We have published complete study results from our Phase I clinical trial with ALN-VSP, an RNAi therapeutic for the treatment of advanced solid tumors with liver involvement. Results from the study – the most comprehensive study of a systemically administered RNAi therapeutic to date – demonstrated proof of RNAi activity in man and evidence of anti-tumor activity, in addition to highlighting the safety and tolerability of multiple doses of ALN-VSP.
Data showed that ALN-VSP was generally well tolerated and showed evidence of tumor regression. Importantly, ALN-VSP was found to mediate RNAi activity in both hepatic and extra-hepatic tumors. Data from the extension study demonstrated disease control lasting more than six months in the majority of patients (who received an average of over 11 months of overall treatment), including a complete response (CR) in an endometrial cancer patient who had multiple liver metastases and who received treatment for over two full years.
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