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更新于：2025-05-07

TRPV3

更新于：2025-05-07

基本信息

别名

Transient receptor potential cation channel subfamily V member 3、transient receptor potential cation channel, subfamily V, member 3、TrpV3

+ [3]

简介

Non-selective calcium permeant cation channel (PubMed:12077604, PubMed:12077606, PubMed:26818531, PubMed:37648856, PubMed:38691614). It is activated by innocuous (warm) temperatures and shows an increased response at noxious temperatures greater than 39 degrees Celsius (PubMed:12077604, PubMed:12077606). Activation exhibits an outward rectification (PubMed:12077604). The channel pore can dilate to provide permeability to larger cations (PubMed:37648856). May associate with TRPV1 and may modulate its activity (PubMed:12077606). Is a negative regulator of hair growth and cycling: TRPV3-coupled signaling suppresses keratinocyte proliferation in hair follicles and induces apoptosis and premature hair follicle regression (catagen) (PubMed:21593771).

关联

项与 TRPV3 相关的药物

KM-001(Kamari-Pharma)

靶点

TRPV3

作用机制

TRPV3拮抗剂

在研机构

Kamari Pharma Ltd.

原研机构

Kamari Pharma Ltd.

在研适应症

丘疹性掌跖角化病 [+3]

非在研适应症-

最高研发阶段临床1期

首次获批国家/地区-

首次获批日期1800-01-20

KM-023 (Kamari)

靶点

TRPV3

作用机制

TRPV3拮抗剂

在研机构

Kamari Pharma Ltd.

原研机构

Kamari Pharma Ltd.

在研适应症

鱼鳞病

非在研适应症-

最高研发阶段临床前

首次获批国家/地区-

首次获批日期1800-01-20

Trpvicin

靶点

TRPV3

作用机制

TRPV3拮抗剂

在研机构

中国医学科学院 [+2]

原研机构

北京大学 [+2]

在研适应症

脱发 [+1]

非在研适应症-

最高研发阶段临床前

首次获批国家/地区-

首次获批日期1800-01-20

项与 TRPV3 相关的临床试验

NCT05956314

/ Completed临床1期

Phase 1b, Open Label Study to Evaluate the Safety, Tolerability, and Efficacy of a 1% Topical Formulation of KM-001 for the Treatment of Type I Punctate Palmoplantar Keratoderma or Pachyonychia Congenita

This Phase 1b, open-label, single-center, prospective trial will be assessing the safety, tolerability, and efficacy of topical KM-001 1% in patients with PPPK1 or PC diseases. In this study 2 cohorts will be recruited:
1. Cohort 1: up to 11 eligible patients, will be enrolled to be treated twice daily, for 12 weeks, with 1% topical KM-001, on the plantar surfaces (2 feet).
2. Cohort 2: up to 8 eligible patients, will be enrolled to be treated twice daily, for 16 weeks, with 1% topical KM-001, on the plantar surfaces (2 feet).
Safety (AEs, blood work [at specific visits], vital signs), tolerability, and efficacy parameters (overall lesion improvement) will be assessed during in-clinic visits (Cohort 1: during Screening, Enrolment, and on Days 7, 28, 42, 63, 84 [end of treatment, EoT], 112 [End of Study, EoS] post first investigational medicinal product (IMP) administration; Cohort 2: during Screening, Enrolment, and on Days 7, 28, 42, 63, 84, 112 [EoT], 140 [EoS] post first investigational medicinal product (IMP) administration).
PK samples will be collected to assess plasma levels of KM-001 on
* Screening (Day -14 to -0): any time during the visit. (or on Day 1 up to 30 minutes pre-dose if missed during Screening)
* Day 7 and at EoT (Cohort 1: Day 84; Cohort 2: Day 112) up to 30 minutes pre-dose, and at 1 h, 2 h, 3 h, 6 h (+15 min) post-dose
* Days 28, 42 for both Cohorts, and Day 84 for Cohort 2: 1 sample after the first dose, before the second dose, as late as possible in the visit.
* End of Study (EoS, Day 112 (Cohort 1) or Day 140 (cohort 2)), or at Early Termination (ET): at any time during the visit.
The patient will complete a patient-reported diary, consisting of treatment compliance and self-assessments for efficacy.
Follow up- 2 weeks after EoT by phone call, and 4 weeks after EoT in clinic visit.

