A Randomized Phase 2 Study of Ompenaclid Versus Placebo in Combination With FOLFIRI Plus Bevacizumab in Patients With Previously Treated RAS Mutant Advanced or Metastatic Colorectal Cancer

The purpose of this study is to measure tumor response to treatment with ompenaclid (RGX-202-01) in patients with previously treated RAS mutant advanced or metastatic CRC. All patients will receive treatment with FOLFIRI and bevacizumab. In addition, patients will be randomized to receive either ompenaclid 3000 mg BID or matching placebo (herein referred to as Study Drug). Each treatment cycle is 28 days in duration.

开始日期2023-10-10

申办/合作机构

Inspirna, Inc.

CTIS2023-503356-27-00

/ Not yet recruiting临床2期

A randomized Phase 2 Study of ompenaclid versus placebo in combination with FOLFIRI plus bevacizumab in patients with previously treated RAS mutant advanced or metastatic colorectal cancer - RGX-202-002

开始日期2023-10-02

申办/合作机构

Inspirna, Inc.

NCT03597581

/ Recruiting临床1期

A Phase 1 Study of RGX-202-01 (Ompenaclid) , a Small Molecule Inhibitor of the Creatine Transporter SLC6a8, as a Single Agent and as Combination Therapy in Patients With Advanced Gastrointestinal Malignancies With Select Expansion Cohorts

This is a Phase 1 study currently evaluating PO administered ompenaclid in combination with FOLFIRI and bevacizumab in patients with advanced (i.e., locally advanced and unresectable, or metastatic) previously treated colorectal adenocarcinoma. The single agent ompenaclid dose escalation stage and the ompenaclid in combination with FOLFIRI and bevacizumab dose escalation stage of the study has been completed; the expansion stage of ompenaclid in combination with FOLFIRI and bevacizumab is ongoing. In April-24 a protocol amendment added a new dose escalation and expansion stage which will evaluate ompenaclid in combination with FOLFOX and bevacizumab in patients with metastatic CRC. It is anticipated that a total of 30 patients will be enrolled in this new dose escalation and expansion stage of the study.

开始日期2018-06-05

申办/合作机构

Inspirna, Inc.

100 项与 Ompenaclid 相关的临床结果

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100 项与 Ompenaclid 相关的转化医学

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100 项与 Ompenaclid 相关的专利（医药）

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项与 Ompenaclid 相关的文献（医药）

2024-11-15·ACS Chemical Biology

Novel Corrector for Variants of SLC6A8: A Therapeutic Opportunity for Creatine Transporter Deficiency

Article

作者: Park, Hyejin ; Barrish, Joel C. ; Gechijian, Lara N. ; Brandon, Nicholas ; Rosskamp, Lea ; Labenski, Matthew T. ; Pullen, Nicholas ; Crotty, William ; Westgate, Christina ; van Kalken, Daniel ; Lyons, Matthew ; Conway, Leslie ; Iantosca, Gianna ; Rabin, Benjamin ; Deshusses, Chloe ; Jones, Lyn H. ; Rusu, Victor ; Brown, Dean G. ; Blanchette, Heather S. ; Rettenmaier, T. Justin ; Muncipinto, Giovanni ; Mathis, Robert ; Gross, Liam

Mutations in creatine transporter SLC6A8 cause creatine transporter deficiency (CTD), which is responsible for 2% of all cases of X-linked intellectual disability. CTD has no current treatments and has a high unmet medical need. Inspired by the transformational therapeutic impact of small molecule "correctors" for the treatment of cystic fibrosis, which bind to mutated versions of the CFTR ion channel to promote its trafficking to the cell surface, we sought to identify small molecules that could stabilize SLC6A8 as a potential treatment for CTD. We leveraged a novel chemoproteomic technology for ligand discovery, reactive affinity probe interaction discovery, to identify small-molecule fragments with photoaffinity handles that bind to SLC6A8 in a cellular environment. We synthesized a library of irreversible covalent analogs of these molecules to characterize in functional assays, which revealed molecules that could promote the trafficking of mutant SLC6A8 variants to the cell surface. Further medicinal chemistry was able to identify reversible drug-like small molecules that both promoted trafficking of the transporter and also rescued creatine uptake. When profiled across the 27 most prevalent SLC6A8 missense variants, we found that 10-20% of patient mutations were amenable to correction by our molecules. These results were verified in an endogenous setting using the CRISPR knock-in of selected missense alleles. We established in vivo proof-of-mechanism for correctors in a novel CTD mouse model with the P544L patient-defined variant knocked in to the SLC6A8 locus, where treatment with our orally bioavailable and brain penetrant tool corrector increased brain creatine levels in heterozygous female mice, validating correctors as a potential therapeutic approach for CTD.

