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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

Key Takeaway: Ascendis Pharma A/S Jan Mikkelsen President & CEO January 2017 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 st

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Ascendis Pharma A/S Jan Mikkelsen
President & CEO January 2017 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, particularly in the sections titled "Risk Factors" and
"Management's Discussion and Analysis of Financial Condition and Results of Operations" as well as our Report on Form 6-K filed with the SEC on October 18, 2016. 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. Ascendis is a trademark that we use in this presentation. Any other trademarks appearing in this presentation are the property of
their respective holders.
Company Overview Create best-in-class
rare disease products addressing unmet medical needs Apply TransCon technology to parent drugs with clinical proof-of-concept Expect higher development success rate compared to traditional drug development Endocrinology rare disease wholly-owned
pipeline and expected milestones TransCon Growth Hormone for pediatric GHD: heiGHt Trial full enrollment Q4 2017 TransCon PTH for hypoparathyroidism: IND/equivalent in 2Q 2017; planning pivotal trial in 2018 TransCon CNP for achondroplasia:
IND/equivalent in 4Q 2017 Commercial focus on the U.S. market with multi-billion dollar revenue potential Established high-value collaborations with Roche/Genentech in ophthalmology and Sanofi in diabetes Pro forma September 30, 2016 cash of ~
190 million1 1 Based on cash and cash equivalents as of September 30, 2016 and approximately $127.4 million in estimated net proceeds from the public offering of our ADSs that was completed in 4Q 2016.
TransCon Technology Predictable release
of parent drug in the body Parent Drug Parent Drug TransCon carrier TransCon linker Inactive Prodrug Predictable release of unmodified parent drugs Parent drugs can be proteins, peptides or small molecules Linker release only dependent on pH and
temperature TransCon carrier enables both systemic and localized drug exposure Maintains same mode-of-action of parent drug molecule Release of parent drug supporting up to half-yearly administration TransCon products eligible for new composition of
matter IP claims Creation of TransCon Prodrug
Ascendis' Approach to Product
Innovation 1 Totals from FDA and EMA Orphan List, public & proprietary databases, advisors and conferences 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 ~4,800 Orphan Drug Designations >500 Endocrine/Metabolic1
Expertise in endocrinology (clinical,
regulatory, medical affairs) Concentrated prescriber audience Small, focused sales force Patient-centric support system Reimbursement and payor knowledge Distribution networks TransCon CNP TransCon Growth Hormone Expected Synergies of Therapeutic
Product Candidate Primary Indication
Development Stage Potential WW Market1 WW Commercial Rights TransCon Growth Hormone Growth hormone deficiency Phase 3 > $3 billion2 TransCon PTH Hypoparathyroidism Pre-IND > $2 billion3 TransCon CNP Achondroplasia Pre-IND > $1 billion
TransCon Ranibizumab Ophthalmology Not disclosed > $7 billion TransCon Peptides Diabetes Not disclosed > $1 billion TransCon Treprostinil PAH Phase 1 > $1 billion Partnering Opportunity Endocrinology Ophthalmology CV Internal Rare Disease
Endocrinology Pipeline Current/Potential Strategic Collaborations Building a Leading Company in Rare Diseases 1 Based on market data and company estimates. 2 Includes all indications. 3 Based on treatment of ~25% of the U.S. patient population of
TransCon Growth Hormone: Once-Weekly
Long-Acting Growth Hormones Human
Growth Hormone (hGH) is a growing ~$3 billion market Largest indication is Growth Hormone Deficiency (GHD), representing ~50% of the market Pediatric indications comprise up to 90% of the market Current growth hormone therapies require daily
injections, which often results in suboptimal compliance and treatment response1 No successful commercialization of a long-acting growth hormone despite ~ 30 years of multiple attempts with different technologies Molecular enlargement technologies
- GH protein fusions, permanent PEGylation of GH Depot formulations - release of unmodified GH from polymer microspheres TransCon Growth Hormone capitalizes on lessons from previous programs Delivers physiological levels of unmodified
