Alnylam Pharmaceuticals Inc. (NASDAQ: ALNY) and Sanofi Genzyme announced on Wednesday its positive results from its phase 3 study of Hereditary ATTR (hATTR) Amyloidosis patients with Polyneuropathy. The company said it met all primary efficacy endpoints and secondary endpoints, causing shares to jump over 30 percent during premarket.
hATTR is one type of the systemic amyloidosis diseases that is caused by inheriting a gene mutation. That genetic mutation produces an amyloid protein that forms into an abnormal shape. It can cause disabilities and affect simple daily life tasks, and in some cases, it can be fatal.
The primary endpoint was to change the modified neuropathy impairment in patients by 18 months. The secondary was to improve the quality of life with patients who suffer from the condition.
The test enrolled 250 patients diagnosed with hATTR. Patients were split into a 2:1 ratio, those receiving the treatment and other receiving a placebo. Patients receiving the treatment were given injections of 0.3mg once every three weeks for 18 months.
By the end of the 18 month trial, the company reported that the patients receiving the treatment reported negative values, meaning an overall improvement. The secondary endpoint was met succeeding in tests such as a walking test, muscle strength, and other daily life tasks.
"We are very proud to report the first ever positive Phase 3 results for an RNAi therapeutic, marking the potential arrival of an entirely new class of medicines." said John Maraganore, Chief Executive Officer of Alnylam. "This is an incredibly exciting milestone for Alnylam and RNAi, and most importantly for patients and their treating physicians and families. We extend our deepest gratitude to all the patients, investigators and study staff who participated in the APOLLO study – they made this important scientific progress possible."
"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." said Akshay Vaishnaw, Executive Vice President, R&D of Alnylam.
Based on the results, Alnylam will file for its first New Drug Application in late 2017 and first Marketing Authorisation Application shortly after. Alnylam is looking to commercialize in the U.S., Canada, and Western Europe.
"This is a significant milestone that supports our belief that RNAi therapeutics have the potential to become an innovative new class of medicines for patients with rare genetic diseases," said Elias Zerhouni, President, Global R&D, Sanofi.