ascendis 2025 JPM Deck - Final version 01.12.25.pdf

1、J.P.Morgan Healthcare ConferenceSan FranciscoJanuary 2025Ascendis Pharma A/SFor investor communication only.Not for use in product promotion.Not for further distribution.This presentation contains forward-looking statements.All statements other than statements of historical facts contained in this p

2、resentation,such as statements regarding our products and prospective product candidates;revenue and growth expectations;PDUFA goal dates;clinical trial results;the expected timing of future clinical trial results,regulatory filings and feedback from regulatory authorities;the scope,progress,results

3、 and costs of developing our product candidates or any other future product candidates;timing and likelihood of success;patient enrollment;timing and expansion of commercial launches,pipelines,and investments;potential payments and royalties relating to investments and partnerships;plans and objecti

4、ves of management for future operations and commercialization and manufacturing activities;and future results of current and/or anticipated products and product candidates are forward-looking statements.These forward-looking statements are based on our current expectations and beliefs,as well as ass

5、umptions concerning future events.These statements involve known and unknown risks,uncertainties and other factors that could cause our actual results to differ materially from the results discussed in the forward-looking statements.These risks,uncertainties and other factors are more fully describe

6、d in our reports filed with or submitted to the Securities and Exchange Commission(“SEC”),including,without limitation,our prospectus supplement filed on September 20,2024 and our current and future reports filed with or submitted to the SEC,including our most recent Annual Report on Form 20-F filed

7、 with the SEC on February 7,2024,particularly in the sections titled“Risk Factors”and“Operating and Financial Review and Prospects.”In light of the significant uncertainties in our forward-looking statements,you should not place undue reliance on these statements or regard these statements as a repr

8、esentation or warranty by us or any other person that we will achieve our objectives and plans in any specified timeframe,or at all.Any forward-looking statement made by us in this presentation speaks only as of the date of this presentation and represents our estimates and assumptions only as of th

9、e date of this presentation.Except as required by law,we assume no obligation to update these statements publicly,whether as a result of new information,future events,changed circumstances or otherwise after the date of this presentation.This presentation concerns Ascendis Pharma approved products a

10、s well as Ascendis Pharma investigational product candidates that are or have been under clinical investigation and which have not yet been approved for marketing by the U.S.Food and Drug Administration,European Medicines Agency or other foreign regulatory authorities.These investigational product c

11、andidates are currently limited by law to investigational use,and no representations are made as to their safety or effectiveness for the purposes for which they are being investigated.This presentation is for investor and insurance carrier communications only.Not for use in product promotion.This p

12、resentation also contains estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry.This data involves a number of assumptions and limitations,and you are cautioned not to give undue weight to such data and estimates.In addit

13、ion,projections,assumptions and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and risk.This presentation contains trademarks,services marks,trade names and copyrights of the company and other

14、companies,which are the property of their respective owners.The use or display of third parties trademarks,service marks,trade name or products in this presentation is not intended to,and does not imply,a relationship with the company,or an endorsement of sponsorship by the company.Cautionary Note o

15、n Forward-Looking Statements2For investor communication only.Not for use in product promotion.Not for further distribution.Three clinically validated Endocrinology Rare Disease medicines:SKYTROFA Approved for pediatric growth hormone deficiency(GHD)1-Full-year 2024 SKYTROFA revenue expected to be 20

16、2 million2,excluding sales deductions related to prior years-sBLA submitted for the treatment of adults with GHD,with a PDUFA goal date of July 27,2025 YORVIPATH Approved for the treatment of hypoparathyroidism in adults1-Launched January 2024 in Germany/Austria;available in the U.S.since late Decem

17、ber 2024-Full-year 2024 YORVIPATH revenue expected to be 29 million3 TransCon CNP Successful pivotal data in achondroplasia-Following pre-NDA meeting,planned filings for treatment of children with achondroplasia in U.S.Q1 2025,EU in Q3 2025 Expanding TransCon technology platform with broad therapeut