growth hormone Safety, tolerability, efficacy and immunogenicity shown to be comparable to daily hGH therapy 1 PLoS ONE 2011, 6(1), e16223
GHD - Not Only About Growth
Children with GHD suffer beyond growth failure1 Increased and abnormal fat distribution (especially truncal fat mass) Abnormal metabolic profile Impaired exercise capacity Decreased quality of life Adults with GHD2 Trunk fat accumulation and
decrease in lean body mass Decreased bone mineral density Dyslipidemia Increased cardiovascular mortality and morbidity Decreased quality of life Daily hGH addresses all the symptoms of the disease 1 BMC Endocrine Disorders 2012, 12:26 2 J Clin
Endocrinol Metab 2006, 91: 1621-1634 Long-acting GH must fully mimic daily hGH to address the totality of the disease
ADIPOSE TISSUE GH breakdown fat via
stimulation of GH receptors in fat, counteracting the lipogenesis effect of IGF-11 Tissue Specific Effects of GH and IGF-1 BONE Optimal growth is achieved via co-stimulation of GH and IGF-1 receptors in bone1 Increasing molecular size through
permanent protein modification alters the ability to distribute outside the blood compartment compared to unmodified drug2 Data continue to support that limiting the distribution of GH alters the therapeutic effect on trunk fat and growth velocity
compared to unmodified/daily hGH GH receptors are expressed on virtually every cell of the body1 IGF-1 in serum is primarily derived from the liver and regulated by GH1 1 J Clin Endocrinol Metab 2007, 92(012): 4529-4535 2 Digestive and liver
disease, 2004, 36 suppl.3, S334-39
Tissue Distribution Associated with
Optimal Profile 1 J Clin Endocrinol Metab 2007,12: 4529-4535 2 J Clin Endocrinol Metab1997, 82(2): 629-633 3 J Clin Endocrinol Metab 2005 90: 6431-40 4 Pituitary. 2013 Sep;16(3):311-8 Additional published data show that long-acting GH
formulations based on unmodified hGH and daily hGH are associated with reduction in fat mass3-4 (1) (2)
TransCon GH Designed to Address
Issues Raised by Academic and Regulatory Experts1 Predictable release of parent drug in the body hGH hGH TransCon carrier TransCon linker Inactive Prodrug Creation of TransCon Prodrug 1 European Journal of Endocrinology (2016) 174, C1-C8
Once-weekly prodrug releasing unmodified GH designed to mimic daily hGH: Tissue distribution Physiological levels Therapeutic effects
Growth Comparable to Daily hGH in
Phase 2 Study1 Same weekly dose 11.5 14.5 0.14 mg 0.21 mg 0.30 mg 0.21 mg Dose hGH/kg/week: 1 Intergroup differences not statistically significant. 2 Conducted with a previous lower strength version of TransCon Growth Hormone. 26-week treatment
period Two thorough PK/PD assessments at weeks 1 and 13 TransCon Growth Hormone2 Genotropin
Dose Proportional IGF-1 Elevation
Observed into the Normal Range in Phase 2 Study Days (Week 13) IGF-1 SDS (+ SEM) 0 1 2 3 4 5 6 7 Transient point values of IGF-1 SDS > +2.0 have been observed in a small number of patients primarily at the highest dose level
Comparable hGH Levels for TransCon
Growth Hormone and Daily hGH in Phase 2 Study Maximum hGH blood concentration comparable between equivalent weekly doses of TransCon Growth Hormone and daily hGH Days (Week 13) hGH serum concentration (+SEM, ng/ml)
Comparable Safety to Daily hGH in
Phase 2 >1100 TransCon Growth Hormone injections administered in the Phase 2 pediatric study No reports of lipoatrophy or nodule formation No Serious Adverse Events (SAEs) related to study drug Immunogenic profile comparable to daily hGH
Injection site tolerability comparable to daily hGH Adverse events consistent with daily hGH therapy observed and not different between cohorts Favorable immunogenic profile comparable to daily hGH No occurrence of neutralizing antibodies
Phase 3 Study - heiGHt Trial Ongoing
Key Inclusion Criteria Prepubertal children with GHD Height SDS -2.0 IGF-1 SDS -1.0 GHD with 2 GH stim. tests (GH 10 ng/mL) Bone age 6 months behind chronological TransCon Growth Hormone (0.24 mg/kg/week) Genotropin
(34 g/kg/d = 0.24 mg/kg/week) Screening 6 weeks Key Endpoints Annualized height velocity (HV) at 52 weeks (primary endpoint) Annualized HV at earlier time points Change in HT SDS over 52 weeks Change in serum IGF-1/IGFBP-3 levels Change
in IGF-1 SDS and IGFBP 3 SDS Normalization of IGF-1 SDS Long-Term Extension Study Week 1 Week 5 Week 13 Week 52 Week 26 Week 39 ~ 150 treatment-na ve children with GHD (2:1 randomization) VISIT SCHEDULE