2024-11-01·FOOD CONTROL

Compound probiotics starter: A solution for aflatoxin B1 reduction and meat quality improvement in fermented chicken jerky

作者: Qiu, Hongli ; Liu, Junhe ; Guo, Hongwei ; Yang, Wenli ; Zhu, Qun ; Chen, Jingyan ; Yin, Qingqiang ; Li, Genfeng ; Ma, Xinjun

This study investigated the effects of compound probiotics (CP) as starter cultures to reduce AFB1 residues and improve safety and quality of chicken jerky.First, Enterococcus faecalis, Candida utilis, Lactobacillus casein, and Bacillus subtilis were subjected to single strain fermentation experimentsThen, a three-factor, three-level response surface methodol. design was used to select the CP based on AFB1 degradation and histamine content.The optimal CP included Candida utilis, Lactobacillus casein and Bacillus subtilis at initial viable counts of 3.0 x 108, 3.0 x 109 and 3.0 x 109 CFU/mL resp., and then mixed at a volume ratio of 1:1:1.Each AFB1-contaminated chicken strip was fermented with 0.15 mL CP at 30 °C for 40 h then dried.AFB1 and histamine contents were reduced by 90.18% and 9.74% in fermented jerky, compared with the unfermented jerky (P < 0.05).Amino acid anal. showed lower serine and histidine contents and higher cysteine content in the fermented jerky. 16S rRNA sequencing unveiled a reduction in the absolute abundance of Staphylococcus saprophyticus.According to metabolomics and proteomics, CP inhibited methionine metabolism and increased lactic acid and alanine accumulation through cysteine and methionine pathways (KEGG map ID: map 00270), thereby maintaining low pH and improving flavor/safety.The results thus confirmed that C. utilis, L. casein, and B. subtilis are potential meat product starters.

2024-11-01·CHEMICAL ENGINEERING JOURNAL

Two-dimensional Ruddlesden-Popper perovskite solar cells with an efficiency exceeding 19 % by inserting a self-assembled monolayer

作者: Dong, Xue ; Zhou, Yipeng ; Tang, Yinhao ; Wu, Zhongbin ; Ran, Chenxin ; Li, Xin

Two-dimensional Ruddlesden-Popper (2DRP) phase perovskites have excellent long-term environmental and structure stability.However, the efficiency of 2DRP perovskite solar cells (PSCs) still lags seriously behind 3D counterparts due to the large exciton binding energy between the large-volume organic spacer and the inorganic plate compared to their 3D analogs.Here, a thin layer of self-assembled monolayer material (2-(3,6-dibromo-9H-carbazol-9-yl)ethyl)phosphonic acid (2PACz-Br) is employed between poly (3,4-ethylene dioxythiophene): poly(styrene sulfonate) (PEDOT: PSS) and β-guanidinopropanoic acid (β-GUA)-based perovskite film (β-GUA)₂(FA)₄Pb₅I₁₆ for efficient and stable 2DRP-based PSCs.The 2PACz-Br-based 2DRP perovskite films show superior crystallinity and quality compared with the control perovskite films.The defect d. in 2PACz-Br-based perovskite films is reduced, and the non-radiative recombination is further suppressed.The exptl. results show that introducing 2PACz-Br can effectively promote the matched energy levels between the perovskite thin film and the charge-carrier transport layer.Benefiting from the conducive collection and transmission of charge carriers, (β-GUA)₂(FA)₄Pb₅I₁₆ (n = 5)-based PSCs can realize an outstanding power conversion efficiency of 19.13%, much higher than that of devices without 2PACz-Br modification (17.70%).Moreover, 2PACz-Br-based 2DRP PSCs show better stability than the control devices.Our work paves the way to efficient and stable 2DRP-based PSCs by inserting a multifunctional self-assembled monolayer.

