Bradykinin is a potent vasodilator that is formed by the activation of the kallikrein-kinin system. We and others have shown that bradykinin increased during hemodialysis; however, the role of bradykinin in dialysis-induced hypotension (DIH) has not been evaluated. Preliminary results from a pilot clinical trial showed that bradykinin B2 receptor blockade with icatibant prevents excessive blood pressure during hemodialysis. Thus, in this study, we will test the overarching hypothesis that blockade plasma kallikrein with lanadelumab would ameliorate the reduction of blood pressure during hemodialysis in patients who are prone to DIH. For this purpose, we will conduct a parallel arm, double-blind placebo-controlled trial, using lanadelumab to evaluate the occurrence of

开始日期2022-07-27

申办/合作机构

Vanderbilt University Medical Center

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100 项与拉那利尤单抗相关的专利（医药）

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137

项与拉那利尤单抗相关的文献（医药）

2025-12-01·JOURNAL OF CLINICAL IMMUNOLOGY

Expanding the Genetic and Clinical Spectrum of Hereditary Angioedema with Normal C1 Inhibitor: Novel Variants and Treatment Insights

Article

作者: He, Shan ; Chen, Zihua ; Yang, Fanping ; Maurer, Marcus ; Zhang, Aiyuan ; Yang, Jin ; Zhao, Ying ; Zheng, Shenyuan ; Xu, Yu ; Luo, Xiaoqun ; Wang, Lanting ; Chen, Shengan ; Tang, Chang ; Lee, Dylan ; Tang, Lin ; Guan, Chenggong ; Gao, Haiqing ; Zhang, Zhen ; Ma, Li

Hereditary angioedema with normal C1 inhibitor (HAE-nC1-INH) is a rare and genetically heterogeneous disorder with an incomplete molecular understanding. This study aimed to identify novel genetic variants associated with HAE-nC1-INH, characterize their clinical manifestations, and evaluate real-world treatment responses. Whole-exome sequencing of 27 HAE patients, including eight with HAE-nC1-INH, identified four previously unreported MYOF variants and additional pathogenic variants in KNG1 and HS3ST6, expanding the genetic spectrum of the disease. MYOF variants were associated with recurrent edema episodes, often with prolonged duration. The HS3ST6 variant was linked to refractory angioedema with non-resolving lower extremity involvement, highlighting atypical, persistent clinical phenotypes beyond the classical self-limiting presentation of HAE. Lanadelumab effectively reduced attack frequency in most patients; however, the variability in treatment response, particularly in MYOF and HS3ST6 carriers, highlights the need for individualized therapeutic approaches. These findings provide new insights into the genetic and clinical complexity of HAE-nC1-INH and emphasize the importance of genetic testing in refining diagnosis and optimizing treatment strategies, contributing to a more precise understanding of hereditary angioedema.

2025-11-01·Journal of Allergy and Clinical Immunology-In Practice

Insights From the First 820 Patients From the Brazilian Multicenter Registry of Hereditary Angioedema: The Key Role of Genetic Testing and Targeted Therapies

Article

作者: Serpa, Faradiba S ; Aun, Marcelo V ; Azoubel-Antunes, Adriana ; Moraes, Maria Stela Y ; Rosário Filho, Nelson A ; Oliva-Alonso, Maria L ; Ferreira, Janaira F S ; Chong-Neto, Herberto J ; Giavina-Bianchi, Pedro ; Fonseca, Joice T ; Dias, Marina M ; Campos, Régis A ; Iwashita, Márcia T ; Tavares, Clarissa L T S V ; Traina, Fabiola ; Ferrel, Luciana M ; Machado, Julianne A ; Kruk, Tatielly ; Pesquero, João B ; Grumach, Anete S ; Mendonça, Leonardo O ; Figueiredo, Mariana R ; Arruda, L Karla ; Moyses, Therezinha R ; Figueiredo, Joanemile P ; Roxo-Junior, Persio ; Tumelero, Melissa T ; Ruy, Patricia C ; Capelo, Albertina V ; Ferriani, Mariana P L ; Ferraroni, Natasha R ; Barbosa, Adriana M S C ; Aragon, Davi C ; Nasser, Nayara M F ; Valle, Solange O R ; da Silva, Jane ; Marcelino, Fernanda C ; Mansour, Eli ; Minafra, Fernanda G ; Moreira, Iramirton F ; Fragnan, Nyla T M L ; Gonçalves, Rozana F ; Toledo, Eliana C ; Teixeira, Ana Julia R ; de Moraes, Caroline G F B ; Dias, Gabriela A C ; Kolarik, Maitê L ; Ferraro, Maria Fernanda ; Batigalia, Vanessa A ; Zanetti, Maria Eduarda T ; Nunes, Fernanda L ; Moreno, Adriana S ; Maia, Luana S M ; Bork, Konrad ; Castro, Ana Paula B M ; Seneda Lemos, José Eduardo ; Jannuzzi, Lucca N P ; Cichon, Sven ; Prado, Alex I F ; Kamoi, Tsukiyo O

BACKGROUND:

Hereditary angioedema (HAE) is a rare autosomal dominant disorder with a prevalence of 1:50,000 individuals. Delayed diagnosis and deaths from asphyxia still occur.

OBJECTIVE:

To identify knowledge and management gaps regarding clinical, genetic, and therapeutic aspects of HAE in Brazil, aiming to improve patient care and outcomes.

METHODS:

A Brazilian multicenter HAE registry was established, with patients' data included by treating physicians using the REDCap (Research Electronic Data Capture) platform.

