Genentech, a member of the Roche Group (OTC: RHHBY) today announced positive data from the pivotal Part 2 of the SUNFISH study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study met its primary endpoint of change from baseline in the Motor Function Measure 32 (MFM-32) scale after one year of treatment with risdiplam, compared to placebo. No treatment-related safety findings leading to study withdrawal have been seen in any risdiplam trial to date. Safety for risdiplam was consistent with its known safety profile and no new safety signals were identified.
"The positive outcome of this trial is an important milestone for people with Type 2 or 3 SMA, too many of whom remain untreated," said Levi Garraway, M.D., Ph.D., chief medical officer and Head of Global Product Development. "SUNFISH is the largest placebo-controlled study ever undertaken in Type 2 or 3 SMA patients. We thank the SMA community for their partnership and look forward to sharing these results with regulators and bringing risdiplam to people living with this condition."
Risdiplam is an investigational, survival motor neuron-2 (SMN2) splicing modifier, designed to durably increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body. Roche and Genentech lead the clinical development of risdiplam as part of a collaboration with the SMA Foundation and PTC Therapeutics. Data from the SUNFISH study will be presented at an upcoming medical congress.
Risdiplam is being studied in a broad clinical trial program in SMA, with patients ranging from birth to 60 years old, and includes patients previously treated with SMA-targeting therapies. The clinical trial population represents the broad real-world spectrum of people living with this disease with the aim of ensuring access for all appropriate patients.