Efzofitimod: a novel anti-inflammatory agent for sarcoidosis.
Robert P. Baughman, Vis Niranjan, Gennyne Walker, Christoph Burkart, Suzanne Paz, Yeeting E. Chong, David Siefker, Eileen Sun, Leslie A. Nangle, Sarah Förster, Michael H. Muders, Carol Farver, Elyse E. Lower, Sanjay Shukla, Daniel A. Culver
Abstract
Efzofitimod is a first-in-class biologic based on a naturally occurring splice variant of histidyl-tRNA synthetase (HARS) that downregulates immune responses via selective modulation of 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. A dose escalating trial 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.