Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Q4 2023 Earnings Presentation
36 slides from Agios Pharmaceuticals's earnings presentation, Feb 15, 2024
Slide 1: Q4 2023 Financial Results
Q4 2023 Financial Results February 15, 2024 1
Slide 2: Agios conference call participants
Agios conference call participants TOPIC PARTICIPANT Chris Taylor, VP Investor Relations and Corporate Introduction Communications Business Update Brian Goff, Chief Executive Officer Sarah Gheuens, M.D., Ph.D., Chief Medical Officer, R & D Update Head of Research and Development Commercial Update Ts
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 the potential benefits of PYRUKYND®
Slide 4: Business Overview
Business Overview Brian Goff Chief Executive Officer 4
Slide 5: Momentum building as we delivered on all 2023 goals
Momentum building as we delivered on all 2023 goals Pediatric PK Deficiency Thalassemia PYRUKYND® Sickle Cell Disease PYRUKYND® Enroll at least half of patients in the PYRUKYND® Pipeline Complete enrollment of Phase 3 Phase 3 ACTIVATE-kids and Begin patient enrollment in Phase 3 File IND for PAH sta
Slide 6: Continuing clinical and regulatory milestone momentum into 2024, with two Phase 3 data readouts
Continuing clinical and regulatory milestone momentum into 2024, with two Phase 3 data readouts 2024 EARLY MID-YEAR YEAR-END Thalassemia Thalassemia Thalassemia PYRUKYND® PYRUKYND® PYRUKYND® Phase 3 data readout for Phase 3 data readout for Filing for FDA Approval ENERGIZE study ENERGIZE-T study (CO
Slide 7: PYRUKYND expansion into diseases with larger patient populations provides significant near-term growth potential
PYRUKYND® expansion into diseases with larger patient populations provides significant near-term growth potential for first- and best-in-class therapies 120-135K patients in the U.S./EU5 18-23K patients ~150K patients in the U.S./EU5 in GCC ~70K patients in GCC >3M patients 3-8K patients worldwide i
Slide 8: Strong beginning of 2024 with positive Thalassemia Phase 3 ENERGIZE readout
Strong beginning of 2024 with positive Thalassemia Phase 3 ENERGIZE readout; four additional Phase 3 readouts expected by the end of 2025 2024 2025 2026 Thalassemia Sickle Cell Disease Sickle Cell Disease PYRUKYND® PYRUKYND® PYRUKYND® Phase 3 ENERGIZE readout Phase 3 RISE UP readout Potential approv
Slide 9: Clinical Overview
Clinical Overview Sarah Gheuens, M.D., Ph.D. Chief Medical Officer, Head of Research and Development 9
Slide 10: Significant advancement building depth and breadth in our rare disease pipeline
Significant advancement building depth and breadth in our rare disease pipeline EARLY-STAGE LATE-STAGE REGULATORY COMPOUND INDICATION PRECLINICAL APPROVAL CLINICAL DEVELOPMENT CLINICAL DEVELOPMENT SUBMISSION US, EU, GB Pyruvate Kinase ACTIVATE KIDS - T Deficiency (PKD) PYRUKYND® ACTIVATE KIDS First-
Slide 11: Fueling growth beyond 2026, an early-stage pipeline addressing rare diseases
Fueling growth beyond 2026, an early-stage pipeline addressing the underlying pathophysiology of rare diseases with high unmet need Phenylketonuria Polycythemia Lower-Risk MDS (PKU) Vera (PV) ~75-80k patients in the U.S. and EU5 ~35-40k patients in the U.S. and EU5 ~100k patients in the U.S. Limited
Slide 12: PYRUKYND: a novel oral therapy with potential to be best-in-class
PYRUKYND: a novel oral therapy with potential to be best-in-class improving anemia, reducing SCPCs and improving how patients feel and function Phase 2 Data Phase 3 Design(2) PYRUKYND • Statistically significant increase in • Phase 3 primary endpoints: • Seamless Phase 2/3 global hemoglobin response
Slide 13: Sickle Cell Disease remains an area of significant need for innovative therapies
Sickle Cell Disease remains an area of significant need for innovative therapies that can demonstrate meaningful benefits beyond hemoglobin increase Disease Overview Lifelong impact on SCD patients Genetic blood disorder that causes sickling of red blood cells ~45 years Caused by mutations in HBB ge
Slide 14: Agios aims to deliver the first therapy approved for all thalassemia subtypes
