VISEN Pharma: TransConTM hGH Biologics License Application (BLA) to United States FDA was submitted

2020-06-30

– European marketing application will be submitted in the third quarter 2020 –

Shanghai, China, June 30, 2020--VISEN Pharma, developing and commercializing through multiple license agreements Ascendis Pharma’s endocrinology rare disease therapies in Greater China, hailed the move and pledged to accelerate the drug’s launch in China, as part of its mission to provide more Chinese patients quick access to the world’s advanced treatment solutions . Ascendis Pharma A/S (Nasdaq: ASND), a biopharmaceutical company that utilizes its innovative TransConTM technologies to address unmet medical needs, announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) on June 26th 2020, for TransConTM hGH (lonapegsomatropin), a long-acting once-weekly prodrug of somatropin (human growth hormone or hGH) for treatment for pediatric growth hormone deficiency (PGHD).

Currently, there is no approved long-acting growth hormone treatment on the market in the U.S. or Europe1. TransConTM hGH has orphan designation for GHD in both the United States and Europe. Ascendis Pharma plans to submit a Marketing Authorisation Application (MAA) for TransConTM 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 TransConTM 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 Pharma.

“TransConTM hGH applies ‘transient conjugation’ technology to release unmodified hGH in a predictable manner. It is designed to maintain the same mode of action as daily hGH therapies by releasing the same growth hormone molecule, somatropin.” Professor Luo Xiaoping, chairman of Department of Pediatrics, Tongji Hospital, Huazhong University of science and technology and vice chairman of pediatric branch of Chinese medicine, addressed, “In the global Phase 3 clinical trial, TransConTM hGH administrated once a week to GHD children for 52 weeks , the AHV for TransConTM hGH was significantly greater than the daily hGH. This result has brought the hope to further optimize the long-term treatment. Now, China Phase 3 clinical trial is ongoing, and we hope this innovative drug could bring new treatment option to Chinese GHD children.”

TransConTM hGH  is the first TransConTM technology product candidate taken from idea stage, through multiple Phase 3 clinical trials, and now to BLA submission. TransConTM technology has the potential to address major unmet medical needs, with multiple programs in clinical development for endocrinology rare disease product candidates, including TransConTM PTH for hypoparathyroidism and TransConTM CNP for achondroplasia.

"We are very pleased to witness the latest progress of TransConTM hGH, the submission of BLA to the U.S. FDA. Meanwhile, we are looking forward to the China Phase 3 clinical trial promising result. VISEN Pharm is trying to bring TransConTM hGH to the China market at an early date in hopes of providing a new treatment option to children who are suffering from GHD in China." Pony Lu, CEO of VISEN Pharma.

Reference:
1. Lal RA, et al. Arch Endocrinol Metab. 2019;63(6):601–607.

About TransConTM Technology
TransConTM refers to “transient conjugation.” The proprietary TransConTM platform is an innovative technology designed to create new therapies that optimize therapeutic effect, including efficacy, safety and dosing frequency. 

TransConTM 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. TransConTM 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, cognitive defect 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 VISEN Pharma

VISEN Pharmaceuticals is committed to the treatment of endocrine-related diseases, introducing the world’s leading treatment methods and drugs into the China market and hoping to provide more Chinese patients quick access to the world's most advanced and reliable treatment solutions.

In 2018, VISEN Pharmaceuticals (VISEN) was formed by Ascendis Pharma A/S (Nasdaq: ASND) and an investor syndicate led by Vivo Capital (along with participation by Sofinnova Ventures), to develop and commercialize Ascendis Pharma’s endocrinology rare disease therapies in Greater China, which includes mainland China, Hong Kong, Macau, and Taiwan.

Ascendis Pharma, headquartered in Copenhagen, Denmark with offices in Heidelberg, Germany and Palo Alto, California, is a leading fully integrated biopharmaceutical company that uses its innovative platform technology to make a real difference to patients’ lives. Guided by its core values of patients, science and passion, the company uses its TransConTM technologies to create new, potentially best-in-class therapies.

Founded in 1996, Vivo Capital is a global healthcare investment firm focused on investing in and building high quality companies. Vivo Capital is headquartered in Palo Alto, California, with offices in Beijing, Shanghai, Hong Kong, and Taipei.

VISEN Pharmaceuticals is headquartered in Shanghai, and its main business centers in mainland China are in Beijing and Shanghai.