blood cancer

US-based Portola Pharmaceuticals has started the Phase I/II clinical study of PRT2070, an oral dual Syk and JAK inhibitor, as targeted therapy for patients with genetically defined hematologic cancers.

Sarah Cannon Research Institute has been selected by Portola Pharmaceuticals to conduct the Phase I/II proof-of-concept study that has been designed to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of PRT2070.

As part of the multi-dose, dose-escalation Phase I part of the study, PRT2070 will be given at increasing dose levels to sequential dose cohorts until identifying the maximum tolerated dose.

Patients with relapsed or refractory chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma and non-Hodgkin lymphoma (NHL) can be included in the Phase I portion of the study.

The Phase II part of the study will assess the safety and efficacy measures in cancer types that are identified based on the responses seen in the dose-escalation phase.

Portola Pharmaceuticals is developing the novel, oral compound PRT2070 for the treatment of patients with genetically-defined hematologic cancers and also for patients who have failed therapy due to relapse or acquired mutations.

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"We have seen promising in vitro activity in cell lines with specific mutations and in tumour cells that have lost sensitivity to other compounds due to acquired mutations."

The dual mechanism of action of PRT2070 targets two validated pathways for tumour survival and proliferation.

Pharmaceuticals research and development executive vice-president Dr John T Curnutte said: "Our goal with PRT2070 is to develop an effective and well-tolerated oral drug for the thousands of lymphoma and leukemia patients who are impacted by difficult-to-treat genetic subtypes or who become unresponsive to available therapies.

"With PRT2070, we have seen promising in vitro activity in cell lines with specific mutations and in tumour 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."

Portola has global rights to develop and commercialise the PRT2070 for hematologic cancers, such as CLL and NHL, and other autoimmune diseases.

Image: Micrograph of a plasmacytoma, a hematological malignancy. Photo: courtesy of Nephron.