When it comes to clinical trials, things rarely go to plan. Just ask Roche, which revealed today that it has pushed back the regulatory timeline for its Duchenne muscular dystrophy (DMD) treatment by a year. In addition, the Big Pharma noted that the conflict in Ukraine has thrown its multiple sclerosis trials into disarray.
Roche had already bet big bucks on its DMD pipeline, stumping up $1 billion in 2019 to buy the ex-U.S. rights to the investigational drug SRP-9001. Despite facing a phase 2 trial failure in 2021, as of February Roche had been expecting to ask for approval in 2023.
But the company’s latest earnings presentation, published today, noted the submission date as 2024.
RBC analyst Brian Abrahams pointed out that Roche’s filing guidance reflects the company's plans in Europe—where it owns the rights— and not necessarily Sarepta’s plans for the U.S.
“We had the opportunity to speak with Sarepta's management this morning, and importantly, they said this does not reflect any changes to the expected timelines for the [phase 3] EMBARK readout or new FDA regulatory requirements,” Abrahams said in a note. “We continue to expect [phase 3] data around mid-2023.”
In an earnings call with investors, Roche Pharmaceuticals' CEO Bill Anderson discussed the phase 3 DMD trial, saying “We hope to have it accrued this year, which means it’s a 12-month endpoint, which would mean we would have a final result from the phase 3 by the end of 2023.”
MS trials suffer in Eastern Europe
A more significant trial complication came in the form of Russia’s war in Ukraine, which has forced Roche to think on its feet to keep its MS trials on track.
A relatively high proportion of patients with the neurological condition come from Russia and Ukraine, Anderson pointed out in the earnings call. Roche’s late-stage trials in this space are focused on Ocrevus in primary progressive and relapsing MS as well as the investigational BTK inhibitor fenebrutinib.
With between 20% and 30% of patients in these trials based in these two countries, Roche has had to open new sites in other countries to continue the studies, Anderson explained.
“In the meantime, we’re doing everything possible to make sure that we can continue to treat and monitor the patients that have already been enrolled,” he said. “But in terms of new patient enrollment, that’s a challenge.”