Ascendis Pharma A/S Price Target | Analysis, Price & Growth Outlook

Ascendis Pharma A/S

NasdaqGS:ASND

$ 207,12

$0,00 (0,00%)

207,12 $

$0,00 (0,00%)

End-of-day quote: 12/09/2025

Ascendis Pharma A/S Stock Value

Analysts currently rate NasdaqGS:ASND as Buy.

Buy

Ascendis Pharma A/S Company Info

EPS Growth 5Y

-5,15%

Market Cap

$12,66 B

Long-Term Debt

$0,29 B

Annual earnings

02/11/2026

Dividend

$0,00

Dividend Yield

0,00%

Founded

2006

Industry

Health Care

Country

Denmark

Website

Ascendis Pharma A/S

ISIN Number

US04351P1012

Website

http://ascendispharma.com

Analyst Price Target

$251,94

21.64%

Last Update: 12/10/2025

Analysts: 15

Highest Price Target $326,97

Average Price Target $251,94

Lowest Price Target $221,33

In the last five quarters, Ascendis Pharma A/S’s Price Target has risen from $190,10 to $249,87 - a 31,44% increase. Twelve analysts predict that Ascendis Pharma A/S’s share price will increase in the coming year, reaching $251,94. This would represent an increase of 21,64%.

Top growth stocks in the health care sector (5Y.)

What does Ascendis Pharma A/S do?

Ascendis Pharma A/S(Ascendis) is a global biopharmaceutical company focused on applying its TransCon technology platform to make a meaningful difference for patients. The company develops TransCon-based therapies that demonstrate best-in-class potential to address unmet medical needs. The company's portfolio of approved Endocrinology Rare Disease products and product candidates addresses hypoparathyroidism, as well as growth disorders, such as growth hormone deficiency and achondroplasia. To cr...

Ascendis Pharma A/S(Ascendis) is a global biopharmaceutical company focused on applying its TransCon technology platform to make a meaningful difference for patients.

The company develops TransCon-based therapies that demonstrate best-in-class potential to address unmet medical needs. The company's portfolio of approved Endocrinology Rare Disease products and product candidates addresses hypoparathyroidism, as well as growth disorders, such as growth hormone deficiency and achondroplasia. To create additional value, the company has established partnerships to develop and bring to market TransCon-based products in large therapeutic areas, including Metabolic, Cardiovascular, and Ophthalmology.

The company's products and product candidates combine its TransCon technologies with clinically validated parent drugs and pathways, with the intention of optimizing efficacy, safety, tolerability and convenience.

The company applies these technologies using its algorithm with the goal of creating product candidates with the potential to be best-in-class. Using this approach, the company plans to expand its pipeline with Endocrinology Rare Disease product opportunities in large addressable markets.

The company currently has two marketed products and a diversified portfolio of four product candidates in clinical development in the areas of Endocrinology Rare Disease and Oncology, and it is working to apply its TransCon technology platform in additional therapeutic areas, such as the glucagon-like peptide 1 (GLP-1) class, where the company has designed a potentially best-in-class, once-monthly program.

SKYTROFA - The company's first marketed product is SKYTROFA (lonapegsomatropin-tcgd), developed as TransCon Growth Hormone (TransCon hGH), which has received regulatory approval in the United States for the treatment of pediatric patients one year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous growth hormone, also known as growth hormone deficiency (GHD). SKYTROFA has been commercially available for prescription in the United States since October 2021. In addition, SKYTROFA (lonapegsomatropin) was granted marketing authorization in the European Union (EU), Norway, Iceland, Liechtenstein, and Great Britain (covering England, Wales, Scotland) as a once-weekly subcutaneous injection for the treatment of children and adolescents aged 3 to 18 years with growth failure due to insufficient secretion of endogenous growth hormone. In the EU, SKYTROFA has been commercially available for prescription in Germany since September 2023.

YORVIPATH - The company's second marketed product is YORVIPATH (palopegteriparatide), developed as TransCon PTH. In the EU, Norway, Iceland, Liechtenstein, and Great Britain (covering England, Wales, and Scotland), YORVIPATH has been granted marketing authorization as a once-daily subcutaneous injection for the treatment of adults with chronic hypoparathyroidism. In the EU, YORVIPATH has been commercially available for prescription in Germany and Austria since January 2024 and has also been available to patients in other countries under named patient programs. In August 2024, YORVIPATH received regulatory approval in the U.S. for the treatment of hypoparathyroidism in adults. YORVIPATH has been commercially available for prescription in the United States since December 2024. As of February 7, 2025, 908 new prescriptions by 539 unique prescribing healthcare providers in the U.S.

Endocrinology Rare Disease Pipeline - Two product candidates in the company's Endocrinology Rare Disease portfolio are currently in development spanning multiple indications and geographies. These product candidates are TransCon hGH (lonapegsomatropin) for adult GHD and children with Turner syndrome and TransCon CNP (navepegritide) for infants, children, and adolescents with achondroplasia. The company is also investigating TransCon CNP in combination with TransCon hGH in children with achondroplasia. In addition, it plans to investigate TransCon hGH in other established daily growth hormone indications. Through its strategic collaboration, Teijin Limited is developing and, if approved, plans to commercialize TransCon hGH, TransCon PTH, and TransCon CNP for endocrinology rare disease in Japan. In addition, through its strategic investment, VISEN Pharmaceuticals (VISEN) is developing and, if approved, plans to commercialize TransCon hGH, TransCon PTH, and TransCon CNP for endocrinology rare diseases in Greater China.

Oncology Pipeline - In Oncology, the company is leveraging its TransCon technologies with the goal of enhancing the anti-tumor effects of clinically-validated parent drugs and pathways and to provide sustained modulation of tumor microenvironments and activate cytotoxic immune cells. The company initiated clinical development of two programs: TransCon TLR7/8 Agonist, an investigational, long-acting prodrug of resiquimod, a small molecule agonist of Toll-like receptors (TLR) 7 and 8, for intratumoral delivery, and TransCon IL-2 b/g (onvapegleukin alfa) for systemic delivery, which is designed for prolonged exposure to an IL-2 variant that selectively activates IL-2 b/g with minimal binding to IL-2R?. During the fourth quarter of 2024, the company closed enrollment in its BelieveIT-201 clinical trial and to dose expansion cohorts involving TransCon TLR7/8 Agonist in the transcendIT-101 and IL-Believe trials to prioritize its efforts on TransCon IL-2 b/g.

TransCon Product Candidates Pipeline

Other than the rights the company has granted to Eyconis, Novo Nordisk, Teijin Limited, and VISEN, as noted in this annual report, the company holds worldwide rights to its TransCon technologies. Additionally, other than its royalty financing arrangements with Royalty Pharma, as noted in this annual report, the company owes no third-party royalty or milestone payment obligations with respect to its TransCon technologies, TransCon hGH, TransCon PTH, or any of its other product candidates.

The company maintains an intellectual property portfolio comprising over 425 issued patents and over 525 patent applications as of December 31, 2024, which includes patents and patent applications applicable to its products and product candidates, with claims directed to composition of matter, process, formulation, and/or methods-of-use for its products and product candidates, including a product-specific device and core TransCon technologies. While the company's TransCon prodrugs may incorporate already approved parent drugs, TransCon hGH, TransCon PTH, and each of its other product candidates are new molecular entities and therefore eligible to be granted new intellectual property rights, including new composition of matter patents.

Global Commercialization Strategy

The company is establishing a global presence to commercialize TransCon products and product candidates, where approved, to address patients' unmet medical needs.

In the U.S., the company has established an integrated organization to commercialize its approved Endocrinology Rare Disease products, SKYTROFA and YORVIPATH. The company's U.S. organization includes sales, market access, patient support, and medical affairs teams. The sales team engages with healthcare providers and presents products, usage, and safety guidelines in accordance with the label. The company's market access team engages with health authorities, insurance companies, and payers to help patients in need of the company's products gain access to them. The company's patient support team facilitates reimbursement support, out-of-pocket assistance, provides educational resources, and product training. The company's medical affairs team provides scientific exchange to the physician and medical community. The company has also established a network of specialty pharmacies to support product distribution.

In Europe, the company is expanding its presence by building integrated organizations in select countries, which it calls Europe Direct, beginning with Germany, where the company has launched its Endocrinology Rare Disease products, SKYTROFA and YORVIPATH. The company is establishing commercial infrastructure in other Europe Direct country clusters, including DACH (Germany, Austria, Switzerland), France & BeNeLux (Belgium, the Netherlands, and Luxembourg), Iberia (Portugal and Spain), Italy, Nordics (Denmark, Norway, Sweden, Iceland, Finland), and the United Kingdom & Ireland.

