Alnylam Pharmaceuticals and Collaborators Publish Results from Phase I Clinical Trial and Extension Study with ALN-VSP, an RNAi Therapeutic for the Treatment of Liver Cancer
Published: Jan 30, 2013
CAMBRIDGE, Mass.--(BUSINESS WIRE)--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.