A Phase 3 Multicenter, Randomized, Placebo-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Eneboparatide (AZP-3601), a Parathyroid Hormone Receptor Agonist, in Patients With Chronic Hypoparathyroidism (CALYPSO)

This study is investigating the safety and efficacy of eneboparatide (AZP-3601) in patients with chronic hypoparathyroidism (cHP).
During the first 24 weeks of the trial, participants will be randomized to receive eneboparatide or placebo. Study treatment is blinded: patients and doctors will not know which group each patient has been randomized to. All patients will start with a fixed dose of study treatment (eneboparatide or placebo), administered subcutaneously with a pre-filled pen. Study treatment will be individually titrated.
After completion of the first 24 weeks, patients will be treated in the open label extension part of the study for 28 weeks. During this phase, all patients (including patients that were in the placebo group) will receive eneboparatide.

开始日期2023-06-07

申办/合作机构

Amolyt Pharma SAS初创企业

NCT05239221 / Completed临床1/2期

A Single and Multiple Ascending Dose Study to Investigate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of AZP-3601, a Synthetic Parathyroid Hormone Analog, in Healthy Subjects and in Subjects With Hypoparathyroidism

This study is investigating the safety, tolerability, pharmacodynamics and pharmacokinetics of AZP-3601 following single and repeated administration in both healthy volunteers and patients with chronic hypoparathyroidism (cHP)
The protocol includes 3 parts:
Part A: first-in-human single ascending dose (SAD) study in healthy volunteers
Part B: multiple ascending dose (MAD) study with 2 weeks of treatment in healthy volunteers
Part C: open-label MAD study with a total treatment duration of 3 months in patients with cHP.

开始日期2020-09-07

申办/合作机构

Amolyt Pharma SAS初创企业

100 项与 Eneboparatide 相关的临床结果

登录后查看更多信息

100 项与 Eneboparatide 相关的转化医学

登录后查看更多信息

100 项与 Eneboparatide 相关的专利（医药）

登录后查看更多信息

项与 Eneboparatide 相关的文献（医药）

2024-08-13·The Journal of clinical endocrinology and metabolism

An Open-label Phase 2 Study of Eneboparatide, a Novel PTH Receptor 1 Agonist, in Hypoparathyroidism

Article

作者: Takacs, Istvan ; Kamenický, Peter ; Lucas, Nadège ; Sumeray, Mark ; Mezosi, Emese ; Galvez Moreno, Maria Angeles ; Borson-Chazot, Francoise ; Ovize, Michel ; Allas, Soraya ; Soto, Alfonso ; Figueres, Lucile ; Ouldrouis, Taha ; Lemoine, Sandrine ; Mannstadt, Michael ; Capel, Ismael

Abstract:

Context:

Hypoparathyroidism is a rare disorder characterized by a deficiency in PTH resulting in hypocalcemia, hyperphosphatemia, and hypercalciuria. Eneboparatide is an investigational peptide agonist of the PTH1 receptor for the treatment of chronic hypoparathyroidism (HP).

Objective:

To evaluate the efficacy, safety, and tolerability of eneboparatide in HP patients.

Design:

Open-label, phase 2 study.

Participants:

Twenty-eight patients (21 women, 7 men), mean age (range): 58 years (28-72), with HP were enrolled into 2 consecutive cohorts (C1, n = 12 and C2, n = 16).

Intervention:

Following an optimization period, daily subcutaneous injections of eneboparatide were administered for 3 months at a 20 µg/day (C1) or 10 µg/day (C2) starting dose. Conventional therapy was progressively removed, and eneboparatide could be titrated up to 60 µg (C1) or 80 µg (C2).

Main outcomes:

Proportion of patients achieving independence from conventional therapy, albumin-adjusted serum calcium (ADsCa), 24-h urine calcium (uCa), serum bone turnover markers (serum carboxy-terminal telopeptide of type I collagen and procollagen 1 intact N-terminal propeptide), bone mineral density (BMD), and adverse events (AEs).

