New 2-Year Data from Pivotal ApproaCH Trial of TransCon® CNP (Navepegritide) Show Pronounced Gains in Growth Outcomes in Children with Achondroplasia Aged ≥5 Years

Rhea-AI Summary

Ascendis Pharma (Nasdaq: ASND) reported 2-year subgroup data from the pivotal ApproaCH trial showing children with achondroplasia aged ≥5 at enrollment treated with once-weekly TransCon CNP (navepegritide) had greater annualized growth velocity (AGV) versus placebo at Week 52 and sustained growth through Week 104.

Key metrics: Week 52 AGV treatment difference +1.78 cm/year (LS mean, 95% CI [1.22,2.33]); ACH-specific height Z-score change +0.31 vs placebo. Safety through two years showed low, mild injection-site reactions, no symptomatic hypotension, and no bone-age acceleration.

Positive

• AGV +1.78 cm/year LS mean treatment difference vs placebo at Week 52
• ACH-specific height Z-score +0.31 LS mean improvement vs placebo at Week 52
• Sustained growth through Week 104 in children ≥5 years of age

Negative

• Week 104 data lacks a concurrent placebo arm due to crossover at Week 52
• Subgroup sizes were small: TransCon CNP n=36, placebo n=17

News Market Reaction – ASND

+4.34%

6 alerts

+4.34% Session close to close

$14.62B Market Cap

0.0x Rel. Volume

In the May 7 session, ASND gained 4.34%, reflecting a moderate positive market reaction. Our momentum scanner triggered 6 alerts that day, indicating moderate trading interest and price volatility.

Data tracked by StockTitan Argus on the day of publication.

Market Context

This announcement adds 2‑year subgroup data from the pivotal ApproaCH trial, showing sustained impro...

Analysis

This announcement adds 2‑year subgroup data from the pivotal ApproaCH trial, showing sustained improvements in annualized growth velocity and height Z-scores in children with achondroplasia aged ≥5 years, alongside a safety profile consistent with earlier reports. It builds on prior COACH trial findings and the FDA-approved status of YUVIWEL, which holds orphan exclusivity through February 27, 2033. Going forward, investors may watch for further real‑world data, longer-term safety signals, and uptake trends in achondroplasia treatment.

Key Figures

ApproaCH enrollment: 84 children aged 2–11 years Subgroup size ≥5 years: 36 TransCon CNP vs 17 placebo Dose: 100 µg/kg once-weekly +5 more

8 metrics

ApproaCH enrollment 84 children aged 2–11 years Pivotal ApproaCH trial population

Subgroup size ≥5 years 36 TransCon CNP vs 17 placebo Children ≥5 years at enrollment in ApproaCH

Dose 100 µg/kg once-weekly TransCon CNP regimen in ApproaCH trial

AGV Week 52 5.79 cm/year vs 4.02 cm/year LS mean AGV, TransCon CNP vs placebo at Week 52 (≥5 years subgroup)

AGV Week 104 5.71 cm/year vs 5.53 cm/year Observed AGV, continuous vs crossover group at Week 104

ACH height Z-score +0.75 vs +0.46 at Week 104 Change from baseline, TransCon CNP vs crossover (≥5 years subgroup)

CDC height Z-score +0.58 vs +0.36 at Week 104 Change from baseline, TransCon CNP vs crossover (≥5 years subgroup)

Orphan exclusivity Through February 27, 2033 FDA orphan drug exclusivity for YUVIWEL (navepegritide)

Historical Context

5 past events · Latest: Apr 30 (Neutral)

Pattern 5 events

Date	Event	Sentiment	24h Move	Catalyst
Apr 30	Earnings date notice	Neutral	-3.7%	Announced timing of Q1 2026 results and business update call.
Apr 21	Debt redemption	Neutral	-5.1%	Redemption of all $575M 2.25% Convertible Senior Notes due 2028.
Apr 20	Listing transition	Neutral	+0.4%	Ordinary shares began direct trading on Nasdaq Global Select Market.
Apr 08	Planned relisting	Positive	+5.8%	Announced plan to list ordinary shares directly on Nasdaq and exchange ADSs.
Apr 08	Clinical trial data	Positive	+5.8%	Reported Week 52 Phase 2 COACH data showing added benefits in achondroplasia.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Pattern Detected

Recent positive or strategic headlines have often seen aligned price gains, while some neutral/technical updates drew negative reactions, showing mixed sensitivity to news.