开始日期2023-03-06

申办/合作机构

Kamari Pharma Ltd.

NCT05435638

/ Completed临床1期

Phase I, Open Label Study Designed to Evaluate the Safety and Efficacy of a 1% Topical Formulation of KM-001 in the Treatment of Type I Punctate Palmoplantar Keratoderma or Pachyonychia Congenital Diseases

In this phase 1 open label study for patients with type I punctate palmoplantar keratoderma or pachyonychia congenital, 2 arms will be recruited to be treated twice daily, with 1% topical KM-001.
Arm 1: up to 10 eligible patients will be treated for 12 weeks. Arm 2: up to 8 eligible patients will be treated for 16 weeks.
Treatment safety and efficacy will be assessed in the clinic visits (for arm 1 up to day 91, for arm 2 up to day 126). In between safety will also be assessed by phone visits.
At the in-clinic visits, treatment efficacy (lesion clearance - IGA, CGI-S, PGI-C, PGI-S and VAS pain) will also be assessed.
PK blood samples will be collected for arm 1: on Days 0, 7, 84 (EoT visit). One week after the end of treatment (EoT) visit, patients will return to the clinic for final safety, efficacy and PK evaluations. For arm 2, PK blood samples will be collected on days 0, 7, 84, 112 (EoT visit). Two weeks after the end of treatment (EoT) visit, patients will return to the clinic for final safety, efficacy and PK evaluations.

开始日期2022-07-17

申办/合作机构

Kamari Pharma Ltd.

NCT05454462

/ Completed临床1期

Double-blind, Vehicle-controlled, Phase I Study to Evaluate Safety and Efficacy of a 0.3% and 1% Topical Formulation of KM-001 for Management of Moderate to Severe Pruritus in Adult Patients With Lichen Simplex Chronicus (LSC)

This is a phase 1, multi-center, randomized, vehicle-controlled, double-blinded, parallel-group study.
Approximately 6 sites will conduct the study at Germany. Approximately 61 patients (male and female) planned to be screened. 51 patient planned to be randomized.
Patients will be randomized to 1 of 3 treatment arms (KM-001 0.3%, KM-001 1%, or vehicle cream) iina a ration of 1:1:1
Patient's duration of participation will be up to 7 weeks,
a screening period with 1 visit (Visit 1) within up to 14 days (Days -14 to -1),
a 4-week treatment period with 3 visits (Visit 2 on Day 0, Visit 3 on Day 7, Visit 4 at Day 28 and 2 phone calls on Days 14 and 21, and
a 1-week follow-up period with 1 visit (Visit 5 on Day 35), as well as unscheduled visits as needed
Since KM-001 is tested in humans for the first time, the safety of KM-001 will be evaluated in a subgroup of 6 patients (sentinel group) at selected sites prior to screening of the remaining sites.
Efficacy assessments will include subjective assessments of itch and investigator assessment of the treatment effect on LSC target lesion using scoring systems.
Safety parameter (including physical examination, vital signs, ECG, standard laboratory test, and PK analysis) will be monitored from the signing of the informed consent form (ICF) until the last follow-up visit. Recording of AEs and serious AEs (SAEs) will be done throughout the study with special attention to local AEs in the treatment area (LSC target lesion, dermal safety).

开始日期2022-05-24

申办/合作机构

Kamari Pharma Ltd.

