aTyr Pharma (ATYR) H.C. Wainwright 26th Annual Global Investment Conference summary

Platform and scientific foundation

• Focused on tRNA synthetase biology, with exclusive IP covering 20 genes and over 200 fragments and splice variants.

• Platform discovered by Dr. Paul Schimmel, who has a track record of translating science into successful medicines.

• Initial focus is on interstitial lung diseases, especially pulmonary sarcoidosis.

Lead asset: efzofitimod

• Efzofitimod targets NRP2 receptor, showing anti-inflammatory and anti-fibrotic effects in preclinical and clinical studies.

• Phase III trial in pulmonary sarcoidosis is the largest and first of its kind, with 268 patients enrolled.

• Phase II trial demonstrated dose-dependent improvements in steroid reduction, lung function, and symptoms, with no safety signals.

• Expanded access program initiated for patients finishing the trial who wish to continue treatment.

• Top-line phase III data expected in Q3 next year.

Pipeline and additional indications

• Pipeline includes ATYR0101 (targeting kidney fibrosis) and ATYR0750, with identified receptors and ongoing background research.

• Phase II trial in scleroderma ILD underway, with interim data expected in Q2 2025.

• Both lead indications have orphan drug and Fast Track designations in the U.S.; efzofitimod is partnered in Japan with Kyorin Pharmaceuticals.