Roche has halted development of olesoxime, the Phase III–bound spinal muscular atrophy (SMA) candidate it acquired three years ago when it bought Trophos.
The company disclosed its decision in a letter Wednesday to SMA patient groups.
“Since we bought olesoxime from Trophos in 2015, we have had many difficulties in developing this molecule for people with SMA,” Sangeeta Jethwa, M.D., Roche’s Head, Patient Partnership, Rare Diseases, said in the letter, dated May 30 and posted by Germany-based patient group SMA Europe on its website.
These difficulties, according to Roche, have included the liquid preparation of olesoxime, the most appropriate dose to be given, and requests from the FDA and European Medicines Agency that the company launch an additional Phase III study.
“We understand the urgency of finding solutions for the SMA community and we have always tried to overcome the difficulties with olesoxime, in the hope of starting a Phase 3 study later this year,” Roche stated.
“Unfortunately, despite all of our efforts and a strong desire to deliver olesoxime as a medicine to people with SMA, we have concluded that this is not going to be possible. Based on all of the available evidence and the continued difficulties described above, we have decided to stop further development of olesoxime.”
Roche added olesoxime (formerly called TRO19622) to its pipeline when it acquired Trophos in 2015 for up to €470 million ($549 million).
Trumpeting Positive Results
As late as April of this year, Roche trumpeted positive results associated with olesoxime.
At the 2018 annual meeting of the American Academy of Neurology, held April 21–27 in Los Angeles, Roche presented results from the OLEOS (NCT02628743) open-label extension study designed to assess the long-term safety and efficacy of olesoxime in patients with Type 2 or nonambulatory Type 3 SMA. The extension study followed up on an earlier Phase II study (NCT01302600) in patients aged 3 to 25 years with Type 2 or nonambulatory Type 3 SMA.
According to the results, maintenance of motor function seen over two years in the Phase II study was followed by a substantial decline in motor function measure (MFM) D1 + D2 (>2 points/year) after patients discontinued the drug. However, the ~2-point MFM treatment difference between olesoxime and placebo at the end of Phase II was maintained at OLEOS baseline. Also, olesoxime open-label treatment stabilized motor function, with a mean change in MFM D1 + D2 from baseline of −0.03 at six months, and of −0.22 at 12 months.
“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 long-term, open-label, follow-up study concluded, according to an abstract that reported the results.
The U.K. SMA patient group TreatSMA issued a response to Roche’s decision stating that it was “disappointed that olesoxime, a drug that once held a lot of promise for the entire SMA community, will no longer see the light of day.”
“We remain hopeful that the promise offered by the new treatments that have since been developed will make our dream of an effective therapy a reality,” TreatSMA added.