Atyr Pharma, Inc. · NASDAQ:ATYR
Atyr Pharma — European Respiratory Society (ERS) Congress 2025
15 slides from Atyr Pharma's conference presentation, September 2025
Slide 1: EFZO-FIT: The Largest Ever Interventional Trial in Pulmonary Sarcoidosis
EFZO-FIT: The Largest Ever Interventional Trial in Pulmonary Sarcoidosis Daniel Culver, DO Clevela nd Cl ini c On beha lf of the EFZO -FIT i nvestigators
Slide 2: Conflict of interest disclosure
Conflict of interest disclosure ❑ I have no real or perceived conflicts of interest that relate to this presentation. x I have the following real or perceived conflicts of interest that relate to this presentation: ❑ Affiliation / Financial interest Commercial company Grants/research support: aTyr (
Slide 3: Efzofitimod Mechanism of Action
Efzofitimod Mechanism of Action MCP-1 TNFα IL-6 If left untreated, persistent macrophage driven inflammation can lead to progressive fibrosis Immune triggers recruit monocytes, which differentiate to pro- inflammatory macrophages expressing NRP2 MCP-1 TNFα IL-6 Efzofitimod acts as an NRP2 agonist Ef
Slide 4: EFZO-FIT Study Design
EFZO-FIT Study Design Treatment Follow-up Efzofitimod 5 mg/kg (n=88) 1:1:1 Randomization Efzofitimod 3 mg/kg (n=88) Placebo (n=88) Dosing Week 0 4 8 12 16 20 24 28 32 36 40 44 48 52 Begin OCS Primary Endpoint taper Population Endpoints Diagnosis of pulmonary sarcoidosis for ≥6 months Primary: Stable
Slide 5: Study Disposition
Study Disposition Screened 331 Excluded 63 Randomized 268 Not dosed 4 ITT placebo ITT 3 mg/kg efzofitimod ITT 5 mg/kg efzofitimod 90 86 88 Discontinued treatment Discontinued treatment Discontinued treatment 11 10 7 Completed study1 Completed study1 Completed study1 80 76 84 1Completed at least 10 d
Slide 6: Baseline Characteristics
Baseline Characteristics Placebo Efzofitimod 3 mg/kg Efzofitimod 5 mg/kg N=90 N=86 N=88 Age (Years); mean (SD) 54.1 (10.5) 54.5 (11.9) 52.7 (9.2) Sex (Male); n (%) 56 (62.2) 47 (54.7) 47 (53.4) Race Black / African American; n (%) 15 (16.7) 12 (14.0) 16 (18.2) Asian; n (%) 15 (16.7) 11 (12.8) 10 (11
Slide 7: Average Change in OCS Dose (mg)
Change from Baseline in OCS Average Change from Baseline in OCS Dose at Week 48 OCS Dose Over Time Placebo Efzofitimod Efzofitimod Average Change in OCS Dose (mg) 3 mg/kg 5 mg/kg N=90 N=86 N=88 LS mean dose at week 3.5 3.5 2.8 48 (mg) LS mean change from -7.1 -7.1 -7.9 baseline (mg) Difference in LS
Slide 8: Proportion of Subjects Steroid Free
Steroid Withdrawal Steroid Free at Week 48 Kaplan Meier Curve of Time to Steroid Free Sustained for 180 Days Placebo Efzofitimod Efzofitimod Proportion of Subjects Steroid Free 3 mg/kg 5 mg/kg N=90 N=86 N=88 Steroid free1; n (%) 36 (40.2) 45 (51.8) 46 (52.6) Odds ratio (95% CI) - 1.6 (0.9, 3.0) 1.7
Slide 9: King's Sarcoidosis Questionnaire-Lung
King’s Sarcoidosis Questionnaire-Lung Change from Baseline in KSQ-L Differences in KSQ-L Change from at Week 48 Baseline vs Placebo* Placebo Efzofitimod Efzofitimod 3 mg/kg 5 mg/kg N=90 N=86 N=88 LS mean change from 6.2 7.3 10.4 baseline Difference; LS mean (95% CI) - 1.1 (-3.1, 5.4) 4.2 (0.0, 8.3)
Slide 10: Consistent Benefit Observed on QOL
Consistent Benefit Observed on QOL Differences in FAS Total Score Change from Differences in KSQ-GH Change from Baseline vs Placebo* Baseline vs Placebo* Time (weeks) Time (weeks) Efzofitimod 3 mg/kg Efzofitimod 5 mg/kg Week 48 difference in LS mean change from Week 48 difference in LS mean change f
Slide 11: King's Sarcoidosis Questionnaire-Lung / Steroid Free Composites
King’s Sarcoidosis Questionnaire-Lung / Steroid Free Composites Steroid Free and KSQ-L Composite Efzofitimod 5 mg/kg vs Placebo from Logistic Endpoints Regression Placebo Efzofitimod Efzofitimod 3 mg/kg 5 mg/kg N=90 N=86 N=88 Steroid-Free Steroid free and stable KSQ-L; 33 (36.7) 41 (47.7) 41 (46.6)
Slide 12: Average Change in FVC (L)
Forced Vital Capacity (FVC) FVC at Week 48 (MMRM) Change from Baseline in FVC (L) over Time Placebo Efzofitimod Efzofitimod 3 mg/kg 5 mg/kg Average Change in FVC (L) N=90 N=86 N=88 FVC (mL), LS mean at week 48 3380.4 3369.7 3395.4 LS mean change from baseline (mL) -84.5 -95.1 -69.4 Difference in LS
Slide 13: Summary of SAE, AE Discontinuations and ADA
Summary of SAE, AE Discontinuations and ADA Placebo Efzofitimod 3 mg/kg Efzofitimod 5 mg/kg N=90 N=86 N=88 SAE 10 12 7 Treatment related (per PI) 2 (pneumonia, scrotal abscess) 2 (PPF, RSV infection) 1 (pneumonia) Discontinuations due to AE 4 3 4 Treatment related (per PI) 2 (hypersensitivity, hypot
Slide 14: Conclusions
Conclusions • EFZO-FIT is the largest-ever RCT in sarcoidosis • Pulmonary sarcoidosis patients can be managed for prolonged periods with lower steroid doses than we are currently using • Efzofitimod is safe and well-tolerated • The trial did not meet its primary endpoint (change from baseline in mea
Slide 15: ERS Congress 2025, Amsterdam
@ ERS CONGRESS|2025 27 September - 1 October | Amsterdam, Netherlands
aTyr Pharma — European Respiratory Society (ERS) Congress 2025 on 6ix