A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children With Achondroplasia

This proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 11 years.

Start Date26 Jul 2024

Sponsor / Collaborator

Ascendis Pharma Growth Disorders A/S

CTIS2023-508341-40-00

/ Not yet recruitingPhase 2

COACH: A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children with Achondroplasia - ASND0042

Start Date26 Jul 2024

Sponsor / Collaborator

Ascendis Pharma Growth Disorders A/S

EUCTR2021-004313-39-DE

/ Not yet recruitingPhase 3

A Multicenter, Open-Label, Extension Trial to Investigate Long Term Efficacy and Safety of Lonapegsomatropin in Adults with Growth Hormone Deficiency

Start Date31 May 2023

Sponsor / Collaborator

Ascendis Pharma Endocrinology Division A/S

100 Clinical Results associated with Lonapegsomatropin-tcgd

100 Translational Medicine associated with Lonapegsomatropin-tcgd

100 Patents (Medical) associated with Lonapegsomatropin-tcgd

Literatures (Medical) associated with Lonapegsomatropin-tcgd

01 Jan 2025·Clinical Pediatric Endocrinology

Current understanding and perspectives on growth and long-acting GH therapy in Japan

Review

Author: Ariyasu, Daisuke ; Amano, Naoko ; Mitani-Konno, Marie ; Ikegawa, Kento ; Hasegawa, Yukihiro

From the perspective of clinical pediatric endocrinology, progress in molecular biology over the past few decades has improved our understanding of growth physiology. This progress was particularly conspicuous in the early era of Sanger-based sequencing and has continued into the current era of next-generation sequencing. The first half of this review summarizes the current understanding about growth. The latter half discusses the development of long-acting GH (LAGH), an important, recent topic in pediatric endocrinology, with a focus on the Japanese market. LAGH is administered weekly over several years in some developed countries as treatment for GH deficiency (GHD). In Japan, somatrogon and somapacitan are available, and lonapegsomatropin has completed a clinical trial. As per recent meta-analyses, these three products were not inferior to conventional, daily GH therapy for short-term growth in pediatric GHD. Although LAGH is promising, some concerns remain. Well-designed clinical research is needed to expand the indications for LAGH to other conditions, such as idiopathic short stature. Finally, future research on orally administered agents may open new avenues for the treatment of short stature.

01 Nov 2024·ADVANCES IN THERAPY

Efficacy and Safety of Somapacitan Relative to Somatrogon and Lonapegsomatropin in Pediatric Growth Hormone Deficiency: Systematic Literature Review and Network Meta-analysis

Review

Author: Odgaard-Jensen, Jan ; Rossi, Annachiara ; de Fries Jensen, Lasse ; Højby, Michael ; Pietropoli, Alberto ; Antavalis, Vasileios

INTRODUCTION:

Since direct comparisons of long-acting growth hormones (LAGHs) are lacking, analyses were performed to indirectly compare the efficacy and safety of somapacitan versus somatrogon and lonapegsomatropin in children with growth hormone deficiency (GHD).

METHODS:

A systematic literature review (SLR) identified studies of once-weekly LAGHs for the treatment of pediatric GHD. Indirect comparisons (ICs) using a Bayesian hierarchical network meta-analysis and a random effects model were performed using daily growth hormone (GH) 0.034 mg/kg/day (base case) or 0.024-0.034 mg/kg/day (alternative analyses) as the common comparator to compare height outcomes to 52 weeks [annualized height velocity, height velocity standard deviation score (SDS), and height SDS]. Identified evidence did not allow IC of safety or longer-term efficacy outcomes so these were qualitatively described.

RESULTS:

The SLR identified two somapacitan trials, three somatrogon trials (one included in alternative analyses only), and one lonapegsomatropin trial comparing the LAGH with daily GH in treatment-naïve pre-pubertal children for IC. ICs revealed no differences at 52 weeks between somapacitan versus somatrogon and lonapegsomatropin, as well as daily GH, with respect to all growth outcomes considered in children with GHD. All three LAGHs had sustained efficacy and were generally well tolerated, with comparable efficacy and safety to daily GH, with the exception of observed injection site pain for somatrogon.

