1、Ascendis Pharma A/S42ndAnnual J.P.Morgan Healthcare ConferenceJanuary 8,2024For investor communication only.Not for use in product promotion.Not for further distribution.Cautionary Note on Forward-Looking StatementsThis presentation contains forward-looking statements.All statements other than state

2、ments of historical facts contained in this presentation,such as statements regarding our products and prospective product candidates;clinical trial results;the expected timing of future clinical trial results and feedback from regulatory authorities;the scope,progress,results and costs of developin

3、g our product candidates or any other future product candidates;timing and likelihood of success;plans and objectives of management for future operations and commercialization activities;and future results of current and anticipated products and product candidates are forward-looking statements.Thes

4、e forward-looking statements are based on our current expectations and beliefs,as well as assumptions 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 f

5、orward-looking statements.These risks,uncertainties and other factors are more fully described in our reports filed with or submitted to the Securities and Exchange Commission(SEC),including,without limitation,our most recent Annual Report on Form 20-F filed with the SEC on February 16,2023 particul

6、arly 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 representation or warranty by us or any other

7、 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 the date of this presentation.Except as req

8、uired 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 as well as Ascendis Pharma investigational

9、 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 candidates are currently limited by law to

10、 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 communication only.Not for use in product promotion.2For investor communication only.Not for use in product promotion.Not

11、for further distribution.Executive SummaryValidated TransCon technology platform and algorithm for product innovationTwo approved products SKYTROFA(TransCon hGH)and YORVIPATH(TransCon PTH)Pivotal data and submission of NDA for TransCon CNP expected in Q4 2024Oncology portfolio Phase 2 data expected

12、in Q4 2024 Creation of Ophthalmology NewCo,financed by institutional investors,expected in Q1 2024SKYTROFA achieved U.S.market value leadership in 2023Expected full year 2023 SKYTROFA revenue of 179 million1Full year 2024 SKYTROFA revenue expected to be 320 to 340 million2YORVIPATHfirst launch in Ge

13、rmany January 2024 Rolling out to Europe Direct and International Markets starting in 2024In the U.S.,PDUFA goal date of May 14,2024,and launch expected the following quarter,if approvedBusiness model built on fast,successful drug development and commercial therapeutic synergiesExpect to be operatin

14、g cash flow breakeven on a quarterly basis by the end of 20243For investor communication only.Not for use in product promotion.Not for further distribution.1Unaudited preliminary estimate.2 Based on average 2023 exchange rates.Vision 3x3:Building a Leading Global Biopharma Company4Our goal is to ach

15、ieve sustainable growth through multiple approachesObtain regulatory approval for three independent Endocrinology Rare Disease products TransConhGH for pediatric growth hormone deficiency TransConPTH for adult hypoparathyroidism TransConCNP for achondroplasiaGrow Endocrinology Rare Disease pipeline

16、through Global clinical reach Pursuing 9 total indications,label optimization,and life cycle managementNew endocrinology productsEstablish global commercial presence for our Endocrinology Rare Disease area Build integrated commercial organization in North America and select European countriesEstabli

17、sh global commercial presence through partners with local expertise and infrastructureAdvance a high-value oncology pipeline with one IND or similar filing each yearCreate a third independent therapeutic area with a diversified pipelineAscendis Pharmas 2020-2025 strategic roadmapFor investor communi

18、cation only.Not for use in product promotion.Not for further distribution.Endocrinology Rare Disease Pipeline&Ascendis Direct CommercializationFor investor communication only.Not for use in product promotion.Not for further distribution.Endocrinology Rare Disease Products Approved in Major Markets6T

19、ransCon hGHPediatric Growth Hormone DeficiencyUnited States1Approved in the U.S.as SKYTROFA(lonapegsomatropin-tcgd)European Union2and Selected Other Countries3Approved in the EU as SKYTROFA(lonapegsomatropin)TransCon PTHAdult Chronic HypoparathyroidismEuropean Union4and Selected Other Countries5Appr

