aTyr Pharma, Inc. (NASDAQ:LIFE), today announced that additional clinical data from aTyr’s Phase 1b/2 Trial (002) in adult patients with FSHD were presented at the 21st International Congress of the World Muscle Society in Granada, Spain. The poster presentation is titled “A Randomized, Double-blinded, Placebo-controlled, Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, Pharmacokinetics, Immunogenicity, and Biological Activity of ATYR1940 (Resolaris™) in Adult Patients with Facioscapulohumeral Muscular Dystrophy.”
“In today’s presentation we have provided additional data analyses which reaffirm encouraging trends in two meaningful clinical assessments from our adult FSHD (002) trial,” commented John Mendlein, PhD, CEO of aTyr Pharma. “In December, we look forward to sharing further top-line updates from our Phase 1b/2 clinical program with Resolaris in three different rare myopathies from our early onset FSHD (003) trial, our LGMD2B/FSHD (004) trial, and our first FSHD extension (005) trial.”
Today’s presentation includes additional detailed data from the adult FSHD (002) trial covering safety, tolerability, PK, and clinical assessments. Encouraging activity was seen in the patient-reported outcomes, known as the Individualized Neuromuscular Quality of Life Assessment (INQoL), and the physician-reported functional assessment Manual Muscle Testing (MMT).
Patients treated with Resolaris were generally improved compared with placebo as assessed by INQoL, with patients in cohort 3 (3.0 mg/kg for 12 weeks) showing the greatest improvement compared with cohort 1 and cohort 2 (0.3 mg/kg and 1.0 mg/kg for 4 weeks respectively). Patients in cohort 3 reported a ~9.9% improvement in INQoL compared with a ~15.6% worsening in the placebo group at week 14. Five out of the six patients on Resolaris in cohort 3 showed overall improvement in their INQoL score at week 14, versus zero out of two patients on placebo.
A trend for improvement in MMT results with Resolaris treatment was also observed compared with placebo, especially in the upper limbs. In general, there was an association between changes in INQoL and MMT, whereby all patients who experienced an improvement in muscle function also showed improvement in INQoL scores. Patients in cohort 3 reported ~0.7% improvement in MMT compared with ~1.4% worsening in the placebo group at week 14. Three out of the six patients on Resolaris in cohort 3 showed overall improvement in their MMT score at week 14, versus zero out of two patients on placebo.