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1、1Fueled by Connections to Transform Rare DiseasesBrian Goff,Chief Executive OfficerJanuary 11,20232Forward-looking statementsThis presentation and various remarks we make during this presentation contain forward-looking statements within the meaning of The Private Securities Litigation Reform Act of
2、 1995.Such forward-looking statements include those regarding the potential benefits of PYRUKYND(mitapivat),AG-946 and its PAH stabilizer;Agios plans,strategies and expectations for its preclinical,clinical and commercial advancement of its drug development,including PYRUKYND,AG-946 and its PAH stab
3、ilizer;Agios strategic vision and goals,including its key milestones for 2023 and potential catalysts through 2026;and the potential benefits of Agios strategic plans and focus.The words“anticipate,”“expect,”“goal,”“hope,”“milestone,”“plan,”“potential,”“possible,”“strategy,”“will,”“vision,”and simil
4、ar expressions are intended to identify forward-looking statements,although not all forward-looking statements contain these identifying words.Such statements are subject to numerous important factors,risks and uncertainties that may cause actual events or results to differ materially from Agios cur
5、rent expectations and beliefs.For example,there can be no guarantee that any product candidate Agios is developing will successfully commence or complete necessary preclinical and clinical development phases,or that development of any of Agios product candidates will successfully continue.There can
6、be no guarantee that any positive developments in Agios business will result in stock price appreciation.Managements expectations and,therefore,any forward-looking statements in this presentation and various remarks we make during this presentation could also be affected by risks and uncertainties r
7、elating to a number of other important factors,including,without limitation:risks and uncertainties related to the impact of the COVID-19 pandemic to Agios business,operations,strategy,goals and anticipated milestones,including its ongoing and planned research activities,ability to conduct ongoing a
8、nd planned clinical trials,clinical supply of current or future drug candidates,commercial supply of current or future approved products,and launching,marketing and selling current or future approved products;Agios results of clinical trials and preclinical studies,including subsequent analysis of e
9、xisting data and new data received from ongoing and future studies;the content and timing of decisions made by the U.S.FDA,the EMA or other regulatory authorities,investigational review boards at clinical trial sites and publication review bodies;Agios ability to obtain and maintain requisite regula
10、tory approvals and to enroll patients in its planned clinical trials;unplanned cash requirements and expenditures;competitive factors;Agios ability to obtain,maintain and enforce patent and other intellectual property protection for any product candidates it is developing;Agios ability to maintain k
11、ey collaborations;the failure of Agios to receive milestone or royalty payments related to the sale of its oncology business,the uncertainty of the timing of any receipt of any such payments,and the uncertainty of the results and effectiveness of the use of proceeds from the transaction with Servier
12、;and general economic and market conditions.These and other risks are described in greater detail under the caption Risk Factors included in Agios public filings with the Securities and Exchange Commission.Any forward-looking statements contained in this presentation and various remarks we make duri
13、ng this presentation speak only as of the date hereof,and Agios expressly disclaims any obligation to update any forward-looking statements,whether as a result of new information,future events or otherwise,except as required by law.3Track record of success in discovering,developing and commercializi
