PPMS Study Does Not Meet Primary Endpoint
On December 1, 2014, Novartis announced that their Phase III INFORMS trial with Gilenya® (fingolimod) for individuals with primary-progressive multiple sclerosis (PPMS) did not meet its primary endpoint. Presently, this disease-modifying therapy, taken orally (by mouth), is approved for the long-term treatment of relapsing forms of MS. The hope was that Gilenya would also prove effective in treating PPMS, a less-common form of MS that presently has no approved treatments available to slow disease activity.
As noted in MSAA’s 2014 edition of the MS Research Update, “Although Gilenya was approved for RRMS [relapsing-remitting multiple sclerosis] in 2010, several large clinical trials of this medication are still ongoing. The 36-month INFORMS study will evaluate the effect of Gilenya relative to placebo on delaying the time to sustained disability progression in patients with PPMS. It will also evaluate safety, tolerability, and the effects on MRI parameters. As there is presently no FDA-approved medicine for PPMS, this is an important study for the field.”
According to a press release from Novartis, the INFORMS study is the largest clinical trial ever conducted in PPMS, enrolling 970 people aged 25-69 years with PPMS. The double-blind, randomized, and placebo-controlled study was conducted at 148 sites across 18 countries, including the United States, Canada, Australia, and several European countries.
Patients were treated for at least three years and the primary endpoint was to evaluate the effect of Gilenya versus placebo on reducing the risk of three-month sustained disability progression based on a composite measure of several standard tests used in evaluating some of the physical effects of MS. These included the Expanded Disability Status Scale (EDSS), an assessment of upper limb function using the 9-Hole Peg Test (HPT), and walking speed as measured by the 25-foot Timed Walk Test (TWT).
Unfortunately, the initial results of the study did not show a significant difference between the group taking Gilenya and the placebo group on these disability measures. However, additional analysis of the data will reveal if any subgroups of individuals with PPMS experienced positive effects from this medication over the three years. Novartis also notes that the safety results were consistent with the safety profile seen with Gilenya in relapsing forms of MS.
MSAA’s Chief Medical Officer Dr. Jack Burks comments, “While the results of the INFORMS trial were not what we had hoped for, further study of the trial’s data may yield important details in regard to the mechanisms involved with PPMS, which may aid in the design of future studies and potential treatments. Additionally, data from magnetic resonance imaging (MRI) may show some results that occurred during this study, so we look forward to hearing further details as they become available. In the meantime, this does not change the usefulness of this medication as a treatment for relapsing forms of MS, and the safety information should also be reassuring to individuals presently taking this disease-modifying therapy. Of particular interest is the fact that the individuals with PPMS who were taking Gilenya in the INFORMS trial were older than the participants in the earlier studies for relapsing forms of MS. These older patients tolerated the medication very well, similar to the younger ones, and these results should be reassuring to older individuals already taking or considering this therapy.”
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Written by Susan Wells Courtney
Reviewed by Jack Burks, MD, MSAA Chief Medical Officer