Mouse studies of siponimod — a potential progressive multiple sclerosis (MS) treatment that’s up for approval in the U.S. and EU — were among presentations given by Novartis at the 34th European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), held Oct. 10-12 in Berlin.
Animal work might seem an odd entry at a major science conference, especially when the investigative treatment it concerns has gone through a pivotal Phase 3 trial and is under regulatory review.
But siponimod is pioneering, said Dan Bar-Zohar, MD, global head of neuroscience development at Novartis, regardless of whatever is decided by the U.S. Food and Drug Administration (FDA) in March, and the European Medicines Agency (EMA) possibly in December 2019. He spoke in an interview with Multiple Sclerosis News Today at the conference.
It’s “the first disease modifying therapy … that has shown for a first time efficacy in a very typical secondary progressive MS population,” Bar-Zohar said, referring to results from the Phase 3 EXPAND study (NCT01665144) that compared oral siponimod, at 2 mg a day, with a placebo in 1,651 secondary progressive MS (SPMS) patients with moderate to severe disability across 31 countries.
Results — previously reported — showed a confirmed 21% lower rate of disability progression in treated patients at three months, and 26% at six months, Bar-Zohar said. But equally important, they did so in people more representative of the SPMS population — those starting treatment in their 40s and 50s, not 30s, with low relapse rates, and mean EDSS scores (disability scale) “of 5.4, not 2.4,” he added. Of note, the higher the EDSS score, the worse the patient’s disability level.
“Now, the fact that we’re talking about secondary progressive MS, and the complexity of secondary progressive MS, make us extremely keen to understand the mechanism of action of the drug, particularly when it comes to its central effects,” he said.
“We are trying to build a story around, and understand, what this molecule — siponimod — does inside the central nervous system, which is relevant to the very remarkable efficacy we’ve seen in secondary progressive MS,” Bar-Zohar added.
Building that understanding is where the animal work comes in, allowing researchers to see effects on brain and spinal tissue in ways impossible in patients. It’s not perfect, due to differences in translating results in the EAE mouse model to people, but it helps to explain the “changes in brain volume loss” seen in trial patients, Bar-Zohar said.
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