Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Q4 2024 Earnings Presentation
37 slides from Agios Pharmaceuticals's earnings presentation, Feb 13, 2025
Slide 1: Q4 2024 Financial Results and Business Update
Q4 2024 Financial Results and Business Update February 13, 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: 2024: Transformative Year, Delivering on All Key Priorities
2024: Transformative Year, Delivering on All Key Priorities 2024 EARLY MID-YEAR LATE Thalassemia Thalassemia Lower-Risk MDS Thalassemia PYRUKYND® PYRUKYND® Tebapivat (AG-946) PYRUKYND® Phase 3 data readout for Phase 3 data readout for Begin patient enrollment of Filing for ENERGIZE study ENERGIZE-T
Slide 7: 2025: Breakout Year
2025: Breakout Year 2024 2025 Transformative Year Breakout Year Progressed pipeline and reached critical clinical and regulatory Maximize potential of milestones PYRUKYND® franchise Bolstered commercialization expertise Progress and diversify key pipeline programs Expanded geographic commercial reac
Slide 8: Clinical and Regulatory Near-Term Catalysts
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 (AG-946) PYRUKYND® Phase 3 data readout for Begin patient enrollment in Potential FDA approval ACT
Slide 9: Clinical Overview
Clinical Overview Sarah Gheuens, M.D., Ph.D. Chief Medical Officer, Head of R&D 9
Slide 10: 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 11: 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 PK Deficiency ACTIVATE-Kids Topline Data February 2025; ACTIVATE-KidsT Topline Data August 2024 N = 30 N = 45 2:1 randomization 2:1 randomization Aged 1 to <18 ye
Slide 12: 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
Slide 13: Thalassemia: High Patient Need with Limited or No Treatments
Thalassemia: High Patient Need with Limited or No Treatments and Significant Disease Burden ~67% No Approved Therapies in the U.S. Increased Mortality Lower survival for thalassemia patients, and significantly worse in patients who remain non-regularly transfused compared to regularly transfused pat
Slide 14: PYRUKYND Poised to Be First Approved Therapy for All Thalassemia Subtypes
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 15: 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 16: 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 17: Tebapivat (AG-946) Provides High-Growth Potential
Tebapivat (AG-946) 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 e
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 Defi
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 knowledge sharing icon capabilities icon Disease State Commercial Market Education Presence Access ✓ Patient and HCP ✓ Right sized customer- ✓ Payer education on targeted education on facing teams Thalassemia unmet need via
Slide 21: Initial PYRUKYND U.S. Launch Focus on ~65% of Thalassemia Patients
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: 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 23: PYRUKYND Q4 2024 Performance Metrics Highlight Continued Progress
PYRUKYND® Q4 2024 Performance Metrics Highlight Continued Progress $10.7M net sales of PYRUKYND® 20% growth over $9.0 million in Q3 2024 223 unique patients completed PYRUKYND® prescription enrollment forms, including 12 in Q4, a 6% increase over Q3 2024 130 patients on PYRUKYND®, which includes new
Slide 24: Financial Overview
Financial Overview Cecilia Jones Chief Financial Officer 24
Slide 25: Fourth Quarter and Full Year 2024 Financial Results
Fourth Quarter and Full Year 2024 Financial Results Three Months Three Months Year Ended Year Ended Statement of Operations Ended 12/31/24 Ended 12/31/23 12/31/24 12/31/23 PYRUKYND® Net Revenue $10.7M $7.1M $36.5M $26.8M Cost of Sales $1.3M $0.6M $4.2M $2.9M Research & Development $82.8M $77.5M $301
Slide 26: Closing Remarks
Closing Remarks Brian Goff Chief Executive Officer 26
Slide 27: Clinical and Regulatory Near-Term Catalysts
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 (AG-946) PYRUKYND® Phase 3 data readout for Begin patient enrollment in Potential FDA approval ACT
Slide 28: Delivering Significant Value Through Strategic Capital Allocation
Delivering Significant Value Through Strategic Capital Allocation $1.5B 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 29: Thank You!
Thank You! 29 All individuals featured are real patients who have been compensated for their time.
Slide 30: Appendix
Zs agios Appendix
Slide 31: 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 32: 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 33: PYRUKYND Potential to Become First 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 34: 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 35: 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 PK Deficiency Completed; data readout N = 30 N = 45 August 2024 2:1 randomization 2:1 randomization Mitapivat BID Placebo BID Mitapivat BID Placebo BID 20-week do
Slide 36: Phase 2b Open-Label Study of Tebapivat in Lower-Risk MDS
Phase 2b Open-Label Study of Tebapivat (AG-946) in Lower-Risk MDS (enrolling) 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 Ke
Slide 37: 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; ongoing limited treatment options Risk of thrombo
Agios Pharmaceuticals — Q4 2024 Earnings Presentation on 6ix