A Phase 1/1b Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of PAS-004, a MAPK/ERK Kinase 1/2 (MEK 1/2) Inhibitor, in Adult Participants With Neurofibromatosis Type 1 (NF1) With Symptomatic and Inoperable, Incompletely Resected, or Recurrent Plexiform Neurofibromas

The main purpose of this clinical trial is to test PAS-004 in people with at least one symptomatic plexiform neurofibroma due to Neurofibromatosis Type 1 (NF1). The main questions it aims to answer are:
* How well participants are able tolerate different doses of PAS-004, and
* What side effects PAS-004 might have.
This study will have two parts, Part A and Part B. The main goal of Part A of this study is to learn more about how participants tolerate different doses of PAS-004, and what side effects PAS-004 might have. What we learn from Part A of the study will help decide what doses of the study drug (PAS-004) should be used in Part B of the study, and if it is safe.
In Part B, two different doses from Part A will be tested. The main goal of this part of the study is to keep studying any side effects of PAS-004 at those two dose levels, and to learn more about if the doses picked for this part of the study might have an effect on plexiform neurofibromas.
Participants in Part A of the study who were taking doses selected for Part B may be able to continue on to Part B and keep taking the same dose of PAS-004 for 6 more months.
Study participants in both parts will have regular visits to the study doctor and be asked to have tests and exams done to check on their health and safety, including blood draws and MRIs. Everyone participating in the study will take PAS-004 by mouth once a day during the study, in 28-day cycles. Participants will be asked to keep a diary to record their daily dose of study drug.
Participants will continue on daily PAS-004 for up to 6 months, or until:
* They decide to withdraw from the study, or
* They experience unacceptable side effects, or
* Their disease progresses, or another illness interferes with taking the study drug, or
* The sponsor selects a dose level to study further in the next part of the study, or
* The sponsor stops the study.

开始日期2025-05-30

申办/合作机构

Pasithea Therapeutics Corp.

[+1]

NCT06299839

/ Recruiting临床1期

A Phase 1 Open-label Study to Assess the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of PAS-004, a MEK (1/2) Inhibitor, in Patients With MAPK Pathway-driven Advanced Solid Tumors With a Documented RAS, NF1, or RAF Mutation or Patients Who Have Failed BRAF/MEK Inhibition

The main purpose of this clinical trial is to test PAS-004 in people with advanced solid tumors with rat sarcoma virus (RAS), neurofibromatosis type I (NF1), or rapidly accelerated fibrosarcoma (RAF) mutations. The main questions it aims to answer are:
* How well participants are able tolerate different doses of PAS-004, and
* What side effects PAS-004 might have.
Study participants will have regular visits to the study doctor and be asked to have tests and exams done to check on their health and safety. Everyone participating in the study will take PAS-004 by mouth as a single dose, followed by one week observation, then once a day during the study, in 28-day cycles. Participants will continue on daily PAS-004 for up to 2 years, or until:
* They decide to withdraw from the study, or
* They experience unacceptable side effects, or
* Their disease progresses, or another illness interferes with taking the study drug, or
* The sponsors stops the study.

开始日期2024-02-29

申办/合作机构

Pasithea Therapeutics Corp.

100 项与 PAS-004 相关的临床结果

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100 项与 PAS-004 相关的转化医学

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100 项与 PAS-004 相关的专利（医药）

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项与 PAS-004 相关的文献（医药）

2026-02-01·JOURNAL OF ETHNOPHARMACOLOGY

Inhibition of melanogenesis by ginseng phenolic Acids: Suppressed melanosome maturation, melanocyte transport, and keratinocyte uptake

Article

作者: Xu, Xiaohao ; Sun, Liwei ; Liu, Jianzeng ; Guo, Yonggang ; Sun, Guang ; Jiang, Rui ; Wang, Jing ; Li, Zhenzhuo ; Zhai, Lu

ETHNOPHARMACOLOGICAL RELEVANCE:

Ginseng phenolic acid extract (GPAE), composed of five phenolic acids (protocatechuic acid, PA; vanillic acid,VA; p-coumaric acid, p-CA; salicylic acid, SA; and caffeic acid, CA), suppresses UVB-induced melanin overproduction. However, UV-induced skin darkening involves broader melanin metabolism, including melanosome maturation, transport to dendrites, and transfer to keratinocytes. GPAE's overall effects on these processes and its key active components remain unclear.