RESULTS:

Of the 820 patients with HAE enrolled, 68.8% were female. Most (72.4%) experienced HAE due to C1 inhibitor deficiency (HAE-C1INH), whereas 19.4% had HAE with normal C1INH caused by variants in the F12 gene (HAE-FXII). Onset of symptoms occurred earlier in HAE-C1INH as compared with HAE-FXII (mean 11.2 years vs 19.4 years, respectively), and time for diagnosis was shorter in patients younger than 18 years, as compared with those 18 years and older (mean 1.8 years vs 14.5 years, respectively). Regarding treatment, 52.8% received first-line on-demand therapies (icatibant or plasma-derived C1INH [pdC1INH]). Only 4.8% used first-line options for long-term prophylaxis (LTP), such as lanadelumab or subcutaneous/intravenous pdC1INH. Attenuated androgens were used for LTP in 52% of patients, with adverse effects reported for 34.8%.

CONCLUSIONS:

Brazilian patients with HAE share common aspects with global patients, including predominance in women, and HAE-C1INH as the most common subtype. Available genetic testing allowed for identification of a notable proportion of HAE-FXII (19.4% of the patients). Despite recent advances, access to first-line therapies for LTP of HAE attacks remains limited.

2025-11-01·ANNALS OF ALLERGY ASTHMA & IMMUNOLOGY

Lanadelumab’s impact on hereditary angioedema control and quality of life across disease activity subgroups

Article

作者: Aygören-Pürsün, Emel ; Martinez-Saguer, Inmaculada ; Zanichelli, Andrea ; Magerl, Markus ; Betschel, Stephen D ; Wuillemin, Walter A ; Andresen, Irmgard ; Busse, Paula J ; Khutoryansky, Natalie ; Wedner, H James ; Estepan, Daniel Nova ; Kessel, Aharon ; Gagnon, Remi ; Goodyear, M Dawn ; Banerji, Aleena ; Juethner, Salomé ; Watt, Maureen ; Recke, Andreas ; Cancian, Mauro ; Kinaciyan, Tamar

BACKGROUND:

Real-world clinical data support effectiveness and safety of lanadelumab in patients with hereditary angioedema (HAE); however, disease activity between patients can vary substantially in the absence of long-term prophylactic treatment.

OBJECTIVE:

To assess the effectiveness of lanadelumab in patients with HAE by baseline HAE attack frequency.

METHODS:

Patients with HAE from the phase 4 EMPOWER (NCT03845400) and ENABLE (NCT04130191) studies with available baseline attack rate data were included in this post hoc analysis. Disease activity subgroups were defined per pre-enrollment/lanadelumab (baseline) HAE attack rate (low, <1; moderate, ≥1 to <2; high, ≥2 to <3; very high, ≥3 attacks/mo).

RESULTS:

The analysis included 152 patients (low disease activity, n = 29; moderate, n = 29; high, n = 15; very high, n = 79). In all 4 subgroups, mean and median HAE attack rates after lanadelumab initiation were low (0.0-0.5 attacks/mo). Clinically meaningful improvements (≥6-point decreases) in mean Angioedema Quality of Life total scores were observed regardless of pre-lanadelumab attack rates. From month 1 after lanadelumab initiation to the end of follow-up, mean Angioedema Control Test Scores were 10 or more (indicating patient perception of well-controlled disease) in all 4 subgroups.

CONCLUSION:

In these real-world data sets, on-treatment lanadelumab attack rates were low regardless of baseline disease activity. Patients from all 4 subgroups experienced improvements in health-related quality of life and disease control. Overall, these findings support long-term prophylaxis with lanadelumab across disease activity levels.

TRIAL REGISTRATION:

EMPOWER: ClinicalTrials.gov Identifier: NCT03845400; ENABLE: ClinicalTrials.gov Identifier: NCT04130191.

140

项与拉那利尤单抗相关的新闻（医药）

2025-12-01

·BioSpace

As New Hereditary Angioedema Drugs Flood the Market, Will Uptake Match Innovation?