项与 Ompenaclid 相关的新闻（医药）

2025-01-15

·Pharmaceutical Technology

Regenxbio and Nippon Shinyaku forge $810m gene therapy deal

The partnership centres on two of Regenxbio’s gene therapies, RGX-121 and RGX-111. Credit: Jinda Noipho via Getty Images. Regenxbio has entered into a strategic collaboration worth $810m with Japan-based Nippon Shinyaku to advance gene therapies targeting the rare metabolic disorder mucopolysaccharidosis (MPS). Under the terms of the deal, which centres on two of Regenxbio’s assets – RGX-121 and RGX-111 – Regenxbio will receive $110m upfront and is eligible for up to $700m in development, regulatory, and sales milestones. Nippon Shinyaku gains rights to co-develop and commercialise the therapies in specified markets. MPS includes a group of rare, inherited metabolic disorders caused by the absence or malfunctioning of specific enzymes required to break down glycosaminoglycans (GAGs), complex sugar molecules. The accumulation of GAGs leads to progressive tissue and organ damage, developmental delays, and in severe cases, early mortality. The most advanced candidate in the partnership, RGX-121 (clemidsogene lanparvovec), targets Hunter syndrome, also known as MPS II, a condition caused by a mutation in the iduronate-2-sulfatase (IDS) gene. RGX-121 is designed to deliver a functional copy of the IDS gene, addressing the underlying cause of the disease. Symptoms of Hunter syndrome include cognitive impairment, skeletal abnormalities, and organ enlargement. In September 2024, Regenxbio reported positive results from the Phase II/III CAMPSIITE trial (NCT03566043) of RGX-121, showing an 85% median reduction of cerebrospinal fluid (CSF) levels of heparan sulphate (HS) D2S6, a key biomarker linked to disease activity. In June 2024, the company announced progress with the US Food and Drug Administration (FDA) to pursue an accelerated approval pathway, with a rolling biologics license application (BLA) submission underway. An accelerated approval decision is expected in 2025, and Regenxbio retains rights to a priority review voucher (PRV) for RGX-121. The second asset, RGX-111, is being developed for mucopolysaccharidosis type I (MPS I), caused by mutations in the α-l-iduronidase (IDUA) gene. The gene therapy aims to deliver a functional copy of the IDUA gene directly to the central nervous system (CNS), potentially slowing cognitive decline and other symptoms. Interim data from an ongoing Phase I/II trial (NCT03580083) has shown a favourable safety profile and promising biomarker data indicative of CNS activity. Regenxbio continues to advance its broader pipeline of gene therapies for rare diseases. The company’s wet age-related macular degeneration (wAMD) programme, ABBV-RGX-314, partnered with AbbVie, is being evaluated in two pivotal trials , ATMOSPHERE (NCT04704921) and ASCENT (NCT05407636). Plans are also underway for a Phase III study of the therapy in diabetic retinopathy. Additionally, the company is progressing RGX-202, a gene therapy for Duchenne muscular dystrophy. Following positive data from a Phase I/II trial, Regenxbio has aligned with the FDA on an accelerated approval pathway, with a BLA submission anticipated in 2026. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic By downloading this Whitepaper, you acknowledge that GlobalData may share your information with Cytiva Thematic and that your personal data will be used as described in their Privacy Policy

