The primary objective of this study is to evaluate the safety, tolerability, MTD and RP2D of LP-184 in patients with advanced solid tumors who have relapsed from or are refractory to standard therapy or for whom no standard therapy is available. The secondary objectives are to characterize the PK of LP-184 and its metabolites in plasma and assess clinical activity of LP-184.
Participants will receive LP-184 infusion during Day 1 and Day 8 of each 21-day cycle, for a minimum of two cycles. Patients will be monitored for safety, PK, and clinical activity

开始日期2023-06-09

申办/合作机构

Lantern Pharma, Inc.

100 项与 Irofulven-2 相关的临床结果

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100 项与 Irofulven-2 相关的转化医学

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100 项与 Irofulven-2 相关的专利（医药）

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项与 Irofulven-2 相关的文献（医药）

2024-05-06·Cancer research communications

LP-184, a Novel Acylfulvene Molecule, Exhibits Anticancer Activity against Diverse Solid Tumors with Homologous Recombination Deficiency

Article

作者: Ewesuedo, Reginald B. ; Banerjee, Partha P. ; Bhatia, Kishor ; Zhou, Jianli ; Sharma, Panna ; Solarczyk, Kamil ; Prucsi, Zsombor ; Kulkarni, Aditya ; Kathad, Umesh ; Biyani, Neha ; Srivastava, Shiv

Abstract:

Homologous recombination (HR)-related gene alterations are present in a significant subset of prostate, breast, ovarian, pancreatic, lung, and colon cancers rendering these tumors as potential responders to specific DNA damaging agents. A small molecule acylfulvene prodrug, LP-184, metabolizes to an active compound by the oxidoreductase activity of enzyme prostaglandin reductase 1 (PTGR1), which is frequently elevated in multiple solid tumor types. Prior work demonstrated that cancer cell lines deficient in a spectrum of DNA damage repair (DDR) pathway genes show increased susceptibility to LP-184. Here, we investigated the potential of LP-184 in targeting multiple tumors with impaired HR function and its mechanism of action as a DNA damaging agent. LP-184 induced elevated DNA double-strand breaks in HR deficient (HRD) cancer cells. Depletion of key HR components BRCA2 or ataxia telangiectasia mutated (ATM) in cancer cells conferred up to 12-fold increased sensitivity to the LP-184. LP-184 showed nanomolar potency in a diverse range of HRD cancer models, including prostate cancer organoids, leiomyosarcoma cell lines, and patient-derived tumor graft models of lung, pancreatic, and prostate cancers. LP-184 demonstrated complete, durable tumor regression in 10 patient-derived xenograft (PDX) models of HRD triple-negative breast cancer (TNBC) including those resistant to PARP inhibitors (PARPi). LP-184 further displayed strong synergy with PARPi in ovarian and prostate cancer cell lines as well as in TNBC PDX models. These preclinical findings illustrate the potential of LP-184 as a pan-HRD cancer therapeutic. Taken together, our results support continued clinical evaluation of LP-184 in a large subset of HRD solid tumors.

Significance::

New agents with activity against DDR-deficient solid tumors refractory to standard-of-care therapies are needed. We report multiple findings supporting the potential for LP-184, a novel alkylating agent with three FDA orphan drug designations, to fill this void clinically: strong nanomolar potency; sustained, durable regression of solid tumor xenografts; synthetic lethality with HR defects. LP-184 adult phase IA trial to assess safety in advanced solid tumors is ongoing.

2023-10-02·Molecular cancer therapeutics

Conditional Dependency of LP-184 on Prostaglandin Reductase 1 is Synthetic Lethal in Pancreatic Cancers with DNA Damage Repair Deficiencies.

Article

作者: Astsaturov, Igor ; Ogier, Charline ; McDermott, Joseph R ; Surumbayeva, Aizhan ; Cai, Kathy Q ; Olszanski, Anthony J ; Restifo, Diana ; Sharma, Panna ; Kathad, Umesh ; Handorf, Elizabeth A ; Ma, Charlie ; Kulkarni, Aditya ; Bhatia, Kishor ; Cvetkovic, Dusica ; Schimke, Caleb ; Dos Santos, Troy

The greater efficacy of DNA-damaging drugs for pancreatic adenocarcinoma (PDAC) relies on targeting cancer-specific vulnerabilities while sparing normal organs and tissues due to their inherent toxicities. We tested LP-184, a novel acylfulvene analog, for its activity in preclinical models of PDAC carrying mutations in the DNA damage repair (DDR) pathways. Cytotoxicity of LP-184 is solely dependent on prostaglandin reductase 1 (PTGR1), so that PTGR1 expression robustly correlates with LP-184 cytotoxicity in vitro and in vivo. Low-passage patient-derived PDAC xenografts with DDR deficiencies treated ex vivo are more sensitive to LP-184 compared with DDR-proficient tumors. Additional in vivo testing of PDAC xenografts for their sensitivity to LP-184 demonstrates marked tumor growth inhibition in models harboring pathogenic mutations in ATR, BRCA1, and BRCA2. Depletion of PTGR1, however, completely abrogates the antitumor effect of LP-184. Testing combinatorial strategies for LP-184 aimed at deregulation of nucleotide excision repair proteins ERCC3 and ERCC4 established synergy. Our results provide valuable biomarkers for clinical testing of LP-184 in a large subset of genetically defined characterized refractory carcinomas. High PTGR1 expression and deleterious DDR mutations are present in approximately one third of PDAC making these patients ideal candidates for clinical trials of LP-184.

