Summary of aTyr Pharma FY Conference Call (December 02, 2025) Company Overview - **Company**: aTyr Pharma (NasdaqCM:ATYR) - **Focus**: Development of efzofitimod for the treatment of sarcoidosis Key Points and Arguments Clinical Data and Results - **EFZO-FIT Study**: Presented full topline results at ERS, highlighting steroid reduction and patient-reported outcomes (PROs) including KSQ Lung, KSQ General Health, and Fatigue Assessment Scale [4][5][6] - **Placebo Response**: Noted a high placebo response with a 60% reduction in the placebo group and 40% of patients getting off steroids, which was unexpected [5][6] - **Steroid Reduction**: The drug achieved over 70% reductions in steroids, with more than 50% of patients able to discontinue steroid use [5][6] - **Safety and Protocol**: The trial's rigorous protocol allowed for fine-tuning of steroid doses, which may have highlighted overdosing issues in real-world settings [6][7] Regulatory Considerations - **FDA Meeting**: Scheduled for Q1 2026, with a focus on presenting a comprehensive data package including pre-specified and post hoc analyses [12][18] - **Endpoints**: Pre-specified endpoints included steroid reduction, PROs, and safety measures. Post hoc analyses will explore different patient phenotypes and biomarker data [13][15] - **Clinical Benefit**: Experts view the data as demonstrating clear clinical benefit, with improvements in quality of life and durable responses observed [17][18] Future Directions - **Treatment Guidelines**: Anticipated changes in treatment guidelines based on trial results, shifting recommendations towards lower steroid doses [27][28] - **Confirmatory Studies**: Discussion on potential confirmatory studies if required by the FDA, with emphasis on validating new endpoints such as the KSQ Lung [25][30] - **Enrollment in Other Trials**: Ongoing enrollment in the EFZO-CONNECT study, targeting newly diagnosed ILD patients, with expected data release in the second half of the year [36][38] Market Outlook - **Investor Sentiment**: Analysts maintain a strong buy rating, indicating confidence in the drug's potential and the company's efforts [39][42] - **Patient Demand**: Increased interest from patients for participation in trials, emphasizing the need for therapies that improve quality of life and reduce steroid dependency [35][42] Additional Important Insights - **Phenotypic Variability**: The trial identified five different patient phenotypes, suggesting that FVC may not be the best primary endpoint for all patients [10][11] - **Quality of Life Focus**: The primary concerns for patients are improvements in quality of life and steroid reduction, rather than changes in pulmonary function tests [35][42] This summary encapsulates the critical aspects of aTyr Pharma's recent conference call, focusing on the company's clinical data, regulatory strategy, and market outlook.

Summary of aTyr Pharma (ATYR) 2025 Conference Call Company Overview - **Company**: aTyr Pharma (ATYR) - **Event**: 2025 Jefferies Global Healthcare Conference - **Focus**: Lead program for pulmonary sarcoidosis and updates on other indications Key Points on Pulmonary Sarcoidosis Program - **Phase Three Study**: Baseline demographics presented at ATS, showing expected characteristics in race, gender, and age of sarcoidosis patients [3][4] - **Steroid Dosage**: Average steroid dose at entry for the phase three trial was 10.5 mg, slightly lower than the 12-13 mg in phase two, aligning with treatment guidelines [4][6] - **Patient Management**: Patients on lower steroid doses are more sensitive to reductions, with a focus on managing flare-ups in the placebo group [12][13] - **FDA Feedback**: FDA recommended simplifying the statistical analysis plan to focus on the last four weeks of treatment, which increases the power to detect smaller differences [17][18] - **Power Assumptions**: The trial is powered to show a statistically significant absolute reduction of 3 mg in steroid use compared to placebo [22][23] - **Market Opportunity**: The target population for the therapy is larger than initially estimated, with 150,000 to 160,000 steroid-dependent patients in the U.S. [48][49] Other Indications and Updates - **Scleroderma-Related ILD Study**: Interim data showed promising results in skin improvement, particularly in diffuse scleroderma patients [60][62] - **Potential for Broader Applications**: Interest in expanding the use of epsofitimod to other forms of interstitial lung disease (ILD) and connective tissue disease ILD, with a potential market value exceeding $5 billion [70][71] Financial Position - **Cash Position**: The company has sufficient cash to support operations through the upcoming readouts and for at least another year beyond that [72] Conclusion - aTyr Pharma is positioned to potentially change treatment paradigms in pulmonary sarcoidosis and other ILDs, with significant market opportunities and a strong financial position to support ongoing and future trials.