Today, Alnylam Pharmaceuticals (NASDAQ:ALNY) revealed positive top-line results for the company’s transthyretin-mediated amyloidosis treatment, patisiran. Alnylam’s drug reportedly met its primary endpoint for selected trial participants, and consequently, ALNY stock is roaring higher today.
Transthyretin-mediated amyloidosis is a rare and debilitating disease that involves the nerves, heart, and gastrointestinal (GI) tract. Different types of transthyretin-mediated amyloidosis affect tens of thousands, or even hundreds of thousands of people worldwide. Alnylam Pharmaceuticals’ drug, patisiran (brand name ONPATTRO), is designed to treat some symptoms of hereditary transthyretin-mediated amyloidosis.
More specifically, Alnylam tested patisiran in transthyretin-mediated amyloidosis patients with cardiomyopathy. In a Phase 3 study, the participants were given a 6-Minute Walk Test and at 12 months, patisiran met the primary endpoint of change from baseline compared to a placebo.
This wasn’t the only positive result of the clinical trial. According to Alnylam Pharmaceuticals, the study also met the first secondary endpoint of change from baseline in quality of life compared to a placebo. This result for patisiran was measured by the Kansas City Cardiomyopathy Questionnaire (KCCQ).
What’s Happening With ALNY Stock?
There’s a whole lot of scientific terminology in Alnylam Pharmaceuticals’ press release. However, it doesn’t take a scientist to figure out the ALNY stock is on the move today. Indeed, it moved so fast and far that trading was halted on the shares at $141.97 during premarket hours today.
$141.97 would only be a pit stop, though. By 10:30 a.m. Eastern, the Alnylam Pharmaceuticals share price had already hit $210 for a single-day gain of around 50%.
With that, ALNY stock headed quickly toward its 52-week high of $220.92. Meanwhile, Alnylam Pharmaceuticals Chief Medical Officer Pushkal Garg took a moment to tout the clinical implications of his company’s clinical-trial outcomes:
These patients have limited treatment options, and disease progression is common. As such, we are encouraged to see the potential of patisiran to improve the functional capacity and quality of life of patients living with this fatal, multi-system disease.
It’s an exciting day, clearly, for Alnylam Pharmaceuticals and the company’s long-term shareholders. For the short-sellers, however, today is probably a day they’d prefer to forget.
On the date of publication, David Moadel did not have (either directly or indirectly) any positions in the securities mentioned in this article. The opinions expressed in this article are those of the writer, subject to the InvestorPlace.com Publishing Guidelines.