A Phase 3, Multicenter, Single Arm, Open-Label Trial, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Japanese Adult Subjects with Hypoparathyroidism - PaTHway Japan Trial

开始日期2021-11-02

申办/合作机构-

NCT05387070

/ Recruiting临床3期

PaTHway CHINA TRIAL: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Trial, With an Open-Label Extension, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Adults With Hypoparathyroidism

This study is limited to conduct in China only. The primary objective is to assess the treatment effect of daily TransCon PTH on serum calcium (sCa) levels within the normal range and stopping from therapeutic doses of active vitamin D (calcitriol) or active vitamin D analogue (alfacalcidol) and calcium at 26 weeks of treatment. All subjects will start with 18 mcg of study drug and will be individually and progressively titrated to an optimal dose over a 26-week double blind period, followed by an open label extension period up to 156 weeks. TransCon PTH or placebo will be administered as a subcutaneous injection using a pre-filled injection pen. Neither trial participants nor their doctors will know who has been assigned to each group. After the 26 weeks, participants will continue in the trial as part of a long-term extension study. During the extension, all participants will receive TransCon PTH, with the dose adjusted to their individual needs.

开始日期2021-07-28

申办/合作机构

维昇药业（上海）有限公司

NCT04701203

/ Active, not recruiting临床3期

PaTHway TRIAL: A Phase 3, Multicenter, Randomized, Double-Blind, Placebo-Controlled, Parallel Group Trial, With an Open-Label Extension, Investigating the Safety, Tolerability and Efficacy of TransCon PTH Administered Subcutaneously Daily in Adults With Hypoparathyroidism

During the first 26 weeks of the trial, participants will be randomly assigned to one of two groups: one group will receive TransCon PTH and one group will receive placebo. All subjects will start with a fixed dose of study drug and will be individually and progressively titrated to an optimal dose over a 10 week period, followed by an individualized dosing period up to 16 weeks. TransCon PTH or placebo will be administered as a subcutaneous injection using a pre-filled injection pen. Neither trial participants nor their doctors will know who has been assigned to each group. After the 26 weeks, participants will continue in the trial as part of a long-term extension study. During the extension, all participants will receive TransCon PTH, with the dose adjusted to their individual needs. This is a global trial that will be conducted in, but not limited to, the United States, Canada, Germany, and Denmark.

开始日期2021-02-16

申办/合作机构

Ascendis Pharma Bone Diseases A/S

100 项与帕罗培特立帕肽相关的临床结果

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100 项与帕罗培特立帕肽相关的转化医学

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100 项与帕罗培特立帕肽相关的专利（医药）

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项与帕罗培特立帕肽相关的文献（医药）

2025-01-01·Revue medicale suisse

Endocrinologie : ce qui a changé en 2024

Article

作者: Cruchon, Karim ; Papadakis, Georgios ; Giordani, Ilaria ; Ameti, Adelina ; Sykiotis, Gerasimos P

In this article, we look at a selection of recent developments in various areas of endocrinology. We focus on advances in endocrine pharmacotherapy and endocrine surgery, addressing several areas: a) the thyroid safety of Glucagon-Like Peptide-1 (GLP1) analogues; b) the efficacy of adrenal surgery for mild autonomous cortisol secretion; c) crinecerfont in the management of congenital adrenal hyperplasia in adults and children; d) paltusotin as a novel oral therapy for acromegaly and e) TransCon PTH (palopegteriparatide) as a novel therapy for chronic hypoparathyroidism.

2024-06-01·ADVANCES IN THERAPY

Article

作者: Tsourdi, Elena ; Imanishi, Yasuo ; Ominsky, Michael S ; Zhao, Carol ; Sprague, Stuart ; Lai, Bryant ; Sibley, Christopher T ; Palermo, Andrea ; Makita, Noriko ; Gosmanova, Elvira O ; Khan, Aliya A ; Rejnmark, Lars ; Rubin, Mishaela R ; Takeuchi, Yasuhiro ; Makara, Michael A ; Kohlmeier, Lynn ; Ukena, Jenny ; Gagnon, Claudia ; Shoback, Dolores M ; Schwarz, Peter ; Shu, Aimee D

INTRODUCTION:

Individuals with chronic hypoparathyroidism managed with conventional therapy (active vitamin D and calcium) have an increased risk for renal dysfunction versus age- and sex-matched controls. Treatments that replace the physiologic effects of parathyroid hormone (PTH) while reducing the need for conventional therapy may help prevent a decline in renal function in this population. This post hoc analysis examined the impact of palopegteriparatide treatment on renal function in adults with chronic hypoparathyroidism.

METHODS:

PaTHway is a phase 3 trial of palopegteriparatide in adults with chronic hypoparathyroidism that included a randomized, double-blind, placebo-controlled 26-week period followed by an ongoing 156-week open-label extension (OLE) period. Changes in renal function over 52 weeks (26 weeks blinded + 26 weeks OLE) were assessed using estimated glomerular filtration rate (eGFR). A subgroup analysis was performed with participants stratified by baseline eGFR < 60 or ≥ 60 mL/min/1.73 m².

RESULTS:

At week 52, over 95% (78/82) of participants remained enrolled in the OLE and of those, 86% maintained normocalcemia and 95% achieved independence from conventional therapy (no active vitamin D and ≤ 600 mg/day of calcium), with none requiring active vitamin D. Treatment with palopegteriparatide over 52 weeks resulted in a mean (SD) increase in eGFR of 9.3 (11.7) mL/min/1.73 m² from baseline (P < 0.0001) and 43% of participants had an increase ≥ 10 mL/min/1.73 m². In participants with baseline eGFR < 60 mL/min/1.73 m², 52 weeks of treatment with palopegteriparatide resulted in a mean (SD) increase of 11.5 (11.3) mL/min/1.73 m² (P < 0.001). One case of nephrolithiasis was reported for a participant in the placebo group during blinded treatment; none were reported through week 52 with palopegteriparatide.