Recent Company History

Over the last month, Ascendis has focused on capital structure, listing changes, and achondroplasia programs. On April 8, 2026, new COACH trial data for TransCon CNP and TransCon hGH coincided with a +5.77% move. The same day, plans to list ordinary shares directly on Nasdaq also saw a +5.77% reaction reported. The subsequent transition to ordinary-share trading on April 20, 2026 had a small gain, while the $575.0M convertible note redemption and an earnings date notice were followed by declines. Today’s 2‑year ApproaCH data extend this achondroplasia efficacy and safety narrative.

Key Terms

achondroplasia, annualized growth velocity, placebo-controlled, open-label extension, +4 more

8 terms

achondroplasia medical

"children with achondroplasia ≥5 years of age at enrollment treated with once-weekly"

A genetic condition that causes the most common form of short stature, where a specific change in a growth-control gene makes the long bones grow more slowly, producing a distinct body proportion and sometimes breathing, spinal or joint issues. It matters to investors because it defines a clear patient population, predictable medical needs and regulatory pathways for drugs or devices—similar to a niche market with steady, long-term demand for effective treatments or supportive care.

annualized growth velocity medical

"demonstrated significantly greater annualized growth velocity (AGV) compared to placebo"

Annualized growth velocity is a way of expressing how fast a financial metric (such as revenue, earnings, or user base) would grow over a full year based on growth observed during a shorter period. Investors use it to compare performance across companies or timeframes and to project future size; think of timing how far a car travels in one minute and converting that to a miles‑per‑hour estimate to understand overall speed.

placebo-controlled medical

"ApproaCH was a randomized, double-blind, placebo-controlled pivotal trial in 84 children"

"Placebo-controlled" describes a testing method where one group receives the actual treatment or intervention, while another group receives a harmless, inactive version called a placebo. This approach helps determine whether the real treatment has genuine effects beyond psychological expectations. For investors, understanding this ensures confidence that reported benefits are real and not influenced by bias or false perceptions.

open-label extension medical

"followed by a 52-week open-label extension (OLE) period in which all participants"

An open-label extension is a continuation of a clinical trial where all participants and researchers know which treatment is being given, often after an initial blinded phase. It allows further study of a drug's long-term safety and effectiveness. For investors, it can indicate ongoing interest and confidence in a product's potential, influencing perceptions of its future value.

injection site reactions medical

"with a low rate of injection site reactions (ISRs, all mild), no symptomatic hypotension"

Injection site reactions are local skin and tissue responses—such as redness, swelling, pain, itching or bruising—that occur where a drug or vaccine is injected. For investors, they matter because frequent or severe reactions can affect patient acceptance, regulatory review, labeling, and overall commercial success in the same way a product with bothersome side effects can struggle to gain long-term customers.

z-score medical

"ACH-Specific Height Z-score, Change from Baseline | CDC-Based Height Z-score"

A z-score is a standardized number that shows how far a single value lies from the average, measured in units of typical variation—like saying someone is two “height-steps” taller than the crowd. For investors it makes different measures comparable and helps spot unusual performance or risk: large positive or negative z-scores flag outliers that may indicate opportunity, mispricing, or potential financial trouble.

ls mean technical

"The observed mean is a simple average of recorded measurements; the LS mean is"

LS mean (least squares mean) is an adjusted average calculated by a statistical model to compare groups while accounting for differences like baseline measurements or other factors. For investors, it matters because trial or study results reported as LS means show the effect of a treatment after removing confounding influences, similar to comparing students’ final scores after correcting for different starting skill levels.

t-test technical

"Treatment differences between TransCon CNP and placebo were estimated from a T-test"

A t-test is a statistical check that asks whether the difference between two average values is likely real or just the result of random variation, like deciding if two coin-toss results truly differ. Investors use it to judge whether observed gaps — for example in returns, earnings changes, or performance before and after an event — are statistically meaningful rather than noise, helping inform confidence in decisions and risk assessments.