Beyond the U.S. and Europe Direct, the company is expanding its global reach for its Endocrinology Rare Disease programs through exclusive distribution agreements with geographic market leaders, which it refers to as International Markets. As of December 31, 2024, the company has established eight such regional agreements:

Acino Pharma Proprietary Limited (South Africa).

Adium Pharma S.A. (Argentina, Brazil, Colombia and Mexico).

Er-Kim ?lac Sanayi ve Ticaret A.S (Central & Eastern Europe, Turkey, and certain countries in Eurasia).

Neopharm (Israel) 1996 Ltd (Israel, Armenia, Azerbaijan, Georgia, Kazakhstan, Kyrgyzstan, Tajikistan, Turkmenistan and Uzbekistan).

Pendopharm, a division of Pharmascience Inc. (Canada).

Specialised Therapeutics Asia Pte Ltd. (Australia, New Zealand, Singapore, Malaysia, Brunei, Thailand, and Vietnam).

SRS Pharmaceuticals Pvt. Ltd. (India and Philippines).

Vector Pharma FZCO (Saudi Arabia, United Arab Emirates, Kuwait, Oman, Qatar, and Bahrain).

Finally, the company is making its products commercially available in select markets through exclusive license agreements with partners with local development and commercialization expertise and infrastructure. In China, VISEN has exclusive license rights to develop and commercialize TransCon hGH, TransCon PTH, and TransCon CNP. In Japan, Teijin Limited has exclusive license rights to develop and commercialize TransCon hGH, TransCon PTH, and TransCon CNP.

TransCon Technologies

The company's TransCon technologies are designed to combine the benefits of conventional prodrug and sustained release technologies to solve the fundamental limitations seen in other approaches to extending duration of a drug's action in the body, with the goal of developing highly differentiated product candidates based on efficacy, safety, tolerability, and convenience.

TransCon molecules can have up to three components: a parent drug, an inert carrier that protects it, and a linker that temporarily binds the two. When bound, the carrier inactivates and shields the parent drug from clearance. When injected into the body, physiologic pH and temperature conditions initiate the release of the active, unmodified parent drug in a predictable release manner.

TransCon Technology Components

TransCon Carriers

The company's TransCon technologies incorporate three carrier platforms that can be used to provide sustained localized or systemic drug exposure. These biocompatible carrier platforms include its TransCon systemic carriers and TransCon localized carriers (self-eliminating hydrogels). The company's carriers inactivate and protect the drug through a shielding effect, which may prevent rapid excretion and degradation of the parent drug, and enable benefits that include improved injection site tolerability, reduced systemic adverse effects, and low immunogenicity.

Systemic - The company's TransCon systemic carriers are used to provide systemic drug exposure and are based on soluble compounds such as methoxypolyethylene glycol (mPEG) or other natural or synthetic polymers, as well as its albumin avidity approach, where 2 or more albumin binding moieties are incorporated into the drug molecule to facilitate sustained exposure. Prodrugs created using its systemic carriers are readily absorbed into the bloodstream after administration. TransCon hGH, TransCon PTH, and TransCon CNP utilize mPEG as a carrier molecule. mPEG is widely used to improve the pharmacokinetic or pharmacodynamic properties of marketed therapeutics. Below is an illustration of the company's systemic carrier:

Localized - The company's TransCon localized carriers include TransCon hydrogels based on PEG, hyaluronic acid, or other biopolymers. TransCon hydrogel is designed to self-eliminate to soluble, biocompatible molecules after the drug payload has been released. When applied for localized delivery, the TransCon hydrogel enables the release of a parent drug at high local concentrations within the target area while minimizing systemic exposure.

TransCon Linkers

The company's reversible TransCon linkers are designed to enable the transient conjugation of a broad range of therapeutics, including proteins, peptides and small molecules, to the company's TransCon carriers. The company has a large library of TransCon linkers that may be applicable to various types of parent drugs, and that can be tailored to potentially achieve half-life extension enabling daily, weekly, monthly, and half-yearly dosing and to customize the potential pharmacokinetic profile for each individual product candidate with the goal of optimizing the potential therapeutic effect. TransCon linkers are self-cleaving through a process called intra-molecular assisted cleavage, which causes the linker to release the unmodified parent drug. The company can tailor the release properties of the linker to a given therapeutic indication and parent drug by modifying the linker structures. The rate of metabolic conversion of prodrugs in these types of processes may differ between patients, and even within different tissues in the same patient. As a result, conventional prodrugs do not always offer predictable release of the parent drug. The company's TransCon linkers are designed to predictably release an unmodified active parent drug at predetermined rates governed by physiological pH and temperature conditions, which are tightly regulated in the body.

Parent Drugs

The company's TransCon technologies are applicable across a broad range of therapeutic classes and are currently used to create long-acting product candidates with best-in-class potential based on proteins, peptides, and small molecules. By primarily focusing on biological targets that have been clinically validated, the company can leverage available knowledge regarding a target's activity. Based on this selective approach, the company understands what drug levels must be maintained in the body for optimal efficacy and safety, and can design the release half-life and dosing frequency of its TransCon prodrugs to maintain these levels to achieve the desired pharmacological effect. The company moves a product candidate into development after it demonstrates the desired profile in non-clinical models. Furthermore, based on the established translational relationships between preclinical animal models and clinical efficacy, this strategy is designed to reduce risk and increase productivity.

TransCon Products - Endocrinology Rare Disease

TransCon Growth Hormone (hGH)

Market Opportunity for Recombinant Human Growth Hormone

Growth hormone deficiency is a serious orphan disease that affects both children and adults. Children with GHD are characterized by short stature, metabolic and cardiovascular abnormalities, cognitive deficiencies, and poor quality of life. GHD in adults is associated with increased adiposity, or fat mass, as well as psychiatric-cognitive, cardiovascular, muscular, metabolic and skeletal abnormalities. In childhood and adolescence, growth hormone plays an essential role in normal longitudinal growth, muscle and bone strength, and distribution of body fat. In adults, growth hormone contributes to body composition, cardiovascular function, and bone health. The current standard of care for GHD has been daily subcutaneous injections of somatropin, a recombinant human growth hormone (hGH). These daily hGH therapies have been shown to be safe and well-tolerated.

In both therapy-compliant children and adults with GHD, daily subcutaneous injections of hGH have resulted in improved body composition parameters, bone density, cardiovascular outcomes, and quality of life. Growth hormone-deficient children who are fully adherent to their daily hGH treatment regimen may achieve a height in adulthood that is comparable to that of their family members and national norms.

Since the introduction of hGH in 1981, a number of the world's largest pharmaceutical companies have developed and marketed daily-administered hGH products. All currently marketed daily hGH products in the United States - Norditropin (Novo Nordisk A/S), Humatrope (Eli Lilly and Company), Genotropin (Pfizer Inc.), Zomacton (Ferring Pharmaceuticals, Inc.) and Omnitrope (Sandoz GmbH) - contain unmodified somatropin and are administered by subcutaneous injections. The global market for daily hGH products is largely composed of products from Novo Nordisk, Pfizer, Eli Lilly, Sandoz, and Merck KGaA, which together account for most of the global market share.

TransCon hGH

TransCon hGH is a prodrug composed of somatropin that is transiently bound to a carrier by a proprietary linker. TransCon hGH is administered once weekly and is designed to maintain the same mode of action as daily therapies by providing sustained release of active, unmodified somatropin, the same recombinant growth hormone molecule used in the daily hGH therapies that have historically been the standard of care.

TransCon Growth Hormone (hGH) for Pediatric GHD

TransCon hGH, marketed under the brand name SKYTROFA (lonapegsomatropin-tcgd), received regulatory approval in the U.S. for the treatment of pediatric patients one year and older who weigh at least 11.5 kg and have growth failure due to inadequate secretion of endogenous growth hormone, also known as GHD. SKYTROFA has been commercially available for prescription in the United States since October 2021. In the EU, Norway, Iceland, Liechtenstein, and Great Britain (covering England, Wales, Scotland), the company received marketing authorization for TransCon hGH - known by its brand name SKYTROFA (lonapegsomatropin) - as a once-weekly subcutaneous injection for the treatment of children and adolescents aged 3 to 18 years with growth failure due to insufficient secretion of endogenous growth hormone. SKYTROFA has been commercially available for prescription in Germany since September 2023.

