Portola Begins POC Cancer Trial

Portola Pharmaceuticals has initiated a Phase I/II study of PRT2070, a novel, oral, dual spleen tyrosine kinase (Syk) and janus kinase (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. PRT2070’s dual mechanism of action targets two validated pathways for tumor survival and proliferation.

The open-label, multicenter, Phase I/II proof-of-concept study will assess the safety, pharmacokinetics, pharmacodynamics and clinical activity of oral PRT2070. In the multi-dose, dose-escalation Phase I 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 I 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 II 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, TN, to conduct the study.