2022-10-01·Clinical lymphoma, myeloma & leukemia

MCL-319 LP-284 – A Highly Potent Small Molecule Targeting Mantle Cell Lymphoma

Article

作者: Zhou, Jianli ; Dave, Sandeep ; Sturtevant, Drew ; Bhatia, Kishor ; Biyani, Neha

Mantle cell lymphoma (MCL) is a rare and aggressive form of B-cell non-Hodgkin lymphoma (NHL). Approximately 40%-50% of MCL patients carry inactivating mutations in the gene ataxia telangiectasia mutated (ATM), a core component of the cell's DNA damage response system. MCL cells with mutations in ATM are known to have increased chromosomal imbalance, copy number loss, and hypersensitivity to DNA-damaging agents. LP-184, a small-molecule DNA-damaging agent, is known to be synthetically lethal in tumors with DNA-repair deficiencies, including tumors with ATM mutations. However, DNA damage by LP-184 is dependent on activation by the oxidoreductase prostaglandin reductase 1 (PTGR1), which is expressed at low levels in many hematological cancers. We synthesized LP-284, an enantiomer of LP-184, which retains synthetic lethality in cancers with impaired DNA damage repair pathway genes (e.g., ERCC1, ERCC2, ERCC3, ERCC6), but LP-284's activity is independent of PTGR1 expression. We demonstrated that LP-284 has enhanced tumoricidal activity in multiple hematological cancer cell lines using a B-cell NHL panel. The IC₅₀ values for all 6 MCL cell lines treated with LP-284 were among the lowest for all hematological cell lines tested with LP-284. These MCL cell lines included cell lines resistant to ibrutinib, zanubrutinib, venetoclax, and bortezomib. Additionally, among the 6 MCL cell lines tested, the 3 cell lines with mutations in the nucleotide excision repair (NER) or homologous recombination (HR) DNA damage repair pathways had lower LP-284 IC₅₀ values than the 3 cell lines without NER or HR mutations. Because ATM orchestrates the activities of the NER and HR pathways, MCL patients with ATM deficiencies are likely to have better responses to LP-284 treatment. Collectively, these data indicate that LP-284 is a promising preclinical DNA-damaging agent that possesses nanomolar-range anti-tumor activities in multiple and diverse MCL cell lines, with the potential to treat MCL patients who are resistant to other therapies.

