Announcement • May 12
aTyr Pharma, Inc. Provides Regulatory And Clinical Update For Efzofitimod In Pulmonary Sarcoidosis aTyr Pharma, Inc. had announced the path forward for its lead therapeutic candidate, efzofitimod, in pulmonary sarcoidosis, a major form of interstitial lung disease (ILD), following the receipt of the official meeting minutes from a Type C meeting with the U.S. Food and Drug Administration (FDA). The purpose of the meeting was to review the results of the Phase 3 EFZO-FIT study and determine the next steps for the program in pulmonary sarcoidosis. Based on feedback from the FDA, the Company plans to continue the development of efzofitimod in pulmonary sarcoidosis in a new Phase 3 study in patients with chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease utilizing forced vital capacity (FVC) as the primary endpoint of the study and the King’s Sarcoidosis Questionnaire (KSQ)-Lung score as the key secondary endpoint. The Company chose these endpoints based on the FDA’s indication that FVC and KSQ-Lung are direct measures of how patients suffering from pulmonary sarcoidosis function and feel, and the Company concluded FVC to be a more appropriate primary endpoint at this time pending further content validation work for the KSQ-Lung as recommended by the FDA. The Company plans to submit an investigational new drug (IND) application for this study in June 2026. Evidence from EFZO-FIT shows that patients with restrictive lung disease (defined as FVC percent predicted = 80%) experienced a clinically meaningful benefit for FVC when treated with efzofitimod compared to the observed decline in placebo. Furthermore, these more severe patients from EFZO-FIT also experienced positive trends of improvement across multiple patient-reported outcomes, including the KSQ-Lung score. As part of the discussion with the FDA regarding the benefit risk profile for efzofitimod, the Company plans to increase the frequency of dosing of 5.0 mg/kg efzofitimod or placebo from once every four weeks in past trials to once every three weeks in this next trial. Considering the consistent safety profile seen for efzofitimod in trials to date, the Company believes this strategy to increase drug exposure, coupled with additional risk mitigation strategies and safety surveillance, may enhance the effects of efzofitimod without incurring additional safety concerns. The Phase 3 trial is expected to be a global, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of efzofitimod in patients with moderate to severe pulmonary sarcoidosis. The 54-week study will consist of two parallel cohorts randomized equally to either 5.0 mg/kg efzofitimod or placebo dosed intravenously once every 3 weeks for a total of 17 doses. The study is intended to enroll up to approximately 372 patients with symptomatic pulmonary sarcoidosis with restrictive lung disease who are receiving a stable dose of = 5.0 mg daily oral corticosteroid (OCS) and/or a background immunosuppressant. All background treatment will remain stable throughout the duration of the study. The primary endpoint of the study will be change from baseline in FVC at week 48 and the key secondary endpoint will be change from baseline in the KSQ-Lung score at week 48. 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 intends to submit an investigational new drug (IND) application in June 2026 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. Announcement • Mar 14
aTyr Pharma, Inc., Annual General Meeting, May 11, 2026 aTyr Pharma, Inc., Annual General Meeting, May 11, 2026. Location: 10240 sorrento valley road, ca 92121, san diego, United States 
