PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced positive results from part 2 of SUNFISH demonstrating that the study met its primary endpoint of change from baseline after 1 year of treatment with risdiplam compared to placebo as measured by the Motor Function Measure 32, a scale monitoring severity and progression of fine and gross motor function in patients with a neuromuscular disease such as spinal muscular atrophy (SMA). Risdiplam has been well tolerated and no treatment-related safety findings leading to withdrawal have been seen in any risdiplam trial to date. Data from part 2 of the SUNFISH study will be presented at an upcoming medical congress.
"We are very pleased with the results of the SUNFISH study and are excited to move one step closer to bringing risdiplam globally to all patients living with SMA. The SUNFISH trial shows that risdiplam continues to demonstrate its disease-modifying properties and compelling safety profile," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics. "The results from the study will be shared first with regulators globally and then will be presented at a SMA conference early next year. We believe that risdiplam has the potential to enter the market with a best-in-class profile for patients with all SMA types. We are particularly grateful to the SMA community, the patients and investigators who participated in the trials."
Risdiplam (RG7916), is an investigational, oral, first-in-class, mRNA splicing modifier for the treatment of SMA. SUNFISH is a double‐blind, two‐part, placebo‐controlled trial. Part 1 enrolled patients with type 2 or 3 SMA to evaluate the safety, tolerability, and PK/PD of several risdiplam dose levels. The pivotal SUNFISH part 2, in non‐ambulant patients with Type 2 or 3 SMA, evaluated safety and efficacy of the risdiplam dose level selected from part 1 for 24 months, followed by an open label extension. Patients from 2-25 years of age were enrolled in the study, representing the broad real-world spectrum of patients living with SMA. The SMA program is a collaboration between PTC, the SMA Foundation, and Roche.