Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — 38th Annual J.P. Morgan Healthcare Conference (January 2020)
26 slides from Agios Pharmaceuticals's conference presentation, Jan 13, 2020
Slide 1: Agios 2025 Vision
AGIOS 2025 VISION 38th Annual J.P. Morgan Healthcare Conference January 13, 2020 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 Agios’ plans, strategies and expect
Slide 3: Our Strategy is Clear
Our Strategy is Clear For more than a decade, our mission has been to create differentiated, small molecule medicines for patients in three focus areas – malignant hematology, solid tumors and rare genetic diseases – based on our unique expertise in cellular metabolism and adjacent areas of biology
Slide 4: Our People and Culture Fuel Incredible Productivity
Our People and Culture Fuel Incredible Productivity, Strategic Focus and Continuity from Early Research to Market “Other Side Of Possible” Culture Patient-centric Approach Creative Clinical Development Productive Research Engine 4 ADDITIONAL MOLECULES IN CLINICAL DEVELOPMENT 2 MEDICINES APPROVED + ~
Slide 5: Agios 2025 Vision: Focused Innovation. Ambitious Development.
Agios 2025 Vision: Focused Innovation. Ambitious Development. Transformative Treatments for Patients Across Three Focus Areas. NOW CASH FLOW POSITIVE 2025 FINANCIAL $105-115M EXPECTED U.S. TIBSOVO® 2020 REVENUE 4 MEDICINES COMMERCIAL 2 MEDICINES 8+ INDICATIONS LABEL EXPANSION 2 INDICATIONS 6+ MOLECU
Slide 6: Multiple Potential Near- and Long-term Value Drivers Across All Focus Areas
Multiple Potential Near- and Long-term Value Drivers Across All Focus Areas 2020 2021 2022 2023 2024 2025 Approval Make go/no-go decision TIBSOVO® R/R AML EU TIBSOVO® IC-ineligible 1L AML (AGILE Phase 3) TIBSOVO® IC-eligible 1L AML (HOVON Phase 3) TIBSOVO® R/R MDS (Phase 1 expansion) AG-636 Advanced
Slide 7: Highly Productive Research Engine with Optionality Across Focus Areas
Highly Productive Research Engine with Optionality Across Focus Areas Program Target Discovery Target Validation Drug Discovery Drug Candidate Solid Tumor Non-Metabolic Target Metabolic Target Metabolic and Non-Metabolic Targets Bristol-Myers Squibb Collaboration Malignant Hematology Rare Genetic Di
Slide 8: Creating Medicines in Three Focus Areas
CREATING MEDICINES IN THREE FOCUS AREAS
Slide 9: Creating Medicines in Three Focus Areas
CREATING MEDICINES IN THREE FOCUS AREAS
Slide 10: Significant Growth Potential in Malignant Hematology
Significant Growth Potential in Malignant Hematology IDH1 Mutant Acute Myeloid Leukemia (AML) IDH1 Mutant Myelodysplastic Syndrome (MDS) Mantle Cell and Diffuse Large B Cell Lymphoma TIBSOVO® R/R AML U.S. Approval; MAA Under Review 1L Monotherapy U.S. Approval 1L HMA Combo Phase 3 1L 7+3 Combo Phase
Slide 11: Successful TIBSOVO Launch in R/R and Frontline AML
Successful TIBSOVO® Launch in R/R and Frontline AML Result of Focused Commercial Effort $105 – 115M U.S. Net Sales Guidance for 2020 >90% Physicians Testing for IDH1/IDH2 mutations ~515 Unique Prescribers as of Q4 2019 >1,000 Patients Treated Since Launch Core commercial capabilities support two add
Slide 12: Advancing Toward Largest Opportunity for mIDH1 AML
50% will relapse or be refractory to treatment AGILE Phase 3 Aza Combo Enrolling ~50% Eligible for intensive therapy ~25% Currently untreated Phase 1 combo data: 61% CR 70% CR+CRh 82% 12-month OS ~25% Currently untreated Phase 1 combo data: 79% CR in de novo AML 53% CR in secondary AML 79% 12-month
Slide 13: Creating Medicines in Three Focus Areas
CREATING MEDICINES IN THREE FOCUS AREAS
Slide 14: Four Distinct Solid Tumor Opportunities Across Three Clinical Molecules
Four Distinct Solid Tumor Opportunities Across Three Clinical Molecules TIBSOVO® Vorasidenib IDH1 Mutant Cholangiocarcinoma IDH Mutant Low Grade Glioma MTAP-Deleted Non-Small Cell Lung Cancer MTAP-Deleted Pancreatic Cancer ~2-3K PATIENTS IN U.S. & EU ~9K PATIENTS IN U.S. & EU ~9K PATIENTS IN U.S. ~1
