Full Press Release Details
Francisco January 2025 For investor communication only. Not for use in product promotion. Not for further distribution.
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; PDUFA goal dates;
clinical trial results; the expected timing of future clinical trial results, regulatory filings and feedback from regulatory authorities; the scope, progress, results and costs of developing our product candidates or any other future product
candidates; timing and likelihood of success; patient enrollment; timing and expansion of commercial launches, pipelines, and investments; potential payments and royalties relating to investments and partnerships; plans and objectives of management
for future operations and commercialization and manufacturing activities; and future results of current and/or anticipated products and product candidates 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 ("SEC"), including, without limitation, our prospectus
supplement filed on September 20, 2024 and our current and future reports filed with or submitted to the SEC, including our most recent Annual Report on Form 20-F filed with the SEC on February 7, 2024, particularly in the sections titled
"Risk Factors" and "Operating and Financial Review and Prospects." 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,
changed circumstances or otherwise after the date of this presentation. This presentation concerns Ascendis Pharma approved products as well as Ascendis Pharma investigational 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 investigational product candidates are currently limited by law to investigational use,
and no representations are made as to their safety or effectiveness for the purposes for which they are being investigated. This presentation is for investor and insurance carrier communications only. Not for use in product promotion. This
presentation also contains estimates and other statistical data made by independent parties and by us relating to market size and other data about our industry. This data involves a number of assumptions and limitations, and you are cautioned not to
give undue weight to such data and estimates. In addition, projections, assumptions and estimates of our future performance and the future performance of the markets in which we operate are necessarily subject to a high degree of uncertainty and
risk. This presentation contains trademarks, services marks, trade names and copyrights of the company and other companies, which are the property of their respective owners. The use or display of third parties' trademarks, service marks,
trade name or products in this presentation is not intended to, and does not imply, a relationship with the company, or an endorsement of sponsorship by the company. 2 For investor communication only. Not for use in product promotion. Not for
further distribution.
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 years - sBLA submitted for the treatment of adults with GHD, with a PDUFA goal date of July 27, 2025 1 YORVIPATH - Approved for the treatment of hypoparathyroidism in adults - Launched January 2024 in
Germany/Austria; available in the U.S. since late December 2024 3 - Full-year 2024 YORVIPATH revenue expected to be ~ 29 million TransCon CNP - Successful pivotal data in achondroplasia - Following pre-NDA meeting, planned
filings for treatment of children with achondroplasia in U.S. Q1 2025, EU in Q3 2025 Expanding TransCon technology platform with broad therapeutic applicability Formed Eyconis for Ophthalmology and partnered with Novo Nordisk for
Metabolic and Cardiovascular diseases Internal development for Oncology with focus on TransCon IL-2 New TransCon protein degrader technology to expand pipeline with additional potential blockbusters Business model
built on fast, successful drug development and commercial therapeutic synergies 1. Approved in the U.S., EU, and other territories, including Norway, Iceland, Liechtenstein, and Great Britain (covering England, Wales, Scotland). 2. Calculated as
unaudited preliminary estimate of full year 2024 SKYTROFA revenue of ~ 197 million plus ~ 5 million of sales deductions related to prior years. 3. Unaudited preliminary estimate. 3 For investor communication only. Not for use in product
promotion. Not for further distribution.
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 in
Growth Disorders and Hypoparathyroidism, pursuing clinical conditions, innovative LCM, and complementary patient offerings Expand pipeline with Endocrinology Rare Disease blockbuster product opportunities Create Value in Additional
Therapeutic Areas through Innovative Business Models Obtain accelerated approval in Oncology with registrational trials ongoing Pursue TransCon product opportunities in >$5B indications Maximize value creation of these
product opportunities through collaboration with therapeutic area market leaders Differentiate with Ascendis Fundamentals Outperform industry drug development benchmarks with Ascendis' product innovation algorithm Remain
independent as a profitable biopharma through lean and flexible ways of working Let our values Patients, Science, Passion drive our decisions to success Ascendis Pharma's 2025 - 2030 strategic roadmap 4 For investor communication only.
Not for use in product promotion. Not for further distribution.
