Novartis announced this week that its fingolimod (Gilenya) phase III INFORMS trial in primary progressive multiple sclerosis (PPMS) failed to meet the primary endpoint.
Fingolimod, marketed as Gilenya and approved in the U.S. for the treatment of relapsing multiple sclerosis (RMS), was no more effective than placebo on a combination of disability measures in patients with PPMS. The safety profile was comparable to the safety profile of fingolimod in RMS.
The INFORMS study, the largest clinical trial ever in PPMS, was based on a hypothesis that fingolimod, which interacts with damage-causing cells in the central nervous system in RMS, would have a comparable effect in PPMS. The primary endpoint evaluated the effect of fingolimod versus placebo on reducing the risk of three-month sustained disability progression based on a composite measure of the Expanded Disability Status Scale, upper limb function, and walking speed. However, the results of the study suggest that PPMS and RMS have different underlying mechanisms.
“We will actively work with the MS community to review and analyze the INFORMS results to help increase the understanding of this devastating disease,” said Vasant Narasimhan, Global Head of Development at Novartis Pharmaceuticals, in a news release. “Gilenya (fingolimod) revolutionized the treatment of relapsing MS as the first oral disease-modifying therapy. We remain strongly committed to continuing to research new treatment options for patients with MS and other neurological conditions.”
The results are disappointing for a community of patients, clinicians, and researchers working hard to understand the disorder, which affects about 10% of the 2.3 million MS patients globally. Unlike RMS, PPMS lacks acute relapses and presents with fewer brain lesions than what is seen in RMS and other types of MS, making it more difficult to diagnose. Loss of nerve cells and function occur rapidly and steadily over time, severely increasing disability and dependence of patients compared to other types of MS.
- News Release: Novartis provides update on fingolimod phase III trial in primary progressive multiple sclerosis (PPMS). Available here: http://www.novartis.com/newsroom/media-releases/en/2014/1875463.shtml