San Diego-based biotech company aTyr Pharma announced today its first clinical trial in FSHD patients of its investigational new drug Resolaris™. The Phase 1b/2 study is a double-blind, placebo-controlled, multiple ascending dose trial in up to 44 FSHD patients at multiple sites in the European Union. The exploratory trial is designed to evaluate safety, tolerability, pharmacokinetics and the biological activity of Resolaris™ in adult patients with FSHD.
Resolaris™ is a first-in-class intravenous protein therapeutic derived from a naturally occurring protein released in vitro by human skeletal muscle cells. aTyr believes Resolaris™ will provide therapeutic benefit to patients with rare myopathies with an immune component characterized by excessive immune cell involvement.
Read more about the trial here. UPDATE: aTyr Pharma Receives EMA Orphan Drug Designation for Resolaris™ in FSHD.
The FSH Society collaborated with aTyr on recruiting volunteers for a blood biomarker study. You can learn more about that here.