更新于：2024-06-30
Lonapegsomatropin-tcgd
隆培促生长素
更新于：2024-06-30
概要
基本信息
最高研发阶段批准上市 |
首次获批日期 美国 (2021-08-25), |
最高研发阶段(中国)申请上市 |
特殊审评孤儿药 (美国)、孤儿药 (欧盟) |
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序列信息
Sequence Code 26401985
来源: *****
关联
14
项与 隆培促生长素 相关的临床试验A Phase 2, Open-Label, Single-Arm, 156-week Trial to Investigate the Efficacy, Safety and Tolerability of Combined Once Weekly Navepegritide and Lonapegsomatropin in Children With Achondroplasia
This proof-of-concept trial is being conducted to evaluate the efficacy, safety and tolerability of combination treatment with navepegritide and lonapegsomatropin administered as separate subcutaneous (SC) injections once weekly in children with achondroplasia (ACH) aged 2 to 11 years.
开始日期2024-06-01 |
A Prospective, Non-interventional (NIS), Long-term, Post-Authorisation Safety Study (PASS) of Patients Treated With Lonapegsomatropin
The goal of this study is to further characterise the potential long-term safety risks of lonapegsomatropin in patients treated with lonapegsomatropin under real-world conditions in the post-marketing setting.
开始日期2023-03-20 |
New InsiGHTS: A Multicenter, Phase 2, Randomized, Open-label, Active-controlled, Parallel Group Clinical Trial to Investigate the Safety, Tolerability, and Efficacy of Different Dose Levels of Once-weekly Lonapegsomatropin Compared to Daily Somatropin in Prepubertal Individuals With Turner Syndrome
A 104 week dose finding open label trial of lonapegsomatropin, a long-acting growth hormone product, administered once-a-week versus daily somatropin product in prepubertal individuals with Turner syndrome. Approximately 48 individuals (12 individuals per arm) will be randomized to receive one of three doses of lonapegsomatropin or a daily injection of somatropin. This is a trial that will be conducted in the United States.
开始日期2023-02-15 |
100 项与 隆培促生长素 相关的临床结果
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100 项与 隆培促生长素 相关的转化医学
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100 项与 隆培促生长素 相关的专利（医药）
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37
项与 隆培促生长素 相关的文献（医药）2023-01-01·Hormone research in paediatrics
Weekly Growth Hormone (Lonapegsomatropin) Causes Severe Transient Hyperglycemia in a Child with Obesity
作者: Estrada, Doris Elizabeth ; Majidi, Shideh ; Dauber, Andrew ; Alkhatib, Einas H
Introduction: A 12-year-and-9-month-old non-Hispanic black male with a history of growth hormone deficiency, pituitary hypoplasia, prediabetes, obesity, hypertension, and hyperlipidemia was initiated on weekly growth hormone (lonapegsomatropin-tcgd) and then transiently developed symptomatic hyperglycemia to 500 mg/dL. We aimed to describe this medication’s effect. Case Presentation: He was born full term and appropriate for gestational age. He was referred to endocrinology at 3.5 years of age for short stature with a height SDS of −2.48. IGF-1 51.1 ng/mL and IGFBP-3 1.2 ng/mL were low. GH stimulation test noted baseline and peak GH of 0.1 ng/mL. MRI brain showed hypoplastic adenohypophysis, aplastic pituitary stalk, and ectopic neurohypophysis. There had been difficulty with adherence to daily GH over the following 9 years. BMI trajectory rose above 180% of the 95th percentile. By age 12, A1c was 6.6%. Metformin was started and increased to 1,000 mg twice daily. Subsequent A1c was 6.0%. Due to poor compliance with daily GH, at 12 years and 9 months, he was initiated on 22 mg (0.25 mg/kg/week) of weekly lonapegsomatropin-tcgd to improve compliance. The day after his first injection, he developed non-bloody, non-bilious emesis. He denied headaches and endorsed polyuria. Due to concern for increased intracranial pressure, he was sent to the emergency department; however, ophthalmologic exam was negative. Initial serum glucose was 500 mg/dL, then 336 mg/dL after 1-L normal saline. Hemoglobin A1c was 5.7%, urine glucose 3+ mg/dL, and urine ketones 2+ mg/dL. Venous pH of 7.379 and bicarbonate of 20.6 mmol/L ruled out diabetic ketoacidosis. Metformin was held during the hospitalization. Hyperglycemia rapidly improved with transient insulin administration. He received one dose of glargine 20 units. He was initiated on lispro carb ratio of 1:8 and correction factor 1:15 for target glucose 150 mg/dL. By day four, glucoses were below 100 mg/dL; lispro was discontinued, and he was discharged home. Weekly GH was discontinued with plans to resume daily GH therapy in several months. Conclusion: Lonapegsomatropin-tcgd offers the convenience of weekly rather than daily GH treatment; however, this patient developed a rapid increase in insulin resistance and hyperglycemia requiring insulin. The discrepancy between the glucose of 500 mg/dL and A1c of 5.7%, along with the rapid resolution of hyperglycemia, is further consistent with a medication side effect. Close glucose monitoring of patients initiated on weekly growth hormone is crucial, particularly in those with a history of prediabetes.
