New InsiGHTS Trial of TransCon® hGH (Lonapegsomatropin) in Turner Syndrome Demonstrated Comparable Efficacy and Safety to Daily Somatropin at Week 52

Ascendis Pharma (Nasdaq: ASND) reported Week 52 topline results from New InsiGHTS, a Phase 2 randomized, open-label trial in 49 prepubertal children with Turner syndrome.

Rhea-AI Summary

Ascendis Pharma (Nasdaq: ASND) reported Week 52 topline results from New InsiGHTS, a Phase 2 randomized, open-label trial in 49 prepubertal children with Turner syndrome.

TransCon hGH (lonapegsomatropin) produced an LS mean annualized height velocity of 9.05 cm/year, versus 9.04 cm/year for daily somatropin, with comparable safety and no SCFE occurrences through up to 143 weeks follow-up.

Positive

• AHV parity: 9.05 cm/yr for TransCon hGH vs 9.04 cm/yr for daily somatropin
• Lower mean weekly dose: TransCon hGH 0.22 mg/kg/week vs daily somatropin 0.29 mg/kg/week
• Extended follow-up: Safety data reported through up to 143 weeks
• No SCFE events observed in the trial

Negative

• Small cohort: randomized and dosed only 49 children
• Open-label design may introduce assessment bias

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News Market Reaction – ASND

On Mar 17, the day this news came out, ASND closed 1.28% below the previous close.

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Market Context

This announcement details positive Phase 2 New InsiGHTS data in Turner syndrome, with TransCon hGH a...

Analysis

This announcement details positive Phase 2 New InsiGHTS data in Turner syndrome, with TransCon hGH achieving an annualized height velocity of 9.05 cm/year, comparable to daily somatropin at 9.04 cm/year, using a lower mean dose of 0.22 mg/kg/week versus 0.29 mg/kg/week. Safety through up to 143 weeks showed only mild to moderate adverse events and no SCFE cases. In the context of recent FDA approval and pivotal data for other TransCon programs, investors may track future Phase 3 results and potential label expansion progress.

Key Figures

TransCon hGH AHV: 9.05 cm/year Daily somatropin AHV: 9.04 cm/year Trial participants: 49 children +5 more

TransCon hGH AHV

9.05 cm/year

LS mean annualized height velocity at Week 52

Daily somatropin AHV

9.04 cm/year

LS mean annualized height velocity at Week 52 comparator arm

Trial participants

49 children

Randomized and treated in New InsiGHTS Phase 2 trial

TransCon hGH dose

0.22 mg/kg/week

Mean dose at Week 52

Daily somatropin dose

0.29 mg/kg/week

Mean dose at Week 52 comparator cohort

Follow-up duration

143 weeks

Maximum safety and tolerability follow-up in trial

SCFE incidence

<1%

Long-term daily somatropin SCFE rate in Turner syndrome literature

Patient age range

1–10 years

Age of prepubertal Turner syndrome children enrolled

Historical Context

5 past events · Latest: Mar 16

5 events

1. Mar 16

   Pivotal trial update

   24h Move

   +2.0%

   Two-year ApproaCH data showed sustained growth and improved body proportionality.

2. Mar 02

   Conference participation

   24h Move

   +3.7%

   Announcement of participation in TD Cowen healthcare conference with investor webcast.

3. Feb 27

   FDA approval

   24h Move

   +3.7%

   FDA accelerated approval of once-weekly YUVIWEL for children with achondroplasia.

4. Feb 11

   Earnings report

   24h Move

   -1.5%

   Q4 and full-year 2025 results with net loss despite growing product revenue.

5. Feb 04

   Earnings date notice

   24h Move

   -1.1%

   Scheduling announcement for upcoming FY2025 results and business update call.

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

Key Terms

annualized height velocity, slipped capital femoral epiphysis, igf-1, adverse events

4 terms

annualized height velocity medical

"At Week 52, children treated with TransCon hGH demonstrated improved annualized height velocity (AHV)..."

Annualized height velocity is a measure of how fast a person grows in height, scaled up to a one‑year rate (for example, centimeters per year) based on shorter observation periods. Investors watch it because it quantifies the effectiveness of therapies or interventions aimed at promoting growth—like reading a car’s miles‑per‑hour to judge performance—so stronger, sustained increases can signal greater clinical value and market potential.

slipped capital femoral epiphysis medical

"TransCon hGH showed a safety and tolerability profile comparable... with no occurrences of slipped capital femoral epiphysis (SCFE)"

Slipped capital femoral epiphysis is a childhood hip disorder where the head of the thigh bone shifts off its growth plate, similar to the top of a ball slipping off its stem, causing pain, limping and limited movement. Investors should care because it often requires surgery, long-term follow-up and rehabilitation, which affects demand for orthopedic devices, hospital services, potential liability and health-care costs, and can influence regulatory reviews and reimbursement decisions for treatments.

igf-1 medical

"They were treated with either TransCon hGH or daily somatropin. Doses were subsequently individualized based on IGF-1."

A naturally occurring protein hormone that helps cells grow, survive and repair, acting like a biological 'fertilizer' for tissues. Investors watch IGF-1 because drugs that raise, block, or mimic it can affect treatments for growth disorders, metabolic diseases and certain cancers, making it a common target in clinical trials; trial results, safety concerns or regulatory decisions tied to IGF-1 activity can materially change a healthcare company’s value.

adverse events medical

"Adverse events (AEs) were mild to moderate in severity, with no AEs leading to discontinuation..."

