 ASCENDIS PHARMA Jan Mikkelsen, President and CEO Company Presentation, Q2 2015 1 Q2 2015 Exhibit 99.1 |
 CAUTIONARY NOTE ON FORWARD LOOKING STATEMENTS: 2 Q2 2015 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, business strategy, prospective products, availability of funding, clinical trial results (including the timing of data from our ongoing Phase 2 pediatric study of TransCon hGH), 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.” 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 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. |
 INVESTMENT HIGHLIGHTS *USD/EUR=1.076 as of March 31, 2015 TransCon technology is applied to create long-acting prodrugs with best-in-class profiles Internal programs targeting multi-billion dollar orphan indications: - Growth disorders and pulmonary arterial hypertension Capital to complete Phase 3 study of TransCon Growth Hormone March 31 , 2015 cash balance: ~€153 million* High-value collaborations with Large Cap Pharma in their key strategic therapeutic areas - Roche Genentech in ophthalmology - Sanofi in diabetes Existing and future collaborations are a potential source of significant revenue - Approximately €202 million* in development and regulatory milestones for three product candidates - Eligible for royalties from mid-single to low teen digit percentages from ophthalmology collaboration All TransCon products eligible for new composition of matter IP TransCon technology is broadly applicable with no third-party royalty or milestone payment obligations Technology & Pipeline Strong Balance Sheet Partners Partner Revenue Intellectual Property 3 Q2 2015 st |
 EXPERIENCED LEADERSHIP TEAM 4 Jan Mikkelsen, Director, President and CEO LifeCycle Pharma (now known as Veloxis), Maxygen, Profound Pharma, Novo Nordisk Martin Auster, MD, SVP & CBO United Therapeutics, Wachovia, GLG Partners Michael Wolff Jensen, LLM, Chairman, SVP & General Counsel Dong Energy, Veloxis, Genmab Grethe Rasmussen, PhD, SVP Development Maxygen, Novo Nordisk Dr. Harald Rau, SVP & CSO Complex Biosystems Thomas P. Soloway, SVP & CFO Transcept, Montreux Equity Partners Lotte Sønderbjerg, SVP & CAO Veloxis, Acadia, Novo Nordisk Management Michael Wolff Jensen, LLM, Chairman, SVP & General Counsel Dong Energy, Veloxis, Genmab Albert Cha, MD, PhD Vivo Capital, Managing Partner Edwin de Graaf Gilde Healthcare Partners, Managing Partner James Healy, MD, PhD Sofinnova Ventures, General Partner Michael Mayer TechnoStart, Managing Partner Jan Mikkelsen, President and CEO LifeCycle Pharma (now known as Veloxis), Maxygen, Profound Pharma, Novo Nordisk Martin Olin Symphogen A/S, CFO Jonathan Silverstein, JD Orbimed, Co-Head of Global Private Equity Rafaèle Tordjman, MD, PhD Sofinnova Partners, Managing Partner Board of Directors Q2 2015 |
 TRANSCON TECHNOLOGY Predictable self-cleaving of TransCon linker Parent drug Inactive long-acting prodrug Unmodified active parent drug released Long-acting prodrugs with predictable release of unmodified parent drugs Parent drugs can be proteins, peptides or small molecules Predictable release of parent drug supporting up to half-yearly administration TransCon linker release dependent only on pH and temperature Enables systemic or localized drug exposure depending upon choice of TransCon carrier Same mode-of-action as parent drug molecule Potential to improve development success rate when incorporating approved parent drug TransCon carrier TransCon linker Parent drug Parent drug 5 Q2 2015 |
 BROAD, BALANCED AND DIVERSE PIPELINE Product Candidate Primary Indication Approved Parent Drug Stage of Development Market Size Worldwide Commercial Rights TransCon Growth Hormone Pediatric Growth hormone deficiency Phase 2 (Topline data expected mid-2015) > $3 billion Adult Growth hormone deficiency Phase 2 completed TransCon Treprostinil Pulmonary Arterial Hypertension Phase 1 > $1 billion TransCon Ranibizumab Ophthalmology Preclinical > $5 billion TransCon Peptide Diabetes Preclinical n/a 6 Q2 2015 |
 TransCon hGH: Once-weekly growth hormone 7 Q2 2015 |