iStock, natrot This year has seen the approval of several first-in-class therapies for HAE, but in a fragmented space, experts question whether they will be enough to net their developers a significant share of the entrenched market. The drug development landscape for hereditary angioedema—a potentially lethal condition characterized by severe swelling, including of the airways—has evolved rapidly in recent decades, with at least one investigational therapy touted to be potentially curative. The disease’s dire nature necessitated the development of both acute treatments for individual attacks and prophylactic therapies designed to reduce the overall number and severity of attacks. The creation of the first FDA-approved prophylactic and on-demand treatments in 2008 and 2009, respectively, changed the outlook for patients with hereditary angioedema (HAE). In the intervening years, progress has continued, with 2025 marked by several novel approvals and the emergence of late-stage candidates that could again change the treatment of the disease. In August, Ionis Pharmaceuticals received FDA approval for Dawnzera, the first RNA-targeting therapy for the prophylactic treatment of HAE. This followed closely behind FDA greenlights in July for KalVista Pharmaceuticals’ Ekterly , the first on-demand pill for the condition, and in June for CSL’s prophylactic factor XIIa inhibitor Andembry , again, the first in its class. Waiting in the wings to join this latest cohort is Intellia Therapeutics’ NTLA-2002, also known as lonvo-z, a CRISPR-based gene therapy aiming to prevent HAE attacks with a single dose. Phase I/II data presented at the American College of Allergy, Asthma & Immunology (ACAAI) 2025 conference last month showed that 97% of patients receiving a 50-mg dose of the therapy were attack-free and long-term prophylaxis-free as of the data cutoff of Aug. 29. Patients in the pooled readout were followed for up to 32 months. All this movement in the pipeline should mean a range of treatments for patients to choose from, but the reality is quite different. Patients are often “sticky” with their existing treatments, Myles Minter, biotech equity research analyst at William Blair, told BioSpace, and often view switching treatments as an unnecessary risk if their current therapies are working. “That’s why investors consider it a fragmented market,” Minter said, “Because great work has been done to keep HAE patients under control, as most people who have access are taking an effective therapy. Any other treatments are just fighting to get a smaller piece of the pie, and there are lots of different therapeutic options here.” That “pie” is projected to be worth nearly $6 billion by 2030, according to Grand View Research. Takeda’s Takhzyro, approved in 2018 , surpassed incumbents to become the market leader and now generates full-year sales of approximately $1.7 billion, according to Minter. “It’s still attractive,” Minter continued, “because if you can command a lion’s share of the market, you can have a blockbuster on your hands.” A Penetrable Market HAE is a rare disease, affecting approximately 1 in 50,000 people globally and around 7,000 in the U.S. About one-third of these patients are still taking C1 esterase inhibitors, Minter said, representing some of the earliest modern therapies for HAE, having been used for on-demand and preventive treatment since 2008. Shire’s Cinryze was approved in 2008 for prophylactic care, with CSL’s Berinert following in 2009 for acute treatment. CSL now has three treatments in its HAE portfolio. This includes the newly approved Andembry, which, according to a company spokesperson, inhibits the protein that plays a key role in attacks of swelling, effectively preventing the HAE cascade “at the top.” Andembry also comes with the advantages of being offered in a once-monthly dosing regimen and delivered in 15 seconds or less via an autoinjector, the spokesperson told BioSpace . In comparison, Takhzyro, administered subcutaneously, has a recommended dosing schedule of every two weeks. This fits the “treat-and-extend paradigm,” as Minter described, where drug developers vie for market share by boosting convenience. Although treatment efficacy is relevant for FDA approval, he explained that it is rare for this to be a deciding factor when it comes to switching therapies. For patients, time between dosing, safety or tolerability are typically more important. But companies in the space beg to differ. In one Ionis-sponsored poll, 9 of 10 patients with HAE who responded to the survey expressed interest in trying a new prophylactic therapy, “with nearly two-thirds reporting they hadn’t yet found the best treatment option for them,” Kyle Jenne, chief global product strategy officer at Ionis, told BioSpace by email. “In the U.S., many people living with HAE remain unsatisfied with their current treatment, continuing to experience painful, unpredictable attacks.” A Truly Differentiated Approach The emergence of new treatment modalities such as RNA-targeting therapies and gene therapies, alongside a deeper understanding of the underlying mechanisms of HAE, have created different angles of attack, Minter said. Ionis’s Dawnzera, an RNA-targeting therapy, achieved a “compelling set of data” because it achieved a high rate of attack freedom, with a 90% reduction over 24 weeks of follow-up, he noted. In addition, Ionis reported a 94% mean reduction in attack rate from baseline after one year in an open label extension study. The treatment is also dosed every four weeks and is delivered by autoinjector in 10 seconds. Dawnzera works by limiting the production of PKK, a protein that plays a role in the cascade that triggers HAE attacks, Jenne explained. Minter noted that Ionis’ launch of Dawnzera would be watched closely by analysts, as the knockdown approach is differentiated to some other treatments that have emerged in the space. On Dawnzera’s uptake so far, Jenne said only that it has been “encouraging.” Intellia’s lonvo-z, which has been said to have “potentially curative” potential, could be another differentiated option, though optimism around the candidate was slightly blunted after two recent setbacks. Earlier Phase II data reported last October showed mean monthly attack reduction of 77% and 81%, which, though effective, meant that not all patients were completely under control with one dose. Then, perhaps more significantly for the company, last month Intellia paused Phase III trials for a separate CRISPR therapy, nexiguran ziclumeran, due to safety concerns. This led Minter to ask whether patients would be interested in a permanent solution, such as gene therapy, if safety risks are present—especially if there are safer and equally effective dosed options. Overall, Minter believes the HAE pipeline is promising, particularly as analysts keep a close eye on a series of new launches to determine patient uptake. “I would never wish a disease on anyone, but if you have HAE and you’re living with the disease today, you have a much, much better prognosis than you did 15 years ago,” he concluded. “The next goal is to make these patients basically forget that they’re living with this rare disease.” .responsive-container { display: flex; flex-wrap: wrap; border: 1px solid #ededed; font-family: Helvetica, Arial, Sans-Serif; padding: 20px 20px 10px 20px; } .column-left { flex: 1; max-width: 45%; padding: 20px 20px 10px 20px; } .column-right { flex: 2; padding: 20px 20px 10px 20px; } @media (max-width: 768px) { .column-left, .column-right { max-width: 100%; flex: 100%; padding: 10px 0; } } Subscribe to ClinicaSpace! Clinical trial results, research news, the latest in cancer, cell and gene therapy hbspt.forms.create({ region: "na1", portalId: "4413123", formId: "674f4dc0-7371-4190-86cc-e1afd00b08ea", onFormSubmitted: function($form, data) { window.dataLayer = window.dataLayer || []; window.dataLayer.push({ 'event': 'GTMevent', 'event_name': 'newsletter_sign_up' }); } });

临床3期上市批准临床结果临床2期

2025-11-06

·ir.ionispharma.com

Ionis presents new data demonstrating long-term disease control with DAWNZERA™ (donidalorsen) at ACAAI 2025 Annual Meeting

