Full Press Release Details
Conference January 9, 2023 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 prospective product candidates; clinical trial results; the expected timing of future clinical
trial results; the scope, progress, results and costs of developing our product candidates or any other future product candidates; timing and likelihood of success; plans and objectives of management for future operations; and future results of
current and 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, including, without limitation, our most recent Annual Report on Form 20-F filed with the SEC on March 2, 2022 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 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 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, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, and SKYTROFA are trademarks owned by the Ascendis Pharma group. January 2023 Ascendis Pharma A/S. 2 For investor
communication only. Not for use in product promotion. Not for further distribution.
Our Values Drive Sustainable Value Creation Making a meaningful
difference in patients' lives: Addressing real unmet medical needs Creating new products with best-in-class potential based on strong scientific rationale Uncompromising focus on execution 3 For investor communication
only. Not for use in product promotion. Not for further distribution.
Positioned to Reach Patients Across the Globe A fully integrated
biopharma company with an expanding global presence Founded in 2007 in Copenhagen, Denmark Global HQ Ascendis Pharma A/S Publicly listed on NASDAQ Hellerup, Denmark since 2015 (ASND) Clinical Site Research Site Commercial
TransCon hGH approved Berlin, Germany Redwood City, CA Princeton, NJ in the US and Europe Research Site US Office Heidelberg, Germany Palo Alto, CA Commercial Affiliate Financials: ~ 935 million VISEN Munich, Germany cash, cash
equivalents Pharmaceuticals Shanghai, China and marketable securities as of September 30, 2022 4 For investor communication only. Not for use in product promotion. Not for further distribution.
Predictable Release of Unmodified Parent Drug from TransCon Prodrug
Sustained Systemic Release Sustained Localized Release Parent drug is Following injection, Designed to distribute Parent drug is Following injection, Designed to provide transiently bound to the linker is designed sustained high local transiently
bound to a the linker is designed released molecule like TransCon linker- to autocleave at a the parent drug; TransCon linker- to autocleave at a drug levels with low hydrogel carrier, specific systemic exposure; soluble carrier moiety, specific
linker-carrier is which inactivates and rate to predictably cleared renally which inactivates, rate to predictably hydrogel degrades shields parent drug release unmodified shields parent drug release unmodified into small polymers and prevents
parent drug that are renally from clearance parent drug clearance cleared Potential to improve drug safety, efficacy, tolerability, and convenience 5 For investor communication only. Not for use in product promotion. Not for further
Strategic Approach for Product Innovation A unique algorithm for
designing clearly differentiated product candidates Higher Value, Lower Risk Pipeline Expected higher success rate compared to traditional drug development 6 For investor communication only. Not for use in product promotion. Not for further
Approved Products & Clinical-Stage Product Candidates APPROVED
PRODUCTS Pediatric Growth Hormone Deficiency Endocrinology rare diseases 1 U.S.: SKYTROFA (lonapegsomatropin-tcgd) EUROPE: SKYTROFA (lonapegsomatropin) PRODUCT CANDIDATES PHASE 1 PHASE 2 PHASE 3 REGULATORY Endocrinology rare diseases 2
Pediatric Growth Hormone Deficiency (Japan) 3 Adult Growth Hormone Deficiency (Global) TransCon hGH 4 Turner Syndrome (U.S.) 5 Adult Hypoparathyroidism (U.S. and Europe) TransCon PTH 6 Adult Hypoparathyroidism (Japan) 7 Achondroplasia (Global)
TransCon CNP Oncology 8 Solid Tumors (including indication specific cohorts) TransCon TLR7/8 Agonist 9 TransCon IL-2 / Solid Tumors 1. Not yet marketed in the EU. 2. Japanese riGHt Trial. 3. Global foresiGHt Trial. 4. New InsiGHTS Trial.
5. NDA submitted to the FDA; PDUFA action date April 30, 2023, European MAA submitted November 2022, decision anticipated Q4 2023. 6. Japanese PaTHway Japan Trial. 7. Global ApproaCH Trial. 8. transcendIT-101 Trial includes four indication-specific
cohorts currently enrolling patients. 9. IL- elie e Trial. For investor communication only. Not for use in product promotion. Not for further distribution. 7
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
hypoparathyroidism TransCon CNP for achondroplasia Grow Endocrinology Rare Disease pipeline through - Global clinical reach - Pursuing 9 total indications, label optimization, and life cycle management - New
endocrinology products Establish global commercial presence for our Endocrinology Rare Disease area Build integrated commercial organization in North America and select European countries Establish global commercial presence
through partners with local expertise and infrastructure Advance a high-value oncology pipeline with one IND or similar filing each year Create a third independent therapeutic area with a diversified pipeline Ascendis Pharma's
2020 - 2025 strategic roadmap 8 For investor communication only. Not for use in product promotion. Not for further distribution.
TransCon hGH Once-weekly growth hormone replacement therapy
Approved as SKYTROFA in the U.S. and Europe For investor communication only. Not for use in product promotion. Not for further distribution.