CONCLUSION:

In this post hoc analysis of the PaTHway trial, palopegteriparatide treatment was associated with significantly improved eGFR at week 52 in addition to previously reported maintenance and normalization of serum and urine biochemistries. Further investigation of palopegteriparatide for the preservation of renal function in hypoparathyroidism is warranted.

TRIAL REGISTRATION:

ClinicalTrials.gov NCT04701203.

2024-04-30·Endocrinology and metabolism (Seoul, Korea)

Review

作者: Rejnmark, Lars

The conventional treatment of hypoparathyroidism (HypoPT) includes active vitamin D and calcium. Despite normalization of calcium levels, the conventional treatment is associated with fluctuations in calcium levels, hypercalciuria, renal impairment, and decreased quality of life (QoL). Replacement therapy with parathyroid hormone (PTH)(1-84) is an option in some countries. However, convincing beneficial effects have not been demonstrated, which may be due to the short duration of action of this treatment. Recently, palopegteriparatide (also known as TransCon PTH) has been marketed in Europe and is expected also to be approved in other countries. Palopegteriparatide is a prodrug with sustained release of PTH(1-34) designed to provide stable physiological PTH levels for 24 hours/day. A phase 3 study demonstrated maintenance of normocalcemia in patients with chronic HypoPT, with no need for conventional therapy. Furthermore, this treatment lowers urinary calcium and improves QoL. Another long-acting PTH analog with effects on the parathyroid hormone receptor (eneboparatide) is currently being tested in a phase 3 trial. Furthermore, the treatment of autosomal dominant hypocalcemia type 1 with a calcilytic (encaleret) is also being tested. All in all, improved treatment options are on the way that will likely take the treatment of HypoPT to the next level.

