Slide 1: COACH Trial Interim Topline Week 26 Data
COACH Trial Interim Topline Week 26 Data June 9, 2025 Combination treatment with investigational TransCon CNP and TransCon hGH is investigational. 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 TransCon hGH’s ability to complement TransCon CNP’s holistic treatment of achondr
Slide 3: Executive Summary
Executive Summary • COACH is the first clinical trial to evaluate once-weekly TransCon CNP and once-weekly TransCon hGH combination treatment in children with achondroplasia (ACH) • Interim topline Week 26 data from the Phase 2 COACH Trial demonstrated: ◦ For TransCon CNP treatment-naïve cohort, mea
Slide 4: Expanding the Treatment Paradigm
Expanding the Treatment Paradigm • TransCon hGH expected to complement TransCon CNP’s holistic treatment of achondroplasia, combining distinct modes of action GH/CNP exert different effects on the Growth Plate1-7 TransCon CNP releases the brake Resting zone Direct effects of GH: Recruitment of stem
Slide 5: First Phase 2 Combination Trial of CNP + hGH in Children with Achondroplasia
First Phase 2 Combination Trial of CNP + hGH in Children with Achondroplasia Week 26 Week 52 Interim Analysis Primary Analysis N=12 TransCon CNP treatment-naïve cohort TransCon CNP-treated in ACcomplisH/AttaCH N=9 TransCon CNP-treated cohort Primary Efficacy Objective Primary Efficacy Endpoint • Eva
Slide 6: Demographics and Baseline Characteristics (1/2)
Demographics and Baseline Characteristics (1/2) TransCon CNP Treatment-Naïve TransCon CNP-Treated Full analysis set at COACH screening Cohort (N=12) Cohort (N=9) Age at screening, years, mean (min, max) 4.67 (1, 9) 7.89 (5, 10) Age group, n (%) < 5 years 6 (50.0) 0 5 to < 8 years 5 (41.7) 3 (33.3) ≥
Slide 7: Demographics and Baseline Characteristics (2/2)
Demographics and Baseline Characteristics (2/2) TransCon CNP Treatment-Naive TransCon CNP-Treated Cohort (N=12) Cohort (N=9) Full analysis set at COACH screening Age at screening, years, mean (min, max) 4.67 (1, 9) 7.89 (5, 10) Years of exposure to TransCon CNP 100 µg/kg/wk, Not Applicable 2.56 (2.3
Slide 8: Overview of Treatment-Emergent Adverse Events
Overview of Treatment-Emergent Adverse Events All Participants (N=21) n (%) Safety Analysis Set Treatment-Emergent Adverse Event 15 (71.4) Grade 1 (Mild) 12 (57.1) Grade 2 (Moderate) 7 (33.3) Grade 3 (Severe) 1 (4.8) Grade 4 & 5 (Life-threatening or Death) 0 Treatment Related Adverse Events 7 (33.3)
Slide 9: Safety and Tolerability
Safety and Tolerability • Combination treatment showed safety data consistent with those observed for TransCon hGH and TransCon CNP monotherapies, and was generally well tolerated, with generally mild TEAEs • Majority of TEAEs were mild (Grade 1) or moderate (Grade 2) and typical for children of the
Slide 10: Combination Treatment Growth at Week 26 TransCon CNP Treatment-Naïve Cohort (N=12)
Combination Treatment Growth at Week 26 TransCon CNP Treatment-Naïve Cohort (N=12) AGV (Mean) ACH Height Z-Score (Mean) 10 1.0 0.99 9.14 0.9 9 0.8 8 0.7 +0.531 7 +4.231 P=0.0009 0.6 AGV 6 P=0.0063 ACH Height 0.5 0.46 (cm/year) Z-Score 5 4.92 0.4 0.3 4 0.2 3 TransCon CNP + TransCon hGH 0.1 Baseline T
Slide 11: Combination Treatment Growth at Week 26 TransCon CNP-Treated Cohort (N=9)
Combination Treatment Growth at Week 26 TransCon CNP-Treated Cohort (N=9) AGV (Mean) ACH Height Z-Score (Mean) 9 8.25 1.8 1.72 8 1.6 +0.441 1.4 7 1.28 P=0.0002 +3.101 1.2 6 P=0.0006 AGV ACH Height 1.0 (cm/year) 5.14 5 Z-Score 0.8 0.6 4 0.4 3 TransCon CNP + TransCon hGH Baseline TransCon 0.2 CNP-Trea
Slide 12: Combination Treatment Mean AGV at Week 26
Combination Treatment Mean AGV at Week 26 20 9.14 cm/year TransCon CNP Treatment-Naïve 15 8.25 cm/year TransCon CNP-Treated 97th AGV 10 (cm/year) 50th Average Stature 3rd 5 95th 50th Achondroplasia 5th 0 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 4.92 cm/year Age 5.14 cm/year Baseline TransCon CNP Treatment
Slide 13: COACH Body Proportionality at Week 26
COACH Body Proportionality at Week 26 0.02 0.02 0.01 TransCon CNP Treatment-Naïve 0.01 TransCon CNP-Treated Cohort (N=12) Cohort (N=9) 0.00 0.00 -0.01 -0.01 -0.02 -0.02 Upper to Lower Body Segment Ratio -0.03 -0.03 Change from Baseline (Mean (±SE)) -0.04 -0.04 Improvement -0.05 -0.05 -0.06 -0.06 -0.
Slide 14: Combination Treatment Program Next Steps
Combination Treatment Program Next Steps • Week 26 data demonstrated the potential to boost growth ~3x above that observed with CNP monotherapies over the same period, supporting the scientific rationale for treating with TransCon CNP and TransCon hGH combined • Week 52 data expected in Q4 2025 • Pl
Slide 15: Thank you
Thank you ascendis LA pharma