BOSTON — RyCarma Therapeutics Inc., a clinical-stage biotechnology company developing treatments for skeletal muscle diseases, announced that the first patient has been dosed in a Phase 2 clinical trial evaluating its investigational drug surlorian for RYR1-related myopathy (RYR1-RM), a rare genetic muscle disorder with no approved treatments.
The company expects to report topline results from the study in the second half of 2027.
RYR1-related myopathy is a congenital skeletal muscle disorder caused by mutations in the RYR1 gene, which plays a critical role in regulating calcium release within muscle cells.
The condition disrupts normal muscle contraction, leading to muscle weakness, fatigue and reduced mobility. According to RyCarma, approximately 80% of affected individuals experience difficulty walking, and many eventually require wheelchairs.
There are currently no approved medications specifically targeting RYR1-related myopathy. Treatment generally focuses on managing symptoms and maintaining mobility and independence through physical therapy, respiratory support and energy conservation.
Surlorian, also known as ARM210, is an investigational small-molecule therapy designed to improve skeletal muscle function by targeting abnormal calcium leakage through ryanodine receptors.
Mutations in the RYR1 gene can destabilize these calcium channels, allowing calcium to leak within muscle cells and interfering with normal muscle contraction. Surlorian is being developed to address this underlying disease mechanism.
Previous Phase 1 clinical studies involving healthy volunteers and patients with RYR1-related myopathy demonstrated favorable safety, tolerability and pharmacokinetic profiles, according to the company.
In an earlier Phase 1b open-label study, patients receiving a higher dose of surlorian for 28 days reported improvements in proximal muscle strength and reductions in fatigue.
The drug has received Orphan Drug and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration (FDA).
“There are currently no approved treatments for RYR1-RM, leaving individuals and families with limited therapeutic options for a condition that can profoundly affect daily life and independence. Encouraging advances in our understanding of RYR1-RM are paving the way for therapies that target the underlying disease mechanisms,” said Lindsay Goldberg, executive director of The RYR-1 Foundation.
“If proven safe and effective, a treatment like surlorian, which targets calcium leak through the ryanodine receptor, could represent an important step forward for the RYR1-RM community,” Goldberg added.
The Phase 2 trial is a randomized, double-blind, placebo-controlled, multicenter study expected to enroll approximately 28 patients with autosomal dominant RYR1-related myopathy and proximal muscle weakness.
Participants will receive 28 days of surlorian and 28 days of placebo in a randomized sequence, with a 21-day washout period between treatments.
Following the double-blind portion of the study, eligible participants will have the option to enter an open-label extension and continue receiving surlorian for up to 12 months.
The study’s primary endpoint will measure changes in performance on the one-minute sit-to-stand test, which evaluates lower-body muscle function. Secondary endpoints include additional physical performance assessments, muscle strength measurements and patient-reported outcomes.
“With surlorian, RyCarma has the opportunity to bring a first-in-class therapy to patients with skeletal muscle disorders who lack suitable treatment options,” said Adam Rosenberg, CEO of RyCarma Therapeutics.
“In addition to our Phase 2 study in RYR1-RM, which is now enrolling patients at leading RYR1-RM investigator sites, RyCarma also has an open IND to evaluate surlorian in Duchenne Muscular Dystrophy patients. With a strong group of investors and experienced drug development team, we are well positioned to advance surlorian through clinical studies and closer to patients,” Rosenberg added.
Beyond RYR1-related myopathy, RyCarma is exploring the potential of surlorian for Duchenne muscular dystrophy, another serious genetic muscle disease characterized by progressive muscle weakness and degeneration.
The company has an active Investigational New Drug application with the FDA to evaluate the therapy in patients with Duchenne muscular dystrophy.
RyCarma said the Phase 2 study represents an important step in determining whether surlorian can provide meaningful improvements in muscle function for patients with RYR1-related myopathy.
The drug remains investigational, and its safety and effectiveness have not yet been established in larger clinical trials.