AI-generated analysis. How Rhea-AI works. Not financial advice.

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COPENHAGEN, Denmark, May 06, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced new data from a subgroup analysis showing that children with achondroplasia ≥5 years of age at enrollment treated with once-weekly TransCon CNP (navepegritide) in its pivotal ApproaCH Trial demonstrated significantly greater annualized growth velocity (AGV) compared to placebo at Week 52, and sustained these growth improvements through up to two years of treatment. The safety profile for this subgroup through up to two years of treatment was similar to the overall population, with a low rate of injection site reactions (ISRs, all mild), no symptomatic hypotension, and no acceleration of bone age. The data follow previously reported Week 104 results showing consistent improvements in growth and body proportionality in the overall population, and expand on data recently presented by M. Jennifer Abuzzahab, M.D. during PES 2026, the annual meeting of the Pediatric Endocrine Society.

“We are pleased to see confirmation of the expected sustained growth improvements in these children, with a consistent safety and tolerability profile,” said Aimee D. Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Science and Chief Medical Officer at Ascendis Pharma. “These results, along with the improved skeletal alignment and body proportionality and positive changes in health-related quality of life previously reported for the overall populationⁱ further highlight TransCon CNP’s ability to promote healthy, proportional growth in children with achondroplasia across age groups.”

ApproaCH Trial Design
ApproaCH was a randomized, double-blind, placebo-controlled pivotal trial in 84 children with achondroplasia aged 2–11 years, investigating TransCon CNP (100 µg/kg once-weekly) versus placebo for 52 weeks, followed by a 52-week open-label extension (OLE) period in which all participants received TransCon CNP through Week 104. Fifty-three of the 84 children were ≥5 years of age at the time of their enrollment in the trial.

Highlights of the ApproaCH Trial Data Through Week 104
Subgroup of children aged ≥5 years at enrollment

	AGV (cm/year)
	LS Mean	Observed Mean3
	Week 52	Week 52	Week 104
TransCon CNP(n=36)	5.79	5.84	5.71
Placebo/TransCon CNP1 (n=17)	4.02	3.88	5.53
TransCon CNP vs. Placebo, Treatment Difference [95% CI]	+1.78 [1.22, 2.33] p<0.0001	+1.972 [1.37, 2.56] p<0.0001	-

	ACH-Specific Height Z-score, Change from Baseline			CDC-Based Height Z-score, Change from Baseline
	LS Mean	Observed Mean3		LS Mean	Observed Mean3
	Week 52	Week 52	Week 104	Week 52	Week 52	Week 104
TransCon CNP (n=36)	+0.38	+0.38	+0.75	+0.28	+0.26	+0.58
Placebo/TransCon CNP1 (n=17)	+0.07	+0.07	+0.46	-0.05	-0.02	+0.36
TransCon CNP vs. Placebo, Treatment Difference2 [95% CI]	+0.31 [0.20, 0.42] p<0.0001	+0.30 [0.18, 0.42] p<0.0001	-	+0.32 [0.20, 0.44] p<0.0001	+0.29 [0.14, 0.44] p=0.0004	-

Note: The observed mean is a simple average of recorded measurements; the LS mean is a model-based estimated average that adjusts for selected variables, typically baseline patient characteristics, enabling a more balanced comparison across arms of a clinical trial.
¹ Week 104 data reflects placebo patients that crossed over to TransCon CNP treatment at Week 52
² Not presented at PES 2026; included for context
³ Treatment differences between TransCon CNP and placebo were estimated from a T-test

Through up to two years of treatment, the safety profile in children ≥5 years was similar to the overall population, with a low rate of ISRs (all mild), no symptomatic hypotension, and no acceleration of bone age. Most adverse events in TransCon CNP-treated children were mild or moderate, with none leading to treatment discontinuation or withdrawal from the trial.

A slide presentation with these data can be found on the Investor Relations & News section of the Ascendis Pharma website: https://investors.ascendispharma.com.

