Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — JPMorgan Healthcare Conference 2017
33 slides from Agios Pharmaceuticals's conference presentation, Jan 9, 2017
Slide 1: Agios: Delivering Our First Medicines to Patients
Agios: Delivering Our First Medicines to Patients JPMorgan Healthcare Conference David Schenkein, M.D. Chief Executive Officer January 9, 2017 Exhibit 99.1
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation and various remarks we make during this presentation contain forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Such forward-looking statements include those regarding the Agios’ plans, strategies and ex
Slide 3: We Are Driven By a Clear Vision and Values
Agios is passionately committed to applying our scientific leadership in the field of cellular metabolism to transform the lives of patients with cancer and rare genetic diseases. We Are Driven By a Clear Vision and Values CANCER METABOLISM METABOLIC IMMUNO-ONCOLOGY RARE GENETIC DISEASES DYSREGULATE
Slide 4: January 2009
January 2009 5
Slide 5: Science Team Discovery 2009
Science Team Discovery 2009 6
Slide 6: Delivering Our First Medicines to Patients
January 9, 2017 Delivering Our First Medicines to Patients Science Team Discovery 7
Slide 7: 2017 Key Priorities & Expected Milestones
PKR Continue to demonstrate leadership in PK deficiency Prepare for 1H 2018 pivotal trial initiation for wholly owned AG-348 in PK deficiency IDH Secure approval and co-commercialize enasidenib for R/R AML in the U.S. Submit NDA for wholly owned AG-120 in R/R AML by YE 2017 Initiate Phase 3 combinin
Slide 8: Commercial Stage Biopharmaceutical Company
Delivering Our First Medicines to Patients Commercial Stage Biopharmaceutical Company Science Team Discovery 450+ employees 2 approved precision medicines in AML 2017 – 2018 4+ clinical-stage molecules 3+ pivotal trials (IDH, PKR) 100% committed to helping patients 3 areas of research expertise 9
Slide 9: IDH
IDH PKR RESEARCH 10
Slide 10: Repairing an IDH Mutant Cancer Cell
Repairing an IDH Mutant Cancer Cell IDHm Inhibitor Differentiation Mature Cell Normal Cell Death / HOMEOSTASIS Differentiation Progenitor Unchecked Cell Proliferation CANCER HSC Progenitor DIFFERENTIATION BLOCKED Me Me Me IDHm IDH Isocitrate aKG 2HG Epigenetic “Re-Wiring” Inhibitor 2HG Day 15 Day 28
Slide 11: Repairing an IDH Mutant Cancer Cell
Repairing an IDH Mutant Cancer Cell IDHm Inhibitor Differentiation Mature Cell Normal Cell Death / HOMEOSTASIS Differentiation Progenitor Unchecked Cell Proliferation CANCER HSC Progenitor DIFFERENTIATION BLOCKED Me Me Me IDHm IDH Isocitrate aKG 2HG Epigenetic “Re-Wiring” Inhibitor 2HG AG-120, ASH,
Slide 12: Our Vision for IDHm Inhibitors: A Roadmap for Speed and Breadth
Goal All IDHm patients screened and treated with an IDHm inhibitor for the entire course of their disease Our Vision for IDHm Inhibitors A Roadmap for Speed and Breadth Now Next
Slide 13: Our Vision for IDHm Inhibitors: Now
Now Next Goal Now Relapsed/Refractory AML Enasidenib NDA submission 2016 AG-120 NDA submission by YE 2017 Our Vision for IDHm Inhibitors A Roadmap for Speed and Breadth
Slide 14: Our Vision for IDHm Inhibitors: Next
Next Frontline AML AG-120 + Vidaza® Phase 3 Enasidenib / AG-120 + (7+3) with maintenance Relapsed/Refractory AML Novel-novel combination Phase 1 studies Solid Tumors AG-120 Phase 3 cholangiocarcinoma AG-120 and AG-881 Phase 1 glioma expansion Glioma development strategy Our Vision for IDHm Inhibitor
Slide 15: Incidence of AML Rising in U.S. and EU5 with Aging Population
Incidence of AML Rising in U.S. and EU5 with Aging Population Sources: American Cancer Society: Cancer Facts and Figures Visser et. Al. Incidence, survival and prevalence of myeloid malignancies in Europe. Eur J Cancer. 2012 Nov;48(17):3257-66 Epiphany Partners Epic Oncology Decision Resources Thous
Slide 16: Shifting the Treatment Paradigm for AML with Precision Medicine
AML Fit for Intensive Treatment (IC) Unfit for IC 7+3 HMA/LDAC Supportive care Clinical trial CR Non-CR Consolidation CTX 2nd induction Transplant if eligible CTX = Chemotherapy HMA = Hypomethylating Agent LDAC = Low dose Cytarabine Shifting the Treatment Paradigm for AML with Precision Medicine Lon
Slide 17: Advancing AG-120 into Frontline Setting
Randomization 1:1 Double Blinded AG-120 500mg (n=196) + Vidaza® Primary Endpoint OS Interim analyses for futility and superiority Advancing AG-120 into Frontline Setting Placebo (n=196) + Vidaza® Final OS Analyses Secondary Endpoints: CR, CR+CRh, EFS, ORR Trial on track for 1H 2017 initiation Global
Slide 18: Building World-Class Commercial Capabilities for IDH Launches
2017 2016 Building World-Class Commercial Capabilities for IDH Launches 2018 Co-commercialization with Celgene for enasidenib Established medical affairs and commercial leadership Chief Commercial Officer VP Market Access VP Sales VP Global Medical Affairs MSL deployment ongoing Field sales recruiti
