Overview

• Efzofitimod missed EFZO-FIT’s primary endpoint in 268 pulmonary sarcoidosis patients, with mean daily steroid use reduced to 2.79 mg for 5.0 mg/kg versus 3.52 mg on placebo at week 48, a difference that was not statistically significant.
• Select secondary and patient-reported outcomes showed nominal gains, including KSQ-Lung improvement (10.36 vs 6.19; p=0.0479) and a higher rate of complete steroid withdrawal with KSQ-Lung improvement (29.5% vs 14.4%; p=0.0199), but the hierarchical design prevents confirmatory claims.
• ATyr’s stock fell more than 80% to roughly $1, leaving its market value near its estimated $113 million cash balance, and analysts at Leerink Partners and Cantor Fitzgerald downgraded the shares.
• The company reported a generally favorable safety profile for efzofitimod and cited a higher-than-anticipated placebo response as it prepares to discuss potential paths forward with the FDA.
• A shareholder rights firm, Hagens Berman, said it opened an investigation into aTyr’s disclosures about Phase 2 data and the Phase 3 trial design, with no findings announced.