Agios aims to deliver the first therapy approved for all thalassemia subtypes Mitapivat Thalassemia Mitapivat Thalassemia Phase 3 program Phase 3 program ~67% ~33% NON-TRANSFUSION TRANSFUSION • Alpha- and Beta- DEPENDENT (NTDT) DEPENDENT (TDT) • Alpha- and Beta- (~4K ADULTS) (~2K ADULTS) thalassemia
Slide 15: ENERGIZE: achieved significance across both primary and all key secondary endpoints
ENERGIZE: achieved significance across both primary and all key secondary endpoints Key findings in Next Steps ENERGIZE trial • Total of 194 patients were randomized 2:1 to 100 mg mitapivat (n=130) or • Full data set to be placebo (n=64) presented at an upcoming medical • Statistically significant i
Slide 16: ENERGIZE data drives positive feedback from thalassemia patients and clinicians
ENERGIZE data drives positive feedback from thalassemia patients and clinicians Thalassemia ENERGIZE results Implications for Mitapivat ▪ Mitapivat has potential to be the first therapy that ▪ Statistically significant increases in hemoglobin improves hemoglobin and makes patients with response rate
Slide 17: Commercial Overview
Commercial Overview Tsveta Milanova Chief Commercial Officer 17
Slide 18: Thalassemia remains an area of high unmet need with few treatment options
Thalassemia remains an area of high unmet need with few treatment options and significant burden of disease regardless of transfusion needs Serious, Irreversible Healthcare Resource Increased Mortality Poor Quality of Life Morbidities Utilization & Cost Lower survival and High rates of Adult patient
Slide 19: PYRUKYND has the potential to become the first therapy approved for all thalassemia subtypes
PYRUKYND® has the potential to become the first therapy approved for all thalassemia subtypes Thalassemia ~8K in US Adult Thalassemia (18+) ~6K (~70%) Alpha Thalassemia Beta Thalassemia ~3K (~47%) ~3K (~53%) U.S. Non-Transfusion Transfusion Non-Transfusion Transfusion Dependent Dependent Dependent D
Slide 20: Strong alignment with trial sites, key centers for thalassemia patients across US
Strong alignment with trial sites, key centers for thalassemia patients across US Patient prevalence heatmap alongside Agios trial sites and /or Centers of Excellence (COE) Availability of newborn Thalassemia screening Well established ICD-10 codes in Thalassemia Agios trial sites aligned to key Hea
Slide 21: Concentration of treating physicians facilitates initial PYRUKYND launch plan
Concentration of treating physicians facilitates initial PYRUKYND® launch plan Initial launch focus represents 65-70% adult thalassemia patients Target Treating Physician Segments CoE • Centers of Excellence and Top Initial Affiliated Practices | Concentrated Launch Top Affiliated ~50% of active adu
Slide 22: Strengthening our commercial capabilities to support Thalassemia launch
Strengthening our commercial capabilities to support Thalassemia launch in a meaningfully larger patient population Market Disease State Internal Market Understanding Education Capabilities Access ✓ Account profiling ✓ Patient and HCP ✓ Field team sizing ✓ Engage with Payers ✓ Claims driven insight
Slide 23: PYRUKYND Q4 2023 performance metrics highlight continued progress
PYRUKYND® Q4 2023 performance metrics highlight continued progress $7.1M net U.S. sales of PYRUKYND® $26.8M in 2023, from $11.7M in 2022 178 unique patients completed PYRUKYND® prescription enrollment forms, a 11% increase over Q3 2023 109 patients on PYRUKYND®, which includes new prescriptions and
Slide 24: PYRUKYND expansion into diseases with larger patient populations provides significant near-term growth potential
PYRUKYND® expansion into diseases with larger patient populations provides significant near-term growth potential for first- and best-in-class therapies 120-135K patients in the U.S./EU5 18-23K patients ~150K patients in the U.S./EU5 in GCC ~70K patients in GCC >3M patients 3-8K patients worldwide i
Slide 25: Financial Overview
Financial Overview Cecilia Jones Chief Financial Officer 25
Slide 26: Fourth quarter and full year 2023 financial results
Fourth quarter and full year 2023 financial results Three Months Three Months Year Ended Year Ended Statement of Operations Ended 12/31/23 Ended 12/31/22 12/31/23 12/31/22 PYRUKYND® Net Revenue $7.1M $4.3M $26.8M $11.7M Cost of Sales $0.6M $0.4M $2.9M $1.7M Research & Development $77.5M $70.3M $295.
