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Research & Development
Board of Directors
We are focused on the discovery and development of important therapeutics for cancer and infectious diseases, with both small molecule and biologics approaches.
We aim to develop first-in-class medicines specifically for the China market and first/best-in-class medicines for worldwide unmet medical needs, which we will partner globally after demonstrating clinical proof-of-concept.
ASC-06 (ALN-VSP), Phase I Trial and Extension Study Designs
ALN-VSP is a systemically delivered RNAi therapeutic comprising of two siRNAs designed 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). The ALN-VSP Phase I trial was designed as a multi-center, open label, dose escalation study in patients with advanced solid tumors with liver involvement who have failed to respond to or have progressed after standard treatment.
The primary objective was to evaluate the safety, tolerability, and pharmacokinetics of intravenous ALN-VSP. Other secondary and exploratory objectives included: assessment of tumor response using Response Evaluation Criteria for Solid Tumors (RECIST), a set of published guidelines that defines when a cancer patients’ disease improves, stabilizes or progresses during treatment; quantitation of change in tumor blood flow and vascular permeability as measured by dynamic contrast-enhanced magnetic resonance imaging (DCE-MRI); and, analysis of pharmacodynamic effects of ALN-VSP on tumors as measured in patients electing to proceed with voluntary pre- and post-treatment biopsies.
The ALN-VSP extension study was designed to enable continued dosing with ALN-VSP in patients who had achieved stable disease or better after completing four months of treatment on the Phase I trial. Patients enrolling onto the extension study were permitted to receive ALN-VSP bi-weekly until disease progression or unacceptable toxicity, with the primary objective to collect long-term safety data and the secondary objective to assess tumor response.
RESEARCH & DEVELOPMENT