CONCLUSION:

No efficacy and safety differences were identified in comparisons of once weekly somapacitan versus somatrogon and lonapegsomatropin, as well as daily GH. All treatments were generally well tolerated, with the exception of observed injection site pain for somatrogon.

02 Jan 2024·Expert Review of Endocrinology & Metabolism

Somatrogon injection for the treatment of pediatric growth hormone deficiency with comparison to other LAGH products

Review

Author: Yuen, Kevin C.J. ; Velazquez, Eric P. ; Miller, Bradley S.

INTRODUCTION:

Somatrogon (NGENLA™) is a long-acting GH (LAGH) formulation that was approved in Canada in October 2021 for the treatment of pediatric growth hormone deficiency (GHD). Somatrogon has also received approval in Australia, Japan, the European Union, the USA, and the UK. Somatrogon is a glycoprotein that utilizes three copies of the C-terminal peptide of human chorionic gonadotropin to delay its clearance allowing for once-weekly administration.

AREAS COVERED:

The purpose of this article is to describe the development of somatrogon for treatment of individuals with GHD. Trials of somatrogon demonstrated positive efficacy results in adults (Phase 2) and children (Phase 2 and 3) with GHD including non-inferiority of height velocity compared to daily GH, with no concerning side effects. Growth responses, pharmacodynamics and safety data are compared to other LAGH products, lonapegsomatropin and somapacitan, in Phase 3 trials in pediatric GHD.

EXPERT OPINION:

New LAGH products, including somatrogon, have the potential to increase patient adherence as well as improve quality of life and clinical outcomes. Clinicians will need to identify the best candidates for LAGH therapy and understand how to safely monitor and adjust therapy. Long-term surveillance studies are necessary to demonstrate adherence, efficacy, cost-effectiveness, and safety of LAGH preparations.

News (Medical) associated with Lonapegsomatropin-tcgd

12 Jan 2025

·www.globenewswire.com

Ascendis Pharma Provides Business and Strategic Roadmap Update at 43rd Annual J.P. Morgan Healthcare Conference