项与 Irofulven-2 相关的新闻（医药）

2025-09-22

·药明康德

临床应答率达100%的创新炎症性肠病疗法；临床获益优于标准疗法的小分子抗癌疗法…… | 一周盘点

本期看点 1. 用于治疗溃疡性结肠炎（UC）的在研药物PALI-2108的早期临床数据亮眼，患者的临床应答率达100%。 2. 靶向广泛BRAF突变体的小分子抑制剂plixorafenib用于初治（未接受过MAPK抑制剂治疗）的BRAF V600突变乳头状甲状腺癌（PTC）患者，中位无进展生存期（mPFS）达到64个月，临床获益率（CBR）达85.7%，与现有标准疗法的历史数据相比表现更为优越，且安全性良好。 PALI-2108：公布1b期临床试验数据 Palisade Bio公司公布了其用于治疗溃疡性结肠炎的在研药物PALI-2108的1b期临床试验数据。在UC和纤维狭窄型克罗恩病（FSCD）中，较高的PDE4B表达与局部炎症活动有关。PALI-2108是一种PDE4抑制剂前药，能在UC和FSCD等疾病部位局部活化，显著提升病变组织内PDE4抑制剂浓度，同时减少全身暴露，从而降低腹泻等传统PDE4抑制剂的不良反应。 1a期安全性和药代动力学（PK）研究结果显示，PALI-2108在临床试验中表现出良好的安全性，未报告严重不良事件，也无实验室指标异常或心电图异常。在1b期临床试验中，PALI-2108用于治疗UC患者的临床应答率达100%，5名患者中有2名根据FDA定义的终点实现了临床缓解，显示出其治疗UC的潜力。生物标志物分析显示，UC患者在基线时升高的186个与纤维化及克罗恩病狭窄相关的基因表达得到正常化，表明PALI-2108在治疗FSCD方面也具有一定潜力。该公司针对FSCD患者的1b期临床试验预计将于2025年下半年启动。 Plixorafenib（FORE8394）：公布1/2a期试验的新数据 FORE Biotherapeutics公司公布了plixorafenib的1/2a期临床试验的新数据。Plixorafenib是一款可口服的下一代小分子BRAF突变体选择性抑制剂，旨在靶向广泛的BRAF突变体，同时不影响野生型RAF蛋白的活性。此次公布的结果显示，plixorafenib用于治疗BRAF基因变异的乳头状甲状腺癌和甲状腺未分化癌（ATC）可实现持久的疾病控制，与现有标准疗法的历史数据相比表现更为优越，且安全性良好，与既往报告的药物安全性特征一致。在初治的BRAF V600突变PTC患者中，接受plixorafenib治疗患者的mPFS达到64个月，CBR达85.7%。 JNJ-1900（NBTXR3）：公布1期联合治疗试验数据 Nanobiotix公司公布了一项正在进行的1期研究的最新结果。该研究评估了JNJ-1900联合免疫检查点抑制剂治疗晚期癌症患者的效果，此次分析重点聚焦于原发性皮肤黑色素瘤患者。JNJ-1900由功能化二氧化铪（HfO2）纳米颗粒组成，经由一次性瘤内注射给药并通过放射疗法激活。它的物理作用机制为：通过放疗激活，诱导被注射肿瘤内大量的肿瘤细胞死亡，随后触发适应性免疫反应和长期的抗癌记忆。得益于该物理作用机制，Nanobiotix公司认为该药物可扩展到任何可以通过放疗治疗的实体肿瘤和任何联合治疗方案中，特别是与免疫检查点抑制剂联合。2023年，Nanobiotix宣布与强生旗下杨森公司（现名为强生创新制药）达成全球共同开发和商业化该药物的许可协议。此次公布的结果显示，在既往接受过多线治疗（包括抗PD-1治疗）后疾病进展的黑色素瘤患者中，JNJ-1900联合疗法表现出良好的安全性及早期疗效信号。在可评估的19例患者中，所有病灶的最佳客观缓解率（ORR）达到47.4%，疾病控制率（DCR）达78.9%（依据RECIST 1.1标准）。所有接受治疗的患者（n=21）的中位总生存期（mOS）为14.6个月。研究者认为，这些数据支持通过随机临床试验进一步验证该方案作为抗PD-1治疗初治或耐药原发性皮肤黑色素瘤患者的潜在新选择。 LP-184：公布1a期临床试验数据 Lantern Pharma公司宣布，其在研疗法LP-184的1a期临床试验达到了所有主要终点，显示出良好的安全性和PK特征，并观察到初步的抗肿瘤活性。目前患者入组已完成，部分患者因持续获得临床获益而继续接受治疗。LP-184是一种可穿透血脑屏障的小分子药物，通过“合成致死”机制靶向DNA损伤修复（DDR）缺陷肿瘤。此次公布的结果显示，在达到或高于治疗剂量阈值的可评估癌症患者中，48%的患者观察到临床受益，其中包括对现有疗法无效或已耗尽治疗选择的患者。值得注意的是，在胶质母细胞瘤（GBM）、胃肠道间质瘤（GIST）和胸腺癌等难治性肿瘤中观察到持久的临床获益。生物标志物分析显示，LP-184在DDR通路相关基因（如CHK2、ATM、STK11/KEAP1）突变的患者中具有显著潜力，此类患者出现了明显的肿瘤缩小。此外，该药良好的安全性和耐受性为其作为单药治疗或与PARP抑制剂、免疫疗法联合使用提供了支持。 ORKA-001：公布1期临床试验的中期数据 Oruka Therapeutics公司公布了其长效IL-23p19抗体ORKA-001的1期临床试验的中期数据。ORKA-001是一种新型皮下注射的半衰期延长型单克隆抗体，靶向IL-23p19，旨在为包括斑块状银屑病（PsO）在内的慢性皮肤病提供新的治疗方法。该药物在设计上与经过验证的risankizumab具有相似的表位结合和亲和力，有望实现每年给药一到两次的给药频率。临床数据显示，ORKA-001的半衰期约为100天，增加了其每年给药一次的潜力。ORKA-001具有良好的PK特征，可实现更高的药物暴露水平，从而可能带来更强的疗效和更长的停药后缓解期。此外，该药物在试验中耐受性良好，安全性特征与IL-23p19类药物一致。参考资料（可上下滑动查看） [1] Lion TCR Achieves Triple FDA Milestones with IND Clearance for Chronic Hepatitis B Following Earlier Fast Track and Orphan Drug Designations. Retrieved September 19, 2025, from https://www.prnewswire.com/news-releases/lion-tcr-achieves-triple-fda-milestones-with-ind-clearance-for-chronic-hepatitis-b-following-earlier-fast-track-and-orphan-drug-designations-302555819.html [2] Myrtelle Announces Nature Medicine Publication of Interim Results from Its Phase 1/2 Clinical Trial of Investigational Gene Therapy rAAV-Olig001-ASPA for Canavan Disease. Retrieved September 19, 2025, from https://www.prnewswire.com/news-releases/myrtelle-announces-nature-medicine-publication-of-interim-results-from-its-phase-12-clinical-trial-of-investigational-gene-therapy-raav-olig001-aspa-for-canavan-disease-302557585.html [3] Navigator Medicines Announces Positive Phase 1a Data from its Program of Potential Best-in-Class Bispecific Antibodies; NAV-240 Poised for Further Development in Inflammatory Disorders and Autoimmune Diseases. Retrieved September 19, 2025, from