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Cautionary Note On Forward-Looking Statements This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this presentation, such as statements regarding o

Key Takeaway: Ascendis Pharma A/S R&D Day June 26, 2019 Exhibit 99.1 Cautionary Note On Forward-Looking Statements This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this presentation, such as statements regarding our

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Ascendis Pharma A/S R&D Day June
26, 2019 Exhibit 99.1
Cautionary Note On Forward-Looking
Statements This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this presentation, such as statements regarding our future results of operations and financial position,
including our business strategy, prospective products, availability of funding, clinical trial results, product approvals and regulatory pathways, collaborations, timing and likelihood of success, plans and objectives of management for future
operations, and future results of current and anticipated products, are forward-looking statements. These 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 forward-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, including, without limitation, our most recent Annual Report on Form 20-F filed with the SEC on April 3, 2019 particularly in the sections titled "Risk
Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". 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 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 required by law, we assume no obligation to update these statements publicly, whether as a result of new
information, future events or otherwise after the date of this presentation. This presentation concerns 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 product candidates are currently limited by U.S. Federal law to investigational use, and no representations are made as to their safety or effectiveness
for the purposes for which they are being investigated. These presentation materials include a presentation from Dr. Ezra Cohen, who is an expert in the field in which he is presenting. He is providing background information about certain diseases,
but his views do not necessarily represent those of the Company. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo and TransCon are trademarks owned by the Ascendis Pharma group. June 2019 Ascendis Pharma A/S.
Today's Agenda 9:00-9:05 a.m.
Welcome & Agenda Overview Scott T. Smith 9:05-9:15 a.m. Vision 3x3 Jan M ller Mikkelsen 9:15-9:30 a.m. TransCon Platform & Product Innovation Kennett Sprog e, Ph.D. 9:30-10:05 a.m. TransCon hGH Jonathan Leff, M.D. 10:05-10:15 a.m.
Connected Healthcare Platform Thomas rts Pedersen 10:15-10:30 a.m. Commercialization Update Tom Larson 10:30-10:40 a.m. Q&A: TransCon hGH 10:40-10:50 a.m. Break 10:50-11:20 a.m. TransCon PTH David Karpf, M.D. & Nyssa Liebermann Noyola
11:20-11:35 a.m. TransCon CNP Kennett Sprog e, Ph.D. 11:35-11:45 a.m. Q&A: TransCon PTH & TransCon CNP 11:45-12:05 p.m. Introducing Oncology Jan M ller Mikkelsen & Juha Punnonen, M.D., Ph.D. 12:05-12:20 p.m. IO & IT
Treatments: Challenges & Promise Ezra Cohen, M.D. 12:20-12:40 p.m. Oncology Product Candidates Juha Punnonen, M.D., Ph.D. 12:40-12:50 p.m. Q&A: Oncology 12:50-1:00 p.m. General Q&A & Closing Remarks Jan M ller
Vision 3x3 Jan Mikkelsen President
Building a Leading Rare Disease Company
Leverage the validated TransCon technology to create best-in-class products Advance the company's pipeline of three rare disease endocrinology product candidates: Phase 3 ongoing for TransCon Growth Hormone File INDs for TransCon PTH and
TransCon CNP in 2017 Obtain approval for at least two products between 2020-2024 Next rare disease therapeutic area to come Identify clinical stage candidate by 2020 Build an integrated commercial business focused on the U.S. market Slide presented
Vision 20/20: Established the
Foundation for a Leading Rare Disease Company Clinical validation of all 3 product candidates in endocrinology rare diseases Positive phase 3 for TransCon Growth Hormone Selection of Oncology as new therapeutic area TransCon CNP TransCon hGH
TransCon PTH Oncology
Vision 3x3: Building a Leading
BioPharma Company Our Goal is to Achieve Sustainable Growth through Multiple Approaches Obtain regulatory approval for 3 Endocrinology Rare Disease products TransCon hGH for pediatric growth hormone deficiency TransCon PTH for adult
hypoparathyroidism TransCon CNP for achondroplasia Growth of Endocrinology Rare Disease pipeline through: Label expansion programs with the goal of obtaining 9 indications in total Global clinical reach directly or through partnerships Build an
integrated commercial business for our Endocrinology Rare Disease franchise in North America and select European countries Establish global commercial presence with partners outside our geographic areas Create 3 independent therapeutic areas each
