ATYR1923 Is Safe, Well-tolerated by Pulmonary Sarcoidosis Patients, Phase 1/2 Trial Shows

ATYR1923, aTyr Pharma’s lead investigational therapy for pulmonary sarcoidosis, is safe and well-tolerated, according to interim data from a Phase 1b/2a clinical trial.

ATYR1923 is a first-in-class disease-modifying therapy  in development for the treatment of different types of interstitial lung diseases (ILDs), collectively characterized by progressive scarring (fibrosis) and impaired function of the lungs. Pulmonary sarcoidosis is an ILD.

The investigational ATYR1923 therapy modulates the activity of the neuropilin-2 (NRP-2) receptor protein to suppress both innate and adaptive immune responses associated with lung inflammation. Innate immunity refers to the general immune response against a trigger, while adaptive immunity refers to a more specialized immune response.

aTyr Pharma is currently investigating the safety, tolerability, preliminary efficacy, immunogenicity (the ability to trigger an immune response), and pharmacokinetics (how the therapy is absorbed, distributed, metabolized, and eliminated from the body) of multiple doses of ATYR1923 in a Phase 1b/2a trial (NCT03824392) in patients with pulmonary sarcoidosis.

The trial, being conducted in partnership with the Foundation for Sarcoidosis Research (FSR), is recruiting participants from multiple sites across the United States.

Once enrolled, patients will be randomly assigned to receive one of three intravenous doses of ATYR1923 (1.0, 3.0, or 5.0 mg/kg), or a placebo, every four weeks.

The study’s main goal is to assess the incidence of treatment-emergent adverse events and serious adverse events from the beginning of the study until week 24.

Data from a pre-planned interim analysis of the first 15 patients enrolled and treated with at least one dose showed that ATYR1923 was generally safe and well-tolerated, with no drug-related serious adverse events reported. These findings are in agreement with previous data from a Phase 1 study (ACTRN12617001446358) in healthy volunteers.

Adverse events observed in the study were only mild or moderate in severity, and deemed unrelated to ATYR1923 by trial investigators. Moreover, up to now, no anti-drug antibodies were identified.

“We are very encouraged by these initial safety findings, which provide an early indication for ATYR1923 meeting our expectations for the primary endpoint of safety and tolerability,” Sanjay Shukla, MD, MS, president and chief executive officer of aTyr Pharma, said in a press release.

“Having accomplished this first important interim step in our study, we can now also begin to focus on demonstrating activity of ATYR1923. Pulmonary sarcoidosis and other interstitial lung diseases present a significant opportunity for novel therapeutic approaches that can ideally slow or halt disease progression and the resulting decline in lung function. These favorable interim safety results allow us to advance our trial to provide evidence of the potential of ATYR1923 as a treatment option to improve the lives of patients with pulmonary sarcoidosis,” Shukla added.