Disappointing Results for Fingolimod Phase III Trial In Primary Progressive MS

Novartis International recently provided an update on its fingolimod Phase III trial evaluating the use of the drug in primary progressive multiple sclerosis (PPMS), reporting that the study did not reveal any significant benefits of prescribing fingolimod over a placebo. However, results regarding safety for the drug were consistent with the safety profile of fingolimod in treating patients with relapsing MS (RMS).

PPMS is a disease that attacks the central nervous system (CNS), and it affects about 10 percent of 2.3 million people already diagnosed with multiple sclerosis worldwide. PPMS is different from relapsing MS because the process of the disease itself is distinct — there are almost no acute relapses, and there are very few active MRI lesions. In PPMS patients, severe and irreversible damage occurs in the CNS; the scientific community thinks that it occurs because of the rapid, continuous loss of nerve cells through different neural pathways. As a result, this form of MS dramatically affects patients’ lives from its onset.

In addition, the disease is usually diagnosed later than other types of MS, which means that when the diagnosis is made, substantial damage to the CNS has already occurred. Researchers are focused on understanding the mechanisms of the disease but, despite that, there are no approved treatments to stop the disease from progressing or to manage the symptoms.

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The study was based on the knowledge that fingolimod penetrates into the CNS and interacts with the cells that cause neural damage in PPMS. Since this effect is clear in relapsing forms of MS it was thought that it could be relevant in PPMS too. Nevertheless, the action mechanisms of these two forms of MS are different.

Vasant Narasimhan, Global Head of Development at Novartis Pharmaceuticals said in a press release: “We understand this news is very disappointing for those affected by PPMS and involved in its management. While PPMS is a focus of the MS community, relatively little is known about the disease so finding effective treatments remains a challenge. We will actively work with the MS community to review and analyze the INFORMS results to help increase the understanding of this devastating disease (…) 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.”