基因疗法临床3期临床结果优先审批引进/卖出

2024-11-24

·药明康德

一次注射有效半年的下一代RNAi疗法；12周减少肝脏脂肪近70%的MASH疗法…… | 一周盘点

▎药明康德内容团队编辑本期看点 1. RNAi疗法nucresiran治疗转甲状腺素蛋白（ATTR）介导淀粉样变性患者的早期临床试验结果亮眼，单次给药后第15天，患者平均血清转甲状腺素蛋白（TTR）水平降低超过90%，并维持至试验第6个月。 2. 抗体偶联药物PYX-201在一项1期临床试验中，在头颈部鳞状细胞癌患者中的疾病控制率（DCR）达100%，客观缓解率（ORR）达50%。 3. 用于治疗代谢功能障碍相关性脂肪性肝炎（MASH）和肥胖症的胰高血糖素样肽-1（GLP-1）/胰高血糖素双重受体激动剂pemvidutide在一项早期临床试验中展现积极疗效，患者用药12周后肝脏脂肪含量（LFC）相对基线减少68.5%。药明康德内容团队整理 Nucresiran（ALN-TTRsc04）：公布1期临床试验的新数据 Alnylam Pharmaceuticals公司公布了其下一代RNAi疗法nucresiran（曾用名ALN-TTRsc04）用于治疗转甲状腺素蛋白介导淀粉样变性患者的1期临床研究的最新数据。Nucresiran通过Alnylam专有IKARIA平台所开发，旨在迅速降低突变型和野生型TTR的水平，从根本上治疗ATTR淀粉样变性。Nucresiran具有实现更深层次和更持久TTR快速降低的潜力，从而能够减少用药频率。此次公布的显示，单次给予300 mg或更高剂量的nucresiran，可在第15天实现患者平均血清TTR水平快速降低超过90%，且这一效果持续至试验第6个月。数据表明，nucresiran具有每半年或每年进行一次皮下注射的潜力。Alnylam计划在2025年第一季度公布3期临床开发方案。 PYX-201：公布1期临床试验数据 Pyxis Oncology公司公布了其候选抗体偶联药物PYX-201治疗多种类型实体瘤的1期剂量递增研究的初步积极数据。PYX-201以微管抑制剂（优化的auristatin）作为有效载荷，能独特地靶向肿瘤细胞外基质（ECM）中的一种非细胞结构成分——纤连蛋白B结构域（Extradomain-B Fibronectin，EDB+FN）。截至2024年10月4日的数据，在6例疗效可评估的头颈部鳞状细胞癌患者中，观察到确认的ORR为50%，包括1例确认的完全缓解（CR）和2例确认的部分缓解（PR），DCR为100%。在所有6种研究者感兴趣的实体瘤（头颈部鳞状细胞癌、卵巢癌、非小细胞肺癌、HR+/HER2-乳腺癌、三阴性乳腺癌和肉瘤）患者（n=31）中，ORR为26%，研究人员还观察到剂量依赖性的缓解。此外，PYX-201通常耐受性良好，具有良好的安全性。 Pemvidutide：公布1b期临床试验数据 Altimmune公司公布了pemvidutide治疗代谢功能障碍相关性脂肪性肝病（MASLD）的1b期试验的最新数据。Pemvidutide是一种GLP-1/胰高血糖素双重受体激动剂，目前正在开发用于治疗MASH和肥胖症。该研究中，94名超重或肥胖且LFC≥10%的受试者按1：1：1：1的比例被分配接受pemvidutide（1.2 mg、1.8 mg和2.4 mg）或安慰剂，每周一次皮下注射，持续12周。数据显示，患者血浆中与心血管不良后果相关的多类炎症脂质有所减少。患者接受pemvidutide治疗12周后，LFC相对基线减少了68.5%，总胆固醇和甘油三酯分别最多降低了12.2%和44.6%。研究结果支持pemvidutide对MASH合并症的潜在益处，包括动脉粥样硬化、心脏病和代谢综合征。 IMA402：公布1期临床试验的初步数据 Immatics公司公布了其下一代半衰期延长的T细胞受体（TCR）双特异性分子IMA402的1期剂量递增试验数据。IMA402靶向由HLA-A*02呈递的黑色素瘤抗原PRAME。PRAME是一种在各种实体瘤中经常表达的蛋白质，因此IMA402具潜力治疗广泛的癌症患者群体。截至2024年11月6日的数据，IMA402在接受治疗的33例患者中表现出良好的耐受性。最常见的治疗相关不良事件多为轻度至中度细胞因子释放综合征（CRS）和短暂的淋巴细胞减少症。早期药代动力学数据表明，IMA402的中位半衰期约为7天，有望支持每两周一次的给药方案。9例PRAME检测阳性或未接受PRAME检测的患者中有7例实现了靶病灶缩小，1例皮肤黑色素瘤患者获得了确认的PR，靶病灶已缩小40.2%。 ▲1期剂量递增试验的初步数据（图片来源：参考资料[6]） Mipletamig：公布1b/2期临床试验的初步数据 Aptevo Therapeutics公司公布了其正在进行的1b/2期临床试验RAINIER中首例患者的治疗情况。该研究针对18岁及以上新确诊为急性髓系白血病且不符合强化诱导化疗条件的成人患者，旨在评估使用mipletamig联用标准治疗维奈托克和阿扎胞苷作为一线疗法的效果。APVO436是一种创新双特异性重组蛋白，它像两个单克隆抗体连接在一起，可以同时靶向肿瘤细胞表面的CD123和T淋巴细胞表面的CD3，从而将宿主免疫系统的T细胞重定向到患者的肿瘤细胞上，以快速和完全摧毁表面表达CD123的肿瘤细胞。此次公布的结果显示，首例患者在治疗的前30天内白血病细胞减少了90%，延续了先前研究中观察到的总体疗效趋势。 ST-503：IND申请获得FDA许可 Sangamo Therapeutics公司宣布，美国FDA批准了其在研表观遗传调节剂ST-503的IND申请，用于治疗特发性小纤维神经病变引起的顽固性疼痛（iSFN），这是一种慢性神经病理性疼痛。大量的证据表明，钠通道在介导神经病理性疼痛的病理生理过程中起着重要作用。开发特异性靶向Nav1.7钠通道的小分子药物具有挑战性，因为不同钠通道之间的结构相似性很高，难以实现选择性并避免脱靶效应。ST-503以腺相关病毒（AAV）为载体，携带工程化的锌指抑制子（ZFR），能够特异性地靶向编码Nav1.7钠通道的人类基因SCN9A，该基因在疼痛信号传导中起关键作用。在动物模型中，通过直接靶向SCN9A基因，ST-503被证明可以特异性地减少感觉神经元中Nav1.7钠通道的表达，并在单次鞘内给药后显著减少对疼痛的高敏感性。临床前研究表明，ST-503在非人灵长类动物中具有良好的耐受性，观察到显著的Nav1.7减少且没有脱靶效应，这显示了ST-503治疗慢性神经病理性疼痛的潜力。Sangamo公司正在准备进行1/2期临床研究，以评估单次剂量ST-503的安全性、耐受性和初步疗效，该药物将通过鞘内给药的方式给予iSFN患者，并计划于2025年中期开始患者招募。 ENS-002：1期临床试验完成首例受试者给药 Concerto Biosciences公司宣布，其用于治疗特应性皮炎的微生物组疗法ENS-002的1期临床试验已完成首例受试者给药。ENS-002是该公司利用一种名为kChip的强大筛选技术，从超过600万个皮肤微生物群落中发现的可缓解湿疹的细菌组合，该组合中包含三种菌株，能够抑制皮肤炎症的关键驱动因素金黄色葡萄球菌（S.aureus）。与类固醇和免疫抑制剂相比，该候选疗法旨在解决特应性皮炎背后的微生物组缺陷，以提供一条缓解更持久且副作用更少的潜在新治疗途径。 ▲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] Alnylam Announces Interim Phase 1 Data of Nucresiran (ALN-TTRsc04) Showing Rapid Knockdown of TTR that is Sustained at Six Months Following a Single Dose. Retrieved November 18, 2024 from https://investors.alnylam.com/press-release?id=28541 [2] Altimmune Presents New Data on the Effect of Pemvidutide on Inflammatory Lipids in Subjects with Metabolic Dysfunction-Associated Steatotic Liver Disease (MASLD) at The Liver Meeting® 2024. Retrieved November 22, 2024 from https://ir.altimmune.com/news-releases/news-release-details/altimmune-presents-new-data-effect-pemvidutide-inflammatory [3] Sangamo Therapeutics Announces U.S. FDA Clearance of IND Application for ST-503 for the Treatment of Idiopathic Small Fiber Neuropathy, a Type of Chronic Neuropathic Pain. Retrieved November 22, 2024 from https://www.businesswire.com/news/home/20241119527555/en/Sangamo-Therapeutics-Announces-U.S.