Phase I Trial of GB1211 (Selvigaltin), an Oral Galectin-3 (Gal-3) Inhibitor, Combined With Standard of Care Treatment in Relapsed/Refractory Multiple Myeloma (RRMM)

This phase I trial studies the side effects and best dose of selvigaltin when given together with standard of care treatment (daratumumab-hyaluronidase, carfilzomib, dexamethasone) in treating patients with multiple myeloma that has come back after a period of improvement (relapsed) or that does not respond to treatment (refractory). Selvigaltin works by blocking the activity of a protein called galectin-3. Galectin-3 is involved in various cellular processes, including inflammation and tissue scarring, which is associated with worse outcomes in several forms of cancer. By blocking the activity of galectin-3, selvigaltin may help reduce inflammation and tissue scarring. Daratumumab-hyaluronidase is a drug composed of daratumumab and hyaluronidase. Daratumumab is in a class of medications called monoclonal antibodies. It binds to a protein called CD38, which is found on some types of immune cells and cancer cells, including myeloma cells. Daratumumab may block CD38 and help the immune system kill cancer cells. Hyaluronidase helps deliver the daratumumab to CD38-expressing cancer cells. Carfilzomib may stop the growth of cancer cells by blocking some of the enzymes needed for cell growth. Dexamethasone is in a class of medications called corticosteroids. It is used to reduce inflammation and lower the body's immune response to help lessen the side effects of chemotherapy drugs. Giving selvigaltin with standard of care treatment may be safe, tolerable, and/or effective in treating patients with relapsed or refractory multiple myeloma.

开始日期2025-09-01

申办/合作机构

Roswell Park Comprehensive Cancer Center

NCT06918990

/ Recruiting临床2期IIT

Targeting the B Cell Response to Treat Antibody-Mediated Rejection With Carfilzomib and Belatacept (CarBel)

The purpose of this study is to see:
1. If using these two drugs (carfilzomib and belatacept) together is safe
2. If the use of these two study drugs in addition to the usual immunosuppression for kidney transplant patients can improve your transplanted kidney function by lowering the antibodies you have against your transplanted kidney
3. If the study drugs effect the immune cells that were responding to your donor kidney. And, whether blood or urine tests can measure signs of inflammation and kidney cell injury
4. If using new computer techniques can help describe important changes seen on biopsy in your donated kidneys
The primary objective is to assess the efficacy of carfilzomib and belatacept therapy when added to current treatment with steroids and maintenance immunosuppression, compared to conventional treatment alone, to improve the clinical outcome of renal transplant patients with active and chronic - active ABMR occurring more than 6 months after renal transplantation or less than 6 months post-transplant with persistent refractory Antibody-Mediated Rejection (ABMR)

开始日期2025-08-25

申办/合作机构

National Institute of Allergy & Infectious Diseases

NCT06892522

/ Recruiting临床1/2期

A Phase 1/2, Open-Label, Platform Study to Evaluate Safety and Efficacy of Etentamig Monotherapy or Etentamig Combinations in Subjects With Multiple Myeloma

Multiple myeloma (MM) is a cancer of the blood's plasma cells. The cancer is typically found in the bones and bone marrow (the spongy tissue inside of the bones) and can cause bone pain, fractures, infections, weaker bones, and kidney failure. Treatments are available, but MM can come back (relapsed) or may not get better (refractory) with treatment. This is a study to determine the safety, efficacy, and pharmacokinetics of Etentamig in adult participants with MM.
Etentamig is an investigational drug being developed for the treatment of MM. This study is broken into 4 substudies and each substudy consists of a dose escalation phase and dose expansion phase. Participants will receive escalating doses of etentamig alone or in combination with daratumumab and lenalidomide (DR), carfilzomib and dexamethasone (Kd) or lenalidomide (R). This will be followed by etentamig at the dose levels established during the escalation phases alone or in combination with DR, Kd, R. The participants can also receive daratumumab, lenalidomide and dexamethasone (DRd), R, or daratumumab, carfilzomib, and dexamethasone (DKd) as a comparator in the dose expansion phases. Around 440 adult participants with MM will be enrolled at approximately 50 sites worldwide
In all substudies, participants will receive escalating doses of etentamig as Intravenous (IV) infusions, alone or in combination with DR, R or Kd, followed by IV infusions of etentamig at the dose levels established during the escalation phases alone or in combination with IV and oral DRd, DKd, or R. The study duration is approximately 130 months.
There may be higher treatment burden for participants in this trial compared to their standard of care. Participants will attend regular visits during the study at a hospital or clinic. The effect of the treatment will be checked by medical assessments, blood tests, checking for side effects and questionnaires.

开始日期2025-06-30

申办/合作机构

AbbVie, Inc.

