Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Q3 2025 Earnings Presentation
28 slides from Agios Pharmaceuticals's earnings presentation, Oct 30, 2025
Slide 1: Q3 2025 Financial Results and Business Highlights
Q3 2025 Financial Results and Business Highlights Conference call for investors and analysts October 30, 2025
Slide 2: Forward Looking Statements
Forward Looking Statements This presentation and various remarks we make during this presentation contains 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 3: Q3 2025 earnings call agenda
Q3 2025 earnings call agenda 1 Introduction Morgan Sanford, VP Investor Relations 2 CEO Opening Remarks Brian Goff, Chief Executive Officer 3 Financial Results Cecilia Jones, Chief Financial Officer 4 Commercial Highlights Tsveta Milanova, Chief Commercial Officer 5 R&D Highlights Sarah Gheuens, MD,
Slide 4: CEO Opening Remarks
CEO Opening Remarks Brian Goff, Chief Executive Officer 4
Slide 5: Unlocking sustainable growth to deliver shareholder value
Unlocking sustainable growth to deliver shareholder value PYRUKYND® – de-risked Accelerating near-term Strong financial position, multi-billion opportunity high-value catalysts strategic capital allocation • Robust Phase 2 or 3 data • PYRUKYND PDUFA – thalassemia • $1.3B cash on hand shown across PK
Slide 6: Q3 2025 - continued portfolio and pipeline delivery
Q3 2025 - continued portfolio and pipeline delivery1 Global thalassemia Strong financial position Pipeline momentum regulatory actions PYRUKYND SFDA approval Enrollment complete in $12.9M net revenues in Saudi for thalassemia tebapivat Phase 2b trial for +44% vs $9.0M in Q3 2024 lower-risk MDS top-l
Slide 7: Financial Results
Financial Results Cecilia Jones, Chief Financial Officer 7
Slide 8: Q3 2025 Financial Results
Q3 2025 Financial Results Statement of Operations Q3 2025 Q3 2024 PYRUKYND Net Revenue $12.9M $9.0M Cost of Sales $1.7M $0.8M Research & Development Expense $86.8M $72.5M Selling, General & Administrative Expense $41.3M $38.5M Net (Loss) Income1 ($103.4M) $947.9M Balance Sheet Q3 2025 Q4 2024 Cash,
Slide 9: Strong balance sheet, disciplined capital allocation strategy
Strong balance sheet, disciplined capital allocation strategy 1 Capital efficient global commercial build out 2 Strategic investment to advance novel pipeline 3 Value-enhancing pipeline expansion Well-capitalized to execute on commercial portfolio and development pipeline 9
Slide 10: Commercial Highlights
Commercial Highlights Tsveta Milanova, Chief Commercial Officer 10
Slide 11: PYRUKYND – continued demand in Q3 2025
PYRUKYND – continued demand in Q3 2025 Key Performance Metrics PYRKUKYND Net Revenue ($m) $12.9 $12.5 $12.9M net sales of PYRUKYND $10.7 compared with $12.5M in Q2 2025 and $9.0M in Q3 2024 $9.0 $8.7 262 unique PK deficiency patients completed prescription enrollment forms since launch in U.S.1 149
Slide 12: Thalassemia – capital-efficient commercial build-out
Thalassemia – capital-efficient commercial build-out United States GCC Europe Positive CHMP opinion New PDUFA goal date SFDA approval in Saudi Arabia October 16th; expected December 7th announced August 4th EC decision early 2026 Launch underway; Launch preparation ongoing patient access granted on
Slide 13: Recent engagement reinforces market readiness following potential PYRUKYND approval
Recent engagement reinforces market readiness following potential PYRUKYND approval Robust engagement with thalassemia community Strong feedback and receptivity from reinforces launch preparedness and market understanding recent community engagement Disease Roughly 6,000 diagnosed adult thalassemia
Slide 14: R&D Highlights
R&D Highlights Sarah Gheuens, MD, PhD, Chief Medical Officer, Head of R&D 14
Slide 15: Continued pipeline momentum in Q3 2025
Continued pipeline momentum in Q3 2025 REGULATORY PRECLINICAL EARLY-STAGE LATE-STAGE APPROVAL SUBMISSION Approved US, EU, UK Pyruvate Kinase Deficiency PYRUKYND Phase 3 completed ACTIVATE – Kids/KidsT NEW – SFDA approval1 First-in-class NTD and TD α- and β- Approved KSA; filing under review US, EU,
