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aTyr Pharma Announces Encouraging Phase 1b/2 Results for Resolaris™ in its First Rare Myopathy Trial
First patient trial of a Physiocrine-based investigational new drug
Preliminary analysis shows potential activity signals in three months of treatment
Safety, tolerability, immunogenicity & PK profile supports Resolaris program advancement in FSHD and potentially other rare diseases
SAN DIEGO, March 30, 2016 /PRNewswire/ — aTyr Pharma, Inc. (LIFE) a biotherapeutics company engaged in the discovery and development of Physiocrine-based therapeutics to address severe rare diseases, today announced results from a Phase 1b/2 clinical trial evaluating Resolaris™ in adult FSHD (facioscapulohumeral muscular dystrophy) patients. The Company is developing Resolaris, a potential first-in-class protein therapeutic, for the treatment of rare myopathies with an immune component (RMICs). The Phase 1b/2 study was designed to evaluate the safety, tolerability, immunogenicity and pharmacokinetic (PK) profile of Resolaris in adult FSHD patients, the Company’s first treated RMIC population. In addition, the study also evaluated the utility of exploratory pharmacodynamic (PD) markers (including MRI measurements to quantitate areas of potential muscle inflammation) and clinical assessments (including patient reported outcomes).
“As the first clinical trial of a Physiocrine-based investigational new drug in patients, our data set provides important insights about the safety, tolerability, immunogenicity and PK profile of Resolaris in a very challenging disease setting, and explores potential PD markers and clinical assessments in FSHD,” said John Mendlein, PhD, Chief Executive Officer of aTyr. “It is our vision to provide patients with FSHD and other muscular dystrophies, such as limb-girdle muscular dystrophy (LGMD) 2B, an innovative treatment that potentially modulates immune components and other important aspects of disease progression.”