18、ic applicability Formed Eyconis for Ophthalmology and partnered with Novo Nordisk for Metabolic and Cardiovascular diseases Internal development for Oncology with focus on TransCon IL-2 New TransCon protein degrader technology to expand pipeline with additional potential blockbusters 3Ascendis Posit

19、ioned to Drive Rapid Revenue Growth1.Approved in the U.S.,EU,and other territories,including Norway,Iceland,Liechtenstein,and Great Britain(covering England,Wales,Scotland).2.Calculated as unaudited preliminary estimate of full year 2024 SKYTROFA revenue of 197 million plus 5 million of sales deduct

20、ions related to prior years.3.Unaudited preliminary estimate.For investor communication only.Not for use in product promotion.Not for further distribution.Business model built on fast,successful drug development and commercial therapeutic synergiesVision 20304For investor communication only.Not for

21、use in product promotion.Not for further distribution.Outperform industry drug development benchmarks with Ascendis product innovation algorithm Remain independent as a profitable biopharma through lean and flexible ways of working Let our values Patients,Science,Passion drive our decisions to succe

22、ss Obtain accelerated approval in Oncology with registrational trials ongoing Pursue TransCon product opportunities in$5B indications Maximize value creation of these product opportunities through collaboration with therapeutic area market leadersAchieve blockbuster status for multiple products and

23、expand our engine for future innovation Achieve blockbuster status($1B)for each of TransCon PTH,TransCon hGH,and TransCon CNP through worldwide commercialization Be the leader in Growth Disorders and Hypoparathyroidism,pursuing clinical conditions,innovative LCM,and complementary patient offerings E

24、xpand pipeline with Endocrinology Rare Disease blockbuster product opportunitiesBe the Leading Endocrinology Rare Disease CompanyCreate Value in Additional Therapeutic Areas through Innovative Business ModelsDifferentiate with Ascendis FundamentalsAscendis Pharmas 2025-2030 strategic roadmap5Overvie

25、w of TransCon TechnologiesAromaticCyclic ImideDKPCarbamateBicinAEGPyroglutamateTransCon Prodrug:3 componentsTransCon LinkerAntibodies,Antibody Fragments,Proteins,Peptides and Small MoleculesSoluble CarriersInsoluble CarriersContinued expansion of TransCon technologies,enabling new product candidates

26、Albumin AvidityProtein DegradersTransCon CarrierParent DrugFor investor communication only.Not for use in product promotion.Not for further distribution.TransCon Protein Degrader Technology6Enabling product opportunities for diseases characterized by excess hormones,cytokines,etc.For investor commun

27、ication only.Not for use in product promotion.Not for further distribution.Linker cleavage dependent upon pH and temperatureTransCon Protein Degrader(long half-life)Protein Degrader(short half-life)Liver rapidly binds,internalizes and degrades the degrader-ligand complexLigand to be degraded TransCo

28、n protein degrader technology designed to enable efficient clearance of hormones,cytokines,and other targets by utilizing hepatic scavenger receptorsEndocrinology Rare DiseasesIndicationStatusRegionLead IndicationTransCon CNP Achondroplasia(children aged 211)Pivotal1 MultinationalLabel ExpansionTran

29、sCon hGH Adult Growth Hormone Deficiency sBLA submitted2 Multinational Turner Syndrome(children aged 110)Phase 23 U.S.TransCon CNP Achondroplasia(infants)Phase 24 MultinationalTransCon CNP Achondroplasia(adolescents)Phase 25 MultinationalTransCon CNP+TransCon hGH Achondroplasia(children aged 211)Pha

30、se 26 MultinationalPartner ProgramsTransCon hGH Pediatric GHD BLA submitted7 ChinaTransCon hGH Pediatric GHD Phase 38 JapanTransCon PTH Hypoparathyroidism in adults Phase 39 ChinaTransCon PTH Hypoparathyroidism in adults J-NDA submitted10 JapanTransCon CNP Achondroplasia Phase 211 ChinaOncologyIndic