100 项与卡非佐米相关的临床结果

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100 项与卡非佐米相关的转化医学

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100 项与卡非佐米相关的专利（医药）

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2,427

项与卡非佐米相关的文献（医药）

2025-12-31·Hematology

Pharmacovigilance and signal detection of adverse drug events associated with proteasome inhibitors in multiple myeloma: a real-world analysis using the FAERS database

Article

作者: Gao, Yao ; Zhang, Meiling ; Luo, JunYun ; Zeng, Yingjian ; Chen, Shupeng

BACKGROUND:

Proteasome inhibitors (PIs) such as Bortezomib, Carfilzomib, and Ixazomib have significantly improved outcomes in multiple myeloma (MM), but their real-world safety profiles require further exploration.

OBJECTIVE:

To assess adverse drug events (ADEs) associated with Bortezomib, Carfilzomib, and Ixazomib in MM patients using the FDA Adverse Event Reporting System (FAERS), and to identify new ADE signals not listed in product labels.

METHODS:

A total of 47,310 ADE reports (Bortezomib: 23,843; Carfilzomib: 9,835; Ixazomib: 13,632) were analyzed from FAERS (2004 - Q4 2024). Signal detection was conducted using Reporting Odds Ratio (ROR), Proportional Reporting Ratio (PRR), Bayesian Confidence Propagation Neural Network (BCPNN), and Multi-item Gamma Poisson Shrinker (MGPS). Onset time was evaluated using Weibull survival analysis.

RESULTS:

All three PIs were associated with significant ADE signals across multiple system organ classes, notably in the hematologic, nervous, cardiovascular, hepatobiliary, and gastrointestinal systems. Newly identified ADEs included muscular weakness (Bortezomib), pancytopenia (Carfilzomib), and cognitive disorder (Ixazomib). Onset time analysis showed that most ADEs occurred within the first 30 days of treatment.

CONCLUSION:

This study highlights both known and newly detected ADEs linked to PIs, emphasizing the importance of early-phase monitoring and ongoing pharmacovigilance. The findings provide crucial real-world evidence for clinical risk management and future label updates.

2025-10-01·VETERINARY JOURNAL

Canagliflozin: Pharmacokinetics, tolerability and glucose/insulin effects of supratherapeutic doses in healthy horses

Article

作者: Fjordbakk, Cathrine ; Bergquist, Jonas ; Lilliehöök, Inger ; Erkas, Malin ; Ekstrand, Carl ; Löwgren, Minerva ; Michanek, Peter ; Bröjer, Johan ; Hedeland, Mikael

Sodium-glucose co-transporter 2 inhibitors like canagliflozin (CFZ) have shown promise in preventing hyperinsulinemia-associated laminitis in horses, but data on pharmacokinetics, tolerability, and controlled studies are limited. This randomized, open-label, placebo-controlled, crossover study evaluated these aspects of CFZ treatment in eight healthy Standardbred mares. Each horse received single supratherapeutic oral doses of CFZ (1.8 mg/kg or 3.6 mg/kg) and placebo, with a two-week washout between treatments. A graded glucose infusion (GGI) was administered post-treatment to evaluate glucose and insulin responses. Plasma CFZ, glucose, insulin, urinary glucose, serum biochemistry, and urinalysis samples were collected over 72 h post-treatment. For CFZ 1.8 mg/kg, median C_max was 2623 ng/mL, T_max 2.2 h, and T_1/2Z 21.8 h; for 3.6 mg/kg, C_max was 4975 ng/mL, T_max 2.8 h, and T_1/2Z 23.0 h. The pharmacokinetics of CFZ displayed dose-proportionality across the two tested doses. Insulin and glucose responses to a GGI, measured by the area under the concentration-time curve (AUC), were similar between CFZ doses but significantly reduced compared to placebo (p < 0.001). Specifically, mean glucose AUC for CFZ treatments was approximately 14-15 % lower, and mean insulin AUC 22-29 % lower, than for placebo. For CFZ-treated horses, mean urinary glucose concentrations ranged from 277 to 347 mmol/L at 24, 48, and 72 h post-administration, with no significant differences between dose levels. No clinical signs of adverse effects were observed, although a significant increase in GLDH levels compared to placebo (p < 0.05) was observed with the CFZ 3.6 mg/kg dose.

2025-10-01·JOURNAL OF PHARMACEUTICAL SCIENCES

Pulmonary drug delivery of clofazimine: A route of administration and pharmacokinetics guided repositioning strategy against drug resistant tuberculosis informed by use for other disease indications

