Atyr Pharma, Inc. · NASDAQ:ATYR
Atyr Pharma — Investor Presentation (January 2017)
37 slides from Atyr Pharma's investor presentation, Jan 9, 2017
Slide 1: Advancing New Therapeutic Horizons: Corporate Presentation January 2017
Advancing New Therapeutic Horizons Harnessing Novel Physiocrine Biology To Promote Homeostasis Corporate Presentation January 2017 NASDAQ: LIFE 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: LIFE Value Proposition and Opportunities
$76M estimated cash 2016 EOY* $51M market capitalization 2016 EOY Pursuing partnership(s) for one or more programs to accelerate clinical and preclinical pipeline Pioneers of new therapeutic intervention points in homeostasis - The World of Physiocrines LIFE Value Proposition LIFE’s Opportunities Cl
Slide 4: The Power of Physiocrines Orchestrating Homeostasis
The Power of Physiocrines Orchestrating Homeostasis New Class of Proteins from Alternative Splicing of Ancient Genes
Slide 5: Orchestrating Homeostatic Pathways for Novel Therapies
Nature 2015 Science 2014 Nature 2013 Nature 2010 Science 1999 Orchestrating Homeostatic Pathways for Novel Therapies Discovery of potential therapeutic intervention points Tapping an Ancient Source of Power Physiocrines may promote homeostasis and provide for novel therapeutic intervention points fo
Slide 6: Evidence for Homeostatic Role of a Physiocrine in Humans
Evidence for Homeostatic Role of a Physiocrine in Humans Disrupting the Resokine Pathway Promotes Muscle and Lung Disease Auto-antibodies Homeostasis Imbalance Immune cell invasion/activity Resokine Pathway Diseased Muscle Diseased Lung Healthy muscle Healthy lung 100% (18 of 18) anti-synthetase syn
Slide 7: Free Resokine Pathway in Anti-Synthetase Patients Diminished
Free Resokine Pathway in Anti-Synthetase Patients Diminished Unpublished data from aTyr and collaborator Statistics: Mann-Whitney test Resokine Pathway in Humans 69% at or below limit of detection
Slide 8: Agonists of the Resokine Pathway in Immune Driven Models
Agonists of the Resokine Pathway in Immune Driven Models Balancing the immune response to tissue insults In vivo administration of Resokine proteins to animal models of T cell driven disease states. Cell type indicates type of cells involved but may not be limited to these cells. Resokine Pathway Im
Slide 9: Resokine: 1st Physiocrine Pathway Harnessed
Resokine: 1st Physiocrine Pathway Harnessed “Resolution of immune activity” Muscle Resokine pathway relates to a secreted 57kD protein from skeletal muscle (full length HARS*) Lung Resokine pathway relates to a 7kD protein (the iMod domain, a splice variant of HARS) MOA Involved in muscle differenti
Slide 10: Resolaris: Harnessing the Resokine Pathway to Treat Multiple Rare Muscle Diseases
Resolaris Harnessing the Resokine Pathway to Treat Multiple Rare Muscle Diseases
Slide 11: Rare Myopathies with an Immune Component (RMIC)
Rare Myopathies with an Immune Component (RMIC) Chronic damage, homeostasis disrupted Genetic Mutation Multiple proteins Dysferlin proteins Dystrophin proteins FSHD LGMD2B DMD Aberrant Protein Expression Localized T Cell Invasion/Proliferation Potential to link genotype to specific T cell phenotype
Slide 12: Resolaris Tempers Activated T cells
Resolaris Tempers Activated T cells Demonstrated effect as an immuno-modulator In Vitro T Cell Modulation Anti-CD3/CD28 Stimulation + Vehicle T cell Activation Marker CD4+ T cell count Anti-CD3/CD28 Stimulation + Resolaris T cell Activation Marker CD4+ T cell count Resolaris appears to work on activ
Slide 13: First Physiocrine For Patients: Resolaris
First Physiocrine For Patients: Resolaris Skeletal muscle secretes Resokine Resokine, an agonist, plays a role in homeostasis & T cell responses in muscle Recombinant version of Resokine Demonstrated favorable safety profile and potential clinical activity in two rare myopathy indications Therapeuti
