Date: 2017-04-24
Type of information: Presentation of results at a congress
phase: 1-2
Announcement: presentation of results at the American Academy of Neurology (AAN) 69th Annual Meeting
Company: Atyr Pharma (USA - CA)
Product: Resolaris® - ATYR1940
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: limb girdle muscular dystrophy (LGMD)
Therapeutic area: Rare diseases - Genetic diseases - Neuromuscular diseases
Country: Denmark, France, USA
Trial details: This open-label, intrapatient dose escalation study will evaluate the safety, tolerability, immunogenicity, and biological activity of ATYR1940 in Patients With Limb Girdle and Facioscapulohumeral Muscular Dystrophies. (NCT02579239)
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 its participation as part of the Emerging Science Platform Session at the upcoming American Academy of Neurology (AAN) 69th Annual Meeting in Boston. The company will present results of a phase 1b/2 study of ATYR1940 in adult patients with limb girdle muscular dystrophy type 2B (LGMD2B) and facioscapulohumeral muscular dystrophy (FSHD) (ATYR1940-C-004). The poster presentation provides further detail on the previously announced results from the completed Phase 1b/2 open-label, intra-patient dose escalation 004 trial testing doses of Resolaris® (ATYR1940) of up to 3.0 mg/kg biweekly in patients with LGMD2B and FSHD. Data from all clinical trials completed to date demonstrate that Resolaris has a favorable safety profile and was generally well-tolerated across all doses tested. There have been no observed signs of general immunosuppression and low-level anti-drug antibody signals did not result in clinical symptoms. 78% of the LGMD2B patients in the trial recorded increases in muscle function at 14 weeks as measured by manual muscle test (MMT) score, a validated assessment tool. 50% of the FSHD patients in the trial recorded increases in muscle function as measured by MMT score. aTyr believes these data are supportive of further advancement of Resolaris. • On October 13, 2015, aTyr Pharma, a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe rare diseases, announced expansion of its Resolaris™ clinical program in rare myopathies with an immune component (\"RMICs\") by initiating a Phase 1b/2 clinical trial in patients with limb girdle muscular dystrophy (LGMD) 2B. This international Phase 1b/2 clinical trial is an open-label, intra-patient dose escalation study designed to assess the safety, tolerability, immunogenicity and activity of Resolaris™ in adult patients with LGMD2B. This new trial includes adult patients with facioscapulohumeral muscular dystrophy (FSHD) to further augment the Company\'s ongoing, blinded Phase 1b/2 clinical trial of Resolaris™ in adult patients with FSHD and to inform subsequent later-stage trial considerations. Both LGMD2B and FSHD are progressive, debilitating muscle diseases characterized by an immune component in the effected skeletal muscles. Clinical trial sites for this study will screen patients for potential enrollment in the United States and Europe with plans to enroll an estimated 16 patients in total, including up to eight patients with LGMD2B and up to eight adult patients with FSHD. The Company expects to initiate a third Phase 1b/2 clinical trial this month to specifically study patients with early onset FSHD. All three trials, adult patients with FSHD, early onset FSHD and LGMD2B, will assess Resolaris\' impact on the immune component using blood borne markers and magnetic resonance imaging (MRI).
