Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Q1 2025 Earnings Presentation
38 slides from Agios Pharmaceuticals's earnings presentation, May 1, 2025
Slide 1: Q1 2025 Financial Results and Business Update
Q1 2025 Financial Results and Business Update May 1, 2025 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: A Rare Blueprint for Success
A Rare Blueprint for Success Multi-Billion-Dollar Robust Pipeline, Proven Executional Strong Financial Market Opportunity Rich with Near-Term Excellence, Powered Position with PYRUKYND® Catalysts by Highly Experienced Team 5
Slide 6: 2025: Breakout Year
2025: Breakout Year 2024 2025 Transformative Year Breakout Year Progressed pipeline and reached critical clinical and regulatory Maximize potential of milestones 1 PYRUKYND® franchise Bolstered commercialization expertise Progress and diversify key 2 pipeline programs Expanded geographic commercial
Slide 7: Clinical and Regulatory Near-Term Catalysts Offer Potential to Significantly Drive Shareholder Value
Clinical and Regulatory Near-Term Catalysts Offer Potential to Significantly Drive Shareholder Value 2025 EARLY MID-YEAR LATE Pediatric PK Deficiency Sickle Cell Disease Thalassemia PYRUKYND® Tebapivat PYRUKYND® Phase 3 data readout for Begin patient enrollment in Potential FDA approval ACTIVATE-Kid
Slide 8: Clinical Overview
Clinical Overview Sarah Gheuens, M.D., Ph.D. Chief Medical Officer, Head of R&D 8
Slide 9: Advancing Therapies for Rare Diseases with Limited or No Treatment Options
Advancing Therapies for Rare Diseases with Limited or No Treatment Options EARLY-STAGE LATE-STAGE REGULATORY COMPOUND INDICATION PRECLINICAL CLINICAL CLINICAL SUBMISSION APPROVAL DEVELOPMENT DEVELOPMENT U.S., EU, GB Pyruvate Kinase Deficiency ACTIVATE - Kids T PYRUKYND® ACTIVATE - Kids First-in-clas
Slide 10: Thalassemia: High Patient Need with Limited or No Treatments and Significant Disease Burden
Thalassemia: High Patient Need with Limited or No Treatments and Significant Disease Burden of thalassemia patients Increased Mortality ~67% with no approved Lower survival for thalassemia patients, and significantly worse in patients who therapies in the U.S. remain non-regularly transfused compare
Slide 11: PYRUKYND Poised to Be First and Only Approved Therapy to Treat All Subtypes of Thalassemia
PYRUKYND® Poised to Be First and Only Approved Therapy Indicated to Treat All Subtypes of Thalassemia ENERGIZE and ENERGIZE-T Phase 3 Results Presented at EHA 2024 and ASH 2024 Established a favorable benefit-risk Population profile for mitapivat in adult patients • Enrolled a total of 452 patients
Slide 12: Sickle Cell Disease: Urgent Need for Multiple Innovative Therapies
Sickle Cell Disease: Urgent Need for Multiple Innovative Therapies that Demonstrate Clinically Meaningful Benefits Increased Mortality 30-year reduction in life expectancy; 48 years median survival in patients with severe sickle cell disease OUR OPPORTUNITY Serious Morbidities 120-135K patients Asso
Slide 13: PYRUKYND Offers Best-in-Class Opportunity in Sickle Cell Disease
PYRUKYND® Offers Best-in-Class Opportunity in Sickle Cell Disease with Potential to Improve Anemia, Reduce VOCs and Improve How Patients Feel and Function Phase 3 RISE UP study topline readout in late 2025; Potential U.S. launch in 2026 STUDY Sickle cell disease patients 16 years of age or older POP
Slide 14: Tebapivat Provides High-Growth Potential with Best- and First-in-Class Opportunities
Tebapivat Provides High-Growth Potential with Best- and First-in-Class Opportunities in Areas of Critical Medical Need PREVALENCE MEDICAL NEED STATUS No oral therapy Phase 2b study Lower-Risk addresses ongoing, with MDS ~75K-80K ineffective patient enrollment Potential first oral patients erythropoi
