aTyr Pharma Secures FDA Type C Feedback, Plans June IND Filing for Efzofitimod Phase 3 Sarcoidosis Study

A Type C meeting with the FDA has given aTyr Pharma a defined regulatory pathway for efzofitimod in pulmonary sarcoidosis, with an IND submission targeted for June 2026, a sequence that will draw close attention from regulatory affairs leads navigating endpoint selection for rare interstitial lung disease programs.

The planned Phase 3 study is a global, randomized, double-blind, placebo-controlled design enrolling up to approximately 372 patients with chronic, symptomatic pulmonary sarcoidosis with restrictive lung disease. The primary endpoint is change from baseline in forced vital capacity (FVC) at week 48; the King's Sarcoidosis Questionnaire (KSQ)-Lung score serves as the key secondary endpoint. The 54-week protocol runs two parallel cohorts dosed intravenously at 5.0 mg/kg once every three weeks for 17 total doses, with all background corticosteroid and immunosuppressant therapy held stable throughout.

The endpoint architecture reflects direct FDA input from the Type C review of the prior Phase 3 EFZO-FIT study. For regulatory affairs teams, the FVC-as-primary selection in a restrictive lung disease population signals the agency's current evidentiary expectations for this indication, a data point relevant to any sponsor designing IND strategy in the ILD space under 21 CFR Part 312.

In parallel, aTyr reports it remains on track to complete enrollment in the Phase 2 EFZO-CONNECT study evaluating efzofitimod in systemic sclerosis-related ILD (SSc-ILD) in the first half of 2026. That proof-of-concept study is a 28-week, randomized, double-blind, placebo-controlled design across three cohorts dosed at 270 mg or 450 mg intravenously monthly, with a target of up to 25 patients at U.S. centers. Promising interim data were reported in Q2 2025.

aTyr closed Q1 2026 with $68.3 million in cash, cash equivalents, restricted cash, and investments, providing a near-term runway that supports both the IND filing and continued EFZO-CONNECT enrollment without immediate financing pressure.

The June IND submission will be the next measurable checkpoint for the efzofitimod program, with Phase 3 site activation timelines and patient enrollment rates in the restrictive lung disease cohort serving as the operational indicators to monitor through the remainder of 2026.

Source: aTyr Pharma, Inc. via GlobeNewswire, May 15, 2026.