Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Q3 2024 Earnings Presentation
41 slides from Agios Pharmaceuticals's earnings presentation, Oct 31, 2024
Slide 1: Q3 2024 Financial Results and Business Update
Q3 2024 Financial Results and Business Update October 31, 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 Co
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: Well-positioned with multiple clinical and regulatory catalysts
Well-positioned with multiple clinical and regulatory 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 dif
Slide 6: Key milestones in Q3 2024 and continued positive momentum
Key milestones in Q3 2024 and continued positive momentum Completed enrollment in Phase 3 RISE UP study of mitapivat in sickle cell disease (10/24) For tebapivat, commenced enrollment of the Phase 2b study in Lower-Risk Myelodysplastic Syndromes (LR-MDS); Granted FDA Orphan Drug Designation for trea
Slide 7: Continuing clinical and regulatory milestone momentum, three Phase 3 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 Thalassemia Thalassemia Lower-Risk MDS Thalassemia PYRUKYND® PYRUKYND® Tebapivat (AG-946) PYRUKYND® Phase 3 data readout for Phase 3 data readout Begin patient enrol
Slide 8: Capturing larger patient populations positions PYRUKYND for near-term growth
Capturing larger patient populations positions PYRUKYND® for significant near- term growth as a first- and best-in-class therapy 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 in the U.S./EU5 >1M patie
Slide 9: Clinical Overview
Clinical Overview Sarah Gheuens, M.D., Ph.D. Chief Medical Officer, Head of R&D 9
Slide 10: 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(1) PYRUKYND • Phase 3 primary endpoints: • Seamless Phase 2/3 global study • Statistically significant increase in Hb response(2
Slide 11: 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 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 12: Agios aims to deliver the first therapy for all thalassemia subtypes
Agios aims to deliver the first therapy for all thalassemia subtypes Mitapivat Thalassemia Mitapivat Thalassemia Phase 3 program Phase 3 program ~67% ~33% NON-TRANSFUSION TRANSFUSION • Alpha- and Beta- DEPENDENT (NTDT) (~4K ADULTS) DEPENDENT (TDT) • Alpha- and Beta- (~2K ADULTS) thalassemia Non- tha
Slide 13: PYRUKYND significantly increases Hb and improves quality-of-life measures
PYRUKYND significantly increases Hb and improves quality-of-life measures, highlighting potential to address unmet need in underserved community • Statistically significant improvements in hemoglobin and fatigue Phase 3 Data observed across the full range of NTDT for both subtypes compared to placeb
Slide 14: PYRUKYND has the potential to reduce transfusion burden
PYRUKYND has the potential to reduce transfusion burden and improve lives with unprecedented durability of effect • Statistically significant transfusion reduction response observed in the mitapivat Phase 3 Data arm (30.4%) compared to placebo (12.6%) • Transfusion independence achieved in a higher
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 Firs
Slide 16: Phase 2b open-label study of Tebapivat (AG-946) in lower-risk MDS
Phase 2b open-label study of Tebapivat (AG-946) in lower-risk MDS Phase Primary endpoint: Transfusion independence, defined as transfusion-free for ≥8 consecutive weeks during the Core 2b Period Secondary endpoints: safety, change in hemoglobin, TI for 12 weeks, additional measures of Key inclusion
Slide 17: Commercial Overview
Commercial Overview Tsveta Milanova Chief Commercial Officer 17
Slide 18: PYRUKYND has the potential to become 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: Thalassemia remains an area of high unmet need
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 20: Strengthening our commercial capabilities to support thalassemia launch
Strengthening our commercial capabilities to support thalassemia launch in a meaningfully larger patient population knowledge sharing icon knowledge sharing icon capabilities icon knowledge icon Market Disease State Internal Market Understanding Education Capabilities Access ✓ Account profiling ✓ Pa
Slide 21: PYRUKYND's initial launch focus within the thalassemia patient population
PYRUKYND’s initial launch focus will address approximately 65% of the thalassemia patient population Initial Launch Focus for Expanded Launch Focus PYRUKYND for PYRUKYND Younger Older Patient Hb<10 g/dL Hb>10 g/dL Co-morbid Transfused with Kidney Patient with Patient with Sickle-Cell Patient on Iron
Slide 22: Disease state education campaigns launched for patients and providers
