Ascendis Pharma A/s · NASDAQ:ASND
Ascendis Pharma A/s — 41st Annual J.P. Morgan Healthcare Conference (January 2023)
44 slides from Ascendis Pharma A/s's conference presentation, Jan 9, 2023
Slide 1: Ascendis Pharma A/S 41st Annual J.P. Morgan Healthcare Conference
Exhibit 99.1 Ascendis Pharma A/S 41st Annual J.P. Morgan Healthcare Conference January 9, 2023 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 prospective product candidates; clinical trial results; the expected timing o
Slide 3: Our Values Drive Sustainable Value Creation
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 fo
Slide 4: Positioned to Reach Patients Across the Globe
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 • TransCo
Slide 5: Predictable Release of Unmodified Parent Drug from TransCon Prodrug
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 loc
Slide 6: Strategic Approach for Product Innovation
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 fu
Slide 7: Approved Products & Clinical-Stage Product Candidates
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
Slide 8: Vision 3x3: Building a Leading Global Biopharma Company
Vision 3x3: Building a Leading Global Biopharma Company Our goal is to achieve sustainable growth through multiple approaches Ø Obtain regulatory approval for three independent Endocrinology Rare Disease products ⎯ TransCon hGH for pediatric growth hormone deficiency ⎯ TransCon PTH for adult hypopar
Slide 9: TransCon hGH
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.
Slide 10: Growth Hormone Supports Overall Endocrine Health
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.
Slide 11: Commercially Launched in the U.S.
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 •
Slide 12: SKYTROFA Selected Highlights of U.S. Prescribing Information
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
Slide 13: 2022 U.S. SKYTROFA Revenue Ramp
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 grow
Slide 14: Adult GHD Trial Primary Objective - Body Composition
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 d
Slide 15: Building a Leading Global Growth Hormone Brand in Value
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
Slide 16: TransCon hGH: Summary & Next Steps
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 ge
Slide 17: TransCon PTH
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.
Slide 18: Hypoparathyroidism: One Disease, Multiple Consequences
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* • Papi
Slide 19: Diverse Etiologies of Disease Covered in Clinical Programs
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
Slide 20: Once-Daily TransCon PTH – Showed Consistent Impact
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 independ
Slide 21: PaTHway Japan Trial: Topline Data at Week 26
PaTHway Japan Trial: Topline Data at Week 26 PaTHway Japan Trial achieved primary objectives 1 • 92% of patients (12 of 13) met the primary composite endpoint • Results consistent across disease etiologies studied (post-surgical, genetic, or idiopathic) • TransCon PTH was generally well-tolerated, w
Slide 22: Serum Phosphate PaTHway Japan
Serum Phosphate PaTHway Japan Released unmodified PTH is filtered by kidney, decreasing phosphate reabsorption 6.0 5.5 5.0 4.5 Serum Phosphate Normal range: 3.4 to 4.5 mg/dL mg/dL 4.0 Mean (± SE) 3.5 3.0 2.5 2.0 0 2 4 6 8 10 12 14 16 18 20 22 24 26 28 Week PaTHway Japan data showed TransCon PTH norm
Slide 23: Diagnosed U.S. Chronic Hypopara Patient Population
Diagnosed U.S. Chronic Hypopara Patient Population Early Œ Adopters 1 PTH ~4,000-5,000 Previously treated with PTH therapy Treated Capturing the 2 ~65,000-85,000 Currently treated with conventional therapy Market On conventional therapy 2 Sustaining growth ~3,000/year Newly diagnosed annually Ž Ne
Slide 24: Latest Clinical Practice Guideline
Latest 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 25: TransCon PTH: Summary & Next Steps
TransCon PTH: Summary & Next Steps Potential new paradigm to treat the underlying cause of hypoparathyroidism 1 • Unmet medical need recognized by FDA Priority Review; PDUFA date April 30, 2023 • Enrollment now open for U.S. Expanded Access Program • First wave of dedicated TransCon PTH U.S. sales f
Slide 26: TransCon CNP
TransCon CNP Investigational once-weekly growth treatment for achondroplasia TransCon CNP is an investigational product candidate. For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 27: Achondroplasia: Primary Skeletal Manifestations and Key Complications
Achondroplasia: 1-10 Primary Skeletal Manifestations and Key Complications Manifestations Complications Interventions • Short stature • Foramen magnum • Cervicomedullary stenosis decompression • Short limbs & rhizomelia • Spine deformities • Grommets • Narrowing of foramen magnum • Spinal stenosis •
Slide 28: TransCon CNP: Phase 2 Trial Design
TransCon CNP: Phase 2 Trial Design Up to 60 children (ages 2–10 years) with achondroplasia (ACH) 12 subjects randomized in each dose cohort in a blinded manner Open Label TransCon CNP Extension vs. placebo Period to 6 µg/kg 20 µg/kg 50 µg/kg 100 µg/kg >100 µg/kg (3:1 randomization) evaluate long- te
