VISEN’s TransConTM Human Growth Hormone Phase-III Clinical Trial in China Completes Target Patient Recruitment Number as Schedule

2021-03-30

 (March 30th, 2021, Shanghai, China) VISEN Pharmaceuticals, a biotech company committed to the treatment of endocrine-related diseases, announced today that it had successfully completed its recruiting target of 150 subjects for the Phase-III registration clinical trial in China to evaluate efficacy safety and tolerability of TransCon™ human growth hormone (hGH) weekly versus daily recombinant human growth hormone (rhGH) in Chinese prepubertal children with growth hormone deficiency (GHD). Initially delayed for four months due to the impact of COVID-19 pandemic, the recruitment was nonetheless completed by its original target date, gaining back valuable time for the new treatment option for pediatric growth hormone deficiency (PGHD) in China.


Pediatric growth hormone deficiency (PGHD) is a disease caused when the pituitary gland does not produce enough growth hormone. Children with PGHD are typically characterized by short stature with normal proportions, being unable to reach their midparental adult height. They also may experience metabolic abnormalities, psychosocial challenges, cognitive 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 abnormality. However, such a treatment approach can place a heavy burden on children with GHD, often leading to poor compliance and decreasing overall treatment efficacy.


This Phase-III clinical trial of TransCon hGH is focused on prepubertal children with GHD in China. The multicenter, randomized, open-label and parallel control study aims to demonstrate noninferiority in annualized height velocity (AHV) following weekly injections of TransCon hGH compared to once daily dosing of hGH after 52 weeks of treatment in prepubertal children with GHD.


 “Currently, a large number of children in China still suffer from growth hormone deficiency or other forms of lagging growth,” said Professor Luo Xiaoping, director of the Department of Pediatrics, Tongji Hospital, Huazhong University of Science and Technology and deputy director of Chinese Pediatric Society, Chinese Medical Association. “Many such children are unable to receive the proper diagnosis and treatment in time, causing significant negative effects that during last their entire lives. VISEN’s TransCon hGH utilizes ‘Transient Conjugation’ technology to release somatropin in a predictable manner, maintaining its original mode of action. In the heiGHt trial, which is used to support the FDA Biologics License Application (BLA), TransCon hGH (longapegsomatropin) showed superiority in AHV at Week 52 compared to daily somatropin, with comparable safety. The results of a two-year global clinical study presented at ENDO 2021 furthermore show that TransCon hGH can continuously improve the patients’ growth and demonstrated continued safety for two years. The sudden outbreak of COVID-19 pandemic last year meant that pediatric investigational sites were unable to carry out Phase-III study in China for more than four months. However, VISEN team worked together with each site to support them in achieving the goal of recruitment as scheduled, which is a very impressive achievement.”


 “The strong support of all researchers and experts involved in the project has been indispensable for the TransCon Phase-III clinical trial to achieve the goal of patient recruitment as scheduled,” said Pony Lu, CEO and board member of VISEN Pharmaceuticals. “The Investigational New Drug Application (IND) approval coincided with the outbreak of COVID-19, leaving the project blocked for four months. However, to enable Chinese patients to benefit from this international innovative treatment as early as possible, all participants in this project rose to the challenge, speeding up each segment of their work to meet the goal of recruitment according to the original timeline. This milestone signifies that the R&D of TransCon hGH in China has entered the next critical stage, and the data from this trial will serve as pivotal study for the upcoming BLA for TransCon hGH in China. VISEN will continue to work hard to ensure TransCon™ hGH is available to patients in China sooner, bringing a new treatment option to children with GHD.”


TransCon hGH has completed the pivotal Phase-III clinical study for PGHD in the United States and Europe, successfully submitted the Biologics License Application (BLA) for PGHD in the US in June 2020, and successfully submitted the Marketing Authorization Application (MAA) for PGHD in the European Union in September 2020.


About TransCon™ Technology


TransCon refers to “transient conjugation.” The TransCon platform is an innovative technology to create new therapies that are designed to potentially 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 is expected to be maintained. TransCon technology can be applied broadly to a protein, peptide or small molecule in multiple therapeutic areas, and can be used systemically or locally.


About VISEN Pharmaceuticals


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 was formed by Ascendis Pharma A/S (Nasdaq: ASND) and an investor syndicate led by Vivo Capital (along with participation by Sofinnova Investments). In January 2021, VISEN Pharmaceuticals completed series B financing, led by Sequoia China with participation from OrbiMed, Sherpa Healthcare Partners, Cormorant, HBM Healthcare Investments, Pivotal bioVenture Partners China, Logos Capital, and CDG Capital, to develop and commercialize endocrinology therapies in Greater China, which includes mainland China, Hong Kong, Macau, and Taiwan.