Therapy Areas: Central Nervous System
Genentech's Risdiplam Meets Primary Endpoint in Pivotal SUNFISH Trial in People with Type 2 or 3 Spinal Muscular Atrophy
13 November 2019 - - US-based biotechnology company Genentech has released 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, the company said.
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.
Genentech is a member of Switzerland's Roche Group (SIX: RO) (OTCQX: RHHBY).
Risdiplam is an investigational, survival motor neuron-2 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.
SUNFISH is a two part, double-blind, placebo controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA.
Part 1 determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using total score of Motor Function Measure 32 (MFM-32) at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA.
Risdiplam is an investigational, orally administered liquid survival motor neuron-2 (SMN-2) splicing modifier for SMA.
It is designed to durably increase and sustain SMN protein levels both throughout the central nervous system and peripheral tissues of the body.
It is being evaluated for its potential ability to help the SMN2 gene produce more functional SMN protein throughout the body.
Risdiplam is currently being evaluated in four multicenter trials in people with SMA.
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