In September 2023, the company announced top-line results from the completed enliGHten Trial, an open-label extension trial evaluating the long-term safety and efficacy of TransCon hGH as a once-weekly treatment for children and adolescents with GHD. The enliGHten Trial enrolled 298 participants (mean age 10.3 years) from the Phase 3 heiGHt Trial of treatment-naïve pediatric GHD patients and the Phase 3 fliGHt Trial of pediatric GHD patients switching from daily somatropin treatment. Patients in these trials received a total of up to 6 years of treatment with TransCon hGH. At the time of the enliGHten Trial closure, 81 participants were designated as treatment completers, based on their physician's determination that treatment for pediatric GHD was no longer required. Of these treatment completers, 59% met or exceeded their average parental height standard deviation score (SDS), with mean TransCon hGH treatment duration of 3.2 years.

Clinical Trial of TransCon hGH in Japanese Pediatric GHD

In the Phase 3 riGHt Trial, the company is evaluating TransCon hGH (N=15) compared to somatropin (N=16) as a treatment in Japanese children with GHD. The trial achieved its primary objective with Week 52 top-line results consistent with its pivotal heiGHt Trial and VISEN's Phase 3 trial. In the riGHt Trial, TransCon hGH was generally well tolerated with a safety profile that was similar to that of somatropin's. Trial subjects continue in the extension period.

Proprietary Auto-Injector

SKYTROFA includes the SKYTROFA Auto-Injector and cartridges. The auto-injector provides for room temperature storage, includes an empty-all design, and is expected to last for at least four years. The device enables a single, low-volume injection of less than 0.6 mL for the majority of patients with a thin, 31-gauge needle that is only 4 millimeters in length, which is comparable to needles used to administer daily hGH. The company is also working on strategies that will enable the auto-injector to integrate with the digital healthcare system, including Bluetooth connectivity features to allow for easy tracking of dosing adherence over time.

TransCon Product Candidates - Endocrinology Rare Diseases

TransCon Growth Hormone (hGH) for Other Indications

Clinical Development in Adult GHD

In September 2024, the company announced the submission of a supplemental Biologics License Application (sBLA) to the FDA for TransCon hGH to expand its currently approved label to include eligible adults for the treatment of GHD. The submission was 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 hGH in adults with GHD. In December, the company announced that the FDA accepted the sBLA for review and set a Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025.

In December 2023, the company announced positive top-line results from foresiGHt. The trial aims to evaluate the metabolic benefits of TransCon hGH in adults, with the primary objective being to evaluate the change in trunk fat percentage.

The foresiGHt Trial evaluated 259 adults with GHD aged 23 to 80 years old, randomized 1:1:1, and 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.

Other Development Plans

During the third quarter of 2025, the company plans to submit an Investigational New Drug (IND) application or similar for a basket trial evaluating other indications, including small for gestational age without catch-up growth (SGA), idiopathic short stature (ISS), and SHOX deficiency (including Turner syndrome).

TransCon PTH

Market Opportunity in Hypoparathyroidism

The company is also aware of several academic groups and companies working on making longer-acting agonists of the PTH receptor. In addition, other companies and groups are developing therapies for hypoparathyroidism at the clinical stage, including Calcilytix (a BridgeBio company), Entera Bio, Extend Biosciences, Massachusetts General Hospital, AstraZeneca, MBX Biosciences, and Septerna.

Forteo (teriparatide, PTH [1-34]), approved since 2002 for the treatment of osteoporosis, has sometimes been used for the treatment of hypoparathyroidism using multiple daily injections, despite not being approved for this indication. Clinical research conducted by the U.S. National Institutes of Health in subjects receiving continuous exposure to PTH (1-34), administered by an infusion pump demonstrated simultaneous normalization of serum calcium and urinary calcium, as well as normalization of bone turnover.

Solution: TransCon PTH

TransCon PTH (palopegteriparatide) is a prodrug of PTH (1-34) that is administered once-daily to achieve and maintain a steady concentration of PTH in the bloodstream within the physiological range. TransCon PTH is designed to provide PTH in the physiological range for 24 hours per day, thereby more fully addressing aspects of the disease, including maintaining normal serum calcium and phosphate levels and normalizing urinary calcium.

TransCon PTH for the Treatment of Hypoparathyroidism

In August 2024, the FDA approved YORVIPATH (palopegteriparatide; developed as TransCon PTH) for the treatment of hypoparathyroidism in adults. In September 2024, the FDA granted Orphan Drug exclusivity to YORVIPATH, providing seven years of market exclusivity for YORVIPATH in the United States for the treatment of hypoparathyroidism in adults. YORVIPATH has been commercially available for prescription since late December 2024 in the United States.

In November 2023, TransCon PTH received regulatory approval in the EU and European Economic Area and is marketed as YORVIPATH (palopegteriparatide), a parathyroid hormone replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. In addition, YORVIPATH was granted Orphan status in the EU in November 2023. In January 2024, the company announced commercial availability of YORVIPATH in Germany and Austria, and it began shipping to customers in February 2024.

In April 2024, TransCon PTH received regulatory approval in Great Britain as a PTH replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism. In addition, in April 2024, the company announced that the United Kingdom's Medicines & Healthcare products Regulatory Agency granted YORVIPATH Orphan Drug status.

In December 2022, the FDA allowed it to initiate a U.S. expanded access program (EAP) for TransCon PTH for eligible adult patients with hypoparathyroidism with prior PTH treatment experience. The EAP closed to new patients on November 29, 2024, in preparation for the U.S. commercial availability of YORVIPATH. Beginning in December 2024, the company started enrolling EAP patients into the Ascendis Signature Access Program Ascendis' patient support program. Patients enrolled in the EAP are now working with their healthcare providers (HCPs) to transition to the commercially available YORVIPATH.

In July 2021, the Ministry of Health, Labour and Welfare in Japan granted Orphan Drug Designation (ODD) to TransCon PTH for the treatment of hypoparathyroidism.

In October 2020, the company were granted Orphan Designation (OD) by the European Commission (EC) for TransCon PTH for the treatment of hypoparathyroidism.

Clinical Development of TransCon PTH for the Treatment of Hypoparathyroidism in Adults

TransCon PTH is being evaluated for the treatment of hypoparathyroidism in adults in the Phase 3 PaTHway Trial, Phase 3 PaTHway Japan Trial, and the Phase 2 PaTH Forward Trial. Last patient, last visit (LPLV) in the Phase 3 PaTHway Trial occurred in January 2024. LPLV in the Phase 2 PaTH Forward Trial is expected in March of 2025.

In September 2024, the company announced results from the ongoing Phase 2 PaTH Forward Trial of adults with hypoparathyroidism showing that long-term treatment with TransCon PTH (palopegteriparatide; marketed as YORVIPATH) through Week 162 drove bone remodeling into the normal range. Deficiency of parathyroid hormone is associated with low rates of bone remodeling, accumulation of overly mature bone, and higher-than-average bone mineral density that may correspond with poorer overall bone quality compared to that seen in the general population. In contrast, these results suggest that long-term palopegteriparatide treatment promotes attainment of skeletal health parameters in line with those expected with states of parathyroid sufficiency. As of December 31, 2024, 10 patients successfully completed the trial, while 46 out of the 59 patients originally enrolled in the trial continued in the open label (OLE) portion and had exceeded four and a half years of follow-up. Three patients withdrew from the trial for reasons unrelated to safety or efficacy of the study drug.

On January 8, 2023, the company announced top-line data from PaTHway Japan, a single-arm Phase 3 trial to evaluate the safety, tolerability, and efficacy of TransCon PTH in adults with hypoparathyroidism. The study achieved its primary objective, with top-line results consistent with the company's trials in North America and the EU. Twelve out of thirteen patients met the primary multi-component endpoint, which was defined as serum calcium levels in the normal range (8.3-10.6 mg/dL) and independence from conventional therapy (no active vitamin D and ?600 mg/day of calcium).

In March 2022, the company announced that top-line data from the randomized, double-blind, placebo-controlled portion of the Phase 3 PaTHway Trial of TransCon PTH in adults with hypoparathyroidism demonstrated statistically significant higher proportion of participants treated with TransCon PTH achieved the primary multi-component endpoint compared to placebo.