AIM OF THE STUDY:

To identify the effects and mechanisms of GPAE and its phenolic acids on melanosome transfer, providing a mechanistic explanation for the antipigmentation properties of ginseng root.

METHODS:

The effects of GPAE or its phenolic acids were tested in UVB-irradiated mice and B16F10 and HaCaT cells. Skin and cell samples were analyzed using various staining, microscopy, and molecular techniques to assess structure, melanin deposition, and protein expression.

RESULTS:

GPAE suppressed UVB-induced skin hyperpigmentation in mice by lightning skin, thinning the epidermis, and decreasing basal-layer pigmentation. GPAE downregulated melanosome transport proteins Myosin Va (Myo Va), melanophilin (Mlph), Rab27a, and the melanophage-associated receptor PAR2. In UVB-exposed B16F10 cells, GPAE reduced melanin content and accumulation, shortened dendrite length, inhibited dendritic proliferation, and disrupted melanosome maturation by decreasing stage III-IV melanosomes and increasing stage I-II populations. A composite mixture, PAs(4) (PA/VA/p-CA/SA), formulated at GPAE-equivalent ratios, similarly downregulated Mlph, Rab27a, and Myo Va expression. Another blend, PAs(3) (PA/VA/SA), replicated GPAE's inhibition of HaCaT cell-mediated phagocytosis. Both GPAE and PAs(3) inhibited UVB-induced PAR2 upregulation, intracellular Ca²⁺ levels, and PI3K, AKT, JNK, ERK, and p38 phospherylation.

CONCLUSION:

GPAE alleviates UVB-induced hyperpigmentation by inhibiting melanosome maturation and dendritic transport in melanocytes and reducing keratinocyte melanosome uptake via the PAR2-Ca²⁺-PI3K-AKT-MAPK pathway. Key active phenolic acids were identified, providing mechanistic insight into ginseng's antimelanogenic effects.

项与 PAS-004 相关的新闻（医药）

2026-01-13

·ir.pasithea.com

Pasithea Therapeutics Provides Outlook on PAS-004 Clinical Programs and Data Release Timelines