-FDA-Clearance-of-IND-Application-for-ST-503-for-the-Treatment-of-Idiopathic-Small-Fiber-Neuropathy-a-Type-of-Chronic-Neuropathic-Pain [4] Pyxis Oncology Announces Favorable Preliminary PYX-201 Clinical Phase 1 Part 1 Data. Retrieved November 22, 2024 from https://www.globenewswire.com/news-release/2024/11/20/2984764/0/en/Pyxis-Oncology-Announces-Favorable-Preliminary-PYX-201-Clinical-Phase-1-Part-1-Data.html [5] First Patient Dosed in Aptevo's Ongoing RAINIER Trial Achieves 90% Reduction in Leukemic Blasts Within the First 30 Days of Treatment, Continues Overall Efficacy Trend Seen in Prior Mipletamig AML Studies. Retrieved November 22, 2024 from https://aptevotherapeutics.gcs-web.com/news-releases/news-release-details/first-patient-dosed-aptevos-ongoing-rainier-trial-achieves-90 [6] Immatics Announces Third Quarter 2024 Financial Results, Business Update and First Clinical Data on TCER® IMA402 Targeting PRAME. Retrieved November 22, 2024 from https://investors.immatics.com/news-releases/news-release-details/immatics-announces-third-quarter-2024-financial-results-business [7] Celldex Announces First Patient Dosed in Phase 1 Healthy Volunteer Study of CDX-622, a Bispecific Antibody, for the Treatment of Inflammatory Diseases. Retrieved November 22, 2024 from https://www.globenewswire.com/news-release/2024/11/20/2984404/0/en/Celldex-Announces-First-Patient-Dosed-in-Phase-1-Healthy-Volunteer-Study-of-CDX-622-a-Bispecific-Antibody-for-the-Treatment-of-Inflammatory-Diseases.html [8] Insilico Medicine receives IND clearance from FDA for ISM5939, an oral ENPP1 inhibitor treating solid tumors. Retrieved November 20, 2024, from https://www.eurekalert.org/news-releases/1065685 [9] AmacaThera Announces Phase 1 Results of First-in-Human Study with AMT-143, Exceeding Expectations and Paving the Way for a Novel Approach to Post-Operative Pain Relief. Retrieved November 20, 2024, from https://www.prnewswire.com/news-releases/amacathera-announces-phase-1-results-of-first-in-human-study-with-amt-143-exceeding-expectations-and-paving-the-way-for-a-novel-approach-to-post-operative-pain-relief-302310726.html [10] LAPIX Therapeutics Announces Positive Topline Phase 1 Clinical Data Results with LPX-TI641 for the Treatment of Autoimmune Diseases. Retrieved November 20, 2024, from https://www.globenewswire.com/news-release/2024/11/20/2984711/0/en/LAPIX-Therapeutics-Announces-Positive-Topline-Phase-1-Clinical-Data-Results-with-LPX-TI641-for-the-Treatment-of-Autoimmune-Diseases.html [11] Ocugen Announces Compelling Preliminary Data for OCU410—a Single Dose Novel Modifier Gene Therapy to Treat Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration. Retrieved November 20, 2024, from https://ir.ocugen.com/news-releases/news-release-details/ocugen-announces-compelling-preliminary-data-ocu410-single-dose [12] Genethon presents positive initial results from a phase 1/2/3 trial of its gene therapy (GNT0004) for Duchenne Muscular Dystrophy at ASGCT Breakthroughs in Muscular Dystrophy in Chicago. Retrieved November 22, 2024, from https://www.eurekalert.org/news-releases/1065424 [13] REGENXBIO INITIATES PIVOTAL PHASE OF AFFINITY DUCHENNE® TRIAL OF RGX-202 GENE THERAPY AND REPORTS POSITIVE FUNCTIONAL DATA. Retrieved November 18, 2024, from https://www.prnewswire.com/news-releases/regenxbio-initiates-pivotal-phase-of-affinity-duchenne-trial-of-rgx-202-gene-therapy-and-reports-positive-functional-data-302307989.html [14] Children's Hospital of Philadelphia Researchers Announce Promising Results from First-of-its-Kind, Multicenter, Phase 1 Gene Therapy Trial for Danon Disease. Retrieved November 22, 2024, from https://www.prnewswire.com/news-releases/childrens-hospital-of-philadelphia-researchers-announce-promising-results-from-first-of-its-kind-multicenter-phase-1-gene-therapy-trial-for-danon-disease-302308857.html [15] Spirovant Sciences Doses First Patient in the SAAVe Phase 1/2 Clinical Trial of Its Investigational, Aerosol-Delivered Genetic Medicine for the Treatment of Cystic Fibrosis. Retrieved November 22, 2024, from https://www.prnewswire.com/news-releases/spirovant-sciences-doses-first-patient-in-the-saave-phase-12-clinical-trial-of-its-investigational-aerosol-delivered-genetic-medicine-for-the-treatment-of-cystic-fibrosis-302304959.html [16] Intellia Announces First Clinical Evidence from Ongoing Phase 1 Study that Nexiguran Ziclumeran (nex-z), an In Vivo CRISPR/Cas9-Based Gene Editing Therapy, May Favorably Impact Disease Progression in Transthyretin (ATTR) Amyloidosis. Retrieved November 22, 2024 from https://www.globenewswire.com/news-release/2024/11/16/2982349/0/en/Intellia-Announces-First-Clinical-Evidence-from-Ongoing-Phase-1-Study-that-Nexiguran-Ziclumeran-nex-z-an-In-Vivo-CRISPR-Cas9-Based-Gene-Editing-Therapy-May-Favorably-Impact-Disease.html [17] CERo Therapeutics, Inc. Receives FDA Clearance of Investigational New Drug Application to Initiate Phase 1 Clinical Trial of Its Lead Compound CER-1236 in Acute Myelogenous Leukemia. Retrieved November 22, 2024 from