Ascendis Pharma A/s · NASDAQ:ASND
Ascendis Pharma A/s — 42nd Annual J.P. Morgan Healthcare Conference (January 2024)
28 slides from Ascendis Pharma A/s's conference presentation, Jan 8, 2024
Slide 1: Ascendis Pharma A/S – 42nd Annual J.P. Morgan Healthcare Conference
Ascendis Pharma A/S 42nd Annual J.P. Morgan Healthcare Conference January 8, 2024 For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 2: Cautionary Note on Forward-Looking Statements
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 products and prospective product candidates; clinical trial results; the expe
Slide 3: Executive Summary
Executive Summary • Validated TransCon technology platform and algorithm for product innovation – Two approved products – SKYTROFA® (TransCon hGH) and YORVIPATH® (TransCon PTH) – Pivotal data and submission of NDA for TransCon CNP expected in Q4 2024 – Oncology portfolio Phase 2 data expected in Q4
Slide 4: Vision 3x3: Building a Leading Global Biopharma Company
Vision 3x3: Building a Leading Global Biopharma Company Our goal is to achieve sustainable growth through multiple approaches ➢ Obtain regulatory approval for three independent Endocrinology Rare Disease products ⎯ TransCon™ hGH for pediatric growth hormone deficiency ⎯ TransCon™ PTH for adult hypop
Slide 5: Endocrinology Rare Disease Pipeline & Ascendis Direct Commercialization
Endocrinology Rare Disease Pipeline & Ascendis Direct Commercialization For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 6: Endocrinology Rare Disease Products Approved in Major Markets
Endocrinology Rare Disease Products Approved in Major Markets TransCon hGH • Pediatric Growth Hormone Deficiency • United States1 Approved in the U.S. as SKYTROFA (lonapegsomatropin-tcgd) • European Union2 and Selected Other Countries3 Approved in the EU as SKYTROFA (lonapegsomatropin) TransCon PTH
Slide 7: TransCon hGH: Expanding Beyond Pediatric GHD
TransCon hGH: Expanding Beyond Pediatric GHD • Pediatric GHD accounts for approximately 50% of the hGH market by patients • Growth hormone plays an essential role in the health of children and adults – Maintains normal body composition and cardiometabolic health – Promotes normal growth in children
Slide 8: SKYTROFA Revenue Development and Expectations
SKYTROFA Revenue Development and Expectations • SKYTROFA was launched in U.S. in Q4 2021, Germany in Q3 2023 – SKYTROFA Q4 2023 revenue 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** Q
Slide 9: TransCon hGH Program Summary and Outlook
TransCon hGH Program Summary and Outlook • Attained U.S. market value leadership in 2023 • Completed enliGHten Trial in pediatric GHD1 – Majority of patients met or exceeded average parental height SDS – Demonstrated the long-term safety in patients treated up to 6 years • Planned sBLA submission to
Slide 10: Hypoparathyroidism: One Disease, Multiple Consequences
Hypoparathyroidism: One Disease, Multiple Consequences 1-4 Absence or deficiency of parathyroid hormone (PTH) is linked to multi-organ manifestations5,6 CENTRAL NERVOUS SYSTEM RENAL OPHTHALMALOGICAL • Seizures • Nephrocalcinosis* • Cataracts • Basal ganglia calcifications • Nephrolithiasis* • Papill
Slide 11: Latest Clinical Practice Guideline
Latest Clinical Practice Guideline • Consider PTH replacement therapy in patients not adequately controlled on conventional therapy • Inadequate control is considered to be any one of the following: – Symptomatic hypocalcemia – Hyperphosphatemia – Renal insufficiency – Hypercalciuria – Poor quality
Slide 12: Significantly Improved Renal Function with TransCon PTH Treatment
Significantly Improved Renal Function with TransCon PTH Treatment in Phase 3 PaTHway Trial Post Hoc Analysis Patients Randomized Patients Randomized to Placebo / to TransCon PTH Switched to TransCon PTH after Week 26 At Week 26 At Week 52 At Week 26 At Week 52 ▲ Change in mean eGFR +11.4 ▲ +11.5 +11
Slide 13: Ascendis Commercial Organization - TransCon PTH Launch
Ascendis Commercial Organization - TransCon PTH Launch • Europe Direct1 – Germany – ~70,000 adults2 with chronic hypoparathyroidism in Germany – Launch of YORVIPATH planned in January 2024, initial list price3 of €105,000 per patient per year • Europe Direct – other selected countries – Over 100,000
Slide 14: TransCon PTH Program Summary and Outlook