14、ng therapiesUnmatched expertise in cellular metabolismThe Leader in Pyruvate Kinase(PK)Activation3DEVELOPED APPROVED THERAPIES IN ONCOLOGY2017&2018FIRST PYRUKYNDAPPROVAL ADULTS WITH PYRUVATE KINASE(PK)DEFICIENCY2022RARE DISEASE FOCUS:POTENTIAL APPROVALS IN THALASSEMIA AND SICKLE CELL DISEASEBy 20264
15、Leader in pyruvate kinase(PK)activation poised for significant growthCompelling and consistent data across connected diseasesRobust clinical data set supports potential of PK activation to transform patient function,quality of life,and long-term outcomesMeaningful commercial opportunities on the hor
16、izonFirst rare disease launch building capabilities to maximize anticipated franchise expansionPotential for two additional PYRUKYNDindications by 2026Well capitalized to advance and expandStrong cash position expected to support completion of ongoing programs and disciplined portfolio expansion5Ped
17、iatric PK DeficiencyNo approved therapy for pediatric PK deficiency patientsOur goal:Deliver the first approved therapy forpediatric PK deficiencyThalassemiaNo approved therapy for 60%of thalassemia patientsOur goal:Deliver the first therapy approved for all thalassemia subtypesSickle Cell DiseaseNo
18、 novel oral therapy improves anemia andreduces sickle cell pain crisesOur goal:Deliver a novel oral therapy that improves anemia and reduces VOCsLower-Risk MDSNo oral therapy addresses ineffective erythropoiesisOur goal:Deliver the first oral therapy that addresses ineffective erythropoiesisFocused
19、on expanding from PK deficiency to other diseases with shared pathophysiology,limited treatment options,and profound unmet needsPYRUKYND is approved in the U.S.,EU,and Great Britain for adult PK deficiency and is under investigation for pediatric PK deficiency,thalassemia,and sickle cell disease.AG-
20、946 is under investigation for LR-MDS.56ADDITIONAL OPPORTUNITIES FOR THE FRANCHISE WITH NOVEL PK ACTIVATOR AG-946PK deficiency Approved for adults in the U.S.and EUThalassemiaPotential U.S.approval in 2025Sicklecell disease Potential U.S.approval in 2026PK activation franchise positioned for meaning
21、ful expansion,with near-term opportunity in thalassemia3-8K patients in the U.S./EU5 18-23K patients in the U.S./EU5120-135K patients in the U.S./EU575-80K patientsin the U.S./EU5Source:Agios internal estimatesLower-risk myelodysplastic syndromeOrphan patient populationsHigh unmet needFocused prescr
22、iber poolDifferentiated product profilePYRUKYND is the first and only disease-modifying treatment approved for adults with PK deficiencyPotential for two additional PYRUKYND indications by 20267RESEARCHEARLY-STAGE CLINICAL DEVELOPMENTLATE-STAGE CLINICAL DEVELOPMENTREGULATORY SUBMISSIONAPPROVALPyruva
23、te Kinase Deficiency-and-ThalassemiaACTIVATE KidsACTIVATE KidsTENERGIZEENERGIZE-TSickle Cell Disease*Myelodysplastic Syndrome(MDS)Healthy Volunteers/Sickle Cell DiseasePhenylketonuria(PKU)RISE UPPHASE 2PHASE 1PYRUKYNDFirst-in-class PK activatorAG-946Novel PK activatorPhenylalanine hydroxylate(PAH)st
24、abilizerUS,EU,GBBuilding a diverse pipeline leveraging our expertise in cellular metabolism*In addition to RISE UP,two investigator-sponsored trials are ongoing with the NIH and University of Utrecht.8Compelling and consistent data across connected diseasesPK activation may address a range of hemoly
25、tic and acquired anemias underpinned by shared pathophysiology9Energy imbalance in red blood cells can lead to severe hematological diseaseInsufficient ATP productionLeads to red blood cell metabolic stress,anemia,and reduced patient quality of lifeEnergy supplyEnergy demandHealthy Red Blood CellATP
26、 production meets demandPK DeficiencyOther AnemiasThalassemia,Sickle Cell Disease,Lower-Risk MDS10Chronic HemolysisDestruction of red blood cells Ineffective ErythropoiesisDecreased output of red blood cellsDisease areas share common pathophysiology and severely impact quality of life ANEMIATRANSFUS
27、ION BURDENIRON OVERLOADREDUCED BONE MINERAL DENSITYLONG-TERM ORGAN DAMAGESignificant near and long-term clinical consequences of chronic hemolysis and ineffective erythropoiesisCHRONIC FATIGUE,SUSCEPTIBILITY TO ILLNESSCHALLENGES WITH BASIC SOCIAL,SCHOOL/WORK ACTIVITIESIMPACT ON EMOTIONAL AND MENTAL