31、ationIndicationStatusStatusRegionRegionLead IndicationTransCon IL-2 Various tumor types Phase 212,13 MultinationalTransCon Clinical Development Pipeline1.Pivotal ApproaCH Trial(NCT05598320).2.sBLA submitted to U.S.FDA,PDUFA goal date July 27th 2025.3.New InsiGHTS Trial(NCT05690386).4.reACHin Trial(N

32、CT06079398).5.teACH(NCT06732895).6.COACH Trial(NCT06433557).7.VISEN Pharmaceuticals Phase 3 trial.8.Japanese riGHt Trial.9.PaTHway China.10.PaTHway Japan.11.ACcomplisH China.12.BelieveIT-201 Trial(NCT05980598).13.IL-Believe Trial(NCT05081609).7For investor communication only.Not for use in product p

33、romotion.Not for further distribution.Growing Worldwide Commercialization in Endocrinology Rare Disease1Multiple approaches:Ascendis direct commercialization in the U.S.and selectEuropean countries Through partners with local expertise and infrastructure outside of U.S.and Europe Direct 2 1.As of De

34、cember 31,2024.2.DACH,France&BeNeLux,Iberia,Italy,Nordics,UK&Ireland.8For investor communication only.Not for use in product promotion.Not for further distribution.Ascendis direct commercializationExclusive distribution agreementExclusive license agreementStrategic investment and exclusive license a

35、greementSKYTROFATransCon hGHOnce-weekly growth hormone therapyFor investor communication only.Not for use in product promotion.Not for further distribution.SKYTROFA-High Value Growth Hormone BrandSKYTROFA Revenue(million)10For investor communication only.Not for use in product promotion.Not for furt

36、her distribution.sBLA=supplemental Biologics License Application.1.Calculated as unaudited preliminary estimate of full year 2024 SKYTROFA revenue of 197 million plus 5 million of sales deductions related to prior years.2.Planned for small for gestational age without catch-up growth“SGA”;Idiopathic

37、short stature“ISS”;SHOX deficiency(including Turner syndrome).Launched for pediatric growth hormone deficiency in U.S.in Q4 21,Germany in Q3 23 Successful Phase 3 riGHt Trial in Japan Planned expansion across multiple countries Planned label expansion to drive growth sBLA accepted for adult growth h

38、ormone deficiency;PDUFA goal date of July 27,2025 Positive topline results from Phase 2 New InsiGHTS Trial in Turner syndrome Submit IND or similar for a basket trial evaluating additional indications2 in Q3 2025Committed to making SKYTROFA the global leading product in value in a growing growth hor

39、mone market 1 36 179 202 1-25 50 75 100 125 150 175 200 225 2021202220232024ETransCon hGH Program Summary and Outlook 2024 SKYTROFA revenue,with single indication in pediatric GHD,expected to be 202 million1 High-value brand in the U.S.,revenue per patient of around 3 times that of daily growth horm

40、one Volume(mg)increased 84%in 2024 resulting in 6.5%market share of total U.S.growth hormone market2 Adult GHD sBLA submitted to FDA;PDUFA goal date of July 27,2025 U.S.commercial launch planned in Q4 2025,pending approval Turner syndrome Phase 2 New InsiGHTS Trial achieved primary objective at Week

41、 26 Plan to submit IND or similar for basket trial evaluating additional indications3 in Q3 2025 Planned commercial launches across multiple indications and countries 111.Calculated as unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of 197 million plus 5 million of sales deductions

42、 related to prior years.2.Based on third party prescription data for 2024.3.Planned for small for gestational age without catch-up growth“SGA”;Idiopathic short stature“ISS”;SHOX deficiency(including Turner syndrome).For investor communication only.Not for use in product promotion.Not for further dis

43、tribution.Committed to making SKYTROFA a blockbuster productYORVIPATHTransCon PTHTreatment of hypoparathyroidism in adultsFor investor communication only.Not for use in product promotion.Not for further distribution.Myopathy Spondyloarthropathy Dry Skin Thinning Hair Brittle Nails Altered Tooth Morp