TransCon CNP

In November 2021, BioMarin Pharmaceutical Inc.'s daily VOXZOGO (vosoritide) was approved by the FDA to increase linear growth in pediatric patients with achondroplasia with open epiphyses. Additionally, BioMarin is developing a long-acting CNP to build on VOXZOGO. Other companies that are developing therapies for achondroplasia include QED Therapeutics (a BridgeBio company), Sanofi, Ribomic, Tyra Biosciences, GeneScience and ProLynx.

Solution: TransCon CNP

TransCon CNP (navepegritide) is an investigational prodrug of CNP administered once weekly and designed to provide sustained release of active CNP supporting continuous exposure for the treatment of achondroplasia. TransCon CNP is designed to provide effective shielding of CNP from neutral endopeptidase degradation in subcutaneous tissue and the blood compartment, minimize binding of CNP to the NPR-C receptor to decrease clearance, reduce binding of CNP to the NPR-B receptor in the cardiovascular system to avoid hypotension, and release unmodified CNP, which is small enough in size to allow effective penetration into growth plates. Shorter-acting CNP and CNP analogs in development have resulted in high maximum serum concentration (Cmax) levels that may cause adverse hypotensive events.

Clinical Development of TransCon CNP for Achondroplasia

The company's ongoing pivotal ApproaCH Trial, its long-term extension trial AttaCH, and COACH, are evaluating the safety and efficacy of TransCon CNP in children with achondroplasia. The reACHin Trial is evaluating the safety, tolerability, and efficacy of TransCon CNP in infants with achondroplasia (aged 0 to < 2 years at the time of randomization). The TeACH Trial is evaluating the safety, tolerability, and efficacy of TransCon CNP in adolescents with achondroplasia (aged 12 to 18).

In January 2025, the company announced data demonstrating improvements in leg bowing, a common complication in achondroplasia, observed with TransCon CNP compared to worsening observed with placebo in the pivotal ApproaCH Trial.

In September 2024, the company announced top-line data from ApproaCH, a pivotal, multicenter, randomized, double-blind, placebo-controlled trial of once-weekly TransCon CNP versus placebo in 84 children (aged 2 to 11 years) with achondroplasia. Participants were randomized 2:1 to receive TransCon CNP 100 µg/kg/week or placebo for 52 weeks in the double-blind period, after which all participants could choose to receive TransCon CNP at the 100 µg/kg/week dose in an ongoing open-label extension. In the trial, children treated with once-weekly TransCon CNP demonstrated annualized growth velocity (AGV) superior to those treated with placebo. TransCon CNP also demonstrated statistically significant improvements in other growth parameters, including height Z-score and change from baseline AGV.

The company analyzed available data for patients who only received TransCon CNP at the 100 µg/kg/week dose in either the blinded or OLE period and were treated for one year (n=19), compared to those administered placebo for one year (n=15). Results showed that these TransCon CNP-treated patients (data available for 9-16 patients) showed improvements (nominal p-value <0.05) in health-related quality of life and disease impacts compared to those receiving placebo (data available for 5-13 patients).

During the third quarter of 2023, the company filed an IND amendment with the FDA to initiate reACHin, a Phase 2, multicenter, double-blind, randomized, placebo-controlled trial, designed to evaluate the safety, tolerability, and efficacy of 100 ?g/kg of TransCon CNP once-weekly for 52 weeks in infants with achondroplasia, aged 0 to < 2 years at the time of randomization.

In November 2022, the company announced top-line results from ACcomplisH, a Phase 2 randomized, double-blind, placebo-controlled, dose-escalation trial evaluating the safety and efficacy of once-weekly TransCon CNP compared to placebo in children with achondroplasia aged 2 to 10 years old.

As of December 31, 2024, 55 patients completed the OLE portion of the ACcomplisH Trial; 2 patients prematurely withdrew during the OLE. From ACcomplisH OLE, 53 patients transitioned into the Phase 2 AttaCH Trial, a multicenter, long-term, open-label extension trial to continue treatment with TransCon CNP 100 µg/kg/week and 2 patients transitioned into COACH, a TransCon CNP/TransCon hGH combination trial. As of December 31, 2024, 43 patients continue in AttaCH, on long-term OLE treatment; 3 patients prematurely withdrew from treatment and 7 patients transitioned into COACH. With the last subject completing the OLE portion of the ACcomplisH Trial, the ACcomplisH Trial has completed.

In 2019, the company initiated the ACHieve Study, a five-year, multi-center natural history study designed to gain insight into the experiences of pediatric patients with achondroplasia. ACHieve was designed to evaluate growth velocity, body proportionality, and comorbidities over time in children with achondroplasia up to eight years old. The study ended in the first quarter of 2024. The company plan to make the results available in 2025.

TransCon Product Candidates-Oncology

The company is currently developing TransCon technology in oncology for a variety of solid tumors, with encouraging early data in platinum resistant ovarian cancer and melanoma. Aside from Proleukin being the only approved IL-2, TransCon IL-2 b/g may face competition from other IL-2 type drug candidates in development, including those being developed by Mural, Anaveon, Medicenna, Roche, Innovent, Synthekine, Dragonfly, Sotio, and Aulos. In addition, TransCon IL-2 b/g may face competition from drug candidates in development for platinum resistant ovarian cancer, including Merck, Corcept, Novartis, Advenchen Laboratories, Genelux, Daiichi Sankyo and Mereo/OncXerna. In melanoma, TransCon IL-2 b/g may face competition from drug candidates in development including from Replimune, Philogen, OncoSec, Immunocore, and Regeneron.

Solution: TransCon Technologies for Oncology

The company is currently investigating one clinical-stage product candidate designed to activate the patient's own immune system to eradicate malignant cells.

The company's TransCon product candidate currently being investigated in solid tumors is designed to provide sustained systemic administration. The company's nonclinical studies have shown sustained activation of cytotoxic immune cells that resulted in robust anti-tumor responses by TransCon product candidates using infrequent administration.

TransCon IL-2 b/g for Sustained Systemic Release

TransCon IL-2 b/g (onvapegleukin alfa) is an investigational long-acting prodrug designed to improve cancer immunotherapy through sustained release of an IL-2 variant that selectively activates IL-2 b/g, with minimal binding to IL-2R?. The IL-Believe Trial, a Phase 1/2 clinical trial to evaluate the safety and efficacy of TransCon IL-2 b/g in locally advanced or metastatic solid tumors, alone or in combination with pembrolizumab or standard of care chemotherapy, has completed dose escalation and is enrolling patients in multiple indication-specific dose expansion cohorts, including platinum-resistant ovarian cancer (PROC), cervical cancer, melanoma, non-small cell lung cancer (NSCLC), and small cell lung cancer (SCLC) at the RP2D.

In September 2024, the company announced initial data showing signs of clinical activity in heavily pre-treated patients with PROC treated (cohort 3) with TransCon IL-2 ?/? in combination with chemotherapy in the ongoing Phase 1/2 IL-Believe Trial of TransCon IL-2 ?/?. As of a cutoff date of July 29, 2024, of the 18 patients (median age 64 years) included in the initial assessment, 14 were efficacy evaluable patients who had one or more post-baseline tumor assessment(s), plus an additional four who discontinued treatment before the first post-baseline tumor assessment due to disease progression or death.

In June 2024, the company reported updated results from its ongoing Phase 1/2 IL-Believe Trial of TransCon IL-2 b/g. Data included the first presentation of Phase 2 dose expansion Cohort 4 (TransCon IL-2 ??? in combination with TransCon TLR7/8 Agonist) in post anti-PD-1 melanoma and new analyses of patients from dose escalation cohorts with prior disease progression on checkpoint inhibitors, along with biomarker studies correlating cytotoxic immune cell expansion and observed clinical benefit. As of the April 16, 2024, data cutoff, confirmed clinical partial responses were observed in 40% (two out of five) of efficacy-evaluable patients from Cohort 4, suggesting potential synergy of the company's two novel immunotherapy candidates in patients who did not derive sufficient benefit from checkpoint inhibitors.

In October 2023, the company announced updated data from the ongoing Phase 1 dose escalation cohort from IL-Believe Trial. Forty-six patients were enrolled into dose escalation cohorts: 25 to monotherapy and 21 to combination therapy. As of the August 15, 2023, data cutoff, anti-tumor clinical responses were observed with TransCon IL-2 b/g monotherapy (colorectal cancer with PR) or in combination with pembrolizumab (small cell lung cancer, one with confirmed PR and one ongoing with unconfirmed complete response) in heavily pre-treated patients who previously progressed on checkpoint inhibitors. TransCon IL-2 b/g every three weeks was generally well-tolerated, with no meaningful effect on Tregs and eosinophils.

