Lonapegsomatropin-tcgd

- For the TransCon CNP treatment-naïve cohort, combination treatment resulted in mean annualized growth velocity (AGV) of 9.14 cm/year, with an improvement in mean ACH height Z-score of +0.53 over 26 weeks - For the TransCon CNP-treated cohort, combination treatment resulted in mean AGV of 8.25 cm/year, with an improvement in mean ACH height Z-score of +0.44 over 26 weeks - The combination of TransCon hGH and TransCon CNP demonstrated accelerated improvement in body proportionality at Week 26, aligning with the increase in linear growth - Safety and tolerability data consistent with those observed for TransCon hGH and TransCon CNP monotherapies; combination treatment was generally well tolerated, with generally mild treatment-emergent adverse events (TEAEs) - Ascendis to host conference call today at 8:00 am ET COPENHAGEN, Denmark, June 09, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced Week 26 interim analysis results from its ongoing COACH Trial, the first clinical trial to evaluate combination treatment with once-weekly investigational TransCon CNP (navepegritide) and once-weekly TransCon hGH (lonapegsomatropin) in children with achondroplasia. Results demonstrated that TransCon hGH boosted treatment benefits of TransCon CNP, resulting in significant growth and proportionality improvements in children with achondroplasia after 26 weeks of combination treatment, with a safety and tolerability profile consistent with those observed for TransCon hGH and TransCon CNP monotherapies. TransCon CNP, which is under priority review as a monotherapy for children with achondroplasia by the U.S. Food & Drug Administration (FDA), is an investigational prodrug of C-type natriuretic peptide (CNP) administered once weekly, providing continuous exposure of active CNP to receptors on tissues throughout the body, including growth plates and skeletal muscle. TransCon hGH is a prodrug of somatropin administered once weekly, providing sustained release of active, unmodified somatropin. TransCon hGH is approved and marketed as SKYTROFA ® for the treatment of pediatric growth hormone deficiency and is in development for other indications. “TransCon CNP as a monotherapy has demonstrated the potential to transform the treatment of achondroplasia, and the COACH Trial at Week 26 demonstrates that TransCon hGH has the potential to boost treatment benefits of TransCon CNP with a safety profile consistent with monotherapies,” said Aimee Shu, M.D., Executive Vice President of Endocrine & Rare Disease Medical Sciences and Chief Medical Officer at Ascendis Pharma. “These results highlight the unique portfolio of once-weekly TransCon CNP and once-weekly TransCon hGH, with complementary modes of action, to improve the treatment landscape for growth disorders and physical functioning.” COACH Trial Design The COACH Trial is an ongoing proof-of-concept prospective Phase 2 open-label trial to investigate the efficacy, safety, and tolerability of combined treatment with once-weekly TransCon CNP at 100 µg/kg/week and once-weekly TransCon hGH at 0.30 mg/kg/week in children with achondroplasia aged 2 to 11 years. The trial included a cohort of TransCon CNP treatment naïve children (N=12, mean age 4.67 years) and a cohort of TransCon CNP-treated children (N=9, mean age 7.89 years) who had received TransCon CNP (100 µg/kg/week) for a mean of 2.56 years in clinical trials. The trial population is representative of children with achondroplasia, except for the observed growth benefit in the TransCon CNP-treated cohort. The interim analysis will be followed by Week 52 data, expected in Q4 2025, and Ascendis plans to initiate a Phase 3 trial in Q4 2025. Highlights of the Interim Topline Week 26 COACH Trial Results For TransCon CNP treatment-naïve children, mean annualized growth velocity (AGV) was 9.14 cm/year, representing an increase from baseline at Week 26 of 4.23 cm/year, with an improvement in mean ACH height Z-score of +0.53 over 26 weeks. For TransCon CNP-treated children, mean AGV was 8.25 cm/year, representing an increase from baseline at Week 26 of 3.10 cm/year, with an improvement in mean ACH height Z-score of +0.44 over 26 weeks. Mean AGV with TransCon CNP and TransCon hGH combination treatment exceeded the 97th percentile of average-stature children. Children treated with TransCon hGH and TransCon CNP demonstrated accelerated improvement in body proportionality at Week 26, aligning with the increase in linear growth. Bone age advanced in line with chronologic age. Safety and tolerability data were consistent with those observed for TransCon hGH and TransCon CNP monotherapies; combination treatment was generally well tolerated, with generally mild TEAEs. A slide presentation with these data can be found on the Investor Relations & News section of the Ascendis Pharma website: https://investors.ascendispharma.com . Conference Call and Webcast Information Ascendis Pharma will host a conference call and webcast today at 8:00 am Eastern Time (ET) to discuss these results. Those who would like to participate may access the live webcast here , or register in advance for the teleconference here . The link to the live webcast will also be available on the Investors & News section of the Ascendis Pharma website at https://investors.ascendispharma.com . A replay of the webcast will be available on that page shortly after the conclusion of the event for 30 days. 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. Forward-Looking Statements This press release contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this press release regarding Ascendis’ future operations, plans and objectives of management are forward-looking statements. Examples of such statements include, but are not limited to, statements relating to (i) TransCon CNP’s potential to transform the treatment of achondroplasia, (ii) TransCon hGH’s potential to boost treatment benefits of TransCon CNP with a safety profile consistent with monotherapies, (iii) the potential for the combination of TransCon CNP and TransCon hGH to improve the treatment landscape for growth disorders and physical functioning, (iv) the expected timing of Week 52 data from the COACH Trial and Ascendis’ plans to initiate a Phase 3 trial in Q4 2025, (v) Ascendis’ ability to apply its TransCon technology platform to make a meaningful difference for patients, and (vi) Ascendis’ application of its TransCon technologies to develop new therapies that