Ocrelizumab has become the first drug treatment to show positive results in primary progressive multiple sclerosis (MS) following phase 3 clinical studies. Previously, all phase 3 studies with MS medication have been negative.
The three studies, presented at the European Committee for Treatment Research in Multiple Sclerosis (ECTRIMS) congress, demonstrate that treating MS with ocrelizumab reduces relapse rate and delay onset of disability progression compared to interferon beta-1a (Rebif) in relapsing forms of MS (RMS) (OPERA I and OPERA II studies) and disability progression in primary progressive MS(PPMS) (ORATORIO study) compared to placebo.
Ocrelizumabis selectively targets B-cells, one of the two immune cells implicated in the inflammatory and neurodegenerative process of MS. MS affects about 100,000 people in the UK.
Professor Gavin Giovannoni, Chair of Neurology at the Blizard Institute, Barts and The London School of Medicine and Dentistry, said: “The phase 3 ocrelizumab results for both PPMS and RMS, in my opinion, are a game changer for the clinical community. These data demonstrate that B-cell targeting can significantly modify the disease, which in effect means a more positive outlook for patients. The important next step is for regulators to enable the use of ocrelizumab across the spectrum of PPMS and RMS, and for treatment to be provided as soon after diagnosis as possible to provide optimal outcomes, with the potential to improve patients’ quality of life in the long-term.”
The phase 3 ORATORIO study in PPMS included five UK sites and met its primary endpoint in this progressively disabling form of MS, which currently has no approved treatments. Top-line results being presented as a late-breaking abstract at ECTRIMS have shown a reduction in the progression of clinical disability with ocrelizumab of 24% compared with placebo sustained for at least 12 weeks, as measured by the Expanded Disability Status Scale (p=0.0321).
Also presented at ECTRIMS were the phase 3 OPERA I and II studies, which included six study sites in the UK. For patients treated with ocrelizumab, the reduction in frequency of their relapses (known as annualised relapse rate (ARR)) was 46% (p=0.0001) in OPERA I and 47% (p=0.001) in OPERA II compared to patients treated with interferon beta-1a.
Ocrelizumab also significantly delayed the progression of clinical disability with a relative risk reduction of 40% (p=0.006) across the two studies (OPERA I: 43%, p=0.0278; OPERA II: 37%, p=0.0370) compared to interferon beta-1a.
This is the first time a single treatment has met this endpoint in two separate phase 3 studies.
Dr Dan Thurley, Medical Director of pharmaceutical company Roche UK, said: “This pioneering science redefines our understanding of the underlying biology of MS – with a clear focus on the role of B-cells in the disease. This is the result of our investment in following the science and we look forward to continuing further research of new treatment options for patients with chronic and potentially devastating diseases. We are excited to be re-entering the neuroscience space with the aim to improve the lives of patients.”