Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — JPMorgan Healthcare Conference 2016
30 slides from Agios Pharmaceuticals's conference presentation, Jan 11, 2016
Slide 1: Agios in 2016
Exhibit 99.1 Agios in 2016 JPMorgan Healthcare Conference January 11, 2016 David Schenkein, M.D. Chief Executive Officer
Slide 2: Cautionary Note Regarding Forward-Looking Statements
Cautionary Note Regarding Forward-Looking Statements This presentation and various remarks we make during this presentation contain forward-looking statements of Agios Pharmaceuticals, Inc. within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements in
Slide 3: Driven By a Clear Vision and Values
We Are Driven By a Clear Vision and Values RARE GENETIC DISORDERS OF METABOLISM CANCER METABOLISM DYSREGULATED METABOLISM VISION Agios is passionately committed to the fundamental transformation of patients’ lives through scientific leadership in the field of cancer metabolism and rare genetic disor
Slide 4: Building a Great Sustainable Biopharmaceutical Company
Building a Great Sustainable Biopharmaceutical Company Building Late-stage Development & Commercial Capabilities Continuing to Expand Research Platform 5th Agios Agios IDH Celgene AG-221 AG-348 medicine 2016 Key Labs Discovery Partnership /AG-120 Proof of entering Priorities Open Proof of Mechanism
Slide 5: Our 2016 Key Priorities: Maturing into a Late-stage Company
Our 2016 Key Priorities: Maturing into a Late-stage Company Rapid and broad late stage clinical development for IDHm inhibitors Demonstrate clinical activity of PKR activators in patients Advance research and initiate preclinical development of next wave research program
Slide 6: Novel First-in-Class Clinical Portfolio
Novel First-in-Class Clinical Portfolio Early Stage Late Stage Candidate Indication Primary Commercial Rights Clinical Development Clinical Development R/R AML Phase 3 R/R AML Phase 1 Dose Escalation Expansion 5th Cohort AG-221 Frontline AML (Fit) Phase 1b Combinations (IDH2m inhibitor) Frontline AM
Slide 7: Key 2016 Milestone Announcements
Today’s Key 2016 Milestone Announcements IDHm Inhibitors Complete enrollment in AG-221 and AG-120 expansion arms in 2H Initiate Phase 3 study of AG-120 in frontline AML in 2H Initiate MDS expansion arm for AG-221 Initiate randomized Phase 2 study of AG-120 in cholangiocarcinoma in 2H PKR Activators
Slide 8: Cancer Metabolism: IDH
Cancer Metabolism: IDH Using a pill once a day to repair a cancer cell agios
Slide 9: Repairing an IDH Mutant Cancer Cell
Repairing an IDH Mutant Cancer Cell DIFFERENTIATION RESTORED BLO Inhibitor IDH Epigenetic “Re-Wiring” Unchecked Cell Proliferation Isocitrate mIDH 2HG CANCER Differentiation aKG HSC Progenitor Progenitor IDHm Inhibitor Leukemia Blast Neutrophil Day 0 Day 15 Day 28 AG-120, EORTC, 2014
Slide 10: What's Possible for IDHm Patients
What’s Possible for IDHm Patients A Roadmap for Speed and Breadth Goal Next Now All IDHm patients screened and treated with an IDHm inhibitor for the entire course of their disease Solid tumors Frontline AML Combination trials Maintenance MDS Other hematologic malignancies Relapsed/ Refractory AML
Slide 11: Three IDHm Inhibitors Provide Maximum Opportunity
Three IDHm Inhibitors Provide Maximum Opportunity AG-221 Potent, selective, reversible inhibitor of mutant isocitrate dehydrogenase-2 (IDH2) In Phase 3 clinical development Oral, once daily dosing, 100mg AG -120 Potent, selective, reversible inhibitor of mutant isocitrate dehydrogenase-1 (IDH1) In P
Slide 12: IDH Mutations Occur in Multiple Cancers
IDH Mutations Occur in Multiple Cancers AML MDS IHCC Chondro Other solid tumors Glioma ~40K New Patients/Year; Mutation Occurs Early and Easily Detected with Genomic Testing
Slide 13: AG-221 and AG-120 Represent a New Treatment Paradigm in AML
AG-221 and AG-120 Represent a New Treatment Paradigm in AML AG-221 AG-120 Median response duration: 5.6 months Median response duration: 6.9 months CR PR CRI Ongoing Progression/death crp mcr first response bone marrow transplant treatment suration (months) death or Progression transplant First-in-C
Slide 14: Phase 1 Programs Rapidly Defined Single-Agent Profile for IDHm Inhibitors
Phase 1 Programs Rapidly Defined Single-Agent Profile for IDHm Inhibitors in R/R AML Phase 1 expansion cohorts designed to demonstrate compelling clinical benefit with registration quality data AG-221 Expansion Phase 2 Expansion Phase 1 Ongoing Dose Escalation Four 25-patient Arms Completed R/R AML
Slide 15: Targeting Multiple Lines of Treatment in IDHm AML
Targeting Multiple Lines of Treatment in IDHm AML and Other Hematologic Malignancies Relapsed AML Newly Diagnosed (Untreated) AML MDS / Other Heme Malig. 2nd+ Relapse Intensive Non-Intensive Frontline to R/R Phase 1 AG-221 Expansion Phase 1 AG-120 Expansion Phase 3 IDHENTIFY AG-221 vs SOC Phase 1 In
