Alnylam's HELIOS-A Trial Meets Secondary Endpoints At 18 Months; Vutrisiran Faces FDA On Apr.14

Alnylam Pharmaceuticals Inc.'s (ALNY) phase III trial of investigational RNAi therapeutic Vutrisiran in patients with hATTR amyloidosis with polyneuropathy has met all secondary endpoints at 18 months.

Hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is an inherited, progressively debilitating, and fatal disease caused by mutations in the TTR gene. Mutations in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory-motor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations.

In the phase III trial, dubbed HELIOS-A, Vutrisiran has demonstrated statistically significant improvements in neuropathy impairment, quality of life, gait speed, nutritional status and overall disability, relative to placebo.

Last April, the company reported that the HELIOS-A met its primary and secondary endpoints at nine months in patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy.

The primary endpoint achieved at 9 months was improvement in the mean change from baseline in the modified Neuropathy Impairment Score compared to external placebo data while the secondary endpoints that were met included improvement in quality of life as assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy instrument and improvement in gait speed compared to the external placebo group.

Vutrisiran, proposed for the treatment of polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults, is under FDA review, with a decision expected on April 14, 2022.

The other approved drugs for the treatment of transthyretin-mediated amyloidosis include Alnylam's Onpattro; Pfizer's Vyndaqel, and Ionis's Tegsedi.

ALNY has traded in a range of $125.08 to $212.00 in the last 1 year. The stock closed Thursday's trading at $138.31, down 0.22%.

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