MIAMI, Jan. 13, 2026 (GLOBE NEWSWIRE) -- Pasithea Therapeutics Corp. (NASDAQ: KTTA) (“Pasithea” or the “Company”), a clinical-stage biotechnology company developing PAS-004, a next-generation oral macrocyclic MEK inhibitor for the treatment of neurofibromatosis type 1 associated plexiform neurofibromas (NF1-PN), today provided updated timelines on its ongoing clinical trials in advanced cancer and adult NF1-PN patients. Ongoing Phase 1/1b clinical trial in adult patients with NF1-PN (NCT06961565): Pasithea has completed enrollment of 12 patients through the first 4 dose cohorts (4, 8, 12 and 18 mg tablets) in Part A of the study. The Company plans to present data in the second half of 2026, including available efficacy data through the six-month timepoint for both plexiform and cutaneous neurofibromas. The planned data release is also expected to include safety, tolerability and pharmacokinetic (PK) data. Ongoing Phase 1 clinical trial in advanced cancer patients (NCT06299839): Pasithea expects to present longer-term follow-up data from patients in Cohort 4 (15mg capsule) through Cohort 8 (45mg capsule) in the second quarter of 2026. “2025 was a pivotal year for Pasithea, highlighted by early evidence of a differentiated safety and tolerability profile and initial signals of clinical activity in our first-in-human dose escalation advanced cancer study of PAS-004, our potentially best-in-class macrocyclic MEK inhibitor,” said Dr. Tiago Reis Marques, Chief Executive Officer of Pasithea. “In November 2025, we announced encouraging results in patients previously treated with a MEK inhibitor, including a partial response and an initial disease control rate of 71.4% among efficacy evaluable patients with BRAF-mutated tumors and met our planned milestone of providing initial NF1-relevant data through the presentation of pharmacokinetic results in the first two cohorts of our NF1 study. We believe these findings from our advanced cancer study support the development of PAS-004 for the treatment of NF1-PN patients. Additionally, in December 2025, we successfully raised $60 million in gross proceeds through a public offering, enabling us to advance PAS-004 through several key milestones and support operations through at least the first half of 2028. We remain steadfast in our mission to deliver safe, tolerable and effective therapies to patients with significant unmet need, especially in indications requiring chronic dosing.” About Pasithea Therapeutics Corp. Pasithea is a clinical-stage biotechnology company primarily focused on the research and development of its lead drug candidate, PAS-004, a next-generation macrocyclic MEK inhibitor intended for the treatment of RASopathies, MAPK pathway-driven tumors, and other diseases. The Company is currently testing PAS-004 in a Phase 1 clinical trial in advanced cancer patients (NCT06299839), and a Phase 1/1b clinical trial in adult patients with neurofibromatosis type 1 (NF1)-associated plexiform neurofibromas (NCT06961565). Forward Looking Statements This press release contains statements that constitute “forward-looking statements” made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include statements regarding the Company’s ongoing Phase 1 clinical trial of PAS-004 in advanced cancer patients, the Company’s ongoing Phase 1/1b clinical trial of PAS-004 in adult NF1 patients, and the safety, tolerability, pharmacokinetic (PK), pharmacodynamics (PD) and preliminary efficacy of PAS-004, as well as all other statements, other than statements of historical fact, regarding the Company’s current views and assumptions with respect to future events regarding its business, as well as other statements with respect to the Company’s plans, assumptions, expectations, beliefs and objectives, the success of the Company’s current and future business strategies, product development, pre-clinical studies, clinical studies, clinical and regulatory timelines, market opportunity, competitive position, business strategies, potential growth and financing opportunities and other statements that are predictive in nature. Forward-looking statements are subject to numerous conditions, many of which are beyond the control of the Company. While the Company believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to the Company on the date of this release. These forward-looking statements are based upon current estimates and assumptions and are subject to various risks and uncertainties, including risks that future clinical trial results may not match results observed to date, may be negative or ambiguous, or may not reach the level of statistical significance required for regulatory approval, as well as other factors set forth in the Company’s most recent Annual Report on Form 10-K, Quarterly Report on Form 10-Q and other filings made with the U.S. Securities and Exchange Commission (SEC). Thus, actual results could be materially different. The Company undertakes no obligation to update these statements whether as a result of new information, future events or otherwise, after the date of this release, except as required by law. Pasithea Therapeutics Contact Patrick Gaynes Corporate Communications pgaynes@pasithea.com Released January 13, 2026