Slide 19: Our Vision for IDHm Inhibitors: Next
Next Frontline AML AG-120 + Vidaza® Phase 3 Enasidenib / AG-120 + (7+3) with maintenance Relapsed/Refractory AML Novel-novel combination Phase 1 studies Solid Tumors AG-120 Phase 3 cholangiocarcinoma AG-120 and AG-881 Phase 1 glioma expansion Glioma development strategy Our Vision for IDHm Inhibitor
Slide 20: Treating Solid Tumors with an IDH1m Inhibitor
Other solid tumor types include colon, melanoma, lung, ovarian. Multiple sources, including market research and SEER. Estimates will continue to evolve with additional future data *excludes primary GBM Treating Solid Tumors with an IDH1m Inhibitor Glioma Intrahepatic Cholangiocarcinoma (IHCC) Chondr
Slide 21: Registration-Enabling Phase 3 Cholangiocarcinoma Study
2:1 Double Blind Randomization (n=186) AG-120 Arm 500mg Registration-Enabling Phase 3 Cholangiocarcinoma Study Placebo Arm Primary Endpoint: PFS Crossover at time of progression Secondary Endpoints: OS, ORR, safety, QoL Trial Initiated in December 2016 Global Phase 3 Previously Treated IDH1m Cholang
Slide 22: AG-120 Data Supports Clinical Development in Glioma
Phase 1 Dose-Escalation and Expansion (Completed) Encouraging Data with AG-120 Supports Clinical Development of IDH1m Inhibitor in Glioma AG-120 IDH1m Inhibition AG-881 High CNS Penetration, Pan-IDHm Inhibition Phase 1 Dose-Escalation (Ongoing) Potential Expansion 22 AG-120 Data Presented at SNO 201
Slide 23: PKR
IDH PKR RESEARCH 23
Slide 24: PK Deficiency Is a Rare Genetic Disease that Affects Red Blood Cells
PK Deficiency Is a Rare Genetic Disease that Affects Red Blood Cells Rare genetic disease of erythrocyte pyruvate kinase PK deficiency often presents at birth with jaundice and can cause lifelong hemolytic anemia and associated morbidities Estimated prevalence ranges from ~1:20K to ~1:485K1–4 PKR re
Slide 25: PK Deficiency Is a Lifelong Disease with Only Supportive Treatments
INFANT CHILD ADULT Supportive Treatments Complications Phototherapy Blood transfusions Removal of spleen Removal of gall bladder Blood transfusions Infection risk à lifelong prophylactic antibiotics Thrombosis risk Blood transfusions Iron overload à iron chelation therapy Disease Burden PK Deficienc
Slide 26: Quality of Life Impact Weighs Heavily for Both Patients & Families
Quality of Life Impact Weighs Heavily for Both Patients & Families “If I say I’m tired, people think I need more sleep... I have fatigue so intense that it wakes me up at night. How do you get someone to understand that?!” “Think of a day when you are sick with a cold. That is me on my best day.”* *
Slide 27: PK Deficiency Strategy
PK Deficiency Strategy Increase Understanding of Disease Burden Drive the Science Develop & Commercialize First Treatment Addressing Underlying Cause of Disease Demonstrate Commitment to PK Deficiency Community & Patients Agios Leadership in PK Deficiency
Slide 28: Compelling Proof-of-Concept for AG-348
Compelling Proof-of-Concept for AG-348, the First Disease Modifying Therapy for PK Deficiency DRIVE PK Learnings Robust hemoglobin increases in 15 / 32 patients; 15 / 26 patients with 1 or more missense mutation Responses are rapid and sustained; median time to response of 1.4 weeks Majority of resp
Slide 29: Key Considerations for AG-348 Pivotal Trial Design
Key Considerations for AG-348 Pivotal Trial Design Design Element Considerations Rationale Patient Population Transfusion dependent adult (TD) Non-Transfusion dependent adult (NTD) Goal to treat all adult patients Size ~100 patients Rare disease Dose Dose titration up to optimal hemoglobin response
Slide 30: Research
IDH PKR RESEARCH 30
Slide 31: Agios' Scientific Research Platform
Agios’ Scientific Research Platform CANCER METABOLISM RARE GENETIC DISEASES METABOLIC IMMUNO-ONCOLOGY DYSREGULATED METABOLISM Inhibit key enzymes in cancer cell specific metabolic pathways to disrupt tumor cell proliferation and survival Restore defective metabolic pathways in disease cells that cau
Slide 32: Development Candidate for MTAP Pathway Selected
Development Candidate for MTAP Pathway Selected IND Expected by Year-End Deletion of metabolic gene adjacent to tumor suppressor p16/p14 p16/p14 tumor suppressor locus deleted in 15% of all cancers Metabolic gene, MTAP, is adjacent to p16/p14 & typically co-deleted MTAP p16/p14 MTAP-deleted tumors c
Slide 33: Commercial Stage Biopharmaceutical Company
Delivering Our First Medicines to Patients Commercial Stage Biopharmaceutical Company Science Team Discovery 450+ employees 2 approved precision medicines in AML 2017 – 2018 4+ clinical-stage molecules 3+ pivotal trials (IDH, PKR) 100% committed to helping patients 3 areas of research expertise 33
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