Review

作者: Bhatti, Gurdeep ; Datta, Pallab ; Singh, Nidhi ; Reddy, Kolimi Prashanth

Current oral therapeutic regime of clofazimine (CFZ) in tuberculosis treatment is limited by poor pharmacokinetics, long t_1/2, and especially exposure-related systemic toxicity. This review critically evaluates pulmonary administration as a targeted delivery approach to reduce the dose and improve therapeutic efficacy. From the primarily anti-leprotic indication, CFZ has evolved for the treatment of multidrug-resistant tuberculosis (MDR-TB), nontuberculous mycobacterial (NTM) diseases, small-cell lung cancer, and as an adjunct in therapies targeting immune checkpoints such as programmed cell death protein 1 (PD-1) and cytotoxic T-lymphocyte-associated antigen 4 (CTLA-4) in cancer patients. Despite broad-spectrum activity, clinical effectiveness is constrained by its poor physicochemical and pharmacokinetic properties. Due to its high lipophilicity, orally administered CFZ has a high volume of distribution (1470 L) and tends to accumulate in fatty tissues, resulting in long times to reach steady-state concentrations, sub-therapeutic drug levels at the infection site, increased risk of systemic toxicity and triggering drug resistance over time. Although novel delivery systems administered through the oral route are also explored extensively, they are inadequate to address the concerns of sustained systemic exposure. Pulmonary delivery enables direct deposition at the diseased site, provides higher local concentrations, and reduces off-target effects. Poor solubility and high lipophilicity are the principal biopharmaceutical disadvantages of CFZ for oral formulation development. However, these properties are advantageous for pulmonary delivery in facilitating prolonged lung retention and reduced dosing frequency. This review evaluates the current advantages and challenges of CFZ inhalation versus oral formulations, discusses their pharmacokinetic profiles, and explores pulmonary macrophage uptake strategies to optimize tuberculosis treatment outcomes.

440

项与卡非佐米相关的新闻（医药）

2025-09-05

·PRNewswire

Florida Cancer Specialists & Research Institute Clinical Expertise And Real-World Evidence Capabilities Advancing Treatment of Blood Cancers

Abstracts Presented at Worldwide Gathering of Hematology ExpertsSOHO 2025 abstracts featuring FCS research: CT087, MM353, MPN566, MM353 FORT MYERS, Fla., Sept. 5, 2025 /PRNewswire/ -- Physicians, clinicians, and senior leaders from Florida Cancer Specialists & Research Institute, LLC (FCS) are among the internationally recognized experts presenting advancements in the treatment of hematologic malignancies at the Society for Hematologic Oncology (SOHO) 2025 annual meeting this week in Houston. Continue Reading Lucio N. Gordan, MD. "We are driving advances in blood cancers by accelerating development of new therapies." Post this Florida Cancer Specialists & Research Institute presenting groundbreaking hematologic research at the annual Society of Hematologic Oncology. FCS President & Managing Physician Lucio N. Gordan, MD. "By combining the care of more than one million patients with our deep clinical, research, and informatics expertise, we are driving meaningful advances in understanding blood cancers and accelerating the development of new therapies." FCS Director of Real-World Evidence Amanda Warner, MS, RN is the presenting author of two abstracts: Myelofibrosis Observational Real-World Experience in Community Oncology (MORE-CO): A Retrospective Analysis of Baseline Clinical Factors Impacting Overall Survival | Co-authors are Lucio N. Gordan, MD, Arsh Singh, MD, FCS hematologist and medical oncologist, and Trevor Heritage, PhD, FCS clinical technology director. Real-World Treatment Patterns and Outcomes of Luspatercept in Treatment-Naïve Patients with Lower-Risk Myelodysplastic Syndromes (LR-MDS) in a Large Community Oncology Practice | Co-authors are Gustavo Fonseca, MD, FACP, FCS director of research & clinical trials, and Christian Okitondo, PhD, FCS real-world evidence biostatistician. "FCS in recent years has made forward-thinking investments in proprietary real-world datasets and advanced data science," noted Heritage. "Our comprehensive approach transforms real-world clinical data into high-quality insights that are driving improvements in clinical decision making, closing gaps in care, and enhancing patient experiences and outcomes." Additionally, the following abstract presentations feature research results with FCS participation: Phase 2 SubQSA Study of Subcutaneous (SC) Isatuximab (Isa) Administered by an On-Body Delivery System With Weekly Carfilzomib and Dexamethasone (Kd) in Patients with Relapsed/Refractory Multiple Myeloma (RRMM) – co-authored by Alexander Philipovskiy, MD, PhD, FCS medical oncologist and hematologist specializing in drug development. Preliminary Insights into CAR-T Therapy for Melanoma: A Systematic Review – co-authored by Uma Iyer, MD, FCS medical oncologist and hematologist. At any point in time, more than 150 active clinical trials are underway at FCS, including early-phase studies at one of three drug development units. Research conducted at FCS is made possible through valuable relationships with Sarah Cannon Research Institute, one of the world's leading clinical research organizations, and Paradigm Health, an AI-enabled clinical trial matching and recruitment platform. In recent years, the majority of new cancer drugs approved for use in the U.S. were studied in clinical trials with FCS participation, prior to approval. About Florida Cancer Specialists & Research Institute, LLC: (FLCancer.com) For more than 40 years, Florida Cancer Specialists & Research Institute (FCS) has embraced innovation to deliver world-class care and drive the dramatic transformation of oncology care through its robust clinical research program. FCS provides patients with access to a wide range of clinical trials, positioning it as a leader in research among private oncology practices in Florida and across the country. In fact, before receiving FDA approval, the majority of new cancer drugs in the U.S. were first made available to patients through participation in clinical trials at FCS. Our outstanding team of highly trained and dedicated physicians is committed to delivering tailored treatment plans that make the best use of cutting-edge precision oncology advancements to enhance patient outcomes. SOURCE Florida Cancer Specialists & Research Institute WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床2期