临床1期临床结果

2025-12-26

·博药

MEK抑制剂：破解RAS通路困局

在肿瘤靶向治疗领域，RAS-RAF-MEK-ERK通路（MAPK/ERK通路）的异常激活是驱动肿瘤发生发展的核心机制之一，尤其在RAS、BRAF等基因高频突变的癌症中，该通路堪称“致癌引擎”。而丝裂原活化的细胞外信号调节激酶（MEK）作为这条通路的核心信号枢纽，其抑制剂的研发与应用，成为破解RAS通路“不可成药”困局、拓展精准治疗边界的关键方向，为肿瘤治疗带来了突破性希望。市场：MEK抑制剂规模迅速增长 MAPK通路是细胞增殖、应激、炎症、分化、功能同步化、转化、凋亡等信号转导通路的共同交汇通路之一。该通路以三级激酶级联为特征，依次为MAPK激酶的激酶（MEKK或MKKK）、MAPK激酶（MEK或MKK）以及MAPK，三者通过顺序磷酸化传递并放大信号。根据结构和功能差异，MAPK家族主要分为ERK、JNK、p38及ERK5四个核心分支，分别响应不同胞外刺激，并在细胞功能调控中发挥关键作用。 RAS-RAF-MEK-ERK通路的异常活化与癌症发生密切相关，约三分之一的实体瘤存在RAS突变，约8%存在BRAF突变，导致信号持续激活，驱动肿瘤恶性进展。基于BRAF突变在黑色素瘤中的高频性，RAF抑制剂成为最早获批的ERK通路靶向药物，但其面临的快速耐药（如CRAF过表达）与“矛盾激活”等难题，揭示了单一节点抑制的不足。因此，靶向MEK成为克服上游耐药的关键，而“RAF+MEK”垂直联合抑制策略通过同时、深度抑制通路上的两个关键节点，不仅显著提升了通路抑制的广度与持久性，确立了BRAF突变黑色素瘤的一线治疗标准，更从机制上验证了针对汇聚节点进行多靶点协同阻断是克服旁路激活与反馈耐药的根本策略。 MEK是一种双特异性激酶，能同时磷酸化底物蛋白的丝氨酸/苏氨酸及酪氨酸残基，是ERK信号通路中的核心信号枢纽。其主要功能是整合并放大上游生长因子与细胞因子等信号，并通过特异性磷酸化ERK1/2的苏氨酸-202/185和酪氨酸-204/187位点（T202/185和Y204/187），激活下游ERK1/2。活化的ERK1/2进而调控转录因子等多种靶蛋白的磷酸化状态，参与细胞增殖、分化、存活与代谢等重要生命过程的调节[1]。 MEK存在两种高度保守的亚型：MEK1（44 kDa）与MEK2（45 kDa），二者在激酶结构域具有约80%的氨基酸同源性，进化上高度保守，但部分功能存在差异且不可互相替代。在多种肿瘤中，RAS或RAF等上游基因的突变可导致MEK/ERK通路持续异常激活，进而驱动肿瘤失控性生长。因此，靶向MEK的抑制剂已成为治疗BRAF突变型黑色素瘤、1型神经纤维瘤病等相关癌症及罕见病的重要策略。图1 四种MAPK信号转导途径数据来源：文献研究[1]，药智咨询整理图2 人源MEK1和MEK2的基因结构图数据来源：文献研究[1]，药智咨询整理根据Market Research Future分析，全球MEK抑制剂市场预计将从2025年的23.0亿美元增长至2035年的46.5亿美元，其间的年复合增长率（CAGR）约为7.26%。驱动因素主要包括：针对I型神经纤维瘤病（NF1）等罕见病的新适应症持续获批并填补治疗空白；MEK抑制剂与BRAF抑制剂等药物在黑色素瘤、结直肠癌等更多实体瘤中的联合应用不断拓展与深入；此外，中国市场的重要性日益凸显，本土制药企业自主研发的新药成功上市，显著提升了药物的可及性并扩大了市场覆盖范围。图3 全球MEK抑制剂市场规模（亿美元）数据来源：Market Research Future 格局：已上市MEK抑制剂与在研管线据药智数据，截至2025年12月23日，全球共有9款已上市的MEK抑制剂，均为小分子靶向药物。