44、hology Cataracts Papilledema Anxiety&Depression Cognitive Impairment(“Brain Fog”)Paresthesia Muscle Cramps Pain Tetany Nephrocalcinosis*Nephrolithiasis*Chronic Kidney Disease*Vascular Calcifications Cardiac Arrhythmias Hypocalcemia-associated Dilated Cardiomyopathy Laryngospasm Seizures Basal Gangli

45、a Calcifications Parkinsonism Or DystoniaUnmet Medical Need in Hypoparathyroidism:One Disease,Multiple Consequences1-4Central Nervous SystemOphthalmologicalPeripheral Nervous SystemCardiovascularDermatologicalRenalNeuropsychiatricMusculoskeletalDentalRespiratoryAbsence or deficiency of parathyroid h

46、ormone(PTH)is linked to multi-organ manifestations5,6*These manifestations are mostly the result of management with active vitamin D and calcium rather than of the disease itself.1.Underbjerg L,Sikjaer T,Mosekilde L,et al.Cardiovascular and renal complications to postsurgical hypoparathyroidism:a Da

47、nish nationwide controlled historic follow-up study.J Bone Miner Res.2013;28(11):2277-2285.doi:10.1002/jbmr.1979.2.Underbjerg L,Sikjaer T,Mosekilde L,et al.The epidemiology of nonsurgical hypoparathyroidism in Denmark:a nationwide case finding study.J Bone Miner Res.2015;30(9):1738-1744.doi:10.1002/

48、jbmr.2501.3.Shoback DM,Bilezikian JP,Costa AG,et al.Presentation of hypoparathyroidism:etiologies and clinical features.J Clin Endocrinol Metab.2016;101(6):2300-2312.doi:10.1210/jc.2015-3909.4.Underbjerg L,Sikjaer T,Mosekilde L,et al.The epidemiology of nonsurgical hypoparathyroidism in Denmark:a na

49、tionwide case finding study.J Bone Miner Res.2015;30(9):1738-1744.doi:10.1002/jbmr.2501.5.Mannstadt M,Bilezikian JP,Thakker RV,et al.Hypoparathyroidism.Nat Rev Dis Primers.2017;3:17055.doi:10.1038/nrdp.2017.55.6.Shoback DM,Bilezikian JP,Costa AG,et al.Presentation of hypoparathyroidism:etiologies an

50、d clinical features.J Clin Endocrinol Metab.2016;101(6):2300-2312.doi:10.1210/jc.2015-3909.For investor communication only.Not for use in product promotion.Not for further distribution.13 Consider PTH replacement therapy in patients not adequately controlled on conventional therapy Inadequate contro

51、l is considered to be any one of the following:Symptomatic hypocalcemia Hyperphosphatemia Renal insufficiency Hypercalciuria Poor quality of life In addition,individuals with poor compliance,malabsorption,or who are intolerant of large doses of calcium and active vitamin D may also benefit from PTH

52、replacement therapyKhan AA,Bilezikian JP,Brandi ML,et al.Evaluation and Management of Hypoparathyroidism Summary Statement and Guidelines from the Second International Workshop.J Bone Miner Res.Dec 2022;37(12):2568-2585.doi:10.1002/jbmr.4691For investor communication only.Not for use in product prom

53、otion.Not for further distribution.Current Clinical Practice Guideline14Vast majority of patients with hypoparathyroidism fulfill guideline for PTH replacement therapyYORVIPATH Commercial UpdateYORVIPATH on track for additional commercial launches in Europe Direct and International Markets in 2025YO

54、RVIPATH Revenue(million)For investor communication only.Not for use in product promotion.Not for further distribution.15 1.5 5.2 8.5 13.6 1-5.0 10.0 15.0 Q1-24Q2-24Q3-24Q4-24E Europe Direct Commercial launch in Germany and Austria in January 2024,with initial list price of 105,000 per patient per ye

55、ar Outside Germany and Austria,providing commercial product through early access routes,such as named patient,until commercial reimbursement established Expect multiple commercial launches across Europe in 2025 International Markets 8 exclusive distribution agreements signed for 50+countries Revenue