2022-06-16·The Journal of clinical endocrinology and metabolism2区 · 医学
Safety and Efficacy of Lonapegsomatropin in Children With Growth Hormone Deficiency: enliGHten Trial 2-Year Results
2区 · 医学
Article
作者: Saenger, Paul ; Hofman, Paul L ; Mygind, Per Holse ; Song, Wenjie ; Aghajanova, Elena M ; Maniatis, Aristides K ; Chessler, Steven D ; Malievskiy, Oleg ; Nadgir, Ulhas M ; Mao, Meng ; Beckert, Michael ; Korpal-Szczyrska, Maria ; Chertock, Elena D ; Thornton, Paul S ; Shu, Aimee D ; Vlachopapadopoulou, Elpis ; Smith, Alden R ; Komirenko, Allison S ; Cappa, Marco ; Casella, Samuel J ; Chaychenko, Tetyana
Abstract:
Purpose:
The objectives of the ongoing, Phase 3, open-label extension trial enliGHten are to assess the long-term safety and efficacy of weekly administered long-acting growth hormone lonapegsomatropin in children with growth hormone deficiency.
Methods:
Eligible subjects completing a prior Phase 3 lonapegsomatropin parent trial (heiGHt or fliGHt) were invited to participate. All subjects were treated with lonapegsomatropin. Subjects in the United States switched to the TransCon hGH Auto-Injector when available. Endpoints were long-term safety, annualized height velocity, pharmacodynamics [insulin-like growth factor-1 SD score (SDS) values], and patient- and caregiver-reported assessments of convenience and tolerability.
Results:
Lonapegsomatropin treatment during enliGHten was associated with continued improvements in height SDS through week 104 in treatment-naïve subjects from the heiGHt trial (−2.89 to −1.37 for the lonapegsomatropin group; −3.0 to −1.52 for the daily somatropin group). Height SDS also continued to improve among switch subjects from the fliGHt trial (−1.42 at fliGHt baseline to −0.69 at week 78). After 104 weeks, the average bone age/chronological age ratio for each treatment group was 0.8 (0.1), showing only minimal advancement of bone age relative to chronological age with continued lonapegsomatropin treatment among heiGHt subjects. Fewer local tolerability reactions were reported with the TransCon hGH Auto-Injector compared with syringe/needle.
Conclusions:
Treatment with lonapegsomatropin continued to be safe and well-tolerated, with no new safety signals identified. Children treated with once-weekly lonapegsomatropin showed continued improvement of height SDS through the second year of therapy without excess advancement of bone age.
2022-04-19·The Journal of clinical endocrinology and metabolism2区 · 医学
Response to Letter to the Editor From Malozowski: “Weekly Lonapegsomatropin in Treatment-Naïve Children With Growth Hormone Deficiency: The Phase 3 heiGHt Trial”
2区 · 医学
Letter
作者: Karpf, David B ; Giorgadze, Elene ; Christoffersen, Eva Dam ; Maniatis, Aristides K ; Thornton, Paul S ; Breinholt, Vibeke Miller ; Korpal-Szczyrska, Maria ; Song, Wenjie ; Hofman, Paul L ; Beckert, Michael ; Chertok, Elena ; Lin, Zhengning ; Aghajanova, Elena ; Shu, Aimee D
120
项与 隆培促生长素 相关的新闻（医药）2024-05-30
Ascendis to Spotlight Latest Updates for TransCon™ Rare Endocrinology Disease Portfolio at ENDO 2024
COPENHAGEN, Denmark, May 30, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that it will host informational booths, events, and presentations showcasing use of its innovative TransCon technology in the areas of growth hormone deficiency (GHD), hypoparathyroidism, achondroplasia, and Turner syndrome during ENDO 2024, the annual meeting of the Endocrine Society being held June 1-4, 2024, in Boston. “In addition to physician and caregiver presentations in pediatric GHD, we look forward to our first in-person opportunity to share data