Ascendis Pharma A/s · NASDAQ:ASND
Ascendis Pharma A/s — 40th Annual J.P. Morgan Healthcare Conference (January 2022)
32 slides from Ascendis Pharma A/s's conference presentation, Jan 10, 2022
Slide 1: Ascendis Pharma A/S 40th Annual J.P. Morgan Healthcare Conference
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
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 future results of operations and financial position, including our business s
Slide 3: Ascendis Pharma, a Global Company
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, Scien
Slide 4: Vision 3x3: Building a Leading Global BioPharma Company
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
Slide 5: Five Independent Product Candidates in Clinical Development
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 Nove
Slide 6: Building a Viable, Sustainable, Long-Term Business Model
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 i
Slide 7: Diverse Pipeline of Independent Product Candidates
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 foresi
Slide 8: Endocrinology Rare Disease
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.
Slide 9: TransCon hGH Once-Weekly Replacement Therapy
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.
Slide 10: SKYTROFA (lonapegsomatropin-tcgd) FDA-Approved in the U.S.
SKYTROFA® (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
Slide 11: SKYTROFA: Selected Highlights of U.S. Prescribing Information
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 endogen
Slide 12: U.S. Commercial Focus: Building the Leading Brand
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
Slide 13: Positioned to Build Market Leadership
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 Cumulat
Slide 14: Snapshot of First Two Months of Launch
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 a
Slide 15: Planned Expansion of Global Reach and Indications for TransCon hGH
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 VIS
Slide 16: TransCon hGH: Potential to Transform the Global hGH Market
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 g
Slide 17: TransCon PTH: PTH Replacement Therapy for Hypoparathyroidism
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.
Slide 18: Chronic Hypoparathyroidism: Significant Patient Population
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 N
Slide 19: TransCon PTH PaTH Forward (Phase 2) Trial Design
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 week
Slide 20: PaTH Forward Trial Data
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
Slide 21: PaTH Forward Safety Summary
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
Slide 22: TransCon PTH PaTHway (Phase 3) Trial Design
TransCon PTH PaTHway (Phase 3) Trial Design 1 Sample size selected to ensure evaluable data for 68. 2 If needed to meet recommended dietary intake of calcium, it is permitted to take calcium supplements ≤600 mg/day as a nutritional supplement. Double-blind, placebo-controlled trial with an open-labe
Slide 23: PaTHway Phase 3 Trial: Baseline Characteristics
PaTHway Phase 3 Trial: Baseline Characteristics Characteristics Total Randomized (N = 82) Age, mean years 49 years Female sex, % 78% Geographic region, % North America 62% Europe 38% Postmenopausal, % 28% Duration of hypoparathyroidism, mean years 12 years Post-surgical etiology of hypoparathyroidis
Slide 24: Potential New Treatment Paradigm for Hypoparathyroidism
Potential New Treatment Paradigm for Hypoparathyroidism TransCon PTH has the potential to be the first hormone replacement therapy for hypoparathyroidism addressing major unmet medical need for a large rare disease patient population Upcoming milestones: Phase 3 PaTHway Trial (North America + EU) to
Slide 25: TransCon CNP: Potential New Once-Weekly Growth Treatment Option
TransCon CNP Potential New Once-Weekly Growth Treatment Option All product candidates other than SKYTROFA® are investigational. For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 26: TransCon CNP: Clinical Development Program
TransCon CNP: Clinical Development Program Natural history study Age 0–8 years N = 234 14 countries Global, multiple dose study Age 2–10 years N = 57 China, multiple dose study Age 2–10 years N ~60 Global, multiple dose, early intervention study Age 0–2 years N ~30 ONGOING ONGOING IND or equivalent
Slide 27: TransCon CNP Clinical Program Is Progressing
TransCon CNP Clinical Program Is Progressing ACcomplisH enrollment completed (N = 57) Interim blinded data informed dose selection of 50 and 100 mg/kg/week for ACcomplisH China Preliminary PK and safety data on TransCon CNP suggest continuous CNP is well-tolerated across all doses administered ACcom
Slide 28: Oncology
Oncology All product candidates other than SKYTROFA® are investigational. For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 29: Initial Interim Data Indicate TransCon TLR7/8 Agonist Was Well-Tolerated
Initial Interim Data Indicate TransCon TLR7/8 Agonist Was Well-Tolerated as Monotherapy or in Combination with Pembrolizumab* Initial data* from ongoing TransCon TLR7/8 Agonist first-in-human trial (transcendIT-101) indicated: Observed low systemic exposure and mean plasma half-life of ~7 days based
Slide 30: Designing a Portfolio to Transform Cancer Immunotherapy Treatment
Designing a Portfolio to Transform Cancer Immunotherapy Treatment TransCon TLR7/8 Agonist has the potential for: Sustained immune activation Systemic anti-tumor response with dosing interval of at least three weeks Create a pipeline using TransCon technologies that may enable a new treatment paradig
Slide 31: Selected Milestones Expected in 2022
TransCon hGH Turner Syndrome Submit protocol to FDA Selected Milestones Expected in 2022 Q1 2022 Q2 2022 Q3 2022 Q4 2022 TransCon CNP Pediatric Achondroplasia Phase 2 top-line results ACcomplisH Trial TransCon hGH Pediatric GHD EC MAA Approval TransCon PTH Adult PTH Phase 3 top-line results PaTHway
Slide 32: Thank you
Thank you Company contact: Tim Lee Senior Director, Investor Relations tle@ascendispharma.com (650) 374-6343
Ascendis Pharma — 40th Annual J.P. Morgan Healthcare Conference (January 2022) on 6ix