aTyr Pharma (ATYR) Life Sciences Virtual Investor Forum summary

Platform and pipeline overview

• Focus on advancing platform biology centered on tRNA synthetase fragments, particularly efzofitimod for pulmonary sarcoidosis.

• Efzofitimod is a novel immunomodulator, now in phase III, with proof of concept established.

• Additional pipeline includes ATYR0101, a preclinical anti-fibrotic candidate, and a small trial in scleroderma ILD.

• Strong intellectual property position and approximately $70 million in cash.

Disease background and unmet need

• Pulmonary sarcoidosis is a serious orphan lung disease affecting nearly 200,000 in the U.S., with higher prevalence in African Americans.

• Current therapies are limited to steroids and toxic immunosuppressants, leading to poor quality of life and progression to fibrosis.

• Efzofitimod is the first therapy in 70 years to show improvement in quality of life measures such as cough, breathlessness, and fatigue.

Clinical trial results and learnings

• EFZO-FIT phase III trial was the first to aggressively taper steroids, aiming to replace them with efzofitimod.

• While the primary endpoint (steroid reduction) was not statistically significant versus placebo, both arms showed substantial steroid reduction, prompting updates to treatment guidelines.

• Significant improvements in quality of life (cough, breathlessness, fatigue) were observed from week 8 onward, with durable effects.

• Safety profile remains favorable, with mostly mild to moderate adverse events and no new safety signals.