with a diversified pipeline built on TransCon technologies and our unique algorithm for product innovation Established oncology as next independent therapeutic area
TransCon Technology Platform
& Product Innovation Kennett Sprog e, Ph.D. SVP, Head of Innovation and Research
TransCon Technology: A Combination of
Technologies Prodrug Technology Predictable Release Technology TransCon Technology Known Biology TransCon combines the benefits of Prodrug and Predictable Release Technologies with Known Biology to create highly differentiated products for the
Transient Conjugation: Flexible and
Versatile Platform Parent Drug Soluble Carriers Insoluble Carriers Antibodies, Antibody Fragments, Proteins, Peptides and Small Molecules Aromatic Cyclic Imide DKP Carbamate Bicin AEG Pyroglutamate TransCon Carrier TransCon Prodrug: 3 components
TransCon Technology: The TransCon
Linker Cleaves in an enzyme-independent fashion, ensuring reproducible drug release; in vitro to in vivo correlation with high predictability TransCon linkers remain covalently bound to the carrier molecule after release of the unmodified parent
drug Enables tunable design of prodrugs with dosing frequency from daily up to six months or more Carrier Drug Drug Carrier pH 7.4; 37 C
TransCon Drug Release is Predictable
In vivo TransCon PTH phase 1 interim data [124 g dose] Free PTH [pg/mL] In vitro TransCon PTH release kinetics at pH 7.4 and 37 C Residual peptide [%] Days High predictability of PK profile from in vitro data enables optimization of PK
profile of lead candidates in vitro prior to initiating in vivo development
TransCon Technology: Sustained
Systemic Delivery Parent drug is transiently bound to a TransCon linker-soluble carrier moiety, which inactivates and shields parent drug from clearance Designed to distribute released drug like the parent molecule; linker-carrier is cleared renally
Following injection, the linker is designed to autocleave at a specific rate to predictably release unmodified parent drug TransCon carrier TransCon linker Parent drug (inactive) Unmodified parent drug Receptor Linker cleavage dependent upon pH and
temperature Renal clearance Prodrug (inactive)
Parent drug is transiently bound to
TransCon linker-hydrogel carrier, which inactivates, shields parent drug and prevents clearance Designed to provide sustained high local drug levels with low systemic exposure; hydrogel degrades into small polymers that are renally cleared Following
injection, the linker is designed to autocleave at a specific rate to predictably release unmodified parent drug TransCon Technology: Sustained Localized Delivery Linker cleavage dependent on pH and temperature Parent drug (inactive) Unmodified
parent drug TransCon hydrogel carrier Receptor Renal clearance TransCon linker Prodrug (inactive)
Sustained Localized Delivery:
Validated Across Multiple Drugs and Administration Sites 1 Patent WO2018175788 Vitreal PK of Fab following intra-vitreous administration in rabbits. TransCon Fab half-life was ~53 days compared to free Fab half-life of ~3.2 days. Plasma PK of
Daptomycin following intra-articular administration in rabbits. TransCon Daptomycin half-life was ~3 days. Excellent local tolerability of TransCon hydrogel carrier Sustained high local concentration with low systemic exposure Small Molecules
Intra-articular administration Antibody Fragments (Fab) Intra-vitreous administration1 Fab Concentration ( g/mL) Day 0 20 40 60 1 10 100 1000 Free Fab TransCon Fab 1000 100 10 1 0 2 4 6 8 10 12 14 TransCon Daptomycin Day Daptomycin (ng/mL)
Why Size Matters: Releasing
Unmodified Drug A molecule's hemodynamic radius predicts tissue distribution1 Albumin (66 kDa) is 3.6 nm, effectively maintaining a high albumin concentration in the blood compartment and low tissue concentrations hGH (22 kDa) is 2 nm,
allowing distribution into adipose, muscle, brain and liver 1 Anat Rec A; 2006, 288A: 91-103. 2 AAPS J. 2016, 18(1): 157-170. kDa = kilodalton, a measure of weight Distribution Coefficient The growth plate is avascular, representing a diffusion
barrier Studies in mice suggest molecules 40 kDa and larger has restricted access to the growth plate2 Relatively higher systemic drug concentrations are required to provide efficacious drug levels Size (Hydrodynamic Radius in nm [nanometer])
Ascendis Algorithm for Product
Innovation Unmet Medical Need Clinically Validated Parent Drug TransCon Technology Suitability Clearly Differentiated Product Established Clinical & Regulatory Pathway Large Addressable Market Higher Value, Lower Risk Pipeline Our unique
algorithm for product innovation has resulted in clinical validation of 3 out of 3 product candidates and positive phase 3 data in Endocrinology Rare Disease We are continuing to apply our algorithm to build a pipeline in oncology and are committed
to entering a 3rd therapeutic area
TransCon Enables Multi-level Patent
Protection As of March 1, 2019 TransCon prodrugs eligible for new composition of matter IP A multi-layered patent strategy is applied to protect our assets Composition of Matter 139 Issued Patents >330 Patent Applications Pending Device Dose