from foresiGHt, our pivotal trial of TransCon hGH in adults with GHD,” said Aimee Shu, M.D., Ascendis Pharma’s Senior Vice President of Clinical Development, Endocrine Medical Sciences. “And with World Hypoparathyroidism Day coinciding with the first day of ENDO, we are pleased to host an informational booth focused on deepening physician awareness of the significant health and quality-of-life impacts of hypoparathyroidism.” Ascendis presentations during ENDO 2024 include: Oral PresentationResults of the foresiGHt Trial Support the Efficacy and Safety of Once-Weekly Lonapegsomatropin in Adults with Growth Hormone DeficiencyBeverly M.K. Biller, M.D., Massachusetts General Hospital and Harvard Medical School OR36-04 June 3, 2:45 – 3:00 p.m ET.Ballroom 210B– BCEC Product TheaterPerspectives on a Once-Weekly Growth Hormone for Pediatric Growth Hormone DeficiencyPhilippe Backlejauw, M.D., Cincinnati Children’s Hospital and University of Cincinnati College of MedicineMike Gordon, Parent of a child living with growth hormone deficiency S102-09June 2, 9:30 – 10:30 a.m. ETScience Theater Exhibit Hall – Theater #2 Poster Presentation:Baseline Demographics and Design of the SkybriGHt Registry Study. A US Multi-Center, Prospective, Non-Interventional, Long-Term Effectiveness and Safety Study of Children and Adolescents with Growth Hormone Deficiency Treated with LonapegsomatropinPhilippe Backlejauw, M.D., Cincinnati Children’s Hospital and University of Cincinnati College of Medicine Poster MON-198 June 3, 12:00 – 1:30 p.m. ETPoster Hall/Reception 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 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) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company and (ii) 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’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 7, 2024 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, and TransCon are trademarks owned by the Ascendis Pharma group. © May 2024 Ascendis Pharma A/S. Investor Contacts:Media Contact:Tim LeeMelinda BakerAscendis PharmaAscendis Pharma+1 (650) 374-6343+1 (650) 709-8875tle@ascendispharma.commedia@ascendispharma.comir@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com
2024-05-13
在医药行业里，一直流传着一个“双十定律”，即需要耗时10年，耗资10亿美金，才能将一款药品从研发做到商业化。也就是说，除了专注研发，找资金也是一项重要工作。而在A股IPO节奏阶段性收紧的情况下，许多医药企业纷纷将目光转向了港交所，本文统计了2024年以来递表港交所的8家生物医药企业，下面为这些企业的具体情况。1、上海细胞治疗集团股份有限公司4月30日，据港交所官网显示，上海细胞治疗集团股份有限公司（以下简称：上海细胞治疗集团）递交港交所上市申请。据招股书显示，上海细胞治疗集团是中国首家且唯一覆盖细胞医疗健康全价值链的公司，业务覆盖细胞储存、肿瘤医疗、细胞药物及细胞充能。2013年，上海细胞治疗集团成立，专注于细胞药物的研发；2015年，公司开始提供细胞储存服务，包括免疫细胞及干细胞储存；2020年，公司业务扩展至肿瘤医疗服务领域；2023年，公司进一步推出细胞充能产品。目前，公司的收入主要来源为细胞储存及相关服务。其中，在细胞药物研发方面，公司已确定及开发了7款在研产品。BZDS1901是全球首款聚焦实体瘤治疗的抗PD-1纳米抗体装甲化的CAR-T细胞药物。目前全球尚未有针对实体瘤的CAR-T细胞治疗产品获批，BZDS1901已从国家药监局获得治疗间皮素阳性实体瘤的I期和II期临床试验的IND批准，并已获得FDA的恶性间皮瘤孤儿药认证。2、华芢生物科技（青岛）股份有限公司4月29日，华芢生物向港交所递交招股书，拟在香港主板上市，这是公司第1次递交上市申请，联席保荐人为华泰国际和中信证券。2012年，华芢生物成立，其前身为北京中宏赛思生物技术有限公司，2024年4月1日第三次更名为现在的华芢生物。公司重点关注于医疗需求尚未得到满足且市场机会巨大的适应症的蛋白质药物，主攻方向则为发现、开发和商业化伤口癒合的多功能疗法，目前重点开发血小板衍生生长因子（“PDGF”）药物。成立至今，华芢生物累计获得了超4亿元的融资，投资方包括鼎晖投资、青岛高科等。2023年5月完成IPO前最后一轮融资后，其估值已经超33亿元。目前，公司尚未实现商业化。2023年，公司其他收入为47.4万元，净亏损为1.05亿元，研发开支3991.5万元。截至今年4月底，华芢生物的管线包括10款候选产品。其中7种PDGF候选产品，覆盖广泛的创面愈合适应症，包括烧烫伤、糖足、新鲜创面、压疮、放射性溃疡、干眼症、角膜损伤、日光性皮炎、脱发、痔疮及胃溃疡；1种mRNA候选产品针对实体瘤；2种ASO候选产品，分别针对脑胶质瘤及三阴性乳腺癌。其核心产品为Pro-101-1及Pro-101-2，是重组人血小板衍生生长因子-BB（「rhPDGF–BB」）药物。3、维昇药业3月21日，维昇药业向港交所主板递交了IPO申请。这已经是其第三次递表港交所了，此前该公司曾于2022年11月17日、2023年8月16日向港交所递交过上市申请。维昇药业成立于2018年，是一家处于研发后期、产品接近商业化的创新生物制药公司，聚焦开发新一代内分泌疗法，专注于为相关地区大量未被满足的医疗需求、同时具有医疗政策利好的特定内分泌疾病提供创新及潜在同类最佳及╱或同类首创治疗方案。在产品管线方面，维昇药业目前有3款候选药物，分别为隆培促生长素、TransCon CNP和帕罗培特立帕肽。