Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — J.P. Morgan Healthcare Conference 2024
28 slides from Agios Pharmaceuticals's conference presentation, Jan 8, 2024
Slide 1: J.P. Morgan Healthcare Conference – Agios Pharmaceuticals
J.P. Morgan Healthcare Conference Agios Pharmaceuticals Brian Goff, Chief Executive Officer January 10, 2024 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: Vision, mission and values
To become a leading rare disease company providing first-in-class and/or best-in-class new therapies for diseases with high unmet need Aim High Come Together Embrace Differences Bring Your Whole Self Blaze New Trails Tamara Pyruvate Kinase Deficiency Sharonda Sickle Cell Disease Fueled by Connection
Slide 4: Well-positioned with multiple near-term catalysts
Well-positioned with multiple near-term catalysts to enter multi-billion dollar markets and deliver significant value Increasing probability of success Proven track record supported by compelling and consistent data to date Differentiated mechanism of action Clearly differentiated PK activation fran
Slide 5: PYRUKYND expansion into diseases with larger patient populations
PYRUKYND® expansion into diseases with larger patient populations provides significant near-term growth potential for first- and best-in-class therapies Sickle cell disease 2026 Potential U.S. approval 120-135K patients in the U.S./EU5 ~150K patients in GCC >3M patients worldwide OUR GOAL Deliver a
Slide 6: Unique PK activation mechanism benefits beyond hemoglobin improvement
Unique PK activation mechanism has demonstrated comprehensive benefits beyond hemoglobin improvement PKR Improves cellular energetics in the tissues Improves overall red blood cell health PKM2 Improvement of hemolytic anemia Early and sustained response Potential long term benefits Consistent, compe
Slide 7: 2020 Pipeline: beginning of a rare disease portfolio
COMPOUND INDICATION PRECLINICAL EARLY-STAGE CLINICAL DEVELOPMENT LATE-STAGE CLINICAL DEVELOPMENT REGULATORY SUBMISSION APPROVAL Pyruvate Kinase Deficiency (PKD) α- and β-Thalassemia Healthy Volunteers / Sickle Cell Disease (SCD) 2020 Pipeline: the beginning of a rare disease portfolio leveraging our
Slide 8: 2024 Pipeline: significant advancement building depth and breadth
COMPOUND INDICATION PRECLINICAL EARLY-STAGE CLINICAL DEVELOPMENT LATE-STAGE CLINICAL DEVELOPMENT REGULATORY SUBMISSION APPROVAL Pyruvate Kinase Deficiency (PKD) α- and β-Thalassemia Sickle Cell Disease (SCD) Lower Risk Myelodysplastic Syndrome (LR-MDS) Healthy Volunteers / Sickle Cell Disease Phenyl
Slide 9: Momentum building as we delivered on all 2023 goals
2023 Evaluate business development opportunities to expand pipeline and build commercial capabilities to efficiently launch additional indications Momentum building as we delivered on all 2023 goals to expand and advance our pipeline and strengthened clinical evidence across our PKa franchise Lower-
Slide 10: First therapy approved for all thalassemia subtypes
Agios aims to deliver the first therapy approved for all thalassemia subtypes NO APPROVED THERAPIES IN THE U.S. ~67% Alpha- and Beta-thalassemia Non-transfusion dependent patients Primary endpoint: Hemoglobin (Hb) response Data announced January 2024 Alpha- and Beta-thalassemia Transfusion dependent
Slide 11: Thalassemia remains an area of high unmet need
Thalassemia in all forms remains an area of high unmet need, high morbidity and significant impact on quality of life Disease Impact 18-23K patients in the U.S./EU5 ~70K patients in GCC >1M patients worldwide Serious, Irreversible Morbidities High rates of morbidities (even compared to TDT – e.g., t
Slide 12: ENERGIZE: significance across primary and key secondary endpoints
ENERGIZE: achieved significance across both primary and all key secondary endpoints Total of 194 patients were randomized 2:1 to 100 mg mitapivat (n=130) or placebo (n=64) Statistically significant increase in hemoglobin response rate (42.3%) compared to patients on placebo (1.6%) Statistically sign
Slide 13: Thalassemia: an attractive global rare disease opportunity
6,000 diagnosed adult patients in US Most patients diagnosed before adulthood Nearly 70% of patients have no treatment options Concentrated providers: ~50% of active adult patients in <150 hem/onc practices Recognized high unmet need Well-established ICD-10 codes Established patient advocacy organiz
Slide 14: Sickle Cell Disease remains an area of significant need
Sickle Cell Disease remains an area of significant need for innovative therapies that can demonstrate meaningful benefits beyond hemoglobin increase Disease Overview Market Opportunity No novel oral therapy improves anemia and reduces sickle cell pain crises (SCPC) Lifelong impact on SCD patients Av