Slide 15: Phase 2 Study of Tebapivat in Sickle Cell Disease
Phase 2 Study of Tebapivat in Sickle Cell Disease Primary endpoint: Phase 2 Hb response, defined as ≥ 1.0 g/dL increase in hemoglobin concentration from Week 10 through Week 12 compared with baseline Status Secondary endpoints: Safety, changes in hemoglobin, additional measures of hemolysis, erythro
Slide 16: Phase 2b Open-Label Study of Tebapivat in Lower-Risk MDS
Phase 2b Open-Label Study of Tebapivat in Lower-Risk MDS Phase Primary endpoint: Transfusion independence, defined as transfusion-free for ≥8 consecutive weeks during the Core 2b Period Status • Completion of enrollment expected Secondary endpoints: safety, change in hemoglobin, TI for 12 weeks, add
Slide 17: Early-Stage Pipeline Offers Opportunity for Advancement
Early-Stage Pipeline Offers Opportunity for Advancement PREVALENCE MEDICAL NEED STATUS Left untreated can cause range of Phase 1 study in Phenylketonuria ~35-40k patients neurocognitive issues healthy volunteers AG-181 in U.S./EU5 and decrease in IQ; progressing to MAD limited treatment in mid-2025
Slide 18: Commercial Overview
Commercial Overview Tsveta Milanova Chief Commercial Officer 18
Slide 19: PYRUKYND Expansion into Larger Patient Populations Provides Multi-Billion-Dollar Market Opportunity
PYRUKYND® Expansion into Larger Patient Populations Provides Multi-Billion- Dollar Market Opportunity 120-135K patients in the U.S./EU5* ~150K patients 18-23K patients in GCC* in the U.S./EU5* >3M patients 3-8K patients ~70K patients in GCC* worldwide* in the U.S./EU5* >1M patients worldwide* PK Def
Slide 20: Commercial Expertise and Capabilities in Place to Deliver Strong U.S. Launch
Commercial Expertise and Capabilities in Place to Deliver Strong U.S. Launch; PDUFA Goal Date September 7, 2025 knowledge sharing icon capabilities icon Disease State Commercial Market Education Presence Access ✓ Patient and HCP ✓ Right sized customer- ✓ Payer education on targeted education on faci
Slide 21: Initial PYRUKYND U.S. Launch will Focus on ~65% of Thalassemia Patient Population
Initial PYRUKYND® U.S. Launch will Focus on Addressing ~65% of Thalassemia Patient Population 6,000 diagnosed diagnosedadults adultswith with thalassemia thalassemia in U.S. in the the U.S. Initial Launch Focus for Expanded Launch Focus PYRUKYND for PYRUKYND Younger Older Patient Hb<10 g/dL Hb>10 g/
Slide 22: Agios Market Research Identified Top Clinical Characteristics Healthcare Providers Will Consider
Agios Market Research Identified Top Clinical Characteristics Healthcare Providers Will Consider When Prescribing PYRUKYND Top Clinical Patient Characteristics HCPs Will Consider When Prescribing PYRUKYND Hb Fe Hemoglobin Transfusion Fatigue Iron Levels Burden Overload Source: US THAL Demand Study (
Slide 23: PYRUKYND has Potential to Transform the Thalassemia Treatment Landscape
PYRUKYND® has Potential to Transform the Thalassemia Treatment Landscape FIRST Phase 3 program to include Alpha- & Beta-thalassemia FIRST oral treatment candidate to show potential for benefits in pivotal Phase 3 FIRST to demonstrate Quality-of-Life improvements in non-transfusion dependent patients
Slide 24: PYRUKYND Q1 2025 Performance Metrics Highlight Continued Progress
PYRUKYND® Q1 2025 Performance Metrics Highlight Continued Progress $8.7M net sales of PYRUKYND® compared with $10.7M in Q4 2024 and $8.2M in Q1 2024 234 unique patients completed PYRUKYND® prescription enrollment forms, including 11 in Q1, a 5% increase over Q4 2024 136 patients on PYRUKYND®, which
