New Genentech data for Evrysdi show improved motor function in pre-symptomatic babies after one year and confirm safety profile in previously treated people with spinal muscular atrophy.

Interim exploratory efficacy data from JEWELFISH also suggest stabilization in motor function at one year of treatment as measured by change from baseline in motor function measure (MFM-32). A recent survey from SMA Europe showed that almost 97% of people living with SMA reported disease stabilization as progress. Preliminary efficacy data from RAINBOWFISH, an ongoing open-label study evaluating Evrysdi in babies from birth to 6 weeks with pre-symptomatic SMA, showed that infants treated for 12 months achieved age-appropriate motor milestones, including sitting, standing and walking, and improvements in motor function. These data will be presented at the 2021 Virtual SMA Research & Clinical Care Meeting from June 9-11, 2021. The JEWELFISH study enrolled the broadest patient population ever studied in an SMA trial, including patients with SMA Types 1-3 who received prior treatment across a wide range of ages and disease severities. Of the 174 people enrolled, 30% were teenagers and 35% adults, 62% had a HFMSE score of less than 10 at baseline, 80% had scoliosis and 47% had undergone scoliosis surgery. Seventy-six people had previously been treated with nusinersen and 14 with onasemnogene abeparvovec. Evrysdi led to a sustained greater than 2-fold increase in median SMN protein levels versus baseline in all patients who received prior treatment, irrespective of which treatment was previously received or SMA type. The overall AE profile of Evrysdi treatment in pre-treated patients was consistent with that of treatment-naïve patients in FIREFISH and SUNFISH. The most common adverse events in all patients were upper respiratory tract infection (17%), pyrexia (17%), headache (16%), nausea (12%), diarrhea (11%), nasopharyngitis (10%) and vomiting (8%). The most common serious adverse events were pneumonia (2%), lower respiratory tract infection (2%), upper respiratory tract infection (2%) and respiratory failure (2%). There were no treatment-related adverse events leading to withdrawal or treatment discontinuation in JEWELFISH, with some patients receiving treatment for more than 3 years. The study is ongoing and the primary analysis will be conducted at month 24. “Data from the JEWELFISH study, which included a diverse patient population with a high degree of motor impairment, show that Evrysdi has a favorable safety profile in patients previously treated with an SMA-targeting therapy,” said Dr. Claudia Chiriboga, Professor of Neurology and Pediatrics, Department of Neurology, Columbia University Medical Center, New York. “Importantly, the data also suggest a stabilization of motor function in trial participants. As a progressive disease, untreated patients with SMA typically show a decline in motor function over time.” Roche also presented preliminary efficacy data from RAINBOWFISH , which showed that of the 5 babies treated with Evrysdi for at least 12 months, all achieved sitting without support, rolling and crawling. Of the 5, 2 had 2 SMN copies and 3 had greater than 2 copies. Four of the infants were able to stand unaided and walk independently. In addition, 4 babies reached a maximum score of 64 on the CHOP-INTEND scale, and 1 scored 63. Data on the primary endpoint, the number of infants sitting without support for at least 5 seconds, will be reported when all primary analysis patients have reached one year of treatment. Recruitment for RAINBOWFISH is ongoing. The most common adverse events were nasal congestion (33%), cough (25%), teething (25%), vomiting (25%), eczema (17%), abdominal pain (17%), diarrhea (17%), gastroenteritis (17%), papule (rash; 17%) and pyrexia (fever; 17%). There were no adverse events leading to withdrawal or study discontinuation.