170

项与帕罗培特立帕肽相关的新闻（医药）

2025-01-14

·CPHI制药在线

闻药识重磅，2024年新药“钱景”几何？

关注并星标CPHI制药在线 2024 年，FDA共批准了 60 种新药（CDER批准50款，CBER10款），但真正能够实现 “九天阊阖开宫殿” 的新药可能只有寥寥数款。追求每年十亿美元以上销售额是大型制药公司孜孜以求的目标。西楼望月几回圆，2024年上市新药中几款能够重磅加身？ Evaluate Pharma 对2024年上市的 10 种高价值药物进行了一项前景分析，结果显示，到 2030 年，预计只有四种药物的年收入能超过 30 亿美元，而默沙东的肺动脉高压治疗药物 Winrevair (sotatercept) 届时的销售额预计会超过 60 亿美元大关。 01 Winrevair (sotatercept-csrk) ● 开发商：默沙东 ● 模态：融合蛋白 ● 获批时间：2024年3月26日 ● 获批适应症：肺动脉高压（PAH） ● 预计2030年销售额：63.2亿美元 ● 药物背景：Winrevair (sotatercept) 是一种激活素信号抑制剂，适用于治疗患有肺动脉高压 (PAH，WHO 第 1 组) 的成年人，以提高运动能力、改善 WHO 功能等级并降低临床恶化事件的风险。肺动脉高压 (PAH) 是一种罕见的进行性血管疾病，其特征是小肺动脉收缩和肺循环血压升高。它是由肺动脉壁细胞过度增殖引起的，导致狭窄和异常收缩。Winrevair通过调节血管细胞增殖来减缓或逆转 PAH 中发生的肺血管重塑过程。 ● 看好原因：肺动脉高压是一种严重的、进展性的肺部疾病，目前的治疗手段只能减轻症状或延缓病情进展，但无法治愈。虽然现有的药物（如内皮素受体拮抗剂、磷酸二酯酶-5抑制剂等）在一定程度上改善了患者的生活质量和预后，但依然存在显著的未满足医疗需求。Sotatercept 的独特机制，通过抑制活化素信号通路来减少肺血管重构和增生，有望改变疾病进程，从根本上改善患者的预后。这使得 Sotatercept 在现有治疗中占有独特的地位。PAH 市场预计将在未来几年继续增长。随着对这种罕见疾病的认识提高和早期诊断的改进，患者数量可能增加，而 Sotatercept 将填补目前治疗中的空白。 02 Cobenfy（xanomeline和trospium chloride） ● 开发商：百时美施贵宝 ● 模态：小分子 ● 获批时间：2024年9月26日 ● 获批适应症：精神分裂症 ● 预计2030年销售额：45.8亿美元 ● 药物背景：Cobenfy是首款靶向胆碱能受体激动剂（胆碱能受体中的毒蕈碱受体）而非多巴胺受体的抗精神病药物。此前针对胆碱能受体开发的中枢神经系统疾病药物大多因为明显的副作用而半途而废，但Cobenfy含有双活性成分：xanomeline 靶向 M1 和 M4 毒蕈碱受体，激活这些受体能减轻精神分裂症的症状，而 trospium chloride则有助于减少由激活该途径引起的副作用，如心血管问题和唾液、泪液分泌增加。 ● 看好原因：Cobenfy是七十年多来首次出现的新型精神分裂症药物，为全球上千万受精神分裂症折磨的患者提供了希望。临床研究显示Cobenfy可以改善认知能力并减少精神分裂症的负性症状，例如社会退缩、缺乏动力或无法表达情感。除此之外，Cobenfy在治疗阿尔茨海默病方面的潜力也被众多分析师看好。 03 Rezdiffra（resmetirom） ● 开发商：Madrigal Pharma ● 模态：小分子 ● 获批时间：2024年3月14日 ● 获批适应症：代谢功能障碍相关脂肪性肝炎（MASH） ● 预计2030年销售额：34.5亿美元 ● 药物背景：Rezdiffra是一种甲状腺激素受体-β (THR-β) 激动剂，用于治疗患有中度至晚期肝纤维化（符合 F2 至 F3 期纤维化）的代谢功能障碍相关脂肪性肝炎（MASH）的成年人。 ● 看好原因：作为第一款针对MASH获得批准的药物，Rezdiffra的前景理所当然地被分析师看好。MASH是一个存在巨大未满足的领域，预计从2022年到2032年的十年间，七个主要市场（7MM，美国、法国、德国、意大利、西班牙、英国和日本）的MASH确诊病例数将增长 450 万例，从2022 年的 2204 万人增加到 2032 年的 2655 万人。美国将成为最大市场，预测美国到2032年将有1045万确诊病例。预测显示，到 2035 年底，MASH 治疗市场规模预计将达到 483 亿美元，在此期间复合年增长率为 18%。2022年，MASH治疗的行业规模已超过52亿美元。Rezdiffra的上市成为了药物开发史上具有里程碑意义的事件。 04 Casgevy（exagamglogene autotemcel） ● 开发商：Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics ● 模态：细胞疗法 ● 获批时间：2024年1月16日 ● 获批适应症：输血依赖性β地中海贫血（TDT） ● 预计2030年销售额：34.5亿美元 ● 药物背景：TDT 是一种严重的、危及生命的遗传病。Casgevy是一种非病毒、体外 CRISPR/Cas9 基因编辑细胞疗法，适用于符合条件的TDT 患者，恢复红血球生成的功能。 ● 看好原因：TDT 需要在人的一生中频繁输血和铁螯合疗法。