Slide 1: TransCon CNP (navepegritide) ApproaCH Trial Topline Results
TransCon™ CNP (navepegritide) ApproaCH Trial Topline Results September 16, 2024 TransCon CNP is an investigational product candidate. 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 expected timing for submission of certain regulatory filings related to Trans
Slide 3: Executive Summary
Executive Summary The pivotal ApproaCH Trial in children aged 2-11 years achieved primary objective TransCon CNP demonstrated AGV superior to placebo with LS mean treatment difference of 1.49 cm/year at Week 52 (p<0.0001) For children aged 5-11 years TransCon CNP demonstrated a change from baseline
Slide 4: ApproaCH Pivotal Trial Design: TransCon CNP in Children with Achondroplasia Ages 2 to 11 years
ApproaCH Pivotal Trial Design: TransCon CNP in Children with Achondroplasia Ages 2 to 11 years Double-blind, placebo-controlled trial with an open-label extension period 84 children (ages 2 to 11 years) with achondroplasia randomized 2:1 (TransCon CNP:Placebo) 57 Participants TransCon CNP 100 µg/kg/
Slide 5: Demographics and Baseline Characteristics (1/2)
Demographics and Baseline Characteristics (1/2) Full Analysis Set TransCon CNP (n=57) Placebo (n=27) Overall (N=84) Age (years), mean (SD) 5.6 (2.6) 6.0 (2.7) 5.7 (2.6) Age (years) group, n (%) 2-4 5-7 ≥8 21 (36.8) 26 (45.6) 10 (17.5) 10 (37.0) 10 (37.0) 7 (25.9) 31 (36.9) 36 (42.9) 17 (20.2) Sex, n
Slide 6: Demographics and Baseline Characteristics (2/2)
Demographics and Baseline Characteristics (2/2) Full Analysis Set TransCon CNP (n=57) Placebo (n=27) Overall (N=84) Height (cm), mean (SD) 88.9 (12.9) 89.1 (11.5) 89.0 (12.4) ACH-specific height Z-score*, mean (SD) 0.18 (0.92) -0.11 (0.73) 0.09 (0.87) CDC (general population) height Z-score**, mean
Slide 7: Overview of Treatment Emergent Adverse Events (TEAEs)
Overview of Treatment Emergent Adverse Events (TEAEs) Safety analysis set TransCon CNP (n=57) n (%) Placebo (n=27) n (%) Any Adverse Event Grade 1 Grade 2 Grade 3 Grade 4 Grade 5 52 (91%) 52 (91%) 16 (28%) 4 (7%) 0 0 26 (96%) 25 (93%) 11 (41%) 1 (4%) 0 0 Any treatment-related adverse events 12 (21%)
Slide 8: TransCon CNP Safety and Tolerability Results
TransCon CNP Safety and Tolerability Results TransCon CNP showed safety results comparable to placebo and was generally well tolerated, with generally mild TEAEs Majority of AEs were mild (Grade 1) or moderate (Grade 2) and typical for children of these ages No AEs led to discontinuation of TransCon
Slide 9: Primary Endpoint: Annualized Growth Velocity at Week 52
Primary Endpoint: Annualized Growth Velocity at Week 52 TransCon CNP achieved the primary objective, demonstrating superiority over placebo ANCOVA model. TransCon CNP (n=57) Placebo (n=27) LS Mean AGV (cm/yr) at Week 52 5.89 4.41 LS Mean Difference [95% CI] (TransCon CNP vs. Placebo) 1.49 [1.05, 1.9
Slide 10: Observed Annualized Growth Velocity Over 52 Weeks
3.95 5.95 3.75 4.29 Observed Annualized Growth Velocity Over 52 Weeks Children treated with TransCon CNP increased their AGV 2.00 cm/year over 52 weeks (Observed values) Annualized Growth Velocity (cm/year) Mean (±SE) TransCon CNP is an investigational product candidate. For investor communication o
Slide 11: Sub-Group Analyses: Primary Endpoint by Age Groups
