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Alnylam says gene silencing therapy works for high cholesterol

ALN-PCS trial data contributes to restored interest in RNA interference

Alnylam Pharmaceuticals has reported encouraging results from a phase I trial of ALN-PCS, a gene silencing therapy for severely elevated cholesterol.

The company claims this is the first time that a therapeutic based on RNA interference (RNAi) has demonstrated efficacy towards "a clinically-validated endpoint, namely LDL-cholesterol, a defined risk factor for coronary artery disease and acute myocardial infarction".

Enthusiasm for RNAi therapies has been reduced after clinical trial failures - often caused by delivery difficulties - as well as Roche's decision to pull out of RNA-targeted therapy research in 2010.

However, continued reporting of positive clinical data has managed to restore interest in the sector.

In Alnylam's latest study, ALN-PCS cut plasma levels of PCSK9 protein in plasma - a regulator of the LDL receptor - by around two thirds. This was accompanied by reductions of around 50 per cent in levels of LDL-C.

The study is ongoing, with data reported on the first 20 of a planned 60 test subjects, according to Alnylam. Based on the initial results it now plans to continue escalating the dose of ALN-PCS to see if greater reductions in LDL-C can be achieved.

The drug exerted long-lasting effects, even though it was given as a single systemic injection, and the duration of the response indicated that it could be administered just once a month.

"Our investment in delivery technologies has paid off," said Alnylam chief executive John Maraganore in a conference call to discuss the results.

The company has developed a series of new delivery systems for its RNAi drugs based on lipid nanoparticles (LNP), and now believes intravenous injections once every two months may feasible.

It is also looking into the potential for subcutaneous administration, with promising results in animal models, he said.

Akshay Vaishnaw, Alnylam's chief medical officer, said the latest results establish "a compelling profile for RNAi therapeutics … in man for two disease programmes".

The company has previously reported positive results in a phase I trials of ALN-TTR01 in transthyretin (TTR)-mediated amyloidosis.

It is now planning to press ahead with clinical studies of a new version (ALN-TTR02) based on the same LNP delivery system as is used in the cholesterol programme.

5th January 2012

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