VISEN Pharmaceuticals announces China Phase 3 clinical study initiation for TransCon human growth hormone, the first unmodified long-acting growth hormone in China

SHANGHAI,Oct. 26,2019 --VISEN Pharmaceuticals,a company focused on the treatment of endocrine-related diseases,announced today the approval of a new drug (IND) application with the China National Drug Administration (NMPA) to initiate a phase 3 study for TransCon human growth hormone (TransCon hGH) with childhood growth hormone deficiency (GHD). TransCon hGH is expected to be the first long-acting growth hormone (LAGH) therapy in China that releases unmodified growth hormone,the same growth hormone used in daily therapies,with convenient once weekly dosing. Recently,the European Commission (EC) has granted Orphan Designation to TransCon hGH for pediatric growth hormone deficiency treatment [1]. Currently,there is no long acting growth hormone therapy available in the US or Europe.

TransCon hGH is the only long-acting growth hormone prodrug that uses innovative "Transient Conjugation" technology globally. The technology differs from the long-acting growth hormone analogues developed with other technologies,is designed to ensure the release of unmodified human growth hormone (hGH) at a predictable rate over one week,resulting in the same tissue distribution as daily growth hormone therapy. The pivotal global phase 3 study (heiGHt Trial) in patients with childhood growth hormone deficiency was completed and the top-line data were presented at the Endocrine Society Annual Meeting (ENDO 2019) in March,and recently at the European Society of Paediatric Endocrinology (ESPE) in Vienna. The annual height velocity (AHV) for TransCon hGH was greater compared to a daily hGH (the treatment difference was 0.86 cm/year between TransCon hGH and daily hGH,p value is 0.0088). The result suggests that this growth hormone may be highly differentiated,reflecting application of the TransCon technology.

GHD refers to the growth deficiency caused by impaired production or secretion of hGH. GHD seriously affects children's overall physical and mental health,with an incidence rate of about 1/10,000 ~ 1/1,000[2]. Children with isolated GHD performance are short in stature,slow to grow,and prone to hypoglycemia. The average adult height of untreated patients with growth hormone deficiency is 143 cm in men and 130 cm in women [3]. Children with GHD have normal body proportions,but small hands and feet and a small mid-face,excess subcutaneous truncal fat,decreased muscle mass,thin hair,a high-pitched voice,delayed puberty,and delayed bone and dental maturation. 5% of GHD pediatric subjects experience hypoglycemic episodes,with the latter decreasing in severity after growth hormone therapy [3]. GHD also influences cognitive functions and children's overall sense of well-being.

Patients with GHD often undergo thousands of daily subcutaneous injections over many years of therapy,resulting in a high treatment burden,poor treatment compliance,and fail to achieve expected treatment goal. According to literature reports,more than 70% of children with GHD have poor adherence to daily GH injections,which leads to sub-optimal efficacy in clinical practice [4] [5] [6]. Professor Luo Xiaoping from Tongji Hospital of Tongji Medical College,the leading principal investigator of the China Phase 3 clinical study,said,"a LAGH with less frequent dosing and treatment burden,superior efficacy with no additional safety concern and comparable to the daily GH are really clinical and patient's unmet needs.

According to the State Council's Healthy China Action (2019-2030) plan,the growth retardation rate for children under age 5 will be less than 7% by 2022 and 5% by 2030[7]. Pony Lu,CEO of VISEN Pharmaceuticals,"Meeting the unmet needs of clinical treatment for children with GHD is closely related to the target of Healthy China Action plan to reduce child growth retardation. As the company with the exclusive authorization to develop and commercialize TransCon hGH in Greater China,VISEN Pharmaceuticals will start from the China Phase 3 study and work closely with Chinese pediatric endocrinologists to speed up the launch of TransCon hGH in China. This world-leading treatment will help children with GHD in China achieve treatment expectation and bring benefit to them for a healthy future."

About TransCon™ Technology

TransCon refers to "transient conjugation." The proprietary TransCon platform is an innovative technology 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 pH and temperature conditions initiate the release of the active,unmodified parent drug in a predictable release 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 (VISEN) was formed by Ascendis Pharma A/S and an investor syndicate led by Vivo Capital,to develop,manufacture 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 biopharma company that uses 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 TransCon™ technologies to create new,potentially best-in-class therapies. Founded in 1996,Vivo Capital is a U.S. based healthcare investment firm focused on investing in and building high quality companies in the U.S. and Greater China.

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

References:

[1] https://ascendispharma.gcs-web.com/news-releases/news-release-details/ascendis-pharma-receives-orphan-designation-transcontm-hgh

[2] Guowei Lin,JiYao Wang,Junbo Ge,etc.,Practice of Internal Medicine,15th Edition

[3] Pediatric Growth Hormone Deficiency. (Accessed 8 March 2019,at https://emedicine.medscape.com/article/923688-overview.)

[4] Rosenfeld RG,Bakker B. Compliance and persistence in pediatric and adult patients receiving growth hormone therapy. Endocr Pract 2008;14:143 -54. (https://doi.org/10.4158/ep.14.2.143)

[5] Bang P,Bjerknes R,Dahlgren J,et al. A comparison of different definitions of growth response in short prepubertal children treated with growth hormone. Horm Res Paediatr 2011;75:335 -45. (https://doi.Org/10.1159/000322878)

[6] Cutfield WS,Derraik JGB,Gunn AJ,et al. Non-compliance with growth hormone treatment in children is common and impairs linear growth. PLoS ONE 2011,6(1): e16223

[7] State Council's Healthy China Action (2019-2030) plan (http://www.gov.cn/xinwen/2019-07/15/content_5409694.htm )