https://www.globenewswire.com/news-release/2024/11/15/2982017/0/en/CERo-Therapeutics-Inc-Receives-FDA-Clearance-of-Investigational-New-Drug-Application-to-Initiate-Phase-1-Clinical-Trial-of-Its-Lead-Compound-CER-1236-in-Acute-Myelogenous-Leukemia.html [18] Acepodia Announces FDA Clearance of Investigational New Drug Application for ACE1831 in IgG4-Related Disease. Retrieved November 22, 2024 from https://www.prnewswire.com/news-releases/acepodia-announces-fda-clearance-of-investigational-new-drug-application-for-ace1831-in-igg4-related-disease-302306506.html [19] Fate Therapeutics Presents 6-Month Follow-up Data on First Patient Treated in Phase 1 Autoimmunity Study with Fludarabine-free Conditioning and FT819 Off-the-shelf, 1XX CAR T-cell Product Candidate at ACR Convergence. Retrieved November 22, 2024 from https://www.globenewswire.com/news-release/2024/11/18/2982757/24675/en/Fate-Therapeutics-Presents-6-Month-Follow-up-Data-on-First-Patient-Treated-in-Phase-1-Autoimmunity-Study-with-Fludarabine-free-Conditioning-and-FT819-Off-the-shelf-1XX-CAR-T-cell-P.html [20] Cabaletta Bio Presents Positive Clinical Safety and Efficacy Data on CABA-201 at ACR Convergence 2024. Retrieved November 22, 2024 from https://www.cabalettabio.com/news-media/press-releases/detail/119/cabaletta-bio-presents-positive-clinical-safety-and [21] Adicet Bio Announces First Lupus Nephritis Patient Dosed in Phase 1 Clinical Trial of ADI-001 in Autoimmune Diseases. Retrieved November 22, 2024 from https://investor.adicetbio.com/news-releases/news-release-details/adicet-bio-announces-first-lupus-nephritis-patient-dosed-phase-1 [22] Fate Therapeutics Highlights FT522 Off-the-shelf, ADR-armed CAR NK Cell Product Candidate at 2024 ACR Convergence. Retrieved November 22, 2024 from https://ir.fatetherapeutics.com/news-releases/news-release-details/fate-therapeutics-highlights-ft522-shelf-adr-armed-car-nk-cell [23] RESTEM Presents Phase 1 Data of its ULSC Program in Polymyositis/Dermatomyositis at ACR Convergence 2024. Retrieved November 22, 2024 from https://www.globenewswire.com/news-release/2024/11/18/2982728/0/en/RESTEM-Presents-Phase-1-Data-of-its-ULSC-Program-in-Polymyositis-Dermatomyositis-at-ACR-Convergence-2024.html [24] Theolytics Doses First Patient in Phase I/IIa Trial of THEO-260 in Ovarian Cancer. Retrieved November 22, 2024 from https://www.theolytics.com/post/theolytics-doses-first-patient-in-phase-i-iia-trial-of-theo-260-in-ovarian-cancer [25] Virion Therapeutics Reports First-Ever Clinical Responses with VRON-0200, a Novel Checkpoint Modifier for HBV Functional Cure, at AASLD's The Liver Meeting. Retrieved November 22, 2024 from https://www.prnewswire.com/news-releases/virion-therapeutics-reports-first-ever-clinical-responses-with-vron-0200-a-novel-checkpoint-modifier-for-hbv-functional-cure-at-aaslds-the-liver-meeting-302304358.html [26] GigaGen Doses First Patient in Phase 1 Trial of Recombinant Polyclonal Drug Candidate, GIGA-2339, for Hepatitis B Virus. Retrieved November 22, 2024 from https://www.globenewswire.com/news-release/2024/11/19/2983574/0/en/GigaGen-Doses-First-Patient-in-Phase-1-Trial-of-Recombinant-Polyclonal-Drug-Candidate-GIGA-2339-for-Hepatitis-B-Virus.html [27] Concerto Biosciences Announces First Participant Dosed with Live Biotherapeutic ENS-002 in Phase 1 Trial for Atopic Dermatitis. Retrieved November 22, 2024 from https://www.prnewswire.com/news-releases/concerto-biosciences-announces-first-participant-dosed-with-live-biotherapeutic-ens-002-in-phase-1-trial-for-atopic-dermatitis-302310102.html [28] Contineum Therapeutics Expands Clinical Development of PIPE-791 With FDA Authorization of Its Investigational New Drug (IND) Application for Chronic Pain. Retrieved November 22, 2024 from https://www.businesswire.com/news/home/20241114778987/en/Contineum-Therapeutics-Expands-Clinical-Development-of-PIPE-791-With-FDA-Authorization-of-Its-Investigational-New-Drug-IND-Application-for-Chronic-Pain/ [29] Karolinska Development’s portfolio company Umecrine Cognition presents positive interim data from Phase 1b/2a clinical study. Retrieved November 22, 2024 from https://www.globenewswire.com/news-release/2024/11/18/2982478/0/en/Karolinska-Development-s-portfolio-company-Umecrine-Cognition-presents-positive-interim-data-from-Phase-1b-2a-clinical-study.html [30] Omega Therapeutics Announces Successful Completion of Phase 1 Trial for Novel Epigenomic Controller, Prioritized Pipeline, Leadership Changes, and Third Quarter 2024 Financial Results. Retrieved November 22, 2024 from https://ir.omegatherapeutics.com/news-releases/news-release-details/omega-therapeutics-announces-successful-completion-phase-1-trial 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

