aTyr Pharma, a biotherapeutics company committed to discovering and developing therapeutics for patients with severe rare diseases based on Physioncrine biology, recently announced the enrollment of the first patient with Facioscapulohumeral muscular dystrophy in their clinical trial of new drug called Resolaris™. Resolaris, is a first-in-class intravenous protein used for the treatment of immune rare myopathies. The drug is derived from a protein released by human skeletal muscle cells.
The Resolaris clinical trial is a phase 1b/2 double blind randomized place-controlled study that is going to examine the efficacy, safety, tolerability, pharmacokinetics and the activity of the new biological drug in the treatment of FSHD. The company aims to conduct the study in a total of 44 patients with FSHD from various clinical settings within the European Union. This is the first study that aTyr Pharm is conducting with an Physiocrine-based drug.
In the United States, Facioscapulohumeral muscular dystrophy (FSHD), a severe genetic myopathy, affects approximately 19,000 people. The symptoms in FSHD are progressive beginning with face and upper body muscles and then progressing to the lower body. Furthermore, patients with FSHD present high levels of chronic fatigue and pain. This condition is associated with premature death normally, with patients usually dying between the age of 20-30 years. At the moment there are no approved treatments for this rare condition.
“FSHD patients suffer from a debilitating skeletal muscle disease, and we would like to thank FSHD patients, caregivers and community for their contributions to this trial. We believe therapeutic levels of Resolaris™ have the potential to promote muscle health in FSHD patients that suffer from chronic triggers of skeletal muscle damage,” said John Mendlein, Ph.D., CEO and executive chairman of aTyr Pharma in a recent news release.
“Our Resolaris™ FSHD trial represents the first patient administration of a naturally occurring protein derived from a new class of physiological modulators, Physiocrines. We believe this trial will be an important step in our plan to develop new medicines that will have a meaningful impact for patients by activating physiological pathways important to skeletal muscle health,” Mendlein added.