VISEN Pharmaceuticals Announces the Successful Completion of China Phase 2 Trial of Navepegritide (TransCon CNP)

(May 12 th , 2025, Shanghai, China) - VISEN Pharmaceuticals (VISEN, 2561.HK), an innovative biopharmaceutical company focused on endocrine diseases, announced the successful completion of China Phase 2 Trial of Navepegritide (TransCon CNP) for achondroplasia (ACH). The trail is designed with 52-week double-blind period and 52-week open-label extension (OLE) period. Its results have been submitted to the drug registration platform of China's National Medical Products Administration (NMPA). The China Phase 2 Trial of Navepegritide focused on children with ACH aged 2 to 10 years old and was administered via once-weekly subcutaneous injections, aiming to provide active CNP at a safe therapeutic level. Results from the double-blind phase showed that 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, with good overall safety and tolerability. Results from the prespecified analysis was consistent with Ascendis Pharma's global phase 2 study [1] . Notably, a 52-week open-label extension (OLE) period was included after the double-blind period to ensure continued treatment. The active participation of patients and their families provided valuable data for the trial, which is crucial for drug approval and launch, and offers important references for future ACH research and treatment. Announced by the National Health Commission on September 20 th , 2023, China’s second catalog of rare diseases includes 86 rare diseases, among which is achondroplasia (ACH) [2] . The prevalence of ACH in China was 51.2 thousand, according to Frost & Sullivan, among whom around 10 thousand are treatable children. In recent years, the government has shown increasing attention to rare diseases, with the NMPA enhancing its evaluation and approval of domestic rare disease drugs, resulting in a significant increase in both the number and speed of approvals [3] . On March 31 st , 2025, Ascendis Pharma A/S, a partner of VISEN, officially submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) for Navepegritide [4] . VISEN will continue to strive for early access of safe and effective innovative treatments to children with ACH, committed to enhancing the quality of life for these children. *VISEN Pharmaceuticals has exclusive rights to develop, manufacture and commercialize TransCon CNP (Navepegritide) in Greater China. Approval No.：COMU2025059/EN/PR 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 21 st , 2025, VISEN officially listed on HKEX main board. For more information, please visit our website: http://www.visenpharma.com/ Contact us [email protected] References: 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 China releases second catalog of rare diseases https://english.www.gov.cn/news/202309/21/content_WS650b7dc6c6d0868f4e8df9f1.html 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 Ascendis Submits U.S. NDA for TransCon CNP (Navepegritide) for the Treatment of Children with Achondroplasia https://investors.ascendispharma.com/news-releases/news-release-details/ascendis-submits-us-nda-transcon-cnp-navepegritide-treatment

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