Results:

After 3 months, ≥ 88% of patients achieved independence from conventional therapy while mean ADsCa was maintained within target range (7.8-9 mg/dL). Eneboparatide induced a rapid and sustained reduction of mean 24-hour uCa, even among patients with hypercalciuria. Bone turnover markers slightly increased, and BMD remained unchanged, consistent with progressive resumption of physiologic bone turnover. Eneboparatide was well tolerated with no serious AEs.

Conclusion:

Eneboparatide allowed independence from conventional therapy and maintenance of serum calcium within a target range while normalizing uCa excretion and producing a balanced resumption of bone turnover.

项与 Eneboparatide 相关的新闻（医药）

2024-11-11

·EndPoints

Yorvipath’s approval showcases biopharma’s interest in rare endocrine disorder

Michele Rayes used to take 64 pills a day to manage her hypoparathyroidism, a rare endocrine disorder that impairs calcium levels, dampens daily quality of life and can sometimes lead to chronic kidney disease. Takeda’s Natpara wasn’t helping much. She continued experiencing a “3 o’clock crash” because of the drug’s short half-life and still relied on taking active vitamin D and what the patient community calls “rescue calcium.” After the Japanese drugmaker recalled Natpara due to manufacturing issues with rubber particulates, Rayes tried a new drug: Ascendis Pharma’s experimental daily injectable that replicates the natural parathyroid hormone in the body. Natpara “was a toy; it was kids’ play,” said Rayes, who’s had the condition for three decades and is now an associate director at the HypoPARAthyroidism Association, which receives industry funding. “I never felt like I had my life back. I never felt that change in quality of life.” This summer, the FDA approved the medicine, marketed as Yorvipath, and Rayes said she feels like she doesn’t “have a disease anymore,” saying she can now do daily activities that she hasn’t “done in decades.” Yorvipath’s approval is one of multiple milestones this year for the hypoparathyroidism market. “Clinical research has really intensified,” said Aliya Khan, a McMaster University endocrinologist who worked on Yorvipath’s late-stage trial and is the author of clinical guidelines . Over the last year, Ascendis snagged FDA approval for Yorvipath. AstraZeneca bought a biotech with a late-stage candidate. MBX Biosciences landed on the Nasdaq in September to help finance a mid-stage injectable. And Septerna went public in October to raise money to support what it believes is the first oral candidate to enter human studies. “You’re seeing the result of huge unmet need and a degree of innovation,” said Nicola Heffron, head of global marketing and market access at AstraZeneca’s Alexion. The condition is thought to be one of the most common rare diseases, affecting about 100,000 people in the US and also in Europe. It’s “the last classical hormone deficiency for which there is no hormone replacement,” according to Aimee Shu, a VP of clinical development at Ascendis, and has been “stuck in a rudimentary situation” compared to other conditions like type 1 diabetes. Multiple sources described the patient community as a vocal one. And the rise in R&D activity, coupled with growing awareness of the condition, could also lead to more diagnoses, said Patty Keating, the executive director of the HypoPARAthyroidism Association. She is also a patient who is participating in one of the blinded clinical