aTyr Pharma Announces Topline Results from Phase 3 EFZO-FIT™ Study of Efzofitimod in Pulmonary Sarcoidosis

Core Insights - The Phase 3 EFZO-FIT™ study of efzofitimod in pulmonary sarcoidosis did not meet its primary endpoint regarding the reduction in mean daily oral corticosteroid (OCS) dose at week 48, although clinical benefits were observed across multiple parameters [1][5][7] - A significant proportion of patients treated with efzofitimod achieved complete steroid withdrawal and showed improvement in the King's Sarcoidosis Questionnaire (KSQ)-Lung score compared to placebo [2][6][16] Study Results - The study involved 268 patients and compared the efficacy and safety of efzofitimod at doses of 3.0 mg/kg and 5.0 mg/kg against placebo over 48 weeks [4][9] - The mean daily OCS dose at week 48 was reduced to 2.79 mg for the 5.0 mg/kg efzofitimod group compared to 3.52 mg for placebo, with a p-value of 0.3313 [5][16] - Complete steroid withdrawal was achieved by 52.6% of patients on 5.0 mg/kg efzofitimod versus 40.2% on placebo (p=0.0919) [1][16] - Improvement in KSQ-Lung score was statistically significant for the efzofitimod group (p=0.0479), with 29.5% of patients achieving both complete steroid withdrawal and improved KSQ-Lung score compared to 14.4% in the placebo group (p=0.0199) [2][6][16] Safety and Tolerability - Efzofitimod was well-tolerated, maintaining a consistent safety profile across both tested doses [2][16] - The study demonstrated that lung function, as measured by forced vital capacity (FVC), was maintained with a change from baseline of -1.81 for efzofitimod versus -2.11 for placebo (p=0.7875) [6][16] Future Plans - The company plans to engage with the U.S. Food and Drug Administration (FDA) to discuss the results and determine the future path for efzofitimod in treating pulmonary sarcoidosis [3][7] - A conference call and webcast to discuss the study results is scheduled, indicating the company's commitment to transparency and stakeholder engagement [3][13] Context of Pulmonary Sarcoidosis - Pulmonary sarcoidosis affects approximately 200,000 Americans and is characterized by granuloma formation, leading to varying degrees of lung function impairment [14] - Current treatment options are limited and often associated with significant side effects, highlighting the need for safer and more effective therapies like efzofitimod [14][17]