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Week 78 COACH Trial Data & Achondroplasia Program Update August 6 , 2026 For investor communication only . Not for use in product promotion . Not for further distribution . ascendis pharma
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This presentation contains forward-looking statements. All statements other than statements of historical facts contained in this presentation, such as, statements regarding our product features, goals, efficacy and safety, future plans, expected patient recruitment and timing, and market position are forward-looking statements. These forward-looking statements are based on our current expectations and beliefs, as well as assumptions concerning future events. These statements involve known and unknown risks, uncertainties and other factors that could cause our actual results to differ materially from the results discussed in the forward-looking statements. These risks, uncertainties and other factors are more fully described in our reports filed with or submitted to the Securities and Exchange Commission (SEC), including, without limitation, our most recent Annual Report on Form 20-F filed with the SEC on February 11, 2026, particularly in the sections titled “Risk Factors” and “Operating and Financial Review and Prospects.” In light of the significant uncertainties in our forward-looking statements, you should not place undue reliance on these statements or regard these statements as a representation or warranty by us or any other person that we will achieve our objectives and plans in any specified timeframe, or at all. Any forward-looking statement made by us in this presentation speaks only as of the date of this presentation and represents our estimates and assumptions only as of the date of this presentation. Except as required by law, we assume no obligation to update these statements publicly, whether as a result of new information, future events, changed circumstances or otherwise after the date of this presentation. TransCon CNP is approved in the United States under the trade name YUVIWEL® to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses. All other uses discussed in this presentation, including treatment with TransCon CNP in combination with TransCon hGH and treatment of infants and adolescents with achondroplasia, are investigational and have not been approved by the U.S. Food and Drug Administration or any other regulatory authority. TransCon hGH is investigational in achondroplasia and is approved and marketed as SKYTROFA® for the treatment of pediatric and adult growth hormone deficiency. No representation is made as to the safety or effectiveness of any investigational use. Cautionary Note on Forward-Looking Statements © 2026 Ascendis Pharma. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, TransCon, SKYTROFA® and YUVIWEL® are trademarks owned by the Ascendis Pharma group. For investor communication only. Not for use in product promotion. Not for further distribution2
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For investor communication only. Not for use in product promotion. Not for further distribution3 Program Overview Combination Therapy Overview & Recent Data • TransCon CNP overview • COACH Trial design • Baseline characteristics • Week 78 efficacy and safety • Key takeaways YUVIWEL® U.S. Launch Update • Key FDA label takeaways • U.S. pediatric achondroplasia market overview • Enrollment update and launch progress highlights • ApproaCH Trial design • Recently presented long-term data from ApproaCH Trial • Broader achondroplasia development program • Key takeaways TransCon CNP Monotherapy Overview & Recent Data
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For investor communication only. Not for use in product promotion. Not for further distribution4 TransCon CNP Monotherapy Overview & Recent Data Combination Therapy Overview & Recent Data YUVIWEL® U.S. Launch Update
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Medical complications of achondroplasia vary and may include spinal cord compression, sleep-disordered breathing, otitis media, hearing loss, abnormal curvature of the spine, spinal stenosis (narrowing), and more. 1-7 For investor communication only. Not for use in product promotion. Not for further distribution.5 Medical Complications Over a Lifespan 1. Ireland PJ, et al. Appl Clin Genet 2014; 7: 117-25. 2. Horton WA, et al. Lancet 2007; 370(9582): 162-72. 3. Sims DT, et al. J Appl Physiol 2018; 124(3): 696-703. 4. de Vries OM, et al. Am J Med Genet A 2021;185(4): 1023-32. 5. Pauli RM. Orphanet J Rare Dis 2019;14(1):1. 6. Dhiman N, et al. Qual Life Res 2017; 26(5): 1337-48. 7. Savarirayan R, et al. Nat Rev Endocrinol 2022; 18(3): 173-89. 8. McGraw SA, et al. Adv Ther 2022; 39(7): 3378-91 9. U.S. FDA. Minutes of a Joint Meeting of the Pediatric Advisory Committee and the Endocrinologic and Metabolic Drugs Advisory Committee. 2018. https://www.fda.gov/media/114640/download. • Leg bowing • Otitis media • Hearing loss • Spinal abnormalities • Spinal stenosis • Foramen magnum stenosis • Obesity • Skeletal dysplasia • Enlargement of specific brain structures Major treatment goals are to decrease the incidence and severity of achondroplasia- related medical complications, increase height, promote proportional growth, and improve quality of life.8,9 • Limited elbow extension • Low muscle tone with weakness • Impaired muscle strength and stamina • Upper airway obstruction • Sleep-disordered breathing • Restricted hip mobility • Speech delay • Misalignment of teeth