trials. Patients can inherit the condition or can acquire it by way of disruptions to their thyroid glands during surgery. It impacts multiple organs, including the brain, bones, kidneys and intestines. Until recently, there have been few treatment options for patients aside from taking calcium and active vitamin D. It’s a “completely different” landscape today compared to five years ago, according to Keating. Since launching Yorvipath in Europe at the beginning of this year, Ascendis has seen a retention rate of about 99%, Ascendis CEO Jan Mikkelsen told Endpoints News . Yorvipath had an “encouraging” launch in Germany and Austria, Jefferies analyst Kelly Shi told investors in early September. More details about the launch are expected in the company’s quarterly earnings on Thursday. Ascendis plans for the treatment to be in more than 10 European countries next year, plus the US, where it set a wholesale acquisition cost of $285,000 per year. Rather than developing a prodrug like Ascendis, AstraZeneca is advancing a PTH receptor 1, or PTHR1, agonist that is also dosed daily. Dubbed eneboparatide, the investigational medicine aims to rid the need for calcium and vitamin D supplements while preserving bone mineral density, which is key for a patient population mainly comprising middle-aged women, according to AstraZeneca’s Heffron. The company got eneboparatide by way of its up to $1.05 billion acquisition of Amolyt Pharma in March. “There’s definitely a need for treatments that deal with the root cause,” Heffron said. “This condition doesn’t go away, so they need something that’s going to be sustainable.” AstraZeneca expects to have results from the Phase 3 trial in the first half of 2025, Heffron said. The small team at Amolyt was “quick off the blocks” in getting all the way to Phase 3, she said, and the UK drugmaker is now waiting with “bated breath” for the late-stage data. Behind Ascendis and AstraZeneca are MBX, Septerna and an earlier-stage private startup called Confo Therapeutics, which declined to disclose details on its preclinical PTHR1 agonist. MBX has a hormone replacement injectable, called MBX 2109 , in Phase 2 and expects to have topline results in the third quarter of 2025, MBX CEO Kent Hawryluk told Endpoints. The Indianapolis-area biotech went through a $163 million IPO in September to help advance the lead asset as well as a post-bariatric  hypoglycemia peptide, a preclinical obesity drug and other programs. “We’re glad to see the progress in the space, the activity — most importantly for patients who really are in need of better treatments for hypoparathyroidism,” Hawryluk said. The company hopes its once-weekly option will be a more accessible one for patients versus taking daily drugs. MBX aims to commercialize the drug on its own in the US, but it could seek partners for other regions, he said. Septerna, meanwhile, entered a healthy volunteer study last month in Australia to test once- and twice-daily versions of its oral candidate called SEP-786. The startup has said it aims for Phase 1 data in mid-2025. After that, Septerna has said it will then test the PTH1R-targeting drug in Phase 2 in the US, Canada, Europe and other regions. “We think that the game-changer for this whole space will be an oral small molecule,” CEO Jeffrey Finer told Endpoints at the time of Septerna’s $150 million Series B in 2023. “We’ve developed compounds that we believe should work as well as those injectable peptides.”

