FDA lifts partial hold on Medivation’s Pidilizumab in hematological malignancies

Medivation can now go ahead with the phase II clinical trial in patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), and other studies that cross refer the IND application.

The company said the investigator brochure, protocols, and informed consent documents associated to the phase II trial have been revised to reflect its understanding that PD-1 (Programmed Death-1) is not the target of pidilizumab.

Medivation did not enroll any patients in the trial, which started in late 2015.

Medivation founder, president & CEO David Hung said: "As we move forward, we also are working to determine the compound’s exact binding mechanism which, we believe, modulates the body’s innate immune response and differentiates it from the heavily crowded immuno-oncology space that targets the adaptive side of immunity."

The company plans to submit an amendment to the chemistry, manufacturing and controls section of the IND in the second quarter to provide for larger manufacturing lot sizes for supporting the existing and proposed clinical activities for pidilizumab.

Pidilizumab is intended for development in other hematologic malignancies like multiple myeloma. The DLBCL trial is expected to restart in the second half of this year.

Under the international, open-label, phase II trial of pidilizumab, about 180 patients are expected to be enrolled with an incomplete response following salvage therapy or autologous stem cell transplantation for relapsed or refractory CD20+ DLBCL, transformed indolent lymphoma, or primary mediastinal B-cell lymphoma.

Medivation will evaluate the patients in two parallel cohorts of about 90 patients each. One cohort will enroll patients who have received an autologous stem cell transplant, and the other will enroll patients who have received salvage chemotherapy, but who are ineligible for transplant.

Pidilizumab will be administered at a dose of 200 mg by IV infusion. Best overall response rate is the primary endpoint of the trial.