Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — J.P. Morgan Healthcare Conference 2025
28 slides from Agios Pharmaceuticals's conference presentation, Jan 13, 2025
Slide 1: J.P. Morgan Healthcare Conference – Agios Pharmaceuticals
J.P. Morgan Healthcare Conference Agios Pharmaceuticals Brian Goff, Chief Executive Officer January 15, 2025 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: Fueled by Connections to Transform Rare Diseases
Fueled by Connections to Transform Rare Diseases All individuals featured are real patients who have been compensated for their time.
Slide 4: Strong Foundation to Power New Era of Growth
Strong Foundation to Power New Era of Growth PYRUKYND® is approved in the U.S. for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency and in the Europe Union and in Great Britian for the treatment of PK deficiency in adult patients. PK deficiency: Pyruvate kinase defici
Slide 5: 2024: Transformative Year, Delivering on All Key Priorities
2024: Transformative Year, Delivering on All Key Priorities Early: January – April; Mid-Year: May – August; End: September – December PAH: Phenylalanine hydroxylase; PK deficiency: Pyruvate kinase deficiency Lower-Risk MDS Tebapivat (AG-946) Begin patient enrollment of Phase 2b study Thalassemia PYR
Slide 6: A Rare Blueprint for Success
A Rare Blueprint for Success Multi-Billion-Dollar Market Opportunity with PYRUKYND® Robust Pipeline, Rich with Near-Term Catalysts Proven Executional Excellence, Powered by Highly Experienced Team Strong Financial Position
Slide 7: Advancing Therapies for Rare Diseases with Limited or No Treatment Options
COMPOUND INDICATION PRECLINICAL EARLY-STAGE CLINICAL DEVELOPMENT LATE-STAGE CLINICAL DEVELOPMENT REGULATORY SUBMISSION APPROVAL PYRUKYND® First-in-class PK activator Pyruvate Kinase Deficiency NTDT and TDT α- and β-Thalassemia Sickle Cell Disease Tebapivat (AG-946) Novel PK activator Lower Risk Myel
Slide 8: 2025: Breakout Year
2025: Breakout Year Maximize potential of PYRUKYND® franchise Progress and diversify key pipeline programs Focus capital deployment priorities to sustain growth Progressed pipeline and reached critical clinical and regulatory milestones Expanded geographic commercial reach Bolstered commercializatio
Slide 9: Maximize Potential of PYRUKYND Franchise
Maximize Potential of PYRUKYND® Franchise Laurice Living with thalassemia, and her son, Ben 1
Slide 10: PYRUKYND Expansion into Larger Patient Populations Provides Multi-Billion-Dollar Opportunity
PYRUKYND® Expansion into Larger Patient Populations Provides Multi-Billion-Dollar Market Opportunity PYRUKYND® is approved in the U.S. for the treatment of hemolytic anemia in adults with pyruvate kinase (PK) deficiency and in the Europe Union and in Great Britian for the treatment of PK deficiency
Slide 11: Thalassemia: High Patient Need with Limited or No Treatments
Thalassemia: High Patient Need with Limited or No Treatments and Significant Disease Burden Sources: Beta-THAL prevalence: HEOR Global THAL Epidemiology SLE (XCENDA, 2021); US: Paramore, et.al; DE: Borchert, et.al; Alpha-THAL prevalence: Agios internal estimates; LEK Analysis | Beta-THAL TD/NTD spli
Slide 12: PYRUKYND Poised to Be First Approved Therapy for All Thalassemia Subtypes
PYRUKYND® Poised to Be First and Only Approved Therapy Indicated to Treat All Subtypes of Thalassemia PYRUKYND® is under investigation for thalassemia and is not approved anywhere for that use. Sources: Taher AT. ENERGIZE: A global, phase 3 study of mitapivat demonstrating efficacy and safety in adu
Slide 13: Initial U.S. Launch Focus on ~65% of Thalassemia Patients
~ 65% of diagnosed thalassemia patients ~ 35% of diagnosed thalassemia patients Initial PYRUKYND® U.S. Launch will Focus on Addressing ~65% of Thalassemia Patient Population *Patients aged 18 years and older Source: U.S. EHR Data Analysis Hb: Hemoglobin Younger Transfused Patient on Iron Chelators*
Slide 14: Commercial Expertise and Capabilities in Place for U.S. Launch