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For investor communication only. Not for use in product promotion. Not for further distribution.6 TransCon® CNP (Navepegritide) CNP = C-type natriuretic peptide; FDA = Food and Drug Administration; FGFR3 = fibroblast growth factor receptor 3; NPR-B = natriuretic peptide receptor B Breinholt VM, et al. J Pharmacol Exp Ther 2019;370(3):459-471. • TransCon CNP (marketed as YUVIWEL® in the U.S.) is a prodrug of CNP administered once weekly and designed to provide continuous exposure to active CNP (amino acid sequence identical to endogenous CNP [89-126]) • Active CNP released from TransCon CNP binds to NPR-B throughout the body to counteract the constitutively active FGFR3 signaling in achondroplasia • TransCon CNP is approved by the FDA to increase linear growth in children 2 years and older with achondroplasia with open epiphyses NPR-B
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TransCon CNP treatment in ACcomplisH / AttaCH N=12 TransCon CNP treatment-naïve cohort N=9 TransCon CNP-treated cohort Week 78 Interim Analysis Screening Week 26 Interim Analysis TransCon CNP + TransCon hGH Combination Therapy COACH Trial Design 7 Data on file, Ascendis Pharma 2026. Primary Efficacy Objective • Evaluate effect of combination treatment with TransCon CNP and TransCon hGH on linear growth compared to TransCon CNP alone Population • Children with achondroplasia, aged 2-11 years, with open epiphyses Treatment • TransCon CNP 100 µg/kg/week + TransCon hGH 0.30 mg hGH/kg/week (starting dose) Primary Efficacy Endpoint • Annualized growth velocity (AGV) at Week 52 Selected Secondary Endpoints • Change from baseline in height Z-score • AGV over time • Upper to lower body segment ratio (body proportionality) • Radiological endpoints including lower limb alignment and interpedicular distance (IPD) • Arm span Safety Endpoints • Treatment-emergent AEs, including injection site reactions Week 104 Interim Analysis Week 52 Primary Analysis Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution
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TransCon CNP + TransCon hGH Combination Therapy Demographics and Baseline Characteristics (1/2) Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution.8 McDonnell CM, Irving M, Nolting LA, et al. Navepegritide combined with lonapegsomatropin for the treatment of children with achondroplasia: 52-week results from the phase 2 COACH trial. Eur J Endocrinol. 2026;194(6):745-755. doi:10.1093/ejendo/lvag082. Data on file, Ascendis Pharma 2026. Full analysis set TransCon CNP Treatment-Naïve Cohort (N=12) TransCon CNP-Treated Cohort (N=9) Age at baseline, years, mean (SD) 5.26 (2.24) 8.32 (1.86) Age group, n (%) < 5 years 6 (50.0) 0 5 to < 8 years 5 (41.7) 3 (33.3) ≥ 8 years 1 (8.3) 6 (66.7) Sex, n (%) Male 8 (66.7) 6 (66.7) Female 4 (33.3) 3 (33.3) Genetic variant, n (%) 1138G>A 11 (91.7) 8 (88.9) 1138G>C 0 1 (11.1) 1144G>A 1 (8.3) 0
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TransCon CNP + TransCon hGH Combination Therapy Demographics and Baseline Characteristics (2/2) Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution.9 1CDC Stature for Age Charts, available at: https://www.cdc.gov/growthcharts/who-growth-charts.htm. 2Hoover-Fong JE, et al. US. Orphanet J Rare Dis. 2021;16(1):522. 3Merker A, Neumeyer L, Hertel NT, et al. Am J Med Genet A 2018;176(9):1819–1829. doi:10.1002/ajmg.a.40356. McDonnell CM, Irving M, Nolting LA, et al. Navepegritide combined with lonapegsomatropin for the treatment of children with achondroplasia: 52-week results from the phase 2 COACH trial. Eur J Endocrinol. 2026;194(6):745-755. doi:10.1093/ejendo/lvag082. Full analysis set TransCon CNP Treatment-Naive Cohort (N=12) TransCon CNP-Treated Cohort (N=9) Years of exposure to TransCon CNP 100 µg/kg/wk, mean (range) Not Applicable 2.56 (2.30, 2.95) AGV (cm/year), mean (SD) 4.92 (2.18) 5.14 (0.53) CDC-based1 height Z-score, mean (SD) -4.46 (0.77) -4.04 (0.66) ACH-specific2 height Z-score, mean (SD) 0.46 (0.70) 1.28 (0.81) ACH-specific3 arm span Z-score, mean (SD) 0.18 (1.12) 0.61 (0.84) IGF-1 SDS, mean (SD) -0.63 (1.32) -0.70 (0.48) Trial population is representative of children with achondroplasia and treatment benefit of TransCon CNP Data on file, Ascendis Pharma 2026.
