Atyr Pharma, Inc. · NASDAQ:ATYR
Atyr Pharma — Investor Presentation (March 2019)
23 slides from Atyr Pharma's investor presentation, Mar 11, 2019
Slide 1: Translating New Immune Pathways into Meaningful Medicines
Translating New Immune Pathways into Meaningful Medicines NASDAQ: LIFE Exhibit 99.1 Corporate Presentation March 2019
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: Accelerating Value Creation from Novel Biology
Accelerating Value Creation from Novel Biology Platform of New Biology: Discovery pipeline of novel therapeutic candidates based on proprietary knowledge of extracellular functions of tRNA synthetases (~300 protein compositions patented) Lead Product Candidate: ATYR1923 Engineered, long acting, prot
Slide 4: Development Pipeline
Development Pipeline Program Discovery Preclinical Phase 1 Phase 2 Phase 3 Pulmonary Sarcoidosis Connective Tissue Disease ILD (CTD-ILD) Chronic Hypersensitivity Pneumonitis (CHP) Up to 4 tRNA Synthetases ATYR1923 Undisclosed CSL Behring Collaboration UNMC Collaboration
Slide 5: Novel tRNA Synthetase Domains Secreted Extracellularly with Non-Catalytic Functions
Novel tRNA Synthetase Domains Secreted Extracellularly with Non-Catalytic Functions Cell Circulation / Tissue Intracellularly full-length HARS catalyzes protein synthesis by transporting amino acid (histidine) to tRNA HARS and splice variants secreted extracellularly to regulate immune activity in c
Slide 6: Extracellular tRNA Synthetase Biology Associated with Disease in Multiple Tissues
Extracellular tRNA Synthetase Biology Associated with Disease in Multiple Tissues tRNA Synthetase Gene Families AARS CARS DARS EPRS FARS FARSB GARS HARS IARS KARS LARS MARS NARS QARS RARS SARS TARS VARS WARS YARS Drug: ATYR1923 Function: Immuno-modulatory Disease: Interstitial Lung Disease Known dis
Slide 7: Interstitial Lung Diseases Share Persistent Immune Engagement
Interstitial Lung Diseases Share Persistent Immune Engagement Fibrosis Pulmonary Sarcoidosis Connective Tissue Disease – ILD (CTD-ILD) Chronic Hypersensitivity Pneumonitis (CHP) Idiopathic Pulmonary Fibrosis (IPF) Inflammation Slide adapted from Dr. Steven Nathan, Medical Director, Advanced Lung Dis
Slide 8: High Unmet Need in Interstitial Lung Disease
High Unmet Need in Interstitial Lung Disease Pulmonary Sarcoidosis Systemic inflammatory disorder characterized by non-caseating granulomas (CD4+ T cell driven) US prevalence: ~200k ~30% of patients have chronic progressive disease, unresponsive to steroid treatment Current SOC: steroids - cytotoxic
Slide 9: Pre-Clinical Translational Estate Supports Clinical Development in ILD
Pre-Clinical Translational Estate Supports Clinical Development in ILD (1) 2017 annual sales of Esbriet® (pirfenidone) ~869M CHF, 13% increase YoY (2) 2017 annual sales of Ofev® (nintedanib) ~920M Euros, 52.3% increase YoY Bleomycin-Induced Lung Injury (Mouse) ATYR1923 vs. pirfenidone(1) ATYR1923 re
Slide 10: ATYR1923 For the Treatment of Pulmonary Sarcoidosis
ATYR1923 For the Treatment of Pulmonary Sarcoidosis
Slide 11: Sarcoidosis: The Most Common Form of Interstitial Lung Disease
Sarcoidosis: The Most Common Form of Interstitial Lung Disease (1) aTyr estimates for inflammatory ILD: Pulmonary Sarcoidosis, CHP, CTD-ILD; excludes IPF 30% with chronic progressive disease despite currently available treatment 50% require systemic therapy About 200,000 patients in United States $2
Slide 12: First-in-Patient Population: Pulmonary Sarcoidosis
First-in-Patient Population: Pulmonary Sarcoidosis Systemic inflammatory disorder characterized by the formation of granulomas (clumps of inflammatory cells) in one or more organs of the body CD4+ (Th1 / Th17) T-cell driven Usually begins in the lungs, skin or lymph nodes Sarcoidosis in the lungs is
Slide 13: ATYR1923: Novel Engineered Protein Therapeutic
ATYR1923: Novel Engineered Protein Therapeutic Active domain (iMod) is naturally occurring splice-variant of HARS that is enriched in the human lung Binds selectively to Neuropilin-2 (NRP2) Regulates a number of immune cell-types, including: T cells, Neutrophils, Macrophages, Dendritic cells HARS Sp
Slide 14: Receptor: Importance of NRP-2 as a Binding Partner for ATYR1923
Receptor: Importance of NRP-2 as a Binding Partner for ATYR1923 Pleiotropic receptor that can bind to a number of different ligands Well-established role in the development of the neural and lymphatic systems Emerging role in the immune system; present on a number of immune cell types Expressed on a
Slide 15: ATYR1923 Intervention in Pulmonary Sarcoidosis
ATYR1923 Intervention in Pulmonary Sarcoidosis ATYR1923 Therapeutic Hypothesis(1): Downregulates inflammatory insult and prevents progression to fibrosis (1) Hypothesized Immunopathogenesis of Sarcoidosis adapted from Iannuzzi et al, NEJM, 2007.