Slide 16: IDH Mutations Occur in Multiple Cancers
IDH Mutations Occur in Multiple Cancers Glioma IHCC Chondro Aml mds other solid tumors ~40K New Patients/Year; Mutation Occurs Early and Easily Detected with Genomic Testing 16
Slide 17: Encouraging AG-120 Phase 1 Data in Solid Tumors
Encouraging AG-120 Phase 1 Data in Solid Tumors Solid Tumors Glioma Other chondrosarcoma cholangiocarcinoma treatment duration (weeks) pr sd pd unk/na ongoing progression/death AG-120 well tolerated (no MTD) and showed signs of clinical activity Reductions in tumor volume observed in some glioma pat
Slide 18: Clinical Development Path in IDH1m Solid Tumors Will Be Data Driven
Clinical Development Path in IDH1m Solid Tumors Will Be Data Driven AG-120 IDH1m inhibition; low CNS penetration Dose-Escalation Expansion (Completed) (Ongoing) Randomized IHCC Phase 2 (2H’16) IHCC Chondro sarcoma Glioma Other IDHm Solid Tumors AG-881 Pan-IDHm inhibition; high CNS penetration AG-881
Slide 19: Rare Genetic Metabolic Disorders: PKR
Rare Genetic Metabolic Disorders: PKR Transforming a metabolic disorder with a small molecule agios
Slide 20: PKR Activation Has Broad Potential in Red Cell Disorders
Pyruvate Kinase-R (PKR) Activation Has Broad Potential in Red Cell Disorders Normal Red Cell PEP wtPKR Pyruvate Cellular demand: ATP production meets demand Pyruvate Kinase Deficiency PEP mPKR Pyruvate Cellular demand: Inadequate production: ATP deficiency Other Hemolytic Anemias PEP wtPKR Pyruvate
Slide 21: PK Deficiency: What We Know Today
PK Deficiency: What We Know Today Disease Overview Rare genetic disease often presenting at birth as neonatal Description jaundice ~2400 diagnosed in U.S. and EU5* Caused by mutations in PK-LR Etiology gene coding for Erythrocyte Pyruvate Kinase Clinical Lifelong hemolytic anemia and Presentation as
Slide 22: PKR Activation Represents Therapeutic Approach to Treating PK Deficiency
PKR Activation Represents Therapeutic Approach to Treating PK Deficiency 2,3-DPG pyruvate Glucose atp AG-348 AG-519 Patient A (R510Q/G511R) Pkr activity (relative to dmso) 2-3 dpg (relative to dmso) atp (relative to dmso) 24 hrs Wt vs pkd atp levels rbcs treated with ag-348 24hrs wt control pkd pati
Slide 23: AG-348 Global Phase 2 DRIVE PK Study
AG-348 Global Phase 2 DRIVE PK Study Open and Enrolling Transfusion-independent PK-deficient adults n=25 in each arm Screening Randomization Stratified by PK-R genotype AG-348 300 mg q12hr Optional 3rd Arm AG-348 50 mg q12hr 6-month dosing PK period PD Primary endpoints Safety and tolerability Secon
Slide 24: AG-519 Healthy Volunteer Study Open and Enrolling
AG-519 Healthy Volunteer Study Open and Enrolling AG-519 Potent, highly selective and orally bioavailable PKR activator Differentiated chemical structure versus AG-348 No activity against the aromatase enzyme AG-519 has similar activity in vitro, in vivo and ex vivo (patient samples) relative to AG-
Slide 25: AG-519 Provides Optionality for Clinical Development
AG-519 Provides Optionality for Clinical Development AG-348 Healthy Volunteer Studies (Completed) AG-348 DRIVE PK PK Deficiency (Ongoing) AG-519 Healthy Volunteer Studies (Q1’16) Efficacy Safety PK PD Pivotal Trials: Adults Adolescents Children Beta-thalassemia Studies (Planned) Clinical Data From B
Slide 26: Research
Research Initiating the development of a new research program
Slide 27: Novel First-in-Class Research Portfolio
Novel First-in-Class Research Portfolio Target Validation Compound Optimization Target C Two Cancer Metabolism Multiple Other Oncology Targets Wave Rare Genetic Multiple RGD Targets Metabolic Disorders Cancer Metabolism Multiple Oncology Three Targets Wave Rare Genetic Multiple RGD Metabolic Disorde
Slide 28: Prevalent Deletions of MTAP Create Sensitivity to a Novel Pathway
Prevalent Deletions of MTAP Create Sensitivity to a Novel Pathway With Multiple Therapeutic Targets Deletion of metabolic gene adjacent to tumor suppressor p16/p14 chr9p21 MTAP p16/p14 genetic selection markers ~75,000 new patients/year in U.S. with MTAP deletion across many indications % mtap homoz
Slide 29: Prevalent Deletions of MTAP Create Sensitivity to a Novel Pathway
Prevalent Deletions of MTAP Create Sensitivity to a Novel Pathway With Multiple Therapeutic Targets Upstream metabolite MTA accumulates when MTAP is deleted MTA (MTAP substrate) Vulnerable pathway with multiple Therapeutic targets Target 1 Target 2 Target 3 Druggable by Agios inhibitors with precisi
Slide 30: Key Takeaways
Key Takeaways Focus on Strategic Priorities and Execution: Continue rapid, broad late-stage clinical development for our IDHm inhibitors Demonstrate clinical activity of our wholly owned, PKR activators in patients Advance research and initiate preclinical development of a new research program Build
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