aTyr Pharma Reports Second Positive DSMB Review for Efzofitimod in Phase 3 Pulmonary Sarcoidosis Study

aTyr Pharma, Inc., a clinical-stage biotechnology company, recently announced a significant milestone for its lead therapeutic candidate, efzofitimod. An independent data and safety monitoring board (DSMB) has recommended that the ongoing Phase 3 EFZO-FIT™ study of efzofitimod in patients with pulmonary sarcoidosis should continue without any modifications. This decision follows a second pre-planned interim analysis, reinforcing the favorable safety profile of efzofitimod.

Dr. Sanjay S. Shukla, President and CEO of aTyr Pharma, expressed enthusiasm about the DSMB's recommendation. He emphasized that this positive review builds on the previously observed safety profile of efzofitimod, and additional DSMB reviews are expected as the study progresses. Dr. Shukla highlighted the limitations of current treatments for pulmonary sarcoidosis, which primarily involve oral corticosteroids known for their significant side effects and toxicity. He noted that efzofitimod could offer a much-needed, safe, non-steroidal treatment alternative for patients.

The EFZO-FIT™ study is a global Phase 3 clinical trial designed to evaluate the efficacy and safety of efzofitimod in treating pulmonary sarcoidosis. The study is randomized, double-blind, and placebo-controlled, involving three parallel cohorts. Participants are assigned to receive either 3.0 mg/kg or 5.0 mg/kg of efzofitimod, or a placebo, administered intravenously once a month for 12 doses over 52 weeks. Up to 264 subjects with pulmonary sarcoidosis are being enrolled across multiple centers in the United States, Europe, Japan, and Brazil. The trial includes a forced steroid taper, with the primary endpoint focused on steroid reduction and secondary endpoints evaluating lung function and sarcoidosis symptoms.

Efzofitimod represents a novel approach to treating interstitial lung disease (ILD), including pulmonary sarcoidosis. It is a first-in-class biologic immunomodulator derived from tRNA synthetase that selectively modulates activated myeloid cells through neuropilin-2. This mechanism aims to resolve inflammation without suppressing the immune system and potentially prevent the progression of fibrosis, or scarring, in the lungs. Besides the EFZO-FIT™ study, aTyr is also investigating efzofitimod in the Phase 2 EFZO-CONNECT™ study for patients with systemic sclerosis (SSc, or scleroderma)-related ILD.

aTyr Pharma is dedicated to developing innovative treatments for fibrosis and inflammation through its proprietary tRNA synthetase platform. tRNA synthetases are ancient proteins with newly discovered domains that regulate various extracellular pathways in humans. By exploring these domains, aTyr aims to identify hidden therapeutic intervention points and develop novel treatments. Efzofitimod is the company's lead therapeutic candidate, targeting immune-mediated disorders that cause lung inflammation and fibrosis.

This ongoing research and development effort is crucial, given the limited treatment options available for ILD, including pulmonary sarcoidosis and SSc-related ILD. There is a significant need for safer and more effective disease-modifying therapies that can improve patient outcomes. Through its innovative approach, aTyr Pharma is striving to address this unmet medical need and bring new hope to patients suffering from these debilitating conditions.