Growth Hormone Supports Overall Endocrine Health 2-4 6,7 BODY
COMPOSITION CARDIOVASCULAR DISEASE 5 8 MENTAL HEALTH FRACTURES ULTIMATE HEIGHT 1 ACHIEVEMENT Growth hormone deficiency may lead to multiple health complications 1. de Boer, H. et al. J. Clin. Endocrinol. Metab. 1997; 82(7): 2032-2036. 2. Rutherford,
O. M. et al. Clin. Endocrinol 1991; 34(6): 469-475. 3. Colle, M., J. Auzerie. Horm. Res. 1993; 39(5-6): 192-196. 4. Johannsson, G., et al. J. Clin. Endocrinol. Metab. 1999; 84(12): 4516-4524. 5. Stabler, B. et al. Horm. Res. 1996; 45(1-2) 30-33. 6.
Leonga, G., Johannsson, G. Horm. Res. 2003; 60(suppl1): 78-85. 7. Colao, A. et al. J. Clin. Endocrinol. Metab. 2002; 87(8): 3650-3655. 8. Bex, M., Bouillon, R. Horm. Res. 2003; 60(suppl3): 80-86. For investor communication only. Not for use in
product promotion. Not for further distribution. 10
Commercially Launched in the U.S. First FDA-approved
once-weekly treatment for pediatric growth hormone deficiency (GHD) - Room temperature storage - Small 31G needle - Single low-volume (<0.60mL) injection for patients 60kg - No waste due to empty-all design -
Device lifespan at least 4 years First FDA-approved product utilizing TransCon technology For investor communication only. Not for use in product promotion. Not for further distribution. 11
SKYTROFA (lonapegsomatropin-tcgd) Selected Highlights of U.S.
Prescribing Information INDICATION SKYTROFA is a human growth hormone indicated for the treatment of pediatric patients 1 year and older who weigh at least & USAGE 11.5 kg and have growth failure due to inadequate secretion of endogenous growth
hormone (GH). DOSAGE & SKYTROFA should be administered subcutaneously into the abdomen, buttock, or thigh with regular rotation ADMINISTRATION of the injection sites. The recommended dose is 0.24 mg/kg body weight once-weekly. See full
prescribing information for instructions on preparation and administration of drug. CONTRAINDICATIONS Acute critical illness Hypersensitivity to somatropin or any of the excipients in SKYTROFA Children with closed epiphyses
Active malignancy Active proliferative or severe non-proliferative diabetic retinopathy Children with Prader-Willi syndrome who are severely obese, have a history of upper airway obstruction or sleep apnea or have severe
respiratory impairment due to the risk of sudden death ADVERSE REACTIONS Most common adverse reactions ( 5%) in pediatric patients include: viral infection, pyrexia, cough, nausea and vomiting, hemorrhage, diarrhea, abdominal pain, and
arthralgia and arthritis. Reference: SKYTROFA (lonapegsomatropin-tcgd) prescribing information, Ascendis Pharma / skytrofa_pi.pdf (ascendispharma.us) To report SUSPECTED ADVERSE REACTIONS, contact Ascendis Pharma, Inc. at
1-844-442-7236 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 12 For investor communication only. Not for use in product promotion. Not for further distribution.
2022 U.S. SKYTROFA Revenue ( ) Ramp Preliminary Q4 2022 U.S.
SKYTROFA estimated revenue of 20.0 17.1 million* 18.0 17.1 16.0 14.0 U.S. 12.0 SKYTROFA 12.3 Revenue 10.0 ( MM) 8.0 6.0 4.0 4.4 2.0
1.9 - Q1:22 Q2:22 Q3:22* Q4:22E* Q4 revenue provides the foundation for growth in 2023 and beyond *Preliminary Q4 revenue based on internal estimates of unaudited preliminary financial results. This includes an estimated negative
foreign currency translation impact of 0.4 million, compared to a benefit of 0.5 million in the third quarter of 2022. 13 For investor communication only. Not for use in product promotion. Not for further distribution.
Adult GHD Trial Primary Objective - Body Composition Phase 3,
double-blind, placebo-controlled main period with open-label daily somatropin arm, followed by open-label extension period Main period (38 weeks) Open-Label Extension period (52 weeks) 12 weeks dose titration 26 weeks target maintenance dose Primary
~ 80 Once-weekly Lonapegsomatropin Once-weekly Lonapegsomatropin Once-weekly Lonapegsomatropin comparison for All regions main period Once-weekly Placebo (double-blind for ~ 80 Once-weekly Placebo Once-weekly Lonapegsomatropin main period)
Open-label ~ 80 Daily Somatropin (Norditropin ) Daily Somatropin (Norditropin ) Once-weekly Lonapegsomatropin Japan only: switch patients previously treated Once-weekly Lonapegsomatropin with commercially available daily somatropin Key
Eligibility Criteria Primary Objective Demonstrate efficacy compared to placebo Adults with GHD Ages 23-80 years Primary Efficacy Endpoint GH treatment-na ve or no GH therapy in past 12 months Change from baseline in
trunk % fat at Week 38 IGF-1 SDS -1.0 at screening Norditropin is a trademark of Novo Nordisk Healthcare. 14 For investor communication only. Not for use in product promotion. Not for further distribution.
