Systemic sclerosis (SSc, or scleroderma) 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 and is a major form of interstitial lung disease (ILD). It is estimated that approximately 100,000 people in the U.S. are affected by SSc and up to 80% may develop ILD. SSc-ILD is caused by chronic inflammation in the lungs and, if left untreated, can result in scarring, or fibrosis, that causes permanent loss of lung function. ILD is the primary cause of death in patients with SSc. Current treatment options are limited. They mainly focus on slowing lung function decline, do not improve patient symptoms and are associated with significant toxicity. New treatments are needed that can stabilize or improve lung function and improve patient quality of life.
Efzofitimod has a novel mechanism of action for treating lung inflammation and fibrosis. By targeting neuropilin-2 on myeloid cells during active inflammation, efzofitimod works upstream of currently available immunomodulators to restore immune homeostasis, thereby resolving chronic inflammation and preventing the progression of fibrosis. These effects have been demonstrated in multiple animal models of ILD, including SSc-ILD. Certain cytokines central to the immune pathology of SSc-ILD, including interleukin-6 (IL-6), were also downregulated in an adjacent ILD, pulmonary sarcoidosis, in a recent clinical trial. Efzofitimod does not exhibit generally immunosuppressive properties and has been generally safe and well tolerated in clinical trials to date, potentially presenting a safer treatment option for SSc-ILD patients.
Efzofitimod has received Orphan Drug designation for SSc and Fast Track designation for SSc-ILD in the U.S. and Orphan Drug designation for SSc in the E.U.
We are conducting a Phase 2 study of efzofitimod in patients with SSc-ILD. This study, which is known as EFZO-CONNECT™, is currently enrolling patients at multiple centers in the U.S. To learn more about this study, please visit https://atyrpharma.com/efzoconnect or clinicaltrials.gov (NCT05892614).