Slide 16: Potential best-in-class oral PK activator franchise for SCD
Potential best-in-class oral PK activator franchise for SCD PYRUKYND and tebapivat - Significant need for novel treatment options novel dual PK activation ~100,000 adult and ped patients diagnosed in the U.S.1,2 PKR PKM2 Improves overall Improves cellular RBC health energetics in tissue Lack of nove
Slide 17: PYRUKYND Phase 3 RISE UP trial designed to meet clinical needs of sickle cell disease
PYRUKYND Phase 3 RISE UP trial designed to meet clinical needs of sickle cell disease community 1 Hb increase Annualized SCPC Fatigue Directly addresses chronic SCPC linked to organ Baseline HRQOL comparable anemia, reducing organ dysfunction, early mortality, to diseases including cancer damage and
Slide 18: PYRUKYND Phase 3 data in SCD on track for late 2025
PYRUKYND Phase 3 data in SCD on track for late 20251 Phase 3 RISE UP Operationally seamless RISE UP Phase 2/3 trial Two primary endpoints2: Hb response rate3 Annualized rate of SCPCs • Defined as ≥1.0 g/dL increase in average Hb concentration • Planned sample size 198 with 90% power to detect from w
Slide 19: Tebapivat – more potent, once-daily PK activator
Tebapivat – more potent, once-daily PK activator Phase 2 trial ongoing for the Phase 2b trial ongoing for the treatment of LR-MDS treatment of SCD LR-MDS >70% of MDS cases; treatment goal to improve quality of life by addressing symptomatic anemia Tebapivat – potential first oral medicine to address
Slide 20: Diversifying our early pipeline with AG-181 and AG-236
Diversifying our early pipeline with AG-181 and AG-236 AG-181 Phenylketonuria AG-236 Polycythemia Vera Small molecule PAH stabilizer – selectively siRNA targeting TMPRSS6 – selectively inhibits Mechanism of Action binds and stabilizes PAH enzyme TMPRSS6 enzyme to improve iron metabolism Safe and tol
Slide 21: CEO Closing Remarks
CEO Closing Remarks Brian Goff, Chief Executive Officer 21
Slide 22: Strong execution against corporate priorities for 2025
Strong execution against corporate priorities for 2025 EARLY MID-YEAR LATE Pediatric PK Deficiency Sickle Cell Disease Thalassemia PYRUKYND tebapivat PYRUKYND Potential FDA approval Phase 3 readout ACTIVATE-Kids Initiate enrollment in Phase 2 trial (Extended PDUFA goal date December 7th) Polycythemi
Slide 23: Agios – foundation to deliver innovation and long-term growth
Agios – foundation to deliver innovation and long-term growth Mid-to-late stage PKa franchise Seasoned leadership team PYRUKYND (mitapivat) tebapivat with diverse rare disease experience PK Deficiency LR-MDS Thalassemia Sickle Cell Disease Sickle Cell Disease Innovative delivery fueled by connection
Slide 24: Q&A session
Q&A session FH agios | 24
Slide 25: Appendix
Appendix 25
Slide 26: Appendix – PYRUKYND MoA in sickle cell disease
Appendix – PYRUKYND MoA in sickle cell disease Glycolytic pathway PYRUKYND (mitapivat) novel pan-PK activation Glucose PKR PKM2 Improves overall Improves cellular RBC health energetics in tissue FBP 1,3-DPG 2,3-DPG PYRUKYND modulates SCD symptomology 3-PG • Decreasing 2,3-DPG reduces HbS polymerizat
Slide 27: Appendix – U.S. thalassemia commercial opportunity
Appendix – U.S. thalassemia commercial opportunity 6,000 diagnosed adult thalassemia patients in U.S. Initial launch focus | 4,000 patients Remaining 2,000 diagnosed adult thalassemia patients Higher frequency of visits, transfusion dependent and/or symptomatic Younger Older patient Co-morbid Hb <10
Slide 28: Appendix – GCC thalassemia launch with NewBridge
Appendix – GCC1 thalassemia launch with NewBridge Adult and pediatric thalassemia patients, ~70,000 estimated prevalence 1 regardless of genotype or phenotype Diagnosed, treated patients Access and patient care spread across 2 multiple institutions Known at institutional level Target population Init
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