Study Objectives:
To evaluate the safety and tolerability of oral SYN-004 in adult allogeneic HCT (allo-HCT) recipients who develop fever after conditioning therapy and are treated with IV β-lactam antibiotics meropenem (MER), piperacillin tazobactam (PIP/TAZO), or cefepime (FEP).
To evaluate potential absorption of oral SYN-004 into the systemic circulation of allo-HCT recipients and potential SYN-004-mediated alterations to systemic levels and efficacy of IV MER, PIP/TAZO or FEP.
To evaluate potential protective effects of SYN-004 on the intestinal microbiome of allo-HCT recipients treated with IV MER, PIP/TAZO or FEP.
To obtain preliminary information on potential therapeutic benefits and patient outcomes of SYN-004 in allo-HCT recipients treated with IV MER, PIP/TAZO or FEP

开始日期2021-02-15

申办/合作机构

Theriva Biologics, Inc.

[+1]

NCT02563106 / Completed临床2期

A Double-Blind, Placebo-Controlled, Multicenter Study of SYN-004 Compared to Placebo for the Prevention of C.Diff in Patients With a Diagnosis of a Lower Respiratory Tract Infection

A Phase 2b Parallel-Group, Double-Blind, Placebo-Controlled, Multicenter Study of SYN-004 Compared to Placebo for the Prevention of Clostridium difficile Infection (CDI) in Hospitalized Patients receiving IV ceftriaxone with a Diagnosis of a Lower Respiratory Tract Infection (LRTI).

开始日期2015-10-01

申办/合作机构

Theriva Biologics, Inc.

NCT02419001 / Completed临床1/2期

A Phase 1b/2a, Randomized, Multi-center, Open-label, Fixed-sequence Study to Evaluate the Effect of Oral SYN-004 on the Pharmacokinetics of Intravenous Ceftriaxone in Healthy Adult Subjects With a Functioning Ileostomy

A Phase 1b/2a, Randomized, Multi-Center, Open-Label, Fixed-Sequence Study to Evaluate the Effect of Oral SYN-004 on the Pharmacokinetics of Intravenous Ceftriaxone in Healthy Adult Subjects with a Functioning Ileostomy.

开始日期2015-03-01

申办/合作机构

Theriva Biologics, Inc.

100 项与 Ribaxamase 相关的临床结果

登录后查看更多信息

100 项与 Ribaxamase 相关的转化医学

登录后查看更多信息

100 项与 Ribaxamase 相关的专利（医药）

登录后查看更多信息

项与 Ribaxamase 相关的文献（医药）

2024-07-01·INTERNATIONAL JOURNAL OF ANTIMICROBIAL AGENTS

Fighting against Clostridioides difficile infection: Current medications

Review

作者: Fang, Qingqing ; Zong, Zhiyong ; Lv, Xiaoju ; Zhang, Xiaoxia ; Wang, Xiaohui ; Quan, Min

Clostridioides difficile (formerly Clostridium difficile) has been regarded as an 'urgent threat' and a significant global health problem, as life-threatening diarrhoea and refractory recurrence are common in patients with C. difficile infection (CDI). Unfortunately, the available anti-CDI drugs are limited. Recent guidelines recommend fidaxomicin and vancomycin as first-line drugs to treat CDI, bezlotoxumab to prevent recurrence, and faecal microbiota transplantation for rescue treatment. Currently, researchers are investigating therapeutic antibacterial drugs (e.g. teicoplanin, ridinilazole, ibezapolstat, surotomycin, cadazolid, and LFF571), preventive medications against recurrence (e.g. Rebyota, Vowst, VP20621, VE303, RBX7455, and MET-2), primary prevention strategies (e.g. vaccine, ribaxamase, and DAV132) and other anti-CDI medications in the preclinical stage (e.g. Raja 42, Myxopyronin B, and bacteriophage). This narrative review summarises current medications, including newly marketed drugs and products in development against CDI, to help clinicians treat CDI appropriately and to call for more research on innovation.

2022-07-01·ANTI-CANCER DRUGS4区 · 医学

In-silico drug-likeness analysis, ADME properties, and molecular docking studies of cyanidin-3-arabinoside, pelargonidin-3-glucoside, and peonidin-3-arabinoside as natural anticancer compounds against acting receptor-like kinase 5 receptor

4区 · 医学

Article

作者: Ellidokuz, Hulya ; Calibasi-Kocal, Gizem ; Kurter, Hasan ; Mert-Ozupek, Nazli

Background:

The aim of the study was in-silico drug-likeness analysis, absorption, distribution, metabolism, and excretion (ADME) properties, and molecular docking studies of anthocyanins as natural anticancer compounds against acting receptor-like kinase 5 (ALK5) receptor. Transforming growth factor-β (TGF-β) plays an essential role in various cellular processes. Increased expression of TGF-β and its receptor TGFβR-I (i.e. ALK5) have been associated with poor prognosis in cancer patients.

Methods:

The drug-likeness activity of anthocyanins was performed using SwissADME tool. Molecular docking studies were carried out by using the Autodock Vina 1.5.6 tool.

Results:

The results revealed that cyanidin-3-arabinoside (C3A), pelargonidin-3-glucoside (P3G), and peonidin-3-arabinoside (P3A) were able to use both Lipinski’s rule of five and Ghose variations. The binding energies of C3A, P3G, and P3A against ALK5 were found as −8.0, −8.3, and −8.4 kcal mol−1, respectively.

Conclusion:

These selected anthocyanins have shown higher binding energies than known inhibitors to the ALK5 receptor. Further in-vitro and in-vivo studies were strongly recommended to clarify the whole mechanism.

2021-12-01·Clinical microbiology and infection : the official publication of the European Society of Clinical Microbiology and Infectious Diseases1区 · 医学

How to: prophylactic interventions for prevention of Clostridioides difficile infection

1区 · 医学

Review

作者: Ed J. Kuijper ; Elena Reigadas ; Maria J.G.T. Vehreschild ; Microbiota interaction ; Marco Falcone ; Emilio Bouza ; Fidelma Fitzpatrick ; Joffrey van Prehn

BACKGROUND:

Clostridioides difficile infection (CDI) remains the leading cause of healthcare-associated diarrhoea, despite existing guidelines for infection control measures and antimicrobial stewardship. The high associated health and economic burden of CDI calls for novel strategies to prevent the development and spread of CDI in susceptible patients.

OBJECTIVES:

We aim to review CDI prophylactic treatment strategies and their implementation in clinical practice.

SOURCES:

We searched PubMed, Embase, Emcare, Web of Science, and the COCHRANE Library databases to identify prophylactic interventions aimed at prevention of CDI. The search was restricted to articles published in English since 2012.

CONTENT:

A toxin-based vaccine candidate is currently being investigated in a phase III clinical trial. However, a recent attempt to develop a toxin-based vaccine has failed. Conventional probiotics have not yet proved to be an effective strategy for prevention of CDI. New promising microbiota-based interventions that bind and inactivate concomitantly administered antibiotics, such as ribaxamase and DAV-132, have been developed. Prophylaxis of CDI with C. difficile antibiotics should not be performed routinely and should be considered only for secondary prophylaxis in very selected patients who are at the highest imminent risk for recurrent CDI (R-CDI) after a thorough evaluation. Faecal microbiota transplantation (FMT) has proved to be a very effective treatment for patients with multiple recurrences. Bezlotoxumab provides protection against R-CDI, mainly in patients with primary episodes and a high risk of relapse.

IMPLICATIONS:

There are no proven effective, evidenced-based prophylaxis options for primary CDI. As for secondary prevention, FMT is considered the option of choice in patients with multiple recurrences. Bezlotoxumab can be added to standard treatment for patients at high risk for R-CDI. The most promising strategies are those aimed at reducing changes in intestinal microbiota and development of a new effective non-toxin-based vaccine.

项与 Ribaxamase 相关的新闻（医药）

2024-10-31

·GlobeNewswire-Health Care

Theriva Biologics Selected as Finalist in International Competition for Merck KGaA's EMEA Advance Biotech Grant

Winner to be Presented at BIO-Europe 2024 in Stockholm, SwedenROCKVILLE, Md., Oct. 31, 2024 (GLOBE NEWSWIRE) -- Theriva™ Biologics (NYSE American: TOVX), a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced selection as one of five finalists for Merck KGaA's EMEA Advance Biotech Grant. Finalists for the grant will present to a six-judge panel from Merck’s Emerging Biotech group during BIO Europe 2024 in Stockholm, Sweden. Manel Cascalló, PhD., General Director - EU Subsidiary, Theriva Biologics, will present an overview of VCN-01, currently being studied in a Phase 2b clinical trial in first-line metastatic pancreatic cancer in combination with standard-of-care chemotherapy. Dr Cascalló will highlight VCN-01’s novel mechanisms of action and Theriva’s internal capabilities to develop its manufacturing process in his presentation on Monday, November 4, 2024 from 15:20-16:40 CET/CEST (GMT+2). Merck KGaA’s EMEA Advance Biotech Grant Program celebrates standout emerging biotech companies, and winners receive financial support and guidance on navigating their path to commercialization, including consultation with M Ventures. After multiple rounds of dynamic competition involving diverse biotech companies and technologies, the five selected finalists, will pitch their breakthroughs to Merck KGaA representatives and the overall BIO-Europe audience. The winner will be announced following the five presentations. About Theriva™ Biologics, Inc. Theriva™ Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company is advancing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. The Company’s lead candidates are: (1) VCN-01, an oncolytic adenovirus designed to replicate selectively and aggressively within tumor cells, and to degrade the tumor stroma barrier that serves as a significant physical and immunosuppressive barrier to cancer treatment; (2) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients; and (3) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at www.therivabio.com. Forward-Looking Statement This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding presenting an overview of VCN-01to a six-judge panel from Merck’s Emerging Biotech group during BIO Europe 2024 in Stockholm, Sweden and highlighting VCN-01’s novel mechanisms of action and Theriva’s internal capabilities to develop its manufacturing process. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to a number of risks and uncertainties, many of which are difficult to predict that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, the Company’s ability to reach clinical milestones when anticipated, the Company’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results, the Company’s ability to complete clinical trials on time and achieve the desired results and benefits, continuing clinical trial enrollment as expected; the Company’s ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s ability to maintain license agreements, the continued maintenance and growth of the Company’s patent estate, the ability to continue to remain well financed and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law. For further information, please contact: Investor Relations: Chris Calabrese LifeSci Advisors, LLC ccalabrese@lifesciadvisors.com 917-680-5608

临床2期免疫疗法

2024-10-16

·GlobeNewswire-Health Care

Theriva Biologics Announces Orphan Medicinal Product Designation Granted by the European Commission to VCN-01 for the Treatment of Retinoblastoma

ROCKVILLE, Md., Oct. 16, 2024 (GLOBE NEWSWIRE) -- Theriva Biologics (NYSE American: TOVX), (“Theriva” or the “Company”), a clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced that the European Commission has adopted the European Medicines Agency (EMA) recommendation to grant orphan medicinal product designation to lead clinical candidate VCN-01, Theriva’s systemic, selective, stroma-degrading oncolytic adenovirus, for the treatment of retinoblastoma. The United States Food and Drug Administration (FDA) has previously granted orphan drug designation and rare pediatric disease designation to VCN-01 for the treatment of retinoblastoma. “We are very pleased with the European Commission’s grant of orphan medicinal product designation to VCN-01, emphasizing the urgent need for new treatment options for retinoblastoma,” said Steven A. Shallcross, Chief Executive Officer of Theriva Biologics. “We have previously reported encouraging results from an investigator sponsored Phase 1 trial evaluating the safety and activity of intravitreal VCN-01 in pediatric patients with refractory retinoblastoma, and we are working closely with leading physicians and regulatory agencies worldwide to refine our clinical strategy for VCN-01 as an adjunct to chemotherapy in children with this challenging disease.” The EMA recommends orphan designation for products intended to treat, prevent or diagnose a disease that is life-threatening or chronically debilitating and either the prevalence of the condition in the European Union (EU) does not exceed 5 in 10,000 or it is unlikely that marketing of the product would generate sufficient returns to justify the investment needed for its development. Additionally, there should be no authorizable method of diagnosis, prevention or treatment of the condition, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition. Orphan designation is designed to provide drug developers with various benefits to support the development of novel therapies, including 10-years of market exclusivity once they receive marketing authorization in the EU, protocol assistance, administrative and procedural assistance, and reduced fees for regulatory activities. About Retinoblastoma Retinoblastoma is a tumor that originates in the retina and is the most common type of eye cancer in children. It occurs in approximately 1/14,000 - 1/18,000 live newborns and accounts for 15% of the tumors in the pediatric population < 1 year old. The average age of pediatric patients at diagnosis is 2, and it rarely occurs in children older than 6. In Europe, retinoblastoma has an estimated incidence rate of 1 per 13,844 live births (14.1 per million children under the age of 5) with approximately 300 children diagnosed per year (Stacey et al. 2021). Preserving life and preventing the loss of an eye, blindness and other serious effects of treatment that reduce the patient’s life span or the quality of life, remains a challenge. In addition, children with retinoblastoma have been more likely to lose their eye and die of metastatic disease in low-resource countries. About VCN-01 VCN-01 is a systemically administered oncolytic adenovirus designed to selectively and aggressively replicate within tumor cells and degrade the tumor stroma that serves as a significant physical and immunosuppressive barrier to cancer treatment. This unique mode-of-action enables VCN-01 to exert multiple antitumor effects by (i) selectively infecting and lysing tumor cells; (ii) enhancing the access and perfusion of co-administered chemotherapy products; and (iii) increasing tumor immunogenicity and exposing the tumor to the patient’s immune system and co-administered immunotherapy products. Systemic administration enables VCN-01 to exert its actions on both the primary tumor and metastases. VCN-01 has been administered to over 140 patients to date in clinical trials of different cancers, including PDAC (in combination with chemotherapy), head and neck squamous cell carcinoma (with an immune checkpoint inhibitor), ovarian cancer (with CAR-T cell therapy), colorectal cancer, and retinoblastoma (by intravitreal injection). More information on these clinical trials is available at Clinicaltrials.gov. About Theriva Biologics, Inc. Theriva Biologics (NYSE American: TOVX), is a diversified clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need. The Company’s wholly-owned Spanish subsidiary Theriva Biologics, S.L., has been developing a new oncolytic adenovirus platform designed for intravenous (IV), intravitreal and antitumoral delivery to trigger tumor cell death, improve access of co-administered cancer therapies to the tumor, and promote a robust and sustained anti-tumor response by the patient’s immune system. In addition to VCN-01, the Company’s clinical-stage candidates include (1) SYN-004 (ribaxamase) which is designed to degrade certain commonly used IV beta-lactam antibiotics within the gastrointestinal (GI) tract to prevent microbiome damage, thereby limiting overgrowth of pathogenic organisms such as VRE (vancomycin resistant Enterococci) and reducing the incidence and severity of acute graft-versus-host-disease (aGVHD) in allogeneic hematopoietic cell transplant (HCT) recipients); and (2) SYN-020, a recombinant oral formulation of the enzyme intestinal alkaline phosphatase (IAP) produced under cGMP conditions and intended to treat both local GI and systemic diseases. For more information, please visit Theriva Biologics’ website at www.therivabio.com. Forward-Looking Statement This release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions, and include statements regarding continuing to work closely with leading physicians and regulatory agencies to refine the Company’s clinical strategy for VCN-01 as an adjunct to chemotherapy in pediatric patients with advanced retinoblastoma and the potential benefits achievable from the grant of orphan drug designation. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to a number of risks and uncertainties, many of which are difficult to predict that could cause actual results to differ materially from current expectations and assumptions from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from current expectations include, among others, the Company’s ability to address the unmet medical needs for treatment of pediatric retinoblastoma, the Company’s ability to take advantage of the potential benefits of orphan drug designation, the Company’s ability to reach clinical milestones when anticipated, the Company’s product candidates demonstrating safety and effectiveness, as well as results that are consistent with prior results; the ability to complete clinical trials on time and achieve the desired results and benefits, continuing clinical trial enrollment as expected; the ability to obtain regulatory approval for commercialization of product candidates or to comply with ongoing regulatory requirements, regulatory limitations relating to the Company’s ability to promote or commercialize their product candidates for the specific indications, acceptance of product candidates in the marketplace and the successful development, marketing or sale of the Company’s products, developments by competitors that render such products obsolete or non-competitive, the Company’s ability to maintain license agreements, the continued maintenance and growth of the Company’s patent estate, the ability to continue to remain well financed and other factors described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023 and its other filings with the SEC, including subsequent periodic reports on Forms 10-Q and current reports on Form 8-K. The information in this release is provided only as of the date of this release, and Theriva Biologics undertakes no obligation to update any forward-looking statements contained in this release on account of new information, future events, or otherwise, except as required by law. For further information, please contact: Investor Relations: Chris CalabreseLifeSci Advisors, LLCccalabrese@lifesciadvisors.com917-680-5608 Source: Theriva Biologics, Inc.

免疫疗法孤儿药临床1期临床结果

2024-10-13

·药明康德

100%患者获临床缓解的CAR-T疗法；晚期癌症疾病控制率达92%的TCR-T疗法…… | 一周盘点

▎药明康德内容团队编辑本期看点 1. T细胞受体T细胞（TCR-T）疗法IMA203在一项1b期临床试验中令92%转移性黑色素瘤患者的疾病获得控制，且患者的中位缓解持续时间（DOR）超过一年。 2. B细胞成熟抗原（BCMA）靶向自体嵌合抗原受体（CAR）-T细胞疗法Ribrecabtagene autoleucel（DRL-1801）用于治疗复发/难治性多发性骨髓瘤患者的1期临床试验结果亮眼，首批8名接受治疗的患者均获得了临床缓解。 3. 用于治疗胶质母细胞瘤（GBM）的树突状细胞疫苗DOC1021相比于标准治疗（SOC）有望大幅延长患者的中位总生存期（OS）。药明康德内容团队整理 IMA203：公布1b期临床试验数据 Immatics公布了其在研TCR-T疗法IMA203用以治疗转移性黑色素瘤患者的最新1b期临床数据，这些患者先前接受过大量治疗。IMA203是由Immatics专有的ACTengine平台所开发的TCR-T细胞疗法，靶向由HLA-A*02呈递的黑素瘤抗原PRAME。PRAME是一种在各种实体瘤中经常表达的蛋白质，因此IMA203具潜力治疗广泛的癌症患者群体。此次公布的结果显示，92%患者的疾病获得控制且患者的中位DOR超过一年。根据此积极数据，Immatics预定在今年12月启动SUPRAME注册性3期试验，预计病患招募将于2026年完成，并在2026年初进行预定的中期分析。 Ribrecabtagene autoleucel（DRL-1801）：公布1期临床试验数据 Aurigene Oncology公司宣布其新型BCMA靶向自体CAR-T细胞疗法Ribrecabtagene autoleucel（DRL-1801）用于治疗复发/难治性多发性骨髓瘤的1期临床试验中首批8名患者的初步结果。所有患者都接受过大量的前期治疗，中位治疗线数为5.5线。大多数患者此前也曾接受过移植手术，并在移植后出现疾病进展。结果显示，100%的患者获得了临床缓解，其中有5名（62.5%）患者获得了严格的完全缓解（CR）。安全性方面，所有患者均未出现高级别的细胞因子释放综合征（CRS）或神经毒性。 DOC1021：公布1期临床试验的中期数据 Diakonos Oncology公司公布了其树突状细胞疫苗DOC1021用于治疗胶质母细胞瘤患者的1期开放标签试验的中期分析结果。该公司的树突状细胞疫苗由患者自身的免疫细胞与从肿瘤样本中提取的RNA和蛋白质结合制成。这种独特的方法可以靶向完整的癌症抗原谱，而无需进行任何基因修饰。通过利用天然的免疫信号通路，这种策略使免疫系统能将癌细胞视同受病毒感染的细胞一样进行识别和清除。此次公布的结果显示，15例患有挑战性肿瘤、MGMT未甲基化（通常预后不良）患者中有14例存活超过12个月。目前，中位随访时间为16个月，中位OS尚未达到。Kaplan-Meier（KM）分析将接受DOC1021治疗的患者与接受完整SOC的年龄匹配历史对照组患者进行比较，两组在统计学上具有差异（p=0.03）。KM预测接受DOC1021治疗的患者的OS为19.7个月，而接受SOC的未甲基化GBM患者的预期OS为12.7个月。安全性方面，没有剂量限制性毒性或令人担忧的严重不良事件（SAE）。 VIP943：公布1期临床试验数据 Vincerx Pharma公司公布了其抗体偶联药物（ADC）VIP943治疗复发/难治性急性髓系白血病（AML）、高危骨髓增生异常综合征（HR-MDS）和B细胞急性淋巴细胞白血病（B-ALL）的1期临床试验的结果。VIP943由抗CD123抗体、一种在细胞内切割的独特连接子和一种新型驱动蛋白纺锤体蛋白抑制剂（KSPi）有效载荷组成。Vincerx的CellTrapper技术可确保KSPi有效载荷积聚在靶细胞中，限制非靶细胞、非分裂细胞的吸收，从而减少非特异性释放。该1期剂量递增研究共招募了22名患者，他们很少对单一疗法产生应答。9名患者（6名AML患者和3名HR-MDS患者）至少接受了3次有效剂量的VIP943治疗。截至目前，1名复发性AML患者获得了完全缓解伴血液学不完全恢复（CRi），1名HR-MDS患者获得了完全缓解伴较少血液学恢复（CRL）。截至2024年8月的数据，VIP943显示出良好的安全性和耐受性，没有报告剂量限制性毒性，SAE与预期一致。 VK0214：公布1b期临床试验数据 Viking Therapeutics公司公布了其新型甲状腺激素受体β（TRβ）小分子激动剂VK0214在X-连锁肾上腺白质营养不良（X-ALD）患者中进行的1b期临床试验的积极数据。X-ALD是一种罕见的致命代谢性疾病，由ABCD1基因突变引起，这种突变会导致肾上腺脑白质营养不良蛋白（ALDP）功能障碍，从而引起脂肪酸的异常累积。临床前研究表明，甲状腺激素受体β型可以调节ABCD2相关基因的表达，ABCD2能够编码一种称为肾上腺脑白质营养不良相关蛋白（ADLRP）的代偿转运体，通过增加ABCD2表达可以使特定脂肪酸水平恢复正常。此次公布的研究结果表明，在为期28天的研究期间，VK0214每天用药一次的安全性和耐受性良好。此外，与安慰剂相比，接受VK0214治疗患者血浆中的超长链脂肪酸（VLCFAs）和其他血脂水平明显降低。 ▲VK0214治疗28天后患者的VLCFA与基线相比的变化（图片来源：参考资料[5]） ▲VK0214治疗28天后患者的其他血脂与基线相比的变化（图片来源：参考资料[5]） Paxalisib：公布1期临床试验的新数据 Kazia Therapeutics公司公布其口服PI3K/mTOR双抑制剂paxalisib联用放射疗法治疗PI3K通路突变实体瘤脑转移（BM）或软脑膜转移（LM）患者的1期临床试验的数据。此次公布的结果显示，paxalisib联用放疗总体耐受性良好。研究中最常报告的不良反应是恶心、呕吐和高血糖。接受45 mg paxalisib联用放疗患者中有67%获得了部分缓解（PR），接受最大耐受剂量治疗的患者中有超过三分之二的人获得了颅内缓解，这与单独使用全脑放疗的历史缓解率相比毫不逊色。 ELVN-001：公布1期临床试验的新数据 Enliven Therapeutics公司公布其小分子激酶抑制剂ELVN-001治疗复发/难治性或对现有酪氨酸激酶抑制剂（TKI）不耐受的慢性髓系白血病（CML）患者的1期临床试验的积极数据。ELVN-001是一种强效、高选择性、潜在“best-in-class”的小分子激酶抑制剂，专门针对CML患者的致癌驱动因子BCR-ABL融合蛋白。ELVN-001还具有针对耐药性最强的BCR-ABL1耐药突变体T315I和其他已知耐药突变体的活性。截至2024年6月25日的数据，ELVN-001的耐受性保持良好，没有患者减少剂量，未报告≥3级治疗相关的非血液学毒性反应。第24周时，患者的初始累积主要分子缓解（MMR）率为44%（8/18），与已获批的BCR-ABL1酪氨酸激酶抑制剂在1期临床试验中的数据相比具有优势。 Camonsertib：公布1期联合治疗试验数据 Repare Therapeutics公司公布了潜在”best-in-class”口服小分子ATR抑制剂camonsertib结合姑息性放疗，治疗携带ATM突变转移性肿瘤的1期临床试验数据。该试验纳入了17例携带ATM突变的转移性肿瘤患者，其中12人携带致病性ATM基因突变，5人携带ATM基因突变的意义不明变异（VUS）。这些患者的原发性癌症组织学来源包括胃肠道（n=5）、胰腺（n=5）、乳腺癌（n=2）、肺（n=2）、膀胱（n=2）和甲状腺（n=1）。此次公布的结果显示，2个月时，致病性ATM突变组有2例CR、5例PR和4例疾病稳定（SD）患者，而VUS组有1例PR和4例SD患者。6个月时，在9名可评估患者中，致病性ATM突变组有2例CR、4例PR和1例SD患者，而VUS组有1例SD和1例疾病进展患者。该早期临床数据表明camonsertib联合放疗治疗有可能对携带致病性ATM突变的肿瘤患者进行放射增敏，从而获得更高的临床益处。 Misetionamide：公布1期临床试验的中期数据 Panavance Therapeutics公司公布了其小分子疗法misetionamide（GP-2250）联用吉西他滨治疗晚期不可切除或转移性胰腺导管腺癌患者1期临床试验的中期数据。Misetionamide具有独特的作用机制，可抑制两种致癌转录因子c-MYC和NFκB。此次公布的结果显示，与单独使用吉西他滨的历史结果相比，接受misetionamide联用吉西他滨治疗患者的无进展生存期更长，约40%的患者达到PR或SD。此外，misetionamide联用吉西他滨的安全性良好。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看） [1] Vincerx Reports Positive Initial Clinical Data from Ongoing VIP943 Phase 1 Dose-Escalation Study and Provides Pipeline and Corporate Updates. Retrieved October 11, 2024, from https://www.globenewswire.com/news-release/2024/10/07/2959307/0/en/Vincerx-Reports-Positive-Initial-Clinical-Data-from-Ongoing-VIP943-Phase-1-Dose-Escalation-Study-and-Provides-Pipeline-and-Corporate-Updates.html [2] CNS 2024 Conference Presentation: Diakonos Oncology’s Autologous Dendritic Cell Vaccine (DOC1021) Yields 93% 12-Month Survival for Glioblastoma Patients. Retrieved October 11, 2024, from https://www.diakonosoncology.com/news/cns-2024-conference-presentation-diakonos-oncologys-autologous-dendritic-cell-vaccine-doc1021-yields-93-12-month-survival-for-glioblastoma-patients/ [3] Enliven Therapeutics Announces Positive Data Update from Phase 1 Clinical Trial of ELVN-001 in Chronic Myeloid Leukemia. Retrieved October 11, 2024, from https://www.globenewswire.com/news-release/2024/09/28/2954762/0/en/Enliven-Therapeutics-Announces-Positive-Data-Update-from-Phase-1-Clinical-Trial-of-ELVN-001-in-Chronic-Myeloid-Leukemia.html [4] Kazia Therapeutics Announces Presentation of Promising Phase I Data Evaluating Concurrent Paxalisib and Radiation Therapy in Patients with Solid Tumor Brain Metastases or Leptomeningeal Metastases Harboring PI3K Pathway Mutations at the American Society for Radiation Oncology 66th Annual Meeting. Retrieved October 11, 2024, from https://www.prnewswire.com/news-releases/kazia-therapeutics-announces-presentation-of-promising-phase-i-data-evaluating-concurrent-paxalisib-and-radiation-therapy-in-patients-with-solid-tumor-brain-metastases-or-leptomeningeal-metastases-harboring-pi3k-pathway-mutations--302265538.html [5] Viking Therapeutics Announces Results from Phase 1b Clinical Trial of VK0214 in Patients with X-ALD. Retrieved October 11, 2024, from https://www.prnewswire.com/news-releases/viking-therapeutics-announces-results-from-phase-1b-clinical-trial-of-vk0214-in-patients-with-x-ald-302270769.html [6] Repare Therapeutics Announces Phase 1 Data Highlighting Camonsertib in Combination with Radiotherapy Treatment Presented at the ASTRO Annual Meeting. Retrieved October 11, 2024, from https://ir.reparerx.com/news-releases/news-release-details/repare-therapeutics-announces-phase-1-data-highlighting [7] Panavance Therapeutics Provides Update on Phase 1 Clinical Trial of Misetionamide in Pancreatic Cancer at 2024 Visceral Medicine Congress. Retrieved October 11, 2024, from https://www.globenewswire.com/news-release/2024/10/09/2960394/0/en/Panavance-Therapeutics-Provides-Update-on-Phase-1-Clinical-Trial-of-Misetionamide-in-Pancreatic-Cancer-at-2024-Visceral-Medicine-Congress.html [8] Aurigene Oncology Limited Announces Promising Results of Phase 1 Study for India’s First Trial for Novel Autologous CAR-T Cell Therapy for Multiple Myeloma. Retrieved October 11, 2024, from https://www.businesswire.com/news/home/20241008755325/en/Aurigene-Oncology-Limited-Announces-Promising-Results-of-Phase-1-Study-for-India%E2%80%99s-First-Trial-for-Novel-Autologous-CAR-T-Cell-Therapy-for-Multiple-Myeloma/ [9] Immatics Announces Updated Phase 1b Clinical Data on ACTengine® IMA203 TCR-T Targeting PRAME in Melanoma Patients and Provides Update on Upcoming SUPRAME Phase 3 Trial. Retrieved October 10, 2024 from https://www.globenewswire.com/news-release/2024/10/10/2961129/0/en/Immatics-Announces-Updated-Phase-1b-Clinical-Data-on-ACTengine-IMA203-TCR-T-Targeting-PRAME-in-Melanoma-Patients-and-Provides-Update-on-Upcoming-SUPRAME-Phase-3-Trial.html [10] BlueRock Therapeutics’ investigational cell therapy bemdaneprocel for Parkinson’s disease shows positive data at 24-months. Retrieved October 11, 2024, from https://www.bayer.com/en/us/cell-therapy-bemdaneprocel [11] Estrella Immunopharma Achieves Complete Response in First Patient Treated with CD19- Redirected ARTEMIS® T-cells. Retrieved October 11, 2024, from https://www.estrellabio.com/news/estrella-immunopharma-achieves-complete-response-in-first-patient-treated-with-cd19--redirected-artemis%C2%AE-t-cells [12] INKmune Demonstrates Excellent Safety and Increased NK-Cell Activity in First Dosing Cohort. Retrieved October 11, 2024, from https://inmunebio.com/index.php/newsroom/2024-news/muneemonstratesxcellentafetyandncreasede20240926050502 [13] Theriva Biologics Announces Positive Outcome of Data and Safety Monitoring Committee (DSMC) Review in Phase 1b/2a Clinical Trial of SYN-004 (ribaxamase) in Allogeneic Hematopoietic Cell Transplant Recipients. Retrieved October 11, 2024, from https://www.globenewswire.com/news-release/2024/10/03/2957647/0/en/Theriva-Biologics-Announces-Positive-Outcome-of-Data-and-Safety-Monitoring-Committee-DSMC-Review-in-Phase-1b-2a-Clinical-Trial-of-SYN-004-ribaxamase-in-Allogeneic-Hematopoietic-Cel.html [14] Alentis Therapeutics Receives FDA IND Clearance for ALE.P02, a Novel CLDN1-ADC for the Treatment of Squamous Cancers. Retrieved October 11, 2024, from https://pipelinereview.com/alentis-therapeutics-receives-fda-ind-clearance-for-ale-p02-a-novel-cldn1-adc-for-the-treatment-of-squamous-cancers/ [15] NeuroBo Pharmaceuticals Announces Positive Top-Line Data From the SAD Part 1 of Its Phase 1 Clinical Trial Evaluating DA-1726 for the Treatment of Obesity. Retrieved October 11, 2024, from https://www.prnewswire.com/news-releases/neurobo-pharmaceuticals-announces-positive-top-line-data-from-the-sad-part-1-of-its-phase-1-clinical-trial-evaluating-da-1726-for-the-treatment-of-obesity-302261698.html [16] Calidi Biotherapeutics Announces FDA Clearance of the Northwestern University IND Application for CLD-101 Clinical Trial in High-Grade Glioma. Retrieved October 11, 2024, from https://www.globenewswire.com/news-release/2024/09/30/2955149/0/en/Calidi-Biotherapeutics-Announces-FDA-Clearance-of-the-Northwestern-University-IND-Application-for-CLD-101-Clinical-Trial-in-High-Grade-Glioma.html [17] Adcendo Announces US FDA Clearance of IND Application for Phase I/II First-in-Human ADCElerate-01 Trial of ADCE-D01 (uPARAP Receptor Targeting ADC) Trial in Patients With Metastatic and/or Unresectable Soft Tissue Sarcoma (STS). Retrieved October 11, 2024, from https://pipelinereview.com/adcendo-announces-us-fda-clearance-of-ind-application-for-phase-i-ii-first-in-human-adcelerate-01-trial-of-adce-d01-uparap-receptor-targeting-adc-trial-in-patients-with-metastatic-and-or-unresectabl/ [18] Safety and immunogenicity of CD40.HIVRI.Env, a dendritic cell-based HIV vaccine, in healthy HIV-uninfected adults: a first-in-human randomized, placebo-controlled, dose-escalation study (ANRS VRI06). Retrieved October 11, 2024, from https://www-thelancet-com.libproxy1.nus.edu.sg/journals/eclinm/article/PIIS2589-5370(24)00424-3/fulltext [19] Myrtelle Announces Significant Reduction in N-Acetylaspartate (NAA), a Key Biomarker, in Patients Treated in Its Phase 1/2 Clinical Trial of the Investigational Gene Therapy rAAV-Olig001-ASPA for Canavan Disease. Retrieved October 11, 2024, from https://www.prnewswire.com/news-releases/myrtelle-announces-significant-reduction-in-n-acetylaspartate-naa-a-key-biomarker-in-patients-treated-in-its-phase-12-clinical-trial-of-the-investigational-gene-therapy-raav-olig001-aspa-for-canavan-disease-302263264.html [20] Kiromic BioPharma Reports Favorable 10-Month Follow-Up Results for the First Patient Treated in its Deltacel-01 Clinical Trial. Retrieved October 11, 2024, from https://www.businesswire.com/news/home/20241001186898/en/Kiromic-BioPharma-Reports-Favorable-10-Month-Follow-Up-Results-for-the-First-Patient-Treated-in-its-Deltacel-01-Clinical-Trial [21] Recursion Announces FDA Clearance of Investigational New Drug Application for REC-1245, a Potential First-In-Class RBM39 Degrader for Biomarker-Enriched Solid Tumors and Lymphoma. Retrieved October 11, 2024, from https://www.globenewswire.com/news-release/2024/10/02/2956875/0/en/Recursion-Announces-FDA-Clearance-of-Investigational-New-Drug-Application-for-REC-1245-a-Potential-First-In-Class-RBM39-Degrader-for-Biomarker-Enriched-Solid-Tumors-and-Lymphoma.html [22] Kymera Therapeutics Announces FDA Clearance of Investigational New Drug Application for KT-621, a First-in-Class, Oral STAT6 Degrader. Retrieved October 11, 2024, from https://investors.kymeratx.com/news-releases/news-release-details/kymera-therapeutics-announces-fda-clearance-investigational-0 [23] Vanqua Bio Announces Positive Interim Results from Phase 1 Clinical Trial of VQ-101, an Orally Administered, Brain-Penetrant, Allosteric Activator of GCase for the Treatment of GBA-Parkinson’s and Related Disorders. Retrieved October 11, 2024, from https://www.globenewswire.com/news-release/2024/10/09/2960411/0/en/Vanqua-Bio-Announces-Positive-Interim-Results-from-Phase-1-Clinical-Trial-of-VQ-101-an-Orally-Administered-Brain-Penetrant-Allosteric-Activator-of-GCase-for-the-Treatment-of-GBA-Pa.html [24] Traws Pharma Announces Positive Topline Phase 1 Data for Flu Candidate, Tivoxavir Marboxil. Retrieved October 11, 2024, from https://www.globenewswire.com/news-release/2024/10/08/2959584/0/en/Traws-Pharma-Announces-Positive-Topline-Phase-1-Data-for-Flu-Candidate-Tivoxavir-Marboxil.html [25] Gain Therapeutics Presents Phase 1 GT-02287 Data at International Congress of Parkinson’s Disease and Movement Disorders Demonstrating Increase in GCase Activity in Healthy Volunteers. Retrieved October 11, 2024, from https://www.globenewswire.com/news-release/2024/09/30/2955049/0/en/Gain-Therapeutics-Presents-Phase-1-GT-02287-Data-at-International-Congress-of-Parkinson-s-Disease-and-Movement-Disorders-Demonstrating-Increase-in-GCase-Activity-in-Healthy-Volunte.html [26] Kura Oncology Announces Publication of Ziftomenib Phase 1 Results in The Lancet Oncology. Retrieved October 11, 2024, from https://www.globenewswire.com/news-release/2024/09/30/2955703/35186/en/Kura-Oncology-Announces-Publication-of-Ziftomenib-Phase-1-Results-in-The-Lancet-Oncology.html [27] Allakos Announces Phase 1 Trial Results of Subcutaneous AK006 in Healthy Volunteers. Retrieved October 11, 2024, from https://investor.allakos.com/news-releases/news-release-details/allakos-announces-phase-1-trial-results-subcutaneous-ak006 [28] Ultragenyx Provides Update on Stage 1 Cohorts in Pivotal Phase 1/2/3 Cyprus2+ Study Evaluating UX701 Gene Therapy for the Treatment of Wilson Disease. Retrieved October 4, 2024, from https://www.globenewswire.com/news-release/2024/10/03/2958032/0/en/Ultragenyx-Provides-Update-on-Stage-1-Cohorts-in-Pivotal-Phase-1-2-3-Cyprus2-Study-Evaluating-UX701-Gene-Therapy-for-the-Treatment-of-Wilson-Disease.html 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

细胞疗法临床结果免疫疗法疫苗临床1期

100 项与 Ribaxamase 相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D11196	-	-	DB16311

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
骨髓移植排斥反应	临床2期	美国	Theriva Biologics, Inc.	2021-02-15
梭菌感染	临床2期	美国	Theriva Biologics, Inc.	2015-10-01
梭菌感染	临床2期	保加利亚	Theriva Biologics, Inc.	2015-10-01
梭菌感染	临床2期	加拿大	Theriva Biologics, Inc.	2015-10-01
梭菌感染	临床2期	匈牙利	Theriva Biologics, Inc.	2015-10-01
梭菌感染	临床2期	波兰	Theriva Biologics, Inc.	2015-10-01
梭菌感染	临床2期	罗马尼亚	Theriva Biologics, Inc.	2015-10-01
梭菌感染	临床2期	塞尔维亚	Theriva Biologics, Inc.	2015-10-01
下呼吸道感染	临床2期	美国	Theriva Biologics, Inc.	2015-10-01
下呼吸道感染	临床2期	保加利亚	Theriva Biologics, Inc.	2015-10-01

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


NCT04692181 (GlobeNewswire) 人工标引	临床1/2期	造血干细胞移植	19	SYN-004 + IV Piperacillin/Tazobactam (Cohort 2)	鏇壓淵獵構襯襯範憲鏇(積襯蓋膚鏇願壓繭艱願) = A total of 15 SAEs were reported among 10 patients, with the most common SAE being infections and infestations, including sepsis. 壓廠獵願範構積願遞蓋 (願淵醖觸糧憲鹹憲獵選 ) 更多	积极	2024-10-03
				Placebo + IV Piperacillin/Tazobactam (Cohort 2)
NCT04692181 (Biospace) 人工标引	临床1/2期	造血干细胞移植	19	Ribaxamase	衊繭醖齋廠壓鏇製願顧(簾鹽壓糧構廠鹽願淵鹹) = 鹹膚顧繭膚糧鏇觸壓製蓋淵願膚鹹構窪鹹願淵 (廠鏇衊築糧範糧鑰衊糧 ) 更多	积极	2022-09-27
				Placebo	-
NCT02563106 (Pubmed) 人工标引	临床2期	艰难梭菌感染	413	Ceftriaxone+ribaxamase	憲淵顧製構窪鹹網鹽獵(襯蓋窪糧餘積糧醖鏇鏇) = 膚構衊觸蓋醖遞醖糧襯糧繭鏇廠鬱觸憲鏇製築 (鏇蓋範構膚齋廠鹽鹹積 ) 更多	积极	2019-05-01
				Ceftriaxone+Placebo	憲淵顧製構窪鹹網鹽獵(襯蓋窪糧餘積糧醖鏇鏇) = 鏇膚鹽壓網衊齋夢醖艱糧繭鏇廠鬱觸憲鏇製築 (鏇蓋範構膚齋廠鹽鹹積 ) 更多
NCT02563106 (CTgov)	临床2期	梭菌感染 \| 下呼吸道感染	413	SYN-004 (SYN-004)	窪鏇顧餘淵壓獵膚顧淵(選積鹽簾蓋鹽鏇顧觸壓) = 鏇簾膚鹽簾鹹構觸選構鬱糧蓋鏇餘願鹽鬱網夢 (繭積繭繭簾襯遞築簾膚, 艱鹽簾鑰獵願積觸醖蓋 ~ 鏇遞衊觸鑰膚衊醖簾齋) 更多	-	2018-03-12
				Placebo (Placebo)	窪鏇顧餘淵壓獵膚顧淵(選積鹽簾蓋鹽鏇顧觸壓) = 繭鹽鬱膚壓餘鬱鬱網積鬱糧蓋鏇餘願鹽鬱網夢 (繭積繭繭簾襯遞築簾膚, 夢憲願獵範餘衊膚鹽淵 ~ 製遞窪夢遞蓋醖壓鏇願) 更多
NCT02419001 (CTgov)	临床1/2期	-	11	Ceftriaxone+SYN-004 (Treatment Sequence AB)	憲網蓋製鹽鹹積築艱積(餘積憲築壓蓋顧製淵願) = 顧顧簾淵選糧淵鹹網製範簾觸壓網構艱壓願遞 (築淵醖鑰簾壓獵醖憲顧, 積願衊選築憲顧醖繭範 ~ 積觸選鹹鏇膚襯鹹壓鏇) 更多	-	2017-01-11
				Ceftriaxone+SYN-004 (Treatment Sequence AC)	憲網蓋製鹽鹹積築艱積(餘積憲築壓蓋顧製淵願) = 窪夢憲艱獵鑰憲觸觸鹹範簾觸壓網構艱壓願遞 (築淵醖鑰簾壓獵醖憲顧, 製夢觸淵觸範繭夢壓艱 ~ 繭壓廠鹹積艱憲製憲鹹) 更多