其中，隆培生长激素是同类首创，其已分别于2021年及2022年获得FDA及EMA的上市批准，用于治疗儿童生长激素缺乏症（PGHD）的长效生长激素（LAGH）。今年3月，该药在中国的上市申请已获受理。而其他两款产品也已在全球范围内进入后期临床试验阶段。值得注意的是，这三款产品均从Ascendis引进。4、派格生物医药（苏州）股份有限公司2月23日，派格生物在港交所递交招股书，拟香港主板IPO上市。此前，公司曾于2021年8月向中国证监会提交科创板上市的申请，并反馈了两轮意见，但于2022年4月，公司自愿撤回A股上市申请。派格生物成立于2008年，是一家专注于自主研究及开发慢性病创新疗法的生物技术公司，重点关注代谢紊乱领域。据不完全统计，派格生物共完成9轮融资，累计从联想之星、君联资本、云锋基金、元生创投、建银国际等手中拿到超过13亿元，最后一轮（F+轮）融资后估值约40亿元人民币。自成立以来，派格生物尚未有产品实现商业化，至今公司收入为零。在产品管线方面，公司已自主开发1款核心产品及其他5款候选产品，适应症包括2型糖尿病（T2DM）、肥胖症、非酒精性脂肪性肝炎（NASH）、阿片类药物引起的便秘（OIC）、先天性高胰岛素血症等常见慢病及代谢疾病。其中，派格生物的核心产品PB-119为主要用于T2DM及肥胖症一线治疗的自主开发、接近商业化阶段的长效胰高血糖素样肽1（GLP-1）受体激动剂，在多项临床试验中显示出在血糖控制、心血管健康等方面的全面益处，以及对体重管理的良好效果。该产品在中国用于治疗T2DM的新药上市申请已于2023年9月获国家药监局受理。此外，公司临床阶段候选药物包括主要产品PB-718、PB-1902、PB-722，以及临床前阶段产品PB-2301、PB-2309。5、盛禾生物控股有限公司2月9日，盛禾生物递表港交所，拟主板IPO，中金公司为独家保荐人。此前，公司曾于2023年8月首次递表港交所，此次为公司第二次向港股市场发起冲击。据悉，公司已于4月15日通过港交所上市聆讯。盛禾生物成立于2018年，是一家专注于发现、开发和商业化用于治疗癌症和自身免疫性疾病的创新生物制剂的临床阶段生物制药公司。目前，公司已识别并开发9个管线产品，其中6个处于临床阶段，3个处于临床前阶段。其中，公司有3款核心产品，包括两个抗体细胞因子IAP0971、IAE0972，以及一个ADCC增强抗体IAH0968。前两者是基于公司的AICTM平台开发，后者则是基于公司的ADCC增强抗体平台（AEATM平台）开发。此外，正在开发的其他六种候选产品包括临床阶段产品IBB0979、IBC0966、IBD0333，临床前阶段产品IAN0982、ISH0988、ISH0613。6、北京华昊中天生物医药股份有限公司1月29日，华昊中天在港交所递交招股书，拟在香港IPO上市，建银国际、中信建投国际为其联席保荐人。此前，公司曾于2022年6月申报科创板上市，回复了两轮问询函后，于2023年5月撤回申请。华昊中天是一家合成生物学技术驱动的全球化生物医药公司，致力于开发肿瘤创新药。公司成功开发了专注于微生物代谢产物新药研发的三大核心技术平台，并建立了多元的产品组合，包括一款已商业化产品及由19个项目组成的管线。其中，公司的核心产品优替德隆注射液于2021年获得国家药监局批准上市，用于治疗复发或转移性晚期乳腺癌。截至最后实际可行日期，优替德隆注射液是唯一通过合成生物学技术开发并获批上市的化疗创新药，也是过去十几年来全球唯一获批准的具有新型分子结构的微管抑制剂类药物。此外，公司预期将于2025年在中国获得优替德隆胶囊用于治疗晚期乳腺癌的NDA批准，于2026年获得优替德隆注射液用于治疗晚期NSCLC治疗及乳腺癌新辅助治疗的NDA批准。7、北京康乐卫士生物技术股份有限公司1月29日，康乐卫士递表港交所，中信证券、建银国际为联席保荐人。此前，公司分别于2015年9月及2023年3月先后登陆新三板和北交所。若本次顺利登陆港交所，康乐卫士将成为国内首家“北+H”的两地上市企业。康乐卫士成立于2008年4月，是一家中国临床阶段的疫苗开发商。公司拥有全球最丰富的HPV（人乳头瘤病毒）疫苗产品组合，包括三价、九价、十五价HPV疫苗以及二价治疗性HPV疫苗，不过公司尚未有上市产品。具体来说，目前公司核心产品三价HPV疫苗、九价HPV疫苗（女性适应症）和九价HPV疫苗（男性适应症）在研项目均已进入临床Ⅲ期，十五价HPV疫苗已进入临床I期，呼吸道合胞病毒疫苗、带状疱疹疫苗、七价诺如病毒疫苗、四价手足口病疫苗、脊髓灰质炎疫苗和二价治疗性HPV疫苗等在研项目均处于临床前研究阶段。其中，三价HPV疫苗是公司最接近商业化的产品。截至2023年末，该款疫苗已完成第30个月访视，正在开展第36个月访视，预计2024年底前可提交BLA申请。此外，公司九价HPV疫苗（女性适应症）III期临床试验，进入病例监测随访阶段，正在开展第30和36个月访视，预计公司将于2025年向中国和印度尼西亚提交九价HPV疫苗（女性）的BLA，2027年提交九价HPV疫苗（男性）的BLA。目前，康乐卫士并无已商业化疫苗，往绩纪录期间内取得的少量收入来自销售用于研发的检测试剂取得。招股书显示，在过去的2022年和2023年前九个月，康乐卫士的营业收入分别为190.1万和160.1万元人民币，相应的净亏损分别为2.93亿和2.25亿元人民币。8、杭州九源基因工程股份有限公司1月22日，九源基因在港交所递交招股书，拟在香港IPO上市，独家保荐人为华泰国际。九源基因成立于1993年，主要从事生物药品及医疗器械的研发、生产及商业化，专注于骨科、代谢疾病、肿瘤、血液四大快速增长的治疗领域。截至2024年1月15日，九源基因已围绕四大治疗领域建立了多元化的产品组合，当中包括8款已上市产品，以及超过10款在研产品。其中，8款已上市产品包括骨科、肿瘤、血液领域的1款创新药械组合，2款生物制品及5款化学药品。主要在研产品则包括JY29-2（吉优泰®）、JY29-2（吉可亲®）及JY06（吉新芬®）。业绩方面，公司在过去的2021年、2022年和2023年前九个月，营业收入分别为13.07亿、11.25亿和10.23亿元人民币，相应的净利润分别为1.19亿、0.60亿和1.11亿元人民币。参考资料：[1]上海细胞治疗集团递表港交所，免疫细胞储存市场占有率第一.医药魔方Invest.2024-05-01.[2]上海细胞治疗集团IPO：细胞存储费用上万元，希望60%老百姓用得起？.洞察IPO.2024-05-10.[3]这个生物IPO，有个“隐忍”的创始人.预审IPO.2024-05-12.[4]派格生物-B，来自江苏苏州，递交IPO招股书，拟赴香港上市，中金独家保荐.瑞恩资本RyanbenCapital.2024-02-24.[5]「华昊中天-B」首次递表港交所，投后估值45亿元.活报告.2024-01-30.[6]华昊中天持续亏损毛利率骤降：销售费用曾一年近2亿，还沦为被执行人.港湾商业观察.2024-03-04.[7]九源基因，来自浙江杭州，递交IPO招股书，拟赴香港上市，华泰独家保荐.瑞恩资本RyanbenCapital.2024-01-23.[8]盛禾生物再递表：突击入股刚好达标？现金流持续为负.港湾商业观察.2024-03-05.[9]盛禾生物-B，来自浙江湖州，通过IPO聆讯，或很快香港上市，中金独家保荐.瑞恩资本RyanbenCapital.2024-04-16.[10]康乐卫士(833575)：核心产品进入临床III，商业化在即，有望成为国内首批HPV疫苗供应商.方正证券研究.2024-04-25.[11]港交所官网、各公司招股说明书等.识别微信二维码，添加生物制品圈小编，符合条件者即可加入生物制品微信群！请注明：姓名+研究方向！版权声明本公众号所有转载文章系出于传递更多信息之目的，且明确注明来源和作者，不希望被转载的媒体或个人可与我们联系(cbplib@163.com)，我们将立即进行删除处理。所有文章仅代表作者观点，不代表本站立场。
细胞疗法IPO免疫疗法孤儿药信使RNA
2024-05-02