在美国，TDT 患者的死亡年龄中位数为 37 岁。美国管理 TDT 的终生医疗保健费用估计在 500 万美元至 570 万美元之间。匹配供体的干细胞移植是一种治愈方法，但由于缺乏可用的供体，只有一小部分 TDT 患者可以使用。Casgevy可以减少或消除TDT 患者的输血需求，因此提供了一种可能的治愈选项，这将极大地改善患者的生活质量，减轻疾病负担。β-地中海贫血是全球范围内尤其在地中海地区、南亚和中东地区常见的遗传性疾病。这些地区的患者对新的治愈性治疗需求强烈。随着全球医疗环境的不断改善，越来越多的国家可能会逐步为此类基因疗法提供监管批准和医保覆盖，从而为 Casgevy 打开广阔的市场。 05 Kisunla (donanemab-azbt) ● 开发商：礼来 ● 模态：单抗 ● 获批时间：2024年7月2日 ● 获批适应症：阿尔茨海默病 ● 预计2030年销售额：24.3亿美元 ● 药物背景：Kisunla是一种针对β淀粉样蛋白的抗体，用于治疗早期有症状的阿尔茨海默病。Kisunla 通过清除大脑中过量的淀粉样斑块来发挥作用，这些斑块可能导致与阿尔茨海默病相关的记忆和思维问题。 ● 看好原因：阿尔茨海默病是全球老龄化社会中最常见的失智类型。随着全球人口的老龄化，阿尔茨海默病的患病人数逐年增加，因此市场对治疗这一疾病的需求非常大。尤其在卫材/渤健的阿尔茨海默病抗体药物Aduhelm主动撤市之后，能够有效治疗和控制阿尔茨海默病的药物屈指可数。在临床试验中，donanemab 显示出较好的效果，尤其是在早期阿尔茨海默病患者中。其临床数据显示，它可以显著减少脑内的淀粉样斑块，并改善患者的认知功能。特别是在早期阿尔茨海默病的治疗中，donanemab 的疗效较为显著，患者的认知评分有所提高或保持稳定，这对阿尔茨海默病的治疗具有重要意义。 06 Ohtuvayre (ensifentrine) ● 开发商：Verona Pharma plc ● 模态：小分子 ● 获批时间：2024年6月26日 ● 获批适应症：成人慢性阻塞性肺病 (COPD) ● 预计2030年销售额：20.8亿美元 ● 药物背景：Ohtuvayre 是一种选择性磷酸二酯酶 3 (PDE3) 和磷酸二酯酶 4 (PDE4) 双重抑制剂，适用于成人慢性阻塞性肺病 (COPD) 的维持治疗。COPD 是一组导致气流阻塞和呼吸相关问题的疾病。它包括肺气肿和慢性支气管炎。Ohtuvayre 吸入悬浮液通过标准喷射雾化器直接输送到肺部，无需高吸气流量。 ● 看好原因：Ensifentrine 是一种双重作用的药物，它同时具有支气管舒张作用和抗炎作用。这使得它能够同时解决COPD患者常见的两个主要问题：呼吸道的收缩和持续的炎症反应。目前市场上的COPD药物主要集中在支气管舒张和抗炎两方面的单一作用上，ensifentrine 的双重机制使其在COPD的治疗中具备了独特的竞争力。Ensifentrine 在临床试验中显示出较低的心血管副作用，这是许多传统COPD药物（尤其是β-2激动剂）可能带来的问题。因此，它能够满足市场上对新型、有效治疗药物的强烈需求。Ensifentrine 是通过吸入装置使用，这种给药方式可以直接作用于肺部，具有较高的生物利用度，减少了全身副作用的风险。此外，药物的给药频率和使用方式简单，能够提高患者的依从性，特别是对于需要长期治疗的COPD患者。 07 mRESVIA (呼吸道合胞病毒mRNA疫苗) ● 开发商：莫德纳 ● 模态：mRNA疫苗 ● 获批时间：2024年5月31日 ● 获批适应症：RSV (呼吸道合胞病毒) ● 预计2030年销售额：19.2亿美元 ● 药物背景：mRESVIA 是一种改良的 RNA 疫苗，编码 RSV F 糖蛋白，可诱导免疫反应并形成针对 RSV pre-F 蛋白的抗体，可保护 60 岁及以上的成年人免受呼吸道合胞病毒 (RSV) 感染引起的下呼吸道疾病。它是首个获批的 RSV mRNA 疫苗，也是第三个获批的 RSV 疫苗。 ● 看好原因：mRNA技术是近年来疫苗研发的一个重大突破，尤其在新冠疫苗的成功应用后，mRNA平台的潜力得到了广泛验证。相比传统疫苗，mRNA疫苗的研发速度更快，能够在短时间内应对新出现的病毒。对于RSV这样的呼吸道病毒，mRNA技术能够精确地设计出有效的免疫反应，促进人体产生对病毒的免疫保护。mRESVIA作为mRNA疫苗，能够在短时间内生成病毒蛋白，从而诱导强烈的免疫反应，这使得它在对抗RSV时表现出优异的保护效果。相比传统疫苗，mRNA疫苗在产生高效的免疫反应方面具有优势。这种疫苗的优势还在于不含活病毒或病毒载体，因此其副作用风险较低，适用于大范围人群，尤其是婴幼儿和老年人群体。RSV 是全球范围内普遍存在的病毒感染，尤其在季节变化时流行，影响广泛的群体。随着人口老龄化，RSV疫苗的市场需求预计将持续增长。mRESVIA能够成为满足这些需求的重要治疗手段。此外，RSV的疫苗市场竞争相对较少，mRESVIA具备独特的技术平台和有效性优势，有潜力在市场上占据主导地位。 08 Yorvipath (palopegteriparatide) ● 开发商：Ascendis Pharma, Inc. ● 模态：多肽 ● 获批时间：2024年8月9日 ● 获批适应症：甲状旁腺功能减退 ● 预计2030年销售额：18.1亿美元 ● 药物背景：Yorvipath (palopegteriparatide) 是一种甲状旁腺激素类似物 (PTH(1-34))，用于治疗成人甲状旁腺功能减退症。