Agios Pharmaceuticals, Inc. · NASDAQ:AGIO

Agios Pharmaceuticals — Q2 2020 Earnings Presentation

19 slides from Agios Pharmaceuticals's earnings presentation, Jul 30, 2020

  1. Slide 1: Second Quarter 2020 Financial Results

    Second Quarter 2020 Financial Results July 30, 2020 1

  2. Slide 2: Agios Conference Call Participants

    Agios Conference Call Participants TOPIC PARTICIPANT Introductions Holly Manning, Director of Investor Relations Business Update Jackie Fouse, Ph.D., Chief Executive Officer Clinical Development Update Chris Bowden, M.D., Chief Medical Officer Darrin Miles, Senior Vice President, U.S. TIBSOVO® Perfo

  3. Slide 3: 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

  4. Slide 4: Agios 2025 Vision: Focused Innovation. Ambitious Development.

    AGIOS 2025 VISION: Focused Innovation. Ambitious Development. Transformative Treatments for Patients Across Three Focus Areas. 4 8+ 6+ $ MOLECULES CASH FLOW MEDICINES INDICATIONS IN THE CLINIC POSITIVE 4

  5. Slide 5: Q2 2020 Highlights

    Q2 2020 Highlights Rare Genetic Hematologic Solid Tumors Diseases Malignancies • Established clinical proof-of- • TIBSOVO® net sales of $27.6 • Published data from ClarIDHy study concept for mitapivat in sickle million, an increase of 22% from of TIBSOVO® in cholangio in The cell disease Q1 2020 Lan

  6. Slide 6: Clinical Development Updates

    Clinical Development Updates Chris Bowden, M.D., Chief Medical Officer 6

  7. Slide 7: Anticipated Upcoming Milestones

    • Mitapivat ACTIVATE and ACTIVATE-T topline data in PK deficiency RARE GENETIC DISEASES between YE 2020 and mid-2021 • Finalize pivotal development plan for mitapivat in thalassemia by the end of 2020; initiate a pivotal program in 2021 Anticipated • Finalize pivotal development plan for mitapivat i

  8. Slide 8: PKR Activation Represents Unique Mechanism of Action

    PKR Activation Represents Unique Mechanism of Action with Potential to Address Broad Range of Hemolytic Anemias GLYCOLYSIS Glucose Mutant PKR Wildtype PKR Normal Red Cell Pyruvate Kinase Deficiency Other Hemolytic Anemias FBP PEP PEP PEP wtPKR mPKR wtPKR 1,3-DPG 2,3-DPG Pyruvate Pyruvate Pyruvate 3-

  9. Slide 9: Mitapivat Potential 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 COMPLICATIONS & COMORBIDITIES HIGH RISK SUPPORTIVE REGARDLESS OF OF IRO

  10. Slide 10: Interim Phase 2 Results in Thalassemia

    Interim Phase 2 Results in Thalassemia: Activation of wPKR by Mitapivat Improved Hb and Associated Markers of Hemolysis and Erythropoiesis Treatment with mitapivat induced Hb increase of ≥1.0 g/dL in 12 of 13 evaluable patients, including 100% α-thalassemia patients; 7 of 8 evaluable patients achiev

  11. Slide 11: Clinical Proof-of-concept for Mitapivat Established in Sickle Cell Disease

    Clinical Proof-of-concept for Mitapivat Established in Sickle Cell Disease 7 of 8 (88%) efficacy evaluable patients experienced a Hb increase, and 5 of 8 (63%) patients achieved a Hb increase of ≥1.0 g/dL from baseline (range 1.0-2.7 g/dL) at doses of 50 mg BID or lower. Treatment with mitapivat was

  12. Slide 12: PKR Activation Has Potential Broad Utility Across Hemolytic Anemias

    PKR Activation Has Potential Broad Utility Across Hemolytic Anemias ~18- ~120- ~3-8K PATIENTS IN 23K 135K U.S. & EU PATIENTS IN PATIENTS IN U.S. & EU U.S. & EU Pyruvate Kinase Deficiency β- and α-Thalassemia Sickle Cell Disease Phase 3 enrollment complete; NTD β- and α- Phase 2 enrollment NIH CRADA;

  13. Slide 13: Significant Growth Potential in Malignant Hematology

    Significant Growth Potential in Malignant Hematology ~4K <1K PATIENTS IN PATIENTS IN U.S. & EU U.S. IDH1 Mutant Acute Myeloid IDH1 Mutant Myelodysplastic Leukemia (AML) Syndrome (MDS) TIBSOVO® TIBSOVO® U.S. Approval; R/R AML R/R MDS Phase 1 Expansion MAA Under Review 1L Monotherapy U.S. Approval 1L

  14. Slide 14: Four Distinct Solid Tumor Opportunities Across Three Clinical Molecules

    Four Distinct Solid Tumor Opportunities Across Three Clinical Molecules ~2-3K ~9K ~9K ~10K PATIENTS IN PATIENTS IN PATIENTS PATIENTS U.S. & EU U.S. & EU IN U.S. IN U.S. IDH1 Mutant IDH Mutant MTAP-Deleted Non- MTAP-Deleted Cholangiocarcinoma Low Grade Glioma Small Cell Lung Cancer Pancreatic Cancer

  15. Slide 15: TIBSOVO Performance Update

    TIBSOVO® Performance Update Darrin Miles, Senior Vice President, U.S. Commercial & Global Marketing 15

  16. Slide 16: Q2 Growth Driven by Increased Demand in R/R and Frontline AML

    Q2 Growth Driven by Increased Demand in Both R/R and Frontline AML Segments and Expanding Customer Base TIBSOVO® Revenue (in millions) $28 22% Growth In Product Revenue Quarter-over-Quarter $23 $20 $105 – 115M $17 U.S. Net Sales Guidance for 2020 $14 $9 15% Increase In Unique Prescribers Quarter-ove

  17. Slide 17: Second Quarter 2020 Financial Results

    Second Quarter 2020 Financial Results Andrew Hirsch, Chief Financial Officer and Head of Corporate Development 17

  18. Slide 18: Second Quarter 2020 Financial Results

    Second Quarter 2020 Financial Results Three Months Three Months Statement of Operations Ended 6/30/20 Ended 6/30/19 Total Revenue $37.3M $26.2M Collaboration Revenue 6.4M 9.8M TIBSOVO® Net Sales 27.6M 13.7M Royalty Revenue 3.3M 2.7M Cost of Sales 0.7M 0.3M Research & Development 90.9M 107.4M Expense

  19. Slide 19: Q&A

    Q&A 19

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