TransCon PTH Program Summary and Outlook • Completed two successful Phase 3 trials: PaTHway and PaTHway Japan • Post hoc analysis showed significantly improved renal function in TransCon PTH-treated patients • Approved in European Union and other territories as YORVIPATH – German launch of YORVIPATH
Slide 15: Changing the Treatment Paradigm of Achondroplasia
Changing the Treatment Paradigm of Achondroplasia Current Consensus Updated View Primary Manifestations Growth disorder Growth disorder Muscle disorder Muscle Muscle Secondary Height Strength/ Height Strength/ Manifestations Stamina Stamina ACH-AE HRQoL ACH-AE HRQoL direct consequence indirect/secon
Slide 16: Treatment Effect of TransCon CNP 100 µg/kg/week, Age 2-10 years
Treatment Effect of TransCon CNP 100 µg/kg/week, Age 2-10 years TransCon CNP Placebo TransCon CNP 0.5 TransCon CNP Placebo TransCon CNP incl 1-year OLE incl 1-year OLE Blinded Period Blinded Period 6 0.3 AHV ACH Height (cm/Year) 5 SDS D 0.1 4 -0.1 N= 10 11 10 11 15 57 -0.3 10 11 10 11 15 57 3 N= Gro
Slide 17: Health-Related Quality-of-Life Measures (HRQoL)
Health-Related Quality-of-Life Measures (HRQoL) SF-10™ Health Survey for Children Achondroplasia Child Experience Measure A 10-item non-disease specific survey of a child’s A condition-specific clinical outcome measure that functional health and well-being.1 assesses the impact of achondroplasia on
Slide 18: Consistent Positive Treatment Effect Across HRQoL Domains
Consistent Positive Treatment Effect Across HRQoL Domains SF-10 Physical Summary Achondroplasia Child Experience Measure 25 20 p=0.002 p=0.047 p=0.045 20 10 Placebo better LS mean 15 LS mean 0 difference to difference to 100µg/kg placebo 10 placebo -10 better (95%CI) by (95%CI) by ANCOVA 5 ANCOVA -2
Slide 19: TransCon hGH / CNP Combination Trial - COACH Trial
TransCon hGH / CNP Combination Trial - COACH Trial • Phase 2 open-label, single-arm trial (age 2-11 years), Clinical Trial Application (CTA) submitted – Primary objectives: evaluate treatment effect on linear growth and safety – Secondary objectives: evaluate treatment effect on quality of life, rad
Slide 20: TransCon CNP Program Summary and Outlook
TransCon CNP Program Summary and Outlook • Pediatric patients: – Topline data from pivotal ApproaCH Trial expected Q4 2024 – COACH Trial (hGH/CNP combination) Week 26 topline data expected Q4 2024 – Plan to submit NDA to FDA in Q4 2024 for the treatment of achondroplasia • Infant patients (age 0-2 y
Slide 21: Global Commercial Presence Through Partners with Local Expertise and Infrastructure
Global Commercial Presence Through Partners with Local Expertise and Infrastructure For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 22: Growing Commercial Presence – Rest of the World
Growing Commercial Presence – Rest of the World • China – VISEN has exclusive license agreement for TransCon hGH, TransCon PTH, and TransCon CNP – VISEN’s TransCon hGH Phase 3 and TransCon CNP Phase 2 trials completed • Japan – Teijin has exclusive license agreement for TransCon hGH, TransCon PTH, a
Slide 23: Financial Update & 2024 Selected Milestones
Financial Update & 2024 Selected Milestones For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 24: Financial Update
Financial Update Preliminary unaudited 2023 results • SKYTROFA Q4 revenue of ~€64 million; full year 2023 SKYTROFA revenue of ~€179 million • Full year 2023 total revenue of ~€267 million – Includes $70 million Teijin upfront payment as non-product revenue in Q4 • December 31, 2023 cash, cash equiva
Slide 25: Selected Milestones Expected in 2024
The Year Ahead Selected Milestones Expected in 2024 Q1 Q1 2024 2024 Q2 Q2 2024 2024 Q3 Q3 2024 2024 Q4 Q4 2024 2024 TransCon PTH TransCon PTH TransCon PTH TransCon CNP TransCon hGH Adults with Chronic Adults with Chronic Adults with Chronic Achondroplasia (age 2-11) Turner Syndrome Hypoparathyroidis
Slide 26: Introducing Vision 2030
Introducing Vision 2030 For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 27: Vision 2030
Vision 2030 Achieve blockbuster status for multiple products and expand our engine for future innovation • Be the Leading Endocrinology Rare Disease Company • Achieve blockbuster status (>$1B) for TransCon PTH, TransCon hGH, and TransCon CNP through worldwide commercialization • Be the leader in Gro
Slide 28: Thank you!
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.
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