aTyr Pharma to Present Final Data for Resolaris Phase 1b/2 Trial in Patients with Early-Onset Facioscapulohumeral Muscular Dystrophy at the 22nd International Annual Congress of the World Muscle Society
- Title: Results of a Phase 1b/2 Study of ATYR1940 in Adolescents and Young Adults With Early-onset Facioscapulohumeral Muscular Dystrophy (FSHD) (ATYR1940-C-003)
- Author and Presenter: Gennyne Walker, Ph.D.,
aTyr Pharma, Inc., San Diego, CA
- Supporting Authors: Russel J Butterfield,
Katherine Mathews, Laurent Servais, John Day, Teresa Gidaro, Sanjay Shukla, Lorenzo Maggi
In this exploratory, open-label study, Resolaris was generally well tolerated at doses up to 3.0 mg/kg once weekly in patients aged 16 to 20 years with early-onset FSHD. 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%. In addition, 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. No signs of general immunosuppression were observed, and low-level ADA signals did not result in clinical symptoms.
aTyr believes these data are supportive of further advancement of Resolaris.
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Sr. Director, Investor Relations
Source: aTyr Pharma Inc.