Syndax_JPM 2025_FINAL_.pdf

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1、Syndax Corporate Presentation43rdAnnual J.P.Morgan Healthcare ConferenceJanuary 14,2025Forward-looking statements disclosureThis presentation contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995.Words such asmay,will,expect,plan,anticipate an

2、d similar expressions(as well as other words or expressions referencing future events,progress,timing or circumstances)are intended to identify forward-looking statements.All statements other than statements of historical facts containedin this presentation,including statements regarding future oper

3、ations,financial results and the financial condition of Syndax Pharmaceuticals,Inc.(“Syndax”or the“Company”),including financial position,strategy and plans,the progress,timing,clinical development and scope ofclinical trials and the reporting of clinical data for Syndaxs product candidates,the prog

4、ress of regulatory submissions and approvals andsubsequent commercialization and the potential use of Syndaxs product candidates to treat various cancer indications and fibrotic diseases,andSyndaxs expectations for liquidity and future operations,are forward-looking statements.Many factors may cause

5、 differences between currentexpectations and actual results,including unexpected safety or efficacy data observed during preclinical studies or clinical trials,clinical siteactivation rates or clinical trial enrollment rates that are lower than expected;changes in expected or existing competition;th

6、e impact ofmacroeconomic conditions(the Russia-Ukraine war,inflation,among others)on Syndaxs business and that of the third parties on which Syndaxdepends,including delaying or otherwise disrupting Syndaxs clinical trials and preclinical studies,manufacturing and supply chain,or impairingemployee pr

7、oductivity;failure of our collaborators to support or advance collaborations or product candidates and unexpected litigation or otherdisputes.Moreover,Syndax operates in a very competitive and rapidly changing environment.Other factors that may cause our actual results todiffer from current expectat

8、ions are discussed in Syndaxs filings with the U.S.Securities and Exchange Commission,including the“Risk Factors”sections contained therein.New risks emerge from time to time.It is not possible for Syndaxs management to predict all risks,nor can Syndaxassess the impact of all factors on its business

9、 or the extent to which any factor,or combination of factors,may cause actual results to differmaterially from those contained in any forward-looking statement.In light of these risks,uncertainties and assumptions,the forward-lookingevents and circumstances discussed in this presentation may not occ

10、ur and actual results could differ materially and adversely from thoseanticipated or implied.Except as required by law,neither Syndax nor any other person assumes responsibility for the accuracy and completenessof the forward-looking statements.Syndax undertakes no obligation to update publicly any

11、forward-looking statements for any reason after thedate of this presentation to conform these statements to actual results or to changes in Syndaxs expectations.23Syndax is a commercial-stage oncology company with two first-in-class medicines with practice-changing and billion-dollar potentialSyndax

12、 delivered on multiple major milestones in 2024,includingtwo FDA approvals within 90 days of each otherLaunched first and onlymenin inhibitor for R/Racute leukemia with a KMT2A translocation in the U.S.Received FDA approval for first and only anti-CSF-1R antibody in chronic GVHD Proforma cash of$750

13、M*expected to fund thecompany to profitability Delivered first positive pivotal dataset in R/R mNPM1 AML with a menin inhibitorAdvanced clinical development programs designed to fuel pipeline expansion*Represents cash,cash equivalents and short-and long-term investments as of September 30,2024 pro f

14、orma,accounting for$350 million in gross proceeds received as a result of the royalty funding agreement with Royalty Pharma announced on November 4,2024;actual cash,cash equivalents and short-and long-term investments on hand at September 30,2024 was$399.6 million.4MENIN INHIBITIONA PROMISING NEW MO

15、DALITY FOR CERTAIN GENETICALLY-DEFINED ACUTE LEUKEMIASSyndax is positioned to lead in menin inhibition,an exciting new class with multi-billion-dollar opportunityUp to 50%of AML patients1-2have genetic alterations which may be susceptible to menin inhibition(e.g.,KMT2Ar,mNPM1,NUP98r)15,000 U.S.patie

16、ntscould potentially be addressed$4B+U.S.market opportunityacross R/R and frontlineOPPORTUNITYUNIQUE POSITIONINGFirst and only approved menin inhibitorPoised to be the first to deliver pivotal frontline combo dataDifferentiated product profile supported by positive pivotal data across the broadest p

