aTyr Pharma's lead therapeutic candidate, efzofitimod, continues to show promise in treating pulmonary sarcoidosis. The company recently announced a positive outcome from the third pre-planned interim safety analysis conducted by an independent data and safety monitoring board (DSMB) for the ongoing Phase 3 EFZO-FIT™ study. The DSMB recommended the study continue without any modifications, reinforcing the drug's safety profile.
The EFZO-FIT™ study is a global, Phase 3, randomized, double-blind, placebo-controlled trial designed to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. The trial enrolled 268 subjects across multiple centers in the United States, Europe, Japan, and Brazil. Participants are randomized into three parallel cohorts receiving either 3.0 mg/kg or 5.0 mg/kg of efzofitimod, or a placebo, administered intravenously once a month for a total of 12 doses over 52 weeks. A key component of the trial design is a forced steroid taper, addressing the need to reduce the reliance on corticosteroids, a standard treatment often associated with significant side effects.
Efzofitimod's Mechanism of Action
Efzofitimod is a first-in-class biologic immunomodulator targeting interstitial lung disease (ILD). It functions by selectively modulating activated myeloid cells through neuropilin-2 (NRP2), aiming to resolve inflammation without broad immune suppression and potentially prevent fibrosis progression. This targeted approach is particularly relevant for pulmonary sarcoidosis, where granulomas form in the lungs, leading to inflammation and impaired lung function.
ATS Conference Presentations
aTyr Pharma is set to present three posters at the American Thoracic Society (ATS) 2025 International Conference, taking place May 16 – 21, 2025, in San Francisco, CA. The presentations will cover:
- EFZO-FIT, Largest Placebo-Controlled Trial in Pulmonary Sarcoidosis – Trial Design and Patient Characteristics: This poster will detail the design and baseline characteristics of patients enrolled in the EFZO-FIT™ study.
- Real-World Treatment Patterns Among Pulmonary Sarcoidosis Patients with Parenchymal Involvement in the US: This presentation will explore current treatment approaches for pulmonary sarcoidosis based on real-world evidence.
- Incidence, Prevalence, and Mortality of Pulmonary Sarcoidosis with Parenchymal Involvement in the US: This poster will provide insights into the epidemiological aspects of pulmonary sarcoidosis in the United States.
The Need for New Treatments
Pulmonary sarcoidosis, a major form of ILD, presents a significant unmet medical need. Current treatments, such as corticosteroids, can have substantial long-term side effects. The EFZO-FIT™ study's primary endpoint is steroid reduction, with secondary endpoints including measures of lung function and sarcoidosis symptoms. If successful, efzofitimod could offer a safer and more effective disease-modifying treatment, improving the quality of life for patients with pulmonary sarcoidosis.
Previous Clinical Data
Prior to the Phase 3 trial, a Phase 1b/2a trial (NCT03824392) demonstrated that efzofitimod could ease symptoms, improve lung function, and reduce the need for corticosteroids. A follow-up analysis suggested that higher doses of efzofitimod (3 and 5 mg/kg) significantly reduced relapse rates or the need to increase corticosteroid dosage.
Topline data from the Phase 3 EFZO-FIT™ study is expected in the third quarter of 2025.