20、oved in the EU as YORVIPATH(palopegteriparatide)First country launch planned in Germany in January 20241.SKYTROFApackage insert.Palo Alto,CA:Ascendis Pharma Inc.October 2022.2.SKYTROFASMPC.Hellerup,Denmark:Ascendis Pharma Inc.January 2022.3.SKYTROFAis also approved in Norway,Iceland,Lichtenstein and

21、 Great Britain(covering England,Wales,Scotland).4.YORVIPATHSMPC.Hellerup,Denmark:Ascendis Pharma Inc.November 2023.5.YORVIPATHis also approved in Norway and Iceland.For investor communication only.Not for use in product promotion.Not for further distribution.TransCon hGH:Expanding Beyond Pediatric G

22、HDPediatric GHD accounts for approximately 50%of the hGH market by patientsGrowth hormone plays an essential role in the health of children and adultsMaintains normal body composition and cardiometabolic healthPromotes normal growth in childrenDaily hGH is approved for multiple indications in adult

23、and pediatric patientsAdult GHD accounts for approximately 10%of the daily hGH marketHowever,fewer than 10%of adults with suspected GHD1are treated with hGHTransCon hGH Phase 3 foresiGHt Trial in adult GHDDemonstrated superiority to placebo on primary and secondary efficacy endpointsDemonstrated com

24、parable results to daily hGH on primary and secondary endpoints2Expected sBLA submission in Q2 20247For investor communication only.Not for use in product promotion.Not for further distribution.1 A.Hoffman et al(2023).SAT611 Prevalence Of Comorbidities Among Treated And Untreated Adults With Suspect

25、ed Growth Hormone Deficiency.Journal of the Endocrine Society.7.10.1210/jendso/bvad114.1344.2 Exploratory post-hoc analysis for patients with IGF-1 SDS levels 1.75 at Week 38.SKYTROFA Revenue Development and ExpectationsSKYTROFA was launched in U.S.in Q4 2021,Germany in Q3 2023 SKYTROFA Q4 2023 reve

26、nue of 64 million*Estimated SKYTROFA U.S.pediatric GHD market penetration exiting 2023 at 16%Expect full year 2024 SKYTROFA revenue of 320-340 million*8 2 4 12 17 32 36 47 64 0 10 20 30 40 50 60 70 1 36 179 320-340 0 50 100 150 200 250 300 350 400202120222023E*2024E*Unaudited preliminary estimate.*R

27、eflects 2024 SKYTROFA revenue expectations announced on Jan.7,2024,based on average 2023 exchange rates.For investor communication only.Not for use in product promotion.Not for further distribution.Quarterly SKYTROFA Revenue(million)Annual SKYTROFA Revenue(million)TransCon hGH Program Summary and Ou

28、tlookAttained U.S.market value leadership in 2023Completed enliGHten Trial in pediatric GHD1Majority of patients met or exceeded average parental height SDSDemonstrated the long-term safety in patients treated up to 6 yearsPlanned sBLAsubmission to FDA for adult GHD Q2 2024Topline results from Phase

29、 2 trial in Turner syndrome expected Q4 2024Expanding reach for SKYTROFA through Ascendis commercial organization building on therapeutic synergiesFull year 2024 SKYTROFA revenue expected to be 320 to 340 million29For investor communication only.Not for use in product promotion.Not for further distr

30、ibution.Committed to making TransCon hGH the global leading product in valuein a growing growth hormone market1Vlachopapadopoulou et al.Poster LB17,presented at ESPE 2023.2Based on average 2023 exchange rates.MyopathySpondylorthropathyDry skinThinning hairBrittle nailsAltered tooth morphologyCatarac

31、tsPapilledemaAnxiety&depressionCognitive impairment(“brain fog”)ParesthesiaMuscle crampsPainTetanyNephrocalcinosis*Nephrolithiasis*Chronic kidney disease*Vascular calcificationsCardiac arrhythmiasHypocalcemia-associated dilated cardiomyopathyLaryngospasmSeizuresBasal ganglia calcificationsParkinsoni