Disease state education (DSE) campaigns have been launched for both patients and health care providers DSE Objectives DSE campaign was well received following Q2 launch DSE Objective Educate on the HCP: "I haven't seen this pathophysiology detail in a long time, it's helpful to be pathophysiology re
Slide 23: eels
eels on ss Even if you don't receive regular transfusions, thalassemia may come with serious risks. Learn More about these risks and what you can do about them. QrA ALASS ») ic Mii
Slide 24: The GCC region represents a significant opportunity for PYRUKYND in thalassemia
The GCC region represents a significant opportunity for PYRUKYND in thalassemia; distribution partner signed to unlock value Approximately 70k Thalassemia Patients in High unmet need given Gulf Cooperation Council (GCC) Countries disproportionally high prevalence Kuwait Entered distribution agreemen
Slide 25: PYRUKYND Q3 2024 performance metrics highlight continued progress
PYRUKYND® Q3 2024 performance metrics highlight continued progress $9.0M net sales of PYRUKYND® 4% growth over Q2 2024 211 unique patients completed PYRUKYND® prescription enrollment forms, including 10 in Q3, a 5% increase over Q2 2024 127 patients on PYRUKYND®, which includes new prescriptions and
Slide 26: 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 27: Financial Overview
Financial Overview Cecilia Jones Chief Financial Officer 27
Slide 28: Third quarter 2024 financial results
Third quarter 2024 financial results Three Months Ended Three Months Ended Statement of Operations 9/30/24 9/30/23 PYRUKYND® Net Revenue $9.0M $7.4M Cost of Sales $0.8M $0.6M Research & Development Expense $72.5M $81.8M Selling, General & Administrative Expense $38.5M $25.8M Net Income (Loss) $947.9
Slide 29: Closing Remarks
Closing Remarks Brian Goff Chief Executive Officer 29
Slide 30: Well-positioned with multiple clinical and regulatory catalysts
Well-positioned with multiple clinical and regulatory 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 dif
Slide 31: Thank You
Zs agios Thank You
Slide 32: Appendix
Zs agios Appendix
Slide 33: PK Activation in Sickle Cell Disease modulates 2,3-DPG and ATP
PK Activation in Sickle Cell Disease modulates 2,3-DPG and ATP, which may improve anemia and reduce sickling ADP = adenosine diphosphate; ATP = adenosine triphosphate; DPG = diphosphoglycerate; FBP = fructose bisphosphate; m = mutant; PEP = phosphoenolpyruvate; PG = phosphoglycerate; PK = pyruvate 3
Slide 34: Two global Phase 3 randomized trials encompass broad range of thalassemia patients
Two global, Phase 3, randomized controlled trials of PYRUKYND® encompass broad range of thalassemia patients N = 194 N = 258 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 exten
Slide 35: Phase 3 ENERGIZE study attained primary and key secondary endpoints
Phase 3 ENERGIZE study attained primary and key secondary endpoints; statistically significant improvements in Hb and fatigue • This global study was the first to enroll patients with α-thalassemia in addition to β- thalassemia • The primary and key secondary endpoints were met, with statistically s
Slide 36: Significant improvements in quality-of-life-related outcomes from ENERGIZE
Significant improvements in quality-of-life-related outcomes data from the Phase 3 ENERGIZE study • In the 24-week double-blind period of ENERGIZE, significant improvements in fatigue, measured by FACIT-Fatigue, were demonstrated in the mitapivat arm compared with the placebo arm ▪ A higher proporti
Slide 37: Phase 3 ENERGIZE-T Study: Primary endpoint achieved
Phase 3 ENERGIZE-T Study: Primary endpoint achieved ▪ Total of 258 patients were randomized 2:1 to 100 mg mitapivat (n=171) or placebo (n=87) ▪ 155 patients (90.6%) in the mitapivat arm and 83 patients (95.4%) in the placebo arm completed the 48-week double-blind period of the study ▪ Transfusion re
Slide 38: ENERGIZE-T: Additional results demonstrate durability of effect
ENERGIZE-T: Additional results demonstrate mitapivat’s durability of effect Efficacy Treatment with mitapivat demonstrated statistically significant improvements on all key secondary endpoints evaluating additional measures of reduction in transfusion burden: • ≥50% reduction in transfused RBC units
Slide 39: Mitapivat demonstrated 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 40: 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 41: Unique PK activation mechanism has demonstrated comprehensive benefits
Unique PK activation mechanism has demonstrated comprehensive benefits beyond hemoglobin improvement Pan-PK activation Consistent, compelling and comprehensive data reported to date* Improves overall red Improvement of Translated to PKR blood cell hemolytic anemia other clinically health • Hemoglobi
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