In September 2023, the company announced completion of Phase 1 dose escalation in combination with pembrolizumab of the IL-Believe Trial with a total of 21 patients enrolled and RP2D determined at 120 µg/kg IV every three weeks. Twenty-one patients were enrolled.

In May 2023, the company announced completion of the Phase 1 monotherapy dose escalation of the IL-Believe Trial with RP2D determined at 120 µg/kg IV every three weeks with 25 heavily pre-treated patients enrolled and a median of four prior lines of systemic therapies.

Strategic Collaborations

The company also engages in strategic collaborations to further leverage its TransCon technologies in certain geographies and therapeutic areas with market-leading biopharmaceutical companies. These collaborations aim to make promising treatment options available to more patients and to further monetize both the company's TransCon technologies and its internal product candidates, particularly in therapeutic areas. In addition, the company may choose to pursue a collaboration to develop and market its internal, wholly owned product candidates in geographic markets outside its core focus areas of the United States and Europe.

Novo Nordisk A/S

In November 2024, the company entered into a research and development collaboration and license agreement with Novo Nordisk A/S (Novo Nordisk) pursuant to which it granted Novo Nordisk an exclusive worldwide license to the TransCon technology platform to develop, manufacture and commercialize Novo Nordisk proprietary products (including Semaglutide) in metabolic diseases (including obesity and type 2 diabetes) and a product-by-product exclusive license in cardiovascular diseases.

Teijin Limited

In November 2023, the company announced that it entered into an exclusive license agreement with Teijin Limited for the further development and commercialization of TransCon hGH, TransCon PTH, and TransCon CNP for endocrinology rare disease in Japan.

In December 2024, Teijin Pharma Limited announced the submission of an application for manufacturing and marketing approval of palopegteriparatide for the treatment of hypoparathyroidism in Japan.

Strategic Investments

VISEN Pharmaceuticals

In November 2018, the company announced the formation of VISEN, a company established to develop and commercialize its endocrinology rare disease therapies in the People's Republic of China, Hong Kong, Macau, and Taiwan (Greater China). In connection with the formation of VISEN, it granted VISEN exclusive rights to develop and commercialize certain product candidates based on its proprietary TransCon technologies, including TransCon hGH, TransCon PTH, and TransCon CNP, in Greater China for use in all human indications, subject to certain exceptions.

In August 2024, VISEN announced top-line data from the 26-week randomized, double-blind, placebo-controlled portion of the Phase 3 PaTHway China Trial of Palopegteriparatide (TransCon PTH) in adults with chronic hypoparathyroidism. VISEN reported a statistically significant higher proportion of patients treated with palopegteriparatide achieved the primary multi-component endpoint compared to placebo.

In March 2024, VISEN announced that the BLA for lonapegsomatropin (TransCon hGH) was accepted by the China National Medical Products Administration.

In November 2023, VISEN announced top-line results from the Phase 2 ACcomplisH China Trial in children with achondroplasia aged 2 to 10 years. VISEN reported that patients dosed with TransCon CNP at the 100 ?g CNP/kg/week showed significantly higher AGV than placebo at Week 52.

In November 2022, VISEN announced data from its pivotal Phase 3 study of TransCon hGH in children with GHD in China. VISEN reported that patients dosed with TransCon hGH demonstrated an annualized height velocity (AHV) of 10.66 cm/year compared to 9.75 cm/year for the daily hGH at 52 weeks (treatment difference at 0.91 cm/year with a 95 percent confidence interval: 0.37 - 1.45 cm/year, p=0.0010), reaching its primary objective, demonstrating that TransCon hGH is non-inferior to the daily hGH.

NOF Manufacturing and Supply Agreement Related to TransCon hGH

On December 21, 2017, the company entered into a multi-year Manufacturing and Supply Agreement (the NOF Agreement) with NOF Corporation (NOF). Under the NOF Agreement, NOF has agreed to manufacture and supply the mPEG Linker (the NOF hGH Product) for its TransCon hGH product candidate. The company has agreed to purchase certain quantities of NOF hGH Product.

The NOF Agreement is effective as of December 21, 2017. The initial term of the NOF Agreement terminates on December 31, 2025 unless earlier terminated.

NOF Manufacturing and Supply Agreement Related to TransCon PTH

On August 31, 2020, the company entered into a multi-year Manufacturing and Supply Agreement (the NOF PTH Agreement) with NOF. Under the NOF PTH Agreement, NOF has agreed to manufacture and supply the PEG maleimide (the NOF PTH Product) for its TransCon PTH product candidate. The company has agreed to purchase certain quantities of NOF PTH Product. The company may purchase NOF PTH Product from other manufacturers and are not obligated to purchase NOF PTH Product from NOF, other than certain quantities that have been forecasted by it in accordance with a mandatory rolling forecast that the company must deliver to NOF from time to time.

The NOF PTH Agreement is effective as of August 31, 2020. The initial term of the NOF PTH Agreement terminates on December 31, 2027, unless earlier terminated.

Carbogen Manufacturing and Supply Agreement Related to TransCon hGH

On October 26, 2018, the company entered into a multi-year Manufacturing and Supply Agreement (the Carbogen Agreement) with Carbogen Amcis AG (Carbogen). Under the Carbogen Agreement, Carbogen has agreed to manufacture and supply Linker A (the Carbogen Product) for its TransCon hGH product candidate. The company may purchase Linker A from other manufacturers and are not obligated to purchase Carbogen Product from Carbogen, other than certain quantities that have been forecasted by the company in accordance with a mandatory rolling forecast that it must deliver to Carbogen from time to time.

The Carbogen Agreement is effective as of October 26, 2018. The initial term of the Carbogen Agreement expires five years after the first commercial launch of its TransCon hGH product candidate (the Carbogen Initial Term) unless earlier terminated.

Carbogen Manufacturing and Supply Agreement Related to TransCon PTH

On May 27, 2021, the company entered into a multi-year Manufacturing and Supply Agreement (the Carbogen PTH Agreement) with Carbogen. Under the Carbogen PTH Agreement, Carbogen has agreed to manufacture and supply Linker F (the Carbogen PTH Product) for the company's TransCon PTH product candidate. The company may purchase Carbogen PTH Product from other manufacturers and is not obligated to purchase Carbogen PTH Product from Carbogen, other than certain quantities that have been forecasted by the company in accordance with a mandatory rolling forecast that it must deliver to Carbogen from time to time.

The Carbogen PTH Agreement is effective as of May 27, 2021. The initial term of the Carbogen PTH Agreement expires five years after the first commercial launch of the company's TransCon PTH product candidate (the Carbogen PTH Initial Term) unless earlier terminated.

Phillips Medisize (formerly B&O Medicom and Medicom Innovation Partner)

On January 12, 2017, the company entered into a multi-year Manufacturing and Supply Agreement (the Medicom Agreement) with Medicom Innovation Partner (Medicom). Under the Medicom Agreement, Medicom has agreed to exclusively manufacture and supply the auto injector injection device (the Medicom Product) for the company's TransCon hGH product candidate. The company is obligated to purchase certain quantities that have been forecasted by it in accordance with a mandatory rolling forecast that it must deliver to Medicom from time to time.

The Medicom Agreement is effective as of January 12, 2017. The term of the Medicom Agreement terminates on June 30, 2025 (Medicom Initial Term) unless earlier terminated or unless extended unilaterally by the company, with notice of extension to be given no later than June 30, 2024, by five years until June 30, 2030 (Extended Term) after which date it shall continue indefinitely unless terminated.

Vetter Pharma International GmbH

On December 14, 2018, the company entered into a multi-year Supply Agreement (the Vetter Agreement) with Vetter Pharma International (Vetter). Under the Vetter Agreement, Vetter has agreed to manufacture and fill-and-finish drug products in dual-chamber cartridges (the Ascendis Product) for the company's TransCon hGH product candidate. Vetter has agreed to supply in accordance with a long-term forecast in addition to a rolling forecast with a binding part that the company must deliver to Vetter from time to time.

The Vetter Agreement is effective as of January 1, 2019. The term of the Vetter Agreement expires on the five-year anniversary of the date of first regulatory approval of the TransCon hGH product (the Initial Term) after which term it shall be automatically renewed for subsequent two-year terms unless terminated.

