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aTyr Pharma, Inc. (NASDAQ:LIFE) (aTyr or the Company), a clinical stage biotechnology company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced that it has dosed the first patient in its Phase 2 EFZO-CONNECT™ study. The proof-of-concept study will evaluate the efficacy, safety and tolerability of the Company's lead therapeutic candidate, efzofitimod, compared to placebo in patients with systemic sclerosis (SSc, or scleroderma)-related interstitial lung disease (ILD).
Efzofitimod is a first-in-class biologic immunomodulator that selectively modulates activated myeloid cells through neuropilin-2 (NRP2) to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. Efzofitimod has been granted U.S. Food and Drug Administration (FDA) and European Union orphan drug and U.S. FDA Fast Track designations for SSc.
"We are very pleased to begin patient dosing in EFZO-CONNECT™, which is our second clinical study for efzofitimod in ILD," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "Efzofitimod has been shown preclinically to reduce lung and skin fibrosis in models of SSc, and NRP2, efzofitimod's binding partner, is expressed in the skin of SSc patients. We believe there is compelling rationale that efzofitimod has the potential to target the underlying disease pathology central to this form of ILD and positively impact lung function and improve outcomes in these patients."
"Patients with SSc-ILD have limited treatment options and poor prognosis, with ILD being their leading cause of death," said Kristin Highland, M.D., Director, Rheumatic Lung Disease Program at the Cleveland Clinic. "This study, which evaluates a therapy that targets the inflammatory and fibrotic characteristics of this disease, is an important step forward towards developing a treatment that can potentially improve the prognosis and quality of life for patients in need."
The Phase 2 study is a randomized, double-blind, placebo-controlled, proof-of-concept study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with SSc-ILD. This is a 28-week study with three parallel cohorts randomized 2:2:1 to either 270 mg or 450 mg of efzofitimod or placebo dosed intravenously monthly for a total of 6 doses. The study intends to enroll 25 patients at multiple centers in the United States. The primary objective of the study is to evaluate the efficacy of multiple doses of intravenous efzofitimod on pulmonary, cutaneous and systemic manifestations in patients with SSc-ILD. Secondary objectives include safety and tolerability.
More information on the EFZO-CONNECT™ study is available at www.clinicaltrials.gov (NCT05892614).
About SSc-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. 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 causes 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 for SSc-ILD are limited, mainly focus on slowing lung function decline and are associated with significant toxicity.
About Efzofitimod
Efzofitimod is a first-in-class biologic immunomodulator in clinical development for the treatment of interstitial lung disease (ILD), a group of immune-mediated disorders that can cause inflammation and fibrosis, or scarring, of the lungs. Efzofitimod is a tRNA synthetase derived therapy that selectively modulates activated myeloid cells through neuropilin-2 to resolve inflammation without immune suppression and potentially prevent the progression of fibrosis. aTyr is currently investigating efzofitimod in the global Phase 3 EFZO-FIT™ study in patients with pulmonary sarcoidosis, a major form of ILD, and in the Phase 2 EFZO-CONNECT™ study in patients with systemic sclerosis (SSc, or scleroderma)-related ILD. These forms of ILD have limited therapeutic options and there is a need for safer and more effective, disease-modifying treatments that improve outcomes.
Posted In: LIFE
