Pivotal trial of Becker MD treatment fails to meet main or secondary goals
Developer says results 'not aligned' with findings in earlier clinical studies
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A large clinical trial evaluating the treatment candidate sevasemten for people with Becker muscular dystrophy (BMD) has failed to meet its main goal, with “no statistically significant benefit” seen on a measure of physical function.
That’s according to top-line data, or the first study results, from GRAND CANYON (NCT05291091), which tested sevasemten against a placebo over 1.5 years in approximately 175 men and boys with BMD. The oral therapy also did not ease muscle damage among participants, a key secondary goal, developer Servier stated in a company press release. The company acquired rights to sevasemten earlier this year from former developer Edgewise Therapeutics.
Servier noted that this Phase 2 study was designed to be pivotal, meaning positive data might support applications seeking approval of sevasemten for people with BMD, a genetic disease characterized by progressive muscle weakness and wasting.
Still, the developer noted that the new results “are not aligned with certain findings observed in prior clinical studies” — including another Phase 2 trial dubbed CANYON. As such, the company said it’s now running “a comprehensive analysis of the GRAND CANYON study dataset, together with relevant prior clinical data, to better understand and further characterize the study findings.”
Per the company, “full results will be presented in the future,” though no timeframe was given.
The main goal of the now-completed GRAND CANYON trial was to see if the therapy would improve scores on the North Star Ambulatory Assessment, a standardized measure of physical function, over 18 months of treatment. Secondary goals included easing of muscle damage and changes in height and skeletal muscle.
The global trial enrolled boys and men — the disease primarily affects males — ages 12 to 50 at 51 study sites across a dozen countries.
However, according to the developer, the study “did not meet the primary endpoint,” and “secondary endpoints also were not met.” Still, Servier noted that sevasemten showed “a continued favorable safety and tolerability profile.”
MDA ‘disappointed,’ but says data adds to understanding of BMD
The announcement was met with disappointment from the Muscular Dystrophy Association (MDA), which advocates for new treatments for muscle disorders such as BMD. The nonprofit emphasized, however, that the GRAND CANYON study still provides important knowledge even if it missed its mark.
“While we are disappointed that this trial did not demonstrate efficacy in adults with BMD, clinical research advances our understanding of the disease and informs the path forward,” the MDA said in a statement. “The GRAND CANYON study has contributed to advancements in outcome measures and clinical trial design that can help inform future research.”
The MDA said knowledge gained in every trial “is an important part of advancing the science” and key to developing future treatments.
“For people and families living with BMD, every clinical trial represents hope and an important investment in the future,” the MDA statement said. “We extend our appreciation to the trial participants, families, caregivers, investigators, and research teams whose commitment and contributions make clinical research possible.”
A type of muscular dystrophy, BMD is marked by reduced levels of dystrophin, a protein that normally helps prevent muscle cells from getting damaged when they contract. Sevasemten is designed to block the activity of a muscle contraction protein called myosin. The therapym designed for daily use, essentially aims to make muscle contractions gentler so that muscles don’t accumulate as much damage.
Sevasemten also being developed for Duchenne MD
In addition to BMD, sevasemten is being developed for Duchenne muscular dystrophy, a related condition in which muscles have virtually no dystrophin.
As a potential therapy for BMD, sevasemten was previously tested in a Phase 1 trial called ARCH (NCT05160415) and the Phase 2 CANYON study, as well as an extension study called MESA (NCT06066580). Data from those earlier trials had suggested that sevasemten might stabilize motor function.
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