SAN DIEGO, Calif. — aTyr Pharma, Inc. (Nasdaq: ATYR) (“aTyr” or the “Company”), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced the publication of results from the Phase 3 EFZO-FIT™ study of its lead therapeutic candidate, efzofitimod, in patients with pulmonary sarcoidosis, a major form of interstitial lung disease, in The Lancet Respiratory Medicine. The publication, entitled, “Efzofitimod versus placebo in patients with pulmonary sarcoidosis (EFZO-FIT): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial,” is available on the journal’s website and at: https://www.thelancet.com/journals/lanres/article/PIIS2213-2600(26)00283-3/abstract.
EFZO-FIT™ was a global Phase 3 interventional study in 268 patients with moderate to severe pulmonary sarcoidosis that was designed to address patient and physician needs by investigating the impact of efzofitimod on steroid reduction and key patient-reported outcomes (PROs) through a protocol guided steroid taper.
The trial demonstrated that a large proportion of all patients in the study could be managed with substantially lower doses of steroids for the duration of the intervention. While treatment with efzofitimod did not result in a statistically significant steroid reduction compared to placebo, it did improve multiple PRO measures, which are priority outcomes for patients with sarcoidosis, and was generally well-tolerated.
“The findings from this largest interventional, randomized, controlled trial ever conducted in pulmonary sarcoidosis represent an important advancement in our understanding of how patients can be appropriately managed with steroids and is likely to lead to a paradigm shift in the way that we treat sarcoidosis patients and evaluate new therapies for this patient population in the future,” said Daniel Culver, D.O., Chair of the Division of Pulmonary Medicine at the Cleveland Clinic and lead author of the publication. “There is an urgent need for safe, effective and tolerable treatments for sarcoidosis that have a positive impact on patient quality of life. The study not only demonstrated that a large reduction of steroids was possible in all patients in the study, but remarkably, efzofitimod improved multiple PRO measures, including those specific to sarcoidosis, and warrants further investigation in this patient population.”
“Publication of the EFZO-FIT™ results strengthens the evidence supporting efzofitimod and brings greater visibility to its differentiated clinical profile,” said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr Pharma. “The improvements across multiple PROs, together with preserved lung function and a favorable safety profile, reinforce our confidence in efzofitimod’s potential and provide a strong foundation for its continued development in pulmonary sarcoidosis.”
About the EFZO-FIT™ Study
EFZO-FIT™ was a global Phase 3 interventional study in 268 patients with moderate to severe pulmonary sarcoidosis that compared the efficacy and safety of efzofitimod at 3.0 mg/kg and 5.0 mg/kg doses versus placebo after 48 weeks of treatment, which included a protocol guided steroid taper in the first 12 weeks of the study, followed by continued taper or rescue until week 48. While the study did not meet its primary endpoint of change from baseline in mean daily oral corticosteroid dose at week 48, clinical benefit for 5.0 mg/kg efzofitimod was observed across multiple pre-specified PROs at week 48 compared to placebo, including the King’s Sarcoidosis Questionnaire (KSQ)-Lung score, Fatigue Assessment Scale score, KSQ-General Health score, and complete steroid withdrawal with KSQ-Lung score improvement. Additionally, treatment with efzofitimod maintained lung function as measured by forced vital capacity and was well-tolerated with a safety profile consistent with prior trials conducted to date.
About Pulmonary Sarcoidosis
Pulmonary sarcoidosis is an inflammatory disease characterized by the formulation of granulomas, clumps of inflammatory cells, in one or more organs of the body. Approximately 160,000 Americans are diagnosed with pulmonary sarcoidosis and the prognosis ranges from benign and self-limiting to chronic, debilitating disease, permanent loss of lung function and death. Current treatment options include corticosteroids and other immunosuppressive therapies, which have limited efficacy and are associated with serious side-effects that many patients cannot tolerate long-term.
About Efzofitimod
Efzofitimod is a novel biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), a group of immune-mediated disorders that can cause inflammation and fibrosis, or scarring, of the lungs. Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. Efzofitimod is currently being investigated in the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis (SSc, or scleroderma)-related ILD, and aTyr recently initiated study-related activities for a global Phase 3 study of efzofitimod in patients with pulmonary sarcoidosis, a major form of ILD. These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.
About aTyr
aTyr is a clinical stage biotechnology company leveraging evolutionary intelligence to translate tRNA synthetase biology into new therapies for fibrosis and inflammation. tRNA synthetases are ancient, essential proteins that have evolved novel domains that regulate diverse pathways extracellularly in humans. aTyr’s discovery platform is focused on unlocking hidden therapeutic intervention points by uncovering signaling pathways driven by its proprietary library of domains derived from all 20 tRNA synthetases. aTyr’s lead therapeutic candidate is efzofitimod, a novel biologic immunomodulator in clinical development for the treatment of interstitial lung disease, a group of immune-mediated disorders that can cause inflammation and progressive fibrosis, or scarring, of the lungs. For more information, please visit www.atyrpharma.com.
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