Slide 29: TransCon CNP Demonstrated Superiority in AHV Compared to Placebo
TransCon CNP 100 µg/kg/week Demonstrated Superiority in AHV Compared to Placebo TransCon CNP Placebo Treatment Group p-value AHV* (cm/year), n (TransCon CNP Dose (TransCon CNP vs. LS Mean [95% CI] Levels or Placebo) Pooled Placebo) 4.09, n=10 6 µg/kg/week 0.6004 AHV* [3.34, 4.84] (cm/year) 4.52, n=1
Slide 30: TransCon CNP Demonstrated Superiority in Change in Height SDS
TransCon CNP 100 µg/kg/week Demonstrated Superiority in Change in ACH-Specific Height SDS Compared to Placebo Placebo TransCon CNP p-value Treatment Group Δ Height SDS*, n (TransCon CNP (TransCon CNP Dose LS Mean [95% CI] vs. Pooled Levels or Placebo) Placebo) Δ Height -0.04, n=10 SDS 6 µg/kg/week 0
Slide 31: Overview of TEAEs (Double-Blind Period)
Overview of TEAEs (Double-Blind Period) TransCon CNP TransCon CNP TransCon CNP TransCon CNP Total 20 µg/kg/week 50 µg/kg/week 100 µg/kg/week 6 µg/kg/week Placebo (n=10) (n=11) (n=10) (n=11) (n=15) Subjects with TEAEs* n (%) 9 (90.0) 11 (100.0) 10 (100.0) 10 (90.9) 14 (93.3) Grade 1 (mild) n (%) 9 (9
Slide 32: Treatment-Related Adverse Events (Double-Blind Period)
Treatment-Related Adverse Events (Double-Blind Period) TransCon CNP TransCon CNP TransCon CNP TransCon CNP Total 20 µg/kg/week 50 µg/kg/week 100 µg/kg/week 6 µg/kg/week Placebo (n=10) (n=11) (n=10) (n=11) (n=15) Subjects with at Least One 3 (30.0) 2 (18.2) 3 (30.0) 2 (18.2) 5 (33.3) Treatment-Relate
Slide 33: Safety Results Summary (Double-Blind Period)
Safety Results Summary (Double-Blind Period) • TransCon CNP was generally well tolerated, with no discontinuations – Frequency of TEAEs in each dose group was similar to placebo • No serious AEs (SAEs) related to treatment were reported – Two unrelated SAEs were reported (febrile convulsion and vira
Slide 34: TransCon CNP: Summary & Next Steps
TransCon CNP: Summary & Next Steps ACcomplisH Trial suggested best-in-class potential in children down to 2 years of age – Safety: TEAEs comparable to placebo; ACH related TEAEs numerically lower in TransCon CNP comparing to placebo prompting further investigation – Efficacy: AHV at Week 52 demonstr
Slide 35: Oncology
Oncology Potential to improve immunotherapy safety and efficacy TransCon TLR7/8 Agonist & TransCon IL-2β/γ are investigational products candidates. For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 36: Oncology: Evaluating Multiple Indication-Specific Cohorts
Oncology: Evaluating Multiple Indication-Specific Cohorts TransCon TLR7/8 Agonist • Enrollment underway in four indication-specific cohorts (transcendIT-101) TransCon IL-2 β/γ • Enrollment continues in the dose escalation portion (IL-βelieγe) Upcoming 2023 milestones: • Topline results from monother
Slide 37: Ophthalmology
Ophthalmology Primed for disruption with TransCon Technology TransCon RBZ is an investigational product candidate. For investor communication only. Not for use in product promotion. Not for further distribution.
Slide 38: Ophthalmology - Primed for Disruption
Ophthalmology - Primed for Disruption Ophthalmology intravitreal treatments (IVT) represent an established, well-understood, and high-value therapeutic area, characterized by high unmet medical need • Leverage TransCon hydrogel technology to create highly differentiated products: – Continuous local
Slide 39: TransCon RBZ (Ranibizumab)
TransCon RBZ (Ranibizumab) Anti-VEGF market is established, well-understood, and a high-value therapeutic area • Vision loss caused by abnormal blood vessel growth and/or fluid build-up in the back of eye – Leading cause of blindness in industrialized nations • Poor patient outcomes correlated to an
Slide 40: TransCon RBZ Exposure and Tolerability in Non-Human Primates
TransCon RBZ Exposure and Tolerability in Non-Human Primates Ranibizumab in aqueous humor (µg/ml) 10.00 • Continuous local release of ranibizumab with half-life of ~100 days µg/ml 1.00 • TransCon RBZ single 2mg Target* intravitreal administration Ranibizumab showed higher than target concentrations
Slide 41: Ophthalmology: Summary & Next Steps
Ophthalmology: Summary & Next Steps TransCon Hydrogel Platform supports continuous local drug release over at least 6 months supporting twice yearly administration • TransCon RBZ (ranibizumab) is in preclinical development for wet AMD (age-related macular degeneration) and other VEGF-related disorde
Slide 42: Sustainable, Profitable Global Biopharma Company
Sustainable, Profitable Global Biopharma Company Ø Promising product candidates in multiple high-value areas • Endocrinology rare disease: Estimated $10B+ opportunity • Oncology & Ophthalmology: Estimated $25B+ addressable market Ø Growing pipeline each with best-in-class potential • Highly differen
Slide 43: The Year Ahead: Selected Milestones Expected in 2023
The Year Ahead Selected Milestones Expected in 2023 Q1 2023 Q2 2023 Q3 2023 Q4 2023 TransCon hGH TransCon PTH TransCon hGH TransCon hGH Pediatric GHD Adult Hypopara Pediatric GHD Adult GHD Enroll first patient in U.S. Registry FDA decision SKYTROFA launch in Germany Phase 3 topline results foresiGHt
Slide 44: Thank you!
ascendis 7 pharma » Thank you!
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