56、 recognized from Er-Kim(Central&Eastern Europe),Neopharm(Israel),and Canada 700 patients on YORVIPATH treatment in Europe Direct and International Markets at the end of 20241Unaudited preliminary estimate.YORVIPATH is the first and only product indicated for the treatment of hypoparathyroidism in ad

57、ults1 Commercially available in the U.S.December 2024YORVIPATH U.S.FDA Approved and Now Commercially Available1.Limitations of Use:YORVIPATH was not studied for acute post-surgical hypoparathyroidism.YORVIPATHs titration scheme was only evaluated in adults who first achieved an albumin-corrected ser

58、um calcium of at least 7.8 mg/dL using calcium and active vitamin D treatment.YORVIPATH package insert.Princeton,NJ:Ascendis Pharma Endocrinology,Inc.August 2024.For investor communication only.Not for use in product promotion.Not for further distribution.16Hypoparathyroidism:U.S.Patient Population

59、WAC=Wholesale Acquisition Cost.1.U.S.prevalence literature review(Powers,Clarke).2.Internal estimates and Symphony Metys data.3.U.S.prevalence literature review and epi meta-analysis(Powers,Clarke,Milliman project,ipm.ai claims project;HCUPnet,Healthcare Cost and Utilization Project.Agency for Healt

60、hcare Research and Quality,Rockville,MD for surgical cohort projection).YORVIPATH annual WAC price of$285,00025k32k4,0005,000 2 Patients previously treated with PTH PTH Experienced25k32k70,00090,000 1,3 Currently on active vitamin D and calcium(“conventional therapy”)Total U.S.Prevalence25k32k3,000

61、3 annually Post-surgical,auto-immune,genetic,or idiopathicNewly DiagnosedFor investor communication only.Not for use in product promotion.Not for further distribution.17For investor communication only.Not for use in product promotion.Not for further distribution.U.S.YORVIPATH Enrollment Since Decemb

62、er 20241841%59%Strong early enrollments across all segments from 150 unique prescribersA.S.A.P.=Ascendis Signature Access Program;EAP=Expanded Access Program.Existing patients from TransCon PTH EAP or Clinical TrialsPatients new to YORVIPATHMajority of new YORVIPATH patients are switching from conve

63、ntional therapy New prescriptions enrolled in A.S.A.P.or direct with specialty pharmacy(n=324)as of January 9,2025TransCon PTH Program Summary and Outlook19 First and only approved product indicated for the treatment of hypoparathyroidism in adults Clinical trial results consistent across geographie

64、s,ethnicities,and etiologies TransCon PTH under regulatory review for market authorization in Australia,Israel,Japan,and Switzerland YORVIPATH 2024 revenue expected to be 29 million1 700 patients on YORVIPATH treatment in Europe Direct and International Markets at the end of 2024 Commercial launch e

65、xpected in 5 additional Europe Direct countries in 2025 U.S.commercial launch status as of January 9,2025 Strong initial enrollments with 324 patients,including patients switching from conventional therapy 150 unique prescribers from 38 statesFor investor communication only.Not for use in product pr

66、omotion.Not for further distribution.1.Unaudited preliminary estimate.YORVIPATHTransCon PTHTreatment of Hypoparathyroidism in AdultsTransCon CNP is an investigational product candidate.For investor communication only.Not for use in product promotion.Not for further distribution.TransCon CNP(navepegr

67、itide)Investigational prodrug of CNP designed to provide sustained release and continuous exposure of active CNP for the treatment of pediatric achondroplasia The pivotal ApproaCH Trial in children aged 2-11 years achieved primary objective TransCon CNP demonstrated LS mean AGV of 5.89 cm/year with

68、LS mean treatment difference of 1.49 cm/year at Week 52 compared to placebo(p0.0001)For children aged 5-11 years TransCon CNP demonstrated LS mean AGV of 5.79 cm/year with a change from baseline AGV superior to placebo with LS mean treatment difference of 1.78 cm/year at Week 52(p0.0001)Other endpoi