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 Degraders Continued
expansion of TransCon technologies, enabling new product candidates 5 For investor communication only. Not for use in product promotion. Not for further distribution.
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-ligand
complex Protein Degrader (short half-life) Ligand to be degraded Linker cleavage dependent upon pH and temperature Enabling product opportunities for diseases characterized by excess hormones, cytokines, etc. 6 For investor communication only. Not
for use in product promotion. Not for further distribution.
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 (children aged 1-10) Phase 2 U.S. 4 TransCon CNP Achondroplasia (infants) Phase 2 Multinational 5 TransCon CNP Achondroplasia (adolescents) Phase 2
Multinational 6 TransCon CNP + TransCon hGH Achondroplasia (children aged 2-11) Phase 2 Multinational 7 Partner Programs TransCon hGH Pediatric GHD BLA submitted China 8 TransCon hGH Pediatric
GHD Phase 3 Japan 9 TransCon PTH Hypoparathyroidism in adults Phase 3 China 10 TransCon PTH Hypoparathyroidism in adults J-NDA submitted Japan 11 TransCon CNP Achondroplasia Phase
2 China Oncology Indication Status Region 12,13 Lead Indication TransCon IL-2 Various tumor types Phase 2 Multinational th 1. Pivotal ApproaCH Trial (NCT05598320). 2. sBLA submitted to U.S. FDA, PDUFA
goal date July 27 2025. 3. New InsiGHTS Trial (NCT05690386). 4. reACHin Trial (NCT06079398). 5. teACH (NCT06732895). 6. COACH Trial (NCT06433557). 7. VISEN Pharmaceuticals' Phase 3 trial. 8. Japanese riGHt Trial. 9. PaTHway China. 10. PaTHway
Japan. 11. ACcomplisH China. 12. BelieveIT-201 Trial (NCT05980598). 13. IL-Believe Trial (NCT05081609). 7 For investor communication only. Not for use in product promotion. Not for further distribution.
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 Exclusive
distribution agreement Strategic investment and exclusive license agreement Exclusive license agreement 1. As of December 31, 2024. 2. DACH, France & BeNeLux, Iberia, Italy, Nordics, UK & Ireland. 8 For investor communication only. Not for
use in product promotion. Not for further distribution.
SKYTROFA TransCon hGH Once-weekly growth hormone therapy For
investor communication only. Not for use in product promotion. Not for further distribution.
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 expansion to drive growth 150 sBLA accepted for adult growth hormone 125 deficiency; PDUFA goal date of July 27, 2025 100 Positive topline results from Phase 2 New
75 InsiGHTS Trial in Turner syndrome 50 36 Submit IND or similar for a basket trial evaluating 2 25 additional indications in Q3 2025 1 - 2021 2022 2023 2024E Committed to making SKYTROFA the
global leading product in value in a growing growth hormone market sBLA = supplemental Biologics License Application. 1. Calculated as unaudited preliminary estimate of full year 2024 SKYTROFA revenue of ~ 197 million plus ~ 5 million of
sales deductions related to prior years. 2. Planned for small for gestational age without catch-up growth ["SGA"]; Idiopathic short stature ["ISS"]; SHOX deficiency (including Turner syndrome). 10 For investor communication
only. Not for use in product promotion. Not for further distribution.
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 share of total U.S. growth hormone market Adult GHD sBLA submitted to FDA; PDUFA goal date of July 27, 2025 U.S. commercial launch planned in Q4 2025, pending approval Turner syndrome Phase 2 New
InsiGHTS Trial achieved primary objective at Week 26 3 Plan to submit IND or similar for basket trial evaluating additional indications in Q3 2025 Planned commercial launches across multiple indications and countries Committed to
making SKYTROFA a blockbuster product 1. Calculated as unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~ 197 million plus ~ 5 million of sales deductions related to prior years. 2. Based on third party prescription
data for 2024. 3. Planned for small for gestational age without catch-up growth ["SGA"]; Idiopathic short stature ["ISS"]; SHOX deficiency (including Turner syndrome). 11 For investor communication only. Not for use in
product promotion. Not for further distribution.
YORVIPATH TransCon PTH Treatment of hypoparathyroidism in adults
For investor communication only. Not for use in product promotion. Not for further distribution.