Slide 27: Closing Remarks
Closing Remarks Brian Goff Chief Executive Officer 27
Slide 28: Well-positioned with multiple near-term catalysts to enter multi-billion-dollar markets
Well-positioned with multiple near-term catalysts to enter multi-billion-dollar markets and deliver significant value PKa franchise with Increasing Differentiated multi-billion-dollar probability of Growing pipeline mechanism of action potential success Large opportunities Clearly differentiated Pro
Slide 29: Thank You
Zs agios Thank You
Slide 30: Appendix
Zs agios Appendix
Slide 31: PYRUKYND: first Phase 3 program to encompass full range of thalassemia patients
PYRUKYND®: first Phase 3 program to encompass full range of thalassemia patients N = 171 N = 240 2:1 2:1 randomization randomization Placebo Placebo 100 mg BID 100 mg BID BID BID 24-week core period 48-week core period Open-label extension (up to 5 years) Open-label extension (up to 5 years) Primary
Slide 32: Phase 3 ENERGIZE study: primary endpoint achieved
Phase 3 ENERGIZE study: primary endpoint achieved ▪ Total of 194 patients were randomized 2:1 to 100 mg mitapivat (n=130) or placebo (n=64) ▪ Hemoglobin response is defined as ≥1.0 g/dL (10 g/L) increase in average Hb concentrations from Week 12 through Week 24 compared with baseline. ▪ Treatment wi
Slide 33: Statistical significance also 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 FACIT-Fatigue (Functional Assessment of Chronic Illness Therapy-Fatigue) subscale score fro
Slide 34: Advancing RISE UP Phase 3 Study of PYRUKYND in sickle cell disease
Advancing RISE UP Phase 3 Study of PYRUKYND® in sickle cell disease with expected readout in 2025 Phase 3 primary endpoints (1): Hb response, defined as a ≥ 1.0 g/dL increase in average Hb concentration over Weeks 24–52 compared with baseline, and annualized rate of SCPCs Key inclusion criteria Key
Slide 35: Treatment with mitapivat demonstrated a statistically significant increase in hemoglobin response rate
Treatment with mitapivat demonstrated a statistically significant increase in hemoglobin response rate compared to placebo Placebo Mitapivat 50 mg BID Mitapivat 100 mg BID N=27 N=26 N=26 Hemoglobin responders, n (%) 1 (3.7) 12 (46.2) 13 (50.0) Difference of response rate (Mitapivat-Placebo), 42.5 46
Slide 36: Annualized rates of sickle cell pain crises lower in mitapivat arms
Annualized rates of sickle cell pain crises for patients in the mitapivat arms were lower compared to patients in the placebo arm CRC Adjudicated Data Negative Binomial Regression Model Placebo Mitapivat 50 mg BID Mitapivat 100 mg BID N=27 N=26 N=26 Annualized Rate of SCPC 1.71 0.83 0.51 95% CI (0.9
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