The San Diego-based biotech company, aTyr Pharma, Inc., released findings on March 30, 2016, from a Phase 1b/2 clinical trial of its experimental therapy Resolaris in adult facioscapulohumeral muscular dystrophy (FSHD) patients. While the number of patients in the study is small, the company stated that the favorable safety, tolerability, immunogenicity and activity profile of Resolaris as demonstrated in this study warrants advancing its clinical programs in adult FSHD patients and potentially other rare diseases.
The randomized, double-blind, placebo-controlled trial studied Resolaris in three dose escalation cohorts (0.3, 1.0, and 3.0 mg/kg) across four sites and a total of 20 patients. The study was designed to evaluate the safety, tolerability, immunogenicity (immune reaction to Resolaris) and pharmacokinetic (PK) profile of Resolaris in adult FSHD patients. The study also evaluated the usefulness of tools that could potentially measure drug effectiveness, such as magnetic resonance imaging (MRI) and patient reported outcomes.
Patients were treated and followed for 1 month for the first two cohorts and 3 months for the third cohort. The study did not observe any difference in the MRI between controls and patients receiving the drug after three months of treatment, but it found that data from a patient reported outcome measure suggests potential improvement, primarily in the categories of activities, independence and emotions. The findings were felt to be encouraging enough to justify continuing the development of Resolaris in FSHD and other rare myopathies with an immune component and potentially expanding the trial to additional clinical trial sites.
The company is currently recruiting for a 12-week trial for patients between the ages of 16 and 25 who had onset of FSHD symptoms before age 10, and is conducting a 12-week trial for adults with FSHD or limb-girdle muscular dystrophy type 2B (LGMD2B). Trial details and sites are posted here.
Read aTyr’s press release here.