[+1]

100 项与 TRPV3 相关的临床结果

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100 项与 TRPV3 相关的转化医学

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0 项与 TRPV3 相关的专利（医药）

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525

项与 TRPV3 相关的文献（医药）

2025-07-01·The Journal of Physiological Sciences

Interaction between thermosensitive TRP channels and anoctamin 1

Review

作者: Takayama, Yasunori

Some thermosensitive transient receptor potential (TRP) channels form a protein complex with anoctamin 1 (ANO1, also called TMEM16A). TRP channels have high calcium permeability, and the calcium entering cells through TRP channel activation activates ANO1, a calcium-activated chloride channel, involved in many physiological and pathological conditions. The physiological significance of TRP channels is often mediated by their ability to activate ANO1, which controls chloride flux across the plasma membrane. This review summarizes the latest understanding on the interactions between ANO1 and thermosensitive TRP channels, including TRPV1, TRPV3, and TRPV4, which are involved in pain sensitization in primary sensory neurons, proliferation and migration of human keratinocytes, and fluid secretion such as sweat, respectively.

2025-06-01·Comparative Biochemistry and Physiology Part D: Genomics and Proteomics

Genome-wide characterization of the TRP gene family and transcriptional expression profiles under different temperatures in gecko Hemiphyllodactylus yunnanensis

Article

作者: Qu, Yanfu ; Li, Peng ; Li, Chao ; Liu, Xiaoying ; Hu, Chaochao ; Zhou, Kaiya ; Yan, Jie ; Li, Hong

Temperature is closely linked to the life history of organisms, and thus thermoception is an important sensory mechanism. Transient receptor potential (TRP) ion channels are the key mediators of thermal sensation. In this study, we analyzed the sequence characteristics of TRPs in gecko Hemiphyllodactylus yunnanensis and compared the phylogenetic relationships of TRP family members among different Squamata species. In addition, we sequenced the transcriptome of skin and brain tissues of H. yunnanensis exposed to 12 °C (cold), 20 °C (cool), 28 °C (warm), and 36 °C (hot). The results showed that a total of 591 TRPs were identified in the genomes of 21 Squamate species, and these genes were classified into six subfamilies. Among them, 26 TRP genes were identified in H. yunnanensis and distributed on 13 chromosomes. Overall, TRP genes were conserved in squamates. Based on the transcriptome results, we found a total of 9 TRP genes expressed in the brain and skin of H. yunnanensis, of which six TRP genes were under positive selection. TRPP1L2, TRPP1L3, and TRPV1 were involved in heat-sensitive responses (> 36 °C), and TRPV3, TRPA1, and TRPM8 were involved in cold-sensitive responses (< 20 °C). TRPM8 and TRPP1L2 were important cold and heat sensors in H. yunnanensis, respectively.

2025-06-01·Molecular Therapy Nucleic Acids

Papillomavirus-like particles as vectors for ex vivo gene therapy of the skin

Article

作者: Diversi, Francesco ; Heppenstall, Paul A ; Rinaldin, Mirko ; Mazza, Elisa ; Dabin, Juliette ; Hackett, Jamie A ; de Castro Reis, Fernanda

Ex vivo gene delivery to the skin utilizing retroviral vectors has been demonstrated to be a viable clinical option for replacement of defective genes. However, because these vectors integrate their cargo into the genome, safety issues arise when utilizing them to deliver gene-editing nucleases. Here, we explored the use of Papillomavirus, a non-integrating viral vector, for ex vivo skin gene editing, exploiting its natural tropism for basal keratinocytes. We demonstrated that Papillomavirus-like particles (PVLPs) can deliver a variety of DNA constructs encoding fluorophores, Cre recombinase, calcium indicators, Cas9, and short hairpin RNA (shRNA) to keratinocytes, offering advantages over other viral vectors such as adeno-associated virus (AAV) and Lentivirus. We further showed that PVLPs can be used for gene therapy for Olmsted syndrome, a genetic skin disease caused by a gain-of-function mutation in the Trpv3 gene. Specifically, PVLP-delivered SaCas9 and shRNA effectively disrupted the Trpv3 gene or reduced its expression, leading to decreased TRPV3 activity and mitigating the hyperactivity associated with Olmsted syndrome. Skin equivalents generated from PVLP-treated keratinocytes exhibited complete transduction, and PVLP-shRNA treatment significantly reduced hyperkeratosis in skin equivalents from mice bearing the Olmsted syndrome mutation. These findings highlight PVLP as a promising tool for ex vivo skin gene therapy.