Easy to Use Device with Optimal
Product Features Auto-injector for commercialization to be used in extension study Simple operation with few user steps A single low-volume injection for all patients (<0.6 mL) Small needle comparable to daily hGH (31G, 4mm) Room temperature
storage No waste due to empty-all design Enabled for Bluetooth connectivity to IT health care solutions Device lifetime at least 4 years Key Device Features
Efficacy Safety (including
immunogenicity) Tolerability TransCon Growth Hormone Target Product Profile Comparable to Daily Growth Hormone = Weekly subcutaneous administration Single injection/dose Convenience 31G needle Room temperature storage Device Easy to use Empty-all
design (controlled substance)
TransCon Growth Hormone: Highlights
Potential best-in-class long-acting growth hormone product profile Phase 3 heiGHt Trial recruiting and initial patients dosed; full enrollment expected 4Q 2017 Commercial scale manufacturing and supply chain established Auto-injector developed and
on-track for launch Multiple patent concepts provide potential protection into 2036
TransCon PTH: Once-Daily for
Hypoparathyroidism affects 77,000
patients in the U.S.1 Patients suffer numerous comorbidities: Hypocalcemia and hypercalcemia Hypercalciuria (stones, nephrocalcinosis) Psychiatric disorders, depression Reduced QOL Until recently, hypoparathyroidism remained among the few hormonal
insufficiency states not treated by the replacement of its missing hormone Natpara [PTH(1-84)] launched in 2015 as once-daily administration but incompletely addresses all aspects of the disease Hypoparathyroidism - Not Only About Serum
Calcium Basal ganglia/CNS calcifications Lenticular calcifications/cataracts Arterial calcifications/atherosclerosis 1 J Clin Endocrinol Metab 2016, 101(6): 2284-2299
FDA Perspective on Optimal PTH PK1 1
FDA presentation Natpara Advisory Committee September 12, 2014
Intermittent PTH Caused Decrease in
Cortical BMD Six years of therapy with Natpara was associated with a progressive decrease in cortical BMD1 Continuous exposure to PTH(1-34) restores bone turnover to normal levels while avoiding the overstimulation of daily or twice daily
injections2 TransCon PTH is expected to normalize bone turnover and BMD, due to continuous PTH exposure 1 J Clin Endocrinol Metab 2016 doi: 10.1210/jc. 2015-4135 2 J Clin Endocrinol Metab 2012, 97(2): 391-399 6 years of Natpara therapy decreases
cortical bone BMD Years of rhPTH(1-84) Treatment % Change from Baseline
Optimal PTH PK Improved Treatment
Outcomes Physiological Effect in Hypoparathyroidism Natpara Once-daily1, 2 PTH (1-34) Infusion3-6 Increase serum calcium Reduce pill burden Normalize urinary calcium excretion X Reduce clinical hypercalcemia X Reduce clinical hypocalcemia X
Normalize serum phosphate (high-normal range) Normalize bone turnover X (cortical bone loss) NIH clinical trials demonstrated superiority of continuous infusion > twice daily injections > once daily injections3-6 1 Natpara Product Label 2 J
Clin Endocrinol Metab 2016, 101(7): 2742-2750 3 J Clin Endocrinol Metab 2009, 93(9): 3389-3395 4 J Clin Endocrinol Metab 1998, 83(10): 3480-3486 5 J Clin Endocrinol Metab 2013, 88(9): 4214-4220 6 JAMA 1996, 276(8): 631-636
Designed Specifically for
Hypoparathyroidism Predictable release of parent drug in the body PTH(1-34) PTH(1-34) TransCon carrier TransCon linker Inactive Prodrug Creation of TransCon Prodrug TransCon PTH is a sustained-release prodrug, providing free PTH levels in the
physiological range over an entire 24 hours TransCon PTH designed to address limitations of current therapies to normalize: Serum and urinary calcium levels Serum phosphate levels Bone turnover
Total PTH [ng/mL] PK profile of
TransCon PTH in cynomolgus monkeys supports infusion-like profile following daily administration PTH levels following SC injections in cynomolgus monkeys (TransCon PTH 5 g/kg, n=3) Hours 0 4 8 12 16 20 24 28 32 36 40 44 48 0 10 20 30 40 50 60
Desired PK Profile Confirmed in Primates
TransCon PTH is designed to maintain
serum PTH levels in the physiological range, mimicking physiological exposure 1 Simulation based on 100 g QD Natpara and 70 g QD TransCon PTH; Simulation of 3 repeat doses of Natpara and TransCon PTH at steady state Physiological Range
Last updated: Jan 9, 2017