Dosing of First Patient In Long-Term Safety Extension Study Of Resolaris for FSHD Initiated
aTyr Pharma, Inc. a company engaged in the discovery and development of Physiocrine-based therapeutics to address severe rare diseases, recently announced it began dosing the first patient in a new long term safety extension trial of Resolaris for adult patients suffering from facioscapulohumeral muscular dystrophy (FSHD), a rare and severe genetic myopathy for which there are currently no approved treatments.
Resolaris is a first-in-class intravenous protein therapeutic for the treatment of rare myopathies with an immune component. This novel therapy is derived from a naturally occurring protein released in vitro by human skeletal muscle cells. aTyr believes Resolaris has the potential to provide therapeutic benefit to patients with rare myopathies with an immune component characterized by excessive immune cell involvement.
The newly-initiated, open label study aims to determine Resolaris’ long-term safety, tolerability, and biological activity. The double-blind, placebo-controlled, multiple ascending dose Phase 1b/2 trial is ongoing and is still recruiting adult FSHD patients. aTyr Pharma expects to arrive at an initial batch of findings before the end of 2015, or within the first quarter of 2016. The extension study is projected to run through mid-2016.
“Our Resolaris extension study takes the next step in developing a new class of Physiocrine-based medicines for patients who currently have few options. The trial will also expand our growing safety and tolerability database over a longer treatment period,” said John Mendlein, Ph.D., CEO and executive chairman of aTyr Pharma. “When given in therapeutic doses, we believe Physiocrine-based therapies may productively modulate immune processes by rebalancing the immune systems in chronic rare muscle diseases to provide new options for these severely affected patients.”
aTyr Pharma is also strategizing a series of clinical trials to test Resolaris in other diseases, including early onset FSHD and limb-girdle muscular dystrophy (LGMD) 2B. Both studies are expected to launch later this year.
To learn more about the ongoing Resolaris study for FSHD, please visit www.clinicaltrials.gov.
The FSH Society, a nonprofit, patient-driven organization supporting research and education for facioscapulohumeral muscular dystrophy (FSHD), one of the most prevalent forms of muscular dystrophy, recently announced it will be the beneficiary of a number of fundraising events during this fall. The events which are aimed to raise awareness for the FSH Society and further FSHD research, will take place all over the country, from Boston to Los Angeles.