aTyr recently released a press statement reporting their initial results from their FSHD study. The treatment was developed for use in myopathies with an immune component. This is a relatively new avenue of study in FSHD and this clinical trial will help add to understanding of FSHD.
They were evaluating multiple criteria including drug safety, immunogenicity (does the drug create an immune or allergic reaction), and pharmakokinetics (how the drug is broken down via metabolism). There were 20 patients at four different study sites. Overall, the drug seems to be tolerated well and shows great promise as they move to the next round of trials.
While there weren't enough patients in this initial trial to assess the ability of the drug to cure or prevent further progression, they did get some encouraging results showing they may be able to measure a decrease in progression and improvement of quality of life based on patient reported outcomes (answers directly from patients, not a physician) using the individualized neuromuscular quality of life (INQoL) questionnaire.
"Given the short duration of Resolaris treatment in our first study in FSHD patients, we are encouraged by the observed improvement in patient reported outcomes, in particular in activity and independence domains. While we need to do additional work to extend and confirm these results which are based on a small number of patients, the data represent our first step forward in our efforts to understand the clinical utility of Resolaris in patients with rare myopathies with an immune component (RMICs)," said Sanjay Shukla, MD, MS, Chief Medical Officer of aTyr.
See the full report here:http://atyrpharma.investorroom.com/2016-03-30-aTyr-Pharma-Announces-Encouraging-Phase-1b-2-Results-for-Resolaris-in-its-First-Rare-Myopathy-Trial