2025-08-31

·汇聚南药

百亿骨髓瘤市场的「新王炸」，来了

在全球百亿骨髓瘤市场的蓝海中，BCMA/CD3双抗正成为改写治疗格局的“王牌武器”。目前该赛道已形成强生、辉瑞和再生元三足鼎立的局面，角逐不断升级。 01 百亿骨髓瘤市场掘金多发性骨髓瘤作为血液系统常见恶性肿瘤，其全球治疗市场正呈现扩张态势。2024年该疾病领域市场规模已达283亿美元，并预计以年均6.6%的增速持续增长，根据行业预测，到2032年多发性骨髓瘤领域市场规模有望突破521亿美元。全球多发性骨髓瘤市场目前已经形成传统药物、生物制剂和细胞疗法并进的竞争格局。 2024-2033年主要市场多发性骨髓瘤关键治疗药物的预测销售额图片来源：Nature reviews.Drug discovery,24(4),244–245. 在核心治疗药物中，免疫调节剂（IMiDs）仍占据重要地位。来那度胺作为第二代药物，因其较低的神经毒性及良好的口服便利性，持续占据市场主导，尤其在中国的IMiDs类市场中约占四分之三的份额。第三代产品泊马度胺自2020年上市后也快速放量，显示出显著的临床替代潜力。尽管该类药品面临专利到期所带来的挑战，但其贯穿多线治疗的基础地位仍将支撑其在2033年时占据约38%的市场份额。与此同时，以CD38为靶点的单克隆抗体表现尤为突出。强生公司的达雷妥尤单抗自2015年获批以来，适应症已全面覆盖从后线到一线的治疗场景，2023年全球销售额高达97亿美元，更在2024年突破百亿美元达到116亿美元，成为该领域首个跨入“百亿级别”的明星药物，显著拉动了整个市场的增长预期。另一方面，蛋白酶抑制剂市场则出现结构性分化。早年一线选择的硼替佐米受到国家集采政策冲击，销售额明显下滑；卡非佐米因存在心脏及肾脏毒性问题，市场表现持续低迷；口服制剂伊沙佐米虽提高了用药便利性，但联用方案较为单一，限制了其进一步发展。传统化疗药物则因不良反应较多，正在被更加精准的靶向药物加速替代。而在创新疗法层面，以BCMA为靶点的CAR-T细胞治疗进展迅速，目前全球已有8款治疗多发性骨髓瘤的BCMA靶点药物上市。仅CAR-T产品就有4款获批上市。其中，西达基奥仑赛表现尤为亮眼，2022年上市后销售额快速攀升，2024年接近10亿美元，展现出极强的市场竞争力。国内获批上市的有驯鹿生物的伊基奥仑赛和科济生物/华东医药的泽沃基奥仑赛。随着CAR-T疗法临床试验阶段不断前移，其在早期治疗中的应用潜力正在被持续挖掘。 02 BCMA/CD3双抗赛道角逐升级近年来，BCMA/CD3双抗赛道竞争激烈，成为全球药企争夺的焦点。目前全球在研的BCMA/CD3双抗已超过10款，随着再生元制药Lynozyfic于2025年7月获FDA加速批准，该赛道已形成强生、辉瑞和再生元三足鼎立的局面，角逐不断升级。多发性骨髓瘤适应症BCMA/CD3双抗药物数据来源：药智数据、公开资料整理其中，强生旗下的特立妥单抗（Teclistamab）率先于海外获批，并于2024年6月获得NMPA批准，用于治疗既往接受过至少三线治疗的RRMM成人患者。该获批基于其关键I/II期MajesTEC-1研究数据，结果显示其客观缓解率（ORR）达到63%，且显示出持久的疗效，在18个月时仍有57%的患者处于缓解状态。基于显著的临床价值，强生对其全球销售峰值给出超过50亿美元的预期，充分印证了该靶点的市场潜力。紧随其后，辉瑞的埃纳妥单抗（Elranatamab）也于2025年3月在中国获批相同适应症。其支持的MagnetisMM-3试验数据显示出深度且持久的疗效，30个月DOR率为61.0%，mPFS为17.2个月，mOS为24.6个月，且安全性可控，未出现新的风险信号。这两款先行产品的接连上市，正式开启了国内百亿级骨髓瘤市场双抗疗法的角逐。 2025年4月，再生元Linvoseltamab获欧盟委员会（EC）有条件批准，用于治疗既往至少接受过三类药物（包括蛋白酶体抑制剂、免疫调节剂及抗CD38单抗）治疗，且在最后一种治疗后出现疾病进展的复发/难治性MM成人患者；2025年7月，再生元Linvoseltamab获FDA批准用于治疗复发或难治性（R/R）多发性骨髓瘤（MM）成年患者。