Ascendis Pharma A/S Announces Filing of the Clinical Trial Notification for Phase 3 Clinical Trial of TransCon hGH for Pediatric Growth Hormone Deficiency in Japan
COPENHAGEN, Denmark, Sept. 23, 2020 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical
needs, today announced that it has filed a Clinical Trial Notification (CTN) with the Pharmaceuticals and Medical Devices Agency (PMDA) in Japan to initiate the company’s phase 3 riGHt Trial
of TransCon hGH (lonapegsomatropin), a long-acting prodrug of somatropin (hGH), for the treatment for pediatric growth hormone deficiency (GHD).
“The initiation of our phase 3 riGHt Trial in Japan for TransCon hGH is an important clinical milestone, bringing us a step closer to extending the global reach of TransCon hGH,” said Jan Mikkelsen, President and CEO at Ascendis Pharma. “This riGHt Trial will evaluate TransCon hGH in treatment naïve children with pediatric GHD and will help support our marketing applications in Japan.”
The primary objective of the riGHt Trial is to evaluate and compare the annualized height velocity (AHV) of 40 Japanese prepubertal treatment naïve children with GHD treated with weekly TransCon hGH to that of a commercially available daily hGH formulation at 52 weeks.
About Pediatric Growth Hormone Deficiency (GHD)1
Pediatric GHD is a serious orphan disease caused when the pituitary gland does not produce enough growth hormone. Children with GHD are not only characterized by short stature, but they also may experience metabolic abnormalities, psychosocial challenges and poor quality of life. For decades, the standard of care for GHD has been a daily subcutaneous injection of hGH, which improves growth and overall endocrine health.
About TransCon Technology
TransCon refers to “transient conjugation.” The proprietary TransCon platform is an innovative technology designed to create new therapies that optimize therapeutic effect, including efficacy, safety and dosing frequency. TransCon molecules have three components: an unmodified 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 conditions (e.g., pH and temperature) initiate the release of the active, unmodified parent drug in a predictable manner. Because the parent drug is unmodified, its original mode of action may be maintained. TransCon technology is designed to be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and to be used systemically or locally.