Building a Leading Global Growth Hormone Brand in Value Pricing
discipline built around product strengths has allowed us to expand overall market value EUROPE UNITED STATES First European launch planned for SKYTROFA positioned to be the leading Germany in Q3 2023 growth hormone brand in
value ASIA LABEL EXPANSION Phase 3 pediatric GHD data in Global adult GHD Phase 3 China, consistent w/ heiGHt Trial enrollment completed Phase 3 pediatric GHD continues Turner Syndrome trial underway in Japan
Combination with TransCon CNP Ascendis aspires to become the global leader in growth hormone and growth disorders 15 For investor communication only. Not for use in product promotion. Not for further distribution.
TransCon hGH: Summary & Next Steps Building a leading global growth
hormone brand in value U.S. Q4 revenue provides foundation for growth in 2023 and beyond Building integrated commercial organization in select European countries - First European launch in Germany expected Q3 2023 Label
and geographic expansion underway: - Adult GHD - Topline data from the global Phase 3 foresiGHt Trial expected in Q4 2023 - Turner Syndrome - Complete enrollment of Phase 2 New InsiGHTS Trial planned for Q3 2023 -
Pediatric GHD - Enrollment continues in the Phase 3 riGHt Trial in Japan Evaluating TransCon hGH in combination with TransCon CNP TransCon hGH has potential to grow size of the estimated $4B* global growth hormone market *Ascendis
Pharma estimate of the 2021 hGH market. 16 For investor communication only. Not for use in product promotion. Not for further distribution.
TransCon PTH Investigational PTH replacement therapy for adult
hypoparathyroidism TransCon PTH is an investigational product candidate. For investor communication only. Not for use in product promotion. Not for further distribution.
1-4 Hypoparathyroidism: One Disease, Multiple Consequences 5,6 Absence
or deficiency of parathyroid hormone (PTH) is linked to multi-organ manifestations. CENTRAL NERVOUS SYSTEM RENAL OPHTHALMALOGICAL Nephrocalcinosis* Seizures Cataracts Basal ganglia calcifications
Nephrolithiasis* Papilledema Chronic kidney disease* Parkinsonism or dystonia DENTAL PERIPHERAL NERVOUS SYSTEM Altered tooth morphology RESPIRATORY Paresthesia Muscle cramps Laryngospasm
DERMATOLOGICAL Pain Tetany Dry skin Thinning hair CARDIOVASCULAR Brittle nails NEUROPSYCHIATRIC Vascular calcifications Cardiac arrhythmias Anxiety & depression MUSCULOSKELETAL
Parathyroid Cognitive impairment Hypocalcemia-associated Thyroid gland Myopathy ("brain fog") dilated cardiomyopathy gland Spondylorthropathy 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. 18 TransCon PTH is an investigational product candidate. For investor communication only. Not for use in product
promotion. Not for further distribution.
Diverse Etiologies of Disease Covered in Clinical Programs Phase 2
Phase 3 Phase 3 PaTH Forward PaTHway (Global) PaTHway Japan Total (Global, OLE) (6 months)* (6 months) at 6-months* All TransCon PTH TransCon PTH Arm TransCon PTH TransCon PTH (n=59) (n=61) (n=13) (n=133) Cause of Hypoparathyroidism (HP) Acquired
from neck surgery 47 52 5 104 Autoimmune disease 1 1 0 2 ADH1 0 1 1 2 Other genetic 0 3 1 4 Idiopathic disease 11 4 6 21 * The safety and efficacy of TransCon PTH have not been established and TransCon PTH is not currently approved by the FDA. 19
TransCon PTH is an investigational product candidate. For investor communication only. Not for use in product promotion. Not for further distribution.
Once-Daily TransCon PTH - Showed Consistent Impact Phase 2 Phase
3 Phase 3 3 PaTH Forward PaTHway (Global) PaTHway Japan (Global, OLE) (6-months)* (6-months) at 6-months* Number of patients who reached 6-month visit, n 58 60 13 Number of patients eliminating conventional therapy (Defined as independence from
active vitamin D and independence from 54 (93%) 57 (95%) 13 (100%) 5 therapeutic levels of calcium supplements ) Number of Patients Meeting Individual Components, n (%): 4 Independence from active vitamin D 56 (100%) 60 (100%) 13 (100%) 4
Independence from therapeutic doses of calcium supplements 57 (95%) 52 (93%) 13 (100%) 1 4 Albumin-adjusted serum calcium within the normal range 51 (91%) 49 (82%) 12 (92%) Number of patients meeting all three components, n (%) 4 1 (Defined as serum
calcium in the normal range , independence from active vitamin D, 48 (86%) 48 (80%) 12 (92%) 2 independence from therapeutic levels of calcium supplements ) * The safety and efficacy of TransCon PTH have not been established and TransCon PTH is not
currently approved by the FDA. 1. The normal range for albumin-adjusted sCa is 8.3-10.6 mg/dL (2.07-2.64 mmol/L). 2. If needed to meet recommended dietary intake of calcium, it was permitted to take calcium supplements 600 mg/day as a