Ascendis Pharma A/s · NASDAQ:ASND
Ascendis Pharma A/s — Investor Presentation (May 2015)
25 slides from Ascendis Pharma A/s's investor presentation, May 13, 2015
Slide 1: Ascendis Pharma Company Presentation Q2 2015
Exhibit 99.1 1 Q2 2015 ASCENDIS PHARMA Jan Mikkelsen, President and CEO Company Presentation, Q2 2015
Slide 2: Cautionary Note on Forward Looking Statements
2 Q2 2015 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, business stra
Slide 3: Investment Highlights
3 Q2 2015 INVESTMENT HIGHLIGHTS TransCon technology is applied to create long-acting prodrugs with best-in-class profiles Technology & Pipeline Internal programs targeting multi-billion dollar orphan indications: - Growth disorders and pulmonary arterial hypertension Strong Capital to complete Phase
Slide 4: Experienced Leadership Team
4 Q2 2015 EXPERIENCED LEADERSHIP TEAM Management Board of Directors Jan Mikkelsen, Director, President and CEO Michael Wolff Jensen, LLM, LifeCycle Pharma (now known as Veloxis), Maxygen, Chairman, SVP & General Counsel Profound Pharma, Novo Nordisk Dong Energy, Veloxis, Genmab Martin Auster, MD, SV
Slide 5: TransCon Technology
5 Q2 2015 TRANSCON TECHNOLOGY TransCon carrier Predictable self-cleaving Parent of TransCon linker Parent TransCon linker drug drug Inactive long-acting prodrug Unmodified active parent drug released Long-acting prodrugs with predictable release of unmodified parent drugs Parent drugs can be protein
Slide 6: Broad, Balanced and Diverse Pipeline
6 Q2 2015 BROAD, BALANCED AND DIVERSE PIPELINE Approved Worldwide Parent Commercial Product Candidate Primary Indication Drug Stage of Development Market Size Rights Pediatric Growth Phase 2 TransCon hormone deficiency (Topline data expected mid-2015) > $3 billion Growth Hormone Adult Growth hormone
Slide 7: TransCon hGH: Once-weekly growth hormone
7 Q2 2015 TransCon hGH: Once-weekly growth hormone
Slide 8: Once-Weekly TransCon Growth Hormone
8 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 g
Slide 9: Growth Hormone Market
9 Q2 2015 GROWTH HORMONE MARKET 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
Slide 10: Poor Compliance Reduces Treatment Outcomes
10 Q2 2015 POOR COMPLIANCE REDUCES TREATMENT OUTCOMES Poor compliance with daily growth hormone therapy is associated with reduced 1 height velocity and impaired quality of life Two out of three of the patients miss more than one injection on average per week Improved growth response Higher complian
Slide 11: TransCon hGH: Long-Acting Unmodified Growth Hormone
11 Q2 2015 TRANSCON hGH: LONG-ACTING UNMODIFIED GROWTH HORMONE TransCon Growth Hormone is designed to provide the efficacy, safety and tolerability of daily hGH with once-weekly dosing Daily hGH treatment characteristics Height First-year height velocity Velocity >10 cm Metabolic Reduced adiposity a
Slide 12: TransCon hGH: Overview of Completed Studies
12 Q2 2015 TRANSCON hGH: OVERVIEW OF COMPLETED STUDIES Dose proportional response in hGH and IGF-I levels Phase 2 Adult GHD hGH & IGF-I Cmax and exposure comparable between equivalent weekly doses of TransCon hGH and daily hGH Phase 1 No treatment-emergent anti-hGH antibody formation High dose enabl
Slide 13: Phase 2 Study in GHD Children Ongoing
13 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) - Efficac
Slide 14: Patient Demographics Comparable to Other hGH Studies
14 Q2 2015 PATIENT DEMOGRAPHICS COMPARABLE TO OTHER hGH STUDIES Phase 2 pediatric study patient demographics are comparable to U.S. and EU studies of daily and long-acting growth hormone products TransCon hGH VRS-317 Lagova* Norditropin Weekly Semi-monthly Weekly Daily Key territory Europe United St
Slide 15: TransCon hGH Produces Robust Growth Comparable to Daily hGH
15 Q2 2015 TRANSCON hGH PRODUCES ROBUST GROWTH COMPARABLE TO DAILY hGH Same weekly dose TransCon hGH compared to Phase 2 pediatric study 6-month interim results Genotropin® control arm
Slide 16: Dose Proportional IGF-I Elevation into the Normal Range
16 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
Slide 17: Comparable hGH Levels for TransCon hGH and Daily hGH
17 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 resul
Slide 18: Safety Comparable Between TransCon hGH and Daily hGH
18 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 dai
Slide 19: TransCon hGH May Improve Real-World Treatment Outcomes
19 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 TransCon hGH Genotropin(KIGS) 0.21 mg/kg/wk Prescr
Slide 20: Long-Acting Growth Hormone Programs
20 Q2 2015 LONG-ACTING GROWTH HORMONE PROGRAMS Ascendis Versartis OPKO Novo 11.9 - 14.5 cm 8.7 cm 12.2-13.6 cm Height velocity No data As daily hGH No active control As daily hGH Mode of action: Unmodified hGH Yes No No No comparable to daily hGH Growth hormone blood levels No No No Yes comparable t
Slide 21: Summary TransCon Growth Hormone
21 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 o
Slide 22: TransCon Treprostinil: Once-daily administration
22 Q2 2015 TransCon Treprostinil: Once-daily administration
Slide 23: Once-Daily TransCon Treprostinil
23 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 trepro
Slide 24: Investment Highlights
24 Q2 2015 Investment Highlights
Slide 25: Investment Highlights
25 Q2 2015 INVESTMENT HIGHLIGHTS 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 appl