17、opulation to dateNear-term opportunity for FDA approval and launch in R/R mNPM1 AMLKTM2Ar,KMT2A rearrangement;mNPM1,mutant NPM1;R/R,relapsed or refractory;1L,frontline;AML,acute myeloid leukemia 1.Issa,GC,et al.Therapeutic implications of menin inhibition in acute leukemias.Leukemia 35,2482 2495(202

18、1);2.Issa G.,et al.Clinical outcomes associated with NPM1 mutations in patients with relapsed or refractory AML.Blood Adv.2023;7(6):933-942 5Syndax is aggressively executing on its menin strategy designed to drive long-term growth6EXPAND APPROVAL INTO R/R mNPM1 AMLADVANCE INTO FRONTLINE SECURE FIRST

19、 MOVER ADVANTAGE1232024-20252025Future7Revuforj(revumenib)is the first and only FDA-approved menin inhibitor First mover advantage positions Syndax for long-term growth and successRevuforj is indicated for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene

20、(KMT2A)translocation in adult and pediatric patients one year and olderApproved for an aggressive form of R/R acute leukemia with an urgent unmet needPlease see full Prescribing Information,including BOXED WARNING,for complete details.Broad indication,including adults and peds 8Prior to launch,Synda

21、x reached 2,000 accounts where 98%of KMT2Ar patients receive treatment Key pre-launch activities have set the foundation for a successful U.S.Revuforj launchPre-launch activities focused on 200 accounts estimated to represent 2/3 of the opportunityNote:Bubble size corresponds to prevalent AML+ALL pa

22、tientsHired highly-experienced commercial teamwith average of 22 yrs of experience,primarily in hem/oncProfiled accountsto understand patient journey,treatment workflows and key stakeholdersEducated payers representing 90%of covered lives on urgent need in R/R KTM2Ar acute leukemiaBuilt dedicated pa

23、tient support program and distribution network,including leading specialty pharmaciesEstablished advanced data analyticsto enable identification of high-risk patients and targeted HCP engagement 8Added to NCCN Guidelines for AML and ALLLaunch boosted by strong presence/engagement at ASHReimbursement

24、 seen from all payer types9 9Drug in channel 5 days after approval with immediate orderingScripts received from institutions across the U.S.(major academic centers&community practices)EarlyMilestonesU.S.launch of Revuforj is underway with encouraging early progressHighly-experienced customer facing

25、teams are laser-focused on drivingbest-in-class patient and customer experience10EXPAND APPROVAL INTO R/R mNPM1 AMLADVANCE INTO FRONTLINESECURE FIRST MOVER ADVANTAGE1232024-20252025FutureSyndax is aggressively executing on its menin strategy designed to drive long-term growthSyndax is well positione

26、d to receive an indication in R/R mNPM1 AML,further securing our competitive advantageReported first positive pivotal data in R/R mNPM1 AML(N=64)ORR=47%and CR/CRh=23%in heavily pre-treated population(75%with prior ven)MRD negative CR/CRh=64%17%of responders proceeded to HSCTPublish pivotal data and

27、submit publication to NCCN GuidelinesPotential FDA approval in 2nd indication,further expanding indicated populations for revumenib Submit sNDA to FDANov 20241H 20251H 2025Dec 2024YE 2025Completed milestonesAnticipated milestonesReported consistent results in a larger population(N=77)ORR=48%;CR/CRh=

28、26%ORR,overall response rate;CR,complete remission;CRh,complete remission with partial hematological recovery;HSCT,hematopoietic stem cell transplantation1112EXPAND APPROVAL INTO R/R mNPM1 AMLADVANCE INTO FRONTLINE SECURE FIRST MOVER ADVANTAGE1232024-20252025FutureSyndax is aggressively executing on

29、 its menin strategy designed to drive long-term growthRev maintenanceRev+“7+3”Rapidly advancing our frontline strategy in KMT2Ar acute leukemia and mNPM1 AML13AUGMENT-102Rev+chemo SAVERev+ven/HMAINTERCEPTRev monotherapyBEAT AMLRev+ven/azaPh 1/2 AUGMENT-101Rev monotherapy Ph 2/3RELAPSED/REFRACTORYMAI