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Adapted from: Hoover-Fong JE, et al. Am J Clin Nutr. 2008 Aug;88(2):364-71. Natural history AGV curves presented for male children; curves for average stature children from 0-3y reflect 10th, 50th, and 90th percentile. The data have been plotted on the growth curves for boys as the majority of participants enrolled in COACH were boys, and the growth curves for girls and boys are similar for the age range enrolled in COACH.; McDonnell CM, Irving M, Nolting LA, et al. Navepegritide combined with lonapegsomatropin for the treatment of children with achondroplasia: 52-week results from the phase 2 COACH trial. Eur J Endocrinol. 2026;194(6):745-755. doi:10.1093/ejendo/lvag082. 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 0 5 10 15 20 Age (years) AGV (cm/year) TransCon CNP + TransCon hGH Combination Therapy COACH Trial Mean AGV at Week 78 Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution10 97th 50th 3rd 95th 50th 5th At Week 78, children on the combination maintained AGV at or above the 97th percentile of children of average stature, with changes over time in line with expected age-related growth patterns Achondroplasia Growth Chart Average-stature Growth Chart Data on file, Ascendis Pharma 2026. W26 W52 W78W26 W52 W78BL BL 8.25 8.42 7.67 9.14 8.80 7.73 5.14 4.92 TransCon CNP Treatment-Naïve Cohort TransCon CNP-Treated Cohort AGV (cm/year) Age (years)
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TransCon CNP + TransCon hGH Combination Therapy Long-Term ApproaCH and COACH Mean AGV 0 1 2 3 4 5 6 7 8 9 10 11 12 13 14 0 5 10 15 20 Age (years) AGV (cm/year) Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution11 97th 50th 3rd 95th 50th 5th Achondroplasia Growth Chart Average-stature Growth Chart W26 W52 W78BL AGV (cm/year) Age (years) TransCon CNP monotherapy improved growth rates in line with children of average stature, while combination therapy enhanced growth even further W26 W52 W78 W104BL Adapted from: Hoover-Fong JE, et al. Am J Clin Nutr. 2008 Aug;88(2):364-71. Natural history AGV curves presented for male children; curves for average stature children from 0-3y reflect 10th, 50th, and 90th percentile. The data have been plotted on the growth curves for boys as the majority of participants enrolled in COACH were boys, and the growth curves for girls and boys are similar for the age range enrolled in COACH.; McDonnell CM, Irving M, Nolting LA, et al. Navepegritide combined with lonapegsomatropin for the treatment of children with achondroplasia: 52-week results from the phase 2 COACH trial. Eur J Endocrinol. 2026;194(6):745-755. doi:10.1093/ejendo/lvag082.; Savarirayan R, et al. American College of Medical Genetics and Genomics Annual Meeting (ACMG). 2026. Oral Presentation; Data on file, Ascendis Pharma 2026. 5.14 6.12 5.95 5.77 5.65 3.95 8.25 8.42 7.67 TransCon CNP-Treated Cohort
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TransCon CNP + TransCon hGH Combination Therapy Mean ACH Height Z-score Through Week 78 Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution12 1Gray arrow indicates change from baseline 2COACH is a single-arm, open-label Phase 2 trial in 21 children and does not include a monotherapy comparator arm. Comparisons to results from ApproaCH, a separate randomized trial, are cross-trial and descriptive only; the trials differ in design, population, and duration, and no statistical inference of relative efficacy should be drawn. 0.46 0.99 1.47 1.75 0.0 0.2 0.4 0.6 0.8 1.0 1.2 1.4 1.6 1.8 2.0 2.2 2.4 Baseline Week 26 Week 52 Week 78 +1.29 Sustained increases in ACH height Z-scores, indicating a tripling of efficacy compared to TransCon CNP monotherapy2 Data on file, Ascendis Pharma 2026. 1.28 1.72 2.15 2.38 0.0 0.2 0.4 0.6 0.8 1.0 1.2 1.4 1.6 1.8 2.0 2.2 2.4 Baseline Week 26 Week 52 Week 78 +1.10 P<0.0001 P<0.0001 TransCon CNP Treatment-Naïve Cohort TransCon CNP-Treated Cohort TransCon CNP + TransCon hGH Baseline TransCon CNP-Treated Cohort TransCon CNP + TransCon hGH Baseline TransCon CNP Treatment-Naïve Cohort ACH Height Z-Score (Mean) 1 1
