Evrysdi Trial Demonstrates Improvement of Motor Function in SMA Patients

PTC Therapeutics announced two-year data from Part 2 of the SUNFISH trial evaluating Evrysdi™ (risdiplam) in children and adults with Type 2 or Type 3 spinal muscular atrophy (SMA) at the 2021 Muscular Dystrophy Association (MDA) Virtual and Scientific Conference. These results demonstrated that Evrysdi patients sustained or improved in motor function after 24 months of treatment. Furthermore, the patients and caregivers reported improvements in their ability to function independently as well as their ability to complete daily tasks.

"We're encouraged by the long-term results from the SUNFISH trial, which reinforce the sustained clinical benefit that Evrysdi offers to a very broad population of patients with SMA, many of whom experience difficulty performing daily functions," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "These data further support the strong U.S. launch and the need for an at home oral treatment option for the SMA community."

Two-year data from SUNFISH Part 2 include efficacy and safety data in the overall population (2-25 years old):

• MFM32 (Motor Function Measure – 32), RULM (Revised Upper Limb Module) and HFMSE (Hammersmith Functional Motor Scale Expanded) scores showed improvement or maintenance of motor function during the second year of treatment with 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 (SMA Independence Scale) upper limb module, and the patient-reported SMAIS score stabilized between months 12 and 24.

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).

Evrysdi is designed to treat SMA by increasing and sustaining the production of the SMN protein, which is found throughout the body and is critical for maintaining healthy motor neurons and movement. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. Evrysdi is marketed in the United States by Genentech, a member of the Roche Group.

Evrysdi™ is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi is designed to distribute evenly to all parts of the body, including throughout the central nervous system (CNS). Evrysdi is administered daily at home in liquid form by mouth or feeding tube. In August 2020, the U.S. Food and Drug Administration approved Evrysdi for the treatment of spinal muscular atrophy for adults and children 2 months and older. Evrysdi is marketed in the United States by Genentech, a member of the Roche Group. Evrysdi is also approved in seven countries and has been filed in more than 50 countries worldwide.