Regimen Process TransCon Carriers TransCon Linkers Product Concepts
TransCon: A New Innovative
Technology Platform TransCon technologies combine the benefits from prodrug and predictable release technologies with known biology - in one single platform TransCon prodrugs release unmodified drug expected to maintain the same mode of action
as parent drug (receptor activation, distribution, etc.) Broad applicability for both systemic (s.c. / i.v.) and localized delivery (intravitreal, intratumoral, inhaled) Product features may include low injection volume, room temperature storage,
small needle size (31G) Daily, weekly, monthly, or twice yearly or longer administration frequencies TransCon has a high success rate in endocrinology with clinical validation of 3 out of 3 product opportunities and positive phase 3 results for
TransCon Growth Hormone:
Once-Weekly Replacement Therapy Jonathan A. Leff, M.D. SVP, Chief Medical Officer
Growth Hormone Deficiency Is Not
Just About Height: Growth Hormone Supports Overall Endocrine Health Sources: 1. de Boer, H. et al. 1997; 2. Rutherford, O. M. et al. 1991. 3. Colle, M., J. Auzerie.1993. 4. Johannsson, Gudmundur, et al. 1999. 5. Stabler, Brian et al. 1996. 6. Leong,
Gary M., Gudmundur Johannsson. 2003. 7. Colao, Annamaria et al. 2002. 8. Bex, M, and R Bouillon. 2003 Fractures Adult peak bone mass is considerably lower, and rates of fractures are significantly higher among adults with GHD who were not treated as
children.8 Ultimate Height Achievement Children with GHD may not obtain full height potential if untreated.1 Cardiovascular Disease Early discontinuation of GH treatment may induce impairment of patients' lipid profiles and cardiac function,
leading to increased risk for CV disease.6,7 Mental Health A high incidence of psychiatric disorders, usually accompanied by poor life quality, is associated with adults who were GHD as children.5 Body Composition Increased fat mass, decreased
muscle mass and decreased bone density can occur soon after treatment discontinuation.2,3,4 Daily hGH addresses all the symptoms of the disease; long-acting growth hormone products must fully mimic daily hGH to adequately address the totality of the
Daily Growth Hormone: The Problem 1
PLoS ONE 2011, 6(1): e16223 2 Clinical Therapeutics 2008, 30(2): 307-316 Increased adherence Improved growth Doses missed per week1 3 2 P< 0.001 P< 0.01 1 Height velocity SDS Poor adherence with daily hGH therapy is associated
with reduced height velocity and impaired quality of life1 Reduced frequency of administration is associated with better adherence2 In the 1st year, two of three patients miss >1 injection on average per week1 0 1 2 3 4
A Decades-Long Pursuit: Long-Acting
Growth Hormones1 1 European Journal of Endocrinology (2016) 174, C1-C8 Challenges Appropriate tissue penetration hindered by large size Immunogenicity (neutralizing antibodies) Viscous and painful formulations Large bore needle requirements
Inadequate pharmacokinetics Depot formulations PEGylated (permanent) formulations GH fusion proteins (molecular enlargement) Non-covalent albumin binding Approaches
TransCon hGH Design TransCon carrier
TransCon linker hGH (inactive) Unmodified hGH Receptor Linker cleavage dependent upon pH and temperature Renal clearance Once-weekly prodrug releases unmodified hGH designed to mimic daily hGH: Tissue distribution Physiological levels Therapeutic
effects: efficacy, safety and tolerability
Growth Comparable to a Daily hGH in
Phase 21,2 1 Intergroup differences not statistically significant 2 J Clin Endocrinol Metab 2017, 102(5): 1673-1682 3 Conducted with a bioequivalent version of TransCon hGH Same weekly dose Dose hGH (mg/kg/week): 11.5 14.5 0.14 0.21 0.30 0.21
TransCon hGH3 Genotropin 26-week treatment period (N=53)
Dose Proportional IGF-1 Response in
Phase 21 1 J Clin Endocrinol Metab 2017, 102(5): 1673-1682 Days (Week 13) IGF-1 SDS (Mean +SE) Transient values > +2.0 observed in a small number of subjects, primarily at the highest dose level 0 1 2 3 4 5 6 7 TransCon hGH 0.14 mg/kg/week
TransCon hGH 0.21 mg/kg/week TransCon hGH 0.30 mg/kg/week
Comparable hGH Levels in Phase 21 1
J Clin Endocrinol Metab 2017, 102(5): 1673-1682 Maximum hGH concentration comparable between equivalent weekly doses of TransCon hGH and a daily hGH hGH serum concentration (ng/mL; Mean +SE) Days (Week 13)
TransCon hGH Phase 3 Program in
Pediatric GHD Subjects previously treated (n=143) and treatment-na ve (<3 years, n=3) Top-line data reported Extension trial (N=~300) Regulatory filings (BLA H1 2020, MAA H2 2020) Treatment-na ve subjects Top-line data reported N=146
Phase 3 heiGHt Trial 161
treatment-na ve children with GHD dosed (2:1 randomization) TransCon hGH (0.24 mg/kg/week) Genotropin (34 g/kg/day = 0.24 mg/kg/week) Long-Term Extension Trial Week 1 Week 5 Week 13 Week 52 Week 26 Week 39 Screening 6 weeks Key
Last updated: Jun 26, 2019