Atyr Pharma, Inc. · NASDAQ:ATYR
Atyr Pharma — Investor Presentation (September 2023)
21 slides from Atyr Pharma's investor presentation, Sep 26, 2023
Slide 1: The Evolutionary Intelligence Biotech
The Evolutionary Intelligence Biotech September 2023 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: Translating tRNA Synthetases into New Therapies for Fibrosis and Inflammation
Translating tRNA Synthetases into New Therapies for Fibrosis and Inflammation Novel extracellular functions gained through evolutionary intelligence Potential new class of medicines IP directed to protein compositions from all 20 tRNA synthetase genes Clinical proof of concept established Phase 3 EF
Slide 4: Evolutionary Intelligence: tRNA Synthetases Evolved to Regulate Complex Systems
Evolutionary Intelligence: tRNA Synthetases Evolved to Regulate Complex Systems Domains are released locally from full-length proteins enabling their function as extracellular signaling molecules Growing evidence that domains function to restore homeostasis through new therapeutic intervention point
Slide 5: Increasing Validation of aTyr Science in Peer Reviewed Journals
Increasing Validation of aTyr Science in Peer Reviewed Journals “Efzofitimod: a novel anti-inflammatory agent for sarcoidosis” – first major review article for efzofitimod (https://doi.org/10.36141/svdld.v40i1.14396); “Efzofitimod for the treatment of pulmonary sarcoidosis” – Phase 1b/2a data public
Slide 6: Growing Pipeline of First-in-Class tRNA Synthetase Derived Biologics
Growing Pipeline of First-in-Class tRNA Synthetase Derived Biologics PROGRAM TARGET/MOA INDICATION RESEARCH PRECLINICAL PHASE 1 PHASE 2 PHASE 3 tRNA synthetase programs Efzofitimod NRP2 modulator Pulmonary Sarcoidosis(1) SSc-ILD Other ILD (CTD-ILD; CHP) ATYR0101 LTBP1 modulator Fibrosis ATYR0750 FGF
Slide 7: Efzofitimod: First-in-Class Biologic Immunomodulator for ILD
Efzofitimod First-in-Class Biologic Immunomodulator for Interstitial Lung Disease (ILD)
Slide 8: Efzofitimod: First-in-Class Biologic Immunomodulator for ILD
Efzofitimod: First-in-Class Biologic Immunomodulator for ILD Fc fusion protein Active domain is naturally occurring, lung enriched domain of HARS Downregulates activated myeloid cells via NRP2 Anti-inflammatory and anti-fibrotic effects demonstrated in multiple ILD models Dosed once-monthly via 60 m
Slide 9: Efzofitimod Therapeutic Hypothesis: Restore Immune Balance to Prevent Fibrosis
Efzofitimod Therapeutic Hypothesis: Restore Immune Balance to Prevent Fibrosis *NRP2 also present on fibroblasts Baughman et al. Efzofitimod: A Novel Anti-inflammatory Agent for Sarcoidosis. Sarc Vasc And Diff Lung Dis. 2023 Diverse immune triggers activate common immune pathways NRP2 upregulated on
Slide 10: aTyr is Advancing Efzofitimod as the Standard-of-Care for ILD
aTyr is Advancing Efzofitimod as the Standard-of-Care for ILD Number of U.S. ILD Patients by Types (‘000) ILD is an umbrella term for >200 types of rare lung diseases that span a spectrum of inflammation and fibrosis Patients experience high morbidity and mortality No disease-modifying therapies ava
Slide 11: Significant Market Opportunity in Pulmonary Sarcoidosis Alone
Significant Market Opportunity in Pulmonary Sarcoidosis Alone Pulmonary Sarcoidosis Pathology / Target Relevance Inflammatory disease characterized by non-caseating granulomas NRP2 upregulated in sarcoid granulomas Epidemiology 200K pts in the U.S.; >1M globally Lung predominant in >90% Up to 20% de
Slide 12: Clinical Proof of Concept Demonstrated in Phase 1b/2a Pulmonary Sarcoidosis Trial
Clinical Proof of Concept Demonstrated in Phase 1b/2a Pulmonary Sarcoidosis Trial Primary objective met: Efzofitimod was safe and well-tolerated (n=37) Secondary objectives met: Dose-response observed across all three families of pre-specified endpoints Dose-dependent improvement of inflammatory bio
Slide 13: Global Phase 3 Trial Enrolling in Pulmonary Sarcoidosis
Global Phase 3 Trial Enrolling in Pulmonary Sarcoidosis Population: moderate to severe pulmonary sarcoidosis Diagnosis of pulmonary sarcoidosis for ≥6 months Stable treatment with ≥ 7.5 and ≤ 25 mg/day OCS Extent of fibrosis < 20% Primary Endpoint Steroid burden: change in daily steroid dose Key Sec
Slide 14: SSc-ILD Represents Expanded Commercial Opportunity
SSc-ILD Represents Expanded Commercial Opportunity SSc-ILD Pathology / Target Relevance Lung manifestation of systemic sclerosis (SSc), an autoimmune disease characterized by scarring of skin and other organs NRP2 upregulated in skin macrophages Epidemiology >60K in the U.S.; >1.5M globally 25-30% d
Slide 15: Phase 2 POC Trial Initiated in SSc-ILD
Phase 2 POC Trial Initiated in SSc-ILD Population: SSc with progressive ILD Patients with SSc (ACR/EULAR criteria), and ILD (baseline HRCT) Progressive disease (recent onset, evidence for inflammation, diffuse cutaneous SSc) On background mycophenolate therapy Primary Endpoint Lung function: forced
Slide 16: Efzofitimod Target Value Proposition
Efzofitimod Target Value Proposition Binds new target for ILD upstream not repurposed or failed Targets innate immunity at site of inflammation downregulates pro-inflammatory and pro-fibrotic pathways addresses complex immune pathology restores immune balance without evidence of suppression Robust e
Slide 17: Pre-Clinical Pipeline Generating New Treatments for Inflammation and Fibrosis
Pre-Clinical Pipeline Generating New Treatments for Inflammation and Fibrosis
Slide 18: tRNA Synthetase Platform
tRNA Synthetase Platform Approach validated through efzofitimod clinical POC HARS derived NRP2 modulator efzofitimod currently in Phase 3 in pulmonary sarcoidosis Unique drug discovery platform leveraging evolutionary intelligence Extracellular tRNA synthetases can unlock new targets and / or signal
Slide 19: Translating tRNA Synthetase Biology into New Therapies for Inflammation and Fibrosis
Translating tRNA Synthetase Biology into New Therapies for Inflammation and Fibrosis
Slide 20: Translating tRNA Synthetases into New Therapies for Inflammation and Fibrosis
Translating tRNA Synthetases into New Therapies for Inflammation and Fibrosis Evolutionary intelligence drug discovery platform Extracellular tRNA synthetases represent potential new class of medicines aTyr owns IP directed to entire class Lead program in pivotal development for untapped blockbuster
Slide 21: Thank You
alyr Thank You