- Ascendis positioned to drive rapid revenue growth COPENHAGEN, Denmark, Jan. 12, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today provided a business and strategic roadmap update, including planned 2025 key corporate milestones. Ascendis President and CEO Jan Mikkelsen will present this update tomorrow, January 13, during the 43rd Annual J.P. Morgan Healthcare Conference. “Ascendis is well-positioned for rapid revenue growth with the launch of our first two Endocrinology Rare Disease medicines, SKYTROFA and YORVIPATH, and, following our pre-NDA meeting, planned filings for our third, TransCon CNP for achondroplasia,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Our expanding TransCon technology platform and partnerships demonstrate that our business model built on fast, successful drug development can create substantial stakeholder value.” Selected Key Updates and Milestones TransCon™ hGH (lonapegsomatropin, marketed as SKYTROFA®) SKYTROFA full-year 2024 revenue, with a single indication in pediatric GHD, expected to be ~€202 million excluding sales deductions related to prior years (based on unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions related to prior years).U.S. SKYTROFA volume (mg) increased 84% in 2024 year-over-year resulting in an estimated 6.5% market share of the total U.S. growth hormone market for 2024 (based on third party prescription data).Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025 for FDA review of supplemental BLA for the treatment of adults with growth hormone deficiency; pending approval, U.S. commercial launch planned in the fourth quarter of 2025.During the third quarter of 2025, plan to submit an Investigational New Drug (IND) application or similar for a basket trial evaluating TransCon hGH in additional indications. TransCon PTH (palopegteriparatide, marketed as YORVIPATH®) YORVIPATH full-year 2024 unaudited preliminary revenue estimate of ~€29 million.YORVIPATH commercially available for prescription in Germany and Austria since January 2024. Outside Germany and Austria, providing product through early access routes, such as ‘named patient,’ until commercial reimbursement established. ~700 patients on treatment in our Europe Direct and International Markets at the end of 2024.YORVIPATH commercially available for prescription since late December 2024 in the U.S. As of January 9, 2025, 324 patients enrolled into the Ascendis Signature Access Program or direct with specialty pharmacy, with over half of prescriptions for patients new to YORVIPATH.Expect commercial launch in at least five additional Europe Direct countries in 2025. TransCon CNP (navepegritide) Following pre-NDA meeting with FDA, plan to submit New Drug Application (NDA) for the treatment of children with achondroplasia during the first quarter of 2025, and submit Marketing Authorisation Application to the European Medicines Agency during the third quarter of 2025.Presented new data demonstrating significant improvements in leg bowing, a common complication in achondroplasia, observed with TransCon CNP compared to worsening observed with placebo in pivotal ApproaCH Trial.Topline Week 26 results from Phase 2 COACH Trial (TransCon CNP in combination with TransCon hGH) expected in the second quarter of 2025.During the fourth quarter of 2025, plan to submit an IND or similar for the treatment of hypochondroplasia. Expanding the TransCon Platform & Pipeline New TransCon protein degrader platform designed to enable efficient clearance of hormones, cytokines, and other targets. First planned TransCon protein degrader product candidate designed to normalize excess FGF-23 hormone levels for patients with X-linked hypophosphatemia. Financial Update Unaudited preliminary estimate of total full-year 2024 product revenue of ~€226 million: SKYTROFA full-year 2024 revenue expected to be ~€202 million excluding sales deductions related to prior years (based on unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions related to prior years).YORVIPATH full-year 2024 unaudited preliminary revenue estimate of ~€29 million. Unaudited preliminary estimate of total full-year total 2024 revenue of ~€364 million Includes $100 million Novo Nordisk milestone payment as non-product revenue. December 31, 2024 pro forma cash balance of ~€655 million (based on unaudited preliminary estimate of December 31, 2024 cash balance of €560 million plus expected payment from Novo Nordisk of $100 million). Presentation at J.P. Morgan Healthcare Conference on Monday, January 13A live webcast of the event will be available via the Investors & News section of the Ascendis Pharma website at https://investors.ascendispharma.com. The presentation will begin at 11:15 a.m. Eastern Time / 8:15 a.m. Pacific Time. A webcast replay will be available for 30 days. The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations website at https://investors.ascendispharma.com. About Ascendis Pharma A/SAscendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. 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 StatementsThis 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. Examples of such statements include, but are not limited to, statements relating to (i) Ascendis’ potential for rapid revenue growth, (ii) planned regulatory submissions for TransCon CNP and TransCon hGH, (iii) the ability of Ascendis’ business model to create substantial stakeholder value, (iv) expected full-year 2024 SKYTROFA, YORVIPATH and total revenues, (v) the PDUFA date of the supplemental BLA for SKYTROFA for the treatment of adults with growth hormone deficiency and Ascendis’ plans for a U.S. commercial launch, if approved, (vi) Ascendis’ expectations regarding the commercial launch of YORVIPATH in additional Europe Direct Countries, (vii) the timing of topline results from the Phase 2 COACH Trial of TransCon CNP, (viii) Ascendis’ expectations regarding its TransCon protein degrader platform and first TransCon degrader product candidate, (ix) the expected payment from Novo Nordisk, (x) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (xi) Ascendis’ use of its TransCon technologies 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 the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors. 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’ prospectus supplement filed on September 20, 2024 and Ascendis’ current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC on February 7, 2024. 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, the company logo, TransCon, SKYTROFA®, and YORVIPATH® are trademarks owned by the Ascendis Pharma group. © January 2025 Ascendis Pharma A/S. Investor Contacts:Media Contact:Scott SmithMelinda BakerAscendis PharmaAscendis Pharmair@ascendispharma.com +1 (650) 709-8875 media@ascendispharma.comPatti Bank ICR Healthcare +1 (415) 513-1284 patti.bank@icrhealthcare.com