临床结果siRNA临床1期临床2期

2024-11-19

·药明康德

潜在“best-in-class”DMD基因疗法启动关键性临床试验，显著改善患者肌肉功能

REGENXBIO公司今日宣布，在研疗法RGX-202的临床试验AFFINITY DUCHENNE已进入关键性阶段并完成首位患者给药。新闻稿指出，RGX-202是一种潜在“best-in-class”杜氏肌营养不良症（DMD）基因疗法。该公司还宣布了这一研究1/2期部分的新数据，包括首次公布患者功能性数据。今日公布的功能性数据包括在AFFINITY DUCHENNE试验的1/2期部分接受治疗的前5名参与者。包含3名接受剂量水平1治疗的4至10岁患者的12个月数据，两名接受剂量水平2（关键性阶段推荐剂量）的8岁和12岁患者的9个月数据。在所有5名参与者中，RGX-202显示出积极影响疾病进程的证据，患者在北极星步行能力评估（NSAA）和计时功能测试中，与匹配的外部自然疾病史对照相比，表现出功能稳定或改善。接受关键性研究推荐剂量RGX-202治疗的参与者在9个月时在NSAA和计时功能测试中表现出改善，超过了外部自然历史对照。该剂量下的NSAA平均得分提高了5.5分。 REGENXBIO还宣布了新的生物标志物数据，结果继续支持RGX-202稳定、高水平表达微抗肌萎缩蛋白。表达的蛋白适当地定位于肌膜（sarcolemma）。截至2024年11月1日，RGX-202耐受性良好，无严重不良事件和特殊关注不良事件。常见的药物相关不良事件包括恶心、呕吐和疲劳。基于与FDA的讨论，关键性临床试验将在约30名1岁及以上的可行走患者中评估剂量水平2的RGX-202的疗效。 RGX-202是一种潜在“best-in-class”的基因疗法，旨在改善杜氏肌营养不良症患者的肌肉功能和预后。RGX-202的独特之处在于编码的微抗肌萎缩蛋白中包含了关键的C末端（CT）结构域，在临床前研究中，CT结构域已被证明可保护肌肉免受收缩引起的压力并改善其自我修复能力。参考资料： [1] REGENXBIO INITIATES PIVOTAL PHASE OF AFFINITY DUCHENNE® TRIAL OF RGX-202 GENE THERAPY AND REPORTS POSITIVE FUNCTIONAL DATA. Retrieved November 18, 2024, from https://www.prnewswire.com/news-releases/regenxbio-initiates-pivotal-phase-of-affinity-duchenne-trial-of-rgx-202-gene-therapy-and-reports-positive-functional-data-302307989.html 内容来源于网络，如有侵权，请联系删除。