Slide 15: Positive ClarIDHy Results
POSITIVE CLARIDHY RESULTS FEW TREATMENT OPTIONS Sources: CDC National Program of Cancer Registries (NPCR); Epiphany Partners Epic Oncology; Decision Resources; Market Research; Borger DR et al. Oncologist 2012;17:72-9.; Kipp BR et al. Hum Pathol 2012;43:1552-8.; Goyal L et al. Oncologist 2015;20:101
Slide 16: Global Phase 3 INDIGO Study of Vorasidenib in IDH Mutant Low-Grade Glioma
Global Phase 3 INDIGO Study of Vorasidenib in IDH Mutant Low-Grade Glioma Open and Enrolling 1:1 double-blind randomization (n=366) *centrally-confirmed progressive disease will permit unblinding and crossover Vorasidenib 50 mg QD orally Continuous 28-day cycles (n=183) Matched placebo* (n=183) Stra
Slide 17: AG-270, MAT2A Inhibitor, Preclinical Data Supports Combination with Taxanes
AG-270, MAT2A Inhibitor, Preclinical Data Supports Combination with Taxanes; Two Phase 1 Combination Arms Enrolling Patients 4 of 8 animals were tumor free at last dose and remained tumor free until the arm was terminated on Day 141 PHASE 1 COMBINATION ARMS INITIATED AG-270 + docetaxel in MTAP-delet
Slide 18: Creating Medicines in Three Focus Areas
CREATING MEDICINES IN THREE FOCUS AREAS
Slide 19: PKR Activation Represents Unique Mechanism of Action
PKR Activation Represents Unique Mechanism of Action with Potential to Address Broad Range of Hemolytic Anemias Normal Red Cell PEP Pyruvate wtPKR Cellular demand: ATP production meets demand Pyruvate Kinase Deficiency PEP Pyruvate mPKR Proof-of-concept achieved Adult PK deficiency approval expected
Slide 20: Mitapivat has Potential to be First Disease-modifying Therapy for PK Deficiency
Mitapivat has Potential to be First Disease-modifying Therapy for Patients with PK Deficiency Improvements in Hemoglobin and Other Hemolysis Markers Maintained for More Than 3 Years in Responding Patients from DRIVE PK Extension Chronic daily dosing with mitapivat for a median of 3 years and up to 4
Slide 21: Clinical Proof-of-concept for Mitapivat Established in Non-transfusion-dependent Thalassemia
Clinical Proof-of-concept for Mitapivat Established in Non-transfusion-dependent Thalassemia In responding patients, the mean hemoglobin increase from baseline was 1.76 g/dL (range, 0.9 – 3.3 g/dL) Majority of adverse events were Grade 1 or 2 and consistent with previously published Phase 2 data for
Slide 22: Therapeutic Hypothesis for Wildtype PKR Activation in Sickle Cell Disease
Therapeutic Hypothesis for Wildtype PKR Activation in Sickle Cell Disease: 2,3-DPG and ATP Modulation Improves Anemia and Reduces Sickling ATP-dependent regulation of ion concentration and cellular shape RBC dehydration3 ↑ HbS concentration and polymerization3 ↓ Deformability4 Water and ion loss3 ↓
Slide 23: PKR Activation Has Potential Broad Utility Across Hemolytic Anemias
PKR Activation Has Potential Broad Utility Across Hemolytic Anemias Pyruvate Kinase Deficiency ~3-8K PATIENTS IN U.S. & EU β- and α-Thalassemia ~18-23K PATIENTS IN U.S. & EU Sickle Cell Disease ~120-135K PATIENTS IN U.S. & EU NTD β- and α-Thalassemia Phase 2 Thalassemia Pivotal Plan by YE NTD Adult
Slide 24: Creating Medicines in Three Focus Areas
CREATING MEDICINES IN THREE FOCUS AREAS
Slide 25: Agios 2020 Key Milestones
HEMATOLOGIC MALIGNANCIES SOLID TUMORS RARE GENETIC DISEASES RESEARCH Agios 2020 Key Milestones Achieve full-year U.S. revenue for TIBSOVO® $105-115M Receive CHMP opinion for TIBSOVO® in mIDH1 relapsed/refractory AML Complete enrollment in AGILE Phase 3 trial of TIBSOVO® + azacitidine in frontline mI
Slide 26: Agios 2025 Vision: Focused Innovation. Ambitious Development.
AGIOS 2025 VISION: Focused Innovation. Ambitious Development. Transformative Treatments for Patients Across Three Focus Areas. 4 MEDICINES 8+ INDICATIONS 6+ MOLECULES IN THE CLINIC $ CASH FLOW POSITIVE
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