aTyr Pharma, Inc. (ATYR) announced regulatory feedback and clinical plans for its lead candidate, Efzofitimod, in pulmonary sarcoidosis following a Type C meeting with the U.S. FDA.
Regulatory Update:
The FDA reviewed results from the Phase 3 EFZO-FIT study and advised that future development should focus on endpoints directly tied to patient function and quality of life. In response, aTyr will prioritize forced vital capacity (FVC) as the primary endpoint and the King's Sarcoidosis Questionnaire (KSQ)-Lung score as the key secondary endpoint in its next Phase 3 trial.
Clinical Strategy:
The company plans to submit an investigational new drug (IND) application in June 2026 for a global, randomized, double-blind, placebo-controlled Phase 3 study enrolling approximately 372 patients with chronic, symptomatic pulmonary sarcoidosis and restrictive lung disease.
Patients will be randomized to receive either 5.0 mg/kg Efzofitimod or placebo intravenously once every three weeks for 54 weeks.
The trial design incorporates FDA recommendations to increase dosing frequency from every four weeks to every three weeks, aiming to enhance drug exposure while maintaining safety.
Supporting Evidence:
Data from EFZO-FIT showed that patients with restrictive lung disease (FVC = 80% predicted) experienced clinically meaningful improvements in FVC when treated with Efzofitimod compared to placebo. Positive trends were also observed across patient-reported outcomes, including KSQ-Lung.
CEO Commentary:
"We are pleased with the feedback and believe focusing on FVC and KSQ-Lung in a defined patient population where Efzofitimod has already demonstrated benefit is the right strategy," said Sanjay S. Shukla, M.D., M.S., President and CEO of aTyr Pharma.
Outlook:
The upcoming Phase 3 trial represents the next step in evaluating Efzofitimod as a potential new treatment for pulmonary sarcoidosis, a debilitating interstitial lung disease (ILD), with limited therapeutic options. aTyr expects to file the IND in June 2026 and begin enrolment later in the year.
ATYR has traded between $0.64 and $7.29 over the past year. The stock closed Monday's trading at $0.95, up 0.41%. During overnight trading, the stock traded at $0.80, down 15.81%.
For comments and feedback contact: editorial@rttnews.com
Business News
May 08, 2026 15:50 ET Manufacturing and services sector survey results and labor market data from main economies were the highlight on the economics news front this week. Factory orders and jobs report dominated the news flow in the U.S. Similarly, industrial production data from German garnered attention in Europe. In Asia, purchasing managers’ survey results from China and the central bank decision from Australia were in focus.