69、nts support that TransCon CNP may provide benefits beyond linear growth Treatment with TransCon CNP resulted in numerical improvements in health-related quality of life compared to placebo as observed in several ACEM domains Treatment benefit in muscle functionality demonstrated in sub-group of chil

70、dren aged 5-8 years Treatment with TransCon CNP showed improvement in body proportionality,leg bowing,and other parameters TransCon CNP was generally well-tolerated,with low frequency of injection site reactions(all mild),and no evidence of hypotensive effectPivotal ApproaCH Trial SummaryOnce-weekly

71、 TransCon CNP may address the need for an efficacious,safe,tolerable,and convenient treatmentData on file.Ascendis Pharma.ACEM=Achondroplasia Child Experience Measure.21TransCon CNP is an investigational product candidate.For investor communication only.Not for use in product promotion.Not for furth

72、er distribution.Leg bowing is a common complication in achondroplasia,resulting in pain,impaired physical functioning,need for corrective surgery,and a negative impact on quality of life1,2,3Impact of TransCon CNP on Leg Bowing22Tibial Femoral Angle(TFA,degrees)Mechanical Axis Deviation(MAD,mm)Trans

73、Con CNP100 g/kg/weekPlacebo-2-1012Absolute Change from Baseline at Week 52LS Mean(95%CI)Degreesp=.0094+0.39(-0.72,1.49)-1.43(-2.23,-0.62)-1.81(-3.16,-0.47)Worsen Improve TransCon CNP100 g/kg/weekPlacebo-2-101234Absolute Change from Baseline at Week 52LS Mean(95%CI)mmp=.0063+1.78(0.03,3.52)-1.01(-1.8

74、7,-0.14)-2.78(-4.71,-0.86)Worsen 5Improve TransCon CNP demonstrated significant improvements in leg bowingData on file.Ascendis Pharma.1.Hunter et al.J Med.Genet.35(9)(1998):705712.2.Matsushita et al.Calcif.Tissue Int.104(2019)364372.3.Nahm et al.Orphanet Journal of Rare Diseases 18(2023)139.TransCo

75、n CNP is an investigational product candidate.For investor communication only.Not for use in product promotion.Not for further distribution.TransCon CNP Program Summary and Outlook In pivotal ApproaCH Trial,TransCon CNP demonstrated significant improvements in linear growth and body proportionality,

76、as well as benefits beyond linear growth Following pre-NDA meeting,planned filings for treatment of children with achondroplasiain U.S.Q1 2025,EU in Q3 2025 Comprehensive development plans continue with ongoing and planned trials to support TransCon CNP in additional patient populations COACH Trial

77、first combination trial of TransCon hGH and TransCon CNP to further accelerate growth in achondroplasia;topline Week 26 data in children aged 2-11 expected in Q2 2025 Plan to submit IND or similar for the treatment of hypochondroplasia in Q4 2025With SKYTROFA and TransCon CNP,Ascendis is well-positi

78、oned to become the leader in growth disorders 23TransCon CNP is an investigational product candidate.For investor communication only.Not for use in product promotion.Not for further distribution.Expanding the Endocrine Rare Disease Pipeline and BeyondFor investor communication only.Not for use in pr

79、oduct promotion.Not for further distribution.X-Linked HypophosphatemiaFGF-23=Fibroblast growth factor 23.XLH=X-linked hypophosphatemia.1.International XLH alliance.2 Beck-Nielsen Orphanet J Rare Dis.2019.3.FDA Multi-Disciplinary Review BLA 761068.4.PadidelaCalcif Tissue Int 2021 5.LinglartJCEM 2022.

