Ascendis Pharma utilizes its proprietary TransCon™ technology platform to develop innovative therapies aimed at addressing unmet medical needs, particularly in the fields of endocrinology and oncology. TransCon, which stands for "transient conjugation," allows for the temporary linking of an inert carrier to a parent drug, enabling the creation of prodrugs that can be designed for systemic or localized therapeutic effects. This technology is particularly beneficial for enhancing the efficacy and tolerability of treatments, as it maintains the original mechanism of action of the parent drug while providing controlled release. The platform is versatile and can be applied to proteins, peptides, and small molecules across various therapeutic areas.

One of the key applications of TransCon technology is in the development of TransCon PTH, an investigational therapy designed to replace parathyroid hormone in patients with hypoparathyroidism. This therapy aims to restore physiological levels of PTH, addressing both the immediate symptoms and long-term complications associated with the condition. By providing a once-daily hormone replacement therapy, TransCon PTH seeks to improve the quality of life for patients who currently face significant challenges with existing treatment options. The technology's ability to optimize dosing frequency and enhance patient adherence underscores Ascendis Pharma's commitment to making a meaningful difference in the lives of patients and caregivers alike.

United States

Ascendis Pharma A/S, a biopharmaceutical company, develops various prodrug therapies for unmet medical needs. The company develops TransCon growth hormone, which has completed Phase III clinical study in children to treat growth hormone deficiency; TransCon parathyroid hormone which is in Phase II clinical study for treating hypoparathyroidism; and TransCon CNP, a long-acting prodrug of C-type natriuretic peptide which is in Phase II clinical for the treatment of achondroplasia. It is also developing preclinical studies in the field of oncology for potential product candidates and evaluate systemic and localized delivery systems using its TransCon technologies. It also has strategic collaborations with Sanofi to develop TransCon technologies for the treatment of diabetes. The company was founded in 2006 and is headquartered in Hellerup, Denmark.

Cancer

Endocrine System Diseases

Rare diseases

Ascendis Pharma 

Pediatric growth hormone deficiency

TransCon refers to “transient conjugation.” The proprietary TransCon &nbsp;platform is an innovative technology designed to create new therapies &nbsp;that optimize therapeutic effect, including efficacy, safety and dosing &nbsp;frequency. TransCon molecules have three components: an unmodified &nbsp;parent drug, an inert carrier that protects it, and a linker that &nbsp;temporarily binds the two. When bound, the carrier inactivates and &nbsp;shields the parent drug from clearance. When injected into the body, &nbsp;physiologic conditions (e.g., pH and temperature) initiate the release &nbsp;of the active, unmodified parent drug in a predictable manner. Because &nbsp;the parent drug is unmodified, its original mode of action may be &nbsp;maintained. TransCon technology is designed to be applied broadly to a &nbsp;protein, peptide or small molecule in multiple therapeutic areas, and to &nbsp;be used systemically or locally.
<a href="https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-announces-us-food-and-drug-administration-fda">Source</a>

SKYTROFA (TransCon hGH) (lonapegsomatropin)

Adult hypoparathyroidism

TransCon™ PTH is an investigational prodrug of parathyroid hormone (PTH) in development as a once-daily hormone replacement therapy. It is designed to restore physiologic levels of PTH 24 hours a day. By doing so, we believe TransCon PTH can address both the short-term symptoms and long-term complications of hypoparathyroidism. Our goal is to provide a PTH replacement therapy that normalizes serum and urinary calcium, as well as serum phosphate levels, and improves quality of life. TransCon PTH has orphan designation for the treatment of hypoparathyroidism in both the U.S. and Europe.
 
 
TransCon refers to “transient conjugation.” The proprietary TransCon &nbsp;platform is an innovative technology designed to create new therapies &nbsp;that optimize therapeutic effect, including efficacy, safety and dosing &nbsp;frequency. TransCon molecules have three components: an unmodified &nbsp;parent drug, an inert carrier that protects it, and a linker that &nbsp;temporarily binds the two. When bound, the carrier inactivates and &nbsp;shields the parent drug from clearance. When injected into the body, &nbsp;physiologic conditions (e.g., pH and temperature) initiate the release &nbsp;of the active, unmodified parent drug in a predictable manner. Because &nbsp;the parent drug is unmodified, its original mode of action may be &nbsp;maintained. TransCon technology is designed to be applied broadly to a &nbsp;protein, peptide or small molecule in multiple therapeutic areas, and to &nbsp;be used systemically or locally.
<a href="https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-announces-us-food-and-drug-administration-fda">Source</a>

YORVIPATH® (palopegteriparatide, developed as TransCon PTH)

Adult growth hormone deficiency

TransCon hGH is designed to deliver unmodified hGH, the same growth hormone used in daily therapies, at a predictable rate over one week.
 
