Genentech announced positive data from the pivotal part 2 of the SUNFISH trial that evaluated the safety and efficacy of risdiplam in adult and pediatric patients with Type 2 or Type 3 spinal muscular atrophy (SMA).

Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN2) splicing modifier designed to increase and sustain SMN protein levels throughout the central nervous system and peripheral tissues of the body.

SUNFISH is a 2 part, multicenter, randomized, double-blind, placebo-controlled phase 2/3 trial in patients aged 2-25 years with Types 2 or 3 SMA (n=231). The objective of Part 1 (n=51) was to determine the risdiplam dose to use for the confirmatory Part 2 of the study. Part 2 evaluated motor function in patients (n=180) for 24 months, with a primary end point of change from baseline using total score of Motor Function Measure 32 (MFM-32) at 12 months. 

Results showed the study met its primary end point of treatment with risdiplam after 12 months compared with placebo. The safety profile of risdiplam was consistent with that seen in previous studies, and no treatment-related adverse events were identified leading to study withdrawal. 

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Full study data will be presented at an upcoming medical congress.

“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, MD, PhD, 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.”

The company is currently evaluating risdiplam in 3 additional clinical trials (FIREFISH, JEWELFISH, RAINBOWFISH) in patients with SMA ranging from birth to 60 years of age.

For more information visit gene.com.

This article originally appeared on MPR