On November 9, 2023 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, reported third quarter 2023 results and provided a corporate update (Press release, aTyr Pharma, NOV 9, 2023, View Source [SID1234637440]).

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"During the third quarter we made meaningful progress with our clinical development program for our lead therapeutic candidate, efzofitimod, in interstitial lung disease (ILD)," said Sanjay S. Shukla, M.D., M.S., President and Chief Executive Officer of aTyr. "We had a positive data and safety monitoring board (DSMB) review for our global pivotal Phase 3 EFZO-FIT study in patients with pulmonary sarcoidosis, a major form of ILD with high unmet medical need. This study continues to enroll in the U.S., Europe and Japan and based on current projections, we expect to complete enrollment in this study early in the second quarter of 2024. Additionally, we dosed the first patient in our Phase 2 EFZO-CONNECT study in patients with systemic sclerosis (SSc, or scleroderma)-related ILD (SSc-ILD), which is currently enrolling in the U.S."

Third Quarter 2023 and Subsequent Period Highlights

Continued enrollment in the global pivotal Phase 3 EFZO-FIT study to evaluate the efficacy and safety of efzofitimod in patients with pulmonary sarcoidosis. This is a randomized, double-blind, placebo-controlled, 52-week study consisting of three parallel cohorts randomized equally to either 3.0 mg/kg or 5.0 mg/kg of efzofitimod or placebo dosed intravenously monthly for a total of 12 doses. The study intends to enroll up to 264 subjects with pulmonary sarcoidosis. The study is open for enrollment at nearly all of the centers intended in the U.S., Europe and Japan and is expected to expand to include centers in Brazil. Based on current enrollment projections, the Company expects to complete enrollment in the study early in the second quarter of 2024.
Completed a positive DSMB review for the Phase 3 EFZO-FIT study. Data from the pre-planned, interim analysis of safety and tolerability of efzofitimod in patients with pulmonary sarcoidosis, which included the evaluation of patients who completed treatment, was evaluated. There were no drug-related serious adverse events, consistent with prior studies. The DSMB assessed that the study could continue unmodified and that the drug does not pose any undue risk to the patient that warrants additional safety measures.
Dosed the first patient in the Phase 2 EFZO-CONNECT study to evaluate the efficacy, safety and tolerability of efzofitimod in patients with SSc-ILD. This proof-of-concept study is a randomized, double-blind, placebo-controlled, 28-week study consisting of 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 U.S. 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.
Peer-reviewed article for efzofitimod published in the journal Frontiers in Pharmacology. The publication, titled, "Exposure-response analyses of efzofitimod in patients with pulmonary sarcoidosis," highlights a positive exposure response demonstrated by efzofitimod across multiple clinically relevant endpoints in the Phase 1b/2a study in patients with pulmonary sarcoidosis.
Presented two posters for efzofitimod at the European Respiratory Society (ERS) International Congress 2023. The posters presented new data from a pooled, post hoc analysis from the Phase 1b/2a study of efzofitimod in patients with pulmonary sarcoidosis that further demonstrates efficacy and findings that identify the expression of neuropilin-2 (NRP2), efzofitimod’s binding partner, in the skin of patients with SSc-ILD.
Poster for efzofitimod accepted for presentation at the upcoming American College of Rheumatology (ACR) Convergence 2023. The conference is scheduled to take place November 10 – 15, 2023, in San Diego, CA. The poster presents new data demonstrating the effects of efzofitimod in preclinical models of rheumatoid arthritis (RA) and RA-associated lung fibrosis.
Poster 1322 – Efzofitimod, a First-in-Class NRP2-targeting Immunomodulator, Ameliorates Rheumatoid Arthritis and Associated Lung Fibrosis in Preclinical Models on Monday, November 13, 2023, from 9:00 a.m. to 11:00 a.m. PST.
Third Quarter 2023 Financial Highlights and Cash Position

Cash & Investment Position: Cash, restricted cash, cash equivalents and investments as of September 30, 2023, were $105.6 million. Based on the Company’s current operational plans and existing cash, the Company updates its prior guidance and believes its cash runway will be sufficient to fund the Company’s operations through the filing of a Biologics License Application (BLA) for efzofitimod in pulmonary sarcoidosis.
R&D Expenses: Research and development expenses were $10.3 million for the third quarter of 2023, which consisted primarily of clinical trial costs for the Phase 3 EFZO-FIT and Phase 2 EFZO-CONNECT studies, manufacturing costs for the efzofitimod program and research and development costs for the efzofitimod and discovery programs.
G&A Expenses: General and administrative expenses were $2.6 million for the third quarter of 2023.
Collaboration and License Revenue: Collaboration and license revenue related to the Kyorin Agreement was $0.4 million for the third quarter of 2023, which consisted of drug product material sold to Kyorin for the Japan portion of the EFZO-FIT study.
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.