New two-year data show Roche’s Evrysdi (risdiplam) continues to demonstrate improvement or maintenance of motor function in people aged 2-25 with Type 2 or Type 3 Spinal Muscular Atrophy (SMA)

• SUNFISH Part 2 study population includes broad range of ages and disease severities, representing a real-world spectrum of people living with Type 2 or 3 SMA
• Evrysdi is the first and only at home SMA treatment approved by the FDA, and has proven efficacy across adults, children and infants 2 months and older
• More than 2,500 patients now treated with Evrysdi in clinical trial, compassionate use and real-world settings

Basel, 16 March 2021 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced new exploratory 2-year longer-term data from Part 2 of SUNFISH, a global placebo-controlled study evaluating Evrysdi™ (risdiplam) in people aged 2-25 years with Type 2 or non-ambulant Type 3 spinal muscular atrophy (SMA). The study suggests that gains in motor function observed with Evrysdi treatment at month 12 continued to improve or were maintained at month 24 across primary and secondary endpoint measures. Based on the natural history of the disease, people with Types 2 and 3 SMA who remain untreated decline in motor function over time. These data will be presented at the 2021 Muscular Dystrophy Association (MDA) Virtual Clinical & Scientific Conference taking place from March 15-18.

“These results build on the one-year findings from the SUNFISH trial and importantly show the durability of improvement or stabilization of motor function through two years of treatment,” said SUNFISH principal investigator Eugenio Mercuri, M.D., Ph.D., Department of Pediatric Neurology, Catholic University, Rome, Italy. “In addition, with no new safety signals identified, these second year results may support the favorable benefit-risk profile of Evrysdi over a longer period of time.”

Patients in SUNFISH Part 2 ranged in age from 2-25 and were treated with Evrysdi (n=120) or placebo and Evrysdi (n=60; patients in the placebo arm received placebo for 12 months followed by Evrysdi treatment for 12 months). The study evaluated a number of exploratory 24-month endpoints, which provide important insights into motor function and its impact on daily life. Findings demonstrated that Evrysdi:

• Maintained motor function improvements between months 12 and 24 as measured by Motor Function Measure (MFM-32)*.
• Increased motor function as measured by Revised Upper Limb Module (RULM)** and the Hammersmith Functional Motor Scale-Expanded (HFMSE)*** between months 12 and 24.
• Stabilized motor function for patients who began treatment with Evrysdi after 12 months of placebo as measured by MFM-32, RULM and HFMSE.
• Increased total score change from baseline, as measured by the caregiver-reported SMAIS**** upper limb module, and the patient-reported SMAIS score stabilized between months 12 and 24.

“These encouraging results confirm that the efficacy and safety of Evrysdi in people with Type 2 and Type 3 SMA can be sustained over time,” said Levi Garraway, M.D., Ph. D., Chief Medical Officer and Head of Global Product Development. “Therefore, these findings further highlight the potential longer-term benefit this first-of-its-kind medicine can have for people of varying ages and levels of SMA disease severity.”

Decreases in serious adverse events, high-grade adverse events and treatment-related adverse events were observed in the second year versus the first year in both treatment arms. The most common adverse events observed in the Evrysdi arm and the placebo and Evrysdi arm from 12-24 months were upper respiratory tract infection (15.8% and 10%, respectively), nasopharyngitis (21.7% and 16.7%, respectively), pyrexia (13.3% and 10%, respectively), headache (10% and 16.7%, respectively), diarrhea (7.5% and 10%, respectively), vomiting (11.7% and 13.3%, respectively) and cough (10% and 8.3%, respectively). The most common serious adverse events were pneumonia (6.7% and 0%, respectively) and influenza (0.8% and 0%, respectively).

Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.