上市批准并购临床3期临床2期IPO

2024-09-06

·药精通Bio

超1355亿美元！全球TOP20创新药并购案汇总

TIS2024第四届自免疾病细胞疗法与干细胞疗法论坛重磅来袭，点击图片查看重磅嘉宾，合作详询：王晨 180 1628 8769 （扫码立即报名获取TIS2024免费票）前言 Preface 文 l 猎药人俱乐部本文对2024年至今（截止2024年7月30日）全球创新药交易/收购案例进行梳理，根据金额（已披露）统计出了全球创新药买家TOP20名单。这20家企业交易/并购金额累计超过1355亿美元，所涉项目数量约119起。其中交易项目达96起，金额超848亿美元；收购项目达23起，金额超过507亿美元。一些趋势包括： MNC仍是主要玩家：在20家企业中，诺华、艾伯维、强生等MNC占据16家，并且前十的位置均由MNC占据。另外，Vertex、Ipsen、BioNTech等企业也进入了榜单。Vertex以58亿美元的交易/并购金额位居榜单第十一位，Ipsen以54亿美金位居榜单第十二位。从药物类型来看，核酸疗法、ADC、环肽、核药、多特异性抗体药物、蛋白降解剂等药物频现交易/收购名单。诺华、默沙东、BMS对环肽领域进行了投资；诺和诺德、诺华、Ipsen、BMS、GSK等出手购买核酸领域资产/技术；在核药领域，礼来、阿斯利康、诺华等企业进行了相关交易。从治疗领域来看，肿瘤领域最吸金，尤其肿瘤免疫疗法备受关注。勃林格殷格翰、安斯泰来、艾伯维等多家企业积极布局中。多家企业对AI技术进行投资：共达成约10笔交易，诺华、礼来、默沙东基于AI技术小分子的开发；武田、BMS、阿斯利康投资集成的人工智能和湿实验室技术。智能制药3D打印药物技术受到青睐：BioNTech与三迭纪医药将基于3D打印技术开发口服RNA疗法达成研究和合作平台技术许可，总金额超过12亿美元。中国生物制药公司的技术/资产受到MNC的认可：诺华、阿斯利康、武田、罗氏等7家MNC重金出手购买中国药企的资产，达成14笔交易，1笔收购，涉及14家中国公司；累计金额超过227亿美元。 TOP交易金额：诺华达成6笔创新药交易，涉及金额超过137.53亿美元，不惜重金在核酸疗法、AI技术、环肽等热门领域投资。其次，GSK今年发生了4笔交易，累计金额近90亿美元；刚刚GSK与生物平台创新公司Flagship Pioneering达成合作发现和开发多达10种新型药物和疫苗的组合，合作金额超过70亿美元。艾伯维的9笔交易花费近乎80亿美元。 TOP收购金额：诺和诺德收购项目2笔合计金额超过176亿美元，今年2月诺和诺德宣布以165亿美元收购CDMO巨头Catalent。其次，诺华收购项目有4笔，累计金额超过50亿美元，包括以29亿美价格收购MorphoSys。 TOP项目数量：截止目前，艾伯维达成交易/收购项目11笔，是今年最热衷“买买买”的企业，主要集中在自免领域；诺华达成10笔，在核药、基因疗法、小核酸领域均有相关动作；阿斯利康、BMS分别达成9笔、8笔交易/收购合作。 2024年全球创新药交易/收购金额TOP20企业（截止2024年7月30日）按照实时汇率计算，1 欧元 ≈ 1.086 美元 01 金额：交易/收购费用超过百亿美元的企业在TOP20企业中，诺和诺德和诺华分别以210亿美元、188亿美元的交易/收购金额成为今年最热衷“买买买”的药企。一、企业名称：诺和诺德交易/收购金额：210.27亿美元项目数量：7 整体来看，诺和诺德的交易/收购分为4类： 1、通过合作交易进行心脏代谢相关疾病的药物研发； 2、加码罕见病药物开发和创新； 3、通过交易获得减肥候选产品，和司美格鲁肽形成互补； 4、通过收购，扩大降糖/减重产品司美格鲁肽产能供应。 2月26日，诺和诺德与Neomorph达成多靶点合作，以发现用于心脏代谢和罕见疾病的新型分子胶降解剂，潜在交易总额为14.6亿美元。这是诺和诺德首次入局靶向蛋白降解领域。近年来，靶向蛋白降解技术逐渐成为研究热点，该技术能够特异性地识别并降解细胞内的目标蛋白质，为疾病治疗提供了新的策略，其中以PROTAC（蛋白降解靶向联合体）与分子胶进展最快。 