甲状旁腺功能减退症是一种罕见的内分泌疾病，其特征是甲状旁腺激素缺乏或缺失，甲状旁腺激素在控制血液和骨骼中的钙、磷酸盐和骨化三醇（活性维生素 D）水平方面起着关键作用。Yorvipath 是一种长效、每日一次的激素替代疗法，可以恢复甲状旁腺激素的生理水平，解决甲状旁腺功能减退症的短期症状和长期并发症。 ● 看好原因：甲状旁腺功能减退症是一种相对罕见的疾病，但其发病率逐渐增加，尤其是一些因手术（如甲状腺切除）而导致的继发性甲状旁腺功能减退。现有的治疗方法主要依赖钙补充和维生素D，但这并不足以完全解决问题，尤其是在患者的症状控制方面。Yorvipath填补了这一治疗空白。相比传统的补充钙和维生素D的治疗方法，它具有更直接和高效的作用，有望提高患者的生活质量，并且有望成为标准治疗方案，因此市场需求较大。 09 Anktiva (nogapendekin alfa inbakicept-pmln) ● 开发商：ImmunityBio, Inc. ● 模态：蛋白 ● 获批时间：2024年4月22日 ● 获批适应症：膀胱癌 ● 预计2030年销售额：16.6亿美元 ● 药物背景：Anktiva (nogapendekin alfa inbakicept-pmln) 是同类首创的白介素 15 超激动剂，可与卡介苗 (BCG) 联合使用，用于治疗对 BCG 无反应的非肌层浸润性膀胱癌。白介素 15 (IL-15) 是一种细胞因子，通过影响自然杀伤 (NK) 和 CD8+ 杀伤 T 细胞的发育、维持和功能，在免疫系统中发挥关键作用。Anktiva 通过与 NK、CD4 和 CD8 T 细胞上的 IL-15 受体结合，驱动记忆杀伤 T 细胞的生成，这些细胞经过专门训练，可以识别癌细胞。 ● 看好原因：卡介苗BCG 对许多患者来说是一种有效的膀胱癌治疗方法，但对约 40% 的非肌层浸润性膀胱癌 (NMIBC) 病例无效。Anktiva 可以治疗对 BCG 无反应的 NMIBC，以避免膀胱切除术。 10 Attruby (Acoramidis) ● 开发商：BridgeBio Pharma, Inc. ● 模态：小分子 ● 获批时间：2024年11月22日 ● 获批适应症：转甲状腺素蛋白介导的淀粉样变性性心肌病（ATTR-CM） ● 预计2030年销售额：13.3亿美元 ● 药物背景：转甲状腺素蛋白介导的淀粉样变性心肌病 (ATTR-CM) 是一种潜在的致命性心肌疾病，其特征是淀粉样蛋白沉积物积聚，导致心肌病并最终导致心力衰竭。Attruby (acoramidis) 是一种转甲状腺素蛋白（TTR）稳定剂，用于治疗成人野生型或变异型转甲状腺素蛋白介导的淀粉样变性心肌病，以减少心血管死亡和心血管相关住院。 ● 看好原因：ATTR淀粉样变性是一种罕见病，但患者群体在全球范围内仍然存在。现有治疗选择具有较大的局限性。由于 ATTR患者仍缺乏有效且安全的治疗方法，Attruby 的上市填补了这一治疗空白。Attruby 的机制是通过稳定转甲状腺素蛋白（TTR），防止其折叠异常并减少淀粉样蛋白沉积，从而减缓或停止器官损伤。这种机制的创新使其成为该领域具有较大疗效潜力的治疗方法。临床试验数据显示 Attruby 在减缓病情进展和改善患者生活质量方面有着良好的效果，且在耐受性上表现出色，相较于传统疗法副作用较小。由于这类疾病的进展较快，尤其是心脏损害的情况，因此 Attruby 有可能成为晚期ATTR淀粉样变性患者的新选择，甚至可以在早期治疗阶段疾病缓解，进而延长患者寿命。 Ref. 1.FDA Approves Drug with New Mechanism of Action for Treatment of Schizophrenia. FDA Press Release. 26. 09. 2024. 2.Cobenfy Approval Brings 20% Upside To Bristol Myers Squibb Stock. Forbes. 27. 09. 2024. 3.Nonalcoholic Steatohepatitis (NASH) Market to Observe Stunning Growth by 2032 Owing to a Robust Pipeline. BioSpace. 01. 11. 2023. 4.Taylor, N. P. NASH market fattening up as wait for liver treatments drags on. FiercePharma. 04. 12. 2023. 5.McConaghie, A. Only Half Of 2024's Biggest Launches Belong To Big Pharma. Scrip. 07. 01. 2024. END 【智药研习社近期直播】扫码领取CPHI & PMEC China 2025展会门票来源：CPHI制药在线声明：本文仅代表作者观点，并不代表制药在线立场。本网站内容仅出于传递更多信息之目的。如需转载，请务必注明文章来源和作者。投稿邮箱：Kelly.Xiao@imsinoexpo.com ▼更多制药资讯，请关注CPHI制药在线▼ 点击阅读原文，进入智药研习社~