About TransCon CNP
TransCon CNP is a prodrug of C-type natriuretic peptide (CNP) administered once weekly, designed to provide continuous exposure of active CNP to receptors on tissues throughout the body to counteract the overactive FGFR3 signaling in achondroplasia. In February 2026, TransCon CNP was approved by the U.S. Food & Drug Administration (FDA) under the trade name YUVIWEL^® to increase linear growth in pediatric patients 2 years of age and older with achondroplasia with open epiphyses. Ascendis Pharma’s Marketing Authorisation Application for YUVIWEL is under review by the European Medicines Agency, with a regulatory decision anticipated in the fourth quarter of 2026.

About Achondroplasia
Achondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, and is associated with an increased risk of muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia can vary from individual to individual and across different stages of life. Throughout infancy and childhood, observed complications include spinal abnormalities, enlarged brain ventricles, impaired muscle strength and reduced stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; some of which persist or worsen in adulthood. These medical complications can affect physical well-being and quality of life, and may be impacted by a range of individual, clinical, and social factors. Some individuals with achondroplasia require multiple procedures and surgeries to address specific functional or anatomical concerns.

About Ascendis Pharma A/S
Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark, and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more.

Forward-Looking Statements
This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Examples of such statements include, but are not limited to, statements relating to (i) TransCon CNP’s ability to promote healthy, proportional growth in children with achondroplasia across age groups, (ii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients and (iii) Ascendis’ use of TransCon to create new and potentially best-in-class therapies. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including, without limitation: dependence on third‑party manufacturers, distributors, and service providers for Ascendis’ products and product candidates; risks related to regulatory review and approval, including the possibility of delays, requests for additional data or analyses, restrictions or limitations on use, approval with labeling that is more limited than expected, or failure to obtain approval in the United States, European Union, or other jurisdictions; clinical development risks, including that results from ongoing or future trials may not confirm earlier data; unforeseen safety or efficacy findings in development programs or on‑market products; manufacturing, supply chain, quality, or logistics issues that could delay development or commercialization; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen research and development or selling, general and administrative expenses and other costs impacting Ascendis’ business generally; market acceptance, pricing, and reimbursement challenges, including payer coverage decisions and health technology assessments; competitive developments, including new or improved therapies; intellectual property protection, freedom‑to‑operate, and litigation risks; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; cybersecurity, data privacy, and information technology disruptions; and the impact of international economic, political, legal, compliance, public health, and business factors, including tariffs, trade policies, currency fluctuations, and geopolitical events. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 11, 2026, and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law.

Ascendis, Ascendis Pharma, the Ascendis Pharma logo, TransCon, and YUVIWEL^® are trademarks owned by the Ascendis Pharma group. © May 2026 Ascendis Pharma A/S.

Investor Contact:	Media Contact:
Chad Fugere	Melinda Baker
Ascendis Pharma	Ascendis Pharma
+1 (650) 519-7494	+1 (650) 709-8875

FAQ

What did Ascendis Pharma (ASND) report about TransCon CNP results for children ≥5 years on May 6, 2026?

TransCon CNP showed greater annualized growth velocity versus placebo at Week 52 and sustained gains to Week 104. According to the company, the Week 52 AGV treatment difference was +1.78 cm/year (LS mean, 95% CI [1.22,2.33]).

How much did TransCon CNP increase ACH-specific height Z-score in the ≥5-year subgroup (ASND)?

The ACH-specific height Z-score improved by +0.31 LS mean versus placebo at Week 52. According to the company, this difference had a 95% CI [0.20,0.42], indicating a measurable subgroup benefit.

What safety findings did Ascendis Pharma (ASND) report for TransCon CNP through two years?

Safety in the ≥5-year subgroup showed mainly mild or moderate events, low injection-site reactions, no symptomatic hypotension, and no bone-age acceleration. According to the company, no events led to treatment discontinuation.

Does the Week 104 data for TransCon CNP include a placebo control for children ≥5 years (ASND)?

No, Week 104 lacks a concurrent placebo arm because placebo patients crossed over to TransCon CNP at Week 52. According to the company, Week 104 reflects open-label treatment after crossover.

What were the subgroup sample sizes for the ≥5-year analysis in the ApproaCH trial (ASND)?

The subgroup included 53 children ≥5 years at enrollment, with TransCon CNP n=36 and placebo/TransCon n=17. According to the company, these counts underpin the reported Week 52 and Week 104 analyses.