The 52-week data of the Phase 3 clinical trial of Longpei auxin China was first released

VISEN Pharmaceuticals, an innovative biopharmaceutical company focusing on endocrine-related therapeutics, announced for the first time on November 17 the main research data
of its phase 3 pivotal clinical trial of long-acting growth hormone (weekly formulation), lonapegsomatropin.
The trial targeted children with growth hormone deficiency (GHD) in China, and longpei auxin is a prodrug
given weekly and releasing unmodified growth hormone.

The results showed that after 52 weeks of treatment with Longpei auxin or growth hormone daily preparation, the annualized growth rate (AHV) was 10.
66 cm/year and 9.
75 cm/year, respectively (difference between groups 0.
91, 95% confidence interval: 0.
37 ~ 1.
45, p=0.
0010), which reached the primary endpoint of the study, that is, longpei auxin was not inferior to growth hormone daily preparation
.
Based on preset statistical tests, the analysis results of the primary endpoint also confirmed that longpei auxin was superior to daily GH preparations
.
Multiple preset sensitivity analyses confirmed the robustness
of the results.
Safety results showed that Longpei auxin was well tolerated and the safety profile was comparable
to that of daily preparations of growth hormone.

At present, the vast majority of growth hormone marketed in China is a short-acting preparation
injected daily.
Longpei auxin is a new long-acting growth hormone introduced from Ascendis Pharma based on the innovative "TransCon temporary connection" technology ^[1], which only needs to be injected once
a week.
After injection, the ligation structure automatically cleaves at a specific rate and releases unmodified growth hormone
in a controlled manner.
Wei Sheng's Phase 3 pivotal clinical trial is a randomized, open, positive controlled clinical trial for the treatment of growth hormone deficiency in children in China, led by Professor Luo Xiaoping, Vice Chairman of the Pediatrics Branch of the Chinese Medical Association and Director of the Department of Pediatrics, Tongji Hospital, Huazhong University of Science and Technology
, as the principal investigator.

Professor Luo Xiaoping further interpreted the main research data, saying: "From week 13, AHV was significantly higher in the growth hormone group than in the growth hormone daily preparation group
.
Compared with baseline, the standard deviation score (SDS) for height at week 52^[2] increased by 1.
01 and 0.
83 (p=0.
0015), respectively, in the growth hormone group and the growth hormone daily preparation group.
At week 13, the height SDS improvement was more obvious
in the growth hormone group.
During the 52-week treatment period, mean insulin-like growth factor-1 (IGF-1) SDS increased to the normal range in both groups and was relatively high
in the Longpei auxin group.
IGF-1 is a polypeptide produced by the liver in response to growth hormone, and its physiological role is mainly to stimulate chondrocytes proliferation, differentiation and collagen synthesis ^[3].

The enhanced/antagonistic system of GH-IGF enhances the anabolic effects of growth hormone while attenuating the potential adverse effects of growth hormone for gluconeogenesis and lipolysis ^[4].

"

Mr.
Anbang Lu, CEO and Executive Director of Veson Pharmaceutical, said: "According to the public data of the marketed products, Longpei auxin is currently the only long-acting product that has been proven to be effective compared to the daily preparation of growth hormone, and the key data of the 52-week phase 3 clinical trial in China announced today, 'head-to-head confirmed that once weekly growth hormone auxin is not inferior and effective in growth hormone daily preparation, and the safety profile is comparable
to that of daily preparation of growth hormone 。 This study is consistent with the results of the pivotal global phase 3 study (heiGHt study), showing that Longpei auxin has significant efficacy and good safety in the treatment of GHD children, and looks forward to providing a new long-acting growth hormone treatment option
for Chinese children.
"

The global clinical development of Longpei auxin outside Greater China is led by Ascendis Pharma A/S, which was approved by the US Food and Drug Administration (FDA) in August 2021 and the European Commission (EC) for childhood growth hormone deficiency in August 2021 and January 2022, respectively
.

[1] TransCon (Transient Conjugation) technology, which temporarily (transiently) connects
carrier molecules to biologically active prototype drugs through a unique linkage structure.
The technology platform is owned by Ascendis Pharma, which is exclusively licensed by Ascendis to develop, manufacture and commercialize
its endocrine disease solutions in Greater China.

[2] Height standard deviation integral (SDS) = ( Actual height - standard average height of the same age and sex ) ÷ standard deviation of height of the same age and sex

[3] Xue Xindong, Pediatrics, Eight-Year Program (Second Edition)[M].
Beijing:People's Medical Publishing House, 2010.
8:459-462

[4] Kaplan SA, Cohen P.
The somatomedin hypothesis 2007: 50 years later.
J Clin Endocrinol metab.
2007; 92(12):4529-4535.
doi:10.
1210/jc.
2007-0526