Swiss specialty pharmaceutical company Santhera Pharmaceuticals has reported positive results from a Phase I/II clinical trial evaluating the safety and therapeutic efficacy of idebenone for the treatment of primary progressive multiple sclerosis (PPMS).

The investigator-initiated, double-blind, placebo-controlled trial (IPPoMS) was sponsored by the US National Institutes of Health (NIH).

The trial was conducted at NIH and has confirmed the safety profile of idebenone at a dose of 2,250mg daily over a treatment period of two years.

During the trial, no difference was found between the active treatment group and placebo while evaluating efficacy of idebenone on disease progression.

A total of 77 patients were randomised, while 66 completed the trial that combined a one-year observational pre-treatment phase, followed by a two-year placebo-controlled intervention period.

“The long-term study in patients with PPMS confirms the favourable safety profile of idebenone”

The trial’s primary outcome to explore the efficacy of idebenone was the change in CombiWISE, a rating scale recently developed by Bibiana Bielekova and colleagues at the National Institute of Neurological Disorders and Stroke (NINDS).

Santhera Pharmaceuticals CEO Thomas Meier said: “The long-term study in patients with PPMS confirms the favourable safety profile of idebenone given at higher dose than the currently approved dose for Raxone in Leber’s hereditary optic neuropathy.

“Clearly, the small sample size is a limitation when studying a therapeutic intervention in such a complex, relentlessly progressing neurological disease.”

Multiple sclerosis is an inflammatory and neurodegenerative disorder of the central nervous system that causes several physical symptoms, including impaired movement, fatigue, numbness, and pins and needles, as well as problems with memory and understanding.