项与 TRPV3 相关的新闻（医药）

2025-04-01

·学术经纬

《自然》子刊：天然精油促进皮肤更新的原因找到了

▎药明康德内容团队编辑（来源：武汉大学）芳香植物来源的天然精油在日常生活中应用广泛，涵盖芳香治疗、精油按摩和皮肤护理等领域。研究表明天然精油能够促进皮肤更新并维持其健康状态，但精油发挥效应的分子靶点及机制尚未明确。近日，Nature Communications在线刊发了武汉大学生命科学学院、病毒学国家重点实验室、泰康生命医学中心姚镜教授课题组的研究进展，论文题目为“Plant essential oil targets TRPV3 for skin renewal and structural mechanism of action”，揭示了天然精油激活TRPV3通道促进皮肤更新的作用机制。天然精油的主要生物活性成分是单萜类化合物，本研究选取了植物源精油中的活性成分香茅醛、柠檬醛、芳樟醇及可可醛等无环单萜类化合物，以小鼠为研究模型，通过涂抹其背部皮肤，免疫组化等实验结果发现这些化合物可通过促进角质形成细胞的增殖，并显著提高小鼠表皮层厚度。表皮层的增厚通常是健康皮肤的特征，常见于年轻个体，随着年龄增长和细胞干性下降而逐渐变薄。为揭示无环单萜类化合物作用于角质形成细胞的分子靶点，研究人员分离新生小鼠皮肤的原代角质细胞，发现无环单萜类化合物激活一种非选择性的阳离子通道，且诱发的电流具有TRPV3通道的敏化特征，推测该类天然活性物激活定位在角质细胞中的TRPV3通道。▲天然精油激活TRPV3通道促进皮肤更新的模式图（图片来源：原始论文[1]）同时，在人源的角质化细胞系中也观察到了精油物质对细胞增殖的显著促进作用。进一步实验发现， TRPV3通道敲除后的小鼠角质形成细胞对无环单萜类化合物无响应，且涂抹皮肤后表皮厚度无显著变化，表明无环单萜类化合物通过激活TRPV3通道促进皮肤更新。为进一步理解天然植物精油化合物激活TRPV3通道蛋白的分子机制，研究人员利用单颗粒冷冻电镜技术解析人源TRPV3与多个无环单萜类化合物复合体的三维结构，发现这些化合物结合在人源TRPV3通道跨膜结构域的疏水口袋。功能验证表明，无环单萜类化合物通过竞争性结合于该口袋，促使静息态通道蛋白中结合的内源性脂质分子解离，引起通道蛋白构象变化，导致通道开放。TRPV3通道开放介导的Ca2+内流激活EGFR信号通路，促进细胞生长因子释放，诱导角质细胞增殖，从而促进皮肤更新。综上，本文明确了植物源无环单萜类化合物在皮肤中的分子靶点为TRPV3通道，揭示了其促进皮肤更新的分子机制，即通过激活TRPV3通道，引发一系列下游信号通路，促进皮肤细胞增殖和更新。此外，本研究还加深了对TRPV3通道的药理学调控和门控机制的理解，为基于结构的药物设计提供了新思路。单颗粒冷冻电镜技术得到了中国科学技术大学的田长麟教授、孙德猛研究员，以及中国科学院武汉病毒研究所的邓增钦研究员的大力支持与帮助。武汉大学生命科学学院的博士研究生李阳和陆徐腾为本文的共同第一作者，程旭峰、夏克玉、胡俊彦、王培宇、庞培源、高博旻等同学也参与了该研究，姚镜教授为通讯作者。此外，武汉大学生命科学学院的张竹珍教授、谢畅高级实验师和法国国家健康与医学研究院的李栋栋研究员也为该项研究的顺利进行提供了帮助。该研究工作得到了国家重点研发计划、国家自然科学基金和中央高校基础研究基金等项目的资助。原始论文：[1] Li, Y., Lu, X., Cheng, X. et al. Plant essential oil targets TRPV3 for skin renewal and structural mechanism of action. Nat Commun 16, 2728 (2025). https://doi.org/10.1038/s41467-025-58033-9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。更多推荐点个“在看”再走吧~