Slide 16: PK Profile Supports Potential Once-Monthly Dosing
PK Profile Supports Potential Once-Monthly Dosing 5 mg/kg 3 mg/kg 1 mg/kg 0.3 mg/kg 0.1 mg/kg 0.03 mg/kg D15 Safety Review D1 1hr IV infusion D29 Final Visit Cohort 1 Cohort 2 Cohort 3 Cohort 4 Cohort 5 Cohort 6 Phase 1 Healthy Volunteer Study Completed Positive data announced in June 2018 Randomize
Slide 17: ATYR1923 Phase 1b/2a Study in Pulmonary Sarcoidosis
ATYR1923 Phase 1b/2a Study in Pulmonary Sarcoidosis Objectives Evaluate safety, tolerability, PK, and immunogenicity of multiple ascending doses of ATYR1923 Evaluate signals of drug activity through steroid dose reduction and FDG-PET/CT changes Design Randomized, double-blind, placebo-controlled, mu
Slide 18: ATYR1923 Phase 1b/2a Study Endpoints
ATYR1923 Phase 1b/2a Study Endpoints Primary Safety and tolerability of multiple ascending IV ATYR1923 doses Secondary Steroid-sparing effect Immunogenicity Pharmacokinetics (PK) Exploratory efficacy measures: FDG-PET/CT imaging; Lung function (FVC); Serum biomarkers; Health-related quality of life
Slide 19: ATYR1923 Phase 1b/2a Study Schema
ATYR1923 Phase 1b/2a Study Schema 0 4 8 12 16 20 24 24-week Study Period Follow-up Week Each Cohort: Dose Escalation Review after 6 patients have received ≥3 doses of study drug (ATYR1923/placebo) Screening ATYR1923 Placebo 5 mg OCS Steroid taper Primary endpoint 5.0 mg/kg 3.0 mg/kg Cohort 1 Cohort
Slide 20: ATYR1923 Phase 1b/2a Study Status and Timelines
ATYR1923 Phase 1b/2a Study in Pulmonary Sarcoidosis Initiated Status Up to 15 leading Pulmonary Sarcoidosis centers in US Site initiation activities ongoing Recruiting activities initiated Timelines Interim data: 4Q 2019 Study completion: mid-2020(1) Possible Future Development Registrational trial
Slide 21: CSL Behring Collaboration
CSL Behring Collaboration Goal Identify new IND candidates from up to four tRNA synthetases from aTyr’s proprietary pipeline of novel proteins (non-HARS derived) Terms CSL Behring to fund all R&D costs aTyr eligible for up to $17m in option fees if CSL Behring advances all four programs ($4.25m per
Slide 22: Mission: Generate Value for Patients and Shareholders
Mission: Generate Value for Patients and Shareholders aTyr owns IP estate directed to a potential pipeline of proteins derived from 20 tRNA synthetase genes ATYR1923 in-vitro and in-vivo studies support clinical development in ILD Identification of NRP-2 receptor for ATYR1923 elucidates greater unde
Slide 23: Thank You
Thank You