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 Calcifications Nephrolithiasis* Chronic Kidney Disease* Parkinsonism Or Dystonia Dental Respiratory Peripheral Nervous System Altered Tooth Morphology Laryngospasm Paresthesia Muscle
Cramps Dermatological Pain Cardiovascular Tetany Dry Skin Vascular Calcifications Thinning Hair Cardiac Arrhythmias Brittle Nails Neuropsychiatric Hypocalcemia-associated Dilated
Cardiomyopathy Anxiety & Depression Musculoskeletal Cognitive Impairment ("Brain Fog") Myopathy Spondyloarthropathy * These manifestations are mostly the result of management with active vitamin D and
calcium rather than of the disease itself. 1. Underbjerg L, Sikjaer T, Mosekilde L, et al. Cardiovascular and renal complications to postsurgical hypoparathyroidism: a Danish nationwide controlled historic follow-up study. J Bone Miner Res.
2013;28(11):2277-2285. doi:10.1002/jbmr.1979. 2. Underbjerg L, Sikjaer T, Mosekilde L, et al. The epidemiology of nonsurgical hypoparathyroidism in Denmark: a nationwide case finding study. J Bone Miner Res. 2015;30(9):1738-1744.
doi:10.1002/jbmr.2501. 3. Shoback DM, Bilezikian JP, Costa AG, et al. Presentation of hypoparathyroidism: etiologies and clinical features. J Clin Endocrinol Metab. 2016;101(6):2300-2312. doi:10.1210/jc.2015-3909. 4. Underbjerg L, Sikjaer T,
Mosekilde L, et al. The epidemiology of nonsurgical hypoparathyroidism in Denmark: a nationwide case finding study. J Bone Miner Res. 2015;30(9):1738-1744. doi:10.1002/jbmr.2501. 5. Mannstadt M, Bilezikian JP, Thakker RV, et al. Hypoparathyroidism.
Nat Rev Dis Primers. 2017;3:17055. doi:10.1038/nrdp.2017.55. 6. Shoback DM, Bilezikian JP, Costa AG, et al. Presentation of hypoparathyroidism: etiologies and clinical features. J Clin Endocrinol Metab. 2016;101(6):2300-2312.
doi:10.1210/jc.2015-3909. 13 For investor communication only. Not for use in product promotion. Not for further distribution.
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 of life In addition, individuals with poor compliance, malabsorption, or who are intolerant of large doses of calcium and active vitamin D may also benefit from PTH replacement therapy Vast majority of
patients with hypoparathyroidism fulfill guideline for PTH replacement therapy Khan AA, Bilezikian JP, Brandi ML, et al. Evaluation and Management of Hypoparathyroidism Summary Statement and Guidelines from the Second International Workshop. J Bone
Miner Res. Dec 2022;37(12):2568-2585. doi:10.1002/jbmr.4691 14 For investor communication only. Not for use in product promotion. Not for further distribution.
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 named patient,' until 1 13.6 commercial reimbursement established Expect multiple commercial launches across Europe in 2025 10.0 8.5 International Markets 8 exclusive
distribution agreements signed for 50+ countries 5.2 5.0 Revenue recognized from Er-Kim (Central & Eastern Europe), Neopharm (Israel), and Canada 1.5 - ~700 patients on YORVIPATH treatment in
Europe Direct and Q1-24 Q2-24 Q3-24 Q4-24E International Markets at the end of 2024 YORVIPATH on track for additional commercial launches in Europe Direct and International Markets in 2025 1 Unaudited preliminary estimate. 15 For investor
communication only. Not for use in product promotion. Not for further distribution.
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
hypoparathyroidism. YORVIPATH's titration scheme was only evaluated in adults who first achieved an albumin-corrected serum calcium of at least 7.8 mg/dL using calcium and active vitamin D treatment. YORVIPATH [package insert]. Princeton, NJ:
Ascendis Pharma Endocrinology, Inc. August 2024. 16 For investor communication only. Not for use in product promotion. Not for further distribution.
Hypoparathyroidism: U.S. Patient Population Total U.S. Prevalence PTH