Lonza Tech Transfer and Manufacturing Agreement

On December 12, 2019, the company entered into a multi-year commercial supply agreement (the Lonza Agreement) with Lonza Ltd (Lonza). Under the Lonza Agreement, Lonza has agreed to manufacture and supply drug substance for the company's TransCon hGH product candidate (the TransCon hGH Drug Substance). Starting in 2023, the company is obligated to purchase a certain minimum annual quantity of TransCon hGH Drug Substance from Lonza. The company may also purchase TransCon hGH Drug Substance from other manufacturers.

The Lonza Agreement secures the company expects any patents granted from this family to expire in May 2045, absent any patent term a certain capacity of TransCon hGH Drug Substance per year.

The Lonza Agreement is effective as of December 12, 2019. The initial term of the Lonza Agreement expires seven years after first approval of a drug product manufactured using the TransCon hGH Drug Substance (the Lonza Initial Term) unless earlier terminated.

Sharp Corporation Packaging and Supply Agreement

On December 1, 2019, the company entered into a multi-year packaging agreement (the Sharp Agreement) with Sharp Corporation (Sharp). Under the Sharp Agreement, Sharp agreed to package, assemble, and label TransCon hGH for commercial use in certain territories, including the United States and the EU. The company is non-exclusive to Sharp and may engage other manufacturers to package, assemble, and label TransCon hGH; however, it is obligated to meet certain minimum spend requirements for TransCon hGH during the first 12-month period after the first shipment of TransCon hGH for commercial sale following regulatory approval.

The Sharp Agreement is effective as of December 1, 2019. The initial term of the Sharp Agreement expires on December 31, 2025, and will be automatically extended for additional two-year periods unless earlier terminated.

Bachem Manufacturing and Supply Agreement

On December 27, 2020, the company entered into a multi-year Manufacturing and Supply Agreement (the Bachem Agreement) with Bachem AG (Bachem). Under the Bachem Agreement, Bachem has agreed to manufacture and supply PTH drug substance (the Bachem Product) for the company's TransCon PTH product candidate. The company may purchase Bachem Product from other manufacturers and is not obligated to purchase Bachem Product from Bachem, other than certain quantities that have been forecasted by the company in accordance with a mandatory rolling forecast that it must deliver to Bachem from time to time.

The Bachem Agreement is effective as of December 27, 2020. The initial term of the Bachem Agreement expires on December 31, 2027 (the Bachem Initial Term) unless earlier terminated.

Intellectual Property

As of December 31, 2024, the company owned a total of 99 patent families, 18 of which are currently in their priority year or international phase. These patent families include granted patents in the United States (62), Europe (36), Australia (54), Brazil (10), Canada (29), China (20), Israel (21), Indonesia (6), India (12), Korea (15), Malaysia (13), New Zealand (18), Japan (52), Mexico (21), Singapore (20), Russia (24), the United Arab Emirates (1) and South Africa (17) and approximately 583 pending national/regional applications in a total of 26 jurisdictions (excluding the member states of the European Patent Convention in which its European patents were validated, Hong Kong, in which certain Europe and China patents are extended, and Macao, in which certain China patents are extended).

TransCon hGH

As of December 31, 2024, the company's patent portfolio related to TransCon hGH includes eight patent families relating to different aspects of TransCon hGH and eleven patent families covering various aspects of the auto-injector device for the administration of TransCon hGH. The first of these patent families is a composition of matter patent family directed to the particular stoichiometry of TransCon hGH and a related TransCon carrier. As of December 31, 2024, this patent family includes one patent granted in the United States, which is expected to expire in February 2027 (accounting for patent term adjustment).

The second of these patent families is a composition of matter patent family directed to a TransCon linker used in TransCon hGH. As of December 31, 2024, this patent family includes patents granted in the United States, Europe, Australia, Brazil, Canada, Japan and Mexico and one pending patent application in the United States. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in March 2025, absent any patent term adjustments or extensions.

The third of these patent families is a composition of matter patent family directed to a broad class of TransCon hGH lead candidate structures. As of December 31, 2024, this patent family includes patents granted in the United States, Europe, Australia, Brazil, Canada, China, Israel, India, Japan, Mexico, Russia and South Africa and pending patent applications in Europe and the United States. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in April 2029, absent any patent term adjustments or extensions.

The fourth of these patent families is a composition of matter patent family directed to specific dry pharmaceutical compositions comprising TransCon hGH. As of December 31, 2024, this patent family includes patents granted in the United States, Europe, Australia, Brazil, Canada, India, Israel, Mexico, Singapore and South Africa and one pending patent application in the United States. The granted patents and any patents granted from the pending patent application in this patent family are expected to expire in December 2030, absent any patent term adjustments or extensions.

The fifth of these patent families is a composition of matter patent family directed to a broad class of TransCon hGH lead candidate structures. As of December 31, 2024, this patent family includes patents granted in the United States, Australia, Europe, Israel, Japan, South Korea, Mexico, New Zealand, Russia, Singapore and South Africa and pending patent applications in the United States, Europe, Australia, Brazil, Canada, Israel, Japan, South Korea, Mexico, Russia and Singapore. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in November 2035, absent any patent term adjustments or extensions. One granted EP patent in this family was also used as a basis for requesting a supplementary protection certificate (SPC) for SKYTROFA in Austria, Belgium, Bulgaria, Cyprus, Czech Republic, Germany, Denmark, Estonia, Spain, Finland, France, Great Britain, Greece, Croatia, Hungary, Ireland, Iceland, Italy, Lithuania, Luxemburg, Latvia, Malta, the Netherlands, Norway, Poland, Portugal, Romania, Sweden, Slovenia and Slovakia. SPCs have already been granted in Bulgaria, Cyprus, Czech Republic, Denmark, Spain, Finland, France, Greece, Hungary, Iceland, Italy, Lithuania, Latvia, Malta, the Netherlands, Norway, Portugal, Sweden, Slovenia and Slovakia, each of which are expected to expire in January 2037.

The sixth of these patent families is directed to a particular dosage regimen for long-acting growth hormone formulations. As of December 31, 2024, this patent family includes granted patents in the United States and Europe and pending patent applications in the United States and in Europe. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in November 2035, absent any patent term adjustments or extensions.

The seventh of these patent families is directed to potential superior efficacy achieved with TransCon hGH treatment. As of December 31, 2024, this patent family includes a granted patent in Japan and pending patent applications in the United States, Europe, Australia, Canada, China, Israel, Japan, Mexico and Singapore. The granted patent and any patents granted from the pending patent applications in this patent family are expected to expire in March 2040, absent any patent term adjustments or extensions.

The eighth of these patent families is directed to the use of TransCon hGH in the treatment of an inflammation-induced disease, in particular of non-alcoholic fatty liver disease and non-alcoholic steatohepatitis. As of December 31, 2024, this patent family includes pending patent applications in the United States, Europe, Australia, Brazil, Canada, China, Indonesia, Israel, Japan, South Korea, Mexico, Malaysia, New Zealand, Russia, Singapore and South Africa. Any patents granted from the pending patent applications in this patent family are expected to expire in March 2042, absent any patent term adjustments or extensions.

Seven of the eleven patent families covering the auto-injector device for the administration of TransCon hGH with a filing date of December 29, 2016, include pending patent applications in the United States, Europe, Australia, Canada, Japan and New Zealand and ten granted patents in the United States, six granted patents in Europe, nine granted patents in Australia, four granted patents in Canada, seven granted patents in New Zealand and thirteen granted patents in Japan as of December 31, 2024. The granted patents in these patent families are expected to expire in December 2036, absent any patent term adjustments or extensions. As of December 31, 2024, another two of the eleven patent families covering the auto-injector device with a filing date of May 23, 2018 and June 29, 2018, respectively, include pending patent applications in the United States, Europe, the United Arab Emirates, Australia, Canada, China, Israel, Japan, South Korea, Mexico, Malaysia, New Zealand, Russia and Singapore and two granted patents in the United States, two granted patents in Australia, two granted patents in Brazil, three granted patents in China, two granted patents in India, three granted patents in Japan, three granted patents in South Korea, one granted patent in Mexico, two granted patents in Malaysia, two granted patents in Russia, two granted patents in Indonesia, two granted patents in Singapore and one granted patent in South Africa. The granted patents in these patent families are expected to expire in March and June 2038, respectively, absent any patent term adjustments or extensions. The tenth patent family includes pending patent applications in the United States, Europe, the United Arab Emirates, Australia, Brazil, Canada, China, Eurasia, Indonesia, Israel, India, Japan, South Korea, Mexico, Malaysia, New Zealand, Singapore, Taiwan and South Africa. The company expects any patents granted from the pending patent applications in this patent family to expire in September 2042, absent any patent term adjustments or extensions. The eleventh patent family is currently in its priority year. The company expects any patents granted from this family to expire in May 2045, absent any patent term adjustments or extensions.