其中，2013年首款药物曲美替尼获批用于BRAF突变黑色素瘤以来，早期药物（如卡比替尼、贝美替尼）主要针对黑色素瘤、非小细胞肺癌等实体瘤；而近年来，研发焦点却已转向精准满足1型神经纤维瘤病及其相关丛状神经纤维瘤（如司美替尼、芦沃美替尼、莫达美替尼）以及低级别浆液性卵巢癌（如阿维替尼联合疗法）等由MAPK通路驱动、既往缺乏有效治疗的罕见病及肿瘤疾病领域。芦沃美替尼作为中国首个且目前唯一获批用于3岁及以上1型神经纤维瘤病相关丛状神经纤维瘤和朗格汉斯细胞组织细胞增生症成人患者的MEK抑制剂，其研发实现了从“快速跟进”到“全球首发”的跨越。为进一步拓展在儿童罕见肿瘤领域的应用，该药已于2025年10月被纳入国家药监局“儿童抗肿瘤药物研发鼓励试点计划（星光计划）”，旨在通过专项审评指导，加速其儿童朗格汉斯细胞组织细胞增生症和低级别脑胶质瘤两项新适应症的研发进程。表1 全球已上市MEK抑制剂数据来源：药智数据（如有错误请指正，点击查看大图）从在研药物看，目前全球处于临床在研阶段的MEK抑制剂靶向药物约有11种。其研发趋势显示出两大核心方向：一是针对现有优势领域的深度拓展，例如艾沃美替尼正推进在浆液性卵巢癌的III期研究，以及TQ-B3234等在1型神经纤维瘤病方向的持续探索；二是对药物作用机制的广度突破，以NST-628等药物为代表，通过设计为MEK/RAF双靶点或泛RAF抑制剂，旨在克服现有MEK抑制剂的耐药性问题。从企业格局看，以辉瑞、中外制药为代表的跨国药企与以正大天晴、璧辰医药为代表的中国生物科技公司正在这一领域并行推进。表2 全球临床在研MEK抑制剂数据来源：药智数据（如有错误请指正，点击查看大图）瓶颈：耐药与疗效局限的核心挑战在罕见病领域，MEK抑制剂（如司美替尼、芦沃美替尼）首次为既往无药可医的1型神经纤维瘤病等MAPK通路驱动疾病提供了靶向疗法，显著提升了治疗可及性；在实体瘤领域，它与BRAF抑制剂的联合方案已成为BRAF突变型黑色素瘤、非小细胞肺癌等癌症的标准治疗。但其在更广泛的RAS或RAF突变实体瘤中疗效有限。主要的临床问题有两个方面：复杂的耐药机制：肿瘤极易通过多种方式逃逸药物抑制。 MAPK通路再激活：研究表明，超过一半对RAF抑制剂耐药的黑色素瘤患者，其肿瘤中出现了新的MAPK通路基因（如MAP2K2）变异，导致通路重新激活[2]。新兴耐药突变：例如，有病例报告发现，肺癌患者在接受EGFR/BRAF/MEK三联疗法后，产生了新的MAP2K1 K57N突变，从而导致治疗失败[3]。替代信号通路激活：在某些难治性BRAF突变癌症中，肿瘤会通过激活MAPK通路之外的其他途径（如PI3K-PDK1-AKT）来产生固有耐药[4]。狭窄的适应症谱与毒性：传统MEK抑制剂单药在常见实体瘤（如胰腺癌、结直肠癌）中疗效不佳，且其治疗指数（疗效与毒性的平衡）较低，限制了临床应用。现有ERK信号通路靶向治疗面临的核心困境是耐药性，而MEK作为该通路的关键枢纽，是多种耐药机制汇聚的核心节点。因此，MEK抑制剂领域的发展正超越传统单一靶点抑制模式，沿着源头创新的新一代药物、协同增效的多通路联合疗法和持续扩大的适应症范围三个维度演进，旨在为更广泛的癌症患者群体提供更持久、更深度的临床获益。新一代药物：从源头设计克服耐药。新一代MEK靶向药物的核心策略是从作用机制源头设计，以规避或阻断经典的耐药路径。如“MEK/RAF分子胶”IK-595，它能将MEK与所有RAF亚型（ARAF、BRAF、CRAF）锁定在非活性状态，从结构上根本性阻止了由CRAF二聚化介导的再激活这一常见耐药机制，其约24小时的长半衰期也支持更持久的通路抑制；PAS-004的其早期临床数据表明，对既往经BRAF/MEK抑制剂治疗失败的耐药患者仍能诱导肿瘤缩小，初步证实了其克服获得性耐药的潜力。创新联合疗法：协同阻断多重致癌依赖。创新联合疗法的逻辑在于通过协同阻断肿瘤生存依赖的多个关键通路，以克服其异质性和旁路激活。例如，Avutometinib与FAK抑制剂Defactinib的联用，已获批用于卵巢癌，旨在同时打击肿瘤生长和微环境。