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January 8, 2026 Phase 2 COACH Trial Topline Week 52 Data Treatment with TransCon CNP and TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution.
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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 TransCon hGH’s ability to complement TransCon CNP’s holistic treatment of achondroplasia; the potential of combination therapy to derive durable growth; the ability of TransCon CNP to enable the complementary effect of growth hormones; the expected timing of Week 78 data from the COACH Trial; our planned new trials to support TransCon CNP + TransCon hGH treatment in additional indications such as hypochondroplasia; our ability to become the leader in growth disorders; plans and objectives of management for future operations and commercialization activities; and future results of current and anticipated products and product candidates, 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 12, 2025, 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. This presentation concerns anticipated products that are under clinical investigation and which have not yet been approved for marketing by the U.S. Food and Drug Administration or any other foreign regulatory authority. These anticipated products are currently limited by Federal law to investigational use, and no representation is made as to their safety or effectiveness for the purposes for which they are being investigated. Cautionary Note on Forward-Looking Statements © 2026 Ascendis Pharma. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, and TransCon are trademarks owned by the Ascendis Pharma group. Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution2
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The first clinical trial to evaluate once-weekly TransCon CNP + TransCon hGH in children with achondroplasia (ACH) • Exceeds historical benchmarks for growth without compromising safety or tolerability at 52 weeks ◦ For treatment-naïve cohort, mean annualized growth velocity (AGV) was 8.80 cm/year with an improvement in mean ACH height Z-score of +1.02 over 52 weeks, indicating a tripling of efficacy compared to TransCon CNP monotherapy ◦ For TransCon CNP-treated cohort (average treatment of 2.56 years), mean AGV was 8.42 cm/year with an improvement in mean ACH height Z-score of +0.86 over 52 weeks ◦ After 52 weeks, children treated with combination therapy exceeded the 97th-percentile of average stature children, while ACH height Z-scores reflect at least tripling of efficacy seen with TransCon CNP monotherapy • Demonstrated benefits beyond linear growth with improvements in body proportionality and arm span, aligning with the increase in linear growth, while bone age remained consistent with chronological age • Safety and tolerability consistent with those observed for monotherapies of TransCon CNP and TransCon hGH; combination therapy was generally well-tolerated, with generally mild treatment-emergent adverse events (TEAEs) • 100% of children completed 52 weeks of treatment and remain on therapy in the extension trial as of today Executive Summary - COACH Trial Data on file, Ascendis Pharma 2026. Results suggest once-weekly TransCon CNP + TransCon hGH raises the bar for treatment of achondroplasia Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution3
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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) 50th 5th 95th 97th 50th 3rd COACH Trial Mean AGV at Week 52 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. 4.92 cm/year Baseline TransCon CNP Treatment-Naïve 8.80 cm/year TransCon CNP Treatment-Naïve 8.42 cm/year TransCon CNP-Treated Achondroplasia Growth Chart Average-stature Growth Chart Results suggest once-weekly TransCon CNP + TransCon hGH raises the bar for treatment of achondroplasia 5.14 cm/year Baseline TransCon CNP-Treated Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution Data on file, Ascendis Pharma 2026. 4
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Combination Therapy Expanding Treatment Paradigm 1. Blum WF, et al. Endocr Connect. 2018;7(6):R212-R222. 2. Devesa J, et al. Clin Med Insights Endocrinol Diabetes. 2016 Oct 12;9:47-71. 3. Rintz E, et al. Int J Mol Sci. 2022; 23(11). 4. Krejci P, et al. PLoS One. 2008; 3(12): e3961. 5. Horton WA, et al. Lancet. 2007; 370(9582):162–72. 6. Miyazawa T, et al. Endocrinology. 2002; 143(9): 3604-10. 7. Yasoda A, et al. Nature medicine. 2004; 10(1): 80-6. TransCon CNP releases the brake Press Accelerator Lift Brake Resting zone Pre-hypertrophic zone Direct effects of GH: Recruitment of stem cells Direct/indirect effects of GH: Chondrocytes increase in numbers Stimulatory effect of CNP: Release the FGFR3-induced BRAKE Proliferative zone Hypertrophic zone GH/CNP exert different effects on the Growth Plate1-7 Calcification zone Ossification zone Indirect effects of GH: Chondrocytes grow and differentiate Figure adapted from: Šromová, V, et al. Cells 2023, 12, 2576. In achondroplasia, overactive FGFR3 signaling acts as a BRAKE, inhibiting bone growth3,4 TransCon CNP releases the brake enabling GH to ACCELERATE outcomes + • TransCon CNP provides holistic treatment of achondroplasia and enables complementary effect of TransCon hGH TransCon hGH presses the accelerator Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution Complementary mechanisms of TransCon CNP and TransCon hGH in growth plates 5