https://www.globenewswire.com/news-release/2025/09/17/3151352/0/en/Navigator-Medicines-Announces-Positive-Phase-1a-Data-from-its-Program-of-Potential-Best-in-Class-Bispecific-Antibodies-NAV-240-Poised-for-Further-Development-in-Inflammatory-Disord.html [4] Oruka Therapeutics Announces Positive Interim Phase 1 Results for ORKA-001. Retrieved September 19, 2025, from https://www.globenewswire.com/news-release/2025/09/17/3151351/0/en/Oruka-Therapeutics-Announces-Positive-Interim-Phase-1-Results-for-ORKA-001.html [5] Skyhawk Therapeutics Announces Positive First Interim Results in Patients from its Phase 1 Clinical Trial of SKY-0515 as a Treatment for Huntington's Disease. Retrieved September 19, 2025 from https://www.prnewswire.co.uk/news-releases/skyhawk-therapeutics-announces-positive-first-interim-results-in-patients-from-its-phase-1-clinical-trial-of-sky-0515-as-a-treatment-for-huntingtons-disease-302559094.html [6] Lantern Pharma’s LP-184 Phase 1a Clinical Trial Achieves All Primary Endpoints with Robust Safety Profile and Promising Antitumor Activity in Multiple Advanced Solid Tumors. Retrieved September 19, 2025 from https://www.businesswire.com/news/home/20250916022448/en/Lantern-Pharmas-LP-184-Phase-1a-Clinical-Trial-Achieves-All-Primary-Endpoints-with-Robust-Safety-Profile-and-Promising-Antitumor-Activity-in-Multiple-Advanced-Solid-Tumors [7] Palisade Bio Reports Positive PALI-2108 Phase 1b Clinical Data. Retrieved September 19, 2025 from https://www.globenewswire.com/news-release/2025/09/17/3151657/0/en/Palisade-Bio-Reports-Positive-PALI-2108-Phase-1b-Clinical-Data.html [8] NANOBIOTIX Announces New Results From a Phase 1 Study Evaluating JNJ-1900 (NBTXR3) in Combination With Immune Checkpoint Inhibitors as a 2L+ Therapy for Patients With Primary Cutaneous Melanoma Resistant to Anti-PD-1. Retrieved September 19, 2025 from https://www.globenewswire.com/news-release/2025/09/17/3152017/0/en/NANOBIOTIX-Announces-New-Results-From-a-Phase-1-Study-Evaluating-JNJ-1900-NBTXR3-in-Combination-With-Immune-Checkpoint-Inhibitors-as-a-2L-Therapy-for-Patients-With-Primary-Cutaneou.html [9] Astria Therapeutics Announces Positive Initial Results from the Phase 1a Healthy Subject Trial of STAR-0310 at the European Academy of Dermatology and Venereology. Retrieved September 19, 2025 from https://ir.astriatx.com/news-releases/news-release-details/astria-therapeutics-announces-positive-initial-results-phase-1a [10] Rubedo Life Sciences annuncia la concessione dell’autorizzazione IND da parte della U.S. FDA per il candidato farmaco principale RLS-1496, modulatore selettivo di GPX4, per la cheratosi attinica, nonché l’espansione del comitato consultivo clinico. Retrieved September 19, 2025 from https://www.businesswire.com/news/home/20250917265149/it [11] ProteinQure Announces First Patient Dosed in Phase I Clinical Trial of PQ203 in Advanced Metastatic Cancer. Retrieved September 19, 2025 from https://www.businesswire.com/news/home/20250917696518/en/ProteinQure-Announces-First-Patient-Dosed-in-Phase-I-Clinical-Trial-of-PQ203-in-Advanced-Metastatic-Cancer [12] Luxa Biotechnology Announces Publication in Cell Stem Cell Describing Positive Clinical Results for First-in-Human Clinical Trial for Dry AMD Therapy. Retrieved September 19, 2025 from https://www.luxabiotech.com/press-release/luxa-biotechnology-announces-publication-in-cell-stem-cell-describing-positive-clinical-results-for-first-in-human-clinical-trial-for-dry-amd-therapy 免责声明：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：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2期临床1期放射疗法临床结果