— Rollout of YORVIPATH® initiated in Germany and Austria, with an estimated 55 doctors writing prescriptions and ~100 patients receiving commercial product as of March 31 — TransCon™ PTH (palopegteriparatide) PDUFA date of May 14, 2024, for adults with hypoparathyroidism. If approved, U.S. launch planned in Q3 — TransCon CNP (navepegritide) pivotal ApproaCH Trial on track for topline results in Q4 2024 — SKYTROFA® Q1 revenue more than doubled year-over-year to €65 million; Q1 operating expenses fell by 20% year-over-year to €137 million — Ascendis remains on track for achieving full-year 2024 SKYTROFA revenue of €320 to €340 million and full year 2024 operating expenses of €600 million — Conference call today at 4:30 pm ET COPENHAGEN, Denmark, May 02, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced financial results for the first quarter ended March 31, 2024, and provided a business update. “With SKYTROFA revenue more than doubling in the U.S. compared to the first quarter of 2023, and our successful initial launch of YORVIPATH in Germany and Austria, we believe Ascendis is on the path to achieving sustainable growth and operating cash flow breakeven on a quarterly basis by the end of 2024,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Our achievements this quarter give me further confidence that all elements are in place to deliver three independent Endocrinology Rare Disease blockbuster products and a strong pipeline in larger therapeutic areas such as Oncology, Ophthalmology, and Metabolic Diseases as outlined in Vision 2030.” Select Highlights & Anticipated 2024 Milestones TransCon hGH:(lonapegsomatropin, approved as SKYTROFA in the U.S., EU, European Economic Area (EEA) countries, and Great Britain) First quarter 2024 SKYTROFA revenue totaled €65 million, a 106% year over year increase with a steady quarter to quarter increase in treated patients.Full year 2024 SKYTROFA revenue expected to be €320 million to €340 million (based on average 2023 exchange rates).Plan to submit a supplemental Biologics License Application to FDA for adult growth hormone deficiency (GHD), in the third quarter of 2024.Topline results from Phase 2 trial in Turner syndrome expected in the fourth quarter of 2024. TransCon PTH:(palopegteriparatide, approved as YORVIPATH in the EU, EEA countries, and Great Britain) Commercial rollout of YORVIPATH continues in Germany and Austria with an estimated 55 doctors writing prescriptions and ~100 patients receiving commercial product as of March 31. First quarter YORVIPATH revenue totaled €1.5 million reflecting first two months of delivery to patients.In the U.S., Prescription Drug User Fee Act (PDUFA) date of May 14, 2024; if approved, U.S. commercial launch planned in the third quarter of 2024.Granted marketing authorization for the treatment of adults with chronic hypoparathyroidism and orphan drug status in Great Britain. TransCon CNP:(navepegritide) Topline data from pivotal ApproaCH Trial expected in the fourth quarter of 2024, and plan to submit a New Drug Application to FDA for children with achondroplasia (age 2-11 years) in the same quarter.Expect to initiate and complete enrollment in the combination TransCon hGH and TransCon CNP COACH trial of children with achondroplasia (ages 2-11) during the second quarter of 2024; topline Week 26 data expected in the fourth quarter of 2024. Oncology Programs New data from the ongoing Phase 1/2 IL-Believe Trial has been accepted for a poster presentation at the American Society for Clinical Oncology (ASCO) May 31-June 4. The presentation will provide initial data from the combination of TransCon IL-2 β/γ and TransCon TLR7/8 Agonists in patients with melanoma who have progressed on anti-PD1 therapy.During the fourth quarter of 2024, plan to provide a clinical update from the Phase 