28、HEALTHPAIN AND FRACTURESECONOMIC BURDEN.that lead to major implications for patient quality of life and how they feel and function.PK DeficiencySickle Cell DiseaseThalassemiaMDS Associated AnemiaRed Blood Cell Metabolic Stress11Phase 3 ENERGIZE and ENERGIZE-T studiesPhase 2 long-term extension study
29、Phase 3 long-term extension studyPhase 2/3 RISE UP studyInvestigator-sponsored trialsConsistency of clinical data to date supports potential of PYRUKYNDto address unmet patient needs across hemolytic anemias1.Al-Samkari et al.N Engl J Med 2022;386:1432-1442;2.Glenthojet al.The Lancet Haematology,202
30、2,Vol 9,e724-e732;3.Van Beers et al.ASH 2021:Abstract 757;4.Al-Samakri et al.ASH 2021.Abstract 924;5.Kuo et al.ASH 2022:Abstract 506;6.Van Beers et al.ASH 2022:Abstract 1021;7.Grace et al.ASH 2022:Abstract 2328;8.Kuo KHM et al.Lancet 2022;400:10351;9.Kuo KHM et al.ASH 2021:Abstract 576;10.Kuo KHM et
31、 al.ASH 2022:Abstract 1030;11.Van Dijk MJ et al.Am J Hematol.2022;97:E226;12.Xu JZ et al.Blood.2022;140:2053;13.Van Dijk MJ et al.EHA 2022:Abstract P1501.Thalassemia(Wild-type PK enzyme)Data through Phase 2Adult PK Deficiency(Mutant PK enzyme)Approved in the U.S.,EU and Great BritainSickle Cell Dise
32、ase(Wild-type PK enzyme)Data through Phase 1 and ISTsHemoglobin improvementHemolysis improvementErythropoiesis improvementTransfusion reductionPRO improvementsSickling reductionONGOING STUDYONGOING STUDIESONGOING STUDIES12Emerging Long-term DataThalassemia(Wild-type PK enzyme)Data through Phase 2Adu
33、lt PK Deficiency(Mutant PK enzyme)Approved in the U.S.,EU and Great BritainSickle Cell Disease(Wild-type PK enzyme)Data through Phase 1 and ISTsConsistency of clinical data to date supports potential of PYRUKYNDto address unmet patient needs across hemolytic anemiasHemoglobin improvementHemolysis im
34、provementErythropoiesis improvementPRO improvementsSickling reductionTransfusion reductionBone health stabilizationIron overload reductionLength of exposureOVER 7 YEARSOVER 4.5 YEARSOVER 2 YEARS1.Al-Samkari et al.N Engl J Med 2022;386:1432-1442;2.Glenthojet al.The Lancet Haematology,2022,Vol 9,e724-
35、e732;3.Van Beers et al.ASH 2021:Abstract 757;4.Al-Samakri et al.ASH 2021.Abstract 924;5.Kuo et al.ASH 2022:Abstract 506;6.Van Beers et al.ASH 2022:Abstract 1021;7.Grace et al.ASH 2022:Abstract 2328;8.Kuo KHM et al.Lancet 2022;400:10351;9.Kuo KHM et al.ASH 2021:Abstract 576;10.Kuo KHM et al.ASH 2022:
36、Abstract 1030;11.Van Dijk MJ et al.Am J Hematol.2022;97:E226;12.Xu JZ et al.Blood.2022;140:2053;13.Van Dijk MJ et al.EHA 2022:Abstract P1501.13Meaningful commercial opportunities on the horizonFDA approval and launch of PYRUKYND in PK deficiency builds capabilities to maximize potential product expa
37、nsion14Optimizing Patient&Provider JourneyPYRUKYNDlaunch in PK deficiency building commercial capabilities to support potential expansion in meaningfully larger patient populationsTherapy OnboardingAccess&AdherenceDiagnostic EfficiencyAwareness&EducationCoverage support,free product eligibility,copa
38、y programFree genetic testing to help confirm diagnosis of hereditary anemiasPK deficiency patient identification via claims-based targetingActivate physicians to prescribe and eligible patients to advocate for treatment Drive awareness and urgency to manage PK deficiency among providers and patient
39、s15High unmet need across patient segments and geographies18-23K Patients in U.S./EU5Significant opportunity outside U.S./EU5Thalassemia:no approved therapies for 60%of patientsBeta-THAL prevalence:HEOR Global THAL Epidemiology SLE(XCENDA,2021);US:Paramore,et.al;DE:Borchert,et.al;IT:Italian Society
40、of Thal&Hemoglobinopathies Patient Registry,Jan 2021,Angelucci,et.al,2017;FR:French registry for thal(Thuret,et.al.);ES:Cela,et.al.;UK Registry for Hemoglobinopathies,2020;Alpha-THAL prevalence:Agios internal estimates;LEK Analysis|Beta-THAL TD/NTD split(60%/40%):Thuret,et.al.,Haematologica2010;Magn