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TransCon CNP + TransCon hGH Combination Therapy COACH Body Proportionality Through Week 78 Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution13 -0.12 -0.11 -0.10 -0.09 -0.08 -0.07 -0.06 -0.05 -0.04 -0.03 -0.02 -0.01 0.00 0.01 0.02 Upper to Lower Body Segment Ratio Change from Baseline (Mean (±SE)) Baseline -0.04 Week 26 -0.07 Week 52 -0.08 Week 78 -0.11 -0.10 -0.09 -0.08 -0.07 -0.06 -0.05 -0.04 -0.03 -0.02 -0.01 0.00 0.01 0.02 Baseline -0.05 Week 26 -0.06 Week 52 -0.09 Week 78 TransCon CNP-Treated Cohort TransCon CNP + TransCon hGH showed continued improvement in body proportionality Data on file, Ascendis Pharma 2026. TransCon CNP-Treatment Naive Cohort Improvement
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TransCon CNP + TransCon hGH Combination Therapy Safety Profile Consistent with Monotherapies Through Week 78 14 Safety analysis set All Participants (N=21) Participants experiencing any treatment-emergent AE, n (%) 20 (95.2) Grade 1 (Mild) Grade 2 (Moderate) Grade 3 (Severe) a Grade 4 & 5 (Life-threatening or Death) 20 (95.2) 14 (66.7) 2 ( 9.5) 0 Participants experiencing any SAE, n (%) b, c Acquired hydrocele Respiratory tract infection viral Syncope 2 (9.5) 1 (4.8) 1 (4.8) 1 (4.8) Participants with at least one treatment-related AEs, n (%) Participants experiencing AE related to TransCon hGH Participants experiencing AE related to TransCon CNP Participants experiencing AE related to TransCon hGH and TransCon CNP d 8 (38.1) 7 (33.3) 5 (23.8) 1 (4.8) Treatment-related SAEs, n (%) 0 Participants discontinuing treatment due to AE, n (%) 0 a One participant reported OSA (not related to treatment by the investigator), 2 weeks after initial dose in COACH (participant had prior medical history of OSA that resolved in Jan 2023). Participant recovered and no action taken with treatment. Refer to footnote c for second participant. b One participant reported 2 SAEs: viral respiratory tract infection (moderate; assessed not related to treatment by the investigator) and acquired hydrocele (moderate; assessed not related to treatment by the investigator). Participant recovered and no action taken with treatment. c One participant reported vasovagal syncope (severe; not related to treatment by the investigator), 14 months after initial dose in COACH, approximately 45 minutes after eating. Participant recovered and no action taken with treatment. d One participant reported injection site atrophy of the right buttock (mild). Because the injection site was used for both drugs, the investigator attributed the atrophy to both drugs rather than a single agent. AE is ongoing and no action taken with treatment. Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution
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TransCon CNP + TransCon hGH Combination Therapy Safety and Tolerability Summary Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution15 Data on file, Ascendis Pharma 2026. • Combination treatment showed safety data consistent with those observed for TransCon CNP and TransCon hGH 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 these ages • No TEAEs led to treatment discontinuation or withdrawal from trial; no SAEs assessed as related to study drugs • No fractures or other bone-related safety events observed • No evidence of hypotensive effect • No deaths were reported • Injection-site reactions were consistent with that observed for TransCon CNP and TransCon hGH monotherapies, with all events adjudicated as mild
