Positive new data for Roche’s Evrysdi in largest trial ever

• New two-year Evrysdi data show improvement or maintenance of motor function in people with SMA, a progressive neuromuscular disease that can be fatal
• The JEWELFISH study enrolled the broadest and most diverse patient population ever studied in an SMA trial
• Longer-term safety data consistent with that previously seen in earlier trials and low study drop-out rate
• Evrysdi has proven efficacy in babies, children and adults, with more than 7,000 patients treated to date worldwide

Basel, 12 October 2022- Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new two-year data from the JEWELFISH study evaluating Evrysdi® (risdiplam) in people with Type 1, 2 or 3 SMA aged 6 months to 60 years at time of enrolment. Patients had been previously treated with other approved or investigational SMA-targeting therapies, including nusinersen (Spinraza^(R)) or onasemnogene abeparvovec (Zolgensma^(R)). Data showed Evrysdi improved or maintained motor function and led to rapid increases in SMN protein levels which were sustained after 2-years of treatment. These data will be presented at the 27^th World Muscle Society (WMS) congress, 11-15 October 2022.

“The consistent safety profile and exploratory efficacy we have seen in the JEWELFISH study, the largest ever conducted in previously treated patients, reinforces Evrysdi as a meaningful treatment option across SMA populations,” said Dr. Claudia Chiriboga, Professor of Neurology and Pediatrics, Department of Neurology, Columbia University Medical Center, New York, USA. “The findings add to our confidence when making treatment decisions for previously-treated patients in need.”

The JEWELFISH study enrolled the broadest and most diverse patient population ever studied in an SMA trial. Of the 174 people enrolled, 36% (n=63) were adults, 63% (n=105) had a Hammersmith Functional Motor Scale Expanded (HFMSE) score of less than 10 at baseline, meaning their disease was very severe, and 83% (n=139) had scoliosis. Forty-four…