aTyr Pharma Publishes Efzofitimod Efficacy in Pulmonary Sarcoidosis in European Respiratory Journal

aTyr Pharma, Inc., a clinical-stage biotechnology company, recently published a post hoc analysis of its Phase 1b/2a clinical trial for efzofitimod, a potential treatment for pulmonary sarcoidosis, in the European Respiratory Journal. The analysis reveals significant findings in the management of this interstitial lung disease (ILD).

Pulmonary sarcoidosis is characterized by the formation of granulomas in the lungs, which can lead to severe lung function impairment. Traditional treatments involve corticosteroids, which, although effective, have long-term side effects that many patients find intolerable.

The Phase 1b/2a clinical trial was a randomized, double-blind, placebo-controlled study involving 37 patients with pulmonary sarcoidosis. The trial aimed to assess the safety, tolerability, and potential efficacy of efzofitimod in comparison to a placebo. Patients were divided into three cohorts receiving 1.0 mg/kg, 3.0 mg/kg, and 5.0 mg/kg doses of efzofitimod or a placebo, administered monthly over six months.

A key finding from the trial was the statistically significant difference in time-to-first-relapse between therapeutic doses (3.0 and 5.0 mg/kg) and subtherapeutic doses (1.0 mg/kg) or placebo. The study showed that 54.4% of patients in the subtherapeutic group experienced a relapse following corticosteroid tapering, compared to only 7.7% in the therapeutic group, highlighting efzofitimod’s potential to maintain disease control while reducing or even discontinuing steroid use.

The post hoc analysis revealed the median time-to-first-relapse for the subtherapeutic group was 126 days. By contrast, only one out of 17 patients in the therapeutic group relapsed by the end of the 24-week study period, underscoring the drug’s potential efficacy.

Dr. Ogugua Ndili Obi, the study’s lead author, emphasized the importance of these findings, noting that maintaining disease control while reducing steroid use would be a significant clinical advancement. The results suggest efzofitimod could offer a viable alternative to corticosteroids, avoiding their long-term toxicities and improving patients' quality of life.

The study’s primary objectives focused on evaluating the safety and pharmacokinetic profile of efzofitimod. Its secondary objectives included examining efzofitimod’s potential steroid-sparing effects and other exploratory assessments like lung function improvement.

Efzofitimod is a first-in-class biologic immunomodulator derived from tRNA synthetase, targeting activated myeloid cells through neuropilin-2 to reduce inflammation without suppressing the immune system. This mechanism could also prevent fibrosis progression. aTyr is currently conducting the global Phase 3 EFZO-FIT™ study involving 268 pulmonary sarcoidosis patients to further investigate efzofitimod’s efficacy and safety.

The drug has received orphan drug designation in the U.S., E.U., and Japan, as well as Fast Track designation in the U.S. for treating pulmonary sarcoidosis. Additionally, aTyr is exploring efzofitimod’s potential in treating systemic sclerosis-related ILD through the Phase 2 EFZO-CONNECT™ study.

aTyr Pharma’s platform leverages tRNA synthetase biology to discover new therapeutic pathways for fibrosis and inflammation. Their lead candidate, efzofitimod, exemplifies this approach by potentially offering a new treatment paradigm for ILD patients, aiming to address the underlying disease mechanisms without the adverse effects associated with current therapies.

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