关键性Ⅰ/Ⅱ期临床研究LINKER-MM1研究数据显示：ORR为70%，其中45%的患者达到完全缓解（CR）或更佳疗效，首次缓解的中位时间为0.95个月。尽管再生元较竞争对手晚进入市场，但其通过临床优势及响应适应每月给药方案实现了差异化优势。值得注意的是，下一代技术布局已经展开，艾伯维斥资7亿美元预付款取得了Ichnos Glenmark Innovation开发的三特异性抗体ISB 2001的权利。该药物同时靶向CD38、BCMA和CD3，相比双特异性抗体可能对BCMA表达量低的肿瘤细胞具有更强结合力，从而降低耐药性风险。一期临床数据显示，ISB 2001在35名复发或难治型多发性骨髓瘤患者中取得了79%的总反应率，近30%患者达到完全缓解。 03 国产力量崛起随着国产创新药企持续发力，中国研发力量正在BCMA/CD3双特异性抗体赛道快速崛起。在研发进展方面，国产BCMA/CD3双抗药物展现出令人瞩目的临床效果。康诺亚的CM336在临床研究中显示52%的患者达到严格完全缓解，目前处于II期临床阶段。2025年6月，其治疗难治性自身免疫性溶血性贫血（AIHA）的突破性案例荣登《新英格兰医学杂志》，两例多次治疗失败的患者经CM336挽救治疗后快速缓解并维持疗效超6个月，安全性良好；智翔金泰的GR1803通过高亲和力靶向BCMA抗原、低亲和力结合CD3的策略，精准激活T细胞杀伤肿瘤细胞。2024年EHA年会数据显示，其对既往经过至少3线治疗的多发性骨髓瘤患者显示出显著客观缓解率，髓外浆细胞瘤患者也表现出较突出疗效。此外，岸迈生物的EMB-06正在开展针对复发或难治多发性骨髓瘤的I/II期研究；正大天晴的TQB2934也新增用于治疗成人系统性轻链型淀粉样变的适应症，为罕见病患者带来新希望。凭借卓越的临床数据，中国研发力量逐渐赢得国际市场的广泛认可。 2024年9月，岸迈生物将其BCMA/CD3双抗EMB-06的全球权益授予Vignette Bio，交易首付款及股权对价达6000万美元，潜在里程碑付款更高达5.75亿美元； 2024年11月，维立志博则凭借其创新性的CD19/BCMA/CD3三抗LBL-051，与Aditum Bio共同创立Oblenio Bio公司并获得资金支持以加速临床推进；同一时期，康诺亚与Platina Medicines达成协议，授予其BCMA/CD3双抗CM336海外权益，获得1600万美元首付款及最高6.1亿美元里程碑款； 2025年1月，先声药业与艾伯维就GPRC5D-BCMA-CD3三特异性抗体SIM0500达成的协议，潜在交易金额高达10.55亿美元，进一步丰富了国产双抗药物的布局。 2025年6月，智翔金泰就GR1803以7.12亿美元总金额授权Cullinan覆盖海外市场，探索GR1803在自免领域的应用，开拓增量市场。 04 结语随着BCMA/CD3双抗赛道的持续升温，中国药企崭露头角，多发性骨髓瘤治疗即将进入一个更精准、更可及的新时代。参考资料 1.药智数据 2.https://www.genrixbio.com/#/science?classId=class1 3.Cohen,Y.C.et al.Talquetamab plus Teclistamab in Relapsed or Refractory Multiple Myeloma.New England Journal of Medicine 392,138–149(2025). 4.Morris,Julia L M,and Rachel M Webster.“The multiple myeloma drug market.”Nature reviews.Drug discovery vol.24,4(2025):244-245. 5.https://www.nmpa.gov.cn/ 喜欢我们文章的朋友点个“在看”和“赞”吧，不然微信推送规则改变，有可能每天都会错过我们哦~ 免责声明 “汇聚南药”公众号所转载文章来源于其他公众号平台，主要目的在于分享行业相关知识，传递当前最新资讯。图片、文章版权均属于原作者所有，如有侵权，请在留言栏及时告知，我们会在24小时内删除相关信息。信息来源：药智网往期推荐本平台不对转载文章的观点负责，文章所包含内容的准确性、可靠性或完整性提供任何明示暗示的保证。

