Genentech’s Risdiplam Showed Significant Improvement in Motor Function in People Aged 2-25 With Type 2 or 3 Spinal Muscular Atrophy
SOUTH SAN FRANCISCO, CA, USA I February 06, 2020 I Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today presented 1-year data from the pivotal Part 2 of SUNFISH, a global placebo-controlled study evaluating risdiplam in people aged 2-25 years with Type 2 or 3 spinal muscular atrophy (SMA). The study showed that change from baseline in the primary endpoint of the Motor Function Measure 32 scale (MFM-32)* was significantly greater in people treated with risdiplam, compared to placebo (1.55 point mean difference; p=0.0156). The Revised Upper Limb Module (RULM),** a key secondary endpoint, also showed an improvement (1.59 point difference; p=0.0028). Safety for risdiplam in the SUNFISH study was consistent with its known safety profile. Data were presented at the 2nd International Scientific and Clinical Congress on Spinal Muscular Atrophy from February 5-7 in Evry, France.
As anticipated, exploratory subgroup analyses showed that the strongest responses in MFM-32 versus placebo were observed in the youngest age group (2-5 years) (78.1% vs. 52.9% achieving ≥3 point increase). Importantly, disease stabilization was observed in the 18-25 years age group (57.1% vs. 37.5%, with stabilization defined as a ≥0 point increase), which is the goal of treatment for those with more established disease.
“Risdiplam is the first potential treatment to have pivotal placebo-controlled data in a broad population of patients, including children, teenagers and adults,” said SUNFISH principal investigator Eugenio Mercuri, M.D., Ph.D., Department of Pediatric Neurology, Catholic University, Rome, Italy. “The data suggest that risdiplam can preserve and potentially enable greater independence through improved motor function in people with Type 2 or non-ambulant Type 3 SMA.”
Safety for risdiplam in the SUNFISH study was consistent with its known safety profile and no new safety signals were identified. The adverse event profile was similar to placebo. The most common adverse events were upper respiratory tract infection (31.7%), nasopharyngitis (25.8%), pyrexia (20.8%), headache (20%), diarrhea (16.7%), vomiting (14.2%) and cough (14.2%). While the rate of lower respiratory tract infections overall was similar in both treatment arms (risdiplam 19%, placebo 20%), serious lower respiratory tract infections occurred in more patients in the risdiplam group (risdiplam 10%, placebo 2%) but were reported as unrelated and resolved without change to study treatment. To date, more than 400 patients have been treated with risdiplam across all studies, with no treatment-related safety findings leading to study withdrawal in any risdiplam trial.
“We are very encouraged by the positive results in this broad group of SMA patients, many of whom are under-served and under-represented in clinical trials,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “This study has helped us understand which measurement scales are the most relevant for patients, as well as the importance of stabilization in people with more established disease.”
Genentech leads the clinical development of risdiplam, an investigational, orally administered survival motor neuron-2 (SMN-2) splicing modifier for SMA, as part of a collaboration with the SMA Foundation and PTC Therapeutics. 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.
In November 2019, the U.S. Food and Drug Administration granted Priority Review for risdiplam with an expected decision on approval by May 24, 2020.
About Genentech in neuroscience
Neuroscience is a major focus of research and development at Genentech and Roche. The company’s goal is to develop treatment options based on the biology of the nervous system to help improve the lives of people with chronic and potentially devastating diseases. Genentech and Roche have more than a dozen investigational medicines in clinical development for diseases that include multiple sclerosis, spinal muscular atrophy, neuromyelitis optica spectrum disorder, Alzheimer’s disease, Huntington’s disease, Parkinson’s disease and autism.
Founded more than 40 years ago, Genentech is a leading biotechnology company that discovers, develops, manufactures and commercializes medicines to treat patients with serious and life-threatening medical conditions. The company, a member of the Roche Group, has headquarters in South San Francisco, California. For additional information about the company, please visit http://www.gene.com.
06 February 2020