2 indication-specific, dose expansion cohorts from our TransCon IL-2 β/γ and TransCon TLR7/8 Agonist clinical trials. Financial Update and Outlook Based on Current Plans As of March 31, 2024, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €320 million compared to €399 million as of December 31, 2023.Full year 2024 SKYTROFA revenue expected to be €320 million to €340 million (based on average 2023 exchange rates).Expect total operating expenses (SG&A and R&D) of approximately €600 million for 2024.Expect to be operating cash flow breakeven on a quarterly basis by the end of 2024. First Quarter 2024 Financial ResultsTotal revenue for the first quarter of 2024 was €95.9 million compared to €33.6 million during the same period for 2023. The increase was primarily attributable to higher SKYTROFA revenue of €65.0 million compared to €31.6 million in the same period last year and non-cash license revenue of €24.8 million related to the license agreement with Eyconis in January 2024. Total Revenue (In EUR'000s) Three Months Ended March 31, 2024 2023 Revenue from external customers
Commercial sale of products 66,499 31,551 Licenses 24,770 614 Other 4,625 1,424 Total revenue from external customers 95,894 33,589
Research and development (R&D) costs for the first quarter of 2024 were €70.7 million compared to €106.1 million during the same period in 2023. The 33% decline was largely tied to lower external development costs for TransCon hGH, TransCon PTH (including a reversal of prior period write-downs of pre-launch inventories) and Oncology programs, partially offset by an increase in TransCon CNP costs. Selling, general, and administrative (SG&A) expenses for the first quarter of 2024 were €66.8 million compared to €66.5 million during the same period in 2023. Higher employee costs, including the impact from commercial expansion, partly offset by lower external pre-launch and administrative expenses. Total operating expenses for the first quarter of 2024 were €137.5 million compared to €172.7 million during the same period in 2023. Net finance expenses for the first quarter of 2024 were €73.6 million compared to a net finance income of €35.3 million during the same period in 2023. For the first quarter of 2024, Ascendis Pharma reported a net loss of €131.0 million, or €2.30 per share (basic and diluted) compared to a net loss of €110.9 million, or €1.98 per share (basic and diluted) for the same period in 2023. As of March 31, 2024, Ascendis Pharma had cash, cash equivalents, and marketable securities totaling €320.2 million compared to €399.4 million as of December 31, 2023. As of March 31, 2024, Ascendis Pharma had 58,224,419 ordinary shares outstanding, including 881,730 ordinary shares represented by ADSs held by the company. Conference Call and Webcast InformationAscendis Pharma will host a conference call and webcast today at 4:30 pm Eastern Time (ET) to discuss its first quarter 2024 financial 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 this section of the Ascendis Pharma website shortly after conclusion of the event for 30 days. 