41、olia TPP MR,April 2020|Alpha-THAL TD/NTD split(5%/95%):Taher,et.al.,Vox Sanguinis,2015;Magnolia TPP MR,April 2020.PYRUKYND is under investigation for thalassemia and is not approved anywhere for that use.PYRUKYND Address full range of thalassemia patients Chronic therapy Oral Improved Hb and reduced
42、 transfusion burden Improved ineffective erythropoiesis Safety profile consistent with prior clinical experienceTARGET PROFILE30%ALPHA-THALASSEMIANON-TRANSFUSION DEPENDENT40%BETA-THALASSEMIATRANSFUSION DEPENDENT28%BETA-THALASSEMIANON-TRANSFUSION DEPENDENT2%ALPHA-THALASSEMIATRANSFUSIONDEPENDENTNO AVA
43、ILABLE THERAPIES60%16PYRUKYND Chronic Therapy Oral Improved hemolytic anemia Improvement in sickle cell pain crises(vaso-occlusive crises;VOCs)QoL data Safety profile consistent with prior clinical experienceTARGET PROFILESickle Cell Disease:no novel oral therapy improves anemia and reduces sickle c
44、ell pain crisesPYRUKYNDis under investigation for sickle cell disease and is not approved anywhere for that use.Estimated 120-135K patients across the U.S.&EU5 Significant opportunity outside of U.S./EU Deliver a novel oral therapy that improves anemia,reduces VOCs and is supported by the largest bo
45、dy of clinical evidence Innovative seamless Phase 2/3 trial RISE UP developed with community input Global approach to clinical development Connections with SCD patient and physician communitiesGlobalReachPYRUKYNDOpportunityCritical Success Factors 17Well capitalized to advance and expandLong-term gr
46、owth and value creation fueled by accomplished leadership team and strong balance sheet18Focused on rare diseases rooted in cellular metabolismStrong cash position expected to support portfolio advancement and expansionPYRUKYND First and only disease-modifying treatment for adults with PK deficiency
47、 Potential for two additional indications by 2026AG-946 Potential to be first oral therapy to address the underlying cause of ineffective erythropoiesis in MDSLEAD RESEARCH PROGRAM PAH stabilizer for the treatment of PKU 15-20K patients in the U.S.;20K in the EU5 Target to file IND by year-end 2023D
48、ISCIPLINED BD STRATEGY Prioritize opportunities based on:Rare disease focus Transformative for patients Identified regulatory pathway Potential to de-risk early Clear path to value creationCLINICAL-STAGE PIPELINEEARLIER-STAGE PIPELINEBUSINESS DEVELOPMENTDeliver transformative therapeutics for patien
49、t communities with profound unmet need*PAH:phenylalanine hydroxylate;PKU:Phenylketonuria 192023 and BeyondClinical development strategy driving toward broader commercial opportunities20Clinical and regulatory milestones targeted in 2023 lay the foundation for transformational data readoutsBuild comm
50、ercial capabilities to efficiently launch additional indications and evaluate business development opportunities to expand pipelineThalassemiaPYRUKYNDPediatric PK DeficiencyPYRUKYNDSickle Cell DiseasePYRUKYNDLower-Risk MDSAG-946(Novel PK Activator)Complete enrollment of Phase 2a study(year-end)Compl
51、ete enrollment of Phase 3 ENERGIZE and ENERGIZE-T studies(mid-year)Phase 2 RISE UP data readout&go/no-go to Phase 3 decision(mid-year)Enroll at least half of patients in the Phase 3 ACTIVATE-kids and ACTIVATE-kidsT studies(year-end)Earlier-stage PipelineFile IND for PAH stabilizer for the treatment
52、of PKU(year-end)21Potential for two additional PYRUKYNDindications by 2026ThalassemiaPYRUKYNDPediatric PK DeficiencyPYRUKYNDSickle Cell DiseasePYRUKYNDLower-Risk MDSAG-946(Novel PK Activator)Phase 3 ACTIVATE-kids and ACTIVATE-kidsT readoutsPhase 3 ENERGIZE(1H)and ENERGIZE-T(2H)readouts Potential app
53、rovalPotential approvalPotential approvalPhase 2a readout202420252026Potential Phase 3 RISE UP readout*Pending Go/NoGo decision in 202322Differentiated Capabilities&Cross-functional ConnectivityHematology franchise spanning 3 hemolytic anemiasExpanded portfolio aligned with our core expertiseCash flow positive2026 VISIONDriven to transform patient outcomes in rare diseases2223Q&A