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TransCon CNP + TransCon hGH Combination Therapy COACH Trial Week 78 Key Takeaways Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution16 Data on file, Ascendis Pharma 2026. At Week 78, combination therapy with once-weekly TransCon CNP + TransCon hGH delivered a tripling of efficacy compared to TransCon CNP monotherapy, without compromising safety or tolerability Potential to establish a new bar for healthy and proportional growth in children with achondroplasia • Combination therapy continued to demonstrates durable unprecedented efficacy ◦ Children on the combination maintained AGV at or above the 97th percentile of children of average stature, with changes over time in line with expected age-related growth patterns ◦ Sustained increases in ACH height Z-scores, indicating a tripling of efficacy compared to TransCon CNP monotherapy • Body proportionality continued to improve through Week 78, aligning with the increase in height • Combination treatment was generally well tolerated, with generally mild TEAEs and safety and tolerability consistent with those observed with TransCon CNP and TransCon hGH monotherapies • 100% of children completed 78 weeks of treatment in COACH and remain in the trial as of today
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For investor communication only. Not for use in product promotion. Not for further distribution17 TransCon CNP Monotherapy Overview & Recent Data YUVIWEL® U.S. Launch Update Combination Therapy Overview & Recent Data
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TransCon CNP Monotherapy Pivotal ApproaCH Trial Design For investor communication only. Not for use in product promotion. Not for further distribution.18 N = number of children that received at least one dose of investigational medicinal product. ACH, achondroplasia; AGV, annualized growth velocity 1For an overview of the trial design see Savarirayan R, et al. JAMA Pediatr. 2026;180(1):18–25. Key Assessments Growth • AGV through Week 104 • Change from baseline in ACH height Z-score Safety • Treatment emergent adverse events (AEs) • Tolerability Exploratory • Change from baseline in key orthopedic features of skeletal dysplasia • Radiographs collected at baseline, Week 52, and Week 104 were assessed by blinded, expert central readers Baseline Characteristics TransCon CNP N=57 placebo N=27 Age, years; mean (SD) [min, max] 5.6 (2.6) [2.0, 11.9] 6.0 (2.7) [2.1, 12.0] Sex, male; n (%) 31 (54.4) 14 (51.9) ACH height Z-score; mean (SD) 0.18 (0.92) -0.11 (0.73) Double-Blind Period Open-Label Extension (OLE) TransCon CNP 100 µg/kg/week Placebo 2:1 Randomization Week 52 Week 104 TransCon CNP 100 µg/kg/week TransCon CNP 100 µg/kg/week N=84 n=55 n=27 Treatment-naïve children with achondroplasia (aged 2 to 11 years) n=53 n=27 n=57 n=27
