Atyr Pharma, Inc. · NASDAQ:ATYR
Atyr Pharma — Investor Presentation (June 2026)
27 slides from Atyr Pharma's investor presentation, Jun 1, 2026
Slide 1: Advancing tRNA synthetase biology: Efzofitimod in Pulmonary Sarcoidosis and Beyond
aTyr Pharma Advancing tRNA synthetase biology Efzofitimod in Pulmonary Sarcoidosis and Beyond June 2026 © 2026 aTyr Pharma Exhibit 99.1
Slide 2: Forward-Looking Statements
Forward-Looking Statements The following slides and any accompanying oral presentation contain forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as “may,” “might,” “will,” “should,” “expect,”
Slide 3: Strategic Outlook: Creating First-In-Class Therapies in Inflammatory and Fibrotic Diseases
aTyr’s Strategic Outlook: Creating First-In-Class Therapies that Modulate New Pathways in Inflammatory and Fibrotic Diseases Efzofitimod is a biologic immunomodulator with a novel complementary mechanism of action: EFZO-FIT: largest interventional pulmonary sarcoidosis trial conducted to date; gener
Slide 4: Sarcoidosis is a Serious Orphan Disease in Need of an FDA-Approved Treatment
Sarcoidosis is a Serious Orphan Disease in Desperate Need of a Safe and Effective FDA-Approved Treatment 1aTyr Pharma, Inc. data on file. Abbreviations: IST, immunosuppressive therapy; QoL, quality of life; SoC, standard of care, TNF, tumor necrosis factor. Pulmonary Sarcoidosis Disease pathology In
Slide 5: A New Candidate for Pulmonary Sarcoidosis: Efzofitimod
A New Candidate for Pulmonary Sarcoidosis Efzofitimod A biologic immunomodulator that targets NRP2 on activated macrophage cells to resolve inflammation without immune suppression and may prevent fibrosis progression 1Culver D, et al., CHEST. 2023;163, 881-890. 2Nangle LA, et al. Sci Transl Med. 202
Slide 6: EFZO-FIT: Largest Interventional Trial Conducted in Pulmonary Sarcoidosis
Follow up EFZO-FIT: Largest Interventional Trial Conducted in Pulmonary Sarcoidosis EFZO-FIT 1268 patients were randomized; 264 patients were randomized and dosed Topline data reported Q3 ‘25. *Time frame BL to W48. Abbreviations: BL, baseline; Dx, diagnosis; KSQ-L, King’s Sarcoidosis Questionnaire-
Slide 7: Substantial Steroid Reduction Observed in all EFZO-FIT Groups
Substantial Steroid Reduction Observed in all EFZO-FIT Groups Provides Important Insights Although the primary endpoint of the study was not met, we learned that patients with chronic, symptomatic pulmonary sarcoidosis can be managed for prolonged periods on lower doses of OCS without worsening of d
Slide 8: Clinical Activity Across Multiple Pre-specified QoL Endpoints
p=0.0479 Weeks Estimates with 95% CIs from MMRM Evidence of Clinical Activity Demonstrated Across Multiple Pre-specified QoL Endpoints EFZO-FIT *Difference in LS Means from Placebo (with 95% CIs) from MMRM. Abbreviations: BL, baseline; CI, confidence interval; KSQ-L, King’s Sarcoidosis Questionnaire
Slide 9: Clinical Activity Across Multiple Pre-specified QoL Endpoints (KSQ-GH, FAS)
Evidence of Clinical Activity Demonstrated Across Multiple Pre-specified QoL Endpoints EFZO-FIT *Difference in LS Means from Placebo (with 95% CIs) from MMRM. Abbreviations: BL, baseline; CI, confidence interval; FAS, fatigue assessment scale; KSQ-GH, King’s Sarcoidosis Questionnaire-General Health
Slide 10: Forced Vital Capacity Maintained Despite Steroid Taper
Forced Vital Capacity Maintained Despite Steroid Taper EFZO-FIT EFZO-FIT enrolled a heterogenous patient population with various pulmonary phenotypes *Difference in LS Means from Placebo (with 95% CIs) from MMRM. Abbreviations: BL, baseline; CI, confidence interval; FVC, forced vital capacity; L, li
Slide 11: Efzofitimod Generally Well-Tolerated with Consistent Safety Profile
Efzofitimod was generally well-tolerated at both the 3.0 mg/kg and 5.0 mg/kg doses, consistent with a previously observed safety profile in all trials conducted to date AEs were mostly mild or moderate in severity and generally assessed as unrelated to the study drug SAEs were limited and balanced b
Slide 12: FDA Type C Meeting Informs Future Efzofitimod Development
FDA Type C Meeting Informs Future Efzofitimod Development FDA indicated support for FVC and KSQ-L as clinically meaningful endpoints in pulmonary sarcoidosis KSQ-L: FDA recommended the inclusion of wheezing and cough severity as separate measures of patient symptoms, along with new anchors to interp
Slide 13: Sarcoidosis Heterogenous Lung Phenotypes; FVC Relevant in Restrictive Phenotype
Sarcoidosis has Heterogenous Lung Phenotypes; FVC Relevant Only in Restrictive Phenotype Path Forward FVC has been used as a registrational endpoint in related ILD indications, with approvals being granted for placebo adjusted differences as low as 45 mL *Sharp M, et al. Ann Am Thorac Soc. 2023 Jan;
Slide 14: EFZO-FIT Data Support FVC as Primary Endpoint in Target Population
