A couple of companies have had a tough twenty-four hours in the biotech space. One discontinuation, and another primary endpoint miss, has served up double digit losses in the companies in question, and both look set for further losses as we close in on the end of the week.
Here’s what happened.
The company has been developing what looks like a pretty robust pipeline of what are called RNAi therapeutics, or RNA interference drugs. This class of drugs was hailed as being a potential game changer across a variety of target indications as near term as just a couple of years ago. Some big names picked up programs, however they subsequently dropped these programs and moved on. Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY) was one of the few that stuck with the drug class, and the biggest of the bunch, and it pretty much gambled its future on RNAi.
Fats forward to last night, and the company announced that it was dropping one of its lead programs, a phase III trial investigating the efficacy of a drug called revusiran in a rare disease target called hereditary transthyretin-related amyloidosis (hATTR) with cardiomyopathy (hATTR-CM).
Right now, we don’t know much. All we know is that the data monitoring committee that was tasked with keeping an eye on the study brought to light some concerning death rates (and recommended halting dosing), and that on analysis of these rates, the company has decided to discontinue the study entirely. Reportedly, eighteen deaths out of a total of a little over 200 patients occurred, and there was some unusually high level of nerve pain in patients in the active arm.
On the news, markets have traded down on Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), and the company is currently trading close to 50% down on its Wednesday evening close. How this is going to play out, nobody is quite sure. The company still has a number of RNAi programs ongoing, and there looks to be some promise in these. If this turns out to be the case, the 50% hit will probably turn out to be an oversell. It’s a real gamble right now, however. It’s another in the long list of failures already racked up in RNAi, and the number of potential positive plays is dwindling.