Additional Trial Data Supports Resolaris for FSHD Treatment Potential

by Özge Özkaya, PhD | October 17, 2016

Resolaris, a potential intravenous protein therapy developed by aTyr Pharma for rare myopathies, was found generally safe and well tolerated in patients with facioscapulohumeral muscular dystrophy (FSHD). The results were presented at the 21^st International Congress of the World Muscle Society, Oct. 4-8, in Spain.

The company presented additional data from the Phase 1b/2 clinical trial, “A Randomized, Double-blinded, Placebo-controlled, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Biological Activity of ATYR1940 (Resolaris™) in Adult Patients with Facioscapulohumeral Muscular Dystrophy.”

Patient-reported outcomes were assessed through the Individualized Neuromuscular Quality of Life Assessment (INQoL), and the physician-reported functional assessment Manual Muscle Testing (MMT). Encouraging activity was recorded in both tests. Patients who experienced improvement in muscle function also showed improved INQoL scores.

“We are excited to have observed signals of activity across two clinical assessments with both INQoL and MMT, which is a first for FSHD patients,” Dr. Sanjay Shukla, the company’s chief medical officer, said in a press release. “Directional improvement in two separate assessments that should otherwise decline over time may be evidence of how clinically meaningful Resolaris could be for FSHD patients.”

The company is hoping to present further results from the Phase 1b/2 clinical program with Resolaris including the early onset FSHD trial, the limb girdle muscular dystrophy 2B/FSHD trial, and the FSHD extension trial later this year.

During the trial, adult FSHD patients were given a placebo or three different doses of Resolaris (0.3 mg/kg for four weeks, 1 mg/kg for four weeks, or 3 mg/kg for 12 weeks). Patients in the placebo group showed a 15.6 percent worsening in the INQoL, while those in the third group (highest dose) showed a 9.9 percent improvement.

In addition, patients in the placebo group showed a 1.4 percent worsening in MMT, while those in the high-dose group showed a 0.7 percent improvement. Patients in the second and third group were allowed to enroll in the FSHD extension trial. Results are expected in December 2016.

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About the Author

Özge Özkaya, PhD Özge has a MSc. in Molecular Genetics from the University of Leicester and a PhD in Developmental Biology from Queen Mary University of London. She worked as a Post-doctoral Research Associate at the University of Leicester for six years in the field of Behavioural Neurology before moving into science communication. She worked as the Research Communication Officer at a London based charity for almost two years.