aTyr Pharma (ATYR) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Company overview and scientific platform

• Focuses on evolutionary intelligence, leveraging tRNA synthetase fragments to develop novel therapies targeting immune regulation.

• Lead asset, efzofitimod, is a fragment of histidyl-tRNA synthetase, highly enriched in lung tissue and acts via neuropilin-2 receptor.

• Pipeline includes over 200 protein fragments, with efzofitimod showing promising clinical results.

Lead program: Pulmonary sarcoidosis

• Pulmonary sarcoidosis affects about 200,000 patients in the US and Europe, with high unmet need due to reliance on steroids and lack of approved therapies.

• Phase II trial showed dose-dependent improvements in steroid reduction, lung function (FVC), and patient-reported outcomes.

• Current phase III trial is a one-year, 268-patient study, powered to detect a 30-35% steroid reduction, with primary endpoint of steroid reduction and secondary endpoints including lung function and quality of life.

• Aggressive steroid tapering protocol aims to bring patients to zero by week 12, with exclusion of highly fibrotic patients.

• Expanded access program implemented for trial participants performing well, with open-label drug provided until unblinding in Q3 next year.

Additional indications and pipeline development

• Efzofitimod is also being tested in systemic sclerosis-associated interstitial lung disease (SSc-ILD), with a six-month trial focusing on early ILD patients and FVC as the primary endpoint.

• Interim readouts for SSc-ILD expected in Q2 next year, with emphasis on skin biomarker changes and lung function.

• Plans exist to explore additional indications and develop a subcutaneous formulation post-approval.