Trial of ATYR1923 in Pulmonary Sarcoidosis Patients Nears Finish
“We are pleased to complete the last patient visit in this important study, which represents a significant milestone for aTyr, our ATYR1923 clinical program and the sarcoidosis community,” Sanjay Shukla, MD, president and CEO of aTyr Pharma, which is developing the candidate therapy, said in a press release.
“We would like to thank all of the patients, investigators and the Foundation for Sarcoidosis Research for their support and efforts that helped make this possible,” Shukla added. “We look forward to sharing the results in September.”
ATYR1923 is an immunomodulatory molecule designed to bind the neuropilin-2 (NRP-2) protein, located at the surface of immune cells and responsible for inflammation and granuloma formation in lung sarcoidosis. Specifically, ATYR1923 aims to reduce the immune responses in inflammatory disease states, and to prevent the formation of scar (fibrotic) tissue. The therapy is currently being tested as a treatment for severe inflammatory lung diseases, including pulmonary sarcoidosis.
The primary goal of this Phase 1/2 study (NCT03824392) is to evaluate the safety and tolerability of multiple ascending ATYR1923 doses — 1 mg/kg, 3 mg/kg, and 5 mg/kg — compared with a placebo in 37 pulmonary sarcoidosis patients. Treatment is administered into-the-vein every four weeks, for a total of 24 weeks.
All trial participants are also required to gradually lower their intake of oral corticosteroids, as an additional trial objective is to evaluate the potential steroid-sparing effects of ATYR1923.
Other goals include assessing the therapy’s pharmacokinetics — its movement into, through, and out of the body — and its immunogenicity (the ability to provoke an immune response).
An interim analysis of the first 15 patients treated with at least one dose of ATYR1923 found the therapy generally safe and well-tolerated, with no drug-related serious side effects reported. These findings are in agreement with those of a Phase 1 study (ACTRN12617001446358) in healthy volunteers, in which the therapy was also well-tolerated, with no significant adverse effects.
The ongoing Phase 1/2 trial is being conducted in partnership with the Foundation for Sarcoidosis Research.