BEAVER临床试验也证实，将BRAF/MEK抑制剂与CDK4/6抑制剂或SHP2抑制剂联用，能有效逆转特定BRAF突变肿瘤的固有耐药[5]。适应症持续拓展：扩大临床受益边界。目前正向低级别脑胶质瘤、儿童罕见肿瘤、浆液性卵巢癌等领域延伸，尤其针对儿童患者的研发（如芦沃美替尼纳入“星光计划”），将进一步扩大临床获益人群。展望：精准化与突破型研发并行 MEK作为MAPK/ERK信号通路的核心枢纽，是破解RAS通路“不可成药”困境的关键靶点。其双特异性激酶特性与在肿瘤信号传导中的核心作用，使其抑制剂成为BRAF突变黑色素瘤、1型神经纤维瘤病等疾病的重要治疗手段，既填补了传统治疗的空白，也为精准医疗提供了有力支撑。两种高度保守且功能互补的MEK亚型，更赋予了靶向药物研发的多元潜力。目前对于MEK抑制剂的探索取得了诸多研究成果，但单药疗效有限及耐药性等问题仍是关键挑战。展望未来，该领域将围绕“精准化”与“突破型”两大方向持续演进：一方面，适应症向更多罕见病与难治性实体瘤拓展，扩大临床获益人群；另一方面，通过多靶点联合、作用机制创新等策略，系统提升药物疗效与耐受性。随着对ERK通路作用机制的理解不断深入，以及候选化合物的持续优化，下一代更具潜力的MEK抑制剂有望加速涌现，在肿瘤治疗领域开拓更广阔的应用前景，为患者带来突破性治疗新选择。参考文献 1.郝佩琪,安输,杨洋,刘莹,郭晓汐,徐天瑞.MEK激酶及其抑制剂的研究进展[J].中国细胞生物学学报,2015,37(10):1425-1431. 2.Van Allen EM, Wagle N, Sucker A, et al. The genetic landscape of clinical resistance to RAF inhibition in metastatic melanoma. Cancer Discov. 2014;4(1):94-109. 3.Tan X, Wu Z, Chen M. MAP2K1 K57N Conferred an Acquired Resistance to Furmonertinib, Dabrafenib and Trametinib Combined Therapy in Advanced Lung Adenocarcinoma with EGFR Mutation and BRAF V600E. Onco Targets Ther. 2024;17:307-312. Published 2024 Apr 9. 4.蒯梦妮,乐祥阳,李乾斌,胡高云.MEK抑制剂的研究进展[J].肿瘤药学,2017,7(5):513-522. 5.Rose AAN, Maxwell J, Rousselle E, Riaud M, Elkholi IE, Mukonoweshuro C, et al. CDK4/6 and SHP2 mediate BRAF/MEK inhibitor resistance in Class 2 and 3 BRAF mutant cancers. medRxiv. 2024 Jul 28. 博腾股份成立于2005年，主要为全球药企、生物科技公司、科研机构等提供从临床前研究到药品上市全生命周期所需的小分子药物、多肽与寡核苷酸药物、蛋白与偶联药物以及细胞与基因治疗药物等一站式服务解决方案，研发、生产、运营场地覆盖中国（重庆、上海、四川、江苏、江西、湖北）、美国、斯洛文尼亚、比利时、瑞士和丹麦等地。我们始终坚持以客户为中心，致力于为客户提供创新、可靠的全球化、端到端CDMO服务，让好药更早惠及大众。免责声明：本文仅作者个人观点的表达，文中观点不代表博腾及其所属子公司立场，亦不对本文所提供信息做任何形式的保证。同时，本文不做治疗方案推荐，也不承担因使用或依赖本文信息所产生的任何直接或间接的后果。如需获得治疗方案指导，请前往正规医院就诊。