细胞疗法上市批准免疫疗法专利到期加速审批

2025-08-28

·求实药社

63亿现金入袋！特许权交易还是百济玩得溜！

创新药又双叒叕要续上了？今天，在市场沉浸在上政指数越来越接近4000点的余热里时，百济冷不丁抛出一则公告：宣布有家爱尔兰的公司要掏8.85亿美元（约63.32亿人民币）购买百济旗下一款双抗产品的“未来潜在收益”。要知道，这个数字相当于百济核心产品泽布替尼全球至少一个Q的销售额。那么问题来了，这笔账什么时候能到百济手里？这类交易和既往的BD有何不同？以及，Tarlatamab这款本来属于安进的产品，为何全球的“销售权益”在百济手里？ -01- Royalty Pharma何许人也？这是一家于1996年由一位PE投资人Pablo Legorreta创立的公司，虽然取了个“pharma”的名字，但是很明显，公司本质上是一家投资机构。据说是因为当年Legorreta 观察到，生物医药行业充满了巨大的机遇，但新药研发也伴随着极高的风险、长周期和巨大的资金需求。许多大学、研究机构和中小型生物科技公司虽然拥有极具潜力的在研药物或已上市药品的专利，但它们急需现金来支持后续的研发或商业化。于是，Royalty Pharma就扮演了创新药“商业化阶段投资人”的角色。它也有一个专业的名词，叫做：特许权交易（RBF，Revenue-based Financing）。这种模式和VC的本质没有区别，只不过相比于很多一级基金赌的是临床数据，而Royalty Pharma赌的是“商业化放量”。 Royalty Pharma这家公司的“投资成绩”一点也不差。《十亿美元分子》的主角Vertex旗下的核心产品——囊性纤维化药物（Kalydeco, Trikafta）、一代BTK之王——强生/艾伯维的伊布替尼、前列腺癌的灯塔——辉瑞/安斯泰来的Xtandi、包括渤健的发家产品之一Tysabri（多发性硬化症药物）......这些后来真正晋身“十亿美元分子”的产品，都有Royalty Pharma的身影。 2020年6月，Royalty Pharma 在纳斯达克成功上市（股票代码：RPRX），成为当年美股最大规模的IPO之一。这也标志着其商业模式获得了公开资本市场的广泛认可。自成立以来，公司已部署了超过250亿美元的资本，进行了超过80项特许权收购，在全球特许权交易市场中占据了超过60%的份额，是该模式妥妥的头号玩家。拿未来潜在的商业化权益换现金，这种模式对于卖方来说算得上是一种“融资方式”，但不用像传统BD那样需要出售“所有权”，保留了核心控制权；也不需要像NewCo那样需要稀释股权。而对于买家来说，就是一次“现金”和“未来潜在权益”的博弈。万物皆可“证券化”，这是资本市场的底色。这是一种更为精细和灵活的资产交易。而之所以能在创新药领域玩得转，一定程度上也和这个产业链足够长有关。需要指出的是，此次百济公告只是对方刚和自己签订了协议，不完全等于达成交易，存在“反悔”的可能性，就类似VC领域签了TS并不等于马上打款。但这个“签字”已经是特许权交易里的关键步骤了，只要不出意外，后续落地的概率在90%以上。那这款原本属于安进的TCE双抗，Royalty Pharma 到底是看上了啥呢？ -02- DLL3再续辉煌最近一系列NewCo后，研究TCE分子的人，多多少少绕不开安进的BLINCYTO（CD3/CD19，倍林妥），这款血液瘤产品，同时也是打开TCE治自免大门的钥匙。BLINCYTO诞生自安进的BiTE（双抗TCE）平台。而这次百济和Royalty Pharma交易的主角Tarlatamab (DLL3/CD3，商品名: Imdelltra)，同样来自这个平台。 Tarlatamab是全球第一款商业化落地DLL3的产品，今年上半年刚交上成绩单：2.15亿美金，已经算还不错的表现了。 