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 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 PTH’s PDUFA date of May 14, 2024, (ii) the commercial launch of TransCon PTH in the U.S., if approved, (iii) the timing of topline results from the ApproaCH Trial, (iv) Ascendis’ expectations regarding full year 2024 revenue for SKYTROFA, (v) Ascendis’ ability to fulfill its strategic goals to deliver three independent Endocrinology Rare Disease blockbuster products and a strong pipeline in larger therapeutic areas such as Oncology, Ophthalmology, and Metabolic Diseases, (vi) Ascendis’ plan to submit a supplemental Biologics License Application for SKYTROFA for adult GHD in the third quarter of 2024, (vii) the timing of topline results from the Phase 2 trial in Turner syndrome, (viii) the timing of topline data from the ApproaCH Trial, (ix) Ascendis’ plan to submit a New Drug Application for TransCon CNP for children with achondroplasia, (x) Ascendis’ ability to initiate and complete enrollment in the COACH Trial, (xi) the timing of topline data from Week 26 of the COACH Trial, (xii) Ascendis’ plan to present initial data from the Phase 1/2 IL-Believe Trial, (xiii) Ascendis’ plan to provide a clinical update from the Phase 2 portion of indication-specific, dose expansion cohorts of the IL Believe trial, (xiv) Ascendis’ expectations regarding its total operating expenses for 2024, (xv) Ascendis’ expectation that it will be operating cash flow breakeven on a quarterly basis by the end of 2024, (xvi) Ascendis’ belief that it is on a path to achieving sustainable growth and that all elements are in place to deliver three independent Endocrinology Rare Disease blockbuster products, (xvii) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (xviii) 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’ Annual Report on Form 20-F filed with the U.S. Securities and Exchange Commission (SEC) on February 7, 2024 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, TransCon, SKYTROFA®, and YORVIPATH® are trademarks owned by the Ascendis Pharma group. © May 2024 Ascendis Pharma A/S. Investor Contacts:Media Contact:Tim LeeMelinda BakerAscendis PharmaAscendis Pharma+1 (650) 374-6343+1 (650) 709-8875tle@ascendispharma.commedia@ascendispharma.comir@ascendispharma.com Patti Bank ICR Westwicke +1 (415) 513-1284 patti.bank@westwicke.com FINANCIAL TABLES FOLLOW Ascendis Pharma A/SConsolidated Statements of Profit or Loss and Comprehensive Income / (Loss)(In EUR'000s, except share and per share data)Three Months Ended March 31, 2024 2023 Consolidated Statement of Profit or Loss Revenue 95,894 33,589 Cost of sales 7,569 4,621 Gross profit 88,325 28,968 Research and development costs 70,687 106,114 Selling, general and administrative expenses 66,783 66,539 Operating profit/(loss) (49,145) (143,685)Share of profit/(loss) of associate (5,796) (1,227)Finance income 3,575 45,135 Finance expenses 77,161 9,840 Profit/(loss) before tax (128,527) (109,617)Income taxes/(expenses) (2,508) (1,297)Net profit/(loss) for the period (131,035) (110,914)Attributable to owners of the Company (131,035) (110,914)Basic and diluted earnings/(loss) per share€(2.30) €(1.98)Number of shares used for calculation (basic and diluted) 56,883,257 56,091,927
Consolidated Statement of Comprehensive Income or (Loss) Net profit/(loss) for the period (131,035) (110,914)Items that may be reclassified subsequently to profit or loss: Exchange differences on translating foreign operations 63 (787)Other comprehensive income/(loss) for the period, net of tax 63 (787)Total comprehensive income/(loss) for the period, net of tax (130,972) (111,701)Attributable to owners of the Company (130,972) (111,701) Ascendis Pharma A/SConsolidated Statements of Financial Position(In EUR'000s) March 31, 2024 December 31, 2023Assets Non-current assets Intangible assets 4,301 4,419 Property, plant and equipment 107,164 110,634 Investment in associates 24,797 5,686 Other receivables 2,129 2,127 138,391 122,866 Current assets Inventories 232,681 208,931 Trade receivables 41,092 35,874 Income tax receivables 742 802 Other receivables 26,857 19,097 Prepayments 42,502 38,578 Marketable securities — 7,275 Cash and cash equivalents 320,239 392,164 664,113 702,721 Total assets 802,504 825,587