30、NTENANCEFRONTLINEHOVON TrialRev+ven/aza“UNFIT”“FIT”Strategic priorities:Addressing populations with greatest unmet needsSpeed(time to endpoints)Regular cadence of potentially practice-changing evidenceRobust pipeline of frontline combo trials to supportNCCN Guideline inclusion and/or label expansion

31、 Pivotal trial expected to initiate in 1Q25 Reported positive data Data expected in 2H25Rev maintenanceRev+I.C.Trials expected to initiate in 2025Revumenib clinical development programs(KMT2Ar or mNPM1 acute leukemias)across the treatment paradigmIC,intensive chemoSAVE and BEAT AML data highlight re

32、vumenibs promising combination potential14SAVE(MD Anderson Cancer Center)BEAT AML(LLS Beat AML Master Clinical Trial)PopulationRelapsed or refractorypediatric and adult AML or MPAL patients withmNPM1,KMT2Ar,or NUP98rNewly diagnosedpatients 60 years of age withmNPM1 or KMT2Ar AMLComboRevumenib,veneto

33、clax and decitabine/cedazuridineRevumenib,venetoclax and azacitidineEfficacyORR=82%(27/33);CR/CRh=48%(16/33)MRD negative rate=65%(17/26*)among responders39%(13/33)of patients proceeded to HSCTORR=100%(37/37);CRc=95%(35/37)MRD negative rate=95%(35/37)27%(10/37)of patients proceeded to HSCTSafetyCombi

34、nation was generally well toleratedRevumenib was generally well tolerated at the 113 mg and 163 mg q12h dose in combination with ven/azaNext stepsNow enrolling a cohort of newly diagnosed patientsEnrollment in the expansion cohort is ongoingat both dose levels1.DiNardo CD,et al.Azacitidine and Venet

35、oclax in Previously Untreated Acute Myeloid Leukemia.N Engl J Med.2020.HSCT,hematopoietic stem cell transplant;CRc,composite complete remission,ORR,overall response rate*One responding pt had inadequate MRD by MFC.Results from pivotal ven/aza trial in 1L unfit AML1:CRc=66%In the frontline unfit popu

36、lation,Syndax will initiate a pivotal trial of revumenib+ven/aza15Newly diagnosed adults with mNPM1 or KMT2Ar AML ineligible for intensive chemotherapy(I.C.)N 415Revumenib+ven/aza Placebo+ven/aza1:1A randomized,double-blind,placebo-controlled,clinical trial in collaboration with the HOVON networkTri

37、al targets a significant unmet need and is anticipated toreadout ahead of I.C.combo trialsIn the frontline fit population,an ongoing Ph 1 trial with I.C.is designed to identify the RP2D and support further developmentRevumenib+“7+3”Revumenib maintenance Newly diagnosed pts withmNPM1 or KMT2Ar acute

38、leukemias eligible for I.C.Primary Endpoint:Overall survival in mNPM1 patientsSecondary endpoints,including:Event-free survival,CR/CRh rate,rates of response without MRD,time to response,duration of responseHistorical vena/aza 1L results in unfit AML patients:Long-term outcomes are poor(mOS=14.7 mon

39、ths)and majority of patients will relapse1Data expected in 2H251.Pratz KW,et al.Long-term follow-up of VIALE-A:Venetoclax and azacitidine in chemotherapy-ineligible untreated acute myeloid leukemia.Am J Hematol.2024;99(4):615-624.RP2D,recommended Phase 2 dose15Syndax is well-positioned to lead in me

40、nin inhibition,a commercial opportunity estimated to exceed$4B in the U.S.alone16Market Opportunity($B)R/R Acute Leukemia with a KMT2A translocationR/R Acute Myeloid Leukemia with mNPM11L“Unfit”Acute Leukemia with KMT2Aror mNPM11L“Fit”Acute Leukemiawith KMT2Ar or mNPM1TotalEst.patient population2,00

41、03,000-4,5003,5005,500Source:Data on file;Redbook 2023$1B market$2B market$4B market$.75B market$.75-$1.25B market17CSF-1R INHIBITIONA NOVEL MECHANISM FOR TARGETING INFLAMMATION AND FIBROSIS IN cGVHD WITH POTENTIAL FOR EXPANSIONSyndax is aggressively executing on its strategy designed to drivelong-t