申请上市

2025-01-12

·GlobeNewswire-Health Care

Ascendis Pharma Provides Business and Strategic Roadmap Update at 43rd Annual J.P. Morgan Healthcare Conference

- Ascendis positioned to drive rapid revenue growth COPENHAGEN, Denmark, Jan. 12, 2025 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today provided a business and strategic roadmap update, including planned 2025 key corporate milestones. Ascendis President and CEO Jan Mikkelsen will present this update tomorrow, January 13, during the 43rd Annual J.P. Morgan Healthcare Conference. “Ascendis is well-positioned for rapid revenue growth with the launch of our first two Endocrinology Rare Disease medicines, SKYTROFA and YORVIPATH, and, following our pre-NDA meeting, planned filings for our third, TransCon CNP for achondroplasia,” said Jan Mikkelsen, Ascendis Pharma’s President and Chief Executive Officer. “Our expanding TransCon technology platform and partnerships demonstrate that our business model built on fast, successful drug development can create substantial stakeholder value.” Selected Key Updates and Milestones TransCon™ hGH (lonapegsomatropin, marketed as SKYTROFA®) SKYTROFA full-year 2024 revenue, with a single indication in pediatric GHD, expected to be ~€202 million excluding sales deductions related to prior years (based on unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions related to prior years).U.S. SKYTROFA volume (mg) increased 84% in 2024 year-over-year resulting in an estimated 6.5% market share of the total U.S. growth hormone market for 2024 (based on third party prescription data).Prescription Drug User Fee Act (PDUFA) goal date of July 27, 2025 for FDA review of supplemental BLA for the treatment of adults with growth hormone deficiency; pending approval, U.S. commercial launch planned in the fourth quarter of 2025.During the third quarter of 2025, plan to submit an Investigational New Drug (IND) application or similar for a basket trial evaluating TransCon hGH in additional indications. TransCon PTH (palopegteriparatide, marketed as YORVIPATH®) YORVIPATH full-year 2024 unaudited preliminary revenue estimate of ~€29 million.YORVIPATH commercially available for prescription in Germany and Austria since January 2024. Outside Germany and Austria, providing product through early access routes, such as ‘named patient,’ until commercial reimbursement established. ~700 patients on treatment in our Europe Direct and International Markets at the end of 2024.YORVIPATH commercially available for prescription since late December 2024 in the U.S. As of January 9, 2025, 324 patients enrolled into the Ascendis Signature Access Program or direct with specialty pharmacy, with over half of prescriptions for patients new to YORVIPATH.Expect commercial launch in at least five additional Europe Direct countries in 2025. TransCon CNP (navepegritide) Following pre-NDA meeting with FDA, plan to submit New Drug Application (NDA) for the treatment of children with achondroplasia during the first quarter of 2025, and submit Marketing Authorisation Application to the European Medicines Agency during the third quarter of 2025.Presented new data demonstrating significant improvements in leg bowing, a common complication in achondroplasia, observed with TransCon CNP compared to worsening observed with placebo in pivotal ApproaCH Trial.Topline Week 26 results from Phase 2 COACH Trial (TransCon CNP in combination with TransCon hGH) expected in the second quarter of 2025.During the fourth quarter of 2025, plan to submit an IND or similar for the treatment of hypochondroplasia. Expanding the TransCon Platform & Pipeline New TransCon protein degrader platform designed to enable efficient clearance of hormones, cytokines, and other targets. First planned TransCon protein degrader product candidate designed to normalize excess FGF-23 hormone levels for patients with X-linked hypophosphatemia. Financial Update Unaudited preliminary estimate of total full-year 2024 product revenue of ~€226 million: SKYTROFA full-year 2024 revenue expected to be ~€202 million excluding sales deductions related to prior years (based on unaudited preliminary estimate of full-year 2024 SKYTROFA revenue of ~€197 million plus ~€5 million of sales deductions related to prior years).YORVIPATH full-year 2024 unaudited preliminary revenue estimate of ~€29 million. Unaudited preliminary estimate of total full-year total 2024 revenue of ~€364 million Includes $100 million Novo Nordisk milestone payment as non-product revenue. December 31, 2024 pro forma cash balance of ~€655 million (based on unaudited preliminary estimate of December 31, 2024 cash balance of €560 million plus expected payment from Novo Nordisk of $100 million). Presentation at J.P. Morgan Healthcare Conference on Monday, January 13A live webcast of the event will be available via the Investors & News section of the Ascendis Pharma website at https://investors.ascendispharma.com. The presentation will begin at 11:15 a.m. Eastern Time / 8:15 a.m. Pacific Time. A webcast replay will be available for 30 days. The Company’s slides from the J.P. Morgan presentation will be available on the same Investor Relations website at https://investors.ascendispharma.com. 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) Ascendis’ potential for rapid revenue growth, (ii) planned regulatory submissions for TransCon CNP and TransCon hGH, (iii) the ability of Ascendis’ business model to create substantial stakeholder value, (iv) expected full-year 2024 SKYTROFA, YORVIPATH and total revenues, (v) the PDUFA date of the supplemental BLA for SKYTROFA for the treatment of adults with growth hormone deficiency and Ascendis’ plans for a U.S. commercial launch, if approved, (vi) Ascendis’ expectations regarding the commercial launch of YORVIPATH in additional Europe Direct Countries, (vii) the timing of topline results from the Phase 2 COACH Trial of TransCon CNP, (viii) Ascendis’ expectations regarding its TransCon protein degrader platform and first TransCon degrader product candidate, (ix) the expected payment from Novo Nordisk, (x) Ascendis’ ability to apply its TransCon technology platform to build a leading, fully integrated biopharma company, and (xi) 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, TransCon, SKYTROFA®, and YORVIPATH® are trademarks owned by the Ascendis Pharma group. © January 2025 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

