Full Press Release Details
Ascendis Pharma A/S 40th Annual J.P.
Morgan Healthcare Conference January 10, 2022 All product candidates other than SKYTROFA are investigational. For investor communication only. Not for use in product promotion. Not for further distribution. Exhibit 99.1
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,
including our business strategy, expectations regarding the potential benefits of SKYTROFA, prospective products, availability of funding, clinical trial results, product approvals and regulatory pathways, collaborations, licensing or other
arrangements, the scope, support progress, results and costs of developing our product candidates or any other future product candidates, the potential market size and size of the potential patient populations for SKYTROFA and our product
candidates, timing and likelihood of success, plans and objectives of management for future operations, the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates, future results of
current and anticipated products, and the future operations of VISEN Pharmaceuticals, 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 preliminary prospectus supplement related to the proposed public offering and our most recent
Annual Report on Form 20-F filed with the SEC on March 10, 2021 particularly in the sections titled "Risk Factors" and "Management's Discussion and Analysis of Financial Condition and Results of Operations". 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. SKYTROFA (lonapegsomatropin-tcgd) has
been approved by the U.S. Food and Drug Administration for the treatment of pediatric growth hormone deficiency. SKYTROFA is and has been under clinical investigation and has not yet been approved for marketing by the European Medicines Agency or
other foreign regulatory authorities. In addition, this presentation concerns other product candidates that are 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 2022 Ascendis Pharma A/S.
Ascendis Pharma, a Global Company
Founded in 2007 in Copenhagen, Denmark Publicly listed on NASDAQ since 2015 (ASND) TransCon : Innovative technology platform utilized in all product candidates Our mission: Develop best-in-class therapeutics addressing unmet medical needs Our
values: Patients, Science and Passion Financials: As of September 30, 2021, cash, cash equivalents and marketable securities of 930 million Committed to making a meaningful difference in patients' lives An expanding global footprint
VISEN Pharmaceuticals was established in 2018 to develop and commercialize endocrinology rare disease therapies in Greater China. Research Site Affiliate VISEN Pharmaceuticals Shanghai, China US Office Palo Alto, CA Redwood City, CA Clinical Site
Berlin, Germany Global HQ Ascendis Pharma A/S Hellerup, Denmark Research Site Heidelberg, Germany US Commercial Princeton, NJ
Vision 3x3: Building a Leading Global
BioPharma Company 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 Our Goal Is to Achieve Sustainable Growth through Multiple Approaches
Five Independent Product Candidates in
Clinical Development Endocrinology Rare Disease TransCon hGH: SKYTROFA (lonapegsomatropin-tcgd) approved in the US by FDA in August 2021 for pediatric growth hormone deficiency (GHD) TransCon hGH in Europe: positive CHMP opinion for pediatric
GHD received November 12, 2021 Pediatric GHD: Phase 3 trials in China1 and Japan ongoing Adult GHD: Global Phase 3 foresiGHt Trial ongoing TransCon PTH: Adult hypoparathyroidism (HP) Phase 3 PaTHway Trial in North America and Europe ongoing TransCon
CNP: Achondroplasia Phase 2 trials: ACcomplisH Trial and ACcomplisH China Trial1 ongoing Oncology TransCon TLR7/8 Agonist: Phase 1/2 transcendIT-101 Trial TransCon IL-2 b/g: Phase 1/2 IL-beliege Trial 1 Trials in China being conducted by VISEN
Pharmaceuticals. Potential to create best-in-class products addressing unmet medical needs by applying TransCon technologies to parent drugs with clinical proof-of-concept or clinically validated pathways
Building a Viable, Sustainable,
Long-Term Business Model * Ascendis Pharma internal estimates. Estimates are subject to significant business, economic, regulatory and competitive uncertainties and contingencies, including the outcome of clinical trials and regulatory review. $10B*
Market Potential in First Therapeutic Area Endocrinology Rare Disease Unmet medical needs; focused on large rare disease - potential multi billion $ product opportunities Aim to achieve global market leadership with highly differentiated
products developed using validated, proprietary technology platform Diversified multiple product candidates in each therapeutic area to leverage synergies Sustainability and long-term growth from additional therapeutic areas The right capabilities
to execute Potential Market Opportunity ($MM)* U.S. Pediatric GHD U.S. Pediatric GHD U.S. Other GH Indications ROW All GH Indications Hypoparathyroidism (global) Achondroplasia (global) Strong anticipated profitability
Diverse Pipeline of Independent Product
Candidates 1Received positive CHMP opinion on November 12, 2021. Final EC decision expected within 67 days, or by end of January 2022. 2In development in Greater China through strategic investment in VISEN Pharmaceuticals. 3Japanese riGHt Trial.
