Date: 2017-04-24
Type of
information: Results
phase: 1b-2
Announcement: results
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: France, Italy, The Netherlands, USA
Trial
details: The 003 trial was an international, multi-center, open-label, intra-patient, placebo run-in, dose escalation Phase 1b/2 study designed to evaluate the safety, tolerability, immunogenicity and exploratory clinical assessments of Resolaris at weekly doses of 0.3, 1.0 and 3.0 mg/kg in patients with early onset FSHD for a total of 12 weeks. Eight patients, ages 16 to 20, participated in the study.
Latest
news:
- On April 24, 2017, aTyr Pharma, a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address rare diseases, announced clinical results from its Phase 1b/2 003 trial assessing the safety and potential activity of Resolaris™ in patients with early onset facioscapulohumeral muscular dystrophy (FSHD).
aTyr’s exploratory trials in rare muscular dystrophies were designed to assess:
- Potential signals of clinical activity informative for developing clinical endpoints to be assessed in later-stage, placebo-controlled efficacy trials;
- Safety and tolerability of a new biologic protein, Resolaris, in patients;
- Biomarkers in these patients; and
- Data that would support further evaluation of Resolaris in rare muscular dystrophies with an immune component.Potential Signals of Clinical Activity: 63% of patients (5 of 8) had increases from baseline in their Manual Muscle Test (MMT), a validated assessment tool that measures muscle strength, with a mean change from baseline of +3.8%.
67% of patients measured (4 of 6) had improvement in their Individualized Neuromuscular Quality of Life (INQoL) score, a validated patient reported outcome measuring a patient’s level of disease burden. On average, patients did not have a worsening of their disease burden as measured by INQoL.
Safety and Tolerability: Resolaris® was generally well-tolerated at doses up to 3.0 mg/kg once weekly in early onset FSHD. There have been no observed signs of general immunosuppression and low-level anti-drug antibody signals did not result in clinical symptoms. Adverse events were mild or moderate in intensity. There were no clinically significant changes in other safety assessments. aTyr believes the observed safety results of Resolaris® to date are supportive of further advancement of Resolaris®.
- • On January 26, 2015, aTyr Pharma announced its first FSHD patient clinical trial of Resolaris®. The study focuses on adult patients with facioscapulohumeral muscular dystrophy (FSHD).
Is
general: Yes
