Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Q3 2023 Earnings Presentation
26 slides from Agios Pharmaceuticals's earnings presentation, Nov 2, 2023
Slide 1: Q3 2023 Financial Results
Q3 2023 Financial Results November 2, 2023 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, Research & Development Update Head of Research and Development Com
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: Opening Remarks
>< agios Opening Remarks
Slide 5: Clinical and regulatory milestones targeted in 2023 lay the foundation for transformational data readouts
Clinical and regulatory milestones targeted in 2023 lay the foundation for transformational data readouts Pediatric PK Deficiency Thalassemia PYRUKYND® Lower-Risk MDS Lower-Risk MDS PYRUKYND® Enroll at least half of patients in the AG-946 (Novel PK Activator) AG-946 Complete enrollment of Phase 3 Ph
Slide 6: Q3 2023 highlights
Q3 2023 highlights Pipeline updates Corporate updates • Dosed first patient in the Phase 3 portion of the RISE UP study of mitapivat in sickle cell • On track to achieve all 2023 disease milestones and deliver 3 mid-to- late stage readouts by the end of • Completed enrollment in the Phase 3 2024 ACT
Slide 7: Potential for two additional PYRUKYND indications by 2026
Potential for two additional PYRUKYND® indications by 2026 2024 2025 2026 Thalassemia Phase 3 ENERGIZE (1H) and ENERGIZE-T (2H) Potential approval PYRUKYND® readouts Pediatric PK Phase 3 ACTIVATE- Deficiency kids and ACTIVATE- Potential approval kidsT readouts PYRUKYND® Sickle Cell Disease Potential
Slide 8: Clinical
1 Clinical 2 3 8
Slide 9: Building a diverse pipeline leveraging our expertise in cellular metabolism
Building a diverse pipeline leveraging our expertise in cellular metabolism EARLY-STAGE LATE-STAGE REGULATORY RESEARCH APPROVAL CLINICAL DEVELOPMENT CLINICAL DEVELOPMENT SUBMISSION Pyruvate Kinase Deficiency US, EU, GB ACTIVATE Kids Enrollment >50% ACTIVATE KidsT Enrollment complete α- and β-Thalass
Slide 10: Highlighting key pipeline progress at ASH 2023
Highlighting key pipeline progress at ASH 2023 American Society of Hematology Annual Meeting December 9-12, 2023 | San Diego PK Sickle Cell Patient Thalassemia Deficiency Disease Advocacy Educational Session on PK Activation Organized by ASH Abstracts go live today, November 2 at 9 a.m. ET. 10
Slide 11: RISE UP Phase 3 Study: first patient dosed
RISE UP Phase 3 Study: first patient dosed Phase 3 primary endpoints (1): Hb response, defined as a ≥ 1.0 g/dL increase in average Hb concentration over Weeks 24–52 compared with baseline, and annualized rate of SCPCs Key inclusion criteria Key exclusion criteria • ≥ 16 years of age • Receiving regu
Slide 12: AG-946: Seamless Phase 2a proof-of-concept + Phase 2b trials
Novel PK activator AG-946: Seamless Phase 2a proof-of-concept + Phase 2b trials focused on establishing proof-of-concept and dose selection in LR-MDS Phase Open Label Phase 2a Double Blind, 1:1:1:1 Randomization 2b High N = 20 Open-Label dose Extension AG-946 High dose 16 weeks Medium dose Open-Labe
Slide 13: PAH program aimed to address the underlying cause of phenylketonuria (PKU)
PAH program aimed to address the underlying cause of phenylketonuria (PKU) Normal Protein Diet PHENYLKETONURIA (PKU) A mixed diet provides your body Phe 1 • Rare, genetic disease with limited treatment options • Prevalence: total of ~35-40K patients in the U.S. and EU5 • Driven by deficiency of phen
Slide 14: Enrollment complete in Phase 3 ACTIVATE-KidsT study