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1.0 2.0 3.0 4.0 5.0 6.0 7.0 Annulaized Growth Velocity, cm/year (mean ± SE) Week 26 Week 52 Week 78 Double-Blind Period Open-Label Extension (all children treated with navepegritide) 5.95 5.65 4.29 5.42 Week 104Week 0 navepegritide placebo navepegritide Treatment group navepegritide/navepegritide: placebo/navepegritide: TransCon CNP Monotherapy Annualized Growth Velocity (AGV) Through Week 104 For investor communication only. Not for use in product promotion. Not for further distribution.19 Note: Data shown are observed mean (±SE) Annualized Growth Velocity (AGV). Children who switched from placebo to TransCon CNP at Week 52 had increased AGV at Week 104, comparable to TransCon CNP-related gains observed in the double-blind period Children continuing TransCon CNP in the Open-Label Extension maintained AGV through 104 weeks Annualized TransCon CNP Treatment: TransCon CNP TransCon CNPTransCon CNP: TransCon CNP)
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In the Open-Label Extension, ACH height Z-score increased with TransCon CNP treatment, consistent with TransCon CNP-related improvements seen in the double-blind period TransCon CNP Monotherapy Change in ACH Height Z-score For investor communication only. Not for use in product promotion. Not for further distribution.20 ACH = achondroplasia Improvement -0.1 0.0 0.1 0.2 0.3 0.4 0.5 0.6 0.7 Achondroplasia-specific Height Z-Score (mean ± SE) Change from Baseline (Week 0) Week 26 Week 52 Week 78Week 0 Week 104 +0.29 +0.57 +0.007 +0.29 Double-Blind Period Open-Label Extension (all children treated with navepegritide) Δ+0.27 Δ+0.28 Change from Week 52 to Week 104 navepegritide placebo navepegritide Treatment group navepegritide/navepegritide: placebo/navepegritide: TransCon CNPTransCon CNP Treatment: TransCon CNP TransCon CNP TransCon CNP)
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TransCon CNP Monotherapy Change in CDC Height Z-score For investor communication only. Not for use in product promotion. Not for further distribution.21 CDC = Centers for Disease Control and Prevention -0.2 -0.1 0.0 0.1 0.2 0.3 0.4 0.5 CDC-based Height Z-Score (mean ± SE) Change from Baseline (Week 0) Week 26 Week 52 Week 78Week 0 Week 104 +0.14 +0.42 -0.13 +0.15 Double-Blind Period Open-Label Extension (all children treated with navepegritide) Δ+0.28 Δ+0.28 Change from Week 52 to Week 104 navepegritide placebo navepegritide Treatment group navepegritide/navepegritide: placebo/navepegritide: CDC height Z-score improved with TransCon CNP treatment in the double-blind period, and continued to increase with TransCon CNP treatment in the Open-Label Extension TransCon CNP Treatment: TransCon CNP TransCon CNP placebo/TransCon CNP TransCon CNP) Improvement
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0 4 8 12 16 20 24 10.1 11.811.5 6.97.7 9.1 Mean (SE) TFA, degrees Week 0 Week 52 n= 52 52 52 Week 104 Week 0 Week 52 26 26 26 Week 104 TransCon CNP Monotherapy TFA Decreased Through Week 104 For investor communication only. Not for use in product promotion. Not for further distribution.22 Data reported are paired analyses for children with data available at all timepoints; error bars indicate standard error. TFA is reported as the average of the absolute values (deviations from straight alignment) from each leg; change from baseline represents absolute change from Week 0. TFA = tibial-femoral angle All children Children with baseline TFA ≥5° Greater effects observed in children with genu varum TFA ≥5° TransCon CNP placebo Improved alignment Consistent improvements for children continuing on or switching to TransCon CNP treatment TransCon CNP placebo 0 4 8 12 16 20 24 14.9 18.718.2 9.6 11.3 13.4 Mean (SE) TFA, degrees Week 0 Week 52 n= 25 25 25 Week 104 Week 0 Week 52 13 13 13 Week 104 -2.2 -1.7 -3.8 -3.8