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TransCon CNP-treated in ACcomplisH/AttaCH N=12 TransCon CNP treatment-naïve cohort N=9 TransCon CNP-treated cohort Week 52 Primary Analysis Screening Week 26 Interim Analysis COACH Trial Design 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 Secondary Endpoints • Change from baseline in height Z-score • AGV over time • Upper to lower body segment ratio (body proportionality) Safety Endpoints • Treatment-emergent AEs, including injection site reactions Week 104 Interim Analysis Week 78 Interim Analysis 6 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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Full analysis set at COACH screening TransCon CNP Treatment-Naïve Cohort (N=12) TransCon CNP-Treated 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) ≥ 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 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 Data on file, Ascendis Pharma 2026. 7
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Full analysis set at COACH screening 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) Age at screening, years, mean (min, max) 4.67 (1, 9) 7.89 (5, 10) AGV (cm/year), mean (SD) 4.92 (2.18) 5.14 (0.53) CDC-based** height Z-score, mean (SD) -4.46 (0.77) -4.04 (0.66) ACH-specific* height Z-score, mean (SD) 0.46 (0.70) 1.28 (0.81) IGF-1 SDS, mean (SD) -0.63 (1.32) -0.70 (0.48) Demographics and Baseline Characteristics (2/2) Trial population is representative of children with achondroplasia and treatment benefit of TransCon CNP Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution *Hoover-Fong JE, et al. US. OrphanetJ Rare Dis. 2021;16(1):522. **CDC Stature for Age Charts, available at: https://www.cdc.gov/growthcharts/who-growth-charts.htm Data on file, Ascendis Pharma 2026. 8
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Safety Profile of Combination Therapy Is Consistent with Safety Profile of Monotherapies Data cutoff date: 24Nov2025 All Participants (N=21) Participants experiencing any treatment-emergent AE, n (%) 18 (85.7) Grade 1 (Mild) Grade 2 (Moderate) Grade 3 (Severe) Grade 4 & 5 (Life-threatening or Death) 18 (85.7) 8 (38.1) 1 (4.8) a 0 Most frequent (≥10%) AEs by preferred term, n (%) Nasopharyngitis Injection site erythema Gastroenteritis Upper respiratory tract infection Gastroenteritis viral Hand-foot-mouth-disease Injection site bruising 5 (23.8) 5 (23.8) 5 (23.8) 4 (19.0) 3 (14.3) 3 (14.3) 3 (14.3) Participants experiencing any SAE, n (%) 1 (4.8) b Treatment-related AEs, n (%) Participants experiencing AE related to lonapegsomatropin Participants experiencing AE related navepegritide 7 (33.3) 4 (19.0) Treatment-related SAEs, n (%) 0 AEs of Special Interest (for navepegritide), n (%) Injection site reactions Symptomatic hypotension Fractures 3 (14.3) 0 0 Participants discontinuing treatment due to AE, n (%) 0 a Participant developed severe obstructive sleep apnea (OSA) 2 weeks after starting combination therapy. Had prior medical history of OSA that resolved in Jan 2023. b One participant had 2 SAEs: viral respiratory tract infection (moderate; assessed not related to IMP by PI) and acquired hydrocele (mild; assessed not related to IMP by PI). 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
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Safety and Tolerability Summary • 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 • Bone age remained consistent with chronological age at Week 52 • No fractures or other bone-related safety events observed • No evidence of hypotensive effect • No deaths were reported • Injection tolerability was consistent with that observed for TransCon CNP and TransCon hGH monotherapies, with all events adjudicated as mild Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution Data on file, Ascendis Pharma 2026. 10
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AGV (Mean) ACH Height Z-Score (Mean) Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution Efficacy Treatment-Naïve Cohort at Week 52 (N=12) 1Gray arrow indicates change from baseline 4.92 9.14 8.80 2.0 2.5 3.0 3.5 4.0 4.5 5.0 5.5 6.0 6.5 7.0 7.5 8.0 8.5 9.0 9.5 10.0 AGV (cm/yr) Baseline Week 26 Week 52 +3.89+4.23 0.46 0.99 1.47 0.0 0.2 0.4 0.6 0.8 1.0 1.2 1.4 1.6 1.8 2.0 2.2 Baseline Week 26 Week 52 +1.02 +0.53 TransCon CNP + TransCon hGH Baseline TransCon CNP Treatment-Naïve Cohort P=0.0015 P<0.0001 Results suggest once-weekly TransCon CNP + TransCon hGH raises the bar for treatment of achondroplasia Data on file, Ascendis Pharma 2026. 1 1 P=0.0063 P=0.0009 1 1 11