Slide 15: Hemoglobin response rate increase in both doses vs placebo
Statistically significant increase in hemoglobin response rate observed in both doses compared to placebo Improvements in markers of hemolysis and erythropoiesis observed at both doses compared to placebo A trend in sickle cell pain crises reduction was observed at both doses compared to placebo No
Slide 16: Early-stage pipeline fueling growth beyond 2026
Fueling growth beyond 2026, an early-stage pipeline addressing the underlying pathophysiology of rare diseases with high unmet need Lower-Risk MDS 40% Achieved Transfusion Independence endpoint in open-label Phase 2a study Expect to initiate Phase 2b study of AG-946 in mid-2024 ~75-80k patients in t
Slide 17: Continuing clinical and regulatory milestone momentum into 2024
Continuing clinical and regulatory milestone momentum into 2024 Pediatric PK Deficiency PYRUKYND® Complete enrollment Phase 3 ACTIVATE kids study Lower-Risk MDS AG-946 (Novel PK Activator) Begin patient enrollment of Phase 2b study Pipeline Begin Phase 1 dosing for PAH stabilizer for the treatment o
Slide 18: Strong beginning of 2024 with four additional Phase 3 readouts
Strong beginning of 2024 with positive Thalassemia Phase 3 ENERGIZE readout and four additional Phase 3 readouts expected by the end of 2025 2024 2026 2025 Thalassemia PYRUKYND® Phase 3 ENERGIZE readout (completed) Thalassemia PYRUKYND® Phase 3 ENERGIZE-T readout Thalassemia PYRUKYND® Potential appr
Slide 19: Well-positioned with multiple near-term catalysts
Well-positioned with multiple near-term catalysts to enter multi-billion dollar markets and deliver significant value PK = pyruvate kinase Pka = pyruvate kinase activation Increasing probability of success Proven track record supported by compelling and consistent data to date Differentiated mechani
Slide 20: Thank you
EX agios Thank you
Slide 21: Appendix
EX agios Appendix
Slide 22: First Phase 3 program to encompass full range of thalassemia patients
PYRUKYND®: first Phase 3 program to encompass full range of thalassemia patients BID = twice daily; Hb = hemoglobin; HbE = hemoglobin E; HbH = hemoglobin H; PK = pharmacokinetics; PD = pharmacodynamics. Primary endpoint Mean Hb ≥ 1 g/dL from baseline Secondary endpoints Fatigue, additional measure
Slide 23: ENERGIZE study: primary endpoint achieved
Phase 3 ENERGIZE study: primary endpoint achieved Primary Endpoint Placebo N=64 Mitapivat 100 mg BID N=130 Hemoglobin responders, n (%) 1 (1.6) 55 (42.3) Adjusted difference of response rate (Mitapivat-Placebo), % 40.9 95% CI (32.0, 49.8) 2-sided p-value <0.0001 Abbreviations: RBC = red blood cell;
Slide 24: Statistical significance achieved for both key secondary endpoints
Statistical significance also achieved for both key secondary endpoints Key secondary endpoints: change from baseline in both hemoglobin concentration and FACIT-Fatigue Score Change from baseline in average hemoglobin concentration from Week 12 to Week 24 Change from baseline in average FACIT-Fatigu
Slide 25: Advancing RISE UP Phase 3 study in sickle cell disease
Advancing RISE UP Phase 3 Study of PYRUKYND® in sickle cell disease with expected readout in 2025 Key inclusion criteria ≥ 16 years of age Documented SCD (HbSS, HbSC, HbSβ0/HbSβ+ thalassemia, other SCD variants) Recurrent VOCs (vaso-occlusive crises) – defined as the occurrence of 2–10 SCPCs (acute
Slide 26: Mitapivat increased hemoglobin response rate compared to placebo
Treatment with mitapivat demonstrated a statistically significant increase in hemoglobin response rate compared to placebo Placebo N=27 Mitapivat 50 mg BID N=26 Mitapivat 100 mg BID N=26 Hemoglobin responders, n (%) 1 (3.7) 12 (46.2) 13 (50.0) Difference of response rate (Mitapivat-Placebo), % 42.5
Slide 27: Annualized rate of sickle cell pain crises
Placebo N=27 Mitapivat 50 mg BID N=26 Mitapivat 100 mg BID N=26 Annualized Rate of SCPC 1.71 0.83 0.51 95% CI (0.95, 3.08) (0.34, 1.99) (0.16, 1.59) Rate ratio (Mitapivat/Placebo) 0.48 0.30 95% CI (0.17, 1.39) (0.08, 1.07) Annualized rates of sickle cell pain crises for patients in the mitapivat arm
Slide 28: PYRUKYND potential to be first therapy approved for all thalassemia subtypes
Thalassemia PYRUKYND® has the potential to become the first therapy approved for all thalassemia subtypes Approved treatment in US* PYRUKYND potential label Alpha Thalassemia Beta Thalassemia Non-Transfusion Dependent Alpha Thalassemia Transfusion Dependent Alpha Thalassemia Non-Tran
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