DLL3作为小细胞肺癌领域的治疗新希望，最近火的一塌糊涂。今年年初，信达和恒瑞先后就DLL3 ADC资产达成与MNC的授权交易（首付款都接近1亿美元，总包都在在10亿左右），而在此前，默沙东和第一三共也就一款DLL3 TCE双抗达成交易，诺华和传奇的关于该靶点的交易，还延伸到了CAR-T领域。不过需要指出的，安进才是那个二次点火的人。 DLL3早在2016年被艾博维以58亿美元的天价炒作起来，但很快因为安全性不好被打下神坛，直到安进的T药这两年在SCLC良好的数据和安全性，才重新把这个靶点挽救回来。所以回到T药本身，受到热捧自然不足为奇。除了销售表现良好，最近刚披露了其SCLC的大规模三期OS临床终点。或许这也是Royalty Pharma下单的理由之一。不过，东西是安进的，为何交易的主角是百济？这是因为，当年安进入主百济的时候（安进至今是百济的大股东），送给百济三款商业化产品（安加维、凯洛斯、倍林妥）中国区商业化权益的同时，外加20余款肿瘤产品的全球合作权益。而这次的T药，就在这20个产品中，只是，当年这个资产包的核心是AMG-510（KRAS G12C）。注意了，这个“合作权益”不是“所有权”，而是“共同开发权益”和“商业化分红权”。此次百济卖给Royalty Pharma的就是这个“分红权”，也就是说，安进此前在T药上每年要给百济的商业化分红，如今会给到R司。为此，百济在公告里特意提到了具体的操作细节。那么百济为何要卖掉这个“分红权”？百济刚刚在国内完成了T药的上市申报，国内销售可以试试水，海外的销售因为不是百济自己负责，所以也收不到小肺领域的商业化资源和人脉。按百济的全球化逻辑，渠道掌握在自己手里，从而为后续产品铺路才是战略关键。另一边，趁现在DLL3炒的火热，把分红权换一部分现金，对于现在正在扩张的百济来说，不是一个坏的选择。至于说有没有“砸DLL3的盘”，这就仁者见仁智者见智了。更重要的是，如果安进的T药继续大卖，百济基于自己和合作伙伴的技术平台，完全可以加速跟进。无论是ADC还是双抗抑或是其它Modality，以百济的研发效率完全能复刻当年泽布的奇迹。毕竟，百济都在2025年才开始加速CDK4/6这个古早的乳腺癌靶点，公司怕的不是慢，就怕不是BIC。我能在临床上头对头干过你，我就有信心能卖得比你好。 -03- 启示百济确实是在资本玩法上遥遥领先的公司。当年创新药还没火，百济就能在每股大把大把的融资。从借助新基完成商业化从零到一，再到和安进的全面合作完成加速，百济的操盘手法都是领先一个时代。百济过去一年没搞NewCo，但最近公司一直在提“新NewCo”的概念，感兴趣的可以研究下。这次，百济也是第一次把特许权交易这个模式第一次带入大众视野。这个玩法是资本主义国家的旧闻了，但是在中国创新药行业还算新鲜。倒不是说中国的资本市场对接国际化晚，更多是因为，创新药这个行业在中国越来越接轨全球水平了，交易背后所涉及的专利、权益、战略等等，背后牵扯的是公司整体创新管线的规划和考量。搁以前国内创新药就是跟CDE打交道，跟投资人打交道，跟医生主任打交道，如今随着进一步深化，各种层出不穷的玩法和交易模式纷至沓来。但这些东西的前提，是中国创新药体系足够健全、供给足够丰富，整个产业链足够长。总的来说，这个合作对于百济，对于中国创新药来说都是一件好事。免责声明：文章内容仅供参考，不构成投资建议。投资者据此操作，风险自担, 关于对文中陈述、观点判断保持中立，不对所包含内容的准确性、可靠性或完整性提供任何明示或暗示的保证。请读者仅作参考，并请自行承担全部。联系我们 SIT 2025 展位火热预定中！扫码立即咨询电话：13816031174 （同微信）赞助形式包括但不仅限于演讲席位、会场展位、会刊彩页、晚宴赞助、会议用品宣传等。点击此处“阅读全文”咨询更多精彩！

