aTyr Pharma, Inc. announced the results of a post-hoc analysis of data from its Phase 1b/2a study of efzofitimod in patients with pulmonary sarcoidosis. The analysis was presented in a poster at the European Respiratory Society (ERS) International Congress 2023, which is taking place September 9 ? 13, 2023, in Milan, Italy.

The poster is available on the Company?s website. The poster presents findings from a pooled, post-hoc analysis of data from a Phase 1b/2a randomized, double-blind, placebo-controlled, multiple ascending dose (1.0, 3.0 and 5.0 mg/kg) 24-week study of efzofitimod in patients with pulmonary sarcoidosis receiving oral corticosteroid (OCS) dose = 10.0 mg/day. Patients were randomized 1:2 (placebo:efzofitimod) and underwent a forced steroid taper in the first 8 weeks of the study.

Dose dependent improvements in steroid burden, FVC and patient reported outcomes (PRO) were noted, though the study was not powered for efficacy. In this pooled analysis, the 3.0 mg/kg (N=8) and 5.0 mg/kg (N=9) efzofitimod arms were considered therapeutic, and pooled. The placebo (N=12) and 1.0 mg/kg (N=8) efzofitimod arm, which was considered subtherapeutic, were pooled.

Time to relapse for steroid use (defined as dose of OCS increased after OCS taper to 5.0 mg or less of prednisone or equivalent for at least five consecutive days), rate of change for FVC and proportion of patients with changes that are multiples of the minimally clinically important difference (MCID) in PRO (Kings Sarcoidosis Questionnaire-Lung, or KSQ-L) were compared. Additionally, a responder endpoint was proposed (defined as reduction in OCS from baseline without worsening in FVC or PRO) and an analysis was performed. Key findings include: 7.7% of patients in the therapeutic group relapsed for steroid use compared to 54.4% of patients in the placebo/subtherapeutic group (p=0.017); The rate of change for FVC was significantly improved for the therapeutic group compared to the placebo/subtherapeutic group (p=0.035); 52.9% of patients in the therapeutic group showed an increase =12 for KSQ-L (3 times MCID) compared with 15.0% in the placebo/subtherapeutic group (p=0.032); and 64.7% of patients in the therapeutic group achieved response compared to 20.0% in the placebo/subtherapeutic group (p=0.008).

aTyr is currently conducting EFZO-FIT?, a global Phase 3 randomized, double-blind, placebo-controlled 52-week study to evaluate the efficacy and safety of 3.0 mg/kg and 5.0 mg/kg of efzofitimod in 264 patients with pulmonary sarcoidosis. The trial design incorporates a forced steroid taper. The primary endpoint of the study is steroid reduction.

Secondary endpoints include measures of lung function and sarcoidosis symptoms. 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.