Once-Weekly TransCon™ CNP Achieved Primary Efficacy Objective in ACcomplisH China Phase 2 Trial

SHANGHAI, Nov. 16, 2023 /PRNewswire/ -- VISEN Pharmaceuticals (VISEN), an innovative biopharmaceutical company focused on endocrine diseases, is pleased to announce the topline results from the ACcomplisH China Phase 2 Trial in children with achondroplasia (ACH) aged 2 to 10 years. In the trial, patients dosed with TransCon CNP at the 100 μg CNP/kg/week showed significantly higher annualized growth velocity (AGV) than placebo (1.180 cm/year, P=0.018) at Week 52.

The ACcomplisH China Phase 2 Trial was a randomized, double-blind, placebo-controlled clinical trial conducted across 6 investigational sites in China. The study aimed to evaluate the safety and efficacy of once-weekly TransCon CNP compared to placebo in children with ACH aged 2 to 10 years old. The study enrolled 24 pediatric patients with ACH from China, with two dose cohorts of 50 µg CNP/kg/week and 100 µg CNP/kg/week, with 12 children for each cohort. The top-line results of the primary efficacy endpoint, annualized growth velocity (AGV) at Week 52, demonstrated a greater AGV of 5.939 cm/year for the cohort dosed at 100 µg CNP/kg/week, compared to 4.760 cm/year for placebo. TransCon CNP was generally safe and well tolerated.  Results from the prespecified analysis was consistent with Ascendis Pharma's global Phase 2 ACcomplisH study, supporting continued development at the selected dose of 100 μg/kg/week.^[1]

"Achondroplasia is the most common genetic form of skeletal dysplasia leading to not only severe disproportionate short stature, but is combined with a series of fatal, disabling and teratogenic clinical complications such as craniocervical junction stenosis, obstructive apnea, hydrocephalus, kyphosis, etc. ACH is relatively easily diagnosed, but there is currently no effective treatment in China. We shall be excited and thankful for ACH being included in the country's second catalog of rare diseases announced by the National Health Commission.^[2] This inclusion will greatly facilitate the introduction of innovative drugs and therapies into China." said Prof. Yu Yongguo, Executive Director of Clinical Genetics Center, Xinhua Hospital, Shanghai Jiao Tong University School of Medicine. "Positive top-line results from the ACcomplisH China Trial in Chinese patients with achondroplasia indicated the potential of TransCon CNP to transform the treatment landscape for achondroplasia. "

"VISEN joined the early phase global development of TransCon CNP in ACH, with the aim of bringing it as a potentially world leading disease-modifying therapy to China ACH patients at its first opportunity. We are thrilled that the China top-line results are consistent with Ascendis Pharma's global data. " said Mr. Pony LU, CEO of VISEN Pharmaceuticals. "It is a joint effort of patients, investigators, Ascendis and our dedicated teams and we thank everyone for their invaluable contributions to this important milestone."

VISEN Pharmaceuticals has exclusive rights to develop, manufacture and commercialize TransCon CNP in Greater China. The drug is owned and globally developed by Ascendis Pharma A/S, who is currently conducting a global randomized, double-blind, placebo-controlled pivotal Phase 3 trial of ~80 children ages 2–11 years with ACH, and who has recently initiated a Phase 2 clinical trial of TransCon CNP in infants aged 0-2 years with ACH.^[3][4] TransCon CNP received Orphan Designation and Orphan Drug Designation (ODD) by the European Commission and the United States Food and Drug Administration (U.S. FDA), respectively. Announced by the National Health Commission on September 20^th 2023, China's second catalog of rare diseases contains achondroplasia (ACH) within 86 rare diseases of 17 medical specialties, including hematology, dermatology and pediatrics.^[2] In recent years, NMPA has improved in both the number and speed of rare disease medicine approvals in China.^[5]

*About Achondroplasia*

Achondroplasia (ACH) is the most common form of skeletal dysplasia, affecting approximately 250,000 people worldwide. Individuals living with ACH may experience severe skeletal complications and comorbidities. For example, abnormal skeletal development can lead to sleep apnea, chronic back and leg pain from lower spine impingement and sudden infant death from cervical compression. Chronic ear infections due to abnormal eustachian tubes can lead to hearing loss and speech delay^[6].

The condition is caused by an autosomal dominant activating mutation in the fibroblast growth factor receptor 3 (FGFR3) gene that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways.  Preclinical and clinical data show that the CNP pathway stimulates growth. Increased CNP counteracts the effects of the FGFR3 mutation downstream, thus promoting bone growth^[7].

*About Ascendis Pharma A/S*

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

Please visit https://www.ascendispharma.com/ to learn more about Ascendis Pharma A/S and TransCon Technology.

Approval No.：COMU20231113/EN/PR

*About VISEN Pharmaceuticals*

VISEN is an innovative biopharmaceutical company focused on endocrine diseases. We are dedicated to providing innovative therapies and compassionate, patient-centric care, because we believe that achieving better treatment processes and outcomes results in living better lives. Putting patients' need first, VISEN is committed to providing first-in-class or best-in-class products and treatments for endocrine diseases. Our therapeutic areas cover endocrine diseases in adults and children, and rare endocrine diseases. VISEN comprises seasoned professionals with multinational pharmaceutical experiences and leverages cutting-edge technologies and leading resources across the world. We are focused on the Chinese market, and have established offices in Shanghai, Beijing, Hong Kong and Taipei. We have also launched our Greater China R&D and manufacturing site in Suzhou to enhance R&D, manufacturing and commercialization efforts. Our goal is to enable Chinese endocrine patients to benefit from the world's most advanced and reliable treatment solutions earlier.

For more information, please visit our website:
http://www.visenpharma.com/ 

Contact us
info@visenpharma.com 

References:

[1].  Ascendis Pharma: Once-Weekly TransCon™ CNP Achieved Primary Efficacy Objective, with Superiority at 100 µg/kg/week, in Ascendis Pharma's Phase 2 ACcomplisH Trial of Children with Achondroplasia Aged 2 to 10 Years https://investors.ascendispharma.com/news-releases/news-release-details/once-weekly-transcontm-cnp-achieved-primary-efficacy-objective 

[2].  China releases second catalog of rare diseases https://english.www.gov.cn/news/202309/21/content_WS650b7dc6c6d0868f4e8df9f1.html 

[3].  Ascendis Pharma Provides Update on Vision 3x3 Strategic Roadmap at 41st Annual J.P. Morgan Healthcare Conference https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-provides-update-vision-3x3-strategic-roadmap-0 

[4].  Ascendis Pharma Reports Second Quarter 2023 Financial Results

https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-pharma-reports-second-quarter-2023-financial-results

[5].  SCIO briefing on strengthening drug regulation to ensure drug safety for the public http://english.scio.gov.cn/pressroom/2023-07/28/content_95954492.htm 

[6].  Ireland PJ, Pacey V, et al. Appl Clin Genet. 2014;7:117–25.

[7].  Horton WA, et al. Lancet. 2007;370(9582):162–172.