Roche scraps €120M SMA drug after hitting ‘many difficulties’ 

Roche pulled the plug on olesoxime, the spinal muscular atrophy drug it acquired for €120 million upfront three years ago. (Roche)

Roche has stopped development of the spinal muscular atrophy (SMA) drug it acquired for €120 million ($140 million) upfront three years ago. The Swiss pharma is walking away from olesoxime after running into “many difficulties in developing” the oral mitochondria-boosting drug. 

In a statement to the SMA community shared online by TreatSMA, Roche walks through the issues that have stopped olesoxime from powering forward. The company has struggled with everything from formulation to dose selection and the demands of regulatory agencies for a phase 3 trial.

Roche spent several years working through these issues, only for data from a midphase trial to raise doubts about whether olesoxime is worth the hassle. The 18-month readout shared in April suggested the efficacy of olesoxime wanes over time. While the drug initially helped participants, by 18 months their motor function was deteriorating. 

The disappointing data look to have deterred Roche from persisting with olesoxime. In explaining the decision, Roche pointed to recent changes in the treatment options available to patients with SMA. When Roche acquired olesoxime, there were no approved drugs for SMA. Now, patients have access to Biogen and Ionis’ Spinraza and can look forward to experimental treatments including AveXis’ gene therapy.

That has changed both the commercial and clinical development environments for SMA drugs. 

“The emergence of effective treatment has raised the hurdle for how effective a new treatment needs to be, and this has an impact on how we design and run our clinical studies,” Sangeeta Jethwa, M.D., Roche's head of patient partnerships and rare diseases, wrote in a letter to the SMA community.

Faced with that level of competition, Roche has decided it makes little sense to struggle forward with a drug that may only offer marginal efficacy. Instead, it is switching attention from SMA to its oral SMN2 splicing modifier RG7916.

The decision means Roche’s gamble on olesoxime has failed to pay off. When Roche paid €120 million and committed €350 million in milestones to buy French biotech Trophos, olesoxime had already failed to move the needle in a phase 3 amyotrophic lateral sclerosis trial. That cost Trophos its chance of being bought by Alexion, but its pivot to SMA paid off four years later when Roche swooped in.