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

Key platform and scientific insights

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

• Lead asset efzofitimod targets pulmonary sarcoidosis, an orphan disease with high unmet need.

• Mechanism involves selective binding to NRP2, down-regulating myeloid cells and reducing lung inflammation and fibrosis.

• Platform supports additional candidates (ATYR0101, ATYR0750) targeting fibrotic diseases like kidney fibrosis.

• Ongoing research into other tRNA synthetase-derived candidates to expand the pipeline.

Clinical development and trial updates

• Phase III trial for efzofitimod in pulmonary sarcoidosis is the largest ever, with 268 patients enrolled; top-line data expected Q3 next year.

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

• Phase II data in pulmonary sarcoidosis showed dose-dependent improvements in steroid reduction, lung function, and symptoms.

• No safety signals observed across multiple trials, including in COVID and Japanese studies.

• Expanded access program initiated for patients completing the phase III trial who wish to remain on treatment.

Market opportunity and strategic positioning

• Pulmonary sarcoidosis and scleroderma ILD represent a $2–3 billion market with little competition, especially in sarcoidosis.

• Efzofitimod holds orphan drug and Fast Track designations in the US and Europe.

• Partnership with Kyorin Pharmaceuticals in Japan valued at $175 million, with $20 million realized to date.

• No approved drugs for pulmonary sarcoidosis; current standard of care is steroids with high toxicity.

• Scleroderma ILD has more competition but lacks disease-modifying therapies, positioning efzofitimod as a potential first- or second-line option.