Sino-U.S. joint venture firm Ascletis Pharmaceuticals inked exclusive rights to develop Alnylam Pharmaceuticals’ early clinical-stage anticancer RNAi candidate ALN-VSP, for the treatment of hepatocellular carcinoma (HCC) and other liver cancers in China (including Hong Kong and Macau) and Taiwan. Alnylam retains all rights to the drug in the rest of the world, and will receive future milestones and royalties based on development and sales of the drug in its partner’s territories.

Ascletis initial focus will be to progress ALN-VSP into a Phase II study for the treatment of HCC. Under a strategic collaboration part of the overall deal, Alnylam may use data generated in China by Ascletis for developing ALN-VSP in the rest of the world, for which Ascletis may be eligible for sublicense payments.

“With this collaboration, we are able to develop ALN-VSP globally through the product’s advancement in a region where HCC is a particular challenge,” comments Laurence Reid, Ph.D., svp and chief business officer at Alnylam. “As we retain all rights in the rest of the world, this partnering strategy provides multiple future opportunities for Alnylam to advance this novel therapeutic in other markets.”

ALN-VSP is a systemically delivered RNAi therapeutic comprising two RNAis designed to target VEGF and kinesin spindle protein (KSP, or eglin 5). The candidate has already undergone a Phase I study in patients with advanced cancers involving the liver. An extension to the study is ongoing, and available data from the extension were presented at ASCO earlier this year. These showed that 42% of patients treated at a higher dose of ALN-VSP achieved stable disease or better after the first two months of therapy. Disease control lasting over six months was in addition achieved in the majority of patients, including a complete response in an endometrial cnacer patient with multiple liver metastases.

Ascletis was founded in 2011 as a specialty therapeutics business focused on cancer and infectious diseases. The firm’s preclinical in-house pipeline includes lead optimization-stage compounds targeting HIV (integrase), HCV (polymerase), and cancer (kinase). 

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