2025-08-10

·药明康德

使病变发生率降低94%的抗病毒疗法；多款胶质母细胞瘤创新疗法进入临床…… | 一周盘点

本期看点 1. Assembly Biosciences公司公布了两款抗病毒疗法的积极早期临床数据，分别用于治疗复发性生殖器疱疹和慢性丁型肝炎病毒（cHDV）感染。 2. 两款针对DNA修复缺陷开发的小分子疗法获美国FDA批准开展临床试验，用于治疗胶质母细胞瘤（GBM）。 ABI-5366：公布1期临床试验的中期数据 Assembly Biosciences公司公布了一种用于治疗复发性生殖器疱疹的长效解旋酶-引物酶抑制剂ABI-5366的1b期临床试验数据。该候选疗法通过靶向病毒解旋酶-引物酶复合体发挥作用。这是一种在HSV-1和HSV-2中都保守的重要病毒酶复合体，并且宿主细胞中没有类似的酶复合体。在短期临床研究中，HSV解旋酶-引物酶抑制剂显示出比核苷类似物更佳的疗效潜力。在具有统计效力的主要抗病毒终点——HSV-2病毒脱落率方面，接受每周350 mg剂量的队列在29天的评估期内，病毒脱落率较安慰剂组显著降低了94%（p<0.01），显示出强效的抗病毒活性。这一降幅超过了Assembly Biosciences公司为本研究设定的80%-85%的降低目标。在一项次要临床终点——生殖器病变发生率方面，相比于安慰剂组，每周350 mg剂量组的发生率降低94%（p<0.01）。此外，在评估ABI-5366每周口服剂量的前两个队列中，观察到了良好的安全性和耐受性特征。 ABI-6250：公布1a期临床试验的中期数据 Assembly Biosciences公司公布了其正在开展的ABI-6250的1a期临床试验中多个队列的中期数据结果。ABI-6250是该公司研发的一种口服小分子HDV进入抑制剂。截至目前已评估的队列数据显示，口服给药后，ABI-6250的平均半衰期为4天，支持其每日一次的口服给药目标。此外，在单次和多次给药队列中均观察到总血清胆汁酸（TBA）呈剂量依赖性升高。这些生物标志物数据表明，ABI-6250能够强效作用于钠-牛磺胆酸共转运多肽（NTCP，HDV用于感染肝细胞的受体），同时也为后续在cHDV感染患者中评估抗病毒活性的潜在剂量范围提供了依据。 GLIX1：IND申请获得FDA许可 Hemispherian公司宣布，其针对胶质母细胞瘤及其他实体瘤DNA修复缺陷的小分子药物GLIX1的IND申请已获得美国FDA批准。公司将启动GLIX1的首次人体1期临床试验，旨在评估该药物在复发性胶质母细胞瘤患者中的安全性、耐受性、药代动力学及初步疗效。GLIX1基于公司专有的GLIX技术平台开发，能够选择性清除癌细胞，同时最大程度减少对健康组织的影响。在临床前研究中，GLIX1能在经过验证的动物模型中清除肿瘤且副作用有限，具有治疗胶质瘤及其他侵袭性癌症的潜力。 STAR-001（LP-184）：IND申请获得FDA许可 Lantern Pharma旗下全资子公司Starlight Therapeutics宣布，其针对胶质母细胞瘤首次进展后患者开展的1b/2a期临床试验已获美国FDA批准。该试验将评估STAR-001（LP-184）联合螺内酯（spironolactone）的疗效。STAR-001是一种可穿透血脑屏障的小分子药物，通过“合成致死”机制靶向DNA损伤修复（DDR）缺陷肿瘤，尤其对高表达PTGR1的肿瘤细胞具有选择性杀伤作用——约60%的GBM细胞具备足够水平的PTGR1来激活STAR-001。而螺内酯作为一种已获批的药物，可通过降解核苷酸切除修复（NER）通路中的关键蛋白ERCC3，诱导NER缺陷（NERD），从而显著增强STAR-001的疗效，使GBM细胞更难以修复药物造成的DNA损伤。这一联合策略具有科学合理性，尤其为在复发阶段已产生耐药的GBM患者带来了新的治疗希望。参考资料： [1] 큐로셀, 'CD19 CAR-T' 루푸스 국내 1/2상 "IND 승인". Retrieved Agugst 8, 2025, from https://www.biospectator.com/news/view/25924 [2] Energenesis Biomedical Announces Positive Phase I Results for ENERGI-F705PD, a Potential Disease-Modifying Treatment for Parkinson's Disease. Retrieved Agugst 8, 2025, from https://www.prnewswire.com/news-releases/energenesis-biomedical-announces-positive-phase-i-results-for-energi-f705pd-a-potential-disease-modifying-treatment-for-parkinsons-disease-302519474.html [3] Nuvectis Pharma Provides Final Clinical Data Update from the NXP800 Phase 1b Study in Ovarian Cancer and Reports Completion of the NXP900 Phase 1a Dose Escalation Study. Retrieved Agugst 8, 2025, from https://nuvectis.com/press-release-view/?i=151930 [4] Hemispherian Receives FDA IND Clearance for First-in-Class Glioblastoma Therapeutic, GLIX1. Retrieved Agugst 8, 2025, from https://www.prnewswire.com/news-releases/hemispherian-receives-fda-ind-clearance-for-first-in-class-glioblastoma-therapeutic-glix1-302520810.html [5] Cocrystal Pharma Presents Phase 1 Results for Pan-Viral Inhibitor CDI-988 at Department of Defense Medical Conference. Retrieved Agugst 8, 2025, from https://www.globenewswire.com/news-release/2025/08/05/3127312/0/en/Cocrystal-Pharma-Presents-Phase-1-Results-for-Pan-Viral-Inhibitor-CDI-988-at-Department-of-Defense-Medical-Conference.html [6] Quantum Biopharma Announces Positive Results of the Clinical Study Report (CSR) for the Phase 1 Multiple Ascending Dose (MAD) Clinical Trial of Experimental Multiple