Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Q2 2024 Earnings Presentation
35 slides from Agios Pharmaceuticals's earnings presentation, Aug 1, 2024
Slide 1: Q2 2024 Financial Results
Q2 2024 Financial Results August 1, 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: Key milestones in Q2 2024 and continued positive momentum
Key milestones in Q2 2024 and continued positive momentum Announced that Phase 3 ENERGIZE-T study of mitapivat met the primary endpoint and all key secondary endpoints in adults with transfusion-dependent alpha- or beta-thalassemia Presented data from positive Phase 3 ENERGIZE study of mitapivat in
Slide 6: 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 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 7: Capturing larger patient populations positions PYRUKYND for significant 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 8: Clinical Overview
Clinical Overview Sarah Gheuens, M.D., Ph.D. Chief Medical Officer, Head of R&D 8
Slide 9: 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
Slide 10: ACTIVATE-KidsT: First clinical study in regularly transfused pediatric patients with PKD
ACTIVATE-KidsT: First clinical study in regularly transfused pediatric patients with PKD Topline Results Enrollment & Completion • A total of 49 patients aged 1 to <18 years were enrolled in the study, with 32 randomized to mitapivat twice-daily and 17 randomized to matched placebo. • 30 patients (9
Slide 11: Two global, Phase 3, randomized controlled trials of PYRUKYND in thalassemia
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 12: 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 13: 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 14: 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 15: ENERGIZE-T: Additional results demonstrate mitapivat's 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 16: 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 17: 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 • Phase 3 primary endpoints: • Seamless Phase 2/3 global • Statistically significant increase in Hb response(2) and
Slide 18: 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 19: Commercial Overview
Commercial Overview Tsveta Milanova Chief Commercial Officer 19
Slide 20: 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 21: 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 22: 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 23: 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 24: Disease state education campaigns launched for patients and health care providers
Disease state education (DSE) campaigns have been launched for both patients and health care providers DSE Objective Example DSE Messaging DSE Objective “Hemolysis and ineffective erythropoiesis are the primary drivers Highlight of downstream complications, including chronic anemia” Disease Burden “
Slide 25: Thalassemia hiding campaign creative
: a 0 cue op sy SEE wan TAT THALASSEMIA 18 HIDING It may not be apparent in all patients, but Serious risk of morbidities can exist regardless of LC y transfusion history.
Slide 26: Market research on clinical characteristics HCPs will consider when prescribing PYRUKYND
Recent market research identified top clinical characteristics healthcare providers will consider when prescribing PYRUKYND Top Clinical Patient Characteristics HCPs Will Consider When Prescribing PYRUKYND Hb F e Hemoglobin Transfusion Iron Overload / Fatigue Levels Burden Ferritin Levels Source: US
Slide 27: 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 28: PYRUKYND Q2 2024 performance metrics highlight continued progress
PYRUKYND® Q2 2024 performance metrics highlight continued progress $8.6M net sales of PYRUKYND® 5% growth over Q1 2024 201 unique patients completed PYRUKYND® prescription enrollment forms, including 13 in Q2, a 7% increase over Q1 2024 128 patients on PYRUKYND®, which includes new prescriptions and
Slide 29: 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 30: Financial Overview
Financial Overview Cecilia Jones Chief Financial Officer 30
Slide 31: Second quarter 2024 financial results
Second quarter 2024 financial results Three Months Ended Three Months Ended Statement of Operations 6/30/24 6/30/23 PYRUKYND® Net Revenue $8.6M $6.7M Cost of Sales $1.5M $1.1M Research & Development Expense $77.4M $68.9M Selling, General & Administrative Expense $35.5M $30.4M Net Loss ($96.1M) ($83.
Slide 32: Closing Remarks
Closing Remarks Brian Goff Chief Executive Officer 32
Slide 33: Growing stack of Phase 3 data readouts positions mitapivat as a multi-billion-dollar franchise
Growing stack of Phase 3 data readouts positions mitapivat to become a multi- billion-dollar franchise 2024 2025 2026 Thalassemia Sickle Cell Disease Sickle Cell Disease PYRUKYND® PYRUKYND® PYRUKYND® Phase 3 ENERGIZE readout Phase 3 RISE UP readout Potential approval Thalassemia Thalassemia Pediatri
Slide 34: 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 35: Thank You
Zs agios Thank You
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