Liver Cancers

ALN-VSP: Liver Cancers

Alnylam has advanced its RNAi therapeutic, ALN-VSP, for the treatment of liver cancers and potentially other solid tumors.

ALN-VSP targets two key genes each involved in the disease pathway of liver cancer: kinesin spindle protein, or KSP is involved in cancer proliferation, and vascular endothelial growth factor, or VEGF, is involved in cancer angiogenesis. We believe that a dual-target approach in cancer increases the potential for a significant therapeutic benefit. In April 2009, Phase I clinical trials were initiated for ALN-VSP.

In pre-clinical studies, ALN-VSP demonstrated the ability to silence both tumor KSP and VEGF and stopped the proliferation of cancer cells. In these studies, ALN-VSP showed a striking reduction in the growth and number of significant tumor masses in the liver. Data reported at the 23rd Annual Meeting of the International Society for Biological Therapy of Cancer demonstrated robust efficacy in an orthotopic liver tumor model, including the inhibition of tumor growth as measured by serum levels of alpha-fetoprotein (AFP) and a significant survival benefit. Specific silencing of KSP and VEGF for ALN-VSP was demonstrated, and an RNAi mechanism of action toward both target gene mRNAs was confirmed using a 5'RACE method.

We filed our IND for ALN-VSP. Human clinical trials with this innovative medicine are expected to start in the first half of 2009

Phase I human clinical trial of ALN-VSP were initiated to evaluate its safety, tolerability, pharmacokinetics, and pharmacodynamics in patients with advanced liver cancers, including hepatocellular carcinoma and other solid tumors with liver involvement.

Primary liver cancer – hepatocellular carcinoma, or HCC – is one of the most common cancers worldwide, with more than 600,000 people diagnosed each year. Secondary liver cancer, also known as metastatic liver cancer, is cancer that spreads to the liver from another part of the body due to other cancers like colon, lung, or breast cancer. Worldwide, more than 500,000 people are diagnosed with secondary liver cancer each year.