3月25日，诺和诺德以10.25亿欧元的价格收购Cardior Pharmaceuticals，加强自己在心血管疾病领域的研发管线。Cardior主要致力于RNA疗法的发现与开发，专注于针对心脏功能失调的根本原因，开发预防、修复和逆转心脏疾病的RNA疗法。通过此次收购，诺和诺德将获得Cardior用于治疗心衰的核心在研产品CDR132L。CDR132L是一款靶向miR-132的特异性反义寡核苷酸（ASO）药物，可选择性阻断miR-132分子异常水平，从而停止并部分逆转细胞病理学，带来心脏功能的长期改善。除了CDR132L外，Cardior还有3款治疗肥厚性和扩张型心肌病等心脏疾病的疗法正在开发中：CDR348T（ncRNA调节剂）、CDR641L（ncRNA干扰剂）以及一款尚未披露具体信息的处于安全性/毒理学研究阶段的项目，它们均是通过和ncRNA相互作用，起到治疗肥厚性心肌病等的作用。 2月5日，控股诺和诺德的Novo Holdings同意通过165亿美元（约1190亿元）现金收购Catalent。作为本次交易的一部分，诺和诺德将以110亿美元的预付款收购Catalent在意大利、比利时和美国印第安纳州的三个灌装工厂和相关资产，以增加GLP-1受体激动剂--司美格鲁肽的产能。诺和诺德交易/收购项目二、企业名称：诺华交易/收购金额：188.48亿美元项目数量：10 在ADC、GLP-1疗法交易并购风靡的当下，诺华似乎在“冷眼旁观”，并没有一味地追求热门领域，而是集中力量聚焦到自己的优势领域来探索创新疗法。诺华目前专注于五大疾病领域，即心血管、免疫学、神经科学、实体瘤和血液医学领域；此外诺华不断推进新兴技术平台（基因治疗、细胞治疗、放射性配体治疗、小核酸等）的开发，对这个方向上的技术和管线采取优先投资。 41.65亿美元买下中国Biotech小核酸产品：2024年1月7日，舶望制药宣布与诺华达成合作协议，将一款1期临床项目的全球权益授权给诺华，诺华还将获得针对心血管疾病的最多额外2个靶点化合物的选择权。此外，诺华还获得一款1/2a期临床项目的大中华区外全球权益。诺华支付1.85亿美元预付款，合作总金额高达41.65亿美元。诺华不断加码核药领域：（1）17.5亿美元收购Mariana Oncology：5月，诺华宣布已达成收购 Mariana Oncology的协议，这是一家专注于开发新型放射性配体疗法的生物技术公司。诺华将支付10亿美元的预付款，并在完成预先指定的里程碑后额外支付7.5亿美元。此次交易涵盖了强大的 RLT 项目组合，涵盖乳腺癌、前列腺癌和肺癌等一系列实体瘤适应症的先导化合物优化和早期开发。（2）以近30亿美元的交易额与PeptiDream合作开发环肽RDC药物。4月，诺华宣布进一步扩大与 PeptiDream公司在肽类发现方面的合作。根据这项多项目合作协议，PeptiDream将利用其肽发现平台系统（PDPS）技术，针对诺华选定的目标，来识别并优化大环肽。超10亿美元合作开发新型AAV基因疗法。1月，诺华宣布与Voyager Therapeutics达成高达12亿美元的战略合作和许可协议，将共同推进亨廷顿氏病（HD）和脊髓性肌萎缩症（SMA）的潜在基因疗法。Voyager的TRACER衣壳发现平台是一个广泛适用、基于RNA的筛选平台，能够在多个物种，包括非人灵长类动物（NHPs）中快速发现具有强大穿透血脑屏障和增强中枢神经系统（CNS）嗜性的AAV衣壳。 30亿美元开发新型肿瘤双特异性抗体。7月24日，诺华和美国加州生物制药公司Dren Bio案达成合作，集中使用Dren Bio专有的靶向髓细胞接合器（Targeted Myeloid Engager）和吞噬（Phagocytosis）平台发现和开发癌症治疗性双特异性抗体。此次合作，Dren Bio将从诺华公司获得1.5亿美元的预付款，以及28.5亿美元的里程碑付款。诺华交易/收购项目 02 数量：交易/收购项目数量TOP企业从交易/收购的项目数量来看，艾伯维可谓是“购买狂人”，截至目前发生11笔（包括9笔交易项目，2笔收购项目）相关交易/收购事件，累计金额超过82亿美元，位居TOP20榜单第五。