In addition to the SPCs, three requests for a patent term extension for SKYTROFA were filed based on one granted U.S. patent from the third patent family and two granted patents from the fourth patent family, from which one will be selected for the patent term extension upon allowance. For all of these three requests a preliminary assessment that the patents would be eligible for extension was received from FDA.

TransCon PTH

The company's patent portfolio related to TransCon PTH includes twelve patent families relating to different aspects of TransCon PTH. The first of these patent families is a composition of matter patent family directed to the TransCon linker used in TransCon PTH. As of December 31, 2024, this patent family includes granted patents in the United States, Europe, the United Arab Emirates, Australia, Canada, China, Israel, Japan, Mexico, Russia and South Africa and pending patent applications in Europe, the United States and Brazil. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in January 2029, absent any patent term adjustments or extensions.

The second of these patent families is a composition of matter patent family directed to a broad class of TransCon PTH candidate structures. As of December 31, 2024, this patent family includes pending patent applications in the United States, Europe, Australia, Brazil, Canada, China, Indonesia, Israel, Japan, South Korea, Mexico, Malaysia, New Zealand, Russia, Singapore and Thailand and granted patents in the United States, Australia, China, Europe, Israel, India, Japan, South Korea, Mexico, Malaysia, New Zealand, Russia, Singapore and South Africa. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in February 2037, absent any patent term adjustments or extensions. The granted EP patent in this family was used as a basis for requesting an SPC for YORVIPATH in Austria, Belgium, Bulgaria, Switzerland, Cyprus, Czech Republic, Germany, Denmark, Estonia, Spain, Finland, France, Great Britain, Greece, Croatia, Hungary, Ireland, Iceland, Italy, Lithuania, Luxemburg, Latvia, Malta, the Netherlands, Norway, Poland, Portugal, Romania, Sweden, Slovenia and Slovakia. An SPC was already granted in the Netherlands and Malta with an expiration date of November 2038.

The third and fourth of these patent families are method of treatment patent families directed to a particular dosage regimen. As of December 31, 2024, one of these patent families includes pending patent applications in the United States, Europe, Australia, Brazil, Canada, China, Israel, Indonesia, Japan, South Korea, Malaysia, New Zealand, Russia and Thailand and granted patents in the United States, Europe, Australia, China, Indonesia, Israel, Japan, South Korea, Mexico, New Zealand, Russia, Singapore and South Africa. The other one of these patent families includes pending patent applications in the United States, Europe, Australia, Canada and Japan and granted patents in Europe, Australia, China and Japan. The company expects any patents granted from the pending patent applications in these patent families to expire in September 2037, absent any patent term adjustments or extensions.

The fifth of these patent families is a composition of matter family directed to PTH compounds exhibiting a beneficial pharmacokinetic profile. As of December 31, 2024, this patent family includes pending patent applications in the United States, Europe, Brazil, Canada, China, Israel, South Korea, New Zealand and Russia and granted patents in the United States, Europe, Australia, China, Japan, South Korea, Mexico, New Zealand, Russia, Singapore and South Africa. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in September 2037, absent any patent term adjustments or extensions.

The sixth patent family relates to a starting dose for treatment with reversible PTH conjugates. As of December 31, 2024, this patent family includes pending patent applications in the United States, Europe, Australia, Brazil, Canada, China, Indonesia, Israel, Japan, South Korea, Mexico, Malaysia, New Zealand, Russia, Singapore, Thailand and South Africa and granted patents in Japan, Malaysia and Russia. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in May 2039, absent any patent term adjustments or extensions.

The seventh patent family relates to a pharmaceutical composition comprising reversible PTH conjugates. As of December 31, 2024, this patent family includes pending patent applications in the United States, Europe, Australia, Brazil, Canada, China, Indonesia, Israel, India, Japan, South Korea, Mexico, Malaysia, New Zealand, the Philippines, Russia, Singapore, Taiwan, Thailand, Vietnam and South Africa and granted patents in Japan and Russia. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in February 2040, absent any patent term adjustments or extensions.

The eighth and ninth patent families relate to a method of titrating hypoparathyroidism patients off of standard of care within four weeks from the beginning of daily treatment with a PTH compound and the treatment of the physical and mental well-being of hypoparathyroidism patients, respectively. As of December 31, 2024, the eighth family includes pending patent applications in the United States, Europe, Australia, Brazil, Canada, China, Indonesia, Israel, Japan, South Korea, Mexico, Malaysia, New Zealand, Russia, Singapore and South Africa and a granted patent in Russia. The ninth patent family includes pending patent applications in the United States, Europe, Australia, Canada, China, Israel, Japan, South Korea and Mexico. The granted patent and any patents granted from the pending patent applications in this patent family are expected to expire in September 2041, absent any patent term adjustments or extensions.

The tenth patent family relates to a reduction in bone mineral density in patients having increased bone mineral density. As of December 31, 2024, this patent family includes pending patent applications in the United States, Europe, the United Arab Emirates, Australia, Brazil, Canada, China, Eurasia, Israel, Japan, South Korea, Mexico, Malaysia, New Zealand, Singapore and South Africa. The company expects any patents granted from the pending patent applications in this patent family to expire in September 2042, absent any patent term adjustments or extensions.

The eleventh patent family covers treatment of hypoparathyroidism initially with a long-acting PTH compound, followed by treatment with an ultra-long-acting PTH compound. As of December 31, 2024, this patent family is in the Patent Cooperation Treaty phase. The company expects any patents granted from this patent family to expire in September 2042, absent any patent term adjustments or extensions.

The twelfth family covers treatment of chronic kidney disease. As of December 31, 2024, this patent family is in the Patent Cooperation Treaty phase. The company expects any patents granted from this patent family to expire in September 2044, absent any patent term adjustments or extensions.

In addition to the SPCs, three requests for a patent term extension for YORVIPATH were filed based on two granted U.S. patents from the third patent family described above and one granted patent from the fifth patent family described above, from which one will be selected for patent term extension upon allowance.

TransCon CNP

The company's patent portfolio related to TransCon CNP includes fifteen patent families relating to different aspects of TransCon CNP. The first of these patent families is a composition of matter patent family directed to the particular stoichiometry of TransCon CNP and a related TransCon carrier. As of December 31, 2024, this patent family includes a patent granted in the United States, which is expected to expire in February 2027 (accounting for patent term adjustment).

The second of these patent families is a composition of matter patent family directed to the TransCon linker used in TransCon CNP. As of December 31, 2024, this patent family includes granted patents in the United States, Europe, the United Arab Emirates, Australia, Canada, China, Israel, Japan, Mexico, Russia and South Africa and includes pending patent applications in Europe, the United States and Brazil.

The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in January 2029, absent any patent term adjustments or extensions.

The third of these patent families is a composition of matter patent family directed to a broad class of TransCon CNP candidate structures. As of December 31, 2024, this patent family includes pending patent applications in the United States, Europe, Australia, Canada, India, Japan, Mexico, Malaysia, Singapore and Thailand and granted patents in China, Israel, South Korea, Malaysia, Russia, Singapore and South Africa. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in January 2036, absent any patent term adjustments or extensions.

The fourth to the ninth patent families are composition of matter patent families directed various CNP compounds having beneficial properties. As of December 31, 2024, the first one of these six patent families includes pending patent applications in the United States, Europe and Japan and granted patents in the United States, Australia, Canada, Japan and South Africa. As of December 31, 2024, the second one includes pending patent applications in the United States, Australia and Japan and granted patents in the United States, Europe, Australia, Canada, Japan and South Africa. As of December 31, 2024, the third one includes pending patent applications in the United States, Europe, Australia, Brazil, Canada, Indonesia, India, Japan, South Korea, New Zealand and Singapore and granted patents in the United States, Australia, China, Israel, India, Japan, Mexico, Malaysia, Singapore and South Africa. As of December 31, 2024, the fourth one includes pending patent applications in the United States, Europe and Israel and granted patents in the United States, Australia, Canada and New Zealand. As of December 31, 2024, the fifth one includes pending patent applications in the United States, Europe, Brazil and China and granted patents in the United States, Australia, Canada, China, Israel, New Zealand, South Korea and Singapore. As of December 31, 2024, the sixth one includes pending patent applications in the United States, Europe and Australia and granted patents in the United States, Australia, Canada and Israel. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in January 2037, absent any patent term adjustments or extensions.