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AGV (Mean) ACH Height Z-Score (Mean) 1Gray arrow indicates change from Baseline 2 Baseline AGV calculated as annualized growth over the 52 weeks preceding the COACH Trial 5.14 8.25 8.42 2.0 2.5 3.0 3.5 4.0 4.5 5.0 5.5 6.0 6.5 7.0 7.5 8.0 8.5 9.0 9.5 10.0 AGV (cm/yr) Baseline2 Week 26 Week 52 +3.28+3.10 Combination therapy demonstrated durable boosted growth in TransCon CNP-treated children 1.28 1.72 2.15 0.0 0.2 0.4 0.6 0.8 1.0 1.2 1.4 1.6 1.8 2.0 2.2 Baseline Week 26 Week 52 +0.86 +0.44 Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution Data on file, Ascendis Pharma 2026. 1 TransCon CNP + TransCon hGH Baseline TransCon CNP-Treated Cohort 1 1 1 P=0.0006 P=0.0002 Efficacy TransCon CNP-Treated Cohort at Week 52 (N=9) P<0.0001 P<0.0001 12
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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) 50th 5th 95th 97th 50th 3rd COACH Trial Mean AGV at Week 52 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. 4.92 cm/year Baseline TransCon CNP Treatment-Naïve 8.80 cm/year TransCon CNP Treatment-Naïve 8.42 cm/year TransCon CNP-Treated Achondroplasia Growth Chart Average-stature Growth Chart After 52 weeks, children on combination therapy exceeded the 97th-percentile AGV of average-stature children 5.14 cm/year Baseline TransCon CNP-Treated Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution Data on file, Ascendis Pharma 2026. 13
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COACH Body Proportionality at Week 52 -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 Week 26 Week 52 -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 Week 26 Week 52 TransCon CNP-Treated Cohort (N=9) TransCon CNP + TransCon hGH enhanced improvement in body proportionality Improvement Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution Data on file, Ascendis Pharma 2026. TransCon CNP-Treatment Naive Cohort (N=12) 14
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COACH Arm Span at Week 52 Arm span of children on combination therapy improved beyond the 84th-percentile at Week 52 Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution Data on file, Ascendis Pharma 2026. Arm span is presented as observed means (SE); Natural history of arm span in boys with ACH as reported from Merker A, et al. Am J Med Genet A. 2018;176(9):1819-1829, mean (solid line) and ±1 SD (shaded gray area) 0 2 4 6 8 10 12 14 60 80 100 120 Age (years) Mean (SE) Arm Span (cm) Navepegritide-experienced Treatment-naïve Mean +1 SD (~84th percentile) Mean Mean −1 SD (~16th percentile) Achondroplasia Arm Span Chart Mean (SE) Arm Span (cm) TransCon CNP Treated TransCon CNP Treatment-Naive TransCon CNP-Treated Age (years) 15
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3.95 6.12 5.95 2.0 2.5 3.0 3.5 4.0 4.5 5.0 5.5 6.0 6.5 7.0 7.5 8.0 8.5 9.0 9.5 10.0 AGV Mean (cm/yr) Baseline Week 26 (Pivotal) Week 52 (Pivotal) Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution TransCon CNP-based regimens drove consistent and durable growth over one year in treatment-naïve children Durable Efficacy Across ACH Trials1 TransCon CNP Monotherapy (ApproaCH)2,3 TransCon CNP + TransCon hGH (COACH)2 4.92 9.14 8.80 2.0 2.5 3.0 3.5 4.0 4.5 5.0 5.5 6.0 6.5 7.0 7.5 8.0 8.5 9.0 9.5 10.0 AGV Mean (cm/yr) Baseline Week 26 (Phase 2) Week 52 (Phase 2) 1. In achondroplasia (ACH) children naïve to TransCon CNP therapy; 2. Ascendis Pharma data on file 2026; 3. Savarirayan R, et al. JAMA Pediatr. 2026;180(1):18-25. doi:10.1001/jamapediatrics.2025.4771 16
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Combination Therapy Program Next Steps Data on file, Ascendis Pharma 2026. With once-weekly TransCon CNP and TransCon hGH, well-positioned to be the leader in growth disorders Treatment with TransCon CNP +TransCon hGH combination therapy is investigational. For investor communication only. Not for use in product promotion. Not for further distribution • Exceeds historical benchmarks for growth without compromising safety or tolerability at 52 weeks ◦ TransCon CNP enables the complementary effect of TransCon hGH • 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 • 100% of children completed 52 weeks of treatment and remain on therapy in the extension trial as of today • Bone age remained consistent with chronological age at Week 52 • In Q4 2025, Ascendis submitted a protocol and held an end-of-Phase 2 meeting with the FDA regarding a Phase 3 trial of TransCon CNP and TransCon hGH in pediatric achondroplasia • Week 78 COACH data expected Q2 2026 • New trials initiated and planned to support TransCon CNP + TransCon hGH treatment in additional indications 17
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Thank you