Slide 25: Financial Overview
Financial Overview Cecilia Jones Chief Financial Officer 25
Slide 26: First Quarter 2025 Financial Results
First Quarter 2025 Financial Results Three Months Ended Three Months Ended Statement of Operations 3/31/25 3/31/24 PYRUKYND® Net Revenue $8.7M $8.2M Cost of Sales $1.1M $0.6M Research & Development Expense $72.7M $68.6M Selling, General & Administrative Expense $41.5M $31.0M Net Loss ($89.3M) ($81.5
Slide 27: Closing Remarks
Closing Remarks Brian Goff Chief Executive Officer 27
Slide 28: Clinical and Regulatory Near-Term Catalysts Offer Potential to Significantly Drive Shareholder Value
Clinical and Regulatory Near-Term Catalysts Offer Potential to Significantly Drive Shareholder Value 2025 EARLY MID-YEAR LATE Pediatric PK Deficiency Sickle Cell Disease Thalassemia PYRUKYND® Tebapivat PYRUKYND® Phase 3 data readout for Begin patient enrollment in Potential FDA approval ACTIVATE-Kid
Slide 29: Delivering Significant Value Through Strategic Capital Allocation
Delivering Significant Value Through Strategic Capital Allocation $1.4B Cash, Cash Equivalents and Marketable Securities* Maximize Advance Expand PYRUKYND® Early- and Mid-Stage Pipeline with Internal Thalassemia Clinical Pipeline and External and Sickle Cell Disease Opportunities Potential Launches
Slide 30: Thank You!
Thank You! 30 All individuals featured are real patients who have been compensated for their time.
Slide 31: Appendix
Zs agios Appendix
Slide 32: 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 Late 2025 Phase Two primary endpoints (1): 3 Hb response defined as a ≥1.0 g/dL increase in Annualized rate of SCPCs: average Hb concentration from Week 24 through The sample size will also provide 90% power
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 Glucose GLYCOLYSIS FBP Decreasing 2,3-DPG reduces HbS polymerization by increasing 1,3-DPG Hb oxygenation and may inhibit the sickling process 2,3-DPG 3-PG PEP PK ADP Mitapivat ATP Increasing
Slide 34: Two Global, Phase 3, Randomized Controlled Trials of PYRUKYND in Thalassemia
Two Global, Phase 3, Randomized Controlled Trials of PYRUKYND® in Thalassemia Across Full Range of Thalassemia Patients N = 194 N = 258 2:1 randomization 2:1 randomization 100 mg BID Placebo BID 100 mg BID Placebo BID N = 130 N = 64 N = 171 N = 87 24-week core period 48-week core period Open-label e
Slide 35: PYRUKYND has the Potential to Become the First and Only Therapy Approved for All Thalassemia Subtypes
PYRUKYND® has the Potential to Become the First and Only 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 De
Slide 36: The GCC Region Represents a Significant Opportunity for PYRUKYND in Thalassemia
The GCC Region Represents a Significant Opportunity for PYRUKYND in Thalassemia; Regulatory Filings Under Review in Saudi Arabia and UAE Approximately 70k Thalassemia Patients in High unmet need given Gulf Cooperation Council (GCC) Countries disproportionally high prevalence Kuwait Entered distribut
Slide 37: opi %a
opi %a OLE f< ar \) ASSEM aI Al iW IAL 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. | "@ ir rh \ Fy lA 10 Til
Slide 38: First Clinical Study to Demonstrate Efficacy of an Oral Therapy for Children with PK Deficiency
First Clinical Study to Demonstrate Efficacy of an Oral Therapy for Children with PK Deficiency who are Not Regularly Transfused Topline Results Enrollment & Completion • A total of 30 patients aged 1 to <18 years were enrolled in the study, with 19 randomized to mitapivat twice-daily and 11 randomi
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