aTyr Pharma Reaches FDA Alignment on Phase 3 Efzofitimod Protocol in Pulmonary Sarcoidosis
核心洞察
aTyr Pharma (搜索) has reached alignment with the FDA on the protocol for a global Phase 3 study of efzofitimod in chronic, symptomatic pulmonary sarcoidosis (搜索) with restrictive lung disease.
The 54-week randomized, double-blind, placebo-controlled trial will enroll up to approximately 372 patients randomized equally to 5.0 mg/kg efzofitimod or placebo given intravenously every three weeks.
The primary endpoint is change from baseline in forced vital capacity at week 48, with the King's Sarcoidosis Questionnaire-Lung score as the key secondary endpoint.
aTyr Pharma (搜索) has reached alignment with the U.S. Food and Drug Administration on the protocol for a Phase 3 study of its lead candidate efzofitimod in patients with chronic, symptomatic pulmonary sarcoidosis (搜索) with restrictive lung disease. The San Diego-based clinical-stage biotechnology company said it received FDA feedback earlier in the week and expects to initiate study-related activities in the fourth quarter of 2026, while focusing on regulatory submissions in the United States and Europe.
"We received feedback earlier this week from the FDA and we are very pleased to have reached alignment on the protocol for our planned Phase 3 study of efzofitimod in pulmonary sarcoidosis (搜索), a major form of interstitial lung disease (搜索)," said Sanjay S. Shukla, President and Chief Executive Officer of aTyr Pharma (搜索). "With limited treatment options available, particularly for patients requiring chronic therapy, we believe efzofitimod has the potential to become an important new treatment option."
Trial Design and Endpoints
The planned study is a global, randomized, double-blind, placebo-controlled trial evaluating the efficacy and safety of efzofitimod in patients with moderate to severe pulmonary sarcoidosis (搜索). 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 three weeks for a total of 17 doses.
The trial is intended to enroll up to approximately 372 patients with symptomatic pulmonary sarcoidosis (搜索) with restrictive lung disease who are receiving a stable dose of no more than 5.0 mg per day of oral corticosteroid and/or a background immunosuppressant. All background treatment will remain stable throughout the study.
The primary endpoint is change from baseline in forced vital capacity (FVC) at week 48. The key secondary endpoint is change from baseline in the King's Sarcoidosis Questionnaire (KSQ)-Lung score at week 48.
Supporting Data from EFZO-FIT
The study design is supported by a subgroup analysis of the Phase 3 EFZO-FIT study. In that analysis, patients with pulmonary sarcoidosis (搜索) with restrictive lung disease, defined as FVC percent predicted of 80% or less with a normal FEV1/FVC ratio, who were treated with 5.0 mg/kg efzofitimod experienced a clinically meaningful benefit in FVC and improvements in multiple patient-reported outcomes, including the KSQ-Lung score, compared with placebo. The findings were presented at the World Association of Sarcoidosis and Other Granulomatous Disorders 2026 Congress.
Efzofitimod is derived from aTyr's proprietary tRNA synthetase (搜索) platform, which the company describes as the source of its first-in-class medicines.
Additional Program and Funding Considerations
Efzofitimod is separately being evaluated in the Phase 2 EFZO-CONNECT study in patients with systemic sclerosis-related interstitial lung disease (搜索).
aTyr has stated that future development of efzofitimod in the planned Phase 3 trial will require additional capital, which the company expects to raise through equity or debt offerings, grants, collaborations, strategic partnerships and/or licensing arrangements.