Ascendis Pharma A/s · NASDAQ:ASND
Ascendis Pharma A/s — J.P. Morgan Healthcare Conference 2025
31 slides from Ascendis Pharma A/s's conference presentation, Jan 13, 2025
Slide 1: Ascendis Pharma A/S J.P. Morgan Healthcare Conference
Exhibit 99.1 Ascendis Pharma A/S J.P. Morgan Healthcare Conference San Francisco January 2025 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; revenue and growth expectations;
Slide 3: Ascendis Positioned to Drive Rapid Revenue Growth
Ascendis Positioned to Drive Rapid Revenue Growth • Three clinically validated Endocrinology Rare Disease medicines: ® 1 ◦ SKYTROFA – Approved for pediatric growth hormone deficiency (GHD) 2 - Full-year 2024 SKYTROFA revenue expected to be ~€202 million , excluding sales deductions related to prior
Slide 4: 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 each of TransCon PTH, TransCon hGH, and TransCon CNP through worldwide commercialization • Be the leader
Slide 5: Overview of TransCon Technologies
Overview of TransCon Technologies TransCon Linker Aromatic Cyclic Imide DKP Carbamate Bicin AEG Pyroglutamate TransCon TransCon Prodrug: Carrier 3 components Soluble Carriers Insoluble Carriers Albumin Avidity Parent Drug Antibodies, Antibody Fragments, Proteins, Peptides and Small Molecules Protein
Slide 6: TransCon Protein Degrader Technology
TransCon Protein Degrader Technology • TransCon protein degrader technology designed to enable efficient clearance of hormones, cytokines, and other targets by utilizing hepatic scavenger receptors Liver rapidly binds, internalizes TransCon Protein Degrader and degrades the (long half-life) degrader
Slide 7: TransCon Clinical Development Pipeline
TransCon Clinical Development Pipeline Endocrinology Rare Diseases Indication Status Region 1 Lead Indication TransCon CNP• Achondroplasia (children aged 2–11)• Pivotal• Multinational 2 Label Expansion• Adult Growth Hormone Deficiency• sBLA submitted• Multinational TransCon hGH 3 • Turner Syndrome (
Slide 8: Growing Worldwide Commercialization in Endocrinology Rare Disease
Growing Worldwide Commercialization in 1 Endocrinology Rare Disease Multiple approaches: • Ascendis direct commercialization in the U.S. and select European countries • Through partners with local expertise and infrastructure outside of U.S. and Europe 2 Direct Ascendis direct commercialization Excl
Slide 9: SKYTROFA TransCon hGH
® SKYTROFA TransCon hGH Once-weekly growth hormone therapy For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 10: SKYTROFA - High Value Growth Hormone Brand
SKYTROFA - High Value Growth Hormone Brand • Launched for pediatric growth hormone deficiency SKYTROFA Revenue (€ million) in U.S. in Q4 21, Germany in Q3 23 ◦ Successful Phase 3 riGHt Trial in Japan € 225 1 € 202 ◦ Planned expansion across multiple countries € 200 € 179 € 175 • Planned label expans
Slide 11: TransCon hGH Program Summary and Outlook
TransCon hGH Program Summary and Outlook 1 • 2024 SKYTROFA revenue, with single indication in pediatric GHD, expected to be ~€202 million ◦ High-value brand in the U.S., revenue per patient of around 3 times that of daily growth hormone 2 ◦ Volume (mg) increased 84% in 2024 resulting in 6.5% market
Slide 12: YORVIPATH TransCon PTH
® YORVIPATH TransCon PTH Treatment of hypoparathyroidism in adults For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 13: Unmet Medical Need in Hypoparathyroidism
Unmet Medical Need in Hypoparathyroidism: 1-4 One Disease, Multiple Consequences 5,6 Absence or deficiency of parathyroid hormone (PTH) is linked to multi-organ manifestations Central Nervous System Renal Ophthalmological • Nephrocalcinosis*• Cataracts • Seizures • Papilledema • Basal Ganglia Calcif
Slide 14: Current Clinical Practice Guideline
Current 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 15: YORVIPATH Commercial Update
YORVIPATH Commercial Update • Europe Direct YORVIPATH Revenue (€ million) ◦ Commercial launch in Germany and Austria in January 2024, with initial list price of €105,000 per patient per year ◦ Outside Germany and Austria, providing commercial product € 15.0 through early access routes, such as ‘name
Slide 16: YORVIPATH U.S. FDA Approved and Now Commercially Available
® YORVIPATH U.S. FDA Approved and Now Commercially Available • YORVIPATH is the first and only product indicated for the treatment of 1 hypoparathyroidism in adults • Commercially available in the U.S. December 2024 1. Limitations of Use: YORVIPATH was not studied for acute post-surgical hypoparathy
Slide 17: Hypoparathyroidism: U.S. Patient Population
