showing notable improvements in diastolic and systolic functions as early as 12 and 18 months, respectively.
By Month 30, patients treated with vutrisiran experienced a 32% reduction in the fold change of NT-proBNP levels compared to placebo in the overall population, with a 43% reduction observed in the monotherapy group. Similarly, troponin I levels saw a reduction of 32% in the overall population and 45% in the monotherapy population.
In a subgroup of patients who were on tafamidis at baseline, there was an 18% reduction in NT-proBNP and a 10% reduction in troponin I levels at Month 30. Notably, patients treated with vutrisiran showed nominally significant reductions in both NT-proBNP and troponin I levels relative to the placebo at 6 months.