aTyr Pharma (ATYR) 17th Annual LD Micro Main Event Conference summary

Market and disease overview

• Interstitial lung diseases (ILDs) are chronic, progressive, and often fatal, with limited safe and effective treatment options.

• Current therapies for IPF generate $5 billion in sales but are not disease-modifying and have significant toxicity.

• Sarcoidosis is the most inflammatory ILD, with 200,000 U.S. patients and high steroid dependence.

• Steroid use is widespread but leads to significant side effects and does not prevent progression to fibrosis.

• There is a major unmet need for therapies that address inflammation early and reduce steroid reliance.

Efzofitimod: Mechanism and clinical data

• Efzofitimod is a novel, multinodal immunomodulator targeting Neuropilin-2 on macrophages, aiming to restore immune balance.

• Demonstrated anti-inflammatory and anti-fibrotic effects in preclinical and phase II studies.

• Phase II data showed improvements in symptoms, lung function, and steroid reduction, with no known safety issues.

• Published in CHEST and highlighted in major conferences, validating the approach.

• Phase III trial in sarcoidosis is fully enrolled (268 patients, 9 countries), with top-line data expected Q3 2025.

Market opportunity and positioning

• Efzofitimod could address a $2–3 billion market opportunity in ILD, especially as a steroid-sparing agent.

• Positioned as a first-in-class, disease-modifying therapy for sarcoidosis, with pricing estimates near $100,000–$120,000/year.

• Potential to expand into other fibrotic and inflammatory diseases, including scleroderma-related ILD.

• Pipeline includes additional protein fragments targeting fibrosis in liver, kidney, and via FGFR4.

• Strong financials with $80 million cash and significant institutional ownership.