Alnylam Pharmaceutical Surges After Rare Disease Drug Met Key Endpoints

Shares of Alnylam Pharmaceuticals, Inc. ALNY surged higher by more than 20 percent early Wednesday morning after the company impressed investors with encouraging data from a phase 3 clinical trial.

Alnylam, a company dedicated towards treating a wide range of debilitating diseases through ribonucleic acid (RNA) interference (RNAi) therapeutics, said after Tuesday's market close that a phase 3 study called APOLLO met its primary efficacy endpoint and all secondary endpoints.

The company's clinical trial explored its therapy called patisiran for patients with hereditary ATTR amyloidosis with polyneuropathy. The primary endpoint for the study was the change from baseline in the modified neuropathy impairment score (mNIS+7) at 18 months. The secondary endpoint was an improvement in quality of life assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy.

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Alnylam's data release "looks clean" and could eliminate some concerns that were reported in other pipeline drugs, STAT News reported. As such, the company is now "on the cusp" of proving that RNAi can be transformed into medicines that can help patients in need.

The company expects to file its first New Drug Application in late 2017 and then follow up with a Marketing Authorisation Application in conjunction with its partner, Sanofi SA (ADR) SNY which is preparing for regulatory filings in Japan, Brazil and other countries.

Alnylam will commercialize patisiran in the U.S., Canada, and Western Europe and Sanofi will handle the product in the rest of the world.

"Patients living with hATTR amyloidosis face an inevitable and painful advancement of their debilitating disease," said Akshay Vaishnaw, M.D., Ph.D., Executive Vice President, R&D of Alnylam. "We believe the very encouraging APOLLO data demonstrate the potential for investigational patisiran to help improve the lives of hereditary ATTR amyloidosis polyneuropathy patients. Our immediate objective is now to submit these data to global health authorities.

The stock traded at $90.51 in the pre-market session.