Alnylam Pharmaceuticals Inc. (NASDAQ: ALNY) saw its shares make a handy gain on Wednesday after the firm gave an update on its late-stage trial in patients with hereditary ATTR amyloidosis with polyneuropathy. In fact this was one of the best performing stocks in the market, despite a relatively slow day. The company is also partnered with Sanofi (NYSE: SNY) in this endeavor.
The Apollo Phase 3 study of patisiran ultimately 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 (mNIS+7) at 18 months. The key secondary endpoint was improvement in quality of life assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QOL-DN).
At 18 months, the mean change from baseline in mNIS+7 was significantly lower in the patisiran group as compared with placebo, and patients in the patisiran group experienced improvement in their quality of life according to the Norfolk questionnaire.
All five other secondary endpoints also demonstrated statistically significant favorable differences in the patisiran arm. These included: improvements in muscle strength, gait speed, Rasch-built overall disability scale, modified body mass index and the Compass-31 questionnaire.
John Maraganore, Ph.D., CEO of Alnylam, commented:
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. This moment is the culmination of a 15-year journey of tireless work by countless contributors who have overcome enormous scientific and business challenges to make RNAi therapeutics a reality. 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.
Shares of Alnylam Pharma were last seen up about 53% at $114.82, with a consensus analyst price target of $86.18 and a 52-week trading range of $31.38 to $116.93.
Sanofi shares traded up 0.6% at $49.38, with a consensus price target of $55.00 and a 52-week range of $36.81 to $50.24.