其次，阿斯利康进行了10笔交易/收购事件，累计金额为51.39亿美元；诺华进行了10笔交易/收购事件，累计金额分别为188.48亿美元；BMS发生8笔交易事件，累计金额18.65亿美元；诺和诺德、吉利德、勃林格殷格翰、BioNTech、默沙东分别发生5笔创新药相关的交易/收购事件。一、企业名称：艾伯维交易/收购金额：82.75亿美元项目数量：11 艾伯维正在寻求新的变革，其创新药交易/收购目的可分为2类： 1、加码自免赛道，为修美乐寻找“接班人”； 2、布局新一代神经科学领域药物。自免领域：自免疾病领域是全球仅次于肿瘤的第二大用药市场，具有庞大的市场规模和增长潜力。艾伯维正在自免领域加紧布局，积极拓展管线，今年有超过6笔相关交易，其中3款管线都涉及IBD领域。这些交易中，除了TL1A属于热门靶点，其他都是艾伯维对下一代自免疗法的布局，要么是first-in-class靶点，要么是下一代的技术平台。 2月，艾伯维以7.13亿美元的价格与OSE Immunotherapeutics建立战略合作关系，来开发旨在解决慢性和严重炎症的潜在first-in-class单克隆抗体OSE-230。 2月，艾伯维和Tentarix Biotherapeutics宣布开展多年合作，双方将利用Tentarix的Tentacle平台共同开发肿瘤/免疫相关生物药。根据协议条款，Tentarix将收到两个项目的预付款，总计6400万美元。 6月，艾伯维以2.5亿美元收购Celsius Therapeutics，后者是一家致力于为炎症性疾病患者开发创新疗法的生物技术公司，其核心资产CEL383是一种潜在的一流抗TREM1药物。TREM1是IBD领域中一个已证实的关键致病基因，在炎症性单核细胞和中性粒细胞中均有表达。在这些细胞类型和其他细胞类型中，TREM1处于多条已知炎症通路的上游位置，起着加剧炎症反应的作用。神经科学领域： 2023年，艾伯维神经科学板块实现收入77.17亿美元，同比增长18.2%，是仅次于自免板块的第二大收入来源，也是艾伯维去年唯一实现正增长的板块。艾伯维在神经科学领域也建立了丰富的产品线，包括3款偏头痛药物Botox、Ubrelvy、Qulipta，精神分裂症药物Vraylar，以及帕金森病药物Duodopa。基于此，艾伯维决定乘势而上、深耕这一领域，以进一步扩大市场份额。 5月，艾伯维斥资20亿美元与Gilgamesh Pharmaceuticals签署了一项协议，开发治疗精神疾病的新一代疗法。该合作将利用艾伯维在精神病学领域的专长和Gilgamesh的创新研究平台，以发现新型神经重塑剂（neuroplastogens）。Gilgamesh利用其创新的研究平台已成功发现了神经重塑剂的先导化合物。艾伯维交易/收购项目二、企业名称：阿斯利康交易/收购金额：51.39亿美元项目数量：10 7月15日，阿斯利康宣布成功完成对Amolyt Pharma 的全面收购，收购总价为10.5亿美元。Amolyt Pharma 是一家专注于开发罕见内分泌疾病新疗法的临床阶段生物技术公司，阿斯利康的这次收购囊获了一款极具潜力的研发管线——肽类治疗药物恩尼博帕肽（AZP-3601），已处于临床3期阶段。2023年，阿斯利康罕见病产品组合的全球销售额已高达77.6亿美元，此举将进一步深化阿斯利康的罕见病相关布局。 3月，阿斯利康宣布将斥资高达24亿美元收购Fusion Pharmaceuticals，这是一家专注于开发新一代放射性偶联药物（RDC）的生物制药公司。其核心产品FPI-2265是一款针对前列腺特异性膜抗原（PSMA）的靶向药物，目前正处于2期临床开发阶段，用于治疗转移性去势抵抗性前列腺癌（mCRPC）。此项收购是阿斯利康加大投资靶向疗法、从而继续改革癌症治疗的重要一步。阿斯利康交易/收购项目三、企业名称：默沙东交易/收购金额：65.18亿美元项目数量：7 基于AI技术快速生成小分子化合物：1月，默沙东宣布与Variational AI公司达成合作，将利用Variational AI的Enki生成式人工智能平台，设计创新和选择性小分子药物。Enki技术平台基于生成式AI算法，旨在简化创新分子结构的生成。