In March of 2016, we announced results from our multi-national exploratory Phase 1b/2 clinical trial of ATYR1940 in adult patients with FSHD in the United States and European Union. This randomized, double-blind, placebo-controlled trial was designed to evaluate the safety, tolerability, pharmacokinetics and exploratory pharmacodynamics markers and clinical assessments of multiple intravenous doses of ATYR1940 in adults with FSHD.
ATYR1940 was studied in three dose escalation cohorts (0.3, 1.0 and 3.0 mg/kg) across four sites and 20 patients. In each cohort, patients were randomized at a ratio of 3:1 to receive ATYR1940 or placebo. Patients in the first two cohorts were dosed weekly over a period of one month, and patients in the third cohort were dosed weekly over a period of three months.
We believe the safety, tolerability, immunogenicity and activity profile of ATYR1940 as demonstrated in this study warrants advancing our program in adult FSHD patients and potentially other rare diseases.