">Overview Investor News Press Releases Company Statements Media Kit Events Stock Information Stock Quote & Chart Analyst Coverage Ownership Profile Financials SEC Filings Annual Reports Governance Governance Documents Committee Composition Investor Resources Investor FAQs Settlement Information Contact Us Investor Email Alerts Press Releases Company Statements Media Kit Stock Quote & Chart Analyst Coverage Ownership Profile SEC Filings Annual Reports Governance Documents Committee Composition Investor FAQs Settlement Information Contact Us DAWNZERA showed durable efficacy, demonstrating 94% overall mean HAE attack rate reduction at one year in OASISplus open-label extension study Patients in OASISplus switch study achieved 68% improvement in mean HAE attack rate compared to prior prophylactic therapy one year after switching to DAWNZERA Long-term efficacy and safety maintained up to four years in Phase 2 open-label extension CARLSBAD, Calif. --(BUSINESS WIRE)--Nov. 6, 2025-- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) today announced new long-term data for DAWNZERA™ (donidalorsen), the first and only RNA-targeted prophylactic medicine for hereditary angioedema (HAE), to be presented at the 2025 American College of Allergy, Asthma & Immunology (ACAAI) Annual Scientific Meeting in Orlando, Florida . Results demonstrate the long-term durability and safety of DAWNZERA, including new data from patients who were followed for one year in the ongoing OASISplus open-label extension (OLE) and switch cohorts, as well as new four-year results from the Phase 2 OLE study. DAWNZERA was recently approved by the U.S. Food and Drug Administration for prophylaxis to prevent attacks of HAE in adult and pediatric patients 12 years of age and older. “These new long-term data support our belief that DAWNZERA, which is now available to people living with HAE in the U.S. , is well-positioned to transform the treatment paradigm for HAE,” said Kenneth Newman , M.D., senior vice president, clinical development, Ionis. “The latest results from across our clinical studies support the strong and durable efficacy and safety profile of DAWNZERA. Presentations at the congress offer patients and physicians valuable insights to support informed treatment decisions, including when transitioning to DAWNZERA from a previous prophylactic therapy.” The OASISplus OLE cohort enrolled adult and adolescent patients continuing from the Phase 3 OASIS-HAE pivotal trial, who received DAWNZERA every four (Q4W) or every eight weeks (Q8W). At Week 52 in the OLE, DAWNZERA demonstrated a 94% and 95% mean attack reduction from baseline for patients in the Q4W (n=69) and Q8W (n=14) dosing groups, respectively. Nearly all (97%) patients reported well-controlled disease as measured by the Angioedema Control Test (AECT score ≥10) at Week 52 compared to baseline. OASISplus also included a switch cohort evaluating DAWNZERA Q4W in patients previously treated with lanadelumab, C1-esterase inhibitor or berotralstat. At Week 52, patients who switched to DAWNZERA (n=64) experienced a 68% improvement in monthly HAE attack rate compared to baseline with prior prophylactic therapy, with 90% reporting well-controlled disease as measured by the AECT. In the Phase 2 OLE study, patients treated with DAWNZERA Q4W showed a 97% mean HAE attack rate reduction over four years. The median attack-free interval was 2.7 years, and 71% of patients were attack-free for more than one year. Across all studies, DAWNZERA demonstrated a favorable long-term safety and tolerability profile. The majority of treatment-emergent adverse events (TEAEs) were mild or moderate, with no serious TEAEs related to DAWNZERA. Ionis will present a total of six posters at the congress, which are available on Ionis' website. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS DAWNZERA is contraindicated in patients with a history of serious hypersensitivity reactions, including anaphylaxis, to donidalorsen or any of the excipients in DAWNZERA. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Hypersensitivity reactions, including anaphylaxis, have been reported in patients treated with DAWNZERA. If signs and symptoms of serious hypersensitivity reactions occur, discontinue DAWNZERA and institute appropriate therapy. ADVERSE REACTIONS Most common adverse reactions (incidence ≥ 5%) are injection site reactions, upper respiratory tract infection, urinary tract infection, and abdominal discomfort. Please see full Prescribing Information for DAWNZERA. About Hereditary Angioedema (HAE) HAE is a rare and potentially life-threatening genetic condition that involves recurrent attacks of severe swelling (angioedema) in various parts of the body, including the hands, feet, genitals, stomach, face and/or throat. HAE is estimated to affect approximately 7,000 people in the U.S. About DAWNZERA™ (donidalorsen) DAWNZERA™ (donidalorsen) is approved by the U.S. Food and Drug Administration for prophylaxis to prevent attacks of hereditary angioedema ( HAE) in adult and pediatric patients 12 years of age and older. DAWNZERA is an RNA-targeted medicine designed to target plasma prekallikrein (PKK), which plays an important role in activating inflammatory mediators associated with acute attacks of HAE. For more information about DAWNZERA, visit DAWNZERA.com. About Ionis Pharmaceuticals, Inc. For three decades, Ionis has invented medicines that bring better futures to people with serious diseases. Ionis has marketed medicines and a leading pipeline in neurology, cardiometabolic and other areas of high patient need. As the pioneer in RNA-targeted medicines, Ionis continues to drive innovation in RNA therapies in addition to advancing new approaches in gene editing. A deep understanding of disease biology and industry-leading technology propels our work, coupled with a passion and urgency to deliver life-changing advances for patients. To learn more about Ionis, visit Ionis.com and follow us on X (Twitter), LinkedIn and Instagram. Forward-looking Statements This press release includes forward-looking statements regarding Ionis' business and the therapeutic and commercial potential of DAWNZERA, our commercial medicines, additional medicines in development and technologies. Any statement describing Ionis' goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties including those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis' forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis' forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. Except as required by law, we undertake no obligation to update any forward-looking statements for any reason. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2024 , and most recent Form 10-Q, which are on file with the Securities and Exchange Commission. Copies of these and other documents are available from the Company. In this press release, unless the context requires otherwise, "Ionis," "Company," "we," "our" and "us" all refer to Ionis Pharmaceuticals and its subsidiaries. Ionis Pharmaceuticals® and DAWNZERA™ are trademarks of Ionis Pharmaceuticals, Inc. View source version on businesswire.com: https://www.businesswire.com/news/home/20251105648807/en/ Ionis Investor Contact: D. Wade Walke , Ph.D. IR@ionis.com 760-603-2331 Ionis Media Contact: Hayley Soffer media@ionis.com 760-603-4679 Source: Ionis Pharmaceuticals, Inc. checkbox label label