4Global foresiGHt Trial. 5North American and European PaTHway Trial, Japanese PaTHway Japan Trial. 6North America, Europe, and Oceania ACcomplisH Trial. 7transcendIT-101 Trial. 8IL-beliege Trial. 7 7 8
Endocrinology Rare Disease All product
candidates other than SKYTROFA are investigational. For investor communication only. Not for use in product promotion. Not for further distribution.
TransCon hGH Once-Weekly Replacement
Therapy All product candidates other than SKYTROFA are investigational. For investor communication only. Not for use in product promotion. Not for further distribution.
(lonapegsomatropin-tcgd) FDA-Approved in the U.S. First FDA-approved once-weekly product for pediatric growth hormone deficiency (GHD) First FDA-approved product utilizing TransCon technology In-use room temperature storage for 6 months
SKYTROFA (lonapegsomatropin-tcgd):
Selected Highlights of U.S. Prescribing Information INDICATIONS AND USAGE SKYTROFA is a human growth hormone indicated for the treatment of pediatric patients 1 year and older who weigh at least 11.5 kg and have growth failure due to inadequate
secretion of endogenous growth hormone (GH) DOSAGE AND ADMINISTRATION SKYTROFA should be administered subcutaneously into the abdomen, buttock, or thigh with regular rotation 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 after open heart surgery, abdominal surgery or multiple accidental trauma, or those with acute
respiratory failure due to the risk of increased mortality with use of pharmacologic doses of somatropin Hypersensitivity to somatropin or any of the excipients in SKYTROFA Children with closed epiphyses Active malignancy
Active proliferative or severe non-proliferative diabetic retinopathy 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 a clinical trial were viral infection (15%), pyrexia (15%), cough (11%), nausea and vomiting (11%), hemorrhage (7%), diarrhea (6%), abdominal pain (6%), and arthralgia and
arthritis (6%). Reference: SKYTROFA (lonapegsomatropin-tcgd) prescribing information, Ascendis Pharma To report SUSPECTED ADVERSE REACTIONS, contact Ascendis Pharma, Inc., at 1-844-442-7236 or FDA at 1-800-FDA-1088 or
In-house teams with deep endocrine
experience maximizing SKYTROFA awareness, patient treatment, and payer coverage U.S. Commercial Focus: Building the Leading Brand Focus on high volume prescribers Broaden SKYTROFA awareness Patient enrollment, activation, access, training, and
overall support Fast Start program Benefits verification including prior authorizations, coverage support, and ongoing reimbursement Out-of-pocket assistance for qualified patients R x 0 PAYER ENGAGEMENT Plans & Sub-Plans Commercial Employer
Gov't PBMs 0 Medical Affairs Market Access Sales AWARENESS & ENGAGEMENT PATIENT TREATMENT
Positioned to Build Market
Leadership Size of established U.S. commercial organization on par with current market leader U.S. sales team covers ~1,400 out of 7,000 prescribers who represent ~80% of the prescriptions Strong foundation established to support current and future
product launches Cumulative prescription volume Top 10 states ~60% Next 19 states ~90% All 50 states 100% U.S. GH Prescriber Landscape* * 2019 IQVIA Xponent data
Snapshot of First Two Months of
Launch* Physician enthusiasm for SKYTROFA reflected in increase in prescriptions, submission of formulary exceptions, and repeat prescribers * As of 12/31/21. Data on file. These numbers have not been verified by any third party and represent the
Company's estimates as of the date indicated. The Company assumes no obligation to update them. **MMIT Data as of 01/07/22 42% Repeat prescribers 369 Prescriptions Initial Payer Coverage for pediatric GHD indication in the U.S. R x 139
Targeted prescribers Plan Formulary Exceptions available while formulary reviews take place 28%** (103MM lives)
Planned Expansion of Global Reach
and Indications for TransCon hGH 1SKYTROFA is a human growth hormone indicated for the treatment of pediatric patients 1 year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous growth hormone (GH)
2Conducted by VISEN Pharmaceuticals. FDA Approved1 for pediatric GHD in the U.S. under brand name SKYTROFA (lonapegsomatropin-tcgd) Planned geographic expansion for pediatric GHD: Europe Received positive CHMP opinion MAA approval expected by
end of January 2022 Japan Pediatric GHD Phase 3 riGHt Trial Enrollment ongoing Greater China2 Pediatric GHD Phase 3 trial in China Target recruitment reached in Q1 2021 Planned label expansion Adult GHD Global foresiGHt Phase 3 trial Enrollment
ongoing Turner Syndrome Protocol submission to FDA planned for Q2 2022
TransCon hGH: Potential to Transform
the Global hGH Market In a Phase 3 clinical trial, once-weekly TransCon hGH demonstrated higher annualized height velocity at 52 weeks with comparable safety and immunogenicity to daily somatropin Additional studies planned and/or underway to
potentially expand geographic reach and label indication beyond pediatric GHD in the U.S. TransCon hGH has the potential to increase the size of the estimated $4B global hGH market through increased adherence, persistence, and penetration Upcoming
milestones: EC MAA approval for pediatric growth hormone deficiency expected by end of January 2022 Planned protocol submission to FDA for Turner Syndrome in Q2 2022 Complete enrollment in Phase 3 foresiGHt Trial in adult GHD expected in Q2 2022
Ascendis aspires to become the global leader in the hGH market
TransCon PTH PTH Replacement Therapy
for Hypoparathyroidism All product candidates other than SKYTROFA are investigational. For investor communication only. Not for use in product promotion. Not for further distribution.
