Efzofitimod: a novel anti-inflammatory agent for sarcoidosis

Efzofitimod is a first-in-class biologic immunomodulator based on a naturally occurring splice variant of histidyl-tRNA synthetase (HARS) that binds to neuropilin-2 (NRP2). Preclinical data found high expression of NRP2 in sarcoidosis granulomas. Treatment with efzofitimod reduced the granulomatous inflammation induced by P. acnes in an animal model of sarcoidosis. An ascending dose study of efzofitimod in sarcoidosis with chronic symptomatic pulmonary disease found that treatment with efzofitimod was associated with improved quality of life with a trend towards reduced glucocorticoid use and stable to improved pulmonary function. These studies have led to a large Phase 3 trial of efzofitimod in symptomatic pulmonary sarcoidosis.