临床结果上市批准临床2期

2025-10-24

·搜狐新闻

信达生物，最大BD的激荡与冷漠，资本变了吗。。。

来源：市场资讯（来源：成长企业常识）后浪森林研究室 | 洛上洲编辑 | 许佳维周三，信达生物披露了其历史以来最大的全球交易案，三个产品IBI363、IBI343和IBI3001，交易方是全球制药50强第十四名的武田制药，首付款12亿美元，最高交易额114亿美元。12亿美元、约85亿元人民币，是什么概念?它等于信达生物2024年总收入94.22亿的90.43%。114亿美元、约809亿元人民币，是什么概念?它等于信达生物2019-2025上半年以来六年半收入总和353亿的2.29倍。很难理解的是，资本似乎对信达生物历史最大商业行为几乎无视，股票反而下跌了1.96%。114亿美元BD，这不是一个天大的利好吗?“最大商业交易案”信达生物与武田制药全球战略合作涉及三个药物——III期临床的新一代免疫肿瘤学（IO）IBI363、III期临床的抗体偶联药物（ADC）IBI343和早期临床的B7-H3/EGFR双抗ADC IBI3001。交易条件是：信达生物获得12亿美元首付款（含1亿美元战略股权投资），潜在里程碑付款最高102亿美元，总交易额达114亿美元。IBI363：1）全球共同开发，美国市场联合开发；2）武田主导全球开发；3）信达保留大中华区权益，获得美国市场40%利润分成。IBI343：1）武田获得大中华区以外独家权益；2）信达保留大中华区开发权，获得海外销售分成。IBI3001：武田获得大中华区以外权益的独家选择权。协议明确，武田制药以现金方式按每股112.56港元认购信达生物相当于1亿美元股份。这是2025年中国最牛药物BD了，在全球也排得上前几位的号。什么药物被武田这般看好呢?IBI363是新一代IO基石疗法，它是全球首创PD-1/IL-2α-bias双特异性抗体融合蛋白，通过双重机制增强抗肿瘤活性——PD-1阻断：解除肿瘤对T细胞的免疫抑制；IL-2α偏向性激活：选择性扩增肿瘤特异性T细胞，避免传统IL-2的全身毒性。IBI363的非小细胞肺癌（NSCLC）临床数据——鳞状NSCLC：3mg/kg剂量组客观缓解率(ORR)36.7%，疾病控制率(DCR)90%，中位总生存期(mOS)未达到（12个月生存率70.9%）；腺癌：ORR 28.0%，DCR 76.0%，mOS 17.5个月。IBI363已获中国突破性疗法认定，大概率在2026年申请FDA加速审批。协议约定，双方联合主导IBI363的全球III期临床（MarsLight-11），覆盖中、美、日等600例患者，以提升信达多中心试验能力。第二个药物IBI343，是靶向CLDN18.2的ADC，用于胃/胃食管交界处腺癌、晚期胰腺癌等消化道肿瘤。据其2025ASCO和2025Nature Medicine所披露的临床数据，晚期胰腺癌6mg/kg剂量组，ORR为22.7%，DCR81.8%，mPFS5.4个月，mOS9.1个月；胃癌6-8mg/kg剂量组，ORR32.6%，DCR81.4%，mPFS5.3个月，mOS尚未成熟。该药物胰腺癌2025年1月获中国NMPA突破性疗法认定，III期临床G-HOPE-001进行中（NCT06238843）；同时该适应症在更早前的2024年6月已获得了美国FDA快速通道资格，计划开展国际多中心III期研究。与武田合作的第三个药物IBI3001是全球首创B7-H3/EGFR双抗ADC药物，增强EGFR信号阻断抑制肿瘤增殖，通过EGFR与B7-H3介导的药物内吞实现高效细胞杀伤，强效旁观者效应可杀伤EGFR+/B7-H3+及EGFR-/B7-H3-癌细胞。临床前数据，在NCI-H508（结直肠癌）、JIMT-1（乳腺癌）等异种移植模型中显示强效肿瘤抑制；治疗窗口高达40倍，半衰期282小时（小鼠模型），食蟹猴耐受性达90mg/kg/周。研发进展：2024年10月在中国开展I期临床，针对不可切除实体瘤。“讨论与分歧”这么重大的一个利好事件，为什么没有得到资本市场的响应呢?披露当日的下跌1.96%，次日下跌0.12%。最新市值1458亿港元。我在前述部分已提到过，基于武田向信达首付款12亿美元、总交易额114亿美元的重要性，前者等于信达生物2024年总收入94.22亿的90.43%，后者等于等于信达生物2019-2025上半年以来六年半收入总和353亿的2.29倍。显然，从药物研发本身来讲，IBI363、IBI343及早期的IBI3001被武田开出114亿美元商业化对价，即证实了其价值和潜力。IBI363是继PD-(L)1后，新一代肿瘤免疫治疗的热门研发方向，IBI343则是同类最优的CLDN18.2 ADC，IBI3003也是全球首创的EGFR/B7H3 ADC。所以，武田花大价钱买下的三款药物，有一个共性：首创及同类最优。这也宣告了“仿制药或盲目跟从”的价值贬值。但市场为什么不买账呢?有一种说法是，信达并非一次性出售管线权益，而后续开发与4：6利益分配比例，还需信达生物与武田按40%和60%比例分摊成本，即认为，信达生物实际获益可能不如只拿销售分成的利润，结论是“信达不划算。”这个问题该怎么理解，或该如何站在信达方面来理解呢。信达有信达的算法，IBI363关键适应症临床进度已经到了后期，后续的投入是有确定性限度的，鳞状非小细胞肺癌全球三期临床申请已获得FDA批准。而其它全球临床项目还包括结直肠癌、黑色素瘤耐药治疗，以及一线非小细胞肺癌、结直肠癌Ib/II期临床试验，花费不会少，而武田承担60%成本，这会极大缓解了信达短期经营压力。重大BD交易而导致股票猛涨逻辑失灵，大概率与市场情绪有关。9月以来，诺诚健华、维立志博、奥赛康、翰森制药对外授权后，都意外地没有得到资本市场的积极回馈。据知信达生物为该BD交易专门开了电话会，解读交易意图。董事长俞德超多次强调“信达和武田的‘Co-Co’共同开发产品模式，BD金额不是首要的，未来走向会如何才是关键。”信达的愿景是，“到2030年成为全球Biopharma。”信达表示，去年1年，很多公司表达了对其PD1/IL2双抗的合作意向，之所以选择武田，主要基于三个方面。 1）武田有实力、很积极、合作意愿高。2）武田对合作项目的认知、后续开发计划和理念都与信达高度一致。 3）武田作为一家全球化的公司，信达可以在与其全球开发以及美国共同商业化过程中，向武田学习，为成为一家国际化公司打下基础。