Efzofitimod: a novel anti-inflammatory agent for sarcoidosis

Efzofitimod: a novel anti-inflammatory agent for sarcoidosis

Authors

  • Robert P. Baughman Editor-in-chief, Sarcoidosis, Vasculitis and Diffuse Lung Diseases
  • 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

Keywords:

sarcoidosis, NRP-2, treatment, mechanism of action

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.

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Published

28-03-2023

Issue

Section

Review

How to Cite

1.
Baughman RP, Niranjan V, Walker G, Burkart C, Paz S, Chong Y, et al. Efzofitimod: a novel anti-inflammatory agent for sarcoidosis. Sarcoidosis Vasc Diffuse Lung Dis [Internet]. 2023 Mar. 28 [cited 2025 Apr. 22];40(1):e2023011. Available from: https://www.mattioli1885journals.com/index.php/sarcoidosis/article/view/14396