TransCon CNP demonstrated superior AGV compared to placebo in both age groups Sub-Group Analyses: Primary Endpoint by Age Groups ANCOVA model. Sub-group analyses by age 2 to <5 years 5-11 years TransCon CNP (n=21) Placebo (n=10) TransCon CNP (n=36) Placebo (n=17) LS Mean AGV (cm/yr) at Week 52 6.07
Slide 12: Sub-Group Analyses: Change from Baseline AGV by Age Group
TransCon CNP demonstrated significant change from baseline AGV compared to placebo by age group Sub-Group Analyses: Change from Baseline AGV by Age Group ANCOVA model. Sub-group analyses by age 2 to <5 years 5-11 years TransCon CNP (n=19) Placebo (n=10) TransCon CNP (n=35) Placebo (n=17) Change from
Slide 13: Secondary Endpoint: Change from Baseline in Height Z-score at Week 52
Secondary Endpoint: Change from Baseline in Height Z-score at Week 52 ACH Height Z-Score TransCon CNP (n=57) Placebo (n=27) LS Mean CFB at Week 52 0.30 0.01 LS Mean Difference [95% CI] (TransCon CNP vs. Placebo) 0.28 [0.18, 0.39] p-value (TransCon CNP vs. Placebo) <0.0001 CDC Height Z-score TransCon
Slide 14: Change from Baseline in Height Z-score Over Weeks
Change from Baseline in Height Z-score Over 52 Weeks ACH* Height Z-score CDC** Height Z-score Children with achondroplasia treated with TransCon CNP exceeded growth rate of general population, providing catch-up growth without accelerated bone age Change from Baseline Mean (±SE) Change from Baseline
Slide 15: Results Beyond Linear Growth
Results Beyond Linear Growth In the total trial population, treatment with TransCon CNP resulted in numerical improvements in health-related quality of life compared to placebo as observed in several ACEM domains At baseline, parents of children generally reported lower burden of health-related qual
Slide 16: Results in Patients with Reported Health-Related QoL Burden at Baseline
n Δ Least Squares Means TransCon CNP Placebo 28 13 -9.80 29 12 -7.49 45 24 -3.36 24 11 -9.96 Results in Patients with Reported Health-Related QoL Burden at Baseline* TransCon CNP Better Placebo Better ACEM Observable Signs Measure ACEM Physical Functioning ACEM Daily Living Functioning ACEM Emotiona
Slide 17: Results in ACEM-PF Sub-Group Supported by Muscle Functionality Endpoint
n Δ Least Squares Means TransCon CNP Placebo 20 10 -11.1 Results in ACEM-PF Predefined Sub-Group Are Supported by Exploratory Muscle Functionality Endpoint TransCon CNP Better Placebo Better ACEM Physical Functioning ˂5 years p=0.037 *Week 52 Body-weight normalized torque in Knee Extension test [N*m
Slide 18: ApproaCH Body Proportionality
ApproaCH Body Proportionality Patients dosed with TransCon CNP demonstrated statistical improvement in body proportionality compared to baseline (p=0.02) while placebo was unchanged from baseline (p=0.43) Upper to Lower Body Segment Ratio (Change from Baseline) Mean (±SE) TransCon CNP is an investig
Slide 19: Summary & Next Steps
Summary & Next Steps The pivotal ApproaCH Trial results support the desired target product profile of once-weekly TransCon CNP delivering continuous exposure of CNP Plan to submit New Drug Application to the U.S. FDA for TransCon CNP for treatment of children with achondroplasia in the first quarter
Slide 20: Vision 2030: Be the Leading Endocrinology Rare Disease Company
Vision 2030 Be the Leading Endocrinology Rare Disease Company Achieve blockbuster status (>$1B) for TransCon PTH, TransCon hGH, and TransCon CNP through worldwide commercialization Be the leader in Growth Disorders and Hypoparathyroidism, pursuing clinical conditions, innovative LCM and complementar