Roche announces 2-year risdiplam data from SUNFISH and new data from JEWELFISH in infants, children and adults with spinal muscular atrophy (SMA)

• SUNFISH Part 1 showed risdiplam significantly improved motor function after 24 months of treatment in people aged 2-25 years with Types 2 or 3 SMA
• JEWELFISH study preliminary 12 month data in previously treated patients showed rapid and sustained increases in SMN protein levels
• Safety in SUNFISH and JEWELFISH was consistent with the safety profile observed to date and no new safety signals were identified
  
  

Basel, 12 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today presented two-year data from Part 1 of its pivotal SUNFISH trial in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA) at the virtual Cure SMA Annual Conference, 8-12 June, 2020. The results of an exploratory efficacy analysis show risdiplam significantly improved motor function after 24 months of treatment compared to natural history data. In addition, preliminary 12 month data from JEWELFISH, a trial in people with all types of SMA aged 6 months to 60 years previously treated with other SMA therapies, showed that treatment with risdiplam led to rapid and sustained increases in SMN protein levels. No new safety signals were observed and the overall adverse event profile was consistent with that of treatment-naive patients.

“These 24 month exploratory data are important as they are consistent with the medically meaningful results we saw after one year in Part 2 of the SUNFISH study, designed to represent a broad, real-world SMA population,” said Levi Garraway, M.D., Ph. D., Roche’s Chief Medical Officer and Head of Global Product Development. “We are also encouraged to see an increase in SMN protein levels across both the Sunfish Part 1 and Jewelfish studies. These data reinforce the potential of risdiplam to make a real difference in the lives of the many people living with SMA.”

SUNFISH is a large (n=231) global two-part study in children and adults. The dose-finding SUNFISH Part 1 (n=51) includes a broad patient population ranging from individuals unable to sit to those capable of walking, as well as people with scoliosis or joint contractures.

The exploratory efficacy analysis of Part 1 of the SUNFISH study assessed motor function, using the Motor Function Measure (MFM) scale. MFM is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. It assesses different motor functions from standing and walking through to use of hands and fingers. In a weighted analysis comparing the data with a robust natural history  comparator cohort, MFM total change from baseline at Month 24 was greater in patients receiving risdiplam (3.99 point difference (95% CI: 2.34, 5.65) p< 0.0001). Even small changes in motor function can result in meaningful differences to daily living.