Lonapegsomatropin-tcgd

自从2020年以来，关于“集采”的传闻，始终是萦绕在长春高新的投资者心头的一块心病。 因为手握生长激素这个百亿大单品，长春高新一度被称为“东北药茅”。 但是也正是因为过于依赖这款大单品，每当有生长激素“纳入集采”的小道消息开始传播，二级市场就干脆地以跌停作为反馈。 长春高新近年来跌停数据 作为曾经市值数千亿的医药白马股，这个股价稳定性，确实差了一点，长春高新投资者这几年的持股体验，想必也不会太好。 但是事实上，“集采”根本不是生长激素这个品类需要担心的事情，因为这款产品的主要支付来源是患者的自费。 2021年5月21日，长春高新在投资者电话沟通会议上表示： 目前公司生长激素大概30%以下在公立医院销售，70%以上在其他合作医疗机构销售；对公立医院的依赖在减弱，预计未来在民营医疗机构销售占比会进一步提升。 A股另一家生产生长激素的上市公司安科生物的公开披露数据也表明：公司生长激素产品在公立医院内销售比例不到30%，生长激素不属于急需采购的产品，该类产品超过90%来自患者自费。 究其原因，目前生长激素产品的主要使用者，并不是对症的“矮小症患者”。 大量的自费买单，来自希望给孩子增高的焦虑型家长。 无论院内集采与否，这部分自费的增高需求并不会因此而有太大的变化。 所以，尽管集采的传闻时有耳闻，部分省市也确实对生长激素采取了一定的限价措施，但是长春高新近年的营收还是保持了相对的稳定。 但是，这并不是说长春高新的生长激素这款大单品就没有任何的潜在危机。 目前，国内拥有生长激素水针剂型产品的只有长春高新、安科生物、诺和诺德三家，而长春高新拥有国内唯一获批上市的长效生长激素产品。 这种产品的独占态势，是长春高新大赚特赚的原因之一。 2022年，长春高新的生长激素产品收入突破100亿，2023年更是达到了110亿。 但是，这种“一家独大”的局面，很可能在近期就将被打破，这可能是长春高新真正的危机。 2024年1月，A股上市药企特宝生物公告，公司在研产品怡培生长激素的注册上市许可申请，于近日收到国家药品监督管理局下发的关于的《受理通知书》。 怡培生长激素注射液，是特宝生物自主研发的长效人生长激素，拟用于治疗内源性生长激素分泌不足所致的儿童生长障碍。 2024年3月，CDE官方网站消息显示，维昇药业长效生长激素——隆培促生长素的上市申请，已获得中国国家药品监督管理局药品审评中心正式受理，用于治疗儿童生长激素缺乏症。 隆培促生长素是首个在美国及欧洲获批上市的长效生长激素（每周注射一次），用于治疗儿童生长激素缺乏症。 2024年9月5日，CDE官网显示，诺和诺德的帕西长效生长素注射液的新药上市申请获得受理。 该药于2020年获批用于治疗成人生长激素缺乏症。2023年4月，FDA又批准其用于治疗2.5岁及以上儿童的生长激素缺乏症。 2024年12月，天境生物与济川药业联合宣布，伊坦长效重组人生长激素注射液治疗儿童生长激素缺乏症（PGHD）的生物制品上市申请，已获NMPA受理。 伊坦长效重组人生长激素，是国内申报阶段和已上市产品中首款且唯一一款融合蛋白长效生长激素。 长春高新长期垄断的长效生长激素市场，终于出现了同类竞品，而且一来就是4家。 与此同时，长春高新的生长激素板块，也已经出现了增长停滞的现象。 2024年前三季度，长春高新承载生长激素业务的核心子公司金赛药业实现收入81.63亿元，同比增长仅0.55%，是多年来最差的增速之一。 对于消费属性极强的生长激素业务来说，在消费萎靡的当下，出现增长乏力也是正常现象。 但是如果一下子再增加4个竞争对手来分蛋糕，那对于长春高新来说，想要保持现有的营收体量，无疑是一个不小的挑战。 从“一家独大”到“五王争霸”，相比于存在于传言中的“集采”，或许这才是真正的危机的开始。 医药研究报告分享营 收集与分享全市场主流医药研究报告 每天仅需0.8元 畅享超过2000篇存量精选医药研究报告 每年新增额外500篇

- Results for all three TransCon hGH starting dose cohorts, in first clinical trial of an indication outside of growth hormone deficiency, showed a safety and tolerability profile comparable to daily somatropin - Annualized height velocity was similar at Week 26 in once-weekly TransCon hGH-treated and daily somatropin-treated children COPENHAGEN, Denmark, Dec. 16, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced positive Week 26 topline results from New InsiGHTS, its Phase 2 randomized, open-label, active-controlled trial in the U.S. to investigate the safety, tolerability, and efficacy of once-weekly TransCon hGH (lonapegsomatropin; approved for pediatric growth hormone deficiency) compared to daily somatropin in prepubertal children with Turner syndrome. New InsiGHTS randomized and dosed 49 children with Turner syndrome aged 1 to 10 years old into one of four treatment groups 1:1:1:1 – one of three starting doses of TransCon hGH (0.24, 0.30, or 0.36 mg/kg/week) or an active comparator of daily somatropin with a starting dose of 0.35 mg/kg/week. Doses were individualized based on IGF-1. On the primary endpoint of annualized height velocity and secondary endpoint of change from baseline in height SDS, children treated with TransCon hGH demonstrated improved growth similar to daily somatropin at Week 