What a difference a year can make. In October last year, Alnylam Pharmaceuticals’ share price was in free fall following a decision to scrap a Phase 3 clinical trial for its revusiran therapeutic for hemophilia in the wake of a disproportionate number of deaths in the active arm of the trial. The pharma company’s share price plummeted 50 percent.
But promising Phase 3 study results for its RNAi treatment, Patisiran, to treat a rare, genetic nerve disorder has significantly increased the chances that the company could be the first to commercialize a RNAi drug.
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RNAi uses segments of genetic code to de-activate disease-causing genes. The genetic nerve disorder Alnylam is targeting with its RNAi treatment is transthyretin-mediated amyloidosis. It is a progressive, debilitating, and frequently fatal disease caused by mutations in the TTR gene. TTR mutations can cause abnormal amyloid proteins which can cause damage to tissue, peripheral nerves and the heart. This can lead to intractable peripheral sensory neuropathy, autonomic neuropathy, and/or cardiomyopathy, according to the news release. The condition affects 50,000 people globally.
The study met two endpoints — the primary endpoint was the change from baseline in the modified neuropathy impairment score (mNIS+7) at 18 months, the news release said. A secondary endpoint was improvement in quality of life assessed by the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy.
John Maraganore, Alnylam CEO, said it was an “incredibly exciting milestone” for the pharma company. He thanked the patients, investigators, physicians and staff who took part in and managed the study.
“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,” he said. “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.”
Reuters referenced Leerink analyst Paul Matteis who said the results were a “big win” for Alnylam and RNAi because they validated the platform. The safety data also looked solid.
In the wake of those Phase results, Alnylam’s shares rocketed nearly 40 percent in extremely heavy morning trade Wednesday, according to Marketwatch.
Alnylam said it plans to file a new drug application with the U.S. Food and Drug Administration this fall followed by an application for market authorization, according to a news release.
As part of an alliance agreement hammered out with Alnylam in 2014, Sanofi Genzyme has rights to the treatment outside of the U.S. It is preparing to apply for regulatory approval in Japan, Brazil, and other countries in the first half of 2018, the news release said. 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.
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