Agios Pharmaceuticals, Inc. · NASDAQ:AGIO
Agios Pharmaceuticals — Q1 2023 Earnings Presentation
24 slides from Agios Pharmaceuticals's earnings presentation, May 4, 2023
Slide 1: Q1 2023 Financial Results
Q1 2023 Financial Results May 4, 2023 1
Slide 2: Agios conference call participants
Agios conference call participants TOPIC PARTICIPANT Introduction Cecilia Jones, Chief Financial Officer Business Update Brian Goff, Chief Executive Officer Sarah Gheuens, M.D., Ph.D., Chief Medical Officer, Research & Development Update Head of Research and Development Commercial Update Tsveta Mila
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
Opening Remarks 4
Slide 5: Q1 2023 highlights
Q1 2023 highlights Pipeline updates Corporate updates • Closed screening of Phase 3 ENERGIZE and • $1.0B in cash, cash equivalents, ENERGIZE-T clinical trials in thalassemia; on and marketable securities as of track for data readouts in 2024 March 31, 2023 • On track to announce data readout of Phas
Slide 6: 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® PYRUKYND® Enroll at least half of patients Lower-Risk MDS Complete enrollment of Phase 3 in the Phase 3 ACTIVATE-kids AG-946 (Novel PK Activator) EN
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 Potential approval PYRUKYND® (2H) readouts Pediatric PK Phase 3 ACTIVATE- Deficiency kids and ACTIVATE- Potential approval kidsT readouts PYRUKYND® Sickle Cell Disease Potential
Slide 8: Clinical
Clinical Commercial Financial 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 ACTIVATE KidsT α- and β-Thalassemia ENERGIZE ENERGIZE-T Sickle Cell
Slide 10: 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 11: RISE UP Phase 2/3 operationally seamless trial of PYRUKYND in sickle cell disease
RISE UP Phase 2/3 operationally seamless trial of PYRUKYND® in sickle cell disease allows for speed and flexibility of clinical program Evaluate totality of available data & external environment to trigger Phase 3 including: Phase 2 PHASE 2 PRIMARY ENDPOINTS Phase 3 • 1:1:1 randomization to Hemoglob
Slide 12: Mitapivat development program in pediatric PK deficiency
Mitapivat development program in pediatric PK deficiency to support potential label expansion to those 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 placebo 5 years plac
Slide 13: Novel PK activator AG-946 provides opportunity to build on PYRUKYND franchise
Novel PK activator AG-946 provides opportunity to build on PYRUKYND® franchise and pursue multiple paths in parallel if data support advancement PHASE 1 HEALTHY VOLUNTEER DATA SUPPORT AG-946 PROFILE: SICKLE CELL DISEASE SICKLE CELL DISEASE OR PHASE 1 COHORT OTHER HEMOLYTIC ANEMIA Novel, highly poten
Slide 14: Lead research program aimed to address phenylketonuria (PKU)
Lead research program aimed to address 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 phenylalanine hydr
Slide 15: Commercial
Clinical Commercial Financial 15
Slide 16: Implementing a comprehensive commercial strategy that addresses each stage of the patient journey
Implementing a comprehensive commercial strategy that addresses each stage of the patient journey Awareness and Access and Adherence and Education Initiation Persistency Increase disease Accelerate access by Support adherence awareness and reducing the time and maintain educate on available between
Slide 17: PYRUKYND Q1 2023 performance metrics highlight continued progress
PYRUKYND® Q1 2023 performance metrics highlight continued progress $5.6M net U.S. sales of PYRUKYND® for fourth full quarter of launch 127 unique patients completed PYRUKYND® prescription enrollment forms, a 21% increase over Q4 2022 89 patients on PYRUKYND®, which includes new prescriptions and tho
Slide 18: PK activation franchise positioned for meaningful expansion
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 Sickle cell disease ADDITIONAL OPPORTUNITIES 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 TARGET PROFILE PYRUKYND® 60% • Address full range of thalassemia ~30% NO APPROVED patients ALPHA-THALASSEMIA THERAPIES IN THE U.S. ~40% NON-TRANSFUSION • Chronic therapy BETA-THALASSEMIA DEPENDENT • Oral TRANSFUSION DEPEND
Slide 20: Thalassemia market is concentrated in select geographies
Thalassemia market is concentrated in select geographies PYRUKYND® has the potential to become the first oral therapy to improve hemolytic anemia and ineffective erythropoiesis across all thalassemia subtypes ~70k PATIENTS ~13k ~8k PATIENTS PATIENTS U.S. EU5 GCC GCC: Kingdom of Saudi Arabia, Oman, U
Slide 21: Financial
Clinical Commercial Financial 21
Slide 22: First quarter 2023 financial results
First quarter 2023 financial results Three Months Ended 3/31/23 Three Months Ended Statement of Operations 3/31/22 PYRUKYND® Net Revenue $5.6M $0.8M Cost of Sales $0.6M $0.3M Research & Development Expense $67.3M $70.1M Selling, General & Administrative Expense $28.4M $31.5M Gain on Sale of Oncology
Slide 23: Closing Remarks
ZS agios Closing Remarks
Slide 24: Q&A
Q&A 24
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