Chronic Hypoparathyroidism:
Significant Patient Population Estimated Prevalence: ~400k in these 5 regions ~86k-223k 2013, Underbjerg et. al., Cardiovascular and Renal Complications to Postsurgical Hypoparathyroidism: A Danish Nationwide Controlled Historic Follow-up
Study 2015, The Epidemiology of Nonsurgical Hypoparathyroidism in Denmark: A Nationwide Case Finding Study 2016, Astor et. al., Epidemiology and Health-Related Quality of Life in Hypoparathyroidism in Norway ~70k-112k 2013, Powers et. al.,
Prevalence and Incidence of Hypoparathyroidism in the United States Using a Large Claims Database, JBMR 2011, Clarke et. al., Co-morbid Medical Conditions Associated with Prevalent Hypoparathyroidism: A Population-Based Study ~12k-13k S.
Korean ICD-10 codes Ascendis market research ~25k-32k 2017. Shishiba et. al., Prevalence of postsurgical hypoparathyroidism in Japan: Estimated from the data of multiple institutes 1999. Nakamura et. al., Prevalence of Idiopathic
Hypoparathyroidism and Pseudohypoparathyroidism in Japan Ascendis market research ~180k-220k 2020, Sui et. al, Time trend analysis of thyroid cancer surgery in China: single institutional database analysis of 15,000 patients 2019, Zhao et.
al., Features and trends of thyroid cancer in patients with thyroidectomies in Beijing, China between 1994 and 2015: a retrospective study World Bank, 2018 Ascendis market research China South Korea Japan Europe USA
TransCon PTH PaTH Forward (Phase 2)
Trial Design FECa, fractional excretion of calcium Khan AA, et al. J Clin Endocrinol Metab. 2021 Aug 4. Epub ahead of print Adults with hypoparathyroidism who required conventional therapy (active vitamin D + calcium) at baseline Primary Composite
Endpoint (4 weeks) Proportion of subjects with: Normal serum calcium; and Independence from active vitamin D; and Requiring 1,000 mg/day calcium supplements; and Normal FECa (or at least 50% decrease from baseline) Blinded Treatment (4
weeks) RANDOMIZATION Screening 4 weeks TransCon PTH 6-60 g/day Titration of TransCon PTH and conventional therapy with the goal to maintain normocalcemia TransCon PTH 15 g/day TransCon PTH 18 g/day TransCon PTH 21
g/day Placebo Open Label Extension Endpoints Intake of active vitamin D and calcium supplements Serum calcium and phosphate 24-hour urine calcium Adverse events Bone mineral density (Week 58 only) Patient-reported outcomes (Week 58 only) ALL
SUBJECTS Week 4 Week 58 Week 214 Week 84
Week 84 Phase 2 PaTH Forward Trial
Data *As of January 5th, 2022, 57 subjects continue in the open-label extension; One subject withdrew after week 97, not related to study drug. **Not taking active vitamin D and taking 600 mg/day of calcium supplements. Data on file, Ascendis
Pharma; PaTH Forward week 84 top-line data; Q4 2021. Week 84 data continue to support TransCon PTH as a potential hormone replacement therapy for adults with hypoparathyroidism 58 subjects completed 84 weeks of follow-up* Continued treatment
with TransCon PTH demonstrated that: Mean serum calcium remained stable and in the normal range 93% of subjects were free from active vitamin D and were taking 600 mg/day of calcium supplements TransCon PTH was well-tolerated at all doses
administered through week 84 in PaTH Forward No treatment-related serious or severe adverse events occurred, and no treatment-emergent adverse events (TEAEs) led to discontinuation of study drug
Week 84 PaTH Forward Safety Summary
TransCon PTH was well-tolerated at all doses administered No drug-related serious TEAEs were reported No TEAEs leading to discontinuation of study drug TEAEs with TransCon PTH reflect known PTH pharmacology Injections were well-tolerated using pen
injector planned for commercial presentation No subjects had TEAEs related to hyper- or hypocalcemia leading to ER/urgent care visit and/or hospitalization Data on file, Ascendis Pharma; PaTH Forward week 84 top-line data; Q4 2021.
TransCon PTH PaTHway (Phase 3) Trial