What happened

Shares of Alnylam Pharmaceuticals (ALNY 0.04%) are poised to post a new all-time high upon market open Wednesday. The RNA interference (RNAi) drug specialist's stock price rose by as much as 53% in premarket action this morning.

What's fueling this supercharged move higher today? Ahead of the opening bell, Alnylam announced that its closely watched phase 3 Apollo-B study of the RNAi therapy, patisiran, in patients with transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy met its primary endpoint of change in baseline in the 6-minute walk test at 12 months compared to placebo. ATTR amyloidosis with cardiomyopathy is a rare but often fatal disease, characterized by the buildup of a misfolded protein in the main pumping chamber of the heart.

A hand draws an ascending bar graph on a chalkboard.

Image source: Getty Images.

So what

The biotech said that this novel gene silencer also met the study's first secondary endpoint of change from baseline in quality of life compared to placebo. Patisiran, however, did not pass the point of statistical significance on the study's secondary composite endpoint of all-cause mortality. Alnylam said additional details on the study's results would be presented at a medical conference next month.  

The big deal, from an investing standpoint, is that this high-value indication ought to generate blockbuster sales for the company in short order. What's more, this successful late-stage readout will likely make Alnylam a red-hot buyout target. 

Now what

Is Alnylam stock still a buy after its meteoric rise this morning? I believe so. The company's novel RNAi platform took a major step forward with this late-stage success, putting it on track for enormous levels of organic growth in the years to come. Moreover, the company offers investors a second way to win through a possible buyout.