Sclerosis Drug Lucid-21-302 (Lucid-MS). Retrieved Agugst 8, 2025, from https://www.globenewswire.com/news-release/2025/08/05/3127241/0/en/Quantum-Biopharma-Announces-Positive-Results-of-the-Clinical-Study-Report-CSR-for-the-Phase-1-Multiple-Ascending-Dose-MAD-Clinical-Trial-of-Experimental-Multiple-Sclerosis-Drug-Luc.html [7] Assembly Biosciences Reports Interim Phase 1a Data from Clinical Study of Oral Entry Inhibitor Candidate ABI-6250 for Hepatitis Delta Virus. Retrieved Agugst 8, 2025, from https://www.globenewswire.com/news-release/2025/08/06/3128674/16259/en/Assembly-Biosciences-Reports-Interim-Phase-1a-Data-from-Clinical-Study-of-Oral-Entry-Inhibitor-Candidate-ABI-6250-for-Hepatitis-Delta-Virus.html [8] 바스테라, '경구 효소모방체' PAH "美1상 IND 승인". Retrieved Agugst 8, 2025, from https://www.biospectator.com/news/view/25968 [9] Lantern Pharma’s Subsidiary, Starlight Therapeutics, Announces U.S. Food and Drug Administration Clearance of IND for Phase Ib/2a Glioblastoma Multiforme (GBM) Trial. Retrieved Agugst 8, 2025, from https://www.businesswire.com/news/home/20250806962549/en/Lantern-Pharmas-Subsidiary-Starlight-Therapeutics-Announces-U.S.-Food-and-Drug-Administration-Clearance-of-IND-for-Phase-Ib2a-Glioblastoma-Multiforme-GBM-Trial [10] K36 Therapeutics Receives FDA Clearance of Investigational New Drug (IND) Application for KTX-2001 in Metastatic Castration-Resistant Prostate Cancer (mCRPC) and Announces Clinical Trial Collaboration with Bayer for Supply of Darolutamide. Retrieved Agugst 8, 2025, from https://www.prnewswire.com/news-releases/k36-therapeutics-receives-fda-clearance-of-investigational-new-drug-ind-application-for-ktx-2001-in-metastatic-castration-resistant-prostate-cancer-mcrpc-and-announces-clinical-trial-collaboration-with-bayer-for-supply-of-daro-302523702.html [11] AbCellera Reports Q2 2025 Business Results & First Participants Dosed in a Phase 1 Clinical Trial of ABCL635 for Vasomotor Symptoms. Retrieved Agugst 8, 2025, from https://www.biospace.com/press-releases/abcellera-reports-q2-2025-business-results-first-participants-dosed-in-a-phase-1-clinical-trial-of-abcl635-for-vasomotor-symptoms [12] AbCellera Reports Q2 2025 Business Results & First Participants Dosed in a Phase 1 Clinical Trial of ABCL635 for Vasomotor Symptoms. Retrieved Agugst 8, 2025, from https://investors.abcellera.com/news/news-releases/2025/AbCellera-Reports-Q2-2025-Business-Results--First-Participants-Dosed-in-a-Phase-1-Clinical-Trial-of-ABCL635-for-Vasomotor-Symptoms/default.aspx [13] Assembly Biosciences Reports Positive Interim Results from Phase 1b Clinical Study of Long-Acting Helicase-Primase Inhibitor Candidate ABI-5366 Showing Reductions in Viral Shedding Rate and Genital Lesion Rate in Recurrent Genital Herpes. Retrieved Agugst 8, 2025, from https://www.globenewswire.com/news-release/2025/08/08/3130113/16259/en/Assembly-Biosciences-Reports-Positive-Interim-Results-from-Phase-1b-Clinical-Study-of-Long-Acting-Helicase-Primase-Inhibitor-Candidate-ABI-5366-Showing-Reductions-in-Viral-Shedding.html 免责声明：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：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1期临床申请

2025-08-07

·PipelineReview

Lantern Pharma's Subsidiary, Starlight Therapeutics, Announces U.S. Food and Drug Administration Clearance of IND for Phase Ib/2a Glioblastoma Multiforme (GBM) Trial

DALLAS, TX, USA I August 06, 2025 I Starlight Therapeutics , a wholly owned subsidiary of Lantern Pharma Inc. (NASDAQ: LTRN), today announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for a Phase Ib/2a clinical trial to evaluate STAR-001 (LP-184) in combination with spironolactone for patients with glioblastoma multiforme (GBM) at first progression. “GBM remains one of the most difficult cancers to treat, with virtually no meaningful therapeutic advances in nearly 17 years. We believe our unique combination approach has the potential to offer new hope for patients and their families.” Dr. Chamberlain Share The planned Phase Ib/2a clinical trial (IND 178511) is designed to investigate the safety, tolerability, and preliminary efficacy of STAR-001 in combination with spironolactone in patients with recurrent GBM, one of the deadliest and most aggressive forms of brain cancer. GBM represents approximately 15% of all brain tumors and has a median survival of approximately 12 months from initial diagnosis, with fewer than 5% of patients surviving beyond five years. “This FDA clearance represents a significant milestone for Starlight Therapeutics and our mission to bring innovative treatment options to patients facing the most challenging forms of brain and CNS cancers,” said Dr. Marc Chamberlain , Chief Medical Officer of Starlight Therapeutics. “GBM remains one of the most difficult cancers to treat, with virtually no meaningful therapeutic advances in nearly 17 years. We believe our unique combination approach has the potential to offer new hope for patients and their families while advancing a novel mechanism to challenge these recurrent brain cancers.” Targeting GBM Through Innovative Synthetic Lethality STAR-001 (also referred to as LP-184 when focused on other solid tumor indications) is a synthetically lethal, brain-penetrant, novel DNA-damaging agent that has demonstrated promising preclinical activity against various solid tumors, including pediatric and adult brain cancers. Lantern Pharma has previously been awarded both FDA Orphan Drug Designation and FDA Fast Track Designation for STAR-001 in GBM, underscoring the significant unmet medical need and the drug’s potential to address it. An existing Phase 1a trial to determine safety, dosing, the maximum tolerated dose (MTD) and tolerability across a range of solid tumors, including GBM, is currently underway and expected to complete enrollment in the current quarter. This trial is expected to help establish a baseline dosing level and expectations of drug-concentration in tumor and plasma for future clinical trials, including those being planned by Starlight Therapeutics. The combination of STAR-001 with spironolactone , an existing FDA approved drug, represents a scientifically rational approach to treating GBM. STAR-001 is a blood-brain barrier permeable small molecule that leverages synthetic lethality to exploit DNA damage repair (DDR) deficiencies, particularly those overexpressing PTGR1. Approximately 60% of GBM cells overexpress PTGR1 levels sufficient to activate STAR-001. Spironolactone significantly enhances this therapeutic effect by inducing degradation of ERCC3, a critical protein involved in nucleotide excision repair (NER). This mechanism leads to NER deficiency (NERD), making GBM cells significantly more sensitive to STAR-001 and less capable of repairing the DNA damage caused by the administration of STAR-001. This synergistic approach is particularly promising in GBM which has often become resistant to therapies in the recurrent setting. AI-Driven Drug Development Platform Starlight Therapeutics was formed to pursue the focused clinical development of central nervous system (CNS) oncology indications developed and advanced through Lantern Pharma’s proprietary artificial intelligence drug development platform, RADR®. This AI-driven approach enables the identification of optimal drug-cancer combinations and patient populations most likely to benefit from treatment, significantly reducing the time and cost typically associated with traditional drug development. “The clearance of this IND demonstrates the power of our efficient, data-driven drug development model in identifying promising therapeutic opportunities in areas of high unmet medical need,” said Panna Sharma, President and CEO of Lantern Pharma. “Through Starlight Therapeutics, we are positioned to dedicate focused resources and expertise to advancing treatments for some of the most challenging cancers affecting the brain and central nervous system in both adults and children, while developing meaningful therapies that can reach patients in an accelerated timeline compared to traditional drug development processes.” Market Opportunity and Next Steps With GBM representing an annual market opportunity of $3 to $5 billion globally and case numbers accelerating worldwide, there is an urgent need for innovative treatment approaches. The current standard of care has remained largely unchanged for over 15 years, highlighting the critical importance of novel therapeutic strategies like the STAR-001 combination. Starlight is targeting commencement of the Phase Ib/2a trial in late 2025 or early 2026, subject to additional funding, and plans to provide regular updates on trial preparation, patient enrollment progress, and preliminary data as they become available. About Glioblastoma Multiforme (GBM) Glioblastoma multiforme is the most common and aggressive form of primary brain tumor in adults, accounting for approximately 15% of all brain tumors. Despite aggressive multimodal treatment including surgery, radiation, and chemotherapy, the prognosis remains extremely poor, with a median survival of only 12 months and a five-year survival rate of less than 5%. The blood-brain barrier presents a significant challenge in treating GBM, as most therapeutic agents cannot effectively penetrate brain tissue. About Starlight Therapeutics Starlight Therapeutics is a wholly owned subsidiary of Lantern Pharma Corp., specifically focused on the clinical development of innovative treatments for central nervous system oncology indications. The company leverages Lantern Pharma’s proprietary AI drug development platform to identify and advance promising therapeutic combinations for adult and pediatric patients with brain and CNS cancers. About Lantern Pharma Lantern Pharma (NASDAQ: LTRN) is an AI company transforming the cost, time, and failure rate of oncology drug discovery and development. Lantern’s proprietary AI platform RADR® uses machine learning and multiomic data to solve complex, billion-dollar drug development challenges. By harnessing the power of AI and with input from world-class scientific advisors and collaborators, Lantern has built a pipeline of promising, clinical stage drug candidates, including molecules that target novel cancer mechanisms and targets. SOURCE: Lantern Pharma