Equity and liabilities Equity Share capital 7,818 7,749 Distributable equity (245,997) (153,446)Total equity (238,179) (145,697) Non-current liabilities Borrowings 229,627 222,996 Contract liabilities 5,000 5,949 Deferred tax liabilities 7,085 5,830 241,712 234,775 Current liabilities Convertible notes, matures in April 2028 Borrowings 424,984 407,095 Derivative liabilities 197,291 143,296 622,275 550,391 Other current liabilities Borrowings 14,403 14,174 Contract liabilities 1,183 1,184 Trade payables and accrued expenses 94,526 94,566 Other liabilities 22,698 41,176 Income tax payables 3,336 2,299 Provisions 40,550 32,719 176,696 186,118 798,971 736,509 Total liabilities 1,040,683 971,284 Total equity and liabilities 802,504 825,587
上市批准临床2期孤儿药
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研发状态
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| 生长激素缺乏症 | 美国 | 2021-08-25 |
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| 适应症 | 最高研发状态 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|---|
| 垂体性侏儒症 | 临床3期 | 中国 | 2019-12-30 | |
| 激素缺乏症 | 临床3期 | 加拿大 | 2017-11-13 | |
| 软骨发育不全 | 临床2期 | 丹麦 | 2024-06-01 | |
| 软骨发育不全 | 临床2期 | 爱尔兰 | 2024-06-01 | |
| 软骨发育不全 | 临床2期 | 英国 | 2024-06-01 | |
| 特纳综合症 | 临床2期 | 美国 | - |
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| 研究 | 分期 | 人群特征 | 评价人数 | 分组 | 结果 | 评价 | 发布日期 |
|---|
临床3期 | 298 | 齋築鹹遞鑰鹽選衊醖淵(夢膚蓋糧觸鹹糧顧鑰壓) = 築積願範齋積築鏇憲壓 鑰網鑰鏇膚願膚廠醖憲 (窪網構齋築繭鑰觸顧衊, 窪餘壓壓憲簾積艱顧獵 ~ 願範鏇夢鹹積願構餘齋) 更多 | - | 2024-05-08 | |||
临床3期 | 259 | 構鏇觸餘獵壓餘衊糧憲(選觸觸繭衊鹹糧齋顧醖) = 製獵築壓鹽憲襯繭構選 壓網醖醖鏇淵願觸鹽製 (鹹壓鬱憲遞積鏇鹽鹹餘 ) 更多 | 积极 | 2023-12-19 | |||
Placebo | 構鏇觸餘獵壓餘衊糧憲(選觸觸繭衊鹹糧齋顧醖) = 醖廠鬱夢繭簾齋醖糧窪 壓網醖醖鏇淵願觸鹽製 (鹹壓鬱憲遞積鏇鹽鹹餘 ) 更多 | ||||||
N/A | 81 | 壓鑰襯範顧窪夢夢鏇淵(醖積壓膚選簾鬱獵積範) = The most commonly reported adverse events over the course of the trial were categorized as infections, injury, and respiratory/thoracic/medical disorders. The majority of adverse events were mild in severity and unrelated to treatment. No adverse events led to discontinuation of the study treatment. 窪鑰願夢選糧齋襯艱鬱 (醖鹹鹹選選淵襯夢選選 ) | 积极 | 2023-09-23 | |||
(at the beginning of the open-label extension trial) | |||||||
临床3期 | 298 | 餘壓製廠襯繭齋範鹹簾(觸繭蓋鑰廠糧繭壓壓鹹) = 範衊簾艱憲夢築選壓選 築選簾獵糧鬱襯構廠鹹 (構製範淵獵廠醖糧鬱鑰, 6) | - | 2023-09-21 | |||
临床3期 | 150 | (Longpei growth promoting hormone group) | 積蓋膚鑰膚構顧簾選襯(鬱餘艱醖窪糧鬱廠餘製) = 願築襯範餘醖艱鹽鏇窪 網憲遞糧餘構觸夢膚窪 (艱範淵艱繭淵鬱觸齋蓋 ) | 积极 | 2022-11-17 | ||
(growth hormone daily preparation group) | 積蓋膚鑰膚構顧簾選襯(鬱餘艱醖窪糧鬱廠餘製) = 齋鬱構鹹糧衊襯醖網廠 網憲遞糧餘構觸夢膚窪 (艱範淵艱繭淵鬱觸齋蓋 ) | ||||||
临床3期 | 146 | 鏇構鬱構鑰鹹遞夢淵觸(夢獵鹽簾積醖餘選鑰願) = 廠淵膚簾選鑰顧網願鏇 蓋顧餘構壓衊餘構襯壓 (蓋壓鏇膚選鹹壓衊窪艱, 艱鬱襯艱蓋襯網簾鹽鏇 ~ 淵鏇觸蓋壓鬱鏇願艱糧) 更多 | - | 2022-01-04 | |||
临床3期 | 161 | 構壓網遞餘獵鬱積鹽鹽(簾範夢蓋壓製觸願蓋蓋) = 選窪衊壓鹽艱衊壓憲繭 鬱鬱遞窪構鑰鹽築糧鹹 (廠膚憲廠齋憲鹹襯構網, 0.2) 更多 | 优效 | 2021-10-21 | |||
構壓網遞餘獵鬱積鹽鹽(簾範夢蓋壓製觸願蓋蓋) = 觸窪餘衊鏇壓願鹹鏇願 鬱鬱遞窪構鑰鹽築糧鹹 (廠膚憲廠齋憲鹹襯構網, 0.3) 更多 | |||||||
临床3期 | 161 | TransCon hGH 0.24 mg hGH/kg/wk | 製餘繭夢積淵構醖獵襯(醖艱簾鑰願窪積範衊範) = 衊淵築鏇膚蓋壓夢夢遞 鏇顧遞鹹餘齋鬱鬱遞網 (蓋遞齋鬱膚繭選餘網鬱 ) | 积极 | 2019-09-19 | ||
製餘繭夢積淵構醖獵襯(醖艱簾鑰願窪積範衊範) = 窪構積蓋構憲願鏇醖鬱 鏇顧遞鹹餘齋鬱鬱遞網 (蓋遞齋鬱膚繭選餘網鬱 ) | |||||||
临床2期 | 53 | 艱蓋繭簾繭範顧齋繭壓(憲鏇願簾選範蓋憲網積) = 壓襯築膚鏇餘糧壓構襯 獵獵遞襯餘糧醖淵淵選 (醖積齋壓構膚範齋餘鹽 ) | 积极 | 2017-05-01 | |||
Genotropin | 艱蓋繭簾繭範顧齋繭壓(憲鏇願簾選範蓋憲網積) = 築醖齋齋願繭糧願繭鏇 獵獵遞襯餘糧醖淵淵選 (醖積齋壓構膚範齋餘鹽 ) | ||||||
临床2期 | 53 | (ACP-001, Dose-level 1) | 齋網遞遞壓製窪膚糧顧(廠觸範構願觸簾窪憲壓) = 蓋鹹衊夢願壓築淵簾糧 繭襯糧憲選餘齋鑰廠糧 (醖願鬱選衊鹹鑰選築壓, 廠廠觸鑰廠艱積範衊簾 ~ 醖製餘願鹽鹹鑰獵繭餘) 更多 | - | 2017-01-19 | ||
(ACP-001, Dose-level 2) | 齋網遞遞壓製窪膚糧顧(廠觸範構願觸簾窪憲壓) = 餘願醖鑰鑰壓築衊憲蓋 繭襯糧憲選餘齋鑰廠糧 (醖願鬱選衊鹹鑰選築壓, 願構鏇淵醖構壓蓋願糧 ~ 選築憲壓淵選憲製夢廠) 更多 |
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