临床2期申请上市临床结果

2025-01-11

·精准药物

2024年FDA批准的50款新药

2024年，美国食品药品监督管理局(FDA)药品评价研究中心(CDER)共批准了50种新药，包括34个化药和16个生物药，获批数量虽然低于2023年，但仍处于历史高位水平。图1 美国FDA历年批准的新药数量数据来源：FDA官网、药智网整理 1 化药：小分子仍是主流从药物类型来看，2024年FDA批准的50个新药中，化药有34个，其中小分子28个，占比56%，可见小分子仍然是新药的最主要类型，远超其他形式药物。另外获批的化药新药中还有3个显像剂，2个小核酸和多1个肽药物。新型药物虽然备受期待，但获批数量还很少，小核酸和多肽药物在经历了2023年的小高峰后，2024年获批数量有所回落，仅有3个获批上市。图2 2024年FDA批准的新药药物类型分布资料来源：FDA官网、药智网整理但是我们也看到这些新型药物正在不断拓展疆域，例如小核酸药物此前主要集中在罕见病领域，然后拓展到慢性病领域，而2024年又将适应症拓展到了肿瘤领域。 2024年6月，美国FDA批准Geron公司的Rytelo（imetelstat）上市，用于治疗患有低风险至中等-1风险的骨髓增生异常综合征（MDS），并伴有输血依赖性贫血（需要在8周内使用4个或更多红细胞单位）的成年患者，这些患者对红细胞生成刺激剂（ESA）没有应答或失去应答或不符合使用ESA的条件。这是首个获得美国FDA批准的端粒酶抑制剂。 Rytelo是一种“first-in-class”的寡核苷酸端粒酶抑制剂，通过抑制端粒酶活性，控制癌变干细胞和祖细胞的无序增殖，诱导癌变细胞凋亡，具有改变疾病进程的活性。它被行业媒体Evaluate列为今年有望获批的十大潜在重磅疗法之一。 2 生物药：双抗抢占实体瘤市场生物药方面，2024年美国FDA共批准16个生物新药，其中单抗10个，双抗3个，融合蛋白和其他蛋白3个。适应症方面，肿瘤和免疫疾病依然是抗体药物的主战场。但与往年不同的是，2024年FDA批准的3个双特异性抗体都是针对实体瘤，而此前除了强生的Amivantamab和Immunocore公司的Tebentafusp适应症为实体瘤，其余获FDA批准的抗肿瘤双抗都是针对血液瘤。 Imdelltra 2024年5月，FDA批准了安进公司的CD3/DLL3双特异性T细胞结合器（BiTE）Imdelltra（tarlatamab）上市，用于治疗广泛期小细胞肺癌（ES-SCLC）成人患者。Imdelltra是第一个也是唯一一个靶向DLL3的双特异性T细胞接合疗法，可激活患者自身的T细胞攻击表达DLL3的肿瘤细胞。随着Imdelltra的获批，针对DLL3靶点的交易持续火热。 2024年12月29日，恒瑞医药与美国IDEAYA Biosciences公司就DLL3 ADC药物SHR-4849达成许可交易，交易总金额可达10.45亿美元（包括7500万美元首付款，研发里程碑款累计不超过2亿美元，销售里程碑款累计不超过7.7亿美元）。 2025年1月2日，信达生物与罗氏就新一代DLL3 ADC药物IBI3009达成全球独家合作与许可协议，信交易总金额可达10.8亿美元（包括8000万美元首付款和最高达10亿美元的开发和商业化潜在里程碑付款，以及销售提成）。 Ziihera 2024年11月，FDA加速批准Jazz Pharmaceuticals与Zymeworks联合开发的Ziihera（Zanidatamab）上市，用于治疗经治的、不可切除或转移性HER2阳性（IHC 3+）的胆道癌（BTC）成人患者。这是首个也是唯一一个用于治疗HER2+BTC的靶向HER2的双特异性单抗。百济神州曾于2018年以4000万美元首付款+3.9亿美元的里程碑付款，获得zanidatamab和另一款在研药物ZW49在亚洲（除日本外）、澳大利亚和新西兰的独家开发和商业化权利。 Bizengri 2024年12月，FDA加速批准Merus公司的HER2/HER3双特异性抗体Bizengri（zenocutuzumab）上市，用于治疗携带NRG1基因融合（NRG1+）的晚期不可切除或转移性胰腺导管腺癌或非小细胞肺癌（NSCLC）成年患者，这些患者在接受既往全身治疗后出现疾病进展。根据Merus公司新闻稿，这是美国FDA批准的首款治疗这一患者群体的靶向疗法。另外，康方生物的PD-1/VEGF双特异性抗体依沃西单抗于2024年5月在中国获批上市，联合培美曲塞和卡铂，用于经表皮生长因子受体（EGFR）酪氨酸激酶抑制剂（TKI）治疗后进展的EGFR基因突变阳性的局部晚期或转移性非鳞状非小细胞肺癌（NSCLC）患者的治疗。 3 企业：小公司脱颖而出从企业来看，FDA获批的新药来源很分散，除了罗氏和礼来斩获了2个新药，其他药企都只有1个，而2023年辉瑞曾一家获批6个新药。从药企类型来看，跨国大药企（MNC）占比较往年有所减少，而小公司表现亮眼。这一变化一方面可能是小公司在新技术中占据重要地位，是创新的主要来源；另一方面也反映了生物技术公司更愿意自己销售产品，而不是被收购或引入大型制药合作伙伴。在2024年凭借新药声名鹊起的生物技术公司不在少数，包括Madrigal拿下首个MASH新药Rezdiffra、Verona推出20年来首款COPD新机制药物Ohtuvayre等。 Rezdiffra 2024年3月，FDA批准Madrigal公司Rezdiffra(resmetirom)联合饮食和运动，用于治疗F2-F3期的代谢功能障碍相关脂肪性肝炎（MASH，曾称为NASH）成人患者。这是美国FDA批准的首款MASH疗法。 Ohtuvayre 2024年6月，FDA批准Verona（VRNA）公司的Ohtuvayre(ensifentrine)上市，作为维持疗法治疗慢性阻塞性肺病（COPD）成人患者。Verona新闻稿指出，该药是COPD治疗领域近20年以来首个获批上市的新机制吸入制剂。另外，Ascendis Pharma的Yorvipath是首款获FDA批准用于治疗成人甲状旁腺功能减退患者的药物，Geron公司的Rytelo是首个获得美国FDA批准的端粒酶抑制剂…… 今年，FDA批准的新药中还有2款来自中国药企，分别为贝达药业的ALK抑制剂恩沙替尼和百济神州的PD-1单抗替雷利珠单抗。 4 结语 2024年已悄然落幕，步入2025年，生物医药行业依旧面临挑战也迎来机遇。随着美国新一届总统及其团队的履新，生物医药行业或将迎来一系列变革，例如《通胀削减法案》（IRA）是否会进一步推进。与此同时，人工智能（AI）及其他前沿技术的蓬勃发展，正以前所未有的动力推动着生物技术领域的创新与突破，引领着行业迈向新的高度。总体而言，尽管生物医药领域面临种种挑战，但依然拥有一个充满希望的未来。附表 2024年美国FDA CDER批准的50款新药注：清单包括CDER批准的多种新药及部分生物制品，不包括疫苗、过敏性产品、血液和血液制品、血浆衍生物、细胞和基因治疗产品，以及生物制品评估和研究中心在2023年批准的其他产品。声明：发表/转载本文仅仅是出于传播信息的需要，并不意味着代表本公众号观点或证实其内容的真实性。据此内容作出的任何判断，后果自负。若有侵权，告知必删！长按关注本公众号粉丝群/投稿/授权/广告等请联系公众号助手觉得本文好看，请点这里↓

抗体药物偶联物上市批准免疫疗法细胞疗法引进/卖出

100 项与帕罗培特立帕肽相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	H05AA	-

研发状态

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
甲状旁腺功能减退症	欧盟	Ascendis Pharma Bone Diseases A/S	2023-11-17
甲状旁腺功能减退症	冰岛	Ascendis Pharma Bone Diseases A/S	2023-11-17
甲状旁腺功能减退症	列支敦士登	Ascendis Pharma Bone Diseases A/S	2023-11-17
甲状旁腺功能减退症	挪威	Ascendis Pharma Bone Diseases A/S	2023-11-17

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临床结果

适应症

分期

评价

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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


