aTyr Pharma (ATYR) Jefferies Global Healthcare Conference summary

Key program updates

• Ticker changed to ATYR, reflecting ongoing innovation and a novel immunobiology platform focused on tRNA synthetases for fibrosis and inflammation.

• Lead asset, efzofitimod, targets interstitial lung disease (ILD) with a first-in-class immunomodulator, showing strong phase 2 data and nearing phase 3 enrollment completion.

• Platform includes 200+ novel protein fragments, with a growing pipeline addressing unmet needs in inflammation and fibrosis.

• Preclinical programs target additional fibrotic diseases, leveraging unique receptor binding and evolutionary biology insights.

• Strong cash position supports upcoming data readouts and regulatory milestones.

Clinical and scientific highlights

• Efzofitimod demonstrated robust steroid-sparing, lung function, and symptom improvements in sarcoidosis, with consistent dose response and biomarker validation.

• Phase 3 trial in pulmonary sarcoidosis is well-powered, using steroid reduction as a primary endpoint, with additional lung function and quality of life measures.

• Compassionate use program initiated due to strong patient demand and positive safety profile, with anecdotal reports of long-term steroid-free patients.

• Scleroderma-related ILD phase 2 trial underway, with interim data expected by year-end, focusing on lung function and skin improvement.

• Platform science validated in high-impact journals, with ongoing publication of clinical and translational findings.

Strategic outlook and pipeline

• Targeting innate immunity at the site of inflammation to avoid systemic toxicity seen with current therapies.

• Pipeline candidates address multiple organ systems, with development driven by receptor biology and preclinical efficacy.

• Research foundation from Scripps and leadership by experienced biotech entrepreneurs.

• Anticipated catalysts include phase 3 data readout, BLA filing, and expansion into additional ILD and fibrotic indications.

• Ongoing engagement with regulators and scientific community to support clinical advancement and access programs.