32、sm or dystoniaHypoparathyroidism:One Disease,Multiple Consequences1-410CENTRAL NERVOUS SYSTEMOPHTHALMALOGICALPERIPHERAL NERVOUS SYSTEMCARDIOVASCULARDERMATOLOGICALRENALNEUROPSYCHIATRICMUSCULOSKELETALDENTALRESPIRATORYThyroid glandParathyroid glandAbsence or deficiency of parathyroid hormone(PTH)is lin

33、ked to multi-organ manifestations5,6*These manifestations are mostly the result of treatment with calcium supplements and activated vitamin D rather than of the disease itself.51.Underbjerg L,SikjaerT,Mosekilde L,et al.Cardiovascular and renal complications to postsurgical hypoparathyroidism:a Danis

34、h nationwide controlled historic follow-up study.J Bone Miner Res.2013;28(11):2277-2285.doi:10.1002/jbmr.1979.2.Underbjerg L,SikjaerT,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/jbmr

35、.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,SikjaerT,MosekildeL,et al.The epidemiology of nonsurgical hypoparathyroidism in Denmark:a nationwi

36、de case finding study.J Bone Miner Res.2015;30(9):1738-1744.doi:10.1002/jbmr.2501.5.Mannstadt M,Bilezikian JP,ThakkerRV,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 and clini

37、cal 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.Latest Clinical Practice Guideline11Consider PTH replacement therapy in patients not adequately controlled on conventional

38、 therapyInadequate control is considered to be any one of the following:Symptomatic hypocalcemia Hyperphosphatemia Renal insufficiency HypercalciuriaPoor quality of lifeIn addition,individuals with poor compliance,malabsorption,or who are intolerant of large doses of calcium and active vitamin D may

39、 also benefit from PTH replacement therapy1.Aliya A Khan et al,The Second International Workshop on the Evaluation and Management of Hypoparathyroidism,Journal of Bone and Mineral Research,10.1002/jbmr.4671.For investor communication only.Not for use in product promotion.Not for further distribution

40、.Significantly Improved Renal Function with TransCon PTH Treatmentin Phase 3 PaTHway Trial Post Hoc Analysis 12eGFR(an assessment of kidney filtering capacity)was calculated by the trials central lab using the Modification of Diet in Renal Disease Study Group(MDRD)equation(Levey,Ann Intern Med 2006)

41、.Patients Randomized to TransCon PTHChange in mean eGFR from Baseline(mL/min/1.73m)Patients Randomized to Placebo/Switched to TransCon PTH after Week 26At Week 26At Week 26At Week 52At Week 52+11.4+11.5+6.3+8.2-4.00.04.08.012.0+0.1+11.7-2.4+6.5 eGFR 60 at baselineeGFR 60 at baselineFor investor comm

42、unication only.Not for use in product promotion.Not for further distribution.*indicates nominal p0.05indicates nominal p$1B)forTransCon PTH,TransCon hGH,and TransCon CNP through worldwide commercialization Be the leader in Growth Disorders and Hypoparathyroidism,pursuing clinical conditions,innovati

43、ve LCM and complementary patient offerings Expand pipeline with Endocrinology Rare Disease blockbuster product opportunitiesCreate Value in Additional Therapeutic Areas through Innovative Business ModelsObtain accelerated approval in oncology with registrational trials ongoing Pursue TransCon produc

44、t opportunities in$5B indicationsMaximize value creation of these product opportunities through collaboration with therapeutic area market leadersDifferentiate with Ascendis FundamentalsOutperform industry drug development benchmarks with Ascendis product innovation algorithmRemain independent as a

45、profitable biopharma through lean and flexible ways of workingLet our values Patients,Science,Passion drive our decisions to successFor investor communication only.Not for use in product promotion.Not for further distribution.Achieve blockbuster status for multiple products and expand our engine for future innovation Thank you!2024 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.