Micromet receives FDA feedback on blinatumomab registration for R/R ALL

Oct 14 2011

Micromet, Inc. (NASDAQ: MITI) today outlined a clinical development plan intended to support U.S. registration of its lead product candidate blinatumomab in patients with B-precursor relapsed/refractory (r/r) acute lymphoblastic leukemia (ALL). The plan incorporates advice received from the U.S. Food and Drug Administration (FDA) within the context of a Type C meeting.

"Recent feedback received from the FDA provides a roadmap to blinatumomab registration in a patient population with limited therapeutic options," said Christian Itin, Ph.D., Micromet's President and Chief Executive Officer. "We look forward to initiating later this quarter the first of two planned clinical trials collectively intended to support a biologics license application for full approval."

Global Phase 2 Single-Arm Trial in Adult Patients with R/R ALL

Later this quarter, the Company plans to initiate a single-arm Phase 2 clinical study that will enroll approximately 65 adult patients with B-precursor r/r ALL. Patients will receive blinatumomab for up to five treatment cycles. Each cycle will consist of 28 days of treatment followed by two weeks off therapy. The primary endpoint of the study is complete remission (CR) and complete remission with only partial recovery of blood counts (CRh). Secondary endpoints include duration of CR and CRh and overall survival. The study will be conducted at leading cancer centers in the U.S. and EU.

"In the event that initial results generated from the global Phase 2 trial are compelling, we plan to discuss with the FDA potential avenues to accelerate blinatumomab's path to market." said Jan Fagerberg, M.D., Ph.D., Micromet's Senior Vice President and Chief Medical Officer.

Global Randomized Controlled Trial in Adult Patients with R/R ALL

The Company also reviewed with the FDA the preliminary design of a planned randomized, controlled, Phase 3 clinical study intended to evaluate the efficacy and safety of blinatumomab in comparison to chemotherapy in adult patients with r/r B-precursor ALL. Efficacy will be assessed based on blinatumomab's effect on a time dependent endpoint. The study will be conducted at leading cancer centers in the U.S. and in Europe. Data from this trial, if positive, are intended to support full approval of blinatumomab in this patient population.

Global Phase 1/2 Single-Arm Trial in Pediatric Patients with R/R ALL

The Company recently received clearance from the FDA to conduct a Phase 1/2 clinical study in pediatric and adolescent patients with r/r ALL. This will be an open label two-part study, conducted in collaboration with the Children's Oncology Group and International BFM study group. The first part is designed to determine the appropriate dose and schedule of blinatumomab according to patient age group. Once the recommended dose and schedule is determined, additional patients will be enrolled in the second part of the study to further assess the safety and antitumor activity of blinatumomab. The Company expects to enroll approximately 80 patients at leading investigative sites in the U.S, Canada and EU. Data from this trial will inform the Company's registration strategy in this patient population.

Blinatumomab Clinical Experience in Adult R/R ALL

Interim results from a Phase 2 trial presented at the 2011 Meeting of the European Hematology Association show that blinatumomab produced a high CR rate in adult patients with ALL who had relapsed following treatment with standard therapy. 75% of patients (9 of 12) achieved a CR or CRh following treatment with blinatumomab. All nine responding patients achieved a complete molecular response, or had no evidence of leukemic cells in their bone marrow, a key prognostic factor for patient survival. Notably, four patients with genetic abnormalities typically associated with poorer outcomes all achieved a CR or CRh. The most common clinical adverse events were fever, peripheral edema and fatigue. Treatment of two of the twelve patients was interrupted due to fully reversible and manageable central nervous system (CNS) events.