Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Q1 2024 Earnings Presentation
37 slides from Agios Pharmaceuticals's earnings presentation, May 2, 2024
Slide 1: Q1 2024 Financial Results
Q1 2024 Financial Results May 2, 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 R&D Commercial Update Tsveta Milanova, Chief
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: Continuing clinical and regulatory milestone momentum, with three Phase 3 data readouts in 2024
Continuing clinical and regulatory milestone momentum, with three Phase 3 data readouts in 2024 EARLY 2024 MID-YEAR 2024 YEAR-END 2024 Q2 Thalassemia Thalassemia Lower-Risk MDS Thalassemia PYRUKYND® PYRUKYND® AG-946 (Novel PK Activator) PYRUKYND® Phase 3 data readout for Phase 3 data readout Begin p
Slide 6: Strong beginning to 2024 with positive Thalassemia Phase 3 ENERGIZE readout
Strong beginning to 2024 with positive Thalassemia Phase 3 ENERGIZE readout; four additional Phase 3 readouts expected by the end of 2025 2024 2025 2026 Sickle Cell Disease Sickle Cell Disease Thalassemia PYRUKYND® PYRUKYND® PYRUKYND® Phase 3 RISE UP readout Potential approval Phase 3 ENERGIZE reado
Slide 7: 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 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: Clinical Overview
Clinical Overview Sarah Gheuens, M.D., Ph.D. Chief Medical Officer, Head of R&D 8
Slide 9: 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 10: 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 11: ENERGIZE topline data received positive feedback from thalassemia patients and clinicians
ENERGIZE topline data received positive feedback from thalassemia patients and clinicians Thalassemia ENERGIZE results Implications for Mitapivat ▪ Mitapivat has the potential to be the first therapy to ▪ Statistically significant increases in hemoglobin improve hemoglobin and make patients with res
Slide 12: ENERGIZE-T: trial design reflects real-world clinical treatment dynamics
ENERGIZE-T: trial design reflects real-world clinical treatment dynamics Key Considerations N = 240 2:1 randomization ▪ Individualized Hb threshold determines the 100 mg BID Placebo Hb value by which a given patient will BID receive a transfusion ▪ Individualized Hb thresholds based on 48-week core
Slide 13: PYRUKYND: a novel oral therapy with potential to be best-in-class in sickle cell disease
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(1) PYRUKYND • Statistically significant increase in • Phase 3 primary endpoints: • Seamless Phase 2/3 global hemoglobin response
Slide 14: 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 15: 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 16: Commercial Overview
Commercial Overview Tsveta Milanova Chief Commercial Officer 16
Slide 17: 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 for High rates of Adult patient
Slide 18: 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 19: 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 | ~50% of active Launch Top Affiliated adult patients c
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: 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 22: PYRUKYND Q1 2024 performance metrics highlight continued progress
PYRUKYND® Q1 2024 performance metrics highlight continued progress $8.2M net sales of PYRUKYND® 15% growth over Q4 2023 188 unique patients completed PYRUKYND® prescription enrollment forms, including 10 in Q1, a 6% increase over Q4 2023 120 patients on PYRUKYND®, which includes new prescriptions an
Slide 23: 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 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 24: Financial Overview
Financial Overview Cecilia Jones Chief Financial Officer 24
Slide 25: First quarter 2024 financial results
First quarter 2024 financial results Three Months Ended Three Months Ended Statement of Operations 3/31/24 3/31/23 PYRUKYND® Net Revenue $8.2M $5.6M Cost of Sales $0.6M $0.6M Research & Development Expense $68.6M $67.3M Selling, General & Administrative Expense $31.0M $28.4M Net Loss ($81.5M) ($81.0
Slide 26: Closing Remarks
Closing Remarks Brian Goff Chief Executive Officer 26
Slide 27: 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 28: Thank You
Zs agios Thank You
Slide 29: Appendix
Zs agios Appendix
Slide 30: 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 31: 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 32: 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 33: RISE UP Phase 2/3 operationally seamless trial of PYRUKYND in sickle cell disease
RISE UP Phase 2/3 operationally seamless trial of PYRUKYND® in sickle cell disease allows for speed and flexibility of clinical program Phase 2 PHASE 2 PRIMARY ENDPOINTS Phase 3 • 1:1:1 randomization to Hemoglobin Safety mitapivat 50 mg BID, 100 response profile mg BID or matched • 2:1 randomization
Slide 34: 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 35: 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
Slide 36: 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 37: Pediatric PK Deficiency Program: Two Phase 3 studies
Pediatric PK Deficiency Program: Two Phase 3 studies evaluating regularly transfused and not regularly transfused pediatric patients with PKD N = 30 N = 45 2:1 randomization 2:1 randomization Mitapivat BID Placebo BID Mitapivat BID Placebo BID 20-week double-blind period 32-week double-blind period
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