Alnylam And Collaborators Publish Results From Phase I Clinical Trial And Extension Study With ALN-VSP, An RNAi Therapeutic For The Treatment Of Liver Cancer

Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), a leading RNAi therapeutics company, and collaborators announced today the publication of complete study results from a Phase I trial with ALN-VSP, a systemically delivered RNAi therapeutic for the treatment of advanced solid tumors with liver involvement. The paper, titled “ First-in-Man Trial of an RNA Interference Therapeutic Targeting VEGF and KSP in Cancer Patients with Liver Involvement” appears as an OnlineFirst publication in the journal Cancer Discovery (Tabernero et al., Cancer Discovery CD-12-0429; Published OnlineFirst January 2013). The study results document anti-tumor activity for ALN-VSP in a heavily pre-treated and advanced patient population, including a complete response in an endometrial cancer patient who had multiple hepatic metastases. In addition, this study provided proof of RNAi mechanism in man based on molecular analysis of biopsy samples from patients. Finally, in this study – the most comprehensive study of a systemically administered RNAi therapeutic to date – chronic dosing of ALN-VSP for up to 26 months was found to be generally safe and well tolerated.

“Our ALN-VSP Phase I clinical trial defines the most comprehensive human experience for RNAi therapeutics delivered with lipid nanoparticle formulations. Results from this study highlight safety and tolerability of multiple doses of ALN-VSP, proof of RNAi activity in man, and evidence for anti-tumor activity in a very advanced, heavily pre-treated cancer patient population,” said Jared Gollob, M.D., Vice President, Clinical Research at Alnylam. “We are encouraged by the anti-tumor activity observed in this study in multiple patients who achieved stable disease or better; this includes a patient with endometrial cancer metastatic to the liver who achieved a complete response. Results from the extension study also give us increased confidence in long-term chronic dosing for RNAi therapeutics delivered with lipid nanoparticle formulations, as patients received an average of over 11 months of treatment overall, including one patient who received treatment for over two full years.”

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