Atyr Pharma, Inc. · NASDAQ:ATYR

Atyr Pharma — Investor Presentation (January 2018)

18 slides from Atyr Pharma's investor presentation, Jan 8, 2018

  1. Slide 1: Corporate Presentation January 2018

    Harnessing Newly Discovered Pathways in Immunology Effected by Extracellular tRNA Synthetases CORPORATE PRESENTATION January 2018 Exhibit 99.1

  2. 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,”

  3. Slide 3: Accelerating Value Creation from Novel Immune Pathways

    Accelerating Value Creation from Novel Immune Pathways Research: Discover innovative therapeutic candidates based on extracellular functionality of tRNA synthetases Initial focus on extracellular histidyl-tRNA synthetase (HARS) Development: ATYR1923 (interstitial lung diseases) in ongoing Phase 1 tr

  4. Slide 4: Therapeutic Candidate Pipeline

    Therapeutic Candidate Pipeline Resokine Pathway DISCOVERY Preclinical Phase 1 Phase 2 Phase 3 ATYR1923 (Engineered HARS)* Interstitial Lung Disease ORCA (HARS antibodies) Various Cancers ATYR1940 (~Full-length HARS) Rare Muscular Dystrophies tRNA Synthetase Pipeline DISCOVERY Preclinical Phase 1 Pha

  5. Slide 5: Resokine: Extracellular Proteins Derived From HARS Gene

    AARS CARS DARS EPRS FARS GARS HARS IARS KARS LARS MARS NARS QARS RARS SARS TARS VARS WARS YARS Full-length HARS Splice variant of HARS One example of multiple splice variant proteins Extracellular: (Circulation) Intracellular: (Cytoplasm) Secretion Resokine: Extracellular Proteins Derived From HARS

  6. Slide 6: Resokine MOA Hypothesis: Regulates T Cell Activation

    Stimulatory pathways at levels closer to a resting T cell Effector functions at levels closer to a resting T cell Resokine MOA Hypothesis: Regulates T Cell Activation Acts on both CD4 and CD8 T cells Shifts trafficking and residence closer to a resting T cell

  7. Slide 7: Resokine Regulates T Cell Activation

    Resokine Regulates T Cell Activation Similar for: INFγ, TNFα… Similar to hitting PD-1 pathway Anti-CD3/CD28 (24 hr.) Stimulation + Vehicle T cell Activation Marker CD4+ T cell count Anti-CD3/CD28 Stimulation + Resokine (100pM) T cell Activation Marker CD4+ T cell count Graphs on Right: T cells were

  8. Slide 8: Engineered HARS Splice Variant (iMod.Fc) ATYR1923 for Interstitial Lung Diseases

    Engineered HARS Splice Variant (iMod.Fc) ATYR1923 for the Treatment of Interstitial Lung Diseases

  9. Slide 9: ATYR1923: Program Snapshot

    ATYR1923: Program Snapshot ATYR1923 (iMod.Fc): Engineered fusion protein with HARS splice variant Refer to splice variant as the “iMod domain” (iMod for immuno-modulatory function) Patients: Interstitial lung diseases (ILDs) characterized by an immune component Mechanism: Regulation of T cell activa

  10. Slide 10: Interstitial Lung Diseases Share Persistent Immune Engagement

    Slide adapted from Dr. Steven Nathan, Medical Director, Advanced Lung Disease and Transplant Program at Inova Fairfax Hospital, Falls Church, VA Interstitial Lung Diseases Share Persistent Immune Engagement Fibrotic Sarcoidosis Systemic Sclerosis – ILD (SSc-ILD) Chronic Hypersensitivity Pneumonitis

  11. Slide 11: ATYR1923 Ameliorates Fibrosis in Bleomycin-Induced Lung Injury

    ATYR1923 Ameliorates Fibrosis in Bleomycin-Induced Lung Injury Experiment 1 Experiment 2 ATYR1923 (iMod.Fc) administered therapeutically at 0.4 mg/kg weekly drives efficacy comparable to or greater than Pirfenidone*, anti-TGF antibodies, and dexamethasone *Pirfenidone: Current approved therapy for I

  12. Slide 12: ATYR1923 Clinical Development for Interstitial Lung Diseases

    ATYR1923 Clinical Development for Interstitial Lung Diseases Clinical Overview Randomized, double-blind, placebo-controlled studies to investigate the safety, tolerability, immunogenicity, pharmacokinetics and pharmacodynamics of intravenous ATYR1923 (iMod.Fc) in healthy volunteers and patients with

  13. Slide 13: Antibodies to the Resokine Pathway: ORCA

    Antibodies to the Resokine Pathway ORCA – Targeting a Novel Immune Set Point for Cancer Patients

  14. Slide 14: Regulating T Cells to Temper or Enhance Anti-Tumor Immunity

    Regulating T Cells to Temper or Enhance Anti-Tumor Immunity Resokine “Agonist” Regulates T cell activation with potential to temper immune system ORCA “Antagonist” Unlocks T cell activity with potential to enhance anti-tumor immunity

  15. Slide 15: Anti-Synthetase Syndrome: Evidence of Resokine Pathway Relevance

    Anti-Synthetase Syndrome: Evidence of Resokine Pathway Relevance in a Human Disease Setting Healthy Tissue T Cells Mobilized in Tissue Resokine acts as homeostatic regulator of immune activity Antibodies to Resokine lower threshold for T cell activation Resokine disruption via auto-antibodies Health

  16. Slide 16: ORCA Program: Snapshot

    ORCA Program: Snapshot Patients: Potentially all cancer types: >450 patient samples in over 10 tumor types tested ~95% of cancer patients tested positive for Resokine Target: Resokine pathway Therapeutic Concept: Antibody to block Resokine activity, increases T cell engagement Rationale: Human evide

  17. Slide 17: ORCA Program: Supportive In Vivo Data and Development Timelines

    ORCA Program: Supportive In Vivo Data and Development Timelines Development Timelines In Vivo Efficacy Data Resokine antibody selection: Panel of antibodies selected and in IND enabling activities Present Data at Scientific Conferences: Abstract at ASCO-SITC in January 2018 Additional presentations

  18. Slide 18: Accelerating Value Creation from Novel Immune Pathways: 2018 Strategic Goals

    Accelerating Value Creation from Novel Immune Pathways 2018 Strategic Goals: Advance Clinical Development ATYR1923 Phase 1 ongoing with data in 2Q 2018 Advance Immuno-Oncology Program IND-enabling activities ongoing for patient trials in 2019 Discovery and Pipeline Enhancement Collaborating with aca

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