 ONCE-WEEKLY TRANSCON GROWTH HORMONE The daily hGH market is a $3+ billion orphan opportunity Current therapies require daily injections, which can result in poor compliance and suboptimal treatment response 1 TransCon Growth Hormone is a long-acting prodrug designed to release unmodified growth hormone in the bloodstream Maintains the same mode-of-action as daily hGH Efficacy, safety, tolerability and immunogenic profile comparable to daily hGH Phase 2 study in adults with growth hormone deficiency: Comparable dose response and tolerability to daily hGH therapy Phase 2 study in children with growth hormone deficiency ongoing 1 PLoS ONE 2011, 6(1), e16223 8 Q2 2015 |
 Global sales of daily hGH products exceeded $3 billion in 2013 The daily hGH market is fragmented and undifferentiated Novo Nordisk, Pfizer, Eli Lilly, Sandoz, Merck KGaA and Roche account for approximately 95% of volume market share Pediatric indications comprise up to 90% of the market Indications for growth hormone treatment include: Growth Hormone Deficiency (GHD) Turner Syndrome (TS) Idiopathic short stature (ISS) Prader-Willi Syndrome (PWS) Small for Gestational Age (SGA) GROWTH HORMONE MARKET Ascendis market research 9 Q2 2015 |
 POOR COMPLIANCE REDUCES TREATMENT OUTCOMES Poor compliance with daily growth hormone therapy is associated with reduced height velocity and impaired quality of life Two out of three of the patients miss more than one injection on average per week Reduced frequency of administration is associated with better compliance Once-weekly TransCon Growth Hormone may improve compliance and overall treatment outcomes 1 PLoS ONE 2011, 6(1), e16223 2 Clinical Therapeutics, 2008, 30(2),307 Doses missed per week Higher compliance Improved growth response 10 Q2 2015 1 2 |
 TRANSCON hGH: LONG-ACTING UNMODIFIED GROWTH HORMONE Q2 2015 11 TransCon Growth Hormone is designed to provide the efficacy, safety and tolerability of daily hGH with once-weekly dosing Height Velocity Metabolic effects Quality of Life Safety Injection Device Daily hGH treatment characteristics TransCon hGH prodrug releases unmodified hGH First-year height velocity >10 cm Reduced adiposity and improved cardiovascular health Improved Safe and well-tolerated Essentially pain-free < 1.0 mL per injection Convenient and easy to use |
 TRANSCON hGH: OVERVIEW OF COMPLETED STUDIES Dose proportional response in hGH and IGF-I levels hGH & IGF-I Cmax and exposure comparable between equivalent weekly doses of TransCon hGH and daily hGH No treatment-emergent anti-hGH antibody formation Transient and generally mild injection site reactions comparable to daily hGH PK and PD data support once-weekly administration Phase 1 First in man Phase 1 High dose enabling Phase 2 Adult GHD 12 Q2 2015 |
 PHASE 2 STUDY IN GHD CHILDREN ONGOING Six-month Phase 2 study in GHD children of TransCon hGH vs. daily hGH - Multicenter, randomized, open label, active-controlled, parallel-group study investigating the safety, tolerability and efficacy in pre-pubertal children with GHD (n=53) - Efficacy endpoint: 6-month mean height velocity - Enrolled children meet internationally recognized guidelines, similar enrollment criteria expected in Phase 3 design - Doses of 0.14 mg, 0.21 mg and 0.30 mg hGH/kg/week administered once-weekly versus daily hGH equivalent to 0.21 mg hGH/kg/week - Study being conducted across Europe and North Africa Positive interim update reported December 2014 (n=25) Fully enrolled: n=53 Top-line study results anticipated mid-2015 13 Q2 2015 |
 Phase 2 pediatric study patient demographics are comparable to U.S. and EU studies of daily and long-acting growth hormone products PATIENT DEMOGRAPHICS COMPARABLE TO OTHER hGH STUDIES Q2 2015 14 VRS-317: Versartis corporate presentation Jan. 2015 Lagova: OPKO presentation ENDO 2014 Norditropin: JCEM 2002 87: 90-98 * Ascendis analysis using weighted average of cohorts 1-4 TransCon hGH Weekly VRS-317 Semi-monthly Lagova* Weekly Norditropin Daily Key territory Europe United States Europe United States Number of subjects 53 64 50 104 Age 7.8 7.8 6.1 7.8 Height SDS -3.1 -2.5 -3.9 -3.0 GH stimulation test 5.0 5.4 3.9 4.9 IGF-I SDS -2.2 -1.7 -2.1 -2.8 |
 TRANSCON hGH PRODUCES ROBUST GROWTH COMPARABLE TO DAILY hGH TransCon hGH compared to Genotropin ® control arm Same weekly dose Phase 2 pediatric study 6-month interim results 15 Q2 2015 |
