Alnylam Shares Shoot Higher Following Positive Clinical Results From Late-Stage ATTR Amyloidosis Therapeutics

Alnylam ALNY announced positive results from the APOLLO-B Phase 3 study of Patisiran for the treatment of transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.

APOLLO-B is a Phase 3 multicenter global study designed to evaluate the effects of patisiran on functional capacity and quality of life in patients with ATTR amyloidosis with cardiomyopathy, enrolled 360 adult patients with ATTR amyloidosis (hereditary or wild-type) with cardiomyopathy at 69 sites in 21 countries.

The phase 3 study has met the primary endpoint of change from baseline in the 6-Minute Walk Test (6-MWT) at 12 months compared to placebo (p-value 0.0162) and also met the first secondary endpoint of change from baseline in quality of life compared to placebo, as measured by the Kansas City Cardiomyopathy Questionnaire (KCCQ) (p-value 0.0397).

Pushkal Garg, Chief Medical Officer, said, “We are thrilled that APOLLO-B successfully met all its major objectives, which we believe for the first time validates the hypothesis that TTR silencing by an RNAi therapeutic can be an effective approach for treating the cardiomyopathy of ATTR amyloidosis.”

With the positive results from the phase 3 clinical study, the company is planning to submit a supplemental NDA for patisiran with the U.S. Food and Drug Administration in late 2022.

The company is also planning to present APOLLO-B study results as part of a late-breaker session at the 18^th International Symposium on Amyloidosis on September 8, 2022, in Heidelberg, Germany.

Patisiran is the established name for ONPATTRO, which is approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults.

Price Action : Alnylam shares are trading around 47 percent higher at $207.46 on Wednesday at the time of publication.