NCT05387070 (PaTHway, PRNewswire) 人工标引	临床3期	甲状旁腺功能减退症	-	Palopegteriparatide	夢繭觸鬱繭窪鑰築構遞(憲顧夢願餘鬱網夢憲積) = 蓋襯餘糧衊製齋選觸淵窪繭餘範選繭齋鹹顧鑰 (範顧廠積餘鑰蓋蓋範醖 ) 更多	积极	2024-08-12
				Placebo	夢繭觸鬱繭窪鑰築構遞(憲顧夢願餘鬱網夢憲積) = 憲範襯鹹選廠鹹製壓鬱窪繭餘範選繭齋鹹顧鑰 (範顧廠積餘鑰蓋蓋範醖 )
NCT04701203 (FDA_CDER) 人工标引	临床3期	甲状旁腺功能减退症	82	YORVIPATH	獵遞顧築遞壓遞鹹鹽構(積夢網淵蓋構淵構鹹糧) = 選窪遞衊鬱壓簾餘製壓積糧夢選齋顧蓋淵膚衊 (淵醖積鹽膚製壓鬱淵鹽 ) 更多	积极	2024-08-09
				Placebo	獵遞顧築遞壓遞鹹鹽構(積夢網淵蓋構淵構鹹糧) = 餘鬱繭餘餘積鬱鏇簾艱積糧夢選齋顧蓋淵膚衊 (淵醖積鹽膚製壓鬱淵鹽 ) 更多
NCT04701203 (PaTHway, GlobeNewswire) 人工标引	临床3期	甲状旁腺功能减退症	82	TransCon PTH (palopegteriparatide)	艱鬱範餘繭憲繭襯鏇簾(繭築顧鏇鹽夢願網糧網) = 艱顧鏇齋鬱醖網餘鬱窪鬱簾選壓顧鬱顧廠選築 (膚餘選膚繭選鑰築範積 ) 更多	积极	2024-05-13
				TransCon PTH (palopegteriparatide) (eGFR < 60 mL/min/1.73m2)	艱鬱範餘繭憲繭襯鏇簾(繭築顧鏇鹽夢願網糧網) = 遞簾鑰憲鑰觸範醖積憲鬱簾選壓顧鬱顧廠選築 (膚餘選膚繭選鑰築範積 ) 更多
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2023)	临床3期	甲状旁腺功能减退症	82	TransCon PTH	糧鹽願壓遞醖夢蓋願鏇(遞構餘醖鑰範遞齋鹹範) = trended toward norms from baseline to Weeks 26 and 52 糧簾簾網觸簾餘窪網齋 (膚襯積醖淵範遞窪繭鬱 ) 更多	积极	2023-10-05
NCT04009291 (PaTHway \| PaTH Forward, CTgov)	临床2期	甲状旁腺功能减退症	59	TransCon PTH (TransCon PTH 15 mcg)	齋齋蓋構齋簾遞鏇蓋積(衊繭積夢範膚憲簾鬱廠) = 壓鬱艱獵艱鏇獵構鹽繭衊網鬱蓋鹹鑰製積窪遞 (願糧觸構網憲壓鏇淵鑰, 齋糧網獵顧顧願願艱鑰 ~ 觸膚壓鹹蓋膚選觸壓蓋) 更多	-	2023-09-01
				TransCon PTH (TransCon PTH 18 mcg)	齋齋蓋構齋簾遞鏇蓋積(衊繭積夢範膚憲簾鬱廠) = 顧蓋襯網構範構襯壓夢衊網鬱蓋鹹鑰製積窪遞 (願糧觸構網憲壓鏇淵鑰, 鹹顧築鑰淵襯鑰範構醖 ~ 淵鑰鬱獵繭齋衊廠選鑰) 更多
PaTH Forward Trial (ENDO2022)	临床2期	甲状旁腺功能减退症	59	TransCon PTH 15 mcg/d	範鑰簾糧衊艱遞遞網觸(構衊鏇艱糧鏇鑰範壓顧) = 餘築窪鑰膚網願衊醖選鏇膚積蓋鏇夢簾範範壓 (選餘顧衊網遞衊網顧鹽 ) 更多	积极	2022-11-01
				TransCon PTH 18 mcg/d	範鑰簾糧衊艱遞遞網觸(構衊鏇艱糧鏇鑰範壓顧) = 願願廠鬱鬱窪衊構築構鏇膚積蓋鏇夢簾範範壓 (選餘顧衊網遞衊網顧鹽 ) 更多
NCT04009291 \| NCT04701203 \| NCT05387070 (PaTHway, ENDO2022)	临床3期	甲状旁腺功能减退症	82	TransCon PTH 18 mcg/d	構顧願積蓋餘窪網製糧(網艱鹽築鹽艱顧艱築簾) = The most commonly reported study drug-related AEs were headaches 範選觸鏇夢醖壓醖醖齋 (齋範鏇獵積蓋構鹹鏇廠 ) 更多	积极	2022-11-01
				Placebo
NCT04009291 (PaTH Forward, Corporate Publications) 人工标引	临床2期	-	57	TransCon™ PTH	網衊網鹽醖糧蓋襯觸艱(壓繭膚夢窪積糧範簾繭) = 窪鏇鬱鹹築顧繭蓋鹹蓋鹹築網蓋夢壓鏇襯艱齋 (觸構繭襯襯鑰襯鑰齋衊 ) 更多	积极	2022-09-12
WCO_IOF_ESCEO2022	N/A	肢端肥大症 PTH \| serum vitamin D metabolites	-	PTH (Acromegaly group)	艱積壓鑰鹹構簾襯繭夢(獵蓋觸築繭憲憲鏇餘範) = lower baseline levels 膚淵醖願遞積選蓋襯壓 (構網齋夢繭製選廠願醖 ) 更多	-	2022-03-24
				PTH (Control group)
NCT04701203 (Corporate Publications) 人工标引	临床3期	甲状旁腺功能减退症	82	Palopegteriparatide	衊鑰範鬱鑰窪艱膚淵範(鬱網製餘壓蓋鬱選憲窪) = 鹽鏇積鬱糧廠鹽獵鏇鹽齋積範蓋夢衊餘齋獵觸 (襯顧築願鑰膚築膚鹽顧, 66.3 ~ 88.1) 更多	积极	2022-03-13
				Placebo	衊鑰範鬱鑰窪艱膚淵範(鬱網製餘壓蓋鬱選憲窪) = 鹽觸糧製簾積憲廠鹹鬱齋積範蓋夢衊餘齋獵觸 (襯顧築願鑰膚築膚鹽顧, 0.1 ~ 23.8) 更多