80、Characterized by excess FGF-23 production leading to excessive renal phosphate wasting1,2Affecting 1:20000Deformity&FracturesPainShort StatureTeeth Loss25Clear biological rationale for applying TransCon protein degrader technology to normalize FGF-23 levelsFor investor communication only.Not for use

81、 in product promotion.Not for further distribution.Increase in serum phosphorous from baseline plateaus at 1.0-1.5 mg/dL,irrespective of burosumab drug exposure3At the recommended doses,serum phosphorous remained in the lower limit of normal3Significant unmet medical need remains within the XLH pati

82、ent population4,5 Limitations of Existing TherapyBackgroundCollaboration with Novo NordiskReflects our Vision 2030 to create value in additional large therapeutic areasFor investor communication only.Not for use in product promotion.Not for further distribution.26 Development and commercialization o

83、f TransCon Technology-based products in metabolic(including obesity and type 2 diabetes)and cardiovascular diseases Highlights of the multi-product collaboration:Upfront fee of$100 million for the exclusive license and funding of the programs by Novo Nordisk Lead program:once-monthly TransCon Semagl

84、utide product candidate initially targeting obesity and type 2 diabetes-Potential to receive additional payments of up to$185 million in development and regulatory milestone payments-Potential to receive escalating tiered,mid-single digit royalties on global net sales and sales milestone payments No

85、vo Nordisk responsible for clinical development,regulatory,commercial manufacturing,and commercializationOther Investments and PartnershipsFor investor communication only.Not for use in product promotion.Not for further distribution.27Creating value through innovative business models Eyconis(exclusi

86、ve global rights for TransCon based ophthalmology products)Formed Eyconis in January 2024 with a$150 million commitment from external investors Teijin(exclusive rights for TransCon hGH,PTH,and CNP in Japan)In December 2024,submitted a J-NDA to the Japanese medical authorities for TransCon PTH for th

87、e treatment of adults in Japan with hypoparathyroidism VISEN Pharmaceuticals(exclusive rights for TransCon hGH,PTH,and CNP in Greater China)Announced in September 2024,Phase 3 PaTHway China Trial success for TransCon PTH,achieving primary and key secondary endpoints in treating adults with hypoparat

88、hyroidismFinancial Update&2025 Selected MilestonesFor investor communication only.Not for use in product promotion.Not for further distribution.Total full-year 2024 product revenue expected to be 2261 million,driven by:SKYTROFA full-year 2024 revenue expected to be 2022 million,excluding sales deduc

89、tions related to prior years YORVIPATH full-year 2024 revenue expected to be 291 million Full-year 2024 total revenue expected to be 3641 million Includes$100 million Novo Nordisk milestone payment as non-product revenue December 31,2024 pro forma cash balance of 6553 million Looking ahead Investmen

90、t in YORVIPATH launch in the U.S.and Europe Direct and TransCon CNP pre-launch activities Stable R&D investment as current portfolio matures and early-stage pipeline renews 2024 Financial UpdateFor investor communication only.Not for use in product promotion.Not for further distribution.291.Unaudite

91、d preliminary estimate.2.Calculated as unaudited preliminary estimate of full year 2024 SKYTROFA revenue of 197 million plus 5 million of sales deductions related to prior years.3.Calculated as unaudited preliminary estimate of December 31,2024 cash balance of 560 million plus expected payment from

92、Novo Nordisk of$100 million.Plan to be well-capitalized through cash flow breakeven and beyondSelected Milestones Expected in 2025Q4 2025TransCon CNPSubmit IND or similar for hypochondroplasiaFor investor communication only.Not for use in product promotion.Not for further distribution.30Q1 2025Q2 20

93、25Q3 2025TransCon CNPSubmit MAA to the EMATransCon CNPTopline Week 26 data from COACH TransCon CNPSubmit NDA to the FDATransCon hGHAdult GHD FDA DecisionTransCon hGHAdult GHD U.S.commercial availability,if approvedTransCon PTHCommercial launch expected in 5 additional Europe Direct countriesTransCon

94、 hGHSubmit IND or similar for basket trial in additional indications11.Planned for small for gestational age without catch-up growth“SGA”;Idiopathic short stature“ISS”;SHOX deficiency(including Turner syndrome)Thank youInvestor Relations 2025 Ascendis Pharma.Ascendis,Ascendis Pharma,the Ascendis Pharma logo,the company logo,TransCon,Skytrofa and Yorvipath are trademarks owned by the Ascendis Pharma group.For investor communication only.Not for use in product promotion.Not for further distribution.https:/