Slide 1: TransCon CNP ACcomplisH Trial Topline Results
TransCon™ CNP ACcomplisH Trial Topline Results November 14, 2022 TransCon CNP is an investigational product candidate. 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: Executive Summary
Executive Summary • In the Phase 2 ACcomplisH Trial in children with achondroplasia aged 2-10, once-weekly TransCon CNP demonstrated the potential to meet patient and caregiver needs for a safe, effective, tolerable, and convenient treatment • The primary endpoint, annualized height velocity (AHV) a
Slide 4: TransCon CNP: Phase 2 Trial Design
TransCon CNP: Phase 2 Trial Design Key Secondary/Additional Endpoints • Change in body proportionality • Patient reported outcome (PRO) measures • Exploratory biomarkers evaluated TransCon CNP is an investigational product candidate. For investor communication only. Not for use in product promotion.
Slide 5: Demographics and Baseline Characteristics
Demographics and Baseline Characteristics TransCon CNP TransCon CNP TransCon CNP TransCon CNP Total Total 6 µg/kg/week 20 µg/kg/week 50 µg/kg/week 100 µg/kg/week TransCon CNP Placebo n=10 n=11 n=10 n=11 n=42 n=15 Age (years) Mean (SD) 6.5 (2.6) 6.3 (2.9) 5.2 (3.0) 5.8 (2.6) 6.0 (2.7) 5.9 (3.1) Media
Slide 6: Demographics and Baseline Characteristics (continued)
Demographics and Baseline Characteristics (continued) TransCon CNP TransCon CNP TransCon CNP TransCon CNP Total Total 6 µg/kg/week 20 µg/kg/week 50 µg/kg/week 100 µg/kg/week TransCon CNP Placebo n=10 n=11 n=10 n=11 n=42 n=15 Height (cm) Mean (SD) 90.63 (8.97) 92.29 (12.10) 86.61 (12.97) 89.23 (12.82
Slide 7: TransCon CNP 100 µg/kg/week Demonstrated Superiority in AHV Compared to Placebo
TransCon CNP 100 µg/kg/week Demonstrated Superiority in AHV Compared to Placebo Treatment Group p-value TransCon CNP Placebo (TransCon CNP AHV (cm/year), n (TransCon CNP vs. Dose Levels LS Mean [95% CI] Pooled Placebo) or Placebo) 4.09, n=10 6 µg/kg/week 0.6004 AHV [3.34, 4.84] (cm/year) 4.52, n=11
Slide 8: Comparable AHV Across Age Groups
Comparable AHV Across Age Groups Age <5 years old Age ≥5 years old Treatment Group (TransCon CNP Dose Levels AHV (cm/year), n AHV (cm/year), n or Placebo) LS Mean, [95% CI] LS Mean, [95% CI] 4.31, n=3 3.79, n=7 6 µg/kg/week [2.52, 6.10] [2.87, 4.71] 4.72, n=5 4.29, n=6 20 µg/kg/week [3.30, 6.15] [3.
Slide 9: TransCon CNP 100 µg/kg/week Demonstrated Superiority in Change in ACH-Specific Height SDS
TransCon CNP 100 µg/kg/week Demonstrated Superiority in Change in ACH-Specific Height SDS Compared to Placebo p-value TransCon CNP Placebo Treatment Group Height SDS*, n (TransCon CNP (TransCon CNP Dose LS Mean [95% CI] vs. Pooled Levels or Placebo) Placebo) Height -0.04, n=10 6 µg/kg/week 0.8207 SD
Slide 10: 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 11: Overview of TEAEs (Double-Blind Period)
Overview of TEAEs (Double-Blind Period) TransCon CNP TransCon CNP TransCon CNP TransCon CNP Total 6 µg/kg/week 20 µg/kg/week 50 µg/kg/week 100 µg/kg/week Placebo (n=10) (n=11) (n=10) (n=11) (n=15) Subjects with TEAEs* 9 (90.0) 11 (100.0) 10 (100.0) 10 (90.9) 14 (93.3) Grade 1 (mild) 9 (90.0) 11 (100
Slide 12: Treatment-Related Adverse Events (Double-Blind Period)
Treatment-Related Adverse Events (Double-Blind Period) TransCon CNP TransCon CNP TransCon CNP TransCon CNP Total 6 µg/kg/week 20 µg/kg/week 50 µg/kg/week 100 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 13: Open Label Extension (OLE) Efficacy and Safety Results
Open Label Extension (OLE) Efficacy and Safety Results* • 57 of 57 patients completed the blinded period of ACcomplisH and continued in the OLE on 100 µg/kg/week with 100% retention • Patients treated ≥6 months at 100 µg/kg/week in the blinded or OLE period demonstrated a consistent and sustained re
Slide 14: Next Steps
Next Steps • IND submitted for ApproaCH, a global Phase 2b trial in 80 children with achondroplasia aged 2-11; enrollment targeted for completion in early 2023 – TransCon CNP 100 µg/kg/week vs. Placebo (2:1) • End of Phase 2 meetings with FDA and EMA planned • Plan to file IND or similar for TransCo
Slide 15: Thank you
Thank you Company contact: Tim Lee Senior Director, Investor Relations tle@ascendispharma.com (650) 374-6343 TransCon CNP is an investigational product candidate. For investor communication only. Not for use in product promotion. Not for further distribution.