On May 31, 2014 Roche reported results from a Phase I open-label study that showed the investigational cancer immunotherapy MPDL3280A (anti-PDL1) shrank tumours (overall response rate) in 43 percent (13/30) of people previously treated for metastatic urothelial bladder cancer (UBC) whose tumours were characterised as PD-L1 (Programed Death Ligand-1) positive by a test being developed by Roche (Press release Hoffmann-La Roche, MAY 31, 2014, View Source [SID:1234500672]). Adverse events (AEs) were consistent with what has been previously reported for MPDL3280A. There were no severe (Grade 4-5) treatment related AEs.
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The FDA has granted MPDL3280A Breakthrough Therapy Designation. This designation is designed to expedite the development and review of medicines intended to treat serious diseases and to help ensure patients have access to them through FDA approval as soon as possible.
"Bladder cancer is the ninth most common cancer worldwide, for which there have been no new treatment advances in nearly 30 years, so we are pleased the FDA has granted breakthrough designation for MPDL3280A in metastatic bladder cancer," said Sandra Horning, M.D., chief medical officer and head of Global Product Development. "We are evaluating MPDL3280A in a broad range of tumours, and have begun pivotal studies that include a companion diagnostic test in lung and bladder cancers."
Full results of the study will be presented today at the 50th Annual Meeting of the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) by Thomas Powles, M.D., clinical professor of Genitourinary Oncology, Barts Cancer Institute at the Queen Mary University of London, UK (Abstract #5011, Saturday, May 31, 3:36–3:48 P.M. Central Time).
About the Phase I MPDL3280A Study
The Phase I study is a single-arm, multi-center, open-label trial with a cohort of 68 people with previously treated, metastatic bladder cancer.
The study included 30 patients who were identified as PD-L1 positive (immunohistochemistry [IHC] 2/3) using an investigational PD-L1 diagnostic test being developed by Roche.
After six weeks of follow-up, the objective response rate (ORR) as measured by RECIST criteria was 43 percent (13/30), and after 12 weeks, ORR was 52 percent (13/25) in people with PD-L1-positive tumours.
A complete response (no radiographic evidence of tumour) was observed in 7 percent of PD-L1-positive patients (2/30).
The ORR was 11 percent (4/35) in people whose tumours were identified as PD-L1-negative (IHC 0/1) by our investigational test.
People in the study experienced a median time to response of 42 days.
Treatment-related Grade 3 AEs occurred in 4 percent (3/68) of people in the study and included weakness (asthenia; 2 percent), low platelet count (thrombocytopenia; 2 percent) and low phosphate levels (blood phosphorus decrease; 2 percent).
The most common AEs observed to date occurring in more than 5 percent of patients were decreased appetite (12 percent), fatigue (12 percent), nausea (12 percent), fever (pyrexia; 9 percent) and weakness (asthenia; 7 percent).