FDA Grants Fast Track Designation for Atyr's Resolaris to Treat Limb Girdle Muscular Dystrophy 2B and Removes Partial Clinical Hold for Resolaris
January 18, 2017 at 08:00 am
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Tyr Pharma, Inc. announced that its product candidate Resolaris was granted Fast Track designation by the U.S. Food and Drug Administration for the treatment of limb girdle muscular dystrophy 2B (LGMD2B), making it the first known therapeutic candidate for the treatment of LGMD2B to receive the designation. In addition, the FDA removed its partial clinical hold on a dosing ceiling for Resolaris in clinical trials. aTyr previously announced results from a completed Phase 1b/2 open-label, intra-patient dose escalation trial testing doses of Resolaris up to 3.0 mg/kg biweekly in patients with LGMD2B. Based on the clinical trials completed to date, Resolaris has demonstrated a favorable safety profile without signs of immuno-suppression of circulating immune cells. 78% of the LGMD2B patients in the trial (7 of 9) recorded increases in their muscle function at 14 weeks as measured by manual muscle test, a validated assessment tool. Overall, the LGMD2B patients had a mean increase of MMT scores from baseline of 6.2%.
aTyr Pharma, Inc. is a clinical-stage biotechnology company. The Company is leveraging intelligence to translate tRNA synthetase biology into new therapies for fibrosis and inflammation. Its lead therapeutic candidate is efzofitimod, a biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), a group of immune-mediated disorders that can cause inflammation and fibrosis, or scarring, of the lungs. Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells through neuropilin-2 (NRP2) to resolve aberrant inflammation without immune suppression and prevent the progression of fibrosis. ATYR0101 is a fusion protein derived from a domain of aspartyl-tRNA synthetase (DARS). ATYR0101 binds directly to latent-transforming growth factor beta-binding protein 1 (LTBP1), which regulates transforming growth factor beta (TGFb). Its ATYR0750 is a fusion protein derived from a domain of alanyl-tRNA synthetase (AARS).
ATYR PHARMA, INC. 
