Portola Pharma Initiates Phase 1/2 Clinical Study Of PRT2070 - Quick Facts

(RTTNews) - Portola Pharmaceuticals Inc. (PTLA) announced Wednesday that it has initiated a Phase 1/2 clinical study of PRT2070, a novel, oral, dual spleen tyrosine kinase or Syk and janus kinase or JAK inhibitor. The compound is being developed for patients with genetically-defined hematologic cancers, as well as for patients who have failed therapy due to relapse or acquired mutations.

The company noted that PRT2070's dual mechanism of action, which targets two validated pathways for tumor survival and proliferation, has strong scientific rationale in these settings, where other compounds have failed to demonstrate clinical benefit.

"With PRT2070, we have seen promising in vitro activity in cell lines with specific mutations and in tumor cells that have lost sensitivity to other compounds due to acquired mutations. We look forward to seeing initial clinical data for this agent in 2014," said John Curnutte, executive vice president of research and development for Portola.

The open-label, multicenter, Phase 1/2 proof-of-concept study will assess the safety, pharmacokinetics, pharmacodynamics and clinical activity of oral PRT2070. In the multi-dose, dose-escalation Phase 1 part of the study, PRT2070 will be administered to sequential dose cohorts at increasing dose levels until the maximum tolerated dose is identified.

The Phase 1 portion of the study can include patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma and non-Hodgkin lymphoma (NHL) (including diffuse large B-cell lymphoma, follicular lymphoma and mantle cell lymphoma).

The Phase 2 part of the study is a cohort expansion that will evaluate measures of safety and efficacy in cancer types identified based on the responses seen in the dose-escalation phase. Portola has engaged the Sarah Cannon Research Institute, a global strategic research organization based in Nashville, Tenn., to conduct the study.