In the Phase I/IB study, only patients with NTRK or ROS1 fusions, or an ALK fusion or mutation responded to entrectinib, while patients without these tumor markers didn’t respond.
Quark Pharmaceuticals last week announced “favorable” interim results from a phase I trial of its siRNA-based ocular neuroprotectant QPI-1007, showing the drug may improve the vision of patients with a rare eye condition.
Alnylam partner Tekmira Pharmaceuticals said it views the data as a validation of its drug-delivery technology, which will be used in two new oncology programs it unveiled this week.
The disclosure comes almost a year after Calando Pharmaceuticals published phase I data demonstrating that its own cancer drug CALAA-01 could knock down its intended target mRNA and protein inside a tumor through an RNA interference mechanism when delivered intravenously into humans.
The phase I drug, ALN-VSP02, comprises two siRNAs, one targeting vascular endothelial growth factor and one targeting kinesin spindle protein, both of which the firm said are critical for the growth and survival of cancer cells.
The data, which appeared in the online edition of Nature, "demonstrate that RNAi can occur in a human from a systemically delivered siRNA, and that siRNA can be used as a gene-specific therapeutic," the investigators wrote.
A bill passed by a US House of Representatives appropriations subcommittee would give scientific agencies including the National Science Foundation boosts in funding.
