Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Q2 2025 Earnings Presentation
27 slides from Agios Pharmaceuticals's earnings presentation, Jul 31, 2025
Slide 1: Q2 2025 Financial Results and Business Highlights
Q2 2025 Financial Results and Business Highlights Conference call for investors and analysts July 31, 2025
Slide 2: 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 3: Q2 2025 earnings call agenda
Q2 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: Q2 2025 – continued delivery of strategic objectives
Q2 2025 – continued delivery of strategic objectives $12.5M PYRUKYND net revenues in Q2 2025 Strong pipeline execution in Q2 2025 +44% vs $8.7M in Q1 2025, +45% vs $8.6M in Q2 2024 tebapivat First patient dosed in Phase 2 novel potent PKa Announced commercialization and SCD | LR-MDS Sickle Cell Dise
Slide 7: Financial Results
Financial Results Cecilia Jones, Chief Financial Officer 7
Slide 8: Q2 2025 Financial Results
Q2 2025 Financial Results Statement of operations Q2 2025 Q2 2024 PYRUKYND Net Revenue $12.5M $8.6M Cost of Sales $1.7M $1.5M Research & Development Expense $91.9M $77.4M Selling, General & Administrative Expense $45.9M $35.5M Net Loss ($112.0M) ($96.1M) Balance sheet Q2 2025 Q4 2024 Cash, Cash Equi
Slide 9: Strong balance sheet, focused capital allocation strategy
Strong balance sheet, focused capital allocation strategy 1 Capital efficient commercial build out 2 Strategic investment to advance novel pipeline 3 Value-enhancing pipeline expansion Well-capitalized to execute on commercial portfolio and development pipeline 9
Slide 10: Commercial Highlights
Commercial Highlights Tsveta Milanova, Chief Commercial Officer 10
Slide 11: PYRUKYND – strong demand growth in Q2 2025
PYRUKYND – strong demand growth in Q2 2025 Key Performance Metrics PYRKUKYND Net Revenue ($m) $12.5 $10.7 $12.5M net sales of PYRUKYND compared with $8.7M in Q1 2025 and $8.6M in Q2 2024 $9.0 $8.7 $8.6 248 unique PK deficiency patients completed prescription enrollment forms since launch in U.S. 142
Slide 12: PYRUKYND – potential to deliver a series of firsts in thalassemia
PYRUKYND – potential to deliver a series of “firsts” in thalassemia1 First medicine to address α- and β-thalassemia First oral medicine First medicine to demonstrate quality of life improvements in non-transfusion dependent patients First medicine to demonstrate up to 36 weeks durability of effect o
Slide 13: Prepared for potential PYRUKYND U.S. launch in Q3 2025
Prepared for potential PYRUKYND U.S. launch in Q3 20251 Thalassemia – well-diagnosed and understood, Robust launch preparation to drive successful launch community in need of new treatment options Patients diagnosed – validated by ICD codes and Disease State Education 1 extensive market research • P
Slide 14: U.S. represents largest commercial opportunity
U.S. represents largest commercial opportunity, driven by favorable market dynamics and defined patient population 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 dep
Slide 15: Tailored commercial build out with ex-US partnerships
Tailored commercial build out with ex-US partnerships GCC commercial strategy Europe commercial strategy NewBridge commercialization and Avanzanite1 commercialization distribution agreement and distribution agreement Anticipate first GCC regulatory approval Anticipate potential EU approval in coming
Slide 16: Tailored commercial build out in GCC with NewBridge
Tailored commercial build out in GCC1 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 I
Slide 17: R&D Highlights
R&D Highlights Sarah Gheuens, MD, PhD, Chief Medical Officer, Head of R&D 17
Slide 18: PYRUKYND – consistent, meaningful clinical data in multiple hemolytic anemias
PYRUKYND – consistent, meaningful clinical data in multiple hemolytic anemias PK Deficiency Thalassemia Sickle Cell Disease Phase 2 open-label study Phase 2 open-label study Phase 2 – RISE UP Phase 3 – adults and pediatric Phase 3 – ENERGIZE/ENERGIZE-T Phase 3 – RISE UP Long-term data in adults Ongo
Slide 19: EHA 2025 data supports strong PKa franchise potential
EHA 2025 data supports strong PKa franchise potential ACTIVATE-KidsT ESTIMATE tebapivat tebapivat PK Deficiency Sickle Cell Disease Sickle Cell Disease Lower-Risk MDS • Phase 3 data • Long-term investigator-led • Preclinical data • Preclinical data • Clinically meaningful open-label trial • tebapiva
Slide 20: PYRUKYND Phase 3 RISE UP in SCD anticipated late 2025
PYRUKYND Phase 3 RISE UP in SCD anticipated late 20251 Operationally seamless RISE UP Phase 2/3 trial, aligned with the clinical priorities of SCD community Phase 3 RISE UP Dual primary endpoints2 Hb response rate3 (week 24-52 vs baseline) Annualized rate of SCPCs • 91% power to detect increase in H
Slide 21: Continued pipeline momentum in Q2 2025
Continued pipeline momentum in Q2 2025 REGULATORY PRECLINICAL EARLY-STAGE LATE-STAGE APPROVAL SUBMISSION Pyruvate Kinase Approved US, EU, UK Deficiency PYRUKYND Phase 3 completed ACTIVATE – Kids/KidsT First-in-class NTD and TD α- and β- Regulatory filing under review US, EU, KSA, UAE PK activator Th
Slide 22: CEO Closing Remarks
CEO Closing Remarks Brian Goff, Chief Executive Officer 22
Slide 23: Strong execution against R&D priorities for 2025
Strong execution against R&D 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 (PDUFA goal date September 7th) Polycythemia Vera Sickle
Slide 24: Agios – foundation to deliver innovation and long-term growth
Agios – foundation to deliver innovation and long-term growth Advancing a diversified rare disease portfolio across broad range Seasoned leadership team of indications with foundation in hematology with diverse rare disease Mid-to-late stage PKa franchise experience PYRUKYND (mitapivat) tebapivat PK
Slide 25: Q&A session
Q&A session FO agios | 25
Slide 26: Appendix
Appendix 26
Slide 27: 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
Agios Pharmaceuticals — Q2 2025 Earnings Presentation on 6ix