Sanofi and Alnylam report topline results of patisiran in ATTR amyloidosis

Sanofi’s (SNY) specialty care global business unit, Sanofi Genzyme, and Alnylam (ALNY) announced that the APOLLO Phase 3 study of patisiran, an investigational RNAi therapeutic being developed for patients with hereditary ATTR amyloidosis with polyneuropathy, met its primary efficacy endpoint and all secondary endpoints.

The primary endpoint for the study was the change from baseline in the modified neuropathy impairment score at 18 months.

The key secondary endpoint was improvement in quality of life.

In healthy people, normal, so-called “wild-type” TTR functions as a transporter of thyroid hormone and vitamin A (retinol) within the bloodstream. People with mutations in the TTR gene produce abnormal, amyloidogenic , “ variant ” TTR throughout their lives.

The APOLLO trial enrolled 225 hATTR amyloidosis patients with polyneuropathy.

The overall safety profile of patisiran was “encouraging”.

Based on these positive results, Alnylam expects to file its first New Drug Application in late 2017 and first Marketing Authorisation Application shortly thereafter.

Sanofi Genzyme is currently preparing for regulatory filings for patisiran in Japan, Brazil and other countries, to begin in 1H18. Pending regulatory approvals, Alnylam will commercialize patisiran in the U.S., Canada and Western Europe, with Sanofi Genzyme commercializing the product in the rest of the world.

STOCKWINNERS

To read timely stories similar to this, along with money making trade ideas, sign up for a membership to Stockwinners.

This article does not constitute investment advice. Each reader is encouraged to consult with his or her individual financial professional and any action a reader takes as a result of information presented here is his or her own responsibility.