Ascendis Pharma A/S Announces Submission of Biologics License Application (BLA) to FDA for TransCon™ hGH in Pediatric Growth Hormone Deficiency

– European marketing application on track for third quarter 2020 submission –

COPENHAGEN, Denmark I June 26, 2020 I Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransCon technologies to address unmet medical needs, today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for TransCon hGH (lonapegsomatropin), a long-acting once-weekly prodrug of somatropin (human growth hormone or hGH) for treatment for pediatric growth hormone deficiency (GHD). 

“TransCon Growth Hormone is the first product candidate we have taken from idea stage, through multiple Phase 3 clinical trials, and now to BLA submission. We believe the TransCon technology has the potential to address major unmet medical needs and we look forward to continuing the late stage clinical development of our other endocrinology rare disease product candidates, TransCon PTH and TransCon CNP, as well as filing an Investigational New Drug application for our first oncology product candidate later this year,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Most importantly, we are very grateful to all of the participants and investigators in our TransCon hGH trials, and we look forward to discussions with the FDA in an effort to bring this potential new treatment option to children with GHD.”

TransCon hGH is designed to maintain the same mode of action as daily hGH therapies by releasing the same growth hormone molecule, somatropin. Currently, there is no approved long-acting growth hormone treatment on the market in the U.S. or Europe. TransCon hGH has orphan designation for GHD in both the United States and Europe.

Ascendis Pharma plans to submit a Marketing Authorisation Application (MAA) for TransCon hGH to the European Medicines Agency in the third quarter, also for pediatric GHD. Additionally, the company expects to initiate a phase 3 trial with TransCon hGH in pediatric GHD in Japan in the fourth quarter, and a phase 3 trial is ongoing in Greater China through the company’s strategic investment in VISEN Pharmaceuticals.

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.

About Pediatric Growth Hormone Deficiency (GHD)

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 metabolic effects. For caregivers and patients, the treatment burden with daily injections is high, which may lead to poor adherence and reduced overall treatment outcomes.

About Ascendis Pharma A/S

Ascendis Pharma is applying its innovative platform technology to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of patients, science and passion, the company utilizes its TransCon™ technologies to create new and potentially best-in-class therapies.

Ascendis Pharma currently has a pipeline of three independent endocrinology rare disease product candidates in clinical development and is advancing oncology as its second therapeutic area of focus. The company continues to expand into additional therapeutic areas to address unmet patient needs.

Ascendis is headquartered in Copenhagen, Denmark, with additional offices in Heidelberg and Berlin, Germany, and in Palo Alto and Redwood City, California.

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SOURCE: Ascendis Pharma

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