Hypoparathyroidism: U.S. Patient Population Total U.S. Prevalence PTH Newly 1,3 ~25k–32k 70,000–90,000 Experienced Diagnosed • Currently on active 2 ~4,000 ~25k–– 5,000 32k vitamin D and calcium 3 ~3,~25k 000 ann–32k ually (“conventional therapy”) • Patients previously • Post-surgical, auto- treated
Slide 18: U.S. YORVIPATH Enrollment Since December 2024
U.S. YORVIPATH Enrollment Since December 2024 • New prescriptions enrolled in A.S.A.P. or direct with specialty pharmacy (n=324) as of January 9, 2025 Existing patients from Patients new to TransCon PTH EAP 59% YORVIPATH 41% or Clinical Trials Majority of new YORVIPATH patients are switching from co
Slide 19: TransCon PTH Program Summary and Outlook
TransCon PTH Program Summary and Outlook • First and only approved product indicated for the treatment of hypoparathyroidism in adults ◦ Clinical trial results consistent across geographies, ethnicities, and etiologies ◦ TransCon PTH under regulatory review for market authorization in Australia, Isr
Slide 20: TransCon CNP
® YORVIPATH TransCon CNP TransCon PTH (navepegritide) Investigatio Treatment of nal Hypo prod parath rug of yroidism CNP designed to provide in sustained Adults release and continuous exposure of active CNP for the treatment of pediatric achondroplasia TransCon CNP is an investigational product cand
Slide 21: Pivotal ApproaCH Trial Summary
Pivotal ApproaCH Trial Summary • The pivotal ApproaCH Trial in children aged 2-11 years achieved primary objective ◦ TransCon CNP demonstrated LS mean AGV of 5.89 cm/year with LS mean treatment difference of 1.49 cm/year at Week 52 compared to placebo (p<0.0001) ◦ For children aged 5-11 years TransC
Slide 22: Impact of TransCon CNP on Leg Bowing
Impact of TransCon CNP on Leg Bowing Leg bowing is a common complication in achondroplasia, resulting in pain, impaired physical functioning, 1,2,3 need for corrective surgery, and a negative impact on quality of life Mechanical Axis Deviation (MAD, mm) Tibial Femoral Angle (TFA, degrees) Absolute C
Slide 23: TransCon CNP Program Summary and Outlook
TransCon CNP Program Summary and Outlook • In pivotal ApproaCH Trial, TransCon CNP demonstrated significant improvements in linear growth and body proportionality, as well as benefits beyond linear growth ◦ Following pre-NDA meeting, planned filings for treatment of children with achondroplasia in U
Slide 24: Expanding the Endocrine Rare Disease Pipeline and Beyond
Expanding the Endocrine Rare Disease Pipeline and Beyond For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 25: X-Linked Hypophosphatemia
X-Linked Hypophosphatemia Background Limitations of Existing Therapy • Increase in serum phosphorous from baseline • Characterized by excess FGF-23 production leading to 1,2 plateaus at 1.0-1.5 mg/dL, irrespective of burosumab excessive renal phosphate wasting 3 drug exposure • Affecting ~1:20000 •
Slide 26: Collaboration with Novo Nordisk
Collaboration with Novo Nordisk • Development and commercialization of TransCon Technology-based products in metabolic (including obesity and type 2 diabetes) and cardiovascular diseases • Highlights of the multi-product collaboration: ◦ Upfront fee of $100 million for the exclusive license and fund
Slide 27: Other Investments and Partnerships
Other Investments and Partnerships • Eyconis (exclusive global rights for TransCon based ophthalmology products) ◦ Formed Eyconis in January 2024 with a $150 million commitment from external investors • Teijin (exclusive rights for TransCon hGH, PTH, and CNP in Japan) ◦ In December 2024, submitted a
Slide 28: Financial Update & 2025 Selected Milestones
Financial Update & 2025 Selected Milestones For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 29: 2024 Financial Update
2024 Financial Update 1 • Total full-year 2024 product revenue expected to be ~€226 million, driven by: 2 ◦ SKYTROFA full-year 2024 revenue expected to be ~€202 million, excluding sales deductions related to prior years 1 ◦ YORVIPATH full-year 2024 revenue expected to be ~€29 million 1 • Full-year 2
Slide 30: Selected Milestones Expected in 2025
Selected Milestones Expected in 2025 Q1 2025 Q2 2025 Q3 2025 Q4 2025 TransCon CNP TransCon CNP TransCon CNP TransCon CNP Submit NDA to the FDA Topline Week 26 data from Submit MAA to the EMA Submit IND or similar for COACH hypochondroplasia TransCon hGH TransCon hGH Adult GHD FDA Decision Adult GHD
Slide 31: Thank you
Thank you Investor Relations ir@ascendispharma.com https://investors.ascendispharma.com/ © 2025 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.
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