Adverse events are any harmful or unwanted medical occurrences experienced by people using a drug, device, or undergoing a treatment, whether or not the problem is caused by the product. Think of them as complaints or breakdowns noticed during a trial or after a product is on the market; regulators record and investigate them. Investors care because clusters or serious adverse events can delay approvals, trigger costly studies or recalls, change labeling, and quickly alter a company’s revenue and risk profile.

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

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- Annualized height velocity of 9.05 cm/year (LS mean) for TransCon hGH-treated children was similar at Week 52 to daily somatropin-treated children 

- TransCon hGH showed a safety and tolerability profile comparable to daily somatropin with no occurrences of slipped capital femoral epiphysis (SCFE)

COPENHAGEN, Denmark, March 17, 2026 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced positive Week 52 topline results from New InsiGHTS, its Phase 2 randomized, open-label, active-controlled trial in the U.S. investigating the safety, tolerability, and efficacy of once-weekly TransCon hGH (lonapegsomatropin; U.S. FDA-approved for pediatric and adult growth hormone deficiency (GHD) and approved in other territories for pediatric GHD) compared to daily somatropin in prepubertal children with Turner syndrome.

New InsiGHTS randomized and dosed 49 children with Turner syndrome aged 1 to 10 years old. They were treated with either TransCon hGH or daily somatropin. Doses were subsequently individualized based on IGF-1.

• At Week 52, children treated with TransCon hGH demonstrated improved annualized height velocity (AHV) similar to daily somatropin, independent of starting dose, with an LS mean AHV of 9.05 cm/year for all TransCon hGH-treated children, compared to 9.04 cm/year for those treated with daily somatropin.
• At Week 52, the mean dose for TransCon hGH was 0.22 mg/kg/week, while the mean dose for the daily somatropin cohort was 0.29 mg/kg/week.

• In the trial, TransCon hGH demonstrated a safety and tolerability profile similar to daily somatropin through follow-up of up to 143 weeks. Adverse events (AEs) were mild to moderate in severity, with no AEs leading to discontinuation of study drug. There were no occurrences of slipped capital femoral epiphysis (SCFE), consistent with the low rate of occurrence (<1%) in long-term safety data of daily somatropin use in Turner syndrome from published literature^1,2.
  
  

“These new results demonstrated safety and efficacy comparable to daily growth hormone with up to 143 weeks of follow-up, and support the potential of TransCon hGH as a differentiated therapy for short stature in the setting of growth hormone sufficiency and is being further studied in our recently initiated Phase 3 HighLiGHts basket trial to support label expansion,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer.

About Turner Syndrome
Turner syndrome is the most common congenital sex chromosomal condition in females, with an estimated prevalence of 1 out of every 2,000 to 2,500 live female births. Short stature, associated with short stature homeobox-containing gene (SHOX) haploinsufficiency, is the most common clinical feature of Turner syndrome. Clinical manifestations of Turner syndrome affect multiple organ systems and are associated with significant and potentially life-threatening complications including cardiovascular disease, ovarian dysfunction, endocrine disease, renal malformation, liver disease, sensorineural hearing loss, and varied neuropsychological manifestations.

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) the potential of TransCon hGH as a differentiated therapy for short stature in the setting of growth hormone sufficiency, (ii) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (iii) Ascendis’ ability to apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. 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, and TransCon are trademarks owned by the Ascendis Pharma group. © March 2026 Ascendis Pharma A/S.

Investor Contacts:	Media Contact:
Chad Fugere	Melinda Baker
Ascendis Pharma	Ascendis Pharma
ir@ascendispharma.com	media@ascendispharma.com
Patti Bank
ICR Healthcare
+1 (415) 513-1284
patti.bank@icrhealthcare.com

¹Darendeliler F, Karagiannis G, Wilton P. Horm Res. 2007;68 Suppl 5:41-47. doi:10.1159/000110474
²Bell J, Parker KL, Swinford RD, et al. J Clin Endocrinol Metab 2010;95(1):167–177. doi:10.1210/jc.2009-0178.

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What were the Week 52 efficacy results for TransCon hGH (ASND) in Turner syndrome?

TransCon hGH showed comparable efficacy to daily somatropin at Week 52 with an LS mean AHV of 9.05 cm/year. According to Ascendis Pharma, this matched daily somatropin (9.04 cm/year) in a randomized trial of 49 children, independent of starting dose.

How did safety for TransCon hGH compare to daily somatropin in the New InsiGHTS trial (ASND)?

Safety and tolerability were similar between treatments through follow-up up to 143 weeks. According to Ascendis Pharma, adverse events were mild to moderate, no AEs led to discontinuation, and no slipped capital femoral epiphysis (SCFE) occurred.

What dosing differences were reported between TransCon hGH and daily somatropin in the ASND trial?

The mean dose at Week 52 was lower for TransCon hGH at 0.22 mg/kg/week versus 0.29 mg/kg/week for daily somatropin. According to Ascendis Pharma, doses were individualized based on IGF-1 during the trial.

How large and long was the New InsiGHTS Turner syndrome study for ASND's TransCon hGH?

New InsiGHTS randomized and dosed 49 prepubertal children aged 1–10 years and reported follow-up up to 143 weeks. According to Ascendis Pharma, Week 52 topline results are presented from this randomized, open-label trial.

Will the ASND Phase 2 results lead to further trials or label changes for TransCon hGH in Turner syndrome?

Ascendis Pharma said the Phase 2 results support further study and informed initiation of a Phase 3 HighLiGHts basket trial for label expansion. According to Ascendis Pharma, Phase 3 work is already underway to pursue broader approval.