Navepegritide Achieved Primary Efficacy Endpoint with Sustained Clinical Benefits in Chinese Children with Achondroplasia: 104 Weeks Topline Results in ACcomplisH China Phase 2 Trial

(Aug 15th, 2025, Guiyang, China) - VISEN Pharmaceuticals (VISEN, 2561.HK), an innovative biopharmaceutical company focused on endocrine diseases, announced the topline data of ACcomplisH China Phase 2 Trial as of the completion of 104-week study, including 52 weeks of double-blind period and 52 weeks of open label extension (OLE) period. Navepegritide (TransCon CNP）is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly and designed to provide sustained release and continuous exposure of active CNP. In the trial, children treated with 100μg CNP/kg/week demonstrated significantly higher annualized growth velocity (AGV). In the OLE period, all participants continuously received navepegritide 100μg CNP/kg/week until week 104, demonstrated statistically significant improvements in other growth parameters, including height Z-score and AGV change from baseline. These results were presented at the 24 th Chinese Society of Pediatric Endocrinology and Metabolism (CSPEM 2025) conference in Guiyang, China. The ACcomplisH China Phase 2 Trial is 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 navepegritide compared to placebo in children with ACH aged 2 to 10 years old. The study enrolled 24 children with achondroplasia (ACH) from China, with two dose cohorts of 50µg CNP/kg/week and 100µg CNP/kg/week (12 children in 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 100µg CNP/kg/week, compared to 4.760 cm/year for placebo. All participants enter into the OLE period and continuously received navepegritide 100μg CNP/kg/week until week 104. The ACH-specific height Z-score continuously improved from OLE baseline over 104 Weeks, with an improvement in LS mean ACH Height Z-score of 0.050 at OLE baseline to 0.199 at Week 104. All participants showed continued reduction in the upper-to-lower body segment ratio from OLE baseline. "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. [ 1 ] 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 navepegritide to transform the treatment landscape for achondroplasia. " "VISEN joined the early phase global development of navepegritide in ACH, with the aim of bringing it as a potentially world leading disease-modifying therapy to China ACH patients at its first opportunity. " said Mr. Pony LU, CEO of VISEN Pharmaceuticals. “The design of OLE in Accomplish China trial aims to generate long-term safety and efficacy data, to provide further clinical data of ACH treatment in Chinese population. We will try out best to combine the global data with local data, in order to accelerate the registration of navepegritide in China." *ACH height Z-score: The height of Achondroplasia patient is converted to the sex and age-standard score in Achondroplasia population. Approval No.:COMU20250813/EN/PR 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 [ 5 ] . 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 [ 6 ] . 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. About VISEN Pharmaceuticals (2561.HK) 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 - have set up HQs in Suzhou and established offices in Shanghai, Beijing, Hong Kong and Taipei. Our goal is to enable Chinese endocrine patients to benefit from the world's most advanced and reliable treatment solutions earlier. On March 21st, 2025, VISEN officially listed on HKEX main board. For more information, please visit our website: http://www.visenpharma.com/ Contact us [email protected] References: [1]. China releases second catalog of rare diseases https://english.www.gov.cn/news/202309/21/content_WS650b7dc6c6d0868f4e8df9f1.html

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