Introduction: EFZO-FIT (ATYR1923-C-004) is a randomized (1:1:1 placebo or efzofitimod 3 or 5 mg/kg) , trial evaluating efficacy and safety of a 48-week (12 doses) treatment with efzofitimod in pulmonary sarcoidosis. A total of 264 patients were enrolled and treated (intent-to-treat, ITT). Here we present data for a subset of patients of pulmonary sarcoidosis with the restrictive phenotype (FVCpp < 80%, FEV1/FVC > 0.7).

Objectives: To evaluate the efficacy, safety of efzofitimod in the restrictive phenotype patients from the EFZO-FIT trial.

Materials and Methods: In this posthoc analysis, the Week 48 change from baseline in FVC and Patient Reported Outcomes was analyzed using a Random Coefficient Regression Model (RCRM) to supplement the Mixed Model for Repeated Measures (MMRM) analysis performed for the ITT data. 

Results: There were 16, 16 and 12 patients in the placebo, 3 mg/kg and 5 mg/kg arms respectively. These baseline characteristics were generally balanced with some minor imbalances between treatment arms, consistent with the small sample size. Weight was overall higher in the restrictive subset than the ITT. Mean FVC was expectedly lower in the restrictive subset than that for the ITT population.

Overall, the magnitude of steroid reduction in the 5 mg/kg was comparable to that in the placebo arm.

In the 5 mg/kg efzofitimod arm, the placebo adjusted week 48 change from baseline for FVC by RCRM was 123.8 ml. The placebo adjusted week 48 change from baseline by RCRM showed improvement for the 5 mg/kg arm versus placebo for King’s sarcoidosis questionnaire-Lung (KSQ-L), KSQ-general health (KSQ-GH), fatigue assessment scale (FAS), Leicester cough questionnaire (LCQ).

Efzofitimod was generally well tolerated, consistent with the ITT population.

Conclusions: The study results warrant further investigation in patients with pulmonary sarcoidosis and the restrictive phenotype.