Alnylam scores efficacy data with PCSK9 drug

Posted under ALN-PCS, Alnylam Pharmaceuticals, Amgen, Blog, cholesterol, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, PCSK9 drugs, Pharmaceuticals, Pipeline, RNAi, Startups, Universities, Videos by rmcbride

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

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Countering skeptics, Alnylam claims pioneering first in RNAi cholesterol study

Posted under ALN-PCS, Alnylam Pharmaceuticals, Blog, Companies, Diagnostics, Funding, Medical Devices, Medical Supply, Pharmaceuticals, RNAi, Startups, Universities, Videos by John Carroll

Facing off against some deep-seated skepticism about the future of RNAi drug development, Alnylam ($ALNY) has pushed out a preliminary assessment of a small Phase I study demonstrating some dramatic results in sharply reducing the levels of bad cholesterol in patients suffering from severe hypercholesterolemia. And tackling doubts about being able to effectively deliver an RNAi treatment, Alnylam touted the use of second-generation lipid nanoparticles in the study.

The bottom line on the study is impressive: Patients taking ALN-PCS experienced a reduction of LDL-C of 50% or more. And the biotech unabashedly claimed an historic first in the study.

"We are extremely pleased with these data from our ALN-PCS trial which represent what we believe is the first ever demonstration of efficacy for an RNAi therapeutic toward a clinically validated endpoint, namely LDL-C, a defined risk factor for coronary artery disease and acute myocardial infarction. Indeed, preliminary results from our ongoing study show robust, statistically significant, and dose-dependent lowering of both PCSK9 and LDL-C levels," said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President and Chief Medical Officer of Alnylam. "The RNAi effects were rapid and durable after a single dose, exemplifying a compelling profile for RNAi therapeutics that we have now established in man for two disease programs."

The RNAi world has been rocked by a number of high-level defections in the past two years as pharma companies assessed the time, costs and efforts needed to get a treatment through clinical trials and on to an approval. Encouraging efficacy data from a small Phase I, while helpful, is unlikely to dramatically change anyone's mind about that. But it is a step forward for a field that has been forced to contend with a bitter chill.

- here's the Alnylam release

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