 
TransCon refers to “transient conjugation.” The proprietary TransCon &nbsp;platform is an innovative technology designed to create new therapies &nbsp;that optimize therapeutic effect, including efficacy, safety and dosing &nbsp;frequency. TransCon molecules have three components: an unmodified &nbsp;parent drug, an inert carrier that protects it, and a linker that &nbsp;temporarily binds the two. When bound, the carrier inactivates and &nbsp;shields the parent drug from clearance. When injected into the body, &nbsp;physiologic conditions (e.g., pH and temperature) initiate the release &nbsp;of the active, unmodified parent drug in a predictable manner. Because &nbsp;the parent drug is unmodified, its original mode of action may be &nbsp;maintained. TransCon technology is designed to be applied broadly to a &nbsp;protein, peptide or small molecule in multiple therapeutic areas, and to &nbsp;be used systemically or locally.
<a href="https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-announces-us-food-and-drug-administration-fda">Source</a>

TransCon hGH

Achondroplasia

TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) in development for the treatment of ACH in children. Designed to provide continuous CNP exposure, the goal of TransCon CNP is to optimize efficacy with a safe and convenient once-weekly dose. TransCon CNP has orphan designation for the treatment of ACH in both the U.S and Europe.
 
 
TransCon refers to “transient conjugation.” The proprietary TransCon &nbsp;platform is an innovative technology designed to create new therapies &nbsp;that optimize therapeutic effect, including efficacy, safety and dosing &nbsp;frequency. TransCon molecules have three components: an unmodified &nbsp;parent drug, an inert carrier that protects it, and a linker that &nbsp;temporarily binds the two. When bound, the carrier inactivates and &nbsp;shields the parent drug from clearance. When injected into the body, &nbsp;physiologic conditions (e.g., pH and temperature) initiate the release &nbsp;of the active, unmodified parent drug in a predictable manner. Because &nbsp;the parent drug is unmodified, its original mode of action may be &nbsp;maintained. TransCon technology is designed to be applied broadly to a &nbsp;protein, peptide or small molecule in multiple therapeutic areas, and to &nbsp;be used systemically or locally.
<a href="https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-announces-us-food-and-drug-administration-fda">Source</a>

TransCon CNP  

Solid tumor/s

Designed for sustained release of a toll-like receptor (TLR)7/8 agonist, resiquimod
Expected to provide potent activation of the innate immune system in the tumor and draining lymph nodes with low risk of systemic toxicity
<a href="https://ascendispharma.com/pipeline/oncology/#transCon-agonist">Source</a>

TransCon TLR7/8 Agonist +/- pembrolizumab

TransCon IL-2 B/y 

Turner syndrome

TransCon refers to “transient conjugation.” The proprietary TransCon &nbsp;platform is an innovative technology designed to create new therapies &nbsp;that optimize therapeutic effect, including efficacy, safety and dosing &nbsp;frequency. TransCon molecules have three components: an unmodified &nbsp;parent drug, an inert carrier that protects it, and a linker that &nbsp;temporarily binds the two. When bound, the carrier inactivates and &nbsp;shields the parent drug from clearance. When injected into the body, &nbsp;physiologic conditions (e.g., pH and temperature) initiate the release &nbsp;of the active, unmodified parent drug in a predictable manner. Because &nbsp;the parent drug is unmodified, its original mode of action may be &nbsp;maintained. TransCon technology is designed to be applied broadly to a &nbsp;protein, peptide or small molecule in multiple therapeutic areas, and to &nbsp;be used systemically or locally.
<a href="https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-announces-us-food-and-drug-administration-fda">Source</a>

Ascendis Pharma

Company Details