临床2期

2025-12-04

·PRNewswire

ALS Association Commits $2 Million to Support Early-Stage Clinical Trials of Two Emerging ALS Therapies

This funding was awarded through the ALS Association's Hoffman ALS Clinical Trial Awards Program, which aims to accelerate the development of new ALS treatments ARLINGTON, Va., Dec. 4, 2025 /PRNewswire/ -- The ALS Association is pleased to announce the recipients of its 2025 Hoffman ALS Clinical Trial Awards. These research grants, worth up to $1 million each, support early-phase trials that generate important data about things like safety, dosing, and biomarkers to help speed up the availability of effective new ALS treatments. "Funding for early-stage clinical testing acts like 'ignition fuel' for the R&D engine," said Kuldip Dave, Ph.D., senior vice president of research at the ALS Association. "The knowledge gained from these initial trials helps propel drug development forward by providing critical data that can be used to inform larger efficacy studies. It also helps make these next steps less risky and more attractive to potential investors and partners." This year's awardees were selected from a competitive group of applicants from around the world. Their applications demonstrated a compelling biological rationale for the investigational therapy, robust preclinical data, an integrated biomarkers program, and a clear plan for future clinical development. 2025 Awardees Mayo Clinic Jacksonville – Consultant Jaimin Shah, M.D., will conduct a phase 1 trial of an investigational therapy called PAS-004 in partnership with Pasithea Therapeutics Corp. PAS-004 targets an enzyme called MEK, which is involved in the inflammation, nerve damage, and nerve loss seen in ALS. The trial will recruit 12 people living with ALS to evaluate the safety and tolerability of PAS-004 and determine the best dosage to test in future studies. Biomarkers will also be measured to identify any early signs of potential disease-modifying effects. "While early phase studies cannot really determine whether a drug significantly slows or stops ALS, they are critical to understanding many important questions to determine a drug's potential to become an effective therapy," Dr. Shah said. "We hope this trial will improve the field's understanding of the role MEK inhibitors could play in the treatment of ALS." Molefy Pharma SL – Co-founder and Scientific Director Ana Martίnez, Ph.D., will lead phase 1 clinical testing of an investigational therapy known as AP-2. AP-2 has shown promise in preclinical studies, helping to restore the balance of an important protein called TDP-43. In approximately 97% of people with ALS, TDP-43 imbalances lead to the buildup of abnormal, disease-causing clumps or aggregates. Results from this trial will help determine the highest safe dose of AP-2, its therapeutic window, and any possible side effects. "This first-in-human trial represents a crucial initial step toward testing in ALS patients and paves the way for the clinical development of a new TDP-43–modifying drug," Dr. Martίnez said. "We will work for the success of this project with all our strength and energy." Clinical trials are the most reliable—and ultimately the fastest—way to translate promising laboratory science into better ways to treat ALS. Through its Hoffman ALS Clinical Trial Awards, the ALS Association is paving the way for more, better, faster ALS clinical trials. "People living with ALS urgently need new treatments," said Dr. Dave. "By the funding early-stage clinical trials like these, we are working to accelerate the development of promising therapeutic candidates so we can make ALS livable until we can cure it." About the ALS Association The ALS Association is the largest ALS organization in the world. The ALS Association funds global research collaborations, assists people with ALS and their families through its nationwide network of care and certified clinical care centers, and advocates for better public policies for people with ALS. The ALS Association is working to make ALS a livable disease while urgently searching for new treatments and a cure. For more information about the ALS Association, visit our website at . About ALS Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord. Over the course of the disease, people lose the ability to move, to speak, and eventually, to breathe. The disease is always fatal, usually within five years of diagnosis. Few treatment options exist, resulting in a high unmet need for new therapies to address functional deficits and disease progression. About the Hoffman ALS Clinical Trial Awards Program Hoffman ALS Clinical Trial Awards support early- to mid-stage (phase 1 or 2a) biomarker-driven clinical trials of novel or repurposed therapeutics for ALS (either disease-modifying or symptomatic). These annual awards are made possible through the historic $58 million gift from the late Hugh Hoffman, a Cincinnati native and philanthropist whose life was shaped by the loss of his father, Herbert, to ALS. Mr. Hoffman's contribution, the largest single philanthropic gift ever made to an ALS organization, established the Hugh and Herbert Hoffman ALS Fund, which is dedicated to advancing new treatments, strengthening clinic infrastructure, and reduce barriers to accessing a multidisciplinary care model. SOURCE The ALS Association 21% more press release views with Request a Demo

