Date: 2015-09-10
Type of information: Treatment of the first patient
phase: 1-2
Announcement: treatment of the first patient
Company: Atyr Pharma (USA - CA)
Product: Resolaris®
Action mechanism: protein/enzyme. Resolaris® is a recombinant human histidyl tRNA synthetase developed for the treatment of rare myopathies with an immune component. It is derived from a naturally occurring protein released in vitro by human skeletal muscle cells.
Disease: facioscapulohumeral muscular dystrophy
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Country: Italy, Netherlands
Trial details: This study is an open-label extension study to evaluate the long-term safety, tolerability, biological activity, and systemic exposure of ATYR1940 in adult patients with facioscapulohumeral muscular dystrophy (FSHD). (NCT02531217)
Latest news: • On September 10, 2015, aTyr Pharma, a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe rare diseases, announced that the first patient has been dosed in a newly initiated long term-safety extension study of Resolaris™ for adult patients with facioscapulohumeral muscular dystrophy (FSHD), a rare and severe genetic myopathy for which there are currently no approved treatments. The open label study is designed to assess the long-term safety, tolerability and biological activity of Resolaris™ and will include adult patients who are currently enrolled in an ongoing, double-blind, placebo-controlled, multiple ascending dose Phase 1b/2 trial of Resolaris™ in adult patients with FSHD. Data from the Phase 1b/2 study are expected in the fourth quarter of 2015 or first quarter of 2016. The extension study is expected to continue through mid-2016. The company is also planning trials in additional indications, including early onset FSHD and limb-girdle muscular dystrophy (LGMD) 2B, and both are expected to initiate later this year.