26, independent of starting dose.In the trial, TransCon hGH was generally safe and well tolerated, with no discontinuations related to study drug and with comparable safety and tolerability to daily somatropin. “Week 26 topline data from the New InsiGHTS Trial showed that once-weekly TransCon hGH provided comparable safety and endocrine benefits to those seen with the established use of daily somatropin in Turner syndrome,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “These positive results lay the foundation for our planned basket trial in 2025 to support label expansion.” Ascendis expects Week 52 results for lonapegsomatropin-treated children compared to daily somatropin-treated children in 2025. About Turner SyndromeTurner syndrome is the most common congenital sex chromosomal condition in females, with an estimated prevalence of 1 out of every 2,000 to 2,500 live female births. Short stature, associated with short stature homeobox-containing gene (SHOX) haploinsufficiency, is the most common clinical feature of Turner syndrome. Clinical manifestations of Turner syndrome affect multiple organ systems and are associated with significant and potentially life-threatening complications including cardiovascular disease, ovarian dysfunction, endocrine disease, renal malformation, liver disease, sensorineural hearing loss, and varied neuropsychological manifestations. About Ascendis Pharma A/SAscendis Pharma is applying its innovative TransCon technology platform to build a leading, fully integrated biopharma company focused on making a meaningful difference in patients’ lives. Guided by its core values of Patients, Science, and Passion, Ascendis uses its TransCon technologies to create new and potentially best-in-class therapies. 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 StatementsThis 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) the timing of Week 52 results for lonapegsomatropin-treated patients compared to daily somatropin-treated patients, (ii) Ascendis’ plans for a basket trial in 2025 and SKYTROFA’s potential label expansion, (iii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (iv) Ascendis’ use of its TransCon technologies to create new and potentially best-in-class therapies. 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. 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’ prospectus supplement filed on September 20, 2024 and Ascendis’ current and future reports filed with, or submitted to, the U.S. Securities and Exchange Commission (SEC), including its Annual Report on Form 20-F filed with the SEC on February 7, 2024. 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, and TransCon are trademarks owned by the Ascendis Pharma group. © December 2024 Ascendis Pharma A/S. Investor Contacts:Media Contact:Scott SmithMelinda BakerAscendis PharmaAscendis Pharmair@ascendispharma.com+1 (650) 709-8875 media@ascendispharma.comPatti Bank ICR Healthcare +1 (415) 513-1284 patti.bank@icrhealthcare.com