与武田合作之后，为信达生物带来的直观成果是——2025年上半年收入43.55亿，净利润13.89亿，2025年大概率利润20多亿，再加上12亿美元首付款及股权认购款，信达的资本储备将相当丰厚。“买方武田”这是信达生物与武田制药第一次商业交易。事实上，武田2018年以来发生过多项重大交易。包括亚盛医药的“奥雷巴替尼交易”。武田制药由武田长兵卫（Takeda Chobei）1781年在日本大阪创立，它最初是一家传统草药店。经过200多年的发展，现已成为全球领先的生物制药企业。武田制药的主要股东以国际金融机构为主，前十大股东合计持股27.23%，其中美国资本占比41.87%。主要股东包括持股6.55%的美国贝莱德、持股4.11%的日本三井住友信括、持股3.70%的日本野村资管及持股3.58%的美国先锋领航等。武田制药的收入在2019年因收购夏尔（Shire）开始收入达到275亿美元，2024年收入达到了306亿美元。其收入51%来自美国，其次欧洲占21%，日本收入占14%。按全球生物医药企业收入排名，武田居全球第6位。武田产品核心覆盖肿瘤、消化、神经科学和罕见病。核心产品包括肿瘤方向的Ninlaro (恩莱瑞)、Alunbrig (安伯瑞)，消化系统方向的Entyvio (安吉优)、Movantik (纳洛酮)，罕见病方向的Takhzyro (达泽优)和Vyvanse (二甲磺酸赖右苯丙胺)。买入全球潜力药物而增加竞争力，为武田制药优先策略。除这一次与信达生物合作补充ADC和IO管线之外，它之前已有多个采购案例。武田在与信达合作的稍早前一周与美国Nabla Bio公司达成了超过10亿美元合作，意在探索AI驱动的蛋白质疗法设计——涵盖抗体、多特异性药物及难靶点攻关。Nabla Bio成立于2021年，由哈佛大学乔治·丘奇(George Church)实验室孵化，总部位于美国。核心团队融合了AI算法专家与生物医药领域资深人士。Nabla Bio建立了Joint Atomic Model (JAM) 多模态生成AI系统，JAM融合公开蛋白质数据库与Nabla自有的高通量实验数据，输入靶点信息→AI生成候选分子→虚拟筛选→湿实验验证。JAM具备三大能力——零样本设计：仅凭靶点氨基酸序列或结构，即可生成皮摩级结合力的抗体；快速迭代：从设计到实验室验证仅需3-4周，远快于传统流程；跨膜蛋白突破：已成功针对Claudin-4、CXCR7等多次跨膜蛋白设计抗体。2024年4月，武田制药、安斯泰来和三井住友银行联合成立专注日本早期药物发现的合资企业，以此弥合学术研究与商业化之间的“死亡谷”。合资企业计划在2024-2025年重点孵化5-8个涵盖肿瘤、神经退行性疾病的早期项目。这一年，其以13亿美元获得同时亚盛医药耐立克（奥雷巴替尼）大中华区之外的全球开发及商业化权益，并认购7500万美元股份成为第二大股东。2019年约以34亿元人民币收购了英国Shire公司，这个创日本药企并购纪录的行为，获得了Shire的血液病、免疫及神经疾病管线，包括血友病药物。合作还包括：1）与上药控股合作在中国市场推广两款新药“助因止”和“维因止”，目标至2030年使中国成为武田全球第二大市场。3）以11.3亿美元获得和黄医药交易呋喹替尼海外权益。武田唯一的失败案例是以5.25亿美元收购Maverick Therapeutics公司获得TAK-186（EGFR TCE）和TAK-280（B7-H3 TCE）项目，后来因研发风险而终止。“疾速的信达”信达生物这次的114亿美元交易行为，正加速推动其成为全球创新药的皇冠企业。往前追溯，2025年其还与罗氏达成了一项10.8亿美元的DLL3靶向ADC药物IBI3009交易事件，其将该药大中华区之外的全球权益授权给了罗氏，首付款8000万美元，总里程碑付款最高10亿美元。该药针对的是晚期小细胞肺癌，已获中美澳三地IND批准，完成Ⅰ期临床首例给药。信达历史上合作最多的是礼来，最重要的莫过于玛仕度肽。事实上，双方不止于产品层面商业合作，而礼来还是持股信达生物约5%的股东。玛仕度肽最初由礼来研发，2019年授权信达生物获得中国区独家开发和商业化权益。礼来选择开发同类药物替尔泊肽（GIP/GLP-1双靶点），而将玛仕度肽的权益转让给信达，形成差异化布局。2025年6月，减重适应症在中国获批，9月糖尿病适应症获批。这是信达生物最重要的核心价值之一。2015年首次合作：双方共同开发信迪利单抗（PD-1抑制剂），礼来支付5600万美元首付款及最高4亿美元里程碑付款。2022年深化合作：信达获得礼来雷莫西尤单抗（VEGFR-2抑制剂）和塞普替尼（RET抑制剂）在中国大陆的独家商业化权益，并取得匹妥布替尼（BTK抑制剂）的优先谈判权。2024年12月：就匹妥布替尼达成商业化协议，信达负责中国市场的销售推广，礼来主导研发及医学事务。另外，重点合作项目还有：1）匹妥布替尼（捷帕力®）：全球首个非共价BTK抑制剂，2024年在中国获批用于套细胞淋巴瘤治疗，信达负责进口与分销；2）信迪利单抗（达伯舒®）：首个进入医保的国产PD-1抑制剂，2023年获批用于EGFR阳性非小细胞肺癌治疗；3）雷莫西尤单抗与塞普替尼：分别针对胃癌和RET基因突变肿瘤，信达拥有中国商业化权益。创始人俞德超2011年创立了信达生物，其毕业于中国科学院遗传学博士，在此之前他担任过成都康弘生物总裁、首席执行官及董事。更早前，俞德超所在的两家生物医药企业去了美国纳斯达克上市，一家是其担任研发副总裁的AGTC，一家是其担任副总裁的CEGE。信达生物股权结构主要包括创始人、国际投资机构及员工持股平台。俞德超持股6.58%，淡马通过TLS BETA PTE. LTD.、Elbrus Investments持股约7.46%，美国资本集团通过Capital Group Companies及Capital Group International Inc.持股约6.92%。企业核心理念来源于“始于信，达于行”。信达生物已有15个药物上市，最核心的是达伯舒，2024年销售收入超过了40亿元，等于信达生物总收入的50%之上。信达曾公开表示，预期2027年收入达到200个亿。从现在看来，2026、2027这两年等于要重造一个现在规模的信达了。需要进一步理解康华生物，请参阅：信达生物，悄然越过平衡点。。。返回搜狐，查看更多