2025-03-23

·知识分子

气温升高也会引发失眠

3.23 知识分子 The Intellectual 跟踪前沿进展，掌握最新动态一手掌握一周重大科技新闻撰文 | 姚湧小学森庐州月责编 | 既来知 ● 　● 　● 1 气温升高也会引发失眠图片来源：pixabay.com 在气候变化的大趋势下，环境温度上升对睡眠的影响越来越令人担忧。睡眠由非快速眼动和快速眼动两期构成，对健康至关重要，任何一个阶段的睡眠不足都可能产生重大影响。近日，复旦大学公共卫生学院Renjie Chen团队基于来自中国大陆214445名参与者的2300万天睡眠监测数据，分析了日平均气温如何影响睡眠。结果显示，环境温度每升高10°C，睡眠不足的几率会增加20.1%，而总睡眠时间减少9.67分钟，深度睡眠下降幅度最大（2.82%）。根据无限制（SSP5-8.5）温室气体排放情景进行预测，到本世纪末，人们的睡眠不足可能会增加10.50%，每人每年损失33.28小时的睡眠。这些发现突显了气候变暖在加剧睡眠剥夺和降低睡眠质量方面的潜在作用，特别是对于老年人、妇女、肥胖者以及华南、华中和华东地区。 ►文章链接： https://doi.org/10.1038/s41467-025-57781-y 2 植物精油如何让皮肤更新？图片来源：pixabay.com 让皮肤保持水润光泽是众多爱美人士的追求。天然精油是一种被广泛用于皮肤护理的化妆品。近日，武汉大学生命科学学院Jing Yao团队证实，香茅醛（一种从植物提纯的无环单萜）可通过激活TRPV3促进角质形成细胞增殖来刺激皮肤更新。该团队还通过冷冻电镜成像技术获得了TRPV3与无环单萜（包括香茅醛、柠檬醛、芳樟醇和异二氢薰衣草醛等精油成分）复合物在分辨率为3.1-3.6Å水平的低温EM结构，分析揭示了TRPV3香草酸位点内一致但略微不同的结合模式。该研究结果表明，精油配体通过竞争性置换香草酸位点的内源性脂质来激活TRPV3通道，促进皮肤更新，为我们理解精油的作用机制提供了科学依据。相关论文于3月19日发表在《自然·通讯》（Nature communications）杂志。 ►文章链接： https://doi.org/10.1038/s41467-025-58033-9 3 利用废二氧化碳合成人体必需氨基酸图片来源：pixabay.com L-苯丙氨酸（L -Phe）是人体的必需氨基酸（不能合成或合成速度远不能适应机体需要）之一，在机体代谢中其关键作用。目前，L -Phe是通过微生物和酶过程在内的生物合成方法获得，但实际产量较低。近日，湖南大学化学化工学院Jianyun Zheng团队设计出一种用于通过废二氧化碳和硝基苯乙烷的光电化学偶联合成L-Phe的分级硅基光电阴极，在一个太阳光强照射且低施加电势下实现了37.5 μg·hour−1·cm−2的高产率和21.2%的法拉第效率（电化学反应中转化电流和物质之间的利用效率）。CuO-TiO2-C混合物分散在非晶TiO2层/n+p-Si中的分层结构产生了内置电场，并形成了丰富的导电通道，有效地将电子注入到Cu和Ti位点。这些Cu和Ti位点分别吸附和活化CO2和硝基苯乙烷，协同促进L-Phe的合成。该研究建立了一种将太阳能转化为必需氨基酸产品的环保、高效方法，相关论文于3月19日发表在《科学·进展》（Science Advances）杂志。 ►文章链接： https://www.science.org/doi/10.1126/sciadv.adr8651 4 全球哪些国家的甲基汞暴露水平更高？图片来源：pixabay.com 甲基汞（MeHg）是一种神经毒素，海产品消费是甲基汞暴露的主要途径。那么，海鲜消费上游的生产和贸易环节是如何影响MeHg暴露的呢？近日，清华大学环境学院王书肖团队对2019年全球海产品生产、贸易和消费中的MeHg水平进行了量化分析。结果显示，海产品MeHg暴露量超过世界卫生组织推荐阈值的主要是高收入国家。与低收入国家相比，由于海产品消费量更高以及常被忽视的MeHg污染，高收入国家的MeHg暴露水平增加了十倍。值得注意的是，生产环节中43%的海产品MeHg通过全球贸易完成了重新分配：高收入国家向低收入国家的出口含有更高浓度MeHg的海产品。据统计，MeHg暴露可能导致全球61800人过早死亡，经济损失约2.87万亿美元。详细数据于3月13日发表在《自然·食品》（Nature Food）杂志。 ►文章链接： https://www.nature.com/articles/s43016-025-01136-9 5 动物通过这个蛋白激发口渴感受图片来源：pixabay.com 口渴是动物最为常见的感觉，促使动物补充液体来恢复机体的水稳态。一般来讲，血液渗透压升高会通过激活表达在大脑穹窿下器官（SFO）的高渗传感器来引起口渴，但这种传感器的真实身份一直是个谜。近日，首都医科大学基础医学院Zhiqiang Yan团队通过一系列实验表明，SFO神经元上的TMEM63B发挥了哺乳动物高渗传感器的作用。研究人员发现，TMEM63B在SFO兴奋性神经元中表达，是神经元对高渗刺激做出反应的必要条件。更重要的是，运用非SFO神经元细胞表达的TMEM63B也能被高渗刺激激活，纯化的TMEM63B能展现出渗透压门控电流。此外，Tmem63b基因敲除小鼠在口渴感知方面存在严重缺陷，SFO神经元特异性Tmem63b缺失同样使小鼠无法感知口渴。该研究表明，TMEM63B是哺乳动物的一种高渗口渴传感器。详细数据于3月18日发表在《神经元》（Neuron）杂志。 ►文章链接： https://doi.org/10.1016/j.neuron.2025.02.012 6 可穿戴超声微针贴片用于癌症免疫治疗图片来源：pixabay.com 癌症的临床治疗一直存在几大挑战，包括药物递送效率低下、副作用频发和可操作性差等。近日，四川大学材料科学与工程学院Jiagang Wu团队研发出一种集成化可穿戴柔性超声微针贴片（wf-UMP），为癌症治疗搭建了方便、高效的便携式平台。wf-UMP将可拉伸无铅超声换能器阵列、生物粘附水凝胶弹性体，以及装载有生物相容性压电纳米颗粒的可溶解微针贴片进行高效集成，能无痛输送药物，生成活性氧物质。凭借柔软的机械性能和强化的机电性能，wf-UMP可以牢固佩戴于组织表面，操作简单。小鼠实验结果表明，wf-UMP通过诱导肿瘤细胞凋亡、增强氧化应激和调节免疫细胞增殖表现出卓越的抗癌效果。此外，wf-UMP和抗PD1诱导的协同免疫治疗通过激活免疫原性细胞死亡和调节巨噬细胞极化、抑制肿瘤生长和肿瘤复发，进一步提高了动物的抗癌免疫力。相关论文于3月18日发表在《自然·通讯》（Nature communications）杂志。 ►文章链接： https://doi.org/10.1038/s41467-025-58075-z 亲爱的读者们，不星标《知识分子》公众号，会错过每日科学新知！星标《知识分子》，紧跟前沿科学，一起探索科学的奥秘吧！请戳上图卡片添加星标关注《知识分子》视频号 get更多有趣、有料的科普内容 END

