Roche unveils long-term data from pivotal spinal muscular atrophy trial

21 Mar 2023
endpts.com
Phase 3Clinical Result
Biogen and Novartis have previously rolled out long-term data with their respective spinal muscular atrophy therapies Spinraza and Zolgensma. Now, Roche is seeking to catch up with four-year data for its blockbuster risdiplam, aka Evrysdi. Presenting at the Muscular Dystrophy Association Clinical and Scientific Conference, the pharma giant’s subsidiary Genentech unveiled positive data Monday in patients ages 2 to 25 from the SUNFISH pivotal study that had either type 2 or type 3 SMA. In short, Genentech said that its spinal muscular atrophy drug showed continued efficacy in patients four years after starting the therapy. Those data demonstrated a sustained increase in motor function after 48 months, in line with the increase in function during the first year of the study and measured by changes in both MFM-32 and RULM. Genentech wrote in a statement that naturally, patients with either type 2 or 3 SMA typically have a decline in motor function. The study also reported safety data, saying that the most common adverse events were upper respiratory tract infection, fever and headache. Genentech also said that the rate of adverse events continued to drop — a reference to SUNFISH data released last spring. Roche said in its three-year data from the SUNFISH trial last spring that patients maintained an increase in the MFM-32 and RULM measures, additionally reporting lesser side effects over time. Genentech did not immediately respond to requests for comment from Endpoints News . Roche also rolled out Phase III data last fall in a wider range of patients — from six months of age all the way to 60 years old, and all were previously treated with Spinraza or Zolgensma. Those data showed that risdiplam improved or maintained motor function, on top of an increase in survival motor neuron (SMN) protein after two years of treatment. The study also suggested that maintenance of motor function was sustained at the two year mark. The therapy modifies the splicing of survival motor neuron 2, increasing and sustaining the production of SMN protein in the central nervous system and surrounding tissues. That protein is necessary for healthy motor neurons. Roche first got its hands on the therapy back in 2011, when it made a deal with PTC TherapeuticsPTC Therapeutics for its SMA program. In exchange for a worldwide license, Roche paid $30 million upfront, with the potential of another $460 million in milestones and additional royalties on commercial sales.
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