引进/卖出临床3期ASCO会议突破性疗法抗体药物偶联物

100 项与拉那利尤单抗相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11094	拉那利尤单抗	B06AC05	DB14597

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
遗传性血管性水肿	美国	Takeda Pharmaceuticals U.S.A., Inc.	2018-08-23

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
血管性水肿	临床3期	美国	Shire Plc	2021-02-05
血管性水肿	临床3期	日本	Shire Plc	2021-02-05
血管性水肿	临床3期	加拿大	Shire Plc	2021-02-05
血管性水肿	临床3期	法国	Shire Plc	2021-02-05
血管性水肿	临床3期	德国	Shire Plc	2021-02-05
血管性水肿	临床3期	匈牙利	Shire Plc	2021-02-05
血管性水肿	临床3期	意大利	Shire Plc	2021-02-05
血管性水肿	临床3期	荷兰	Shire Plc	2021-02-05
血管性水肿	临床3期	波兰	Shire Plc	2021-02-05
血管性水肿	临床3期	西班牙	Shire Plc	2021-02-05

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临床结果

适应症

分期

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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


NCT03845400 (EMPOWER, Pubmed \| Adv Ther)	临床4期	遗传性血管性水肿 C1 inhibitor deficiency type 1	109	Newly treated with Lanadelumab	壓觸構網願範廠遞觸網(憲獵願網築製願夢蓋構) = 齋願觸鏇餘獵製鏇鹹壓繭鏇蓋積淵鏇鏇膚膚淵 (醖廠膚鹽齋願廠齋選觸, 0.34 ~ 2.50)	积极	2025-06-12
NCT03845400 (EMPOWER, Pubmed \| Adv Ther)	临床4期	遗传性血管性水肿 C1 inhibitor deficiency type 1	109	Established on Lanadelumab	壓觸構網願範廠遞觸網(憲獵願網築製願夢蓋構) = 獵鏇選淵鹹廠繭繭顧鹹繭鏇蓋積淵鏇鏇膚膚淵 (醖廠膚鹽齋願廠齋選觸, 0.10 ~ 0.30)	积极	2025-06-12
NCT04206605 (CASPIAN Study, Pubmed \| Front Immunol)	临床3期	遗传性血管性水肿Ⅲ型	77	Lanadelumab 300 mg every 2 weeks	積鬱淵衊壓廠範餘繭鹽(願願蓋艱壓齋繭蓋製構) = 鏇築蓋夢選窪範構糧範遞鬱淵鹹憲夢簾網獵觸 (夢願鏇願選鬱積網廠獵 )	-	2025-05-21
NCT04206605 (CASPIAN Study, Pubmed \| Front Immunol)	临床3期	遗传性血管性水肿Ⅲ型	77	Placebo	積鬱淵衊壓廠範餘繭鹽(願願蓋艱壓齋繭蓋製構) = 顧選觸築簾構壓願淵遞遞鬱淵鹹憲夢簾網獵觸 (夢願鏇願選鬱積網廠獵 )	-	2025-05-21
NCT04130191 \| NCT03845400 (Phase 4 studies (EMPOWER, ENABLE), Pubmed \| Pediatr Allergy Immunol)	临床4期	遗传性血管性水肿	-	Lanadelumab 300 mg	餘膚簾膚範選築網積鏇(齋醖網鏇願獵艱積顧鑰) = Nine new patients reported 42 TEAEs, mostly mild/moderate in severity, with 3 TEAEs reported as serious. Seven established patients reported 12 TEAEs (all mild/moderate and non‐serious). No TEAEs were related to lanadelumab. 鹹獵製築襯鑰範窪繭繭 (廠衊製餘積壓遞窪願糧 )	积极	2025-04-01