demonstrate best-in-class potential to address unmet medical needs. Ascendis may not actually achieve the plans, carry out the intentions or meet the expectations or projections disclosed in the forward-looking statements and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions, expectations and projections disclosed in the forward-looking statements. Various important factors could cause actual results or events to differ materially from the forward-looking statements that Ascendis makes, including the following: dependence on third party manufacturers, distributors and service providers for Ascendis’ products and product candidates; unforeseen safety or efficacy results in Ascendis’ development programs or on-market products; unforeseen expenses related to commercialization of any approved Ascendis products; unforeseen expenses related to Ascendis’ development programs; unforeseen selling, general and administrative expenses, other research and development expenses and Ascendis’ business generally; delays in the development of its programs related to manufacturing, regulatory requirements, speed of patient recruitment or other unforeseen delays; Ascendis’ ability to obtain additional funding, if needed, to support its business activities; the impact of international economic, political, legal, compliance, social and business factors, including tariffs and trade policies. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking statements, as well as risks relating to Ascendis’ business in general, see Ascendis’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 12, 2025, and Ascendis’ other future reports filed with, or submitted to, the SEC. Forward-looking statements do not reflect the potential impact of any future licensing, collaborations, acquisitions, mergers, dispositions, joint ventures, or investments that Ascendis may enter into or make. Ascendis does not assume any obligation to update any forward-looking statements, except as required by law. Ascendis, Ascendis Pharma, the Ascendis Pharma logo, the company logo, SKYTROFA, and TransCon are trademarks owned by the Ascendis Pharma group. © June 2025 Ascendis Pharma A/S. Investor Contacts: Media Contact: Sarada Weerasinghe Melinda Baker Ascendis Pharma Ascendis Pharma ir@ascendispharma.com media@ascendispharma.com Patti Bank ICR Healthcare +1 (415) 513-1284 patti.bank@icrhealthcare.com Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

The microbial manufacturing site in Chengdu will be equipped with a 15,000L fermenter, enabling the production of 80 to 110 drug substance (DS) batches annually with expansion potential to 60,000L. It will house China's first dual-chamber lyophilization production line and a vial filling line, with an overall drug product (DP) manufacturing capacity exceeding 10 million vials annually. The new site will feature WuXi Biologics' recently launched microbial expression platform EffiXTM for the development and manufacturing of biologics with high yield, consistent quality, and superior stability and scalability. CHENGDU, China, June 9, 2025 /PRNewswire/ -- WuXi Biologics (2269.HK), a leading global Contract Research, Development and Manufacturing Organization (CRDMO), today announced that it has kicked off construction of its new microbial manufacturing site for commercial production in the Wenjiang district of Chengdu, a renowned, vibrant hub for pharmaceutical development. Spanning an area of 95,000 square meters, the new site is dedicated to a drug substance (DS) facility and a drug product (DP) facility for commercial manufacturing, encompassing a wide array of modalities, such as peptides,antibody fragments, plasmid DNA, enzymes, cytokines, and virus-like particles (VLPs). Scheduled to complete GMP release for production by the end of 2026, the site will be equipped with a 15,000L fermenter, enabling an annual production capacity of 80 to 110 DS batches. In the long run, capacity will be able to expand to 60,000L. Notably, the facility will house China's first dual-chamber lyophilization production line alongside a vial filling line, with overall DP manufacturing capacity exceeding 10 million vials annually, a significant enhancement of WuXi Biologics' commercial manufacturing capabilities for the global market. The state-of-the-art facilities will feature WuXi Biologics' recently launched microbial expression platform EffiXTM for the development and manufacturing of biologics with high yield, consistent quality, and superior stability and scalability, achieving titers that exceed 15 g/L for non-mAb recombinant proteins. Equipped with advanced automated systems to help ensure regulatory compliance, operational efficiency, and uncompromising quality, the facilities have also been designed with a strong focus on energy conservation and sustainability. Through the optimization of process development approaches and implementation of comprehensive carbon tracking mechanisms, the company aims to minimize its environmental footprint while maintaining high production standards. WuXi Biologics will utilize the manufacturing site in Chengdu to produce VISEN Pharmaceuticals' inaugural commercial product, Lonapegsomatropin, a long-acting growth hormone for the treatment of pediatric growth hormone deficiency (PGHD). In addition to this landmark partnership, the company has also established strategic partnership with industry-leading biotech firm Virogen Biotechnology. These collaborations — centered around commercial manufacturing operations — underscore WuXi Biologics' expanding portfolio of partnerships and its drive to meet the growing demand for advanced biologic therapies. Dr. Chris Chen, CEO of WuXi Biologics, commented, "We are excited to kick off the construction of Chengdu microbial manufacturing site, a powerful enhancement to add to our comprehensive end-to-end microbial solutions. With the booming global market for microbial products presenting unprecedented potential for next-generation therapies, our Chengdu site — with its robust production scale and cutting-edge technologies — is ideally positioned to capitalize on this trend. The strategic partnerships with leading pharmaceutical companies not only validate the capabilities of our new site but also represent a pivotal milestone in our unwavering commitment to accelerating the development and delivery of innovative therapies to patients worldwide." SOURCE WuXi Biologics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED