This randomized clinical trial will assess corneal astigmatism and visual outcomes in participants who have undergone corneal transplantation for keratoconus with ex vivo cross-linked donor corneal tissue versus participants who have undergone corneal transplantation for keratoconus with non-cross-linked donor corneas. Crosslinking is a procedure that stabilizes the biomechanical properties of the cornea; as a result, the cornea stiffens. It has been shown that this procedure stabilizes the cornea of patients with keratoconus or corneal ectasias. The FDA currently approves crosslinking for patients with progressive keratoconus and corneal ectasia following refractive surgery. Ex vivo crosslinking of donor corneal tissue for patients with keratoconus undergoing PK or DALK could stabilize the cornea and reduce the risk of high astigmatism and improve vision in patient with keratoconus.

开始日期2023-12-01

申办/合作机构

Massachusetts Eye & Ear Infirmary, Inc.

NCT04481841

/ Unknown status早期临床1期IIT

Head Yuanshi Dian Therapy in the Treatment of Burning Mouth Syndrome: a Randomized Controlled Trial

Burning mouth syndrome is one of the most common oral mucosal diseases in clinic. It is a chronic pain syndrome with extensive burning pain of oral mucosa as its main symptoms. There are no pathological changes in oral mucosa and no characteristic histopathological changes. Patients often have accompanying symptoms such as depression and xerostomia. Although the patient does not have obvious oral lesions, the pain symptoms are more serious and the mental pressure is greater.
BMS, as a complex clinical syndrome associated with multiple factors, mainly occurs in people aged 27-87, with an average age of 61. BMS is rare among people under 30 years old. BMS is predominant in women, the ratio of male to female is 1:5 to 1:7, the incidence is 0.7%-15%, and increases with age. Up to 90% of female patients are in perimenopausal period. Symptoms occur from 3 years before menopause to 12 years after menopause.
The causes of BMS are complex, and the treatment is difficult, easy to relapse and protracted. Studies have confirmed that the occurrence and development of BMS are directly related to mental factors. Therefore, psychosocial factors are the most important pathogenic factors of BMS. If we intervene in these factors, it is hopeful to improve the curative effect of BMS.
Traditional psychotherapy methods include drug treatment, psychotherapy, surgical treatment, traditional Chinese medicine treatment, etc. Drug treatment is mainly based on different types of mental and psychological diseases, choose different pharmacological effects of drugs, so as to effectively control the disease. However, these drugs are prone to adverse reactions such as sleepiness, weight gain, headache, physical weakness, etc. The basic principle of psychotherapy is to let patients fully expose symptoms, listen to their complaints patiently, carry out explanatory psychotherapy according to their medical history or take other psychological training, so as to relieve patients' mental stress and alleviate symptoms. But this method has a long course of treatment and needs the cooperation of the patients' family members; the basic principle of surgical treatment is to resect the corresponding areas of the brain or adopt endoscopy and micro-current to treat them, but the risks and injuries caused by the operation are greater, and the adverse reactions after the operation are larger; the treatment of traditional Chinese medicine needs a long course of treatment, and the treatment of some patients. The effect is not stable enough.
The causes of BMS are complex, there is no objective disease in clinic, and the patients suffer from abnormal pain, but the treatment methods are not uniform, and the curative effect is not good, which makes the patients unable to get effective treatment in the early stage of the disease, and easy to relapse, resulting in the aggravation and development of BMS into intractable sensory abnormalities, and protracted! Literature reports confirm that the tri-drug of oryzanol-riboflavin-vitamin E (oryzanol-riboflavin-vitamin E) is a classic treatment for BMS and has been included in the classic book in China, Pharmacotherapy for oral mucosal disease . However, its long-term clinical application has found that its efficacy is unstable, and clinical symptoms after drug withdrawal. The symptoms are prone to recurrence or even aggravation. Therefore, it is necessary to use the classical program on the basis of a combination of interventions to promote the efficacy of stable and safe.
Over the past two years, the investigators has treated nearly 100 cases of BMS with head yuanshi dian therapy, and achieved satisfactory results. It can obviously relieve burning pain of BMS oral mucosa, promote saliva secretion, improve dry mouth and bitter mouth, and improve sleep to a certain extent. However, due to the limited number of cases treated, the classification of BMS is not meticulous enough, and there are still vague areas in the classification of BMS, which affects the rigorous evaluation of the therapeutic effect of BMS.
Therefore, the investigators propose a hypothesis: can the head yuanshi dian therapy be used as the main adjuvant therapy for BMS? By consulting Pubmed, OVID, CNKI, Wanfang and other major databases at home and abroad, the investigators found that there is no relevant report at home and abroad. In view of this, the investigators intend to design this randomized positive controlled clinical trial, using conventional valley-nucleus-E triple drug therapy as the positive control group, to observe the efficacy and safety of head yuanshi dian therapy for BMS, in order to find a safe and effective green non-invasive therapy, effectively alleviate or eliminate oral mucosal pain, dry mouth, etc.

开始日期2020-07-27

申办/合作机构

浙江大学医学院附属第二医院（浙江省第二医院）

[+1]

NCT03922542

/ Recruiting临床2/3期

Randomized Comparison of Standard vs. Accelerated Corneal Crosslinking for Treatment of Progressive Keratoconus or Ectasia

The study objective is to compare accelerated and standard corneal crosslinking for treatment of progressive keratoconus or corneal ectasia.

开始日期2019-04-16

申办/合作机构

Price Vision Group

100 项与核黄素磷酸钠相关的临床结果

登录后查看更多信息

100 项与核黄素磷酸钠相关的转化医学

登录后查看更多信息

100 项与核黄素磷酸钠相关的专利（医药）

登录后查看更多信息

4,477

项与核黄素磷酸钠相关的文献（医药）

2026-02-01·TISSUE & CELL

Formononetin enhances angiogenesis in diabetic wounds by inhibiting ferroptosis through suppression of mtROS-mediated xCT/GPX4 upregulation

Article

作者: Wang, Yanguo ; Li, Xinying ; Zhu, Ding ; Zhao, Chuanqi ; Lu, Yuangang

BACKGROUND:

Impaired angiogenesis critically contributes to delayed wound healing in diabetic patients. Mitochondrial damage in endothelial cells worsens this vascular dysfunction. Formononetin (FMN), a phytoestrogen from Astragalus roots, is traditionally used to enhance blood function and microcirculation; however, its mechanism remains unclear. This study investigates whether FMN protects human umbilical vein endothelial cells (HUVECs) from high glucose (HG)-induced ferroptosis, and we also evaluated its potential to enhance vascular repair and accelerate diabetic wound healing.

METHODS:

We used Western Blotting to measure ferroptosis markers including the cystine/gluta-mate antiporter system (xCT) and glutathione peroxidase 4 (GPX4) in both HUVECs and diabetic mouse wound tissue. To assess oxidative stress, we measured glutathione content (GSH), malondialdehyde production (MDA), and intracellular iron accumulation. Mitochondrial changes were evaluated through MitoSOX, TOMM20 protein expression, and JC-1 fluorescence. Wound tissue sections were processed for H&E staining to examine healing stages, and CD31 immunofluorescence was performed to visualize angiogenesis in the wound tissue.

RESULTS:

FMN effectively reduced ferroptosis markers in HG-treated HUVECs, and Erastin treatment abolished this protective effect. The compound appeared to block ferroptosis through two mechanisms: restoration of mitochondrial integrity and reactivation of the xCT/GPX4 antioxidant system. When we tested FMN in diabetic mice, wound closure rates improved substantially, the expression of xCT and GPX4 was increased, and CD31 expression in wound vessels increased, which matched what we observed in vitro.

CONCLUSION:

Ferroptosis is critically involved in the pathogenesis of diabetic wounds. FMN exerts protective effects against HG-induced ferroptosis in HUVECs via restoration of mitochondrial function and mitigation of mitochondrial reactive oxygen species (mtROS) accumulation through xCT/GPX4 activation. This evidence suggests FMN could serve as a promising natural therapeutic agent for diabetic wound treatment.

2025-12-01·Rice

A Dual-localized Fructose Bisphosphate Aldolase is Essential for Chloroplast Development and Carbon Metabolism in Rice

Article

作者: Gao, Yingbo ; Yao, Youli ; Zhou, Yong ; Lin, Lingshang ; Tang, Siyuan ; Liu, Xin ; Dong, Guichun ; Huang, Jianye ; Zhang, Xiaoxiang ; Ben, Linli ; Zhou, Juan ; Yang, Zefeng

Fructose-1,6-bisphosphate aldolase (FBA) stands as a pivotal enzyme involved within the Calvin cycle and glycolytic pathways in bacteria and higher plants, but the specific function of OsFBA in rice is still unclear. Here, we identified a chloroplast and mitochondria dual-localized FBA protein, OsFBA1, in rice. Experimental evidence showed that the functionally deficient osfba1 mutants featured a notable decline in chlorophyll content, photosynthetic rate, and severe growth impediment by the three-leaf stage, leading to eventual plant demise. Up-regulation of photosynthetic-pathway genes in the osfba1 mutants indicated the essential role of OsFBA1 in chloroplast development and suggested a compensatory mechanism of other genes in the process. Furthermore, the absence of OsFBA1 impaired the carbon assimilation in young rice seedlings, and supplying exogenous glucose could partially sustain the survival of osfba1 mutant for a few more days. Pathway-specific metabolomics analysis revealed a systemic change of metabolites in the glycolytic pathway, and consequential carbohydrates accumulation due to OsFBA1 disruption. Transcriptomics profiling corroborated the expression changes of photosynthesis, and carbon metabolism pathway genes. We further demonstrated that OsFBA1 serves as the primary FBA enzyme governing energy generation, photosynthesis and carbon metabolism. These results prove that OsFBA1 is an essential core gene in supporting the life cycle of rice, its expression has to be tightly regulated.

2025-11-01·INTERNATIONAL JOURNAL OF BIOLOGICAL MACROMOLECULES

Structural characteristics of Pleurotus eryngii polysaccharide and its protective mechanisms against mastitis in mice

Article

作者: Song, Pengtao ; Shi, Liang ; Dong, Haiju ; Liu, Fang ; Dai, Hongyu ; Wu, Yi ; Zhou, Jiahao ; Chang, Zhihai ; Jiang, Xiaoyu ; Guo, Jinjie ; Yang, Jieyan ; Li, Liangliang ; Qu, Weijie ; Sun, Jitao ; Diao, Xiaoqian ; Chen, Guizhen ; Zuo, Shoujun ; Li, Aoyun

Mastitis, a common inflammatory condition in female mammals, increases the risk of breast cancer and causes substantial economic losses. Although antibiotics effectively treat mastitis, their overuse poses a threat to public health and safety. Pleurotus eryngii polysaccharides (PEPs) have emerged as a promising alternative to reduce antibiotic abuse; however, their protective mechanisms against mastitis remain unclear. This study aimed to elucidate the structural features of PEP and explore its protective effects against mastitis. Structural analysis revealed that PEP (Mw = 15.9 kDa) consists of galactose (60.66 %), mannose (37.51 %), glucose (1.11 %), and fucose (0.72 %), with a backbone composed of →2,6)-α-D-Galp-(1→ and →6)-α-D-Galp-(1→ residues, branched at C-2. In a lipopolysaccharide (LPS)-induced mouse mastitis model, PEP substantially reduced pro-inflammatory cytokine levels, enhanced antioxidant enzyme activity, alleviated mammary epithelial cell apoptosis, and restored blood-milk barrier integrity by modulating the nuclear factor-kappa B and Nrf2/HO-1 signaling pathways. Gut microbiota analysis showed PEP treatment increased the abundance and diversity of beneficial bacteria while reducing potentially harmful taxa. Metabolomic analysis results revealed the downregulation of metabolites such as riboflavin-5-phosphate and progesterone. Overall, PEP protects against mastitis by modulating the gut microbiota and metabolism, potentially via the gut-mammary axis, providing a theoretical basis for its clinical application.

项与核黄素磷酸钠相关的新闻（医药）

2025-10-26

·大药时记

FDA一周回顾：口服司美格鲁肽获批心血管保护等

阿斯利康与第一三共在欧洲医学肿瘤学会（ESMO）大会上公布，其联合开发的TROP2靶向抗体偶联药物（ADC）Datroway，显著延长了免疫治疗无效的难治性乳腺癌患者的生存期。此次公布的数据来自TROPION-Breast02的III期临床试验，近650例无法手术切除且免疫治疗无效的转移性三阴性乳腺癌患者参与了研究。接受Datroway治疗的患者中位总生存期达到23.7个月，明显优于接受传统化疗的18.7个月。无进展生存期（PFS）几乎翻倍，Datroway组为10.8个月，而化疗组仅为5.6个月。在323名接受Datroway治疗的患者中，约63%的患者肿瘤明显缩小，远高于化疗组的29%。此外Datroway治疗组的死亡风险降低了21%，整体疾病进展或死亡风险降低了43%，结果具有高度统计学和临床意义。目前Datroway已获批用于既往接受治疗的HR阳性/HER2阴性转移性乳腺癌患者。今年6月该药还获得批准，用于治疗特定类型的晚期非小细胞肺癌成人患者。此次新数据将推动其在三阴性乳腺癌领域的监管审批，拓宽适应症范围。在安全性方面，Datroway治疗组约33%的患者出现3级及以上治疗相关不良事件，稍高于化疗组的29%。常见副作用包括口腔炎、疲劳、贫血及部分白细胞计数下降。试验中有一例因间质性肺病死亡，独立委员会认定与药物相关，但一位调查员认为死亡原因是疾病进展。未来如何有效管理不良反应，将是提升患者治疗体验和药物竞争力的关键。一旦获得监管批准，Datroway将直接面对吉利德的Trodelvy竞争。Trodelvy同样靶向TROP2，已获批用于至少接受过两种系统治疗的三阴性乳腺癌患者。今年ESMO大会上，Trodelvy作为一线治疗的III期数据也显示出优于化疗的疗效，中位无进展生存期为9.7个月，对比化疗的6.9个月。尽管两款药物的临床试验设计存在差异，难以直接比较，但整体来看Datroway在无进展生存期和总体生存期上略占优势。生物技术公司Exelixis因一项针对难治性结直肠癌患者的关键临床试验数据未能完全满足市场预期，导致其股价在周一早盘大跌超过10%。此次试验评估了Exelixis新药第三代口服酪氨酸激酶抑制剂（TKI）zanzalintinib与免疫药物Tecentriq联合用药的疗效，STELLAR-303试验共纳入901名转移性结直肠癌患者，这些患者均在接受过其他治疗后病情进展。试验将患者随机分为两组：一组接受目前晚期结直肠癌常用药物Stivarga（瑞戈非尼）单药治疗，另一组则接受Tecentriq（阿替利珠单抗）联合zanzalintinib治疗。Exelixis宣布该试验达成主要终点，数据显示联合治疗组的中位总生存期（OS）为10.9个月，较Stivarga组的9.4个月延长了1.5个月。该结果已发表在《柳叶刀》并于欧洲医学肿瘤学会（ESMO）年会上公布。尽管联合用药在统计学上实现了生存期的改善，但临床意义却引发质疑。此次数据基于意向治疗人群（包含所有随机患者），通常更难显示明显疗效。Exelixis指出，这种生存获益在不同患者亚群中均有体现，包括不同地区、不同RAS基因状态以及既往接受抗VEGF治疗的患者。罗氏在ESMO会议上首次详细披露选择性雌激素受体降解剂（Selective Estrogen Receptor Degraders，SERDs）giredestrant治疗ER阳性、HER2阴性乳腺癌的具体数据。evERA试验中，罗氏将giredestrant与依维莫司（everolimus， Afinitor）联合使用，依维莫司是ER阳性乳腺癌二线治疗的常用药物。该试验以联合方案对比传统内分泌治疗加依维莫司，评估其在ESR1突变患者及整体人群中的疗效。最新公布的结果显示，giredestrant联合方案在ESR1阳性患者中将疾病进展风险降低了62%，在整体患者群体中降低了44%。针对无ESR1突变患者的探索性分析（统计学效力较弱）显示，吉瑞司特与其他内分泌治疗在疾病进展风险上无显著差异。Inluriyo在其关键注册试验中未能显著延缓包括ESR1阳性和阴性患者在内的整体人群疾病进展。Orserdu则在类似人群中将疾病进展风险降低了30%，但因疗效主要体现在ESR1阳性亚组，FDA未批准其用于非ESR1突变患者。诺华公布了其治疗IgA肾病（IgAN）药物补体因子B（CFB）抑制剂Fabhalta的最新三期临床数据，结果显示该药物能显著延缓患者肾功能的下降。三期临床试验Applause-IgAN研究比较了IgAN患者每日两次服用Fabhalta与安慰剂的疗效。经过两年治疗，Fabhalta组患者在肾小球滤过率（GFR）等关键指标上表现明显优于对照组，显示出其在延缓肾功能恶化方面的显著优势。早在2024年Fabhalta就基于初步数据获得了美国FDA的加速批准，适用于减少患者尿蛋白。此次最终数据的发布，进一步支持诺华计划于明年申请该药物在IgAN患者中的全面常规批准。诺华对Fabhalta寄予厚望，预计其全球市场潜力可达30亿美元。除IgAN外，Fabhalta还于2023年获FDA批准用于治疗阵发性睡眠性血红蛋白尿症，2025年获批用于C3肾小球病变，展现了其在多种罕见肾病中的广泛适应症潜力。2025年第二季度，Fabhalta实现销售收入1.2亿美元，成为诺华重点推广的品牌之一。 GSK与Spero Therapeutics联合公布了其口服抗生素候选药物，替比培南盐酸盐（tebipenem HBr）的一项三期临床试验结果，显示该口服疗法在治疗复杂性尿路感染（cUTIs）方面，与现有静脉注射药物疗效相当，有望支持未来上市申请。该项针对住院成年复杂性尿路感染患者的临床试验因疗效显著提前终止。中期分析结果显示，口服替比培南盐酸盐在主要终点临床治愈率及微生物根除率方面，表现不逊于静脉注射亚胺培南-西拉司他丁（imipenem-cilastatin）。在2025年亚特兰大感染病学周（ID Week 2025）上，两家公司公布了详细数据：服用600毫克替比培南盐酸盐的患者总体成功率为58.5%，而接受500毫克亚胺培南-西拉司他丁的患者成功率为60.2%。两组差异在预设的10%非劣效界限内，达成了主要研究目标。次要终点同样支持替比培南盐酸盐的疗效，数据显示其组内93.5%的患者症状消失，亚胺培南-西拉司他丁组为95.2%；微生物学反应率方面，替比培南组为60.3%，对比亚胺培南组的61.3%。此外，针对耐药肠杆菌科（Enterobacterales）患者，两药的临床及微生物反应率与整体人群数据一致。替比培南盐酸盐对产超广谱β-内酰胺酶（ESBL）的菌株有效，这类菌株能分解多种抗生素；同时对左氧氟沙星敏感性较低的病原体也显示出疗效，这些特性为其临床应用增添潜力。早在2022年，FDA曾以缺乏足够美国本土患者数据为由，拒绝了Spero的上市申请，并要求其开展另一项三期试验。随后，GSK以6600万美元预付款获得了该药物的权益，并承担后续的上市申请工作。目前，GSK计划于今年内提交监管审批申请，但FDA强调，申请资料中需包含更多美国患者数据，而此次临床试验主要依赖东欧研究中心，未来是否能满足监管要求仍需观察。若获批准，替比培南盐酸盐将使医生能够为更多复杂性尿路感染患者提供院外口服治疗，减少对静脉注射治疗的依赖，降低患者住院率和医疗成本，提升患者治疗便利性。美国生物技术公司Neuphoria Therapeutics的核心候选药物BNC210（soclenicant）在治疗社交焦虑障碍（SAD）的三期临床试验中未能达到预期效果，导致公司决定终止该项目，并启动全面的战略评估，重新规划未来发展方向。BNC210是一种针对α7烟碱型乙酰胆碱受体的负向调节剂，曾被寄予厚望，期望能为社交焦虑患者带来快速且副作用较少的治疗选择。在这项名为Affirm-1的三期临床试验中，研究团队对比了单剂量225毫克BNC210与安慰剂在公众演讲挑战测试中的疗效，主要依托痛苦程度量表来评估症状改善。试验未能达到主要终点，患者在焦虑程度上的改善未显示出统计学显著差异，次要终点如患者自评和临床医师评估同样未见明显效果。尽管如此，BNC210的安全性和耐受性表现依旧良好，且未出现传统抗焦虑药物常见的镇静或认知障碍副作用。消息公布后，Neuphoria股价应声大跌67%。诺和诺德旗下的口服司美格鲁肽制剂Rybelsus获得美国FDA批准，适应症范围得到扩展。该药物现可用于2型糖尿病高风险患者的主要或次要预防，帮助降低重大不良心血管事件（MACE），包括心肌梗死、中风和心血管死亡的发生风险。此次适应症扩展于上周五正式获批，明确将Rybelsus定位为一种GLP-1受体激动剂，适用于心血管风险管理，标志着其产品定位从单纯的降糖药向综合心血管保护药物的转变。这项批准基于IIIb期SOUL研究的结果。SOUL研究共纳入9600多名患者，评估在标准治疗基础上增加Rybelsus对MACE风险的影响。数据显示，接受Rybelsus治疗的患者中，有12%经历了MACE事件，而安慰剂组为13.8%，相对风险降低约14%，且具有统计学意义。作为全球首款口服GLP-1受体激动剂，Rybelsus长期面临注射型司美格鲁肽（Ozempic）及其他GLP-1药物的竞争。此次心血管适应症获批，使Rybelsus从单纯的糖尿病控制药物转型为心血管风险管理药物，极大提升其临床价值和市场竞争力。特别是在心血管风险较高的2型糖尿病患者中，Rybelsus的应用场景将显著扩大。美国生物技术公司Glaukos宣布，其研发的创新药物Epioxa获得美国FDA批准，用于治疗罕见且可能致盲的眼病圆锥角膜。该药物预计将于2026年第一季度在美国上市，为患者带来全新的治疗选择。圆锥角膜是一种罕见的眼科疾病，发病率约为每10万人中50至200例。其特点是角膜逐渐变薄并向外突起，呈现类似圆锥的形状，而非正常的圆顶形。这种异常导致患者视力模糊、复视、视物变形和光晕等症状，且对光线极为敏感。如果不及时治疗，圆锥角膜可能导致视力严重受损，甚至失明。Glaukos指出，这种疾病对患者生活影响巨大，尤其是在30岁以下的年轻人群中进展更快。虽然早期患者可以通过眼镜或隐形眼镜改善视力，但约有20%的患者最终需要接受角膜移植手术。目前，治疗圆锥角膜的主流方法是角膜交联术。传统的角膜交联需要先通过手术去除或松解角膜最表层，然后滴入含有核黄素（维生素B2）的药液，再用紫外线激活以加强角膜结构。Glaukos在2016年推出的Photrexa即是此类治疗药物，但其手术过程较为痛苦，恢复时间较长。而Epioxa则开创了无切口交联治疗新模式。该药物通过局部涂抹一种富含氧气的新型治疗剂于角膜表面，随后同样利用紫外线激活。此方法避免了传统手术切除角膜表层的步骤，大大减少了患者的疼痛和恢复时间，降低了手术风险。两项涉及400多名患者的Ⅲ期临床试验结果显示，Epioxa显著改善了患者的最大角膜曲率，且安全性和耐受性均良好。康方生物与Summit Therapeutics联合开发的PD-1/VEGF双特异性抗体ivonescimab在晚期非小细胞肺癌（NSCLC）患者中的无进展生存期（PFS）数据表现优异。ivonescimab作为晚期NSCLC一线治疗方案，显著延长了患者的无进展生存期。此次数据公布于2025年欧洲肿瘤内科学会（ESMO）年会，来自中国开展的HARMONi-6Ⅲ期临床试验。该研究共纳入530多名未接受过系统治疗的鳞状非小细胞肺癌患者。数据显示，接受ivonescimab联合化疗的患者中位无进展生存期为11.4个月，对照组使用BeOne公司Tevimbra联合化疗的患者仅为6.9个月。Akeso报告称，这相当于ivonescimab组相比对照组风险降低了40%，且统计学意义高度显著。在安全性方面，Akeso报告显示，ivonescimab组有99.2%的患者出现治疗相关不良事件，对照组为98.5%。ivonescimab组共有8例患者死亡，对照组为10例。尽管数据令人振奋，但ivonescimab在美国的审批前景依然存在不确定性。今年9月康方生物与Summit公布了PD-1/VEGF双特异性抗体在亚洲和西方患者中的生存差异数据。HARMONi晚期研究显示，ivonescimab联合化疗在西方患者中带来了22%的总生存期获益，但未达到统计学显著性。早在今年5月，Summit就曾宣布FDA将在批准上市前要求看到具有统计学显著性的总生存期获益。亲爱的读者，如果您对生物医药的最新动态感兴趣，或希望了解更多前沿生物科技资讯，请关注公众号。您的每一次关注和转发，都是最大的支持和鼓励！ 👉 关注大药时记，掌握更多前沿生物科技！ 👉 分享文章，让更多人受益！

免疫疗法临床结果临床3期抗体药物偶联物

2025-10-21

·科睿唯安生命科学与制药

2025 年 Q3 癌症指数大涨 24.15%，并购与临床进展成重要驱动力

本文翻译自科睿唯安旗下 BioWorld 新闻： Cancer stocks rebound, outpacing broader markets in Q3 2025 • BioWorld 癌症指数 (BCI) 在 2025 年第三季度 (Q3) 收盘时上涨 24.15%，创下年内最高水平。此次上涨扭转了早前的跌势，包括第一季度末下跌 12.81%，以及半年业绩为 2.21%。大盘也呈现类似的反弹势头，纳斯达克生物技术指数上涨 13.21%，道琼斯工业平均指数上涨 9.06%，两者在第三季度末均收于 2025 年的最高点。由于并购 (M&A)，BCI 在第三季度有四家公司发生变动： Blueprint Medicines Corp. 被 Sanofi SA 以 95 亿美元的价格收购。该交易为 Sanofi 带来了一款处于商业化阶段、具有准重磅炸弹潜力的罕见病免疫疗法，以及一款 II/III 期后续候选药物和另一款处于中期阶段的自身免疫资产。 Cargo Therapeutics Inc. 被 Concentra Biosciences LLC 收购，Cargo 成为其全资子公司。 Tuhura Biosciences Inc. 是一家专注于克服癌症免疫疗法耐药性的 III 期免疫肿瘤学公司，该公司收购了 Kineta Inc.。该交易将 II 期就绪的 VISTA 抑制性单克隆抗体 TBS-2025（前称 KVA-1213）加入到 Tuhura 的管线中。 Springworks Therapeutics Inc. 被 Merck KGaA 以每股 47 美元的现金交易收购，该公司总部位于康涅狄格州斯坦福，公司股权价值约 39 亿美元，或根据年终现金计算的企业价值为 34 亿美元。该价格较 Springworks 截至 2 月 7 日的 20 日平均股价 37.38 美元溢价 26%。被纳入指数以替代已被收购公司的企业：Day One Biopharmaceuticals Inc.、Kura Oncology Inc.、Recursion Pharmaceuticals Inc. 和 Relay Therapeutics Inc.。在第三季度，BCI 中上涨股占主导地位，有 22 只股票上涨，仅 7 只股价下跌。在该三个月期间，指数中表现最佳的公司为 Olema Pharmaceuticals Inc.（上涨 130%）、Coherus Biosciences Inc.（上涨 125%）、Verastem Inc.（上涨 113%）、Terns Pharmaceuticals Inc.（上涨 102%）、Cellectis SA（上涨 86%）、Atara Biotherapeutics Inc.（上涨 85%）、Puma Biotechnology Inc.（上涨 55%）、Kura Oncology Inc.和Karyopharm Therapeutics Inc.（均上涨 53%）、Relay Therapeutics Inc.（上涨 51%）以及 Genelux Corp.（上涨 46%）。第三季度 BCI 跌幅最大的公司是 Adaptimmune Therapeutics plc（下跌 46%）、Beyondspring Inc.（下跌 23%）、Mural Oncology plc（下跌 17%）和 Agenus Inc.（下跌 15%）。 BioWorld 癌症指数注：历年数据请查看原文链接 Olema 第三季度股价上涨得益于管线进展和试验更新 Olema Pharmaceuticals 是第三季度 BCI 中涨幅最大的公司，该季度股价上涨 130%。这家总部位于旧金山的公司于 8 月 12 日公布了 2025 年第二季度财报，其股价上涨 16%，收于 5.31 美元。公司重点介绍了 Palazestrant (pala) 联合 Ribociclib (ribo) 的临床进展，更新的数据将于 10 月在 ESMO 上公布。H.C. Wainwright 的 Emily Bodnar 指出，该数据集将包含超出 2025 年 2 月截止日期后约六个月的额外数据。在入组的 62 例患者中，有 24 例仍在研究中，其中包括 14 例未使用过 CDK4/6 抑制剂的患者。在总人群和接受过 CDK4/6i 治疗的组中，中位无进展生存期 (PFS) 均已超过 13 个月，由于近 40% 的患者仍在接受治疗，因此该数据仍有进一步改善的可能。结果优于其他口服 SERD 联合疗法，Bodnar 写道，分析师认为“pala + ribo 联合疗法在包括 ESR1 突变型 (ESR1m) 和 ESR1 野生型 (ESR1wt) 患者在内的不同患者亚型中，显示出与同类药物相比可能具有差异化的疗效特征，预示着公司计划于 2025 年第三季度启动的 III 期 (OPERA-02) 潜在 1L 联合疗法前景良好。” 公司其他新闻： III 期 OPERA-02 试验计划于 2025 年第三季度启动，将在约 1,000 例一线 ER+/HER2-转移性乳腺癌患者中，检验 pala 90 mg 联合 ribo 与来曲唑联合 Ribociclib 的疗效。主要终点是研究者评估的无进展生存期，次要终点包括总生存期、总缓解率、安全性和患者报告结局。分层将考虑绝经状态、内脏疾病以及复发与新发病例。Novartis 将提供 ribo，并在获得顶线结果后拥有优先谈判权。 Olema 将 OPERA-01 顶线数据的发布指引更新至 2026 年下半年。该 III 期试验 (n=510) 分为两部分：第 1 部分将 120 例患者随机分配至 90 mg 或 120 mg Palazestrant 组与标准治疗 (SOC) 内分泌疗法组，以选择剂量；第 2 部分将在 390 例 2L/3L ER+/HER2-转移性乳腺癌患者中测试所选的 90 mg 剂量。尽管之前大多数患者接受的是 120 mg 剂量，但 FDA 建议评价 90 mg 剂量，理由是该剂量在暴露量和疗效相似的情况下，可能降低胃肠道 (GI) 毒性。 Olema 正在为 OP-3136 的 I/II 期试验 (n=90) 招募患者，该试验针对 ER+/HER2-转移性乳腺癌、转移性去势抵抗性前列腺癌和晚期非小细胞肺癌，初步数据预计于 2026 年公布。剂量递增阶段将测试最多五个剂量水平，然后选择推荐剂量。根据 Bodnar 的说法，计划的联合用药方案包括 OP-3136 与氟维司群、Palazestrant 的联合用药，以及 Palazestrant 联合 Ribociclib 的三联疗法，这些策略可以产生“管线协同效应，并提供差异化的特征”。 Olema 报告 2025 年第二季度运营亏损 4,790 万美元，即每股亏损 52 美分。与 2025 年第一季度相比，支出有所增加，主要原因是向 Aurigene 支付了 1,000 万美元的研发里程碑付款，这与 OP-3136 进入 I/II 期临床试验有关；下一个里程碑付款要到关键性试验时才会支付。排除该一次性成本，随着 III 期 Palazestrant 试验、III 期 OPERA-02 启动和 OP-3136 开发的持续招募，预计 2025 年下半年支出将增长。全年运营支出预计为 1.719 亿美元。截至 2025 年第二季度末，Olema 拥有 3.619 亿美元现金，可支持公司运营至 2027 年下半年。 Terns 有望从日益增长的 GLP-1 和 THR-β 需求中获益 Terns Pharmaceuticals 在第三季度上涨 102%，其中包括 9 月 18 日上涨 11%。次日，Mizuho Securities USA 分析师 Graig Suvannavejh 重申了“跑赢大盘”的评级和 14 美元的目标价，理由是行业动态更新对 Terns（总部位于 Foster City，California）有利。其中包括礼来公司 (Eli Lilly and Co.) 的 Orforglipron 的积极数据、诺和诺德公司 (Novo Nordisk AG) 的 Ozempic（司美格鲁肽）的新心血管结果，以及罗氏制药 (Roche AG) 宣布以 35 亿美元收购 89bio Inc.。Suvannavejh 写道：“随着肥胖领域的积极势头持续，以及我们看来 NASH/MASH 领域的情绪和兴趣似乎正在改善，我们认为 TERN 凭借其 TERN-601（一种口服 GLP-1）和 TERN-501（一种口服 THR-β 激动剂）处于理想的受益位置，”他们认为这些资产对潜在合作伙伴具有战略价值。 Atara 因 tab-cel 预期和超预期盈利在第三季度上涨 Atara Biotherapeutics 在第三季度跃升 85%，其中包括在 8 月 12 日发布第二季度财报后上涨 12%。H.C. Wainwright 分析师 Robert Burns 指出，投资者对 Atara 的关注点集中在 Tabelecleucel (tab-cel) 的 2026 年 1 月 10 日《处方药使用者付费法案》 (PDUFA) 日期上。重新提交的生物制品许可申请 (BLA) 旨在为成人和 2 岁及以上儿童 Epstein-Barr 病毒阳性移植后淋巴增殖性疾病患者寻求批准，这些患者既往至少一种治疗失败。该申请得到了来自超过 430 例患有危及生命疾病的患者数据的支持，其中包括关键的 Allele 试验结果，该结果显示客观缓解率为 48.8% (p<0.0001)。FDA 的批准将触发 Pierre Fabre 支付的 4,000 万美元里程碑付款，以及两位数的分级特许权使用费和额外的商业里程碑付款。 Atara（总部位于 Oaks，California）公布 2025 年第二季度每股净收益为 19 美分，远超 Wainwright 预测的每股亏损 4.04 美元。营收达到 1,760 万美元，而早前预估为 20 万美元，这促使公司将 2025 年全年指引更新为每股净收益 1.72美元，而此前的预测为每股亏损 8.41 美元。该公司本季度末拥有 2,230 万美元的现金和现金等价物。然而，尽管 tab-cel 的业绩和开发进展有所改善，Wainwright 仍维持“中性”评级，未设定目标价，理由是对近期的投资案例信心有限。 Adaptimmune 在出售资产后股价跌至历史低点 7 月 28 日，在宣布将向美国 Worldmeds 出售其细胞疗法，包括 Tecelra (afamitresgene autoleucel)、lete-cel、afami-cel 和 uza-cel 后，Adaptimmune Therapeutics plc 的股价暴跌 71%，收于 10 美分。根据于 8 月完成的资产购买协议条款，美国 Worldmeds 在交易完成时支付了 5,500 万美元现金，并将根据商业和监管成果支付最高达 3,000 万美元的绩效里程碑付款。位于美国费城和英国牛津的 Adaptimmune 保留了其临床前项目的权利，包括 PRAME 和 CD70 靶向的 T 细胞疗法，以及其异体细胞疗法管线。 // 本文翻译自科睿唯安旗下 BioWorld 新闻： Cancer stocks rebound, outpacing broader markets in Q3 2025 点击文末阅读原文查看英文版产品推介 BioWorld™ 是行业领先的一站式生物医药行业资讯平台，专注于为处于研发阶段的最具创新性的药物和医疗技术提供切实可行的情报。扫码申请免费试用产品咨询： Lulice.zhao@Clarivate.com +86 21 2026 8063 推荐阅读关于科睿唯安科睿唯安是全球领先的变革性资讯提供商。我们提供丰富的数据、洞见、工作流解决方案和专业服务，涵盖学术研究和政府机构，知识产权、生命科学与健康各个领域。如需了解更多信息，请访问www.clarivate.com.cn 敬请关注科睿唯安生命科学与制药微信公众号 “科睿唯安生命科学与制药”微信公众号与本文作者对本文的全部内容以及可能附带的全部资料拥有全部知识产权，并受法律保护。网络转载请注明作者及内容来源。商业合作请联系： lulice.zhao@clarivate.com +86 21 2026 8063

并购免疫疗法财报临床3期

2025-10-20

·Business Wire

Glaukos Announces FDA Approval of Epioxa™

ALISO VIEJO, Calif.--(BUSINESS WIRE)--Glaukos Corporation (NYSE: GKOS), an ophthalmic pharmaceutical and medical technology company focused on novel therapies for the treatment of glaucoma, corneal disorders, and retinal diseases, announced today the U.S. Food and Drug Administration (FDA) approved its Epioxa™ HD / Epioxa™ (“Epioxa”) New Drug Application (NDA). Epioxa is a groundbreaking advancement in corneal cross-linking for the treatment of keratoconus, a rare, sight-threatening disease that is currently far too often undiagnosed and untreated. Epioxa represents a transformative innovation in keratoconus care, offering an incision-free alternative to traditional corneal cross-linking procedures as it does not require the removal of the corneal epithelium, the outermost layer of the front of the eye. This novel, oxygen-enriched topical therapeutic, bioactivated by UV light, is designed to eliminate the pain associated with removal of the epithelium, streamline the procedure, and minimize recovery, all while delivering clinically meaningful outcomes and exceptional value to patients, providers, and the healthcare system. Epioxa is based on two formulations, Epioxa HD and Epioxa, that are sequentially administered during the cross-linking procedure followed by UV activation in an oxygen-enriched environment. “The FDA approval of Epioxa ushers in a new standard-of-care for patients suffering from keratoconus with the first FDA-approved topical drug therapy that does not require removal of the corneal epithelium,” said Thomas Burns, Glaukos chairman and chief executive officer. “Epioxa is designed to significantly improve patient comfort and minimize recovery time, representing a game-changing new treatment for patients suffering from keratoconus. We appreciate the clinical investigators and study participants in the clinical trials for their instrumental roles in helping us reach this important advancement. This approval marks a major milestone in our mission to improve patient access to sight-saving therapies, and we are excited to bring this transformative therapy to market for the benefit of patients.” “Keratoconus is currently an underdiagnosed and undertreated disease. For keratoconus patients who are fortunate enough to be diagnosed, the current standard-of-care requires removal of the epithelium, the top layer of the cornea. The pain and extended healing time associated with the current surgical procedure are major barriers to adoption,” said W. Barry Lee, MD, corneal specialist at Eye Consultants of Atlanta and President of the Cornea Society. “As an incision-free treatment that does not require removal of the epithelium, I expect the newly approved Epioxa treatment to address both of these major concerns.” The FDA approval of Epioxa is based on results from two prospective, randomized, multicenter, double-masked, Phase 3 pivotal trials that randomized a total of over 400 patients. Both trials successfully achieved their pre-specified primary efficacy endpoints and demonstrated favorable tolerability and safety profiles. Glaukos intends for Epioxa to be commercially available in the first quarter of 2026. With this approval, Glaukos plans to make substantial investments in patient awareness and access while addressing the longstanding challenges of underdiagnosis and undertreatment that have affected this rare disease patient community. These efforts will be focused on supporting patients and families across every step of the journey, from awareness to diagnosis and treatment. Key initiatives include streamlined patient access support programs, a co-pay assistance program to reduce financial barriers, integrated healthcare professional (HCP) and patient-centric strategies to improve education and engagement, and broad awareness and detection programs aimed at earlier and more widespread screening for and diagnosis of the disease. Keratoconus is a debilitating eye condition characterized by progressive thinning and weakening of the cornea that is often most aggressively advancing in patients under the age of 30. If left untreated, keratoconus can lead to loss of visual function and even blindness and is one of the leading causes of corneal transplants (penetrating keratoplasty) in the United States. Approximately 90% of cases of keratoconus are bilateral and as many as 20% of untreated keratoconus patients ultimately require a corneal transplant. Conventional keratoconus treatments such as eyeglasses or contact lenses address visual symptoms only and do not slow or halt underlying disease progression. Glaukos’ first-generation corneal cross-linking therapy, known as Photrexa® Viscous / Photrexa®, which requires removal of the corneal epithelium, received U.S. FDA approval in 2016 as an orphan drug and has since been the first-and-only FDA-approved corneal cross-linking therapy for the treatment of keratoconus. There are more than 300 peer-reviewed publications supporting the performance and safety of Glaukos’ cross-linking therapies. Glaukos plans to provide additional details on the FDA approval of Epioxa and related matters as part of its upcoming third quarter 2025 financial results conference call scheduled for Wednesday, October 29, 2025, at 4:30 p.m. ET. A link to the webcast is available on the company’s website at http://investors.glaukos.com. To participate in the conference call, please dial 800-715-9871 (U.S.) or 646-307-1963 (international) and enter Conference ID 5255602. A replay of the webcast will be archived on the company’s website following completion of the call. For more information about Epioxa HD / Epioxa and Full Prescribing Information, please visit www.Epioxa.com. About Glaukos Glaukos (www.glaukos.com) is an ophthalmic pharmaceutical and medical technology company focused on developing and commercializing novel therapies for the treatment of glaucoma, corneal disorders, and retinal diseases. Glaukos first developed Micro-Invasive Glaucoma Surgery (MIGS) as an alternative to the traditional glaucoma treatment paradigm, launching its first MIGS device commercially in 2012. In 2024, Glaukos commenced commercial launch activities for iDose® TR, a first-of-its-kind, long-duration, intracameral procedural pharmaceutical designed to deliver 24/7 glaucoma drug therapy inside the eye for extended periods of time. Glaukos also markets the only FDA-approved corneal cross-linking therapy utilizing a proprietary bio-activated pharmaceutical for the treatment of keratoconus, a rarely diagnosed corneal disorder. Glaukos continues to successfully develop and advance a robust pipeline of novel, dropless platform technologies designed to meaningfully advance the standard-of-care and improve outcomes for patients suffering from chronic eye diseases. Epioxa HD / Epioxa Indication and Important Safety Information Indication: EPIOXA™ HD (riboflavin 5’-phosphate ophthalmic solution) 0.239% and EPIOXA™ (riboflavin 5’-phosphate ophthalmic solution) 0.177% are photoenhancers indicated for use in epithelium-on corneal collagen cross-linking for the treatment of keratoconus in adults and pediatric patients aged 13 years and older, in conjunction with the O2n™ System and the Boost Goggles®. Dosage and Administration: EPIOXA HD and EPIOXA are for topical ophthalmic use. NOT for injection or intraocular use. EPIOXA HD and EPIOXA are supplied in single-dose syringes. Discard opened syringes after use. EPIOXA HD and EPIOXA are for use with the O2n System and Boost Goggles only. Refer to the O2n System Operator’s Manual and Boost Goggles User Guide for device instructions. Contraindications: EPIOXA HD and EPIOXA are contraindicated in patients with known hypersensitivity to benzalkonium chloride or any ingredients in EPIOXA HD and EPIOXA. Epithelium-on corneal collagen cross-linking is contraindicated in aphakic and pseudophakic patients without a UV-blocking intraocular lens. Warnings and Precautions: Corneal collagen cross-linking should be used with caution in patients with a history of herpetic keratitis due to the potential for reactivation of herpes keratitis. Adverse Reactions: The most common adverse reaction was conjunctival hyperaemia (31%). Other adverse reactions, occurring in 5% to 25% of eyes included: corneal opacity (haze), photophobia, punctate keratitis, eye pain, eye irritation, increased lacrimation, corneal epithelium defect, eyelid oedema, corneal striae, visual acuity reduced, dry eye, and anterior chamber flare. Forward-Looking Statements All statements other than statements of historical facts included in this press release that address activities, events, or developments that we expect, believe, or anticipate will or may occur in the future are forward-looking statements. Although we believe that we have a reasonable basis for forward-looking statements contained herein, we caution you that they are based on current expectations about future events affecting us and are subject to risks, uncertainties, and factors relating to our operations and business environment, all of which are difficult to predict and many of which are beyond our control, that may cause our actual results to differ materially from those expressed or implied by forward-looking statements in this press release. These potential risks and uncertainties include, without limitation, our ability to successfully commercialize Epioxa HD / Epioxa and other investigational products, including by maximizing patient access and addressing the challenges of underdiagnosis and undertreatment, and the continued efficacy and safety profile of our products when commercially marketed as compared to their pre-approval clinical trial results. These and other risks, uncertainties, and factors related to Glaukos, and our business are described in detail under the caption “Risk Factors” and elsewhere in our Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, which was filed with the SEC on August 4, 2025. Our filings with the SEC are available in the Investor Section of our website at www.glaukos.com or at www.sec.gov. In addition, information about the risks and benefits of our products is available on our website at www.glaukos.com. All forward-looking statements included in this press release are expressly qualified in their entirety by the foregoing cautionary statements. You are cautioned not to place undue reliance on the forward-looking statements in this press release, which speak only as of the date hereof. We do not undertake any obligation to update, amend, or clarify these forward-looking statements whether as a result of new information, future events or otherwise, except as may be required under applicable securities law.

