Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Investor Presentation (January 2023)
23 slides from Agios Pharmaceuticals's investor presentation, Jan 9, 2023
Slide 1: Fueled by Connections to Transform Rare Diseases
Fueled by Connections to Transform Rare Diseases Brian Goff, Chief Executive Officer January 11, 2023 Exhibit 99.2
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 potential benefits of PYRUKYND®
Slide 3: Track record of success in discovering, developing and commercializing therapies
Track record of success in discovering, developing and commercializing therapies Unmatched expertise in cellular metabolism The Leader in Pyruvate Kinase (PK) Activation DEVELOPED APPROVED THERAPIES IN ONCOLOGY 2017 & 2018 FIRST PYRUKYND® APPROVAL ADULTS WITH PYRUVATE KINASE (PK) DEFICIENCY 2022 RAR
Slide 4: Leader in pyruvate kinase (PK) activation poised for significant growth
Leader in pyruvate kinase (PK) activation poised for significant growth Compelling and consistent data across connected diseases Robust clinical data set supports potential of PK activation to transform patient function, quality of life, and long-term outcomes Meaningful commercial opportunities on
Slide 5: Focused on expanding from PK deficiency to other diseases
Pediatric PK Deficiency No approved therapy for pediatric PK deficiency patients Our goal: Deliver the first approved therapy for pediatric PK deficiency Thalassemia No approved therapy for ~60% of thalassemia patients Our goal: Deliver the first therapy approved for all thalassemia subtypes Sickle
Slide 6: PK activation franchise positioned for meaningful expansion
ADDITIONAL OPPORTUNITIES FOR THE FRANCHISE WITH NOVEL PK ACTIVATOR AG-946 PK deficiency Approved for adults in the U.S. and EU Thalassemia Potential U.S. approval in 2025 Sickle cell disease Potential U.S. approval in 2026 PK activation franchise positioned for meaningful expansion, with near-term o
Slide 7: Building a diverse pipeline leveraging our expertise in cellular metabolism
RESEARCH EARLY-STAGE CLINICAL DEVELOPMENT LATE-STAGE CLINICAL DEVELOPMENT REGULATORY SUBMISSION APPROVAL Pyruvate Kinase Deficiency α- and β-Thalassemia ACTIVATE Kids ACTIVATE KidsT ENERGIZE ENERGIZE-T Sickle Cell Disease* Myelodysplastic Syndrome (MDS) Healthy Volunteers / Sickle Cell Disease Pheny
Slide 8: Compelling and consistent data across connected diseases
Compelling and consistent data across connected diseases PK activation may address a range of hemolytic and acquired anemias underpinned by shared pathophysiology
Slide 9: Energy imbalance in red blood cells can lead to severe hematological disease
Energy imbalance in red blood cells can lead to severe hematological disease Insufficient ATP production Leads to red blood cell metabolic stress, anemia, and reduced patient quality of life Energy supply Energy demand Healthy Red Blood Cell ATP production meets demand PK Deficiency Other Anemias Th
Slide 10: Disease areas share common pathophysiology and severely impact quality of life
Chronic Hemolysis Destruction of red blood cells Ineffective Erythropoiesis Decreased output of red blood cells Disease areas share common pathophysiology and severely impact quality of life ANEMIA TRANSFUSION BURDEN IRON OVERLOAD REDUCED BONE MINERAL DENSITY LONG-TERM ORGAN DAMAGE Significant near
Slide 11: Consistency of clinical data supports potential of PYRUKYND across hemolytic anemias
Phase 3 ENERGIZE and ENERGIZE-T studies Phase 2 long-term extension study Phase 3 long-term extension study Phase 2/3 RISE UP study Investigator-sponsored trials Consistency of clinical data to date supports potential of PYRUKYND® to address unmet patient needs across hemolytic anemias 1. Al-Samkari
Slide 12: Emerging Long-term Data
Emerging Long-term Data Thalassemia (Wild-type PK enzyme) Data through Phase 2 Adult PK Deficiency (Mutant PK enzyme) Approved in the U.S., EU and Great Britain Sickle Cell Disease (Wild-type PK enzyme) Data through Phase 1 and ISTs Consistency of clinical data to date supports potential of PYRUKYND
Slide 13: Meaningful commercial opportunities on the horizon
Meaningful commercial opportunities on the horizon FDA approval and launch of PYRUKYND in PK deficiency builds capabilities to maximize potential product expansion
Slide 14: Optimizing Patient & Provider Journey
Optimizing Patient & Provider Journey PYRUKYND® launch in PK deficiency building commercial capabilities to support potential expansion in meaningfully larger patient populations Therapy Onboarding Access & Adherence Diagnostic Efficiency Awareness & Education Coverage support, free product eligibil
Slide 15: High unmet need across patient segments and geographies
High unmet need across patient segments and geographies ~18-23K Patients in U.S./EU5 Significant opportunity outside U.S./EU5 Thalassemia: no approved therapies for ~60% of patients Beta-THAL prevalence: HEOR Global THAL Epidemiology SLE (XCENDA, 2021); US: Paramore, et.al; DE: Borchert, et.al; IT:
Slide 16: Sickle Cell Disease: target profile and opportunity
PYRUKYND® Chronic Therapy Oral Improved hemolytic anemia Improvement in sickle cell pain crises (vaso-occlusive crises; VOCs) QoL data Safety profile consistent with prior clinical experience TARGET PROFILE Sickle Cell Disease: no novel oral therapy improves anemia and reduces sickle cell pain crise
Slide 17: Well capitalized to advance and expand
Well capitalized to advance and expand Long-term growth and value creation fueled by accomplished leadership team and strong balance sheet
Slide 18: Focused on rare diseases rooted in cellular metabolism
Focused on rare diseases rooted in cellular metabolism Strong cash position expected to support portfolio advancement and expansion PYRUKYND® First and only disease-modifying treatment for adults with PK deficiency Potential for two additional indications by 2026 AG-946 Potential to be first oral th
Slide 19: 2023 and Beyond
2023 and Beyond Clinical development strategy driving toward broader commercial opportunities
Slide 20: Clinical and regulatory milestones targeted in 2023
Clinical and regulatory milestones targeted in 2023 lay the foundation for transformational data readouts Build commercial capabilities to efficiently launch additional indications and evaluate business development opportunities to expand pipeline Thalassemia PYRUKYND® Pediatric PK Deficiency PYRUKY
Slide 21: Potential for two additional PYRUKYND indications by 2026
Potential for two additional PYRUKYND® indications by 2026 Thalassemia PYRUKYND® Pediatric PK Deficiency PYRUKYND® Sickle Cell Disease PYRUKYND® Lower-Risk MDS AG-946 (Novel PK Activator) Phase 3 ACTIVATE-kids and ACTIVATE-kidsT readouts Phase 3 ENERGIZE (1H) and ENERGIZE-T (2H) readouts Potential a
Slide 22: Differentiated Capabilities & Cross-functional Connectivity
Differentiated Capabilities & Cross-functional Connectivity Hematology franchise spanning 3 hemolytic anemias Expanded portfolio aligned with our core expertise Cash flow positive 2026 VISION Driven to transform patient outcomes in rare diseases
Slide 23: Q&A
Q&A
Agios Pharmaceuticals — Investor Presentation (January 2023) on 6ix