Although we don't believe in timing the market or panicking over market movements, we do like to keep an eye on big changes -- just in case they're material to our investing thesis.
What's happening: Shares in Alnylam Pharmaceuticals (NASDAQ:ALNY) are surging higher by as much as 10% earlier today after data from an ongoing phase 2 trial for patisiran as a treatment for familial amyloidotic polyneuropathy, or FAP.
Why it's happening: Patisiran is Alnylam's most advanced clinical drug and a big reason that Sanofi (NYSE:SNY), Alnylam's partner on the drug, took a 12% equity stake in Alnylam last year.
The updated patisiran data show that patients who have taken patisiran had a 2.5 point decrease in modified neuropathy impairment score after 12-months. That is better than the typical 13 to 18 point increase for untreated FAP patients.
FAP is a rare genetic disorder that can cause a buildup of the amyloid protein in tissue and organs. Patients have few treatment options and typically live five to 15 years after symptoms appear.
The tough-to-treat nature of this disease means that patisiran's phase 2 results are encouraging; however, it will be far more important to see patisiran succeed in ongoing phase 3 trials.
If patisiran puts up similarly strong results in those late stage trials, then it could clear the way for a filing for FDA approval. It would also help validate Alnylam's RNA targeting approach, which is being leveraged across a pipeline of drug prospects for other indications, including cardiovascular conditions.
Regardless, investors may want to keep an eye toward later this year when 18-month phase 2 data will be presented. That 18-month data could provide insight into the phase 3 results given that the endpoint for phase 3 is a drop in impairment score at the 18 month mark.
Todd Campbell has no position in any stocks mentioned. Todd owns E.B. Capital Markets, LLC. E.B. Capital's clients may or may not have positions in the companies mentioned. The Motley Fool recommends Alnylam Pharmaceuticals. Try any of our Foolish newsletter services free for 30 days. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.