Alnylam Pushes First RNAi Drug That Circulates Through Body Into Human Test

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two critical genes for this type of tumor. The first, kinesin spindle protein, or KSP, is essential for helping tumors spread. The second, vascular endothelial growth factor, or VEGF, is a gene that helps form blood vessels that nourish growing tumors.

Alnylam has run tests in mice that show its drug can suppress the intended genes, shrink tumors, and help the cancerous animals to live longer. ALN-VSP also passed safety tests in rats and non-human primates, Maraganore says.

The drug is designed to be given via a 15-minute intravenous infusion, which could be given once every couple weeks, Maraganore says. The clinical trial is expected to enroll its first patient before the end of June, he says.

This is the first time Alnylam has pursued a cancer drug program. I asked Maraganore why he would pursue liver cancer, in particular, since a relatively new drug, sorafenib (Nexavar) from Onyx Pharmaceuticals and Bayer, has been shown to prolong lives of liver cancer patients, at least partially by blocking the same VEGF target. One big reason is the belief that an RNAi drug can be more effective, because it shuts down the disease earlier in the disease-forming process, rather than after the troublesome proteins get blocked by the competing drug, Maraganore says.

This milestone won’t immediately turn lucrative for Tekmira, but that company will stand to receive an undisclosed milestone payment when Alnylam delivers the first dose of this product to a patient, Maraganore says. The deal between the companies says that Tekmira can get up to $16 million of milestone payments for every product Alnylam develops with its lipid-delivery technology, although most of the payments are tilted toward success in later stages of development, Maraganore says. Tekmira also stands to receive royalty payments if Alnylam can develop any of these drugs into an FDA approved product, he says.

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