Slide 14: Few Treatment Options: FSHD, LGMD2B, & DMD
Few Treatment Options: FSHD, LGMD2B, & DMD DMD LGMD2B Genetics Immune Pathology Clinical Standard of Care Disease Progression Toxic gain of function (DUX4 region) Immune infiltration1 by activated T cells (CD8+) Loss of function mutations (Dysferlin gene) Immune infiltrates2 of CD4+, CD8+ and macrop
Slide 15: RMIC Disease Progression Case History (LGMD2B)
RMIC Disease Progression Case History (LGMD2B) Difficulty rising from a chair, raising hands above head Uses a cane and leg braces Walks normally, active childhood Abnormal gait Knees locked Difficulty climbing stairs and running Age <15 16 – 18 19 – 27 28 - 34 1https://www.youtube.com/watch?v=JLqHi
Slide 16: LGMD Patients Manual Muscle Strength Progressively Declines
LGMD Patients Manual Muscle Strength Progressively Declines Treatment with Deflazacort was for 6 months in each arm. Single site, placebo controlled, cross over design (n=25) Manual muscle strength assessed bilaterally by the modified Medical Research Council Scales (MRC) CIDD (Clinical Investigatio
Slide 17: Resolaris Phase 1b/2 Clinical Trials
Resolaris Phase 1b/2 Clinical Trials Trial Indication(s) Patients Highest Dose Design 002 Adult FSHD 3 dose cohorts (n=20 Total) 3.0 mg/kg Weekly (12 weeks) Placebo controlled, Double blinded; Interpatient Dose Escalation up to 12 weeks 003 Early onset FSHD Stage 1 (n=8) 3.0 mg/kg Weekly (6 weeks) O
Slide 18: Relatively Stable or Improved Muscle Function Observed
Relatively Stable or Improved Muscle Function Observed Change from baseline overall MMT scores at week 14 *Early onset FSHD (003) Trial represents interim data results (4 patients of a total of 8) **One patient in 004 Trial did not have an MMT measurement due to being wheelchair bound at baseline 50
Slide 19: Patients Reported Relatively Stable or Decreased Disease Burden
Patients Reported Relatively Stable or Decreased Disease Burden Change from baseline overall INQoL scores at week 14 *Early onset FSHD (003) Trial represents interim data results (4 patients of a total of 8) One patient in 003 Trial did not have an INQoL measurement taken at baseline 004 FSHD 3.0 mg
Slide 20: Robust Safety & Tolerability Dossier
No clinical symptoms observed with low-level anti-drug antibody assay signals and protocol discontinuations were primarily driven by transient infusion related reactions (IRR) Robust Safety & Tolerability Dossier 44 patients have received Resolaris for a total drug exposure of 149 patient months No
Slide 21: Resolaris Status and 2017 Development Goals
Second Half First Half Resolaris Status and 2017 Development Goals Clinical Results: Early Onset FSHD Patients (003) Clinical Trial: Kick off next trial post partnership* Regulatory: Advance interactions with regulatory agencies Biomarker/MOA: Introduce Mechanistic/PD Assay Established a favorable s
Slide 22: Resolaris: One Product, Multiple RMICs
Resolaris: One Product, Multiple RMICs Promise for severely afflicted myopathy patients FSHD: Average prevalence rates of FSHD are approximately 1/17,000. Applying this rate to the US population based on recent census data equals approximately 19,000. LGMD: 16,000 cases estimated in US population. 1
Slide 23: Stalaris: Lung Physiocrine Engineered to Treat Multiple Pulmonary Diseases
Stalaris Lung Physiocrine Engineered to Treat Multiple Pulmonary Diseases
Slide 24: Interstitial Lung Disease Opportunity
Adapted from: Thannickal VJ, et al. Ann Rev Med. 2004;55:395-417 (and) 2013 ATS Statement: Update of the International Multidisciplinary Classification of the Idiopathic Interstitial Pneumonias *IPF = Idiopathic Pulmonary Fibrosis Interstitial Lung Disease Opportunity Driven by a combination of immu