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0 2 4 6 8 10 6.35 7.56 6.13 4.364.67 5.40 Mean (SE) TFA Z-score Week 0 Week 52 n= 25 25 25 Week 104 Week 0 Week 52 13 13 13 Week 104 0 2 4 6 8 10 4.284.76 4.08 2.963.153.62 Mean (SE) TFA Z-score Week 0 Week 52 n= 52 52 52 Week 104 Week 0 Week 52 26 26 26 Week 104 TransCon CNP Monotherapy TFA Z-scores Continued to Decrease Through Week 104 For investor communication only. Not for use in product promotion. Not for further distribution.23 Data reported are paired analyses for children with data available at all timepoints; error bars indicate standard error. TFA Z-scores were calculated as the average of the absolute values of the TFA Z-scores from both legs, using reference data from children of average stature (Sabharwal S and Zhao C, J Bone Joint Surg Am 2009); change from baseline represents absolute change from Week 0. TFA = tibial-femoral angle TransCon CNP placebo TransCon CNP placebo Improved alignment All children Children with baseline TFA ≥5° -0.66 -1.04 -0.48 -1.21
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TransCon CNP Monotherapy Change in Upper-to-Lower Body Segment Ratio For investor communication only. Not for use in product promotion. Not for further distribution.24 0 -0.14 -0.12 -0.10 -0.08 -0.06 -0.04 -0.02 0.00 0.02 Upper to Lower Body Segment Ratio Change from Baseline Mean (±SE) Week 26 Week 52 Week 78 Week 104 -0.04 -0.10 Double-Blind Period Open-Label Extension navepegritide/navepegritide n=55 n=54 n=52n=57 0 -0.14 -0.12 -0.10 -0.08 -0.06 -0.04 -0.02 0.00 0.02 Week 26 Week 52 Week 78 Week 104 -0.02 -0.10 placebo/navepegritide Double-Blind Period Open-Label Extension n=27 n=27 n=27n=27 Continued improvement in body proportionality through Week 104 TransCon CNP TransCon CNP Improvement
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TransCon CNP Monotherapy Safety and Tolerability Through 104 Weeks For investor communication only. Not for use in product promotion. Not for further distribution.25 N = number of participants with observation. • The overall rate of ISRs was low and all were mild o ISRs per person-year of exposure is equivalent to ~1 ISR for every 3 years of treatment • No symptomatic hypotension occurred through up to 104 weeks of TransCon CNP treatment Most adverse events in TransCon CNP-treated children were mild or moderate, with none leading to treatment discontinuation or withdrawal from the trial Total TransCon CNP-Treated Population (N=84)
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Ascendis clinical program for TransCon CNP covers age groups from newborns through growth plate closure AGE TRIAL NAME DESCRIPTION STATUS Natural history (aged 0 to 8 years) ACHieve1 Observational study of untreated children with achondroplasia Completed Infants (aged 0 to <2 years) reACHin2 Pivotal trial evaluating the safety, tolerability, and efficacy of TransCon CNP in infants with achondroplasia Ongoing Children (aged 2 to 10 years) ACcomplisH3 Phase 2 dose-finding trial Completed Children (aged 2 to 11 years) ApproaCH4 Pivotal trial Completed Adolescents (aged 12 to <18 years) teACH5 Pivotal trial evaluating the safety, tolerability, and efficacy of TransCon CNP in adolescents Ongoing Long-term outcomes AttaCH6 Long-term open-label extension trial following participants from other Ascendis TransCon CNP monotherapy trials to near-final adult height Ongoing For investor communication only. Not for use in product promotion. Not for further distribution.26 TransCon CNP Monotherapy Achondroplasia TransCon CNP Monotherapy Program CNP, C-type natriuretic peptide; 1-6. Clinicaltrials.gov. Accessed January 23, 2025. 1. https://clinicaltrials.gov/study/NCT03875534. 2. https://clinicaltrials.gov/study/NCT06079398 3. https://clinicaltrials.gov/study/NCT04085523. 4. https://clinicaltrials.gov/study/NCT05598320. 5. https://clinicaltrials.gov/study/NCT06732895. 6. https://clinicaltrials.gov/study/NCT05929807.
