Resolaris (ATYR1940) improved the muscle strength of nearly two-thirds of adolescents and young adults with early-onset facioscapulohumeral muscular dystrophy (FSHD), according to final results of a Phase 1/2 clinical trial.
The therapy’s maker, aTyr Pharma, presented the findings at the 22nd International Annual Congress of the World Muscle Society (WMS) in Saint Malo, France, according to a press release. The conference ran from Oct. 3-7. FSHD is a genetic that causes muscle weakness and atrophy.
Trial participants, who ranged from 16 to 20 years of age, also tolerated Resolaris well, the exploratory open-label study (NCT02603562) indicated.
Sixty-three percent of patients improved their muscle strength scores between the start and end of treatment. The mean change in scores was more than 3.8 percent, researchers said.
In addition, 67 percent of patients improved their Individualized Neuromuscular Quality of Life (INQoL) scores. The patient self-reporting tool is used to assess a disease’s burden.
Researchers said most participants’ disease did not worsen, and they saw no signs of immunosuppression in the patients. The results support advancing Resolaris to additional clinical trials, aTyr said.
Gennyne Walker of aTyr made the presentation, which was titled “Results of a Phase 1b/2 Study of ATYR1940 in Adolescents and Young Adults with Early-onset Facioscapulohumeral Muscular Dystrophy (FSHD) (ATYR1940-C-003).”
Resolaris is derived from a naturally occurring protein, the histidine aminoacyl tRNA synthetase (HARS). Scientists believe the protein can reset the immune system in diseased tissue to a more normal state without interfering with overall immunity.
FSHD is a genetic condition that is named after the muscles affected the most. They are the face (facio-), around the shoulder blades (scapula-), and the upper arms (humeral). Symptoms usually start during adolescence but milder cases can occur later.
The FSH Society says FSHD is the most prevalent of the nine primary types of muscular dystrophy affecting children and adults. A 2014 Dutch study reported that one in 8,333 people develops FSHD — or about 870,000 individuals worldwide. There is no cure for it.
ATyr is a biotherapeutics company developing immunology-based treatments for rare and severe immune-mediated diseases and cancers.