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Alnylam Advances RNAi Therapeutics Pipeline and Files Company’s First Systemic Delivery IND
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Alnylam Pharmaceuticals, Inc. has announced that it has submitted an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for ALN-VSP, an RNAi therapeutic for the treatment of liver cancers, including hepatocellular carcinoma and other solid tumors with liver involvement.
ALN-VSP contains two small interfering RNAs (siRNAs), the molecules that mediate RNAi, formulated in a lipid nanoparticle developed by Tekmira Pharmaceuticals Corporation.
ALN-VSP is designed to target two genes critical in the growth and development of cancer: kinesin spindle protein, or KSP, required for tumor proliferation; and vascular endothelial growth factor, or VEGF, required for tumor growth. Pre-clinical data in mouse tumor model studies have demonstrated robust efficacy of ALN-VSP, including suppression of these targeted genes, demonstration of an RNAi mechanism of action, tumor reduction, and extension of survival.
“ALN-VSP represents Alnylam’s first IND for a systemically delivered RNAi therapeutic, which is a testament to the very strong progress we’ve made in achieving systemic delivery of siRNAs, including our efforts with Tekmira,” said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President, Clinical Research at Alnylam. “We’re also pleased, with this IND filing, to have met one of our key pipeline goals for 2008. Following FDA review of our submission, we expect to initiate patient dosing in the first half of 2009, which positions us solidly on track to meet our goal of having three programs in clinical trials next year.”
“Based on the encouraging pre-clinical data we’ve seen to date with this program, we believe our strategy of using an RNAi therapeutic targeting two well-validated genes critical for tumor proliferation and survival has the potential of achieving meaningful clinical benefit for patients with liver cancer,” said Dinah Sah, Ph.D., Vice President, Research, CNS and Oncology, at Alnylam.
“We believe that this is the first dual targeting RNAi therapeutic program to advance to this stage of development across the entire industry. This is an important milestone, as we view the ability to design and formulate siRNAs that achieve dual targeting as a very attractive feature of our RNAi therapeutics platform, especially in the setting of oncology drug discovery,” said Sah.
The proposed Phase I study is a multi-center, open label, dose escalation trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous ALN-VSP in patients with advanced solid tumors with liver involvement. Additional study design details will be provided upon initiation of the Phase I trial.
ALN-VSP contains two small interfering RNAs (siRNAs), the molecules that mediate RNAi, formulated in a lipid nanoparticle developed by Tekmira Pharmaceuticals Corporation.
ALN-VSP is designed to target two genes critical in the growth and development of cancer: kinesin spindle protein, or KSP, required for tumor proliferation; and vascular endothelial growth factor, or VEGF, required for tumor growth. Pre-clinical data in mouse tumor model studies have demonstrated robust efficacy of ALN-VSP, including suppression of these targeted genes, demonstration of an RNAi mechanism of action, tumor reduction, and extension of survival.
“ALN-VSP represents Alnylam’s first IND for a systemically delivered RNAi therapeutic, which is a testament to the very strong progress we’ve made in achieving systemic delivery of siRNAs, including our efforts with Tekmira,” said Akshay Vaishnaw, M.D., Ph.D., Senior Vice President, Clinical Research at Alnylam. “We’re also pleased, with this IND filing, to have met one of our key pipeline goals for 2008. Following FDA review of our submission, we expect to initiate patient dosing in the first half of 2009, which positions us solidly on track to meet our goal of having three programs in clinical trials next year.”
“Based on the encouraging pre-clinical data we’ve seen to date with this program, we believe our strategy of using an RNAi therapeutic targeting two well-validated genes critical for tumor proliferation and survival has the potential of achieving meaningful clinical benefit for patients with liver cancer,” said Dinah Sah, Ph.D., Vice President, Research, CNS and Oncology, at Alnylam.
“We believe that this is the first dual targeting RNAi therapeutic program to advance to this stage of development across the entire industry. This is an important milestone, as we view the ability to design and formulate siRNAs that achieve dual targeting as a very attractive feature of our RNAi therapeutics platform, especially in the setting of oncology drug discovery,” said Sah.
The proposed Phase I study is a multi-center, open label, dose escalation trial to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of intravenous ALN-VSP in patients with advanced solid tumors with liver involvement. Additional study design details will be provided upon initiation of the Phase I trial.