Phase 2NDAClinical Result

16 Dec 2024

·www.globenewswire.com

New InsiGHTS Trial of TransCon™ hGH (Lonapegsomatropin) in Turner Syndrome Achieved Primary Objective at Week 26

- Results for all three TransCon hGH starting dose cohorts, in first clinical trial of an indication outside of growth hormone deficiency, showed a safety and tolerability profile comparable to daily somatropin - Annualized height velocity was similar at Week 26 in once-weekly TransCon hGH-treated and daily somatropin-treated children COPENHAGEN, Denmark, Dec. 16, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced positive Week 26 topline results from New InsiGHTS, its Phase 2 randomized, open-label, active-controlled trial in the U.S. to investigate the safety, tolerability, and efficacy of once-weekly TransCon hGH (lonapegsomatropin; approved for pediatric growth hormone deficiency) 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 into one of four treatment groups 1:1:1:1 – one of three starting doses of TransCon hGH (0.24, 0.30, or 0.36 mg/kg/week) or an active comparator of daily somatropin with a starting dose of 0.35 mg/kg/week. Doses were individualized based on IGF-1. On the primary endpoint of annualized height velocity and secondary endpoint of change from baseline in height SDS, children treated with TransCon hGH demonstrated improved growth similar to daily somatropin at Week 26, independent of starting dose.In the trial, TransCon hGH was generally safe and well tolerated, with no discontinuations related to study drug and with comparable safety and tolerability to daily somatropin. “Week 26 topline data from the New InsiGHTS Trial showed that once-weekly TransCon hGH provided comparable safety and endocrine benefits to those seen with the established use of daily somatropin in Turner syndrome,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “These positive results lay the foundation for our planned basket trial in 2025 to support label expansion.” Ascendis expects Week 52 results for lonapegsomatropin-treated children compared to daily somatropin-treated children in 2025. About Turner SyndromeTurner 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/SAscendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. 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 StatementsThis 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. Examples of such statements include, but are not limited to, statements relating to (i) the timing of Week 52 results for lonapegsomatropin-treated patients compared to daily somatropin-treated patients, (ii) Ascendis’ plans for a basket trial in 2025 and SKYTROFA’s potential label expansion, (iii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (iv) Ascendis’ use of its TransCon technologies 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 the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors. 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’ prospectus supplement filed on September 20, 2024 and Ascendis’ current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC on February 7, 2024. 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, the company logo, and TransCon are trademarks owned by the Ascendis Pharma group. © December 2024 Ascendis Pharma A/S. Investor Contacts:Media Contact:Scott SmithMelinda BakerAscendis PharmaAscendis Pharmair@ascendispharma.com+1 (650) 709-8875 media@ascendispharma.comPatti Bank ICR Healthcare +1 (415) 513-1284 patti.bank@icrhealthcare.com

Clinical ResultDrug ApprovalPhase 2

12 Dec 2024

·www.globenewswire.com

FDA Accepts Ascendis Pharma’s Supplemental Biologics License Application for TransCon™ hGH for the Treatment of Adults with Growth Hormone Deficiency