临床1期

100 项与 PAS-004 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
神经纤维瘤病1型	临床1期	美国	Pasithea Therapeutics Corp.	2025-05-30
神经纤维瘤病1型	临床1期	澳大利亚	Pasithea Therapeutics Corp.	2025-05-30
神经纤维瘤病1型	临床1期	韩国	Pasithea Therapeutics Corp.	2025-05-30
丛状神经纤维瘤	临床1期	美国	Pasithea Therapeutics Corp.	2025-05-30
丛状神经纤维瘤	临床1期	澳大利亚	Pasithea Therapeutics Corp.	2025-05-30
丛状神经纤维瘤	临床1期	韩国	Pasithea Therapeutics Corp.	2025-05-30
肥大细胞肉瘤	临床1期	美国	Pasithea Therapeutics Corp.	2024-11-22
肥大细胞肉瘤	临床1期	保加利亚	Pasithea Therapeutics Corp.	2024-11-22
肥大细胞肉瘤	临床1期	罗马尼亚	Pasithea Therapeutics Corp.	2024-11-22
晚期恶性实体瘤	临床1期	美国	Pasithea Therapeutics Corp.	2024-02-29

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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


NCT06299839 (Company_Website) 人工标引	临床1期	晚期癌症	27	PAS-004	願襯築齋鬱製簾顧觸積(鏇願築製壓觸觸網鏇艱) = 憲繭鏇觸構糧鏇鬱觸網蓋襯憲鹹齋蓋繭網觸獵 (鏇獵餘製糧簾鹹襯壓淵 ) 更多	积极	2025-11-20
NCT06299839 (Phase I dose-escalation study of PAS-004, ASCO2025)	临床1期	MAPK突变实体瘤 RAS \| NF1 \| RAF mutation ... 更多	9	PAS-004 2mg	餘廠廠願窪鹽夢積網築(築遞淵選遞顧鹽醖鑰積) = 繭選餘糧齋鬱艱觸窪糧蓋鹹衊鹹襯憲餘淵選壓 (餘壓廠繭顧選壓襯醖壓 ) 更多	积极	2025-05-30
NCT06299839 (Company_Website) 人工标引	临床1期	晚期恶性实体瘤	-	PAS-004 (8mg and 15mg capsules)	構壓遞築窪艱鏇醖醖築(鹽鹽網鏇簾衊夢積製蓋) = 積鹹壓積壓壓遞獵範簾壓憲繭廠顧艱窪選夢製 (築餘構築獵餘衊願淵壓 ) 更多	积极	2025-05-06
NCT06299839 (Company_Website) 人工标引	临床1期	晚期癌症	19	PAS-004 capsule (15 patients) or tablet (four patients)	糧蓋顧壓窪築壓製範鏇(鬱範觸窪憲選鹽憲蓋簾) = None 艱壓廠選簾餘夢齋醖製 (壓觸築觸製積製鹽憲觸 )	积极	2025-04-10
NCT06299839 (Company_Website) 人工标引	临床1期	晚期癌症	6	PAS-004	衊繭製鏇鏇餘遞觸鬱鏇(餘願糧淵鏇衊鬱醖鏇鑰) = none 蓋窪鏇膚觸憲窪選簾衊 (衊範積齋鹽壓壓襯網糧 ) 更多	积极	2024-09-26