Ascendis Pharma A/S

— Data demonstrated multiple clinical benefits beyond linear growth —  NDA supported by data from three randomized, double-blind, placebo-controlled clinical trials in children with achondroplasia, with up to three years of open-label extension data —  MAA in EU on track for submission during Q3 2025 COPENHAGEN, Denmark I March 31, 2025 I Ascendis Pharma A/S (Nasdaq: ASND) today announced that it has submitted its New Drug Application (NDA) to the U.S. Food & Drug Administration (FDA) for TransCon CNP (navepegritide) for the treatment of children with achondroplasia. TransCon CNP is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly and designed to treat individuals with achondroplasia by providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle. The filing is based on data from three randomized, double-blind, placebo-controlled clinical trials and up to three years of open-label extension data, including results from the pivotal ApproaCH Trial of children with achondroplasia. “We are pleased to share clinical data with the FDA demonstrating that, in addition to increased growth velocity, treatment with TransCon CNP was associated with reduced health-related burden, stronger muscle function, and straightening of abnormal leg bowing for the majority of treated children,” said Aimee Shu, M.D., Executive Vice President and Chief Medical Officer at Ascendis Pharma. “In addition to once-weekly administration, these outcomes and a safety and tolerability profile comparable to placebo support TransCon CNP’s potential to be recognized as a best-in-class treatment for achondroplasia.” Ascendis is on track to submit its Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) during Q3 2025. About Achondroplasia Achondroplasia is a rare genetic condition arising from a systemic fibroblast growth factor receptor 3 (FGFR3) variant that leads to an imbalance in the effects of the FGFR3 and CNP signaling pathways, estimated to affect more than 250,000 people worldwide. While historically considered a bone growth disorder, the FGFR3 variant seen in achondroplasia is expressed in tissues throughout the body, causing serious muscular, neurological, and cardiorespiratory complications in addition to skeletal dysplasia. Medical complications of achondroplasia vary across different stages of life. Throughout infancy and childhood, observed complications include spinal deformities, enlarged brain ventricles, impaired muscle strength and stamina, hearing deficits and chronic ear infections, upper airway obstructions, sleep-disordered breathing, hip problems, leg bowing, and chronic pain; many of these persist or worsen in adulthood. These medical complications can have detrimental effects on quality of life, physical functioning, and psychosocial function. Individuals with achondroplasia often require multiple surgeries and procedures to alleviate the condition’s many complications. About Ascendis Pharma A/S Ascendis Pharma is a global biopharmaceutical company focused on applying our innovative TransCon technology platform to make a meaningful difference for patients. Guided by our core values of Patients, Science, and Passion, and following our algorithm for product innovation, we apply TransCon to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis is headquartered in Copenhagen, Denmark and has additional facilities in Europe and the United States. Please visit ascendispharma.com to learn more. SOURCE: Ascendis Pharma

Visen Pharmaceuticals is designed to develop and commercialize Ascendis’ endocrinology therapies in greater China. Endocrine-disease-focused Visen Pharmaceuticals is making its public market debut in Hong Kong, filing papers for an $86.5 million IPO.The China-based biotech was formed in 2018 by Ascendis Pharma and an investor syndicate led by Vivo Capital. The biotech is designed to develop and commercialize Ascendis’ endocrinology therapies in greater China.Now, Visen is going public, with expected gross proceeds of $100 million, according to March 20 regulatory filings (PDF). The company has previously raised about $190 million, closing a $150 million series B financing in early 2021. That fundraise included OrbiMed, Logos Capital, Ascendis Pharma, Vivo Capital and Sofinnova Investments, among others.The company hosts three programs in its pipeline: Yorvipath (palopegteriparatide) for adults with chronic hypoparathyroidism; Skytrofa (lonapegsomatropin) for pediatric growth hormone deficiency; and navepegritide, an investigational prodrug of C-type natriuretic peptide for achondroplasia.   

Visen Pharmaceuticals, a biotech developing much of Ascendis Pharma’s endocrine-related disease pipeline for the China market, will go public on the Hong Kong Stock Exchange on Friday. The company expects net proceeds of about 672 million HKD ($86.5 million) from the initial public offering, which will take place Friday local time or Thursday afternoon New York time, according to a regulatory filing . Investors in the IPO include AnkeBio, Suzhou Harvest, Vivo Capital and WuXi Biologics HealthCare Venture, among others, according to the filing. There have been few HKEX biopharma IPOs in recent quarters. A couple listing hopefuls have let their filings lapse after six months of no action, including Insilico Medicine, which went with a $110 million Series E instead. Duality Biologics, an ADC biotech partnered with multiple big-name drugmakers, let its HKEX IPO ambitions stall last month, but the Shanghai-based startup quickly refiled days later. Visen was approaching its own six-month deadline, having filed for the IPO last September. Ascendis formed Visen in 2018 and initially held onto 50% of the company. That stake gradually fell to about 40%, according to regulatory filings. Ascendis continued devoting capital to the joint venture, including an equity investment as part of Visen’s $150 million Series B in January 2021. Visen had raised about $190 million prior to the IPO, according to regulatory filings. It’s already used about 86% of those funds, per the documents. In addition to Ascendis, Visen’s other key pre-IPO investors are Vivo Capital (32.1%), Sofinnova Ventures (4.6%), HongShan Growth (3.6%), OrbiMed (1.6%) and Sherpa Healthcare Partners (1.6%), according to regulatory filings . Visen is working on assets licensed from Ascendis for pediatric growth hormone deficiency, achondroplasia and hypoparathyroidism. Ascendis has received European and FDA approvals for some of the medicines already. Denmark-based Ascendis has done other business development deals, including a Japan venture , spinning out its ophthalmology work and partnering with Novo Nordisk . Visen is led by CEO Pony Lu, who was previously president of Takeda’s China unit. He’s also been in leadership roles at Servier’s Taiwan, China and France units and AstraZeneca Taiwan.