 DOSE PROPORTIONAL IGF-I ELEVATION INTO THE NORMAL RANGE Transient point values of IGF-I SDS > +2 have been observed in a small number of patients and only in the high-dose treatment arm Phase 2 pediatric study 6-month interim results 16 Q2 2015 |
 COMPARABLE hGH LEVELS FOR TRANSCON hGH AND DAILY hGH Maximum hGH blood concentration is comparable between equivalent weekly doses of TransCon hGH and daily hGH (0.21 mg/kg/week) Dose-proportionality observed for TransCon hGH across dose range Phase 2 pediatric study 6-month interim results 17 Q2 2015 |
 SAFETY COMPARABLE BETWEEN TRANSCON hGH AND DAILY hGH No Serious Adverse Events (SAEs) related to study drug Adverse events consistent with hGH therapy observed and not different between cohorts Low immunogenicity comparable to daily hGH Transient injection site reactions comparable to daily hGH - >950 TransCon hGH injections administered in the Phase 2 pediatric study - No reports of lipoatrophy or nodule formation Phase 2 pediatric study 6-month interim results 18 Q2 2015 |
 TRANSCON hGH MAY IMPROVE REAL-WORLD TREATMENT OUTCOMES Phase 2 interim results demonstrate improved height velocity compared to age-matched historical controls (KIGS database) Improved height velocity translates into improved adult height Sources: Ranke JCEM 2010 KIGS database/CDC/Company analysis TransCon hGH 6-month HV vs. KIGS 12-month HV No statistical comparison conducted between the two studies Genotropin (KIGS) Prescribed 0.22 mg/kg/wk TransCon hGH 0.21 mg/kg/wk 19 Q2 2015 |
 LONG-ACTING GROWTH HORMONE PROGRAMS 20 Ascendis Versartis OPKO Novo Height velocity 11.9 - 14.5 cm As daily hGH 8.7 cm No active control 12.2-13.6 cm As daily hGH No data Mode of action: Unmodified hGH comparable to daily hGH Yes No No No Growth hormone blood levels comparable to daily hGH Yes No (Supra-physiological) No (Supra-physiological) No (Supra-physiological) Injection volume <1 mL for most GHD patients Yes No Yes Yes Injection frequency Weekly Every two weeks Weekly Weekly Source: Versartis, OPKO, Novo Q2 2015 |
 SUMMARY TRANSCON GROWTH HORMONE Efficacy, safety profile and mode-of-action comparable to daily hGH with once-weekly administration TransCon hGH has the potential to improve real-world treatment outcomes One Phase 3 study in GHD children may enable approval for several indications based on 505(b)(2) strategy Clinical milestones: – Phase 2 pediatric study: Top-line data expected mid-2015 – Phase 3 pediatric study: Planned initiation of 12-month height velocity trial in mid-2016 Strong IP position with patent life extending until 2035 21 Q2 2015 |
 TransCon Treprostinil: Once-daily administration 22 Q2 2015 |
 ONCE-DAILY TRANSCON TREPROSTINIL Global sales of prostacyclin therapy for Pulmonary Arterial Hypertension (PAH) represented a ~$1.2 billion orphan drug market in 2013 TransCon Treprostinil is being designed as a long-acting prodrug to be inactive and continuously release unmodified treprostinil after administration To maintain same mode-of-action as treprostinil and preserves the benefits of continuous exposure To address the administrative burden that prevents patient and physician acceptance of currently approved prostacyclin therapies Phase 1 proof-of-concept study completed Demonstrated predictable, extended release of unmodified treprostinil supporting infusion-like pharmacokinetics Injection-site tolerability of TransCon Treprostinil did not meet the criteria defined in the target product profile New product formulations are currently being explored to improve injection site tolerability 23 Q2 2015 |
 Investment Highlights 24 Q2 2015 |
 INVESTMENT HIGHLIGHTS Q2 2015 25 Internal pipeline focused on developing best-in-class products for orphan markets TransCon Growth Hormone: Phase 2 pediatric study top-line results expected mid 2015 High-value partnerships in place Roche Genentech in ophthalmology and Sanofi in diabetes Broadly applicable and proven TransCon technology platform Validated with proteins, peptides and small molecules Strong financial position Capitalized to complete Phase 3 study of TransCon Growth Hormone |