aTyr Pharma Publishes Phase 3 Efzofitimod Results in Lancet

San Diego, California — October 29, 2026

EFZO-FIT Evaluated Steroid Reduction and Patient-Centred Outcomes

EFZO-FIT was a multicentre, randomised, double-blind, placebo-controlled Phase 3 study designed to evaluate the efficacy and safety of efzofitimod at 3.0 mg/kg and 5.0 mg/kg compared with placebo. Participants received treatment over 48 weeks, with a protocol-guided corticosteroid taper during the first 12 weeks, followed by continued tapering or rescue treatment through Week 48. The primary endpoint assessed the change from baseline in mean daily oral corticosteroid dose at Week 48. The study did not demonstrate a statistically significant difference between efzofitimod and placebo for this endpoint. Nevertheless, the trial showed that a substantial proportion of participants across the study groups could be managed with considerably lower corticosteroid doses during the intervention period. This observation provides important information about steroid management in pulmonary sarcoidosis, although it does not establish a treatment-specific steroid-sparing benefit for efzofitimod. The study also included several patient-reported measures intended to capture symptoms, fatigue and overall health, reflecting the importance of quality of life in a chronic disease where treatment burden and long-term corticosteroid exposure can significantly affect patients.

Improvements Observed Across Multiple Patient-Reported Measures

At the 5.0 mg/kg dose, efzofitimod demonstrated clinical benefit across multiple pre-specified PRO measures at Week 48 compared with placebo. These included the King’s Sarcoidosis Questionnaire (KSQ)-Lung score, the Fatigue Assessment Scale, the KSQ-General Health score and complete corticosteroid withdrawal accompanied by improvement in the KSQ-Lung score. These findings are relevant because pulmonary sarcoidosis can affect respiratory function, energy levels and general wellbeing, making patient-reported improvements important measures of potential therapeutic value. Efzofitimod treatment also maintained lung function, as assessed by forced vital capacity (FVC), and was generally well tolerated. Its safety profile was consistent with findings from earlier clinical studies. Daniel Culver, D.O., Chair of the Division of Pulmonary Medicine at Cleveland Clinic and lead author of the publication, described the trial as an important contribution to understanding corticosteroid management in pulmonary sarcoidosis. He highlighted the improvements in multiple patient-reported outcomes and the need for safer, effective and tolerable treatments that can improve patients’ quality of life. Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr Pharma, said the publication strengthens the evidence supporting efzofitimod’s differentiated clinical profile, pointing to improvements across multiple PROs, preserved lung function and a favourable safety profile. The findings remain encouraging clinical observations, but they do not overturn the missed primary endpoint.

Efzofitimod Development Continues Across Interstitial Lung Diseases

Efzofitimod is an investigational biologic immunomodulator derived from the biology of transfer RNA synthetases and is being developed for interstitial lung diseases (ILDs), a group of immune-mediated conditions that can cause lung inflammation and progressive fibrosis. The candidate is designed to selectively modulate activated myeloid cells through neuropilin-2, with the aim of resolving inflammation without broadly suppressing the immune system and potentially limiting fibrotic progression. In addition to pulmonary sarcoidosis, efzofitimod is being evaluated in the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis-associated ILD. aTyr has also initiated study-related activities for a new global Phase 3 study in pulmonary sarcoidosis. Pulmonary sarcoidosis is characterised by the formation of granulomas, or clusters of inflammatory cells, in organs including the lungs. Its clinical course ranges from self-limiting disease to chronic illness, permanent loss of lung function and, in severe cases, death. Corticosteroids and other immunosuppressive therapies remain important treatment options, but long-term use can be limited by adverse effects and variable effectiveness. The EFZO-FIT publication adds to the clinical evidence base for efzofitimod, while further studies will be needed to establish its efficacy, safety and potential role in treatment. aTyr’s continued development programme will help determine whether the improvements in patient-reported outcomes observed in EFZO-FIT can translate into meaningful benefits for people living with pulmonary sarcoidosis and other ILDs.

Source:aTyr Pharma,Press release