Commercial Expertise and Capabilities in Place to Deliver Strong U.S. Launch Disease State Education Patient and HCP targeted education on unmet need via digital and personal channels Synergistic omni-channel approach Commercial Presence Right sized customer-facing teams Focused targeting and HCP pr
Slide 15: Sickle Cell Disease: Urgent Need for Multiple Innovative Therapies
Sickle Cell Disease: Urgent Need for Multiple Innovative Therapies that Demonstrate Clinically Meaningful Benefits *Prevalence figures Sources: Agios internal estimates. Faro EZ, et al. Am J Prev Med. 2016;51(suppl 1):S48-S54. National Academies of Sciences, Engineering, and Medicine. Addressing Sic
Slide 16: PYRUKYND Offers Best-in-Class Opportunity in Sickle Cell Disease
PYRUKYND® Offers Best-in-Class Opportunity in Sickle Cell Disease with Potential to Improve Anemia, Reduce VOCs and Improve How Patients Feel and Function *Powering details in appendix PYRUKYND® is under investigation for sickle cell disease and is not approved anywhere for that use. Source: Andemar
Slide 17: Progress and Diversify Key Pipeline Programs
Progress and Diversify Key Pipeline Programs Teonna Living with sickle cell disease 2
Slide 18: Tebapivat (AG-946) Provides High-Growth Potential
Tebapivat (AG-946) Provides High-Growth Potential with Best- and First-in-Class Opportunities in Areas of Critical Medical Need Sources: Agios internal estimates. Greenberg PL, Tuechler H, Schanz J, et al. Revised international prognostic scoring system for myelodysplastic syndromes. Blood. 2012;120
Slide 19: Early-Stage Pipeline Offers Opportunity for Advancement
Early-Stage Pipeline Offers Opportunity for Advancement Sources: Agios internal estimates. van Wegberg et al. Orphanet Journal of Rare Diseases (2017) 12:162. Spivak JL. Myeloproliferative Neoplasms: Challenging Dogma. J Clin Med. 2024;13(22):6957. doi:10.3390/jcm13226957. EU: European Union; CV: Ca
Slide 20: Focus Capital Deployment Priorities to Sustain Growth
Focus Capital Deployment Priorities to Sustain Growth Golie Living with sickle cell disease 3
Slide 21: Delivering Significant Value Through Strategic Capital Allocation
Maximize PYRUKYND® Thalassemia and Sickle Cell Disease Potential Launches Advance Early- and Mid-Stage Clinical Pipeline Expand Pipeline with Internal and External Opportunities Delivering Significant Value Through Strategic Capital Allocation *As of September 30, 2024 $1.7B Cash, Cash Equivalents a
Slide 22: Clinical and Regulatory Near-Term Catalysts
Clinical and Regulatory Near-Term Catalysts Offer Potential to Significantly Drive Shareholder Value Early: January – April; Mid-Year: May – August; End: September – December PK deficiency: Pyruvate kinase deficiency; MDS Myelodysplastic syndromes; IND: Investigational new drug; PDUFA: Prescription
Slide 23: 2025: Breakout Year
Maximize Potential of PYRUKYND® Franchise Progress and Diversify Key Pipeline Programs Focus Capital Deployment Priorities to Sustain Growth 1 3 2 2025: Breakout Year
Slide 24: Thank you
> agios Thank you
Slide 25: Appendix
Zs agios Appendix
Slide 26: PYRUKYND Potential to Be First Therapy Approved for All Thalassemia Subtypes
PYRUKYND® has the Potential to Become the First and Only Therapy Approved for All Thalassemia Subtypes Beta-THAL prevalence: HEOR Global THAL Epidemiology SLE (XCENDA, 2021); US: Paramore, et.al; Alpha-THAL prevalence: Agios internal estimates; LEK Analysis | Beta-THAL TD/NTD split (60% / 40%): Thur
Slide 27: Advancing RISE UP Phase Study of PYRUKYND in Sickle Cell Disease
Advancing RISE UP Phase 3 Study of PYRUKYND® in Sickle Cell Disease with Expected Readout in Late 2025 Abbreviations: BID = twice daily; Hb = hemoglobin; SCPC = sickle cell pain crises; HU = hydroxyurea (1) Phase 2 and phase 3 components are part of a single study/protocol; (2) Patients who receive
Slide 28: Phase 2b Open-Label Study of Tebapivat in Lower-Risk MDS
Phase 2b open-label study of Tebapivat (AG-946) in lower-risk MDS (enrolling) Dose Level 1 10mg QD Core Period Dose Level 2 15mg QD Core Period Dose Level 3 20mg QD Core Period Dose Level 1 10mg QD Extension Period Dose Level 2 15mg QD Extension Period Dose Level 3 20mg QD Extension Period Tebapivat
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