ATYR

• efzofitimod's Phase 3 EFZO-FIT study completed patient enrollment, with top-line data expected in the third quarter. It's a large randomized, double-blind, placebo-controlled study with 268 patients at 85 centers in 9 countries. Four positive data and safety monitoring board reviews identified no safety concerns. - Individual Patient Expanded Access Program (EAP) implemented for patients completing the study, with patients receiving 5 milligrams per kilogram of efzofitimod while blinded to treatment assignments. - Held a Type C meeting with the FDA to discuss the statistical analysis plan for the study, changing the measurement of steroid reduction to absolute change from baseline to week 48. - Will present posters at the American Thoracic Society Conference in May regarding the sarcoidosis market in the U.S. - Appointed Eric Benevich to the Board of Directors, who has experience in launching high-value pharmaceuticals. - EFZO-CONNECT Phase 2 study evaluating efzofitimod in SSc-ILD, with interim data expected in the second quarter focusing on skin assessments. - Published an extensive manuscript in Science Translational Medicine outlining efzofitimod's mechanisms of action. Other tRNA synthetase-derived candidates ATYR0101 and ATYR0750 are being explored for fibrosis-related indications.

• Updated financial guidance indicates the cash runway is expected to be sufficient to fund the company’s operations through 1 year following the Phase 3 EFZO-FIT readout. - Plans to use some of the proceeds from the at-the-market offering program to help fund the commercial readiness plan.

In 2024, collaboration and license revenue related to the Kyorin agreement was $0.2 million. Research and development expenses were $54.4 million, general and administrative expenses were $13.8 million. The company ended 2024 with $75.1 million in cash, restricted cash, cash equivalents, and investments. Subsequent to the end of the fourth quarter 2024, it raised approximately $18.8 million in gross proceeds from its at-the-market offering program. No specific product segment revenue contribution percentages are detailed for product segments other than the general financials mentioned.

Forward-looking statements made during the call involve risks and uncertainties that can cause actual results to differ materially from those in the forward-looking statements. Please refer to the company’s press release, SEC filings (including the most recent annual report on Form 10-K, quarterly reports on Form 10-Q, and other SEC filings) for detailed risk factors.

Q: Derek Archila asked about how measuring the absolute change in steroid reduction at baseline now to week 48 versus the current way that you had set it up in terms of the average cumulative steroid dose and its impact on the trial.

A: Sanjay Shukla explained that previously looking at the average steroid dose in the week 12 through week 48 period was conservative. After discussion with the FDA, changing to measuring steroid reduction as the absolute change from baseline to week 48 simplifies analysis. Powering remains over 90% that either 3 or 5 milligram dose shows STAT-SIG steroid reduction compared to placebo.

Q: Derek Archila followed up on the percent of patients rolling over into the expanded access program.

A: Sanjay Shukla said interest in the EAP is robust but not all countries and centers can participate due to local regulatory requirements. Can't give specific numbers but interest is growing.

Q: Yasmeen Rahimi asked about the reason for the change in statistical analysis plan with the FDA and what to look for in baseline demographics at ATS.

A: Sanjay Shukla said it's about biostatistics pre-hoc analysis to avoid post-hoc cherry picking. At ATS, look for average prednisone dose, background immunomodulator use, and duration of disease.

Q: Faisal Khurshid asked about what happens if a patient is mid taper at week 48 and the durability of the drug impact.

A: Sanjay Shukla said placebo patients have time to unmask disease and multiple steroid tapers. Durability was shown in pooled analysis where 93% of patients on 3 or 5 mg doses didn't relapse in 6 months vs 55% on sub-therapeutic and placebo.

Q: Prakhar Agrawal asked about the Phase 1/2 steroid reduction under new definition, test powering, and FDA discussion on FEV1.

A: Sanjay Shukla said little discussion on PFTs, test powering remains over 90%, and qualitatively delta would be larger than 1.6 mg/day from Phase 1/2.

Q: Gregory Renza asked about expectations for the EFZO-CONNECT study's skin analysis and its guidance for other fibrotic diseases.

A: Sanjay Shukla said scleroderma ILD patients' primary morbidity is skin improvement. Looking at skin pathology, immune biomarkers, and neuropilin expression in skin plaques.

Q: Joe Pantginis asked about EAP data in BLA and current manufacturing readiness.

A: Sanjay Shukla said not slowing BLA timelines, EAP data may help BLA discussions if there are long-term durable effects. Made investments in manufacturing readiness for commercial supply.

Q: Liang Chang asked about drivers of statistical plan change with FDA and impact of scleroderma program interim analysis.

A: Sanjay Shukla said it's about simplification and working closely with regulators. Interim analysis of scleroderma program looks at skin histopathology, immune biomarkers, and Rodnan skin score.

Q: Dev Prasad asked about EAP data in BLA and next pipeline product plans.

A: Sanjay Shukla said EAP data may benefit BLA discussions if there are long-term durable effects. efzofitimod could have relevance in other ILDs, and pipeline includes ATYR0101 and ATYR0750 for anti-fibrotic and liver disorders.