The presentation will be April 26. It will be titled “A Long-Term, Open-Label Follow-Up Study of Olesoxime in Patients with Type 2 or Non-Ambulatory Type 3 Spinal Muscular Atrophy who Participated in a Placebo-Controlled Phase 2 Trial“.
Olesoxime is an oral compound designed to protect the health of movement nerve cells. Preclinical-trial studies have shown that daily doses can extend the survival of mice with severe SMA better than a placebo.
But the therapy maintained patients’ movement function for two years, while the function of those treated with a placebo declined.
The Phase 2 OLEOS trial (NCT02628743) is evaluating olexoxime’s long-term benefit to Type 2 and non-ambulatory Type 3 SMA patients. The trial involves 125 patients who had been treated with olesoxime in the previous Phase 2 trial.
In the expansion study, participants are receiving 10 mg/kg of olesoxime once or twice a day for five years or until they develop serious toxicity to treatment.
The trial’s primary objective is to see if olesoxime is safe. The secondary objective is to see if its improves patients’ Motor Function Measure scores.
One hundred four of the 125 patients have been taking it for 12 months. So far, it appears safe — in line with previous reports.
When the previous Phase 2 trial ended, patients experienced a significant decline in movement function, researchers said. A year of treatment under the current trial showed that olesoxime had prevented their function from declining further.
“These data suggest that olesoxime offers the potential to provide meaningful clinical benefit and may play a role in the future therapeutic management of SMA,” the researchers wrote.
Olesoxime has received an orphan drug designation as an SMA treatment from both the European Medicines Agency and U.S. Food and Drug Administration. In April 2016, the European Medicines Agency changed the clinical trial plan for investigating olesoxime in children.