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Alnylam concludes liver cancer drug Phase I trial

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US based drug developer Alnylam Pharmaceuticals has concluded an open label, multi-center, dose escalation Phase I trial evaluating ALN-VSP as a treatment for advanced solid tumors with liver involvement.

ALN-VSP is a systemically delivered RNAi therapeutic which includes two siRNAs intended to target two genes critical for the growth and development of cancer cells: vascular endothelial growth factor (VEGF) and kinesin spindle protein (KSP), also known as eglin 5 (Eg5).

In the trial, the patients were treated at doses ranging from 0.1 to 1.5 mg/kg. Alnylam Clinical Research senior director Jared Gollob said the results of ALN-VSP Phase I trial demonstrated safety and tolerability of multiple doses of ALN-VSP in addition to evidence for anti-tumor activity and proof of RNAi mechanism from tissue biopsy samples.

 

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Strategic early-stage planning and rigorous regulatory readiness serve as the foundation for accelerating pharmaceutical timelines and optimizing long-term R&D efficiency.

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