Efzofitimod: a novel anti-inflammatory agent for sarcoidosis

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Robert P. Baughman
Vis Niranjan
Gennyne Walker
Christoph Burkart
Suzanne Paz
Yeeting Chong
David Siefker
Eileen Sun
Leslie Nangle
Sarah Forster
Michael Muders
Carol Farver
Elyse Lower
Sanjay Shukla
Daniel A Culver


sarcoidosis, NRP-2, treatment, mechanism of action


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.

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