Olesoxime (TRO19622) is an investigational therapy, initially developed by the biopharmaceutical company Trophos, that is designed to protect the health of motor nerve cells. The use of olesoxime in treating spinal muscular atrophy patients has shown success in stabilizing motor functions in non-ambulant SMA Type 2 and 3 patients in Phase 2 trials.

The therapy, which was acquired along with Trophos by Roche in early 2015, has been granted orphan medicinal product designation for the treatment of SMA by the European Medicines Agency, and orphan drug designation by the U.S. Food and Drug Administration. In April 2016, the EMA published a decision regarding changes to a pediatric investigation plan for olesoxime.

How Olesoxime Works

Olesoxime is part of a cholesterol oximes family, with neuroprotective features that helps to slow the progression of SMA, preserving mitochondrial function. The therapy’s exact mechanism of action is not completely understood, though preclinical studies suggests that it can promote myelination and decrease mitochondrial membrane fluidity in cell and animal models.

Clinical trials for olesoxime

Olesoxime was developed to treat amyotrophic lateral sclerosis (ALS), but it did not show the efficacy in ALS that it has in SMA patients.

The safety and efficacy of olesoxime was studied in a Phase 2 (NCT01302600), multicenter, randomized, adaptive, double-blind, placebo-controlled clinical trial in 3- to 25-year-old SMA patients, which finished in October 2013. Another Phase 2 trial is underway and a Phase 3 study in Type 2 and 3 SMA patients is planned for 2017 to establish clinical evidence of olesoxime’s benefit-risk profile as an SMA treatment. (The completed Phase 2 trial results showed a beneficial effect in the maintenance of neuromuscular function in SMA patients, as well as a reduction in medical complications associated with the disease, according to a release by Cure SMA. But the evidence was not considered sufficient for a “conclusive determination” by regulatory agencies, Roche announced.)

A new Phase 2 study (NCT02628743) to evaluate the long-term safety, tolerability and effectiveness of olesoxime in patients with SMA is now recruiting participants from previous studies in Europe;more information is available on this study’s clinic trials.gov website.

Next Steps for Olesoxime

Olesoxime is part of a group of 14 new molecular entities from Roche that are currently in a late-stage development program, but regulatory filings for olesoxime won’t begin before 2020, after conclusion of the ongoing Phase 2 and, possibly, the planned Phase 3 study.

Review the current list of clinical trials for Olesoxime at clinicaltrials.gov.

Read the latest Olesoxime news.

Follow development of experimental Spinal Muscular Atrophy drugs on the SMA Therapy Tracker.

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