Slide 25: Stalaris Program: Opportunity for Lung Patients
Stalaris Program: Opportunity for Lung Patients Leverages Knowledge of Resokine Pathway in Lung Stalaris Program iMod domain Fc Domain of an antibody Engineered Physiocrine Stalaris iMod domain: Resokine splice variant relatively more expressed in lung than other tissues Fc Domain: increased exposur
Slide 26: Discovery of the iMod Domain Promotes Survival more than Steroids
Discovery of the iMod Domain Promotes Survival more than Steroids Rodent survival model of severe immune cell activity induced by administration of trinitrobenzene sulfonic acid (TNBS) --- Untreated --- TNBS + iMod 1mg/kg daily dosing --- TNBS + Budesonide 5mg/kg daily dosing --- TNBS + PBS iMod pro
Slide 27: iMod Domain in Lung
iMod Domain in Lung Splice Variant Express Data for iMod in Lung Splice variant for the iMod domain is relatively more expressed in lung than other tissues iMod Transcript Stalaris Program
Slide 28: Knockout of Resokine Pathway Increases T Cell Invasion
Knockout of Resokine Pathway Increases T Cell Invasion Post Disease Induction Rodent functional knockout inducing idiopathic pulmonary disease using Bleomycin T cell Invasion Impairment of lung function Stalaris Program * p < .05 Mouse Resokine vaccine Mouse iMod vaccine Naïve Sharm vaccine
Slide 29: Stalaris Tempers Activated T Cells at High Affinity
Stalaris Tempers Activated T Cells at High Affinity Stalaris Program Stalaris inhibits Th2 type cytokines from activated T cells Th2 cytokines play a role in promoting fibrosis in certain interstitial lung diseases Vehicle or Stalaris IL – 13 (pg/ml) Stalaris 10 pM to 10 nM Th2 Cytokine (pg/ml) ***
Slide 30: Stalaris Outperforms Current Treatments
Stalaris Program Stalaris Outperforms Current Treatments Established Rodent Model for Idiopathic Pulmonary Fibrosis (IPF) *The Ashcroft scale for the evaluation of bleomycin-induced lung fibrosis is the analysis of stained histological samples by visual assessment Improvement in Ashcroft Score (%)*
Slide 31: Stalaris: Status and 2017 Development Goals
Second Half First Half Biomarker/MOA: Introduce mechanistic/PD assay GMP Manufacturing: Complete clinical trial supply IND Enabling: Initiate preclinical safety studies Activity in industry proven model of IPF (approved drugs Pirfenidone & Nintedanib) GMP manufacturing kicked off Rat/non-human prima
Slide 32: Building a New Class of Therapeutics
Building a New Class of Therapeutics Foundation for the Future
Slide 33: LIFE Leaders
LIFE Leaders Foundation for the Future Andrea Cubitt, Ph.D. VP, Product Protection John Blake, CPA VP, Finance Sanjay Shukla, M.D. Chief Medical Officer Holly D. Chrzanowski VP, Enterprise Talent and Organization Ashraf Amanullah, Ph.D. VP, Manufacturing John Mendlein, Ph.D. Chief Executive Officer
Slide 34: LIFE Numbers
Discovered Physiocrines tRNA synthetase genes LIFE Numbers Foundation for the Future 3 Programs 300 Physiocrines 4,000,000,000 Years 23 genes 1 LIFE
Slide 35: LIFE Partnering Strategy
LIFE Partnering Strategy “Partner One or More Programs to Accelerate Clinical Development” Lower cost of capital to strategically advance pipeline Accelerate our programs for greater value to stakeholders Leverage complementary resources and capabilities of partner organizations 3rd Physiocrine-base
Slide 36: LIFE 2017 Goals and Financial Guidance
LIFE 2017 Goals and Financial Guidance *Estimated cash, cash equivalents, and investments provided pending completion of year-end financial close and external audit **Operational cash burn only, excludes cash from financings 2017 Goals Partner One or More Programs Advance Pipeline with Two Molecules
Slide 37: Thank you!
Thank you!