研究人员只需要提供化合物的目标产品特征，包括靶点、脱靶效应等对小分子化合物的要求，Enki技术平台可在几天时间内合成符合条件的小分子结构。 10亿美元利用编程技术开发新型智能生物制剂：3月，默沙东斥资10亿美金与Pearl Bio达成许可、合作和期权协议，以发现包含非标准氨基酸的生物疗法。此次合作将专注于发现和开发用于癌症治疗的生物疗法，利用Pearl Bio独家的基因组编码生物（GRO）技术、在基于细胞和无细胞系统中工作的独特能力、专有的栓系核糖体来编码合成单体，以靶向以前难以靶向的表位。默沙东交易/收购项目四、企业名称：BMS 交易/收购金额：18.65亿美元项目数量：8 BMS在AI制药领域达成了2笔合作：（1）2月13日，BMS宣布与VantAI达成合作，借助VantAI的生成式人工智能平台，将分子胶设计成小分子药物，并计划未来扩展更多项目。此次合作将结合VantAI在几何深度学习方面的积累以及BMS在靶向蛋白降解剂开发方面的经验。VantAI将有资格从BMS获得高达6.74亿美元的发现、开发、临床、监管和销售里程碑付款以及分层版税，并可选择进一步扩展到其他治疗项目。（2）5月，BMS与致力于设计新一代环肽疗法的 Menten AI达成合作，利用后者的生成式 AI 平台来加速大环肽的设计和优化。Menten AI的专有平台可在几周内设计出有效的和膜渗透性的大环肽。根据近日路透社的消息，BMS正在中国寻找并购机会。在6月底苏州举办的2024首届美国华人生物医药科技协会中国年会（CBA-China）上，BMS的一位BD负责人表示，BMS正面临着专利到期的挑战，正在寻找“补强机会”，在中国主要在看ADC药物。 BMS交易/收购项目 03 TOP20创新药买家：买入的中国生物制药技术/资产截止2024年7月底，TOP20创新药买家与中国生物制药公司共发生14笔交易，1笔收购，涉及金额超227.44亿美元。 BioNTech是今年在中国“进货”最多的买家，达成3笔交易，花费超30亿美元。（1）5月，BioNTech与宜联生物达成新的战略合作，BioNTech将获得利用宜联生物TMALIN® ADC 技术平台开发针对限定的某几个前沿创新靶点的 ADC 产品的独家选择权及相应全球独家许可。（2）5月8日，BioNTech获得由普米斯自主研发的临床前双特异性抗体候选药物的全球开发、生产和商业化权利。（3）7月23日，BioNTech与三迭纪医药达成研究合作与平台技术许可协议。双方将在合作中基于3D打印药物技术开发口服RNA药物，旨在提供一种突破性疗法，通过方便的口服制剂的方式来解决未满足的临床需求。最高交易额来自恒瑞医药--Hercules公司。5月，美国 Hercules 公司获得恒瑞医药GLP-1产品组合在除大中华区以外的全球范围内开发、生产和商业化的独家权利，此次交易金额高达60.35亿美元；另外恒瑞将取得美国 Hercules 公司 19.9%的股权，且将从美国Hercules 公司获得 GLP-1 产品组合授权许可费。宜联生物与两家国外药企达成合作，均与ADC相关，其他中国企业仅一次。（1）罗氏：罗氏引进宜联生物靶向间质表皮转化因子（c-MET）下一代抗体偶联药物候选产品YL211项目在全球范围内的开发、制造和商业化的独家权益。合作费用超过10亿美元。（2）BioNTech：达成ADC技术授权合作，BioNTech将获得某几个前沿创新靶点ADC产品的独家选择权及相应全球独家许可。核酸技术出海。（1）2月，环码生物与百时美施贵宝达成一项可行性研究合作，探索环形RNA技术在某治疗领域应用的可行性。（2）1月，舶望制药与诺华达成独家合作，开发多款心血管siRNA药物。该交易潜在总价值高达41.65亿美元。 TOP20创新药买家购买的中国生物制药公司的资产/技术参考资料：药智头条、XPharma、医药魔方、药渡 END 免责声明：本文仅作知识交流与分享及科普目的，不涉及商业宣传，不作为相关医疗指导或用药建议。文章如有侵权请联系删除。 TIS2024第四届免疫细胞疗法与干细胞疗法论坛重磅来袭，合作详询：王晨 180 1628 8769

