Study results released this week show that an investigational drug, ocrelizumab, significantly reduces the progression of clinical disability in patients with primary progressive multiple sclerosis (PPMS).This study is a phase 3 study called ORATORIO and was conducted by Genentech, a member of the Roche Group. Ocrelizumab is a drug designed to target CD20-positive B cells. B cells are immune cells that are thought to be a key contributor to neurological damage in MS patients. "Ocrelizumab is the first investigational medicine to show a clinically meaningful and statistically significant effect on the progression of disease in primary progressive MS," Sandra Horning, MD, Roche's chief medical officer and head of global product development, said in a press release. This study evaluated the efficacy and safety of ocrelizumab through a randomized, double-blind, multicenter study. It compared ocrelizumab with placebo in 732 patients with PPMS. Its primary endpoint, which was met, was time to onset of confirmed disability progression, defined as an increase in Expanded Disability Status Scale (EDSS) that is sustained for at least 12 weeks. The safety and efficacy of ocrelizumab has also been evaluated with promising results in Phase III clinical in patients with relapsing forms of MS. In those trials, ocrelizumab treatment significantly reduced the annualized relapse rate, the progression of clinical disability assessed by EDSS, and the number of MS lesions in the patient’s brain in comparison to interferon beta-1a treatment. “These positive results of this study are a critical development for MS patients, and they further validate our hypothesis that B cells are central to the underlying biology of the disease,” explains Dr. Timothy Vollmer, Rocky Mountain MS Center Medical Director and Co-Director of Rocky Mountain MS Center at University of Colorado. Once approved by the FDA, ocrelizumab will become an important treatment option for patients with either relapsing or progressive forms of MS, which is a major advancement in the treatment of MS. Top-line data from this latest pivotal study will be presented as a late-breaking abstract at the 31st congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) by Xavier Montalban, M.D., Ph.D, in Barcelona, Spain on October 10th.
According to their press release, Genentech plans to pursue marketing authorization for ocrelizumab in relapsing MS and in PPMS. Data from the three Phase 3 clinical trials will be submitted to the U.S. Food and Drug Administration in early 2016.