aTyr Pharma, Inc. provided additional details regarding its plans to initiate a Phase 2 study of its lead therapeutic candidate, efzofitimod, in patients with systemic sclerosis (SSc, also known as scleroderma)-associated interstitial lung disease (ILD) following the clearance of an Investigational New Drug (IND) application by the U.S. Food and Drug Administration (FDA) announced earlier this year. Efzofitimod is a potential first-in-class immunomodulator that downregulates innate and adaptive immune responses incontrolled inflammatory disease states via selective modulation of neuropilin-2 (NRP2). The company is currently investigating efzofitimOD in patients with pulmonary sarcoidosis, a major form of ILD, in a global Phase 3 study called EfzofitimOD. The pathology of SSc-ILD is driven by the same immune cells that are central to pulmonary sarcoidosis pathology, and NRP2 is upregulated on these cells. EfzofitIMod has also been shown to reduce key pro-inflammatory markers that are central to this pathology in a clinical study in patients with pulmonary sarcoIDosis. Efzofitamod has been granted U.S. FDA orphan drug designation for the treatment of SSc and Fast Track designation for the treatment of S Sc-ILD. Systemic sclerosis is a chronic, progressive, autoimmune disease characterized by inflammation and fibrosis of connective tissues throughout the body, including the skin and other internal organs. SSc that occurs in the lungs is called SSc-ILD. About Efzofitimod aTyr is developing efzofitimod as a potential therapeutic for patients with fibrotic lung disease. Efzofitimod, a
fusion protein comprised of the immunomodulatory domain of histidyl-tRNA synthetase fused to the FC region of a human
antibody, is a selective modulator of neuropilin-2 that downregulates innate and adaptive immune response in
inflammatory disease states. aTyr's lead indication for efzofitimod is pulmonary sarcoidosis, a major form of
interstitial lung disease. Clinical proof-of-concept for efzofitimod was recently established in a Phase 1b/2a multiple-
ascending dose, placebo-controlled study of efzofitimod in patients with pulmonary sarcoidosis, which demonstrated
safety and a consistent dose response and trends of benefit of efzofitimod compared to placebo on key efficacy
endpoints, including steroid reduction, lung function, clinical symptoms and inflammatory biomarkers. aTyr is currently
conducting EFZO-FIT(TM), a global pivotal Phase 3 study of efzofitimod in pulmonary sarcoidosis. About aTyr aTyr is a biotherapeutics company engaged in the discovery and development of first-in-class medicines from its
proprietary tRNA synthetase platform. aTyr's research and development efforts are concentrated on a newly discovered
area of biology, the extracellular functionality and signaling pathways of tRNA synthetases. aTyr has built a global
intellectual property estate directed to a potential pipeline of protein compositions derived from 20 tRNA synthetase
genes and their extracellular targets. aTyr's primary focus is efzofitimod, a clinical-stage product candidate which
binds to the neuropilin-2 receptor and is designed to downregulate immune engagement in fibrotic lung disease.