并购抗体药物偶联物信使RNA细胞疗法疫苗

2024-08-26

·FierceBiotech

MBX files for IPO, seeking cash to take challenger to Ascendis' Yorvipath into phase 3

In addition to supporting its lead drug, MBX has earmarked some money to take an obesity candidate into the clinic. MBX Biosciences has added to the recent flurry of IPO filings. The biotech, which filed its paperwork weeks after raising $63.5 million privately, is seeking funding to take a would-be challenger to Ascendis Pharma’s rare endocrine disease drug Yorvipath into phase 3 development.Indiana-based MBX is built on technology designed to address the limitations of both unmodified and modified peptide therapies. By engineering peptides to improve their druglike properties, the biotech is trying to reduce the frequency of dosing, ensure consistent drug concentrations and otherwise establish product characteristics that improve clinical outcomes and simplify the management of diseases.MBX used the platform to create the hypoparathyroidism candidate MBX 2109. The biotech is trying to provide continuous exposure to parathyroid hormone (PTH) with once-weekly dosing. MBX 2109 was generally well tolerated in phase 1, with no serious drug-related effects, and is now in phase 2.Management is aiming to report top-line data in the third quarter of 2025 and advance the molecule into phase 3 using the IPO cash. The strategy puts the biotech on a collision course with Ascendis, a biotech that sells a once-daily PTH replacement therapy. MBX sees a need for a more convenient treatment that can normalize serum and urine calcium. AstraZeneca has a once-daily asset, eneboparatide, in phase 3. GLP-1, the peptide at the heart of the obesity drug boom, is central to the rest of MBX’s pipeline. The company has a once-weekly GLP-1 receptor antagonist, MBX 1416, in development. MBX sees the asset as a potential treatment of post-bariatric hypoglycemia, a chronic complication of weight loss surgery. The drug is in phase 1 testing. Data are due this year, and MBX plans to move into phase 2 using the IPO cash.MBX has also earmarked some money to take an obesity candidate into the clinic. The prospect, MBX 4291, is a GLP-1/GIP receptor co-agonist prodrug. Eli Lilly already sells a GLP-1/GIP receptor co-agonist, tirzepatide, as Mounjaro in diabetes and Zepbound in obesity. However, tirzepatide is given once a week. MBX is aiming to achieve once-monthly dosing when it files to test its asset in humans next year.Amgen’s bispecific GLP-1/GIP drug candidate AMG 133 could also support once-monthly dosing, but most molecules are targeting once-weekly administration. MBX is trailing Amgen, which is running a phase 2 trial of its once-monthly prospect.The biotech submitted its paperwork the day after Bicara Therapeutics and Zenas Biopharma filed to go public. Like MBX, Bicara and Zenas are seeking cash to take candidates into and through late-phase trials.

临床2期临床1期临床3期IPO

100 项与 Eneboparatide 相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
甲状旁腺功能减退症	临床3期	美国	Amolyt Pharma SAS初创企业	2023-06-07
甲状旁腺功能减退症	临床3期	加拿大	Amolyt Pharma SAS初创企业	2023-06-07
甲状旁腺功能减退症	临床3期	丹麦	Amolyt Pharma SAS初创企业	2023-06-07
甲状旁腺功能减退症	临床3期	法国	Amolyt Pharma SAS初创企业	2023-06-07
甲状旁腺功能减退症	临床3期	德国	Amolyt Pharma SAS初创企业	2023-06-07
甲状旁腺功能减退症	临床3期	匈牙利	Amolyt Pharma SAS初创企业	2023-06-07
甲状旁腺功能减退症	临床3期	意大利	Amolyt Pharma SAS初创企业	2023-06-07
甲状旁腺功能减退症	临床3期	荷兰	Amolyt Pharma SAS初创企业	2023-06-07
甲状旁腺功能减退症	临床3期	波兰	Amolyt Pharma SAS初创企业	2023-06-07
甲状旁腺功能减退症	临床3期	葡萄牙	Amolyt Pharma SAS初创企业	2023-06-07