42、erm growth with Niktimvo18ADVANCE INTO EARLIER LINES OF cGVHD TREATMENTEXPAND INTO ADDITIONAL INDICATIONS,STARTING WITH IPFESTABLISH NIKTIMVOIN 3L cGVHD123FDA approved for treatment of chronic GVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing

43、 at least 40 kgIncluded in NCCN Guidelines Preparing for U.S.launch in early 1Q25,in partnership with IncyteIncyte-driven trials underway:Ph 2 frontline combo trial with Jakafi Ph 3 frontline combo trial with steroidsSyndax-driven trial underway:Ph 2 MAXPIRe trial in idiopathic pulmonary fibrosis(IP

44、F)topline data anticipated in 2026NCCN Clinical Practice Guidelines in Oncology(NCCN Guidelines)for Hematopoietic Cell Transplantation(HCT).Version 2.2024 August 30,2024.$350M Royalty Pharma deal highlights Niktimvos multi-billion-dollar potential19Upfront$350M payment in exchange for a 13.8%capped

45、royalty on U.S.net sales allows Syndax to maintain revenue and retain upsideVALUATION REFLECTS MULTIPLE FACTORSSignificant cGVHD Population 17,000 cGVHD patients in U.S.1 Nearly 50%require at least 3 lines of treatment2 Clinical trials underway could support advancement into earlier lines of treatme

46、ntHigh Unmet Need&Chronic Nature cGVHD is a debilitating,difficult-to-treat disease Complete responses are rare;many patients cycle through therapiesLarge,Growing Market 3L cGVHD drug is annualizing at$500M in U.S.sales within 3 years of launch3Differentiated Product Profile Novel MoA in cGVHD to ad

47、dress inflammation and fibrosis High response rates(ORR=75%)in heavily pretreated pts.Responses were rapid,durable&observed across all organs studiedStrong Commercial Partner&Synergies Advantages to partnership with Incyte,the leader in GVHD Overlapping call points with Revuforj targetsOpportunities

48、 Beyond cGVHD Preclinical and clinical data provide rationale for CSF-1R inhibition in IPF and potentially other diseases1.Internal data on file;2.Bachier CR,et al.Epidemiology and Treatment of Chronic Graft-versus-Host Disease Post-Allogeneic Hematopoietic Cell Transplantation:A US Claims Analysis.

49、Transplant Cell Ther.2021;3.Sanofi Third Quarter 2024 Results20LOOKING AHEAD21Expected upcoming milestonesRevuforj(revumenib)Menin-KMT2A inhibitionNiktimvo(axatilimab-csfr)CSF-1R inhibitionMaximize U.S.adoption of Revuforj as the preferred menin inhibitorSubmit sNDA in R/R mNPM1 AML in 1H25,with pot

50、ential approval around YE 2025Publish pivotal R/R mNPM1 AML data in 1H25 Initiate a pivotal frontline trial of revumenib with ven/aza in unfit mNPM1 or KMT2Ar acute leukemia patients in 1Q25 Report Ph 1 data from a frontline trial evaluating revumenib with I.C.(7+3)in 2H25Initiate multiple frontline

51、 trials evaluating revumenib in combination with I.C.,starting in 2025Present additional data at medical congresses from ongoing trials of revumenib in combination with SOC agentsLaunch Niktimvo in the U.S.in cGVHD in early 1Q25Complete enrollment in MAXPIRe Phase 2 IPF trial in 2025 with topline da

52、ta expected in 2026Syndax is positioned for long-term growth with two first-in-class therapies with multi-billion-dollar potentialSignificant lead in menin,an exciting new class supported by a growing mountainof clinical evidence Revuforj positioned for a unique launch trajectory with near-term pote

53、ntial for a second indicationPoised for success in cGVHD,with a differentiated product and commercial partnership with the leader in GVHDClear path to expanding Revuforj and Niktimvo into additional indications with large unmet needsStrong leadership team with a successful track record of execution

54、through development,approval and commercialization*Represents cash,cash equivalents and short-and long-term investments as of September 30,2024 pro forma,accounting for$350 million in gross proceeds received as a result of the royalty funding agreement with Royalty Pharma announced on November 4,202

55、4;actual cash,cash equivalents and short-and long-term investments on hand at September 30,2024 was$399.6 million.Well-capitalized with proformacash of$750M*expected to fundthe company to profitability 22Determined to realize a future in which people with cancer live longer and better than ever before.23