孤儿药快速通道临床申请

100 项与 Irofulven-2 相关的药物交易

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适应症	最高研发状态	国家/地区	公司	日期
晚期恶性实体瘤	临床2期	美国	Lantern Pharma, Inc.	2023-06-09
多形性胶质母细胞瘤	临床2期	美国	Lantern Pharma, Inc.	2023-06-09
转移性实体瘤	临床2期	美国	Lantern Pharma, Inc.	2023-06-09
非小细胞肺癌	临床2期	美国	Lantern Pharma, Inc.	2023-06-09
胰腺腺癌	临床2期	美国	Lantern Pharma, Inc.	2023-06-09
三阴性乳腺癌	临床2期	美国	Lantern Pharma, Inc.	2023-06-09
KEAP1突变非小细胞肺癌	临床申请批准	美国	Lantern Pharma, Inc.	2025-05-12
STK11突变非小细胞肺癌	临床申请批准	美国	Lantern Pharma, Inc.	2025-05-12
中枢神经系统横纹肌瘤	临床前	美国	Lantern Pharma, Inc.	2022-01-24
膀胱癌	临床前	美国	Lantern Pharma, Inc.	2021-07-29

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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


NCT05933265 (Company_Website) 人工标引	临床1期	晚期恶性实体瘤 KEAP1 Mutation \| CHEK2 Mutation \| BRCA1 Mutation \| ... 更多	63	LP-184	鑰窪襯獵觸選遞獵積製(鹹鏇廠顧壓廠鹹獵膚範) = LP-184 exhibited a robust safety profile, with no dose-limiting toxicities in the majority of cohorts and low incidence of discontinuations, interruptions, or delays due to drug-related adverse events. 築構鏇顧築衊簾簾選廠 (鹹積壓構夢製觸艱壓醖 ) 更多	积极	2025-09-16
				LP-184 (dose levels ≥8 (≥0.25 mg/kg))
AACR2025	临床1期	肿瘤 PTGR1 \| BRCA1/2 \| ATM/ATR ... 更多	-	LP-184	夢築觸網醖襯鏇淵繭壓(簾獵鹹鹹積獵繭鏇窪範) = 夢衊廠鹹鏇膚艱構築簾顧襯遞憲積艱顧獵蓋鑰 (鏇鏇構願顧繭獵製遞範 )	-	2025-04-28
SNO2023	临床1期	多形性胶质母细胞瘤 MGMT-methylated \| MGMT-unmethylated	-	LP-184	築餘餘齋鬱夢範壓壓廠(築餘廠製願艱顧簾窪鏇) = 觸顧鬱鑰選憲網繭膚積顧壓觸鏇簾鬱廠積構鏇 (衊鹹網蓋鏇壓壓餘膚簾 )	积极	2023-11-10
				Spironolactone	淵鏇淵鑰網醖齋願醖鹹(選廠艱鑰選鏇鏇獵繭衊) = 糧構簾壓築襯艱窪窪窪蓋膚糧鹹構淵餘遞獵廠 (醖鬱鬱廠範糧鏇積構築 )