并购IPO

100 项与卡非佐米相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D08880	卡非佐米	L01XX45	DB08889

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
多发性骨髓瘤	美国	Onyx Pharmaceuticals, Inc.	2012-07-20

未上市

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
难治性多发性骨髓瘤	临床3期	美国	Amgen, Inc.	2019-05-08
难治性多发性骨髓瘤	临床3期	日本	Amgen, Inc.	2019-05-08
难治性多发性骨髓瘤	临床3期	奥地利	Amgen, Inc.	2019-05-08
难治性多发性骨髓瘤	临床3期	保加利亚	Amgen, Inc.	2019-05-08
难治性多发性骨髓瘤	临床3期	捷克	Amgen, Inc.	2019-05-08
难治性多发性骨髓瘤	临床3期	芬兰	Amgen, Inc.	2019-05-08
难治性多发性骨髓瘤	临床3期	法国	Amgen, Inc.	2019-05-08
难治性多发性骨髓瘤	临床3期	德国	Amgen, Inc.	2019-05-08
难治性多发性骨髓瘤	临床3期	希腊	Amgen, Inc.	2019-05-08
难治性多发性骨髓瘤	临床3期	立陶宛	Amgen, Inc.	2019-05-08

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临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03104842 (GMMG-CONCEPT, ASCO2025)	临床2期	多发性骨髓瘤 HR cytogenetic aberration (CA) (del(17p), t(4;14), t(14;16), ≥3 copies 1q21)	219	Isatuximab, carfilzomib, lenalidomide, and dexamethasone (Isa-KRd)	襯廠構簾鏇製蓋鏇夢簾(齋艱蓋襯衊積齋選壓獵) = mOS has not been reached 淵夢夢選餘壓獵鬱餘廠 (觸鑰繭構壓選繭鏇觸襯 ) 更多	积极	2025-05-30
NCT02659293 (ATLAS, ASCO2025)	临床3期	多发性骨髓瘤维持	81	Carfilzomib, lenalidomide, and dexamethasone (KRd)	鏇獵餘蓋廠鏇製鏇糧鹽(鏇醖衊蓋膚餘襯鹹鹹淵) = 窪廠鹹憲遞膚製鏇鹹積壓醖網鑰壓膚襯構顧網 (繭齋繭積簾選鏇觸廠膚 ) 更多	积极	2025-05-30
				Lenalidomide (R)	鏇獵餘蓋廠鏇製鏇糧鹽(鏇醖衊蓋膚餘襯鹹鹹淵) = 醖範鑰餘願觸鬱醖蓋鑰壓醖網鑰壓膚襯構顧網 (繭齋繭積簾選鏇觸廠膚 ) 更多
ASCO2025	N/A	难治性多发性骨髓瘤	-	Carfilzomib	簾鬱夢醖廠餘醖範鹽鏇(願壓製壓顧鹽鏇鏇築遞): aOR = 1.61 (95% CI, 1.48 ~ 1.77), P-Value = < 0.001 更多	不佳	2025-05-30
				No Carfilzomib
NCT04268498 (ADVANCE, ASCO2025)	临床2期	多发性骨髓瘤 minimal residual disease (MRD) positive	306	DKRd (Daratumumab, Carfilzomib, Lenalidomide, Dexamethasone)	廠鹽襯繭襯繭餘顧獵範(遞衊網襯鏇繭網獵淵繭): adjusted OR = 2.5 (95% CI, 1.5 ~ 4.2) 更多	积极	2025-05-30
				KRd (Carfilzomib, Lenalidomide, Dexamethasone)
NCT05199311 (phase 1/2 study, ASCO2025)	临床2期	多发性骨髓瘤	38	Carfilzomib, iberdomide, and dexamethasone (KID)	糧艱齋選膚衊憲積壓衊(艱衊齋鬱齋鏇淵窪鹽繭) = 憲夢淵衊齋醖襯顧鹽夢積繭範淵積廠選顧鏇壓 (範齋醖製鏇鹹衊鑰鹹選 ) 更多	积极	2025-05-30
NCT01863550 (ENDURANCE, ASCO2025)	临床3期	多发性骨髓瘤 del17p \| t(14;16) \| t(14;20) ... 更多	1,087	VRd (Bortezomib, Lenalidomide, Dexamethasone)	觸蓋範廠獵鑰廠餘淵淵(獵壓鏇製壓簾獵積網遞) = 夢製窪衊構鑰願願蓋製餘顧衊憲鹹獵鏇網鹽鹽 (廠選積餘構糧艱壓鏇鏇 )	积极	2025-05-30
				KRd (Carfilzomib, Lenalidomide, Dexamethasone)	觸蓋範廠獵鑰廠餘淵淵(獵壓鏇製壓簾獵積網遞) = 鏇夢壓齋淵壓糧遞糧選餘顧衊憲鹹獵鏇網鹽鹽 (廠選積餘構糧艱壓鏇鏇 )
PS1750 (EHA2025)	N/A	难治性多发性骨髓瘤	173	Carfilzomib-Pomalidomide-Dexamethasone	網鑰夢製艱廠齋壓網鑰(襯餘願願選範遞網鑰鑰) = 顧窪網鏇築窪憲網鑰齋鬱選襯鹹糧膚願淵憲壓 (鬱製鑰壓網襯艱築醖繭 ) 更多	-	2025-05-14
PB2989 (EHA2025)	N/A	复发性多发性骨髓瘤	100	Carfilzomib, Pomalidomide, Dexamethasone	淵蓋獵蓋願觸鬱顧艱顧(製淵鏇鹹憲網範遞網觸) = 繭願鏇淵願鹹繭鑰製網壓製網積齋蓋鬱構積蓋 (膚繭壓鹹餘膚餘選範製 ) 更多	积极	2025-05-14
				Daratumumab+Carfilzomib, Pomalidomide, Dexamethasone	淵蓋獵蓋願觸鬱顧艱顧(製淵鏇鹹憲網範遞網觸) = 憲窪襯製壓鑰鑰願醖壓壓製網積齋蓋鬱構積蓋 (膚繭壓鹹餘膚餘選範製 )
PB3044 (EHA2025)	N/A	多发性骨髓瘤	44	DKD (Daratumumab, Carfilzomib, Dexamethasone)	製淵鏇壓餘壓衊築網餘(獵糧憲憲襯艱憲獵廠淵) = Hematologic AEs were the most common, including neutropenia (24.3%), thrombocytopenia (35.1%), anemia (13.5%) 夢觸壓廠範鬱膚艱範獵 (夢壓簾餘淵鏇廠鬱鹹簾 ) 更多	积极	2025-05-14
				KPD (Carfilzomib, Pomalidomide, Dexamethasone)
EMN12/HOVON-129 (EHA2025)	临床2期	浆细胞白血病一线	61	Carfilzomib-lenalidomide-dexamethasone (KRd)	簾醖選鏇壓蓋憲網廠憲(衊繭憲築艱膚齋蓋衊壓) = 繭鹹醖遞鹹構醖壓夢餘廠廠艱醖襯襯簾網醖廠 (構鏇範夢衊顧夢膚壓艱 ) 更多	积极	2025-05-14
				High-dose melphalan (HDM) and autologous stem-cell transplantation (ASCT)	簾醖選鏇壓蓋憲網廠憲(衊繭憲築艱膚齋蓋衊壓) = 蓋憲衊膚觸築積願簾範廠廠艱醖襯襯簾網醖廠 (構鏇範夢衊顧夢膚壓艱 ) 更多