上市批准临床结果临床3期

100 项与核黄素磷酸钠相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
-	核黄素磷酸钠	S01XA26	DB03247

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
角膜疾病	美国	Glaukos Corp.	2016-04-15
圆锥角膜	美国	Glaukos Corp.	2016-04-15
核黄素缺乏	日本	TOA Eiyo Ltd.	1971-10-20

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
角膜新生血管	临床3期	美国	Price Vision Group	2021-04-15

登录后查看更多信息

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


NCT00674661 (CXL, CTgov)	临床3期	角膜扩张	130	UVA Irradiation+riboflavin ophthalmic solution (Corneal Collagen Cross-linking (CXL) Treatment Group)	醖繭壓壓壓醖鹽齋淵蓋(憲範構顧壓繭鬱窪鏇繭) = 鏇鬱鏇艱糧艱鹹廠簾積獵網選鑰築鏇鬱觸遞齋 (憲願獵夢簾簾網積壓廠, 2.2) 更多	-	2017-01-27
				riboflavin ophthalmic solution (Control Group)	醖繭壓壓壓醖鹽齋淵蓋(憲範構顧壓繭鬱窪鏇繭) = 齋餘壓鏇鬱鏇遞窪簾獵獵網選鑰築鏇鬱觸遞齋 (憲願獵夢簾簾網積壓廠, 2.3) 更多
NCT01893359 (CTgov)	临床1/2期	远视 \| 散光	2	Laser-assisted in situ keratomileusis+riboflavin+dextran (LASIK Followed by Cross-linking (Continuous Wave))	願餘繭製餘築膚鹽選構(鬱鑰願膚鹹願鹹膚遞蓋) = 鏇壓蓋鏇艱鑰鏇醖餘繭壓壓廠襯憲鹽選憲遞艱 (範齋壓蓋願製構夢廠鬱, 製壓築糧鹹廠範鏇範鑰 ~ 觸艱衊鬱壓構艱遞窪襯) 更多	-	2016-10-24
				Laser-assisted in situ keratomileusis+riboflavin+dextran (LASIK Followed by Cross-linking (Pulsed))	願餘繭製餘築膚鹽選構(鬱鑰願膚鹹願鹹膚遞蓋) = 衊顧衊鹹遞淵選範憲簾壓壓廠襯憲鹽選憲遞艱 (範齋壓蓋願製構夢廠鬱, 襯簾選範齋衊蓋鏇獵鏇 ~ 選襯觸積鏇遞觸選網鬱) 更多
NCT00647699 (CXL, CTgov)	临床3期	圆锥角膜	147	UVA Irradiation+riboflavin ophthalmic solution (Corneal Collagen Cross-linking (CXL) Treatment Group)	築齋選鏇齋構鏇膚蓋艱(艱襯餘製壓壓鏇範壓憲) = 餘選鏇醖簾廠艱簾壓繭鏇廠選觸廠淵夢壓艱鹽 (鏇簾餘製製獵齋餘繭鑰, 4.7) 更多	-	2016-07-07
				riboflavin ophthalmic solution (Control Group)	築齋選鏇齋構鏇膚蓋艱(艱襯餘製壓壓鏇範壓憲) = 鹹襯遞襯鬱蓋鬱廠憲遞鏇廠選觸廠淵夢壓艱鹽 (鏇簾餘製製獵齋餘繭鑰, 2.8) 更多
NCT05516004 \| NCT00841386 \| NCT01325298 (Pubmed \| J Cataract Refract Surg)	临床2/3期	圆锥角膜	10	Riboflavin-ultraviolet type A rays induced cross-linking of corneal collagen	鹹積窪鏇簾遞蓋夢網積(鏇獵糧淵鑰鹽積選餘醖) = 簾糧齋鏇糧醖獵積獵餘壓淵選衊襯餘獵鏇獵製 (鏇淵憲範築鬱餘獵範鬱 ) 更多	-	2006-05-01