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TransCon CNP Monotherapy Monotherapy Key Takeaways & Next Steps Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution27 Data on file, Ascendis Pharma 2026. • Consistent and durable improvements in proportional growth seen through 2 years in ApproaCH ◦ Durable height improvements as measured by annualized growth velocity (AGV) and height Z-scores ◦ Durable improvements in tibial-femoral angle and upper-to-lower body segment ratio • High retention rate in completed and ongoing trials, with 96% of the 140 enrolled in AttaCH long-term open-label extension trial remain on TransCon CNP monotherapy or in the ongoing COACH combination therapy trial • Completed target enrollment for pivotal reACHin Trial supporting planned regulatory filings for infants 0 to <2 years of age with achondroplasia • TransCon CNP monotherapy was generally well tolerated, with most adverse events being mild to moderate ◦ No AEs led to treatment discontinuation or withdrawal from trial, no occurrences of symptomatic hypotension, and an overall rate of injection-site reaction of 0.35 per person year of exposure Once-weekly TransCon CNP positioned to be a foundation of care for children with achondroplasia
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For investor communication only. Not for use in product promotion. Not for further distribution28 TransCon CNP Monotherapy Overview & Recent Data YUVIWEL® U.S. Launch Update Combination Therapy Overview & Recent Data
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For investor communication only. Not for use in product promotion. Not for further distribution. • Indicated to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses • LS mean treatment difference between YUVIWEL and placebo was estimated from an analysis of covariance (ANCOVA) model1 ◦ AGV of 1.5 cm/year2 at week 52 for YUVIWEL vs placebo in all children (primary endpoint) ◦ AGV of 1.8 cm/year2 at week 52 for YUVIWEL vs placebo in children aged ≥5 years ◦ AGV was maintained among those who had received 2 years of treatment with YUVIWEL3 • Once-weekly dosing results in continuous systemic exposure of CNP over the dosing interval 4 • Low incidence of injection-site reactions (ISRs) at 0.4 events/child/year observed for YUVIWEL • Switch guidance: Start once-weekly YUVIWEL on the day after completing the last dose of daily CNP therapy • No food or fluid intake required when administering YUVIWEL Key FDA Label Takeaways 1. Included treatment, strata (defined by sex and age), baseline age, and baseline achondroplasia-specific height Z-score as covariates. Results from clinical trial of TransCon CNP consisting of a randomized, double-blind, placebo-controlled 52-week period, followed by a single-arm 52-week OLE period (Trial 1; NCT05598320). 2. Least-squares mean difference in annualized growth velocity. 3. Results from 52-week randomized, double-blind, placebo-controlled Phase 2 dose-finding trial (Trial 2; NCT04085523) where all 57 patients entered the single-arm OLE and continued treatment with TransCon CNP. 4. Mean apparent elimination half-life of released CNP was 5.3 days. YUVIWEL [package insert]. Princeton, NJ: Ascendis Pharma Growth Disorders A/S. February 2026. 29
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For investor communication only. Not for use in product promotion. Not for further distribution.30 Achondroplasia U.S. Landscape at Time of YUVIWEL Launch (April 2026) 1. Children with achondroplasia aged ≤ 18 years. Stevenson et al. Am J Med Genet A 2012; 158A(5): 1046–1054.; 2. Waller DK, et al. Am J Med Genet A 2008; 146A(18): 2385–2389. 30 U.S. Pediatric Achondroplasia Market1,2 Potential to address the unmet needs for those living with achondroplasia: • Currently on a daily therapy • Previously tried a daily therapy and have since discontinued due to tolerability, adherence challenges, or perceived lack of benefit • Naïve to pharmacological treatment Not currently on treatment, 70% On daily treatment, 30% ~2,600 YUVIWEL is expected to grow the therapeutic class with uptake from both treated and untreated patients
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31 YUVIWEL: U.S. Launch Update through June 30 HCPs: healthcare providers UNIQUE PATIENT ENROLLMENTS PRESCRIBING HCPs >170 ~90 Launch Progress Highlights REIMBURSEMENT APPROVAL RATE >65% as of June 30, 2026 • Demand Trajectory: Pace of enrollments steady through June, with all patients new to YUVIWEL therapy • Payor Approval: Encouraging early reimbursement experience; approval rate for Q2 enrollments of >65% • Patient Breadth: Demand from switch, naïve and return-to-treatment patients indicates significant unmet need and broad-based appeal while pointing to market expansion For investor communication only. Not for use in product promotion. Not for further distribution.
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We are committed to providing therapies to address the medical complications of achondroplasia • A strong foundation backed by unparalleled clinical trial monotherapy and combination therapy data • Positioned to offer the broadest choice to address individual treatment goals from infancy through adulthood • Anticipated next steps: ◦ In the European Union, decision anticipated for TransCon CNP Marketing Authorisation Application in the fourth quarter of 2026 ◦ COACH Week 104 results with radiographic data around year end 2026 ◦ Expect to begin patient recruitment for Phase 3 combination therapy trial in children with achondroplasia during the fourth quarter of 2026 Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution32 Summary and Next Steps Data on file, Ascendis Pharma 2026. Ascendis is well-positioned to be leader in growth disorder care with two foundational therapies
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Thank you Ascendis ®