临床结果

2024-05-17

·BioSpace

Kamari Pharma Announces New Data Presented at the 81st Annual Meeting of the Society for Investigative Dermatology

Phase 1b clinical trial of KM-001 demonstrated favorable safety pro high responder rate of 87% in patients with palmoplantar keratoderma and pachyonychia congenita In vitro and in vivo data of KM-001 and KM-023 demonstrated significant improvements in skin histology and significant reductions in scratching NESS ZIONA, Israel, May 17, 2024 (GLOBE NEWSWIRE) -- Kamari Pharma, a privately-held clinical stage biotechnology company developing first and best-in-class treatments for rare and severe genetic skin diseases, today announced two presentations reporting results for its novel TRPV3 inhibitors, KM-001 (topical) and KM-023 (oral), were given at the 81st Annual Meeting of the Society for Investigative Dermatology (SID) that is taking place May 15-18, 2024 in Dallas, TX, USA. Below are summaries of the presentations: Poster #LB941: “KM-001, a novel TRPV3 inhibitor, demonstrates safety and preliminary efficacy in Phase 1b clinical study for the treatment of palmoplantar keratoderma” presented by Edel O’Toole, M.D., Ph.D. Professor of Molecular Dermatology and Centre Lead of the Centre for Cell Biology and Cutaneous Research, Blizard Institute, Queen Mary University of London and lead investigator in the study. Presented results from twelve-week, Phase 1b open-label studies (UK and Israel) assessing 4g/day of KM-001 1% cream applied to each foot twice daily for the treatment of pachyonychia congenita (PC) and punctate palmoplantar keratoderma type 1 (PPPK1). Eight patients in the UK (7 PC;1 PPPK1) and seven patients in Israel (3 PC;4 PPPK1) completed the treatment period. Safety: No drug-related severe adverse events nor systemic side effects were observed. Minimal treatment emergent adverse events occurred (n=11); all were mild and did not lead to patient discontinuation. Efficacy: Responder rate at the end of treatment was 88% (CI 52.9-97.8%) in UK (n=8) and 86% (CI 48.7-97.4%) in Israel (n=7), as defined by an improvement in at least one of six pre-defined disease parameters. Additionally, 47% of patients improved in at least two parameters with a disease severity reduction trend and pain score measured by VAS was reduced in 60% of PC patients; PPPK1 patients do not have pain as a symptom. Presentation #771: “Novel TRPV3 inhibitors developed for the treatment of palmoplantar keratodermas, demonstrate safety and efficacy in preclinical models” presented by Liora Braiman-Wiksman, Ph.D., Chief Scientific Officer at Kamari. Presented preclinical results for KM-001 and KM-023, Kamari’s specific and highly selective TRPV3 inhibitors designed to address both molecular and local damage to the skin by regulating Ca²⁺ influx into the cell. In 3D skin models of PC and PPPK1, KM-001 and KM-023 normalized proliferation and differentiation markers and enhanced epidermal barrier formation. In in vivo efficacy studies performed in DS-Nh mice, a gain-of-function TRPV3 mutation model, KM-001 showed significant improvement in skin histology vs vehicle (70% vs 30%) and exhibited significant reduction in scratching bouts (80%, p>0.04) and KM-023 showed a significant reduction in keratoderma severity score (p=0.003) and reduced scratching significantly (p<0.001) vs vehicle. Based on these results, Kamari plans to initiate Phase 2 development of KM-001 for the treatment of palmoplantar keratoderma and advance KM-023 into first-in-human studies for the oral treatment of Olmstead syndrome, severe keratoderma and Ichthyosis. The full content of these posters is available in the News page of Kamari's corporate website ( ). About Kamari Pharma Kamari Pharma is a privately-held clinical stage biotechnology company developing first and best in class treatments for rare and severe genetic skin diseases. Kamari’s lead molecules, KM-001 (topical) and KM-023 (oral) are novel, highly specific and selective TRPV3 inhibitors that are initially being developed to treat palmoplantar keratodermas, Olmstead syndrome and Ichthyosis. Kamari’s management team is comprised of industry leaders highly experienced in drug discovery, dermatological pharmaceutical development and rare disease drug development. Contact Information Investors and Media Marcy Nanus Managing Partner Trilon Advisors, LLC mnanus@trilonadvisors.com

临床结果临床1期AACR会议