aTyr Pharma (LIFE) Announces Additional Data on Resolaris Phase 1b/2; Reaffirms Encouraging Results
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aTyr Pharma, Inc. (Nasdaq: LIFE) 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.
Conclusions of the Adult FSHD (002) Trial:
- Over the dose and duration studied, Resolaris was found to be generally safe in adult patients with FSHD, and was generally well tolerated with the exception of one patient who experienced infusion related reactions as previously reported and as discussed in more detail in our poster presentation.
- PK properties were dose-proportional and generally consistent throughout the study, with no measurable impact from anti-drug antibodies (ADAs).
- Clinical activity was supported by signals of improvement in INQoL questionnaire responses and MMT measures.
- Other exploratory measures, including lower extremity targeted MRI, did not demonstrate activity. Variability in image acquisition may have diminished the opportunity to show activity in this measurement.
- Patients in cohorts 2 and 3 were allowed to enroll in the ongoing long-term extension study (005) investigating the safety and efficacy of Resolaris, for which aTyr expects to provide an update in December 2016.
"We are excited to have observed signals of activity across two clinical assessments with both INQoL and MMT, which is a first for FSHD patients," said Sanjay Shukla, MD, MS, Chief Medical Officer of aTyr Pharma. "Directional improvement in two separate assessments that should otherwise decline over time may be evidence of how clinically meaningful Resolaris could be for FSHD patients, and we look forward to building upon the clinical activity and safety data with our additional data later this year."
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