NCT05460325 (CTgov)	临床3期	遗传性血管性水肿	20	Lanadelumab	壓構網鏇鹹選製淵夢積 = 願製衊獵鬱衊遞構簾製遞鬱窪鏇鹽鏇鹹積艱憲 (繭鑰網膚衊積壓範範餘, 鹹窪鏇鬱範積製醖顧餘 ~ 淵齋衊蓋膚遞願範餘衊) 更多	-	2024-12-13
NCT02741596 (HELP Study and HELP OLE, Pubmed \| Ann Allergy Asthma Immunol)	临床3期	遗传性血管性水肿	21	Lanadelumab 300 mg	鹹鑰顧襯廠膚築顧繭襯(壓衊窪夢鏇壓衊遞壓糧) = 12 (57.1%) patients reported treatment-related treatment-emergent adverse events; however, there were no treatment-related serious adverse events. 選範鬱淵齋範齋繭艱構 (蓋鑰選衊餘願選憲築製 ) 更多	积极	2024-12-01
NCT04444895 (CTgov)	临床3期	血管性水肿 \| 遗传性血管性水肿Ⅲ型	73	Lanadelumab	鹹廠蓋廠淵製蓋艱鏇築(壓網繭範膚願蓋鏇願網) = 網構鏇遞選顧膚網鏇願夢壓齋廠顧夢範願顧鑰 (觸築積範積憲廠艱鏇築, 襯衊觸選壓鬱淵遞顧鏇 ~ 遞壓簾構鑰積廠衊鑰壓) 更多	-	2024-06-17
NCT03845400 (EMPOWER, AAAAI2024) 人工标引	临床4期	遗传性血管性水肿	113	New users of Lanadelumab	鏇糧窪構糧鹹壓範鬱網(鬱艱窪鹹醖齋餘廠觸鹹) = 壓廠糧艱艱築網艱遞鹹鹽選齋範夢觸觸構築鏇 (醖積糧壓壓襯鏇鏇積蓋, 0.88 ~ 2.81)	积极	2024-02-23
NCT03845400 (EMPOWER, AAAAI2024) 人工标引	临床4期	遗传性血管性水肿	113	Prevalent users of Lanadelumab	鏇糧窪構糧鹹壓範鬱網(鬱艱窪鹹醖齋餘廠觸鹹) = 醖衊窪廠艱壓繭鹽夢遞鹽選齋範夢觸觸構築鏇 (醖積糧壓壓襯鏇鏇積蓋, 0.25 ~ 0.70)	积极	2024-02-23
NCT04130191 \| NCT03845400 (EMPOWER, AAAAI2024) 人工标引	临床4期	遗传性血管性水肿 HAE Type I	19	lanadelumabumab users	鬱齋鑰夢糧醖艱鏇遞窪(廠襯獵選積築廠艱選觸) = There were 29 treatment-emergent adverse events (TEAEs; mild/moderate:82.8%, non-serious:93.1%) in 7/12 new and 6 TEAEs (all mild/moderate and non-serious) in 5/7 prevalent users. No TEAEs were related to lanadelumab. 構艱構築鹹餘選醖夢簾 (選窪觸獵艱鬱廠衊遞積 )	积极	2024-02-23
NCT05147181 (CHOPIN, AAAAI2024) 人工标引	N/A	遗传性血管性水肿一线 HAE type-1	16	Lanadelumab	顧齋窪膚鏇齋餘鏇簾壓(壓觸選憲網構獵鑰鏇獵) = 製鬱窪醖醖衊鏇構襯願蓋獵範築淵夢鹽鹽鹹齋 (選憲齋鬱選獵艱糧壓淵 ) 更多	积极	2024-02-23
AAAAI2024	N/A	遗传性血管性水肿	-	Lanadelumab	築構積夢餘糧顧壓積鏇(願網蓋餘遞夢願醖淵獵) = 襯網鹽獵鹽襯憲壓築餘蓋遞艱膚淵鬱糧壓淵繭 (餘鬱網憲鏇製積餘窪壓 ) 更多	-	2024-02-23
AAAAI2024	N/A	遗传性血管性水肿	-	Lanadelumab (Other Long-Term Prophylaxis)	築構積夢餘糧顧壓積鏇(願網蓋餘遞夢願醖淵獵) = 構壓襯壓範餘窪鹽醖鹹蓋遞艱膚淵鬱糧壓淵繭 (餘鬱網憲鏇製積餘窪壓 ) 更多	-	2024-02-23