Alnylam scores efficacy data with PCSK9 drug

Alnylam Pharmaceuticals' ($ALNY) stock rose this morning after the developer of gene-silencing drugs touted early-stage trial data on its RNA-interference drug that zeros in on one of the hottest targets in biotech. The data show that the small company has a horse in the race to advance PCSK9-targeting drugs as a brand new category of treatments for lowering LDL cholesterol.

The Cambridge, MA-based company's stock had jumped 5.6% as of 10:48 a.m. ET after Alnylam revealed the Phase I trial results early this morning.

The Phase I study met its primary goal of safety and tolerability of the treatment, called ALN-PCS, which was given to patients without cholesterol-lowering statins. And patients on the drug in the 32-person study had an average drop in LDL or "bad" cholesterol of 41% compared with baseline and placebo as well as a mean reduction in PCSK9 protein levels of 68% in the highest dose group. Alnylam says it plans to find a partner for the program before moving ahead with mid-stage trials with the drug.

Alnylam isn't as advanced in clinical development with its PCSK9-targeting drug as AMG 145, Amgen's ($AMGN) contender, or REGN727, which is Sanofi ($SNY) and Regeneron's ($REGN) drug that targets the protein. However, Alnylam's program could attract a partner interested in having a stake in PCSK9 drugs, which could offer an alternative cholesterol-lowering treatment for patients who don't respond to existing drugs such as statins. Analysts say the PCSK9-targeting class could be worth billions of dollars per year if they make it onto the market.

Alnylam also noted that ALN-PCS uses the company's next-generation lipid nanoparticle delivery technology, and the upbeat results from the company's small study could bode well for other drugs in its pipeline that use the delivery tech, such as ALN-TTR02. Delivery challenges have dogged the RNAi field, and Alnylam has been working on providing data that its next-gen drugs can overcome some of the hurdles that plagued programs of the past.

- here's Alnylam's release
- and an RTTNews report