Enrollment complete in Phase 3 ACTIVATE-KidsT study aimed to support potential label expansion to PK deficiency patients under 18 Not Regularly Transfused PK Deficiency N=30 Regularly Transfused PK Deficiency N=45 Randomize 2:1 Randomize 2:1 Mitapivat Mitapivat Mitapivat Mitapivat OLE OLE placebo pl
Slide 15: Two global, Phase 3, randomized controlled trials of PYRUKYND in thalassemia
Two global, Phase 3, randomized controlled trials of PYRUKYND® in thalassemia encompass broad range of thalassemia patients N = 171 N = 240 2:1 randomization 2:1 randomization 100 mg BID Placebo BID 100 mg BID Placebo BID 24-week core period 48-week core period Open-label extension (up to 5 years) O
Slide 16: Commercial
1 2 Commercial 3 16
Slide 17: PYRUKYND Q3 2023 performance metrics highlight continued progress
PYRUKYND® Q3 2023 performance metrics highlight continued progress $7.4M net U.S. sales of PYRUKYND® a 10% increase over Q2 2023 160 unique patients completed PYRUKYND® prescription enrollment forms, a 9% increase over Q2 2023 100 patients on PYRUKYND®, which includes new prescriptions and those con
Slide 18: PK activation franchise positioned for meaningful expansion, with near-term opportunity in thalassemia
PK activation franchise positioned for meaningful expansion, with near-term opportunity in thalassemia PYRUKYND® is the first and only disease- modifying treatment approved for adults with PK deficiency ADDITIONAL OPPORTUNITIES Sickle cell disease FOR THE FRANCHISE WITH NOVEL Potential U.S. approval
Slide 19: Agios aims to deliver the first therapy approved for all thalassemia subtypes
Agios aims to deliver the first therapy approved for all thalassemia subtypes Thalassemia (~8k patients US) Alpha Thalassemia Beta Thalassemia Non-Transfusion Transfusion Non-Transfusion Transfusion Dependent Dependent Dependent Dependent Alpha Thalassemia Alpha Thalassemia Beta Thalassemia Beta Tha
Slide 20: US Thalassemia market has higher diagnosed prevalence and disease awareness compared to PK deficiency
US Thalassemia market has higher diagnosed prevalence and disease awareness compared to PK deficiency Patient Journey Continuum Prevalence Diagnosis Treatment Treatment Setting Patient Engagement • Ultra-rare • ~30% diagnosed • PYRUKYND only • Highly dispersed in • New advocacy FDA approved communit
Slide 21: Initial launch focus represents 65-70% of adult thalassemia patients
Initial launch focus represents 65-70% of adult thalassemia patients Focus Areas for Launch CoE Initial Top Affiliated Launch Practices Focus Community Hem/Onc Regularly monitored patients : 3+ Heme/Onc Visits Annually Expanded Community Hem/Onc Launch Not Regularly monitored patients: <3+ Heme/Onc
Slide 22: Financial
1 2 3 Financial 22
Slide 23: Third quarter 2023 financial results
Third quarter 2023 financial results Three Months Ended Three Months Ended Statement of Operations 9/30/23 9/30/22 PYRUKYND® Net Revenue $7.4M $3.5M Cost of Sales $0.6M $0.5M Research & Development Expense $81.8M $65.0M Selling, General & Administrative Expense $25.8M $29.1M Gain on Sale of Oncology
Slide 24: Closing Remarks
>< agios Closing Remarks
Slide 25: Building a leading hematology franchise
Building a leading hematology franchise Clinical Development Commercial Financial • Excellent execution, on track • Maximize the current launch • Flexibility enables continued to deliver six mid-to-late-stage in PK deficiency investment towards our vision data readouts by the end of to expand portfo
Slide 26: Q&A
Q&A 26
Agios Pharmaceuticals — Q3 2023 Earnings Presentation on 6ix