Roche announced positive results for three pivotal Phase III studies of experimental MS therapy ocrelizumab in relapsing multiple sclerosis and primary progressive multiple sclerosis (PPMS) patients at this year’s ECTRIMS 2015 conference. The results, particularly for treating PPMS, indicate that the novel therapy may represent a viable therapeutic option for progressive forms of the disease.
Multiple sclerosis (MS), a progressive neurodegenerative disorder, has no cure and is estimated to affect more than 2.3 million people worldwide. Relapsing Multiple Sclerosis is the most common type of MS, affecting approximately 85% of all patients with the disease. Primary progressive MS (PPMS) is characterized by escalating worsening symptoms and affects 1 in every 10 MS patients. No approved treatments currently exist for PPMS.
Ocrelizumab, an investigational, humanized monoclonal antibody was designed to target a selective group of immune cells — CD20-positive B cells — which have been shown to damage myelin, the protective layer that covers neurons and that once destroyed leads to motor function impairment, irreversible neurological disability and paralysis.
The therapy was tested in three major clinical trials – OPERA I, OPERA II and ORATORIO. The OPERA I and OPERA II studies tested ocrelizumab in patients with relapsing MS, which showed increased efficacy in reducing three key markers of disease activity when compared to interferon beta-1a (Rebif®), a well-established MS therapy.
The ORATORIO study focused on patients with PPMS where ocrelizumab significantly reduced disease progression — particularly patients’ disability — for at least 12 weeks (primary endpoint), when compared to a placebo control group. Additionally, ocrelizumab achieved positive results in other parameters (secondary endpoints), including reducing the time required to walk 25 feet, the volume of chronic inflammatory brain lesions and brain volume loss.
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Sandra Horning, M.D., Roche’s Chief Medical Officer and Head of Global Product Development commented on ocrelizumab’s major breakthrough: “The results of these three pivotal trials have the potential to transform the treatment of MS. Ocrelizumab is the first investigational medicine to significantly reduce disability progression in people with relapsing MS and people with primary progressive MS – a form of MS with no approved treatments. We are eager to work with regulatory authorities to bring this investigational medicine to the MS community as soon as possible.”
Stephen Hauser, M.D., Chair of the Scientific Steering Committee of the OPERA studies and Chair of the Department of Neurology at the University of California San Francisco School of Medicine added, “These results redefine our understanding of MS by highlighting the central role of the B cell. The findings may also encourage the MS community to look more closely at earlier treatment of the disease. Currently, many doctors reserve what are considered highly effective MS medicines until a patient’s disease becomes more advanced. Patients and their doctors need new treatment options that offer the potential for greater efficacy than a standard-of-care interferon with a similar safety profile.”
Xavier Montalban, M.D., Ph.D., Chair of the Scientific Steering Committee of the ORATORIO study and Professor of Neurology and Neuroimmunology at Vall d’Hebron University Hospital and Research Institute, Barcelona, Spain also noted, “This is an important moment for the MS community. For decades, trial after trial has failed to show the benefit of any medicine for people with primary progressive MS. Now, for the first time, we have a positive Phase III study result for people with this debilitating form of the disease.”
Roche will now seek marketing authorization for ocrelizumab in both relapsing MS and PPMS.