Drug Development

Roche dumps its late-stage SMA drug after gambling up to $545M-plus on the program

After running into repeated setbacks with its experimental drug for spinal muscular atrophy, Roche has opted to scrap the therapy it grabbed three years ago with a $545 million buyout deal.

Sangeeta Jethwa

In a note to a patients group — a favorite avenue for the Swiss pharma giant — a Roche representative explained that regulators at the FDA and the EMA were demanding a new Phase III study of olesoxime. That followed issues with manufacturing and their analysis of the OLEOS study which “actually showed a worsening in motor function” among patients on the drug.

Roche paid $140 million upfront for the company and the drug, with more than $400 million on the table in milestones.

The OLEOS study is now being scrapped and Roche’s Sangeeta Jethwa, head of their patient partnership for rare diseases, had this to say to TreatSMA:

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.

In a statement to Endpoints News, the company added:

Since acquiring Olesoxime from Trophos in 2015, we have undertaken preclinical studies, consulted with health authorities and experts, reformulated the investigational molecule, performed bioavailability studies to optimize dose, and attempted to optimize the study design to enable a phase III trial.

It is with regret that we now conclude that we do not have the necessary body of clinical and scientific evidence to support further development in the new SMA landscape.

We remain committed to SMA. Our investigational medicine RG7916, an oral SMN2 splicing modifier that is systemically distributed throughout the body, is currently being evaluated in three multi-centre Phase II /III trials, SUNFISH, FIREFISH and JEWELFISH.

It couldn’t have come as a complete surprise. The drug — acquired in Roche’s acquisition of Trophos — had failed an earlier mid-stage study, focusing on motor function. And even before the pharma giant bought the biotech, the drug had failed a trial for ALS.

Roche isn’t getting to the finish line on SMA, but it’s sounding a retreat at a time of progress in the field. Biogen and Ionis scored an approval for Spinraza, and promptly made it one of the most expensive therapies in the world, with a starting price of $750,000 for the first year. AveXis, meanwhile, has begun clinical work on a gene therapy for SMA, and others are also jumping in.

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Recursion Pharmaceuticals Salt Lake City, UT
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Atlas Venture Cambridge, MA

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