PDUFA goal date is July 27, 2025COPENHAGEN, Denmark, Dec. 12, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the U.S. Food & Drug Administration (FDA) has accepted for review its supplemental Biologics License Application (sBLA) in adult growth hormone deficiency (GHD) for TransCon hGH (lonapegsomatropin-tcgd; marketed as SKYTROFA® for pediatric GHD). The FDA set a Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025. “This marks another step towards achieving our objective to expand SKYTROFA’s label beyond pediatric GHD and expand its reach to address new groups of patients,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Adult GHD is an undertreated condition associated with significant comorbidities and higher annual healthcare costs compared to the 5-10% of patients who receive treatment, indicative of the high unmet need.” The sBLA submission is based on results from foresiGHt, a Phase 3 randomized, parallel-arm, placebo-controlled (double-blind) and active-controlled (open-label) trial that compared the efficacy and safety of weekly TransCon hGH with weekly placebo and daily human growth hormone (hGH) in adults with GHD. The trial evaluated 259 adults with GHD aged 23 to 80 years old, randomized 1:1:1, titrated to receive a target fixed dose of TransCon hGH, placebo, or daily hGH based on age and oral estrogen intake with approximately equivalent hGH mg/week for TransCon hGH and daily hGH. TransCon hGH demonstrated superiority on its primary efficacy and key secondary efficacy endpoints at Week 38, with TransCon hGH-treated participants showing a statistically significant reduction from baseline in trunk fat and increase in total body lean mass at Week 38 compared to placebo. In the trial, TransCon hGH was generally safe and well tolerated, with no discontinuations related to study drug and with comparable safety and tolerability to daily hGH treatment. About Adult Growth Hormone DeficiencyGrowth hormone plays an essential role in the health of children and adults, promoting normal growth in children and maintenance of normal body composition and cardiometabolic health throughout adulthood. In adults, growth hormone boosts protein production, promotes fat utilization, enhances muscle mass, and helps regulate blood sugar levels. Adult GHD is a condition in which an individual’s body does not produce enough growth hormone. Symptoms and morbidity can include central obesity, metabolic syndrome, decreased bone density, alterations in lipid profile and markers of cardiovascular risk, fatigue, general weakness, lack of muscle tone, and psychological symptoms such as cognitive impairment, social isolation, lack of motivation, and depression.1 About Ascendis Pharma A/SAscendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. 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 StatementsThis 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. Examples of such statements include, but are not limited to, statements relating to (i) the PDUFA goal date for SKYTROFA, (ii) Ascendis’ objective to expand SKYTROFA’s label and reach to address new groups of patients, (iii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (iv) Ascendis’ use of its TransCon technologies 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 the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors. 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’ prospectus supplement filed on September 20, 2024 and Ascendis’ current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC on February 7, 2024. 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, the company logo, TransCon, and SKYTROFA®, are trademarks owned by the Ascendis Pharma group. © December 2024 Ascendis Pharma A/S. Investor Contacts:Media Contact:Scott SmithMelinda BakerAscendis PharmaAscendis Pharmair@ascendispharma.com+1 (650) 709-8875 media@ascendispharma.comPatti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@icrhealthcare.com 1.Hoffman AR, Mathison T, Andrews D, Murray K, Kelepouris N, Fleseriu M. Adult Growth Hormone Deficiency: Diagnostic and Treatment Journeys From the Patients' Perspective. J Endocr Soc. 2022;6(7):bvac077. Published 2022 May 12. doi:10.1210/jendso/bvac077

Phase 3Clinical ResultNDA

100 Deals associated with Lonapegsomatropin-tcgd

External Link

KEGG	Wiki	ATC	Drug Bank
-	Lonapegsomatropin-tcgd	-	DB16220

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Growth hormone deficiency	US	Ascendis Pharma Endocrinology Division A/S	25 Aug 2021

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Endocrine System Diseases	Phase 3	CN	VISEN Pharmaceuticals (Shanghai) Co., Ltd.	30 Dec 2019
Endocrine System Diseases	Phase 3	CN	Ascendis Pharma A/S	30 Dec 2019
Pituitary Diseases	Phase 3	CN	Ascendis Pharma A/S	30 Dec 2019
Pituitary Diseases	Phase 3	CN	VISEN Pharmaceuticals (Shanghai) Co., Ltd.	30 Dec 2019
Achondroplasia	Phase 2	DK	Ascendis Pharma Growth Disorders A/S	26 Jul 2024
Achondroplasia	Phase 2	IE	Ascendis Pharma Growth Disorders A/S	26 Jul 2024
Achondroplasia	Phase 2	GB	Ascendis Pharma Growth Disorders A/S	26 Jul 2024
Turner Syndrome	Phase 2	US	Ascendis Pharma A/S	-