基因疗法临床结果引进/卖出临床研究

100 项与 Ompenaclid 相关的药物交易

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研发状态

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适应症	最高研发状态	国家/地区	公司	日期
结肠转移性腺癌	临床2期	比利时	Inspirna, Inc.	2023-10-10
结肠转移性腺癌	临床2期	法国	Inspirna, Inc.	2023-10-10
结肠转移性腺癌	临床2期	西班牙	Inspirna, Inc.	2023-10-10
RAS 突变结直肠癌	临床2期	美国	Inspirna, Inc.	2023-06-01
KRAS突变肿瘤	临床1期	美国	Inspirna, Inc.	2018-06-05
转移性结直肠癌	临床1期	美国	Inspirna, Inc.	2018-06-05
胃癌	临床1期	美国	Inspirna, Inc.	2018-06-05

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临床结果

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT03597581 (ESMO2024) 人工标引	临床1/2期	RAS 突变结直肠癌二线 RAS mutated (RASmt)	51	Ompenaclid 2400 mg BEV	膚願蓋糧顧築醖襯觸選(蓋艱壓淵鏇醖齋獵鏇積) = 蓋鏇鹽觸鹽顧壓襯糧鏇範鹹簾繭壓窪鹽繭襯築 (簾淵鹹淵窪壓繭鬱鹽夢 ) 更多	积极	2024-09-16
				Ompenaclid 3000 mg BEV	膚願蓋糧顧築醖襯觸選(蓋艱壓淵鏇醖齋獵鏇積) = 淵積衊構襯顧構選觸夢範鹹簾繭壓窪鹽繭襯築 (簾淵鹹淵窪壓繭鬱鹽夢 ) 更多
Corporate Publications 人工标引	临床2期	RAS 突变结直肠癌二线 RAS-mutated	41	Ompenaclid+FOLFIRI +bevacizumab	衊觸窪淵餘簾繭遞製壓(夢襯憲齋築鏇壓製網顧) = 齋窪築構鹽積衊鹽鑰製餘範糧衊淵齋蓋艱網鹹 (獵積艱簾顧觸積願衊膚 ) 更多	积极	2024-01-04
NCT03597581 (RGX-202-01, ESMO 12023 \| ESMO 22023) 人工标引	临床1/2期	RAS 突变结直肠癌二线 RAS mutation	33	Ompenaclid + FOLFIRI+bevacizumab	糧壓獵願壓膚淵鬱鏇築(蓋蓋願鬱顧膚憲夢願顧) = nausea (50%), diarrhea (44%) 襯繭淵觸壓齋簾艱淵艱 (醖網淵積顧齋簾鹹積構 ) 更多	积极	2023-10-22
				Ompenaclid + FOLFIRI+bevacizumab (RASm)
NCT03597581 (P-125, ESMO_GI2022)	临床1期	晚期结直肠腺癌二线	17	RGX-202-01 2400mg BID	繭築醖窪築夢衊淵蓋醖(夢壓積窪獵繭選簾醖膚) = 鹹淵築選觸鑰襯選窪膚艱糧鬱鏇蓋餘選範鑰顧 (範夢壓觸簾積壓餘遞夢 ) 更多	积极	2022-07-02
				RGX-202-01 3000mg BID	繭築醖窪築夢衊淵蓋醖(夢壓積窪獵繭選簾醖膚) = 醖築簾壓襯願獵範廠範艱糧鬱鏇蓋餘選範鑰顧 (範夢壓觸簾積壓餘遞夢 ) 更多
NCT03597581 (ESMO2022 \| ASCO2022) 人工标引	临床1期	晚期结直肠腺癌二线	-	bevacizumab+FOLFIRI+RGX-202	襯艱選齋築膚簾憲願鏇(淵膚淵築襯糧顧範廠淵) = 2 events (neutropenia, pulmonary embolus) 壓構鑰蓋構顧遞鑰鹹鏇 (顧衊願蓋選繭夢餘夢網 ) 更多	积极	2022-06-02
NCT03597581 (ASCO2022) 人工标引	临床1期	晚期结直肠腺癌二线 KRAS Mutation	16	FOLFIRI+RGX-202-01+bevacizumab (dose escalation)	膚鑰網鹹壓憲選艱鑰鹽(願選鑰選蓋膚憲衊壓衊) = 壓鬱顧願廠醖範簾積壓糧獵襯顧糧繭築鬱獵獵 (製蓋鹽鬱壓顧蓋獵襯蓋 ) 更多	积极	2022-06-02
				FOLFIRI+RGX-202-01+bevacizumab (dose expansion)	-
MDA2022	N/A	杜氏肌营养不良症	-	RGX-202 gene therapy	築範簾齋廠齋築製遞醖(築範築醖範遞壓範衊願) = 範構範齋獵窪衊繭鏇衊願夢襯鏇膚顧齋淵膚淵 (淵鏇餘衊獵構鏇積憲範 )	-	2022-03-13
NCT03597581 (ASCO 12020 \| ASCO 22020) 人工标引	临床1期	胃肠道肿瘤	17	FOLFIRI+RGX-202	願鏇繭網觸廠蓋選夢蓋(鏇齋膚築廠網窪獵壓糧) = Not reached 壓製獵遞淵蓋齋積膚範 (衊餘艱艱襯襯築構蓋簾 ) 更多	积极	2020-05-29