Data from EFZO-FIT Support Our Rationale for FVC as Primary Endpoint in Target Patient Population Baseline characteristics were generally similar to the ITT population with a few notable exceptions Expectedly, the FVCpp in the restrictive subset (~70%) was lower when compared to the ITT population (
Slide 15: Benefit on PROs Remains Consistent in Restrictive Population
Benefit on PROs Observed in EFZO-FIT in ITT Remains Consistent in Restrictive* Population Path Forward *Restrictive by FVCpp: Baseline FVCpp ≤80% & FEV1/FVC ration ≥0.7. †RCRM model est. adj. for Baseline MMLUNGSS. ‡RCRM model est. adj. for Baseline MMGENHSS. §RCRM model est. adj. for Baseline MMFAS
Slide 16: Go-Forward Development Plan Leverages EFZO-FIT Insights and FDA Engagement
Go-Forward Development Plan Leverages Insights from EFZO-FIT and Engagement with FDA Path Forward Study design to incorporate key changes intended to demonstrate compelling evidence of efficacy and safety on clinically meaningful endpoint Abbreviations: FVC, forced vital capacity; Q3W, every three w
Slide 17: Phase 3 Randomized Double-Blind Placebo-Controlled Study Design
Continue on stable background therapy (OCS 0 to ≤5 mg/d) Continue on stable background IS Screening A Phase 3 Randomized Double-Blind Placebo-Controlled Study to Evaluate Efzofitimod in Patients with Pulmonary Sarcoidosis Target Population: Moderate to severe pulmonary sarcoidosis with restrictive d
Slide 18: Proposed Key Inclusion and Exclusion Criteria
Proposed Key Inclusion and Exclusion Criteria Path Forward Study focuses on the right patients for the right endpoints Abbreviations: DLCOpp, diffusing capacity of the lungs for carbon monoxide, percent predicted; FEV1, forced expiratory volume in 1 second; FVCpp, forced vital capacity, percent pred
Slide 19: US Pulmonary Sarcoidosis Target Population
*<80% FVCpp; FEV1/FVC ≥0.7. †>20% on HRCT; †Diagnosed pulmonary means ICD10 code D86.0 or D86.2. Sharp M, et al. Ann Am Thorac Soc. 2023 Jan;20(1):30-37; Obi O, et al. Chest. 2024 Apr; 165(4):892-907. Abbreviations: FEV1, forced expiratory volume in 1 second; FVC, forced vital capacity; FVCpp, force
Slide 20: Efzofitimod Targets NRP2 Biology to Reset Pro-Inflammatory Macrophages
Efzofitimod Targets NRP2 Biology to Reset Pro-Inflammatory Macrophages MOA Nangle LA, et al. Sci Transl Med. 2025 Mar 12;17(789):eadp4754. Abbreviations: CCR2, C-C Chemokine Receptor Type 2; CD14, cluster of differentiation 14; IL-6, interleukin-6; MCP-1, monocyte chemoattractant protein-1; MOA, mec
Slide 21: Interstitial Lung Disease Requires New Treatment Approaches
ILD is an umbrella term including >200 rare lung disorders characterized by inflammation and scarring of the lung interstitium Many ILDs share common clinical presentations and pathologic features, and are managed similarly to pulmonary sarcoidosis with immunomodulatory therapy Treatment goals are t
Slide 22: SSc-ILD is a Common and Deadly Manifestation of Systemic Sclerosis
SSc-ILD is a Common and Deadly Manifestation of Systemic Sclerosis SSc-ILD Abbreviations: BAL, bronchoalveolar lavage; IL-6, interleukin-6; ILD, interstitial lung disease; MCP-1, monocyte chemoattractant protein-1; SSc, systemic sclerosis; SSc-ILD, systemic sclerosis-associated interstitial lung dis
Slide 23: EFZO-CONNECT: Phase 2 Study in SSc-ILD
Target Population: SSc with progressive ILD Enrollment: N=25 Patients with SSc (ACR/EULAR criteria), and ILD (BL HRCT) Progressive disease (recent onset, evidence for inflammation, diffuse cutaneous SSc) On background mycophenolate therapy or equivalent Primary Objective: Assess the efficacy of efzo
Slide 24: Our tRNA Synthetase Library: Potential Candidates in a New Class of Medicine
Our tRNA Synthetase Library: Potential Candidates in a New Class of Medicine Full-length tRNA synthetases Natural fragments and variants Novel extracellular functions gained through evolutionary intelligence Pipeline products Pipeline | © 2026 aTyr Pharma WHEP WHEP Fc Fc Efzofitimod D-N1 D-N1 Fc Fc
Slide 25: Efzofitimod Leads a Growing Pipeline of tRNA Synthetase-Derived Biologics
PROGRAM tRNA SYNTHETASE MOA INDICATION PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Efzofitimod HARS NRP2 modulator Pulmonary Sarcoidosis SSc-ILD Other ILD ATYR0101 DARS Myofibroblast depleter Fibrosis ATYR0750 AARS FGFR4 modulator Fibrosis tRNA Synthetase Candidates1 Multiple Multiple Multiple Efzofitimod L
Slide 26: Pulmonary Sarcoidosis, Efzofitimod Opportunity, Pipeline, IP and Cash
Pulmonary sarcoidosis Efzofitimod opportunity Pipeline, IP and cash Efzofitimod is a biologic immunomodulator with a novel and complementary mechanism of action Efzofitimod in position to be first to market in pulmonary sarcoidosis without near term competition aTyr retains a robust IP-protected tRN
Slide 27: Thank You
Thank You © 2026 aTyr Pharma = Branded 2D science visualization