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04615273 (foresiGHt, CTgov)	Phase 3	Growth hormone deficiency	264	Lonapegsomatropin (Lonapegsomatropin)	idmluruzal(edheglkome) = otrtqmecwq wpiathhulc (xgijcblqec, zefgbrhnli - dnmanluncj) View more	-	15 Jan 2025
				Placebo (Placebo)	idmluruzal(edheglkome) = kldzmujarh wpiathhulc (xgijcblqec, kmhymbqdgf - rbqesssded) View more
NCT03344458 (enliGHten, CTgov)	Phase 3	Growth hormone deficiency	298	Lonapegsomatropin	sksiqtcdlr(wvjsriwvyr) = nryezobyvk zugbtqondb (gcbiwhwjrt, bvgotbwpbz - peoxvpwqds) View more	-	08 May 2024
NCT04615273 (foresiGHt, Biospace) Manual	Phase 3	Growth hormone deficiency	259	TransCon hGH	vwpzeocvks(vyzjkhvkpd) = mojyuvoqav xjonafqsdj (klwaggxflc ) View more	Positive	19 Dec 2023
				Placebo	vwpzeocvks(vyzjkhvkpd) = ugfkgcuuul xjonafqsdj (klwaggxflc ) View more
NCT03344458 (enliGHten, GlobeNewswire) Manual	Not Applicable	Growth hormone deficiency	81	TransCon hGH	bqocfdlyad(gvghiwitaq) = The most commonly reported adverse events over the course of the trial were categorized as infections, injury, and respiratory/thoracic/medical disorders. The majority of adverse events were mild in severity and unrelated to treatment. No adverse events led to discontinuation of the study treatment. qnhrglbvxu (vnpllbgyjo )	Positive	23 Sep 2023
				TransCon hGH (at the beginning of the open-label extension trial)
ESPE2023	Phase 3	Growth hormone deficiency	298	Lonapegsomatropin	slqeptyxay(ouwfktmsmj) = rjblwcexfj wkevvmayij (jjxuenywth, 6)	-	21 Sep 2023
NCT04326374 (PRNewswire) Manual	Phase 3	Growth hormone deficiency	150	human growth hormone (Longpei growth promoting hormone group)	pucmxsktgd(ahfrpphgyx) = vbcmdhwljw zkymwumesn (kwkgtqigge )	Positive	17 Nov 2022
				human growth hormone (growth hormone daily preparation group)	pucmxsktgd(ahfrpphgyx) = ezwelwjwfj zkymwumesn (kwkgtqigge )
enliGHten Trial (ENDO2022)	Phase 3	Growth hormone deficiency	298	Lonapegsomatropin via vial/syringe	wibefiuxac(whpnbhukep) = With continued lonapegsomatropin treatment, the AE profile remained consistent with what was observed in the parent trials, with no new safety signals sucdvawber (pdavjdpoew )	Positive	01 Nov 2022
NCT04326374 (PRNewswire) Manual	Phase 3	Growth hormone deficiency	150	lonapegsomatropin	epbdwkhuxs(jrejjlskzr): P-Value = 0.0010	Superior	23 May 2022
				daily growth hormone
NCT03305016 (CTgov)	Phase 3	Growth hormone deficiency	146	Lonapegsomatropin	djctprkxco(ofwxhvzkik) = zibagavjwa ughlcqftlh (haapjnujnu, dobvbnxsvu - budlsbuczc) View more	-	04 Jan 2022
heiGHt Trial (ENDO2020)	Phase 3	Growth hormone deficiency Maintenance	159	TransCon hGH	odywrbowmn(jcilhvwzwh) = zlbmbtzmfx zooqbqclwx (rzsqnnitzf ) View more	Positive	08 May 2020
				Genotropin	odywrbowmn(jcilhvwzwh) = rrvafiztqt zooqbqclwx (rzsqnnitzf )

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