The tenth patent family covers a combination therapy of TransCon CNP. As of December 31, 2024, this patent family includes pending patent applications in the United States, Europe, Brazil, Canada, Israel, South Korea and Thailand and granted patents in the United States, Europe, Australia, China, Indonesia, Israel, Japan, South Korea, Mexico, Malaysia, New Zealand, Russia, Singapore and South Africa. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in September 2037, absent any patent term adjustments or extensions.

The eleventh patent family relates to a pharmaceutical composition comprising reversible CNP conjugates. As of December 31, 2024, this patent family includes pending patent applications in the United States, Europe, Australia, Brazil, Canada, China, Indonesia, Israel, India, Japan, South Korea, Mexico, Malaysia, New Zealand, the Philippines, Russia, Singapore, Taiwan, Thailand, Vietnam and South Africa and granted patents in China, Japan and Russia. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in February 2040, absent any patent term adjustments or extensions.

The twelfth patent family relates to a pharmacologically effective dose of TransCon CNP. As of December 31, 2024, this patent family consists of pending patent applications in the United States, Europe, Australia, Canada, China, Israel, Japan, South Korea and Singapore. The company expects any patents granted from the pending patent applications in this patent family to expire in December 2042, absent any patent term adjustments or extensions. One of the priority applications within this family was also claimed by a Patent Cooperation Treaty application relating to improving muscle function. The company expects any patents granted from this branch of the patent family to expire in November 2043, absent any patent term adjustments or extension.

The thirteenth patent family, which relates to the treatment of spinal deformities, claims priorities from the twelfth patent family. The company expects any patents granted from this patent family to expire in March 2044, absent any patent term adjustments or extensions.

The fourteenth patent family relates to a liquid formulation of TransCon CNP. As of December 31, 2024, this patent family includes pending patent applications in the United States, Europe, Australia, Brazil, Canada, China, Indonesia, Israel, India, Japan, South Korea, Mexico, Malaysia, New Zealand Russia, Singapore and South Africa. The company expects any patents granted from the pending patent applications in this patent family to expire in May 2043, absent any patent term adjustment or extensions.

The fifteenth patent family relates to the use of TransCon CNP for the prevention or reduction of leg bowing. As of December 31, 2024, this patent family is in its priority year and the company expects any patents granted from this patent family to expire in December 2045, absent any patent term adjustments or extensions.

TransCon IL-2 b/g

As of December 31, 2024, the company's patent portfolio related to TransCon IL-2 b/g includes four patent families. The first of these patent families is a composition of matter patent family directed to a TransCon linker used in TransCon IL-2 b/g. As of December 31, 2024, this patent family includes patents granted in the United States, Europe, Australia, Brazil, Canada, Japan and Mexico and one pending patent application in the United States. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in March 2025, absent any patent term adjustments or extensions.

The second and third patent family are composition of matter patent families directed to a broad class of TransCon IL-2 b/g lead candidate structures. As of December 31, 2024, the second patent family includes pending patent applications in the United States, Europe, Australia, Brazil, Canada, China, Indonesia, Israel, India, Japan, South Korea, Mexico, Malaysia, New Zealand, Russia, Singapore, Thailand and South Africa and granted patents in Australia, China, Israel, Japan and Russia. The granted patents and any patents granted from the pending patent applications in this patent family are expected to expire in March 2039. As of December 31, 2024, the third patent family includes pending patent applications in the United States, Europe, Argentina, the United Arab Emirates, Australia, Brazil, Canada, China, Eurasia, Egypt, Indonesia, Israel, India, Japan, South Korea, Mexico, Malaysia, New Zealand, the Philippines, Singapore, Taiwan, Thailand, Vietnam and South Africa and a granted patent in the United States. The granted patent and any patents granted from the pending patent applications in this patent family are expected to expire in June 2041, absent any patent term adjustments or extensions.

The fourth patent family relates to a pharmaceutical dose of TransCon IL-2 b/g. As of December 31, 2024, this patent family includes one Patent Cooperation Treaty application. The company expects any patents granted in this patent family to expire in May 2044, absent any patent term adjustments or extension.

TransCon Technologies

The company's patent portfolio also includes patents and pending patent applications generally relating to its TransCon technologies, including TransCon linkers, TransCon carriers and certain soluble conjugates. The company owns an aggregate of 16 patent families relating to TransCon linkers, the material components of which are described above. The company also owns an aggregate of 11 patent families relating to TransCon carriers, the material components of which are described above.

Government Regulation and Product Approval

The Federal Food, Drug, and Cosmetic Act; the Public Health Service Act; and their implementing regulations set forth, among other things, requirements for the research, testing, development, manufacture, quality control, safety, effectiveness, approval, labeling, storage, record keeping, reporting, distribution, import, export, advertising and promotion of the company's products.

As the company receives and, processes personal and confidential data, the company become subject to numerous laws and regulations relating to data privacy, AI and security, such as data breach notification laws, health information laws, AI regulations and consumer protection laws in the United States and abroad, including the General Data Protection Regulation (GDPR) and the U.K. GDPR.

History

Ascendis Pharma A/S was incorporated in 2006.

Ascendis Pharma A/S Questions and Answers

Which sectors generate sales and which are the top 3 markets?

Revenue distribution by industry: Biotechnology: 100% (2025) TOP 3 markets: USA: approx. 65% Europe: approx. 25% Asia-Pacific: approx. 10% Ascendis Pharma A/S is a biopharmaceutical company focusing on developing drugs for the treatment of rare diseases. The majority of revenues come from the bi...

At which locations are the company’s products manufactured?

Production Sites: Germany, Denmark Ascendis Pharma A/S mainly produces its products in Germany and Denmark. The company strategically located its production capacities in Europe to benefit from the proximity to key markets and a well-developed infrastructure. These sites enable Ascendis Pharma to ef...

What strategy does Ascendis Pharma A/S pursue for future growth?

Focus on Endocrinology: Ascendis Pharma is strongly focused on the development of therapies in the field of endocrinology. Pipeline Expansion: The company is investing in expanding its product pipeline, particularly through the development of new drugs and therapies. TransCon Technology: Ascendis re...

Which raw materials are imported and from which countries?

Main raw materials: Biotechnological materials, chemicals for pharmaceutical production Countries of origin: USA, Germany, China Ascendis Pharma A/S is a biopharmaceutical company specializing in drug development. The production of biopharmaceutical products requires specific biotechnological materi...

How strong is the company’s competitive advantage?

Market share: Estimated 10-15% in the field of endocrinology (2025) R&D expenses: 35% of revenue (2024) Patent portfolio: Over 100 active patents (2025) Ascendis Pharma A/S has gained a significant competitive advantage through its innovative TransCon technology. This technology allows for contr...

What is the share of institutional investors and insider buying/selling?

Institutional Investor Share: approx. 85% (estimated for 2025) Insider Buys/Sells: No specific data available for 2025 The institutional investor share in Ascendis Pharma A/S is typically high, as large investment funds and institutional investors often invest in biotechnology companies to benefit f...

What percentage market share does Ascendis Pharma A/S have?

Market share of Ascendis Pharma A/S: Estimate: approximately 2-3% in the field of endocrinology (2025) Main competitors and their market shares: Novo Nordisk A/S: around 30% Eli Lilly and Company: around 25% Pfizer Inc.: around 15% Sanofi S.A.: around 10% Roche Holding AG: around 8% Merck & Co....

Is Ascendis Pharma A/S stock currently a good investment?

Revenue Growth: 18% (2024) Research and Development Expenses: 45% of Revenue (2024) Net Loss: $150 million USD (2024) Ascendis Pharma A/S recorded a revenue growth of 18% in 2024, attributed to the successful launch of new products and expansion into international markets. The company is heavily inv...

Does Ascendis Pharma A/S pay a dividend – and how reliable is the payout?

Dividend payment: No dividend (as of 2025) Ascendis Pharma A/S is a biopharmaceutical company that focuses on developing drugs for the treatment of rare diseases. Traditionally, biotech and pharmaceutical companies in the development phase do not pay dividends as they reinvest their profits into res...

Ascendis Pharma A/S Stock Value

Ascendis Pharma A/S Company Info

Analyst Price Target

Top growth stocks in the health care sector (5Y.)

What does Ascendis Pharma A/S do?

Ascendis Pharma A/S Questions and Answers

Offer & Services

Service & Collaboration

Company & Legal