A Phase I/II Open-Label, Multi-centre Study to Assess the Safety and Tolerability of Roginolisib in Combination With Ruxolitinib in Patients With Myelofibrosis (MF) Who Are Unresponsive to an Approved JAK Inhibitors (HEMA-MED)

The goal of this clinical trial is to learn how roginolisib works in comparison to standard treatment in adult patients with Myelofibrosis. The main questions it aims to answer is to evaluate the safety and tolerability of roginolisib when administered in combination with ruxolitinib.

开始日期2025-11-17

申办/合作机构

iOnctura SA

NCT07018752

/ Recruiting临床1/2期IIT

A Platform Trial, Evaluating New Drugs or Combination in Relapsed or Refractory Peripheral T-cell Lymphomas

This study is a platform trial for the evaluation of new drugs or combination of drugs in relapsed or refractory peripheral T-cell lymphomas.
The objective of the study is to generate exploratory data on new drugs or combination of drugs to treat refractory/relapse peripheral T-cells lymphoma to better identify the population of interest and design future correct clinical trials.
Primary objectives of the different sub-studies :
* phase 1 sub-studies: determine the safety and tolerability of escalating doses of the sub-study treatment
* phase 2 sub-studies: identify drugs that will improve significantly the outcome in target patients Secondary objectives of both sub-studies: analyze the response rate, the clinical benefit rate, the progression-free survival, the duration of response, the time to next treatment or death, the overall survival, the rate of transplantation following study treatment and the safety profile of the drugs used

开始日期2025-08-20

申办/合作机构

Le LYSARC

[+3]

100 项与 Roginolisib 相关的临床结果

登录后查看更多信息

100 项与 Roginolisib 相关的转化医学

登录后查看更多信息

100 项与 Roginolisib 相关的专利（医药）

登录后查看更多信息

项与 Roginolisib 相关的文献（医药）

2024-07-22·Journal of Chemical Information and Modeling

Challenging Complexity with Simplicity: Rethinking the Role of Single-Step Models in Computer-Aided Synthesis Planning

Article

作者: Lai, Luhua ; Lin, Kangjie ; Pei, Jianfeng ; Li, Junren

Computer-assisted synthesis planning has become increasingly important in drug discovery. While deep-learning models have shown remarkable progress in achieving high accuracies for single-step retrosynthetic predictions, their performances in retrosynthetic route planning need to be checked. This study compares the intricate single-step models with a straightforward template enumeration approach for retrosynthetic route planning on a real-world drug molecule data set. Despite the superior single-step accuracy of advanced models, the template enumeration method with a heuristic-based retrosynthesis knowledge score was found to surpass them in efficiency in searching the reaction space, achieving a higher or comparable solve rate within the same time frame. This counterintuitive result underscores the importance of efficiency and retrosynthesis knowledge in retrosynthesis route planning and suggests that future research should incorporate a simple template enumeration as a benchmark. It also suggests that this simple yet effective strategy should be considered alongside more complex models to better cater to the practical needs of computer-assisted synthesis planning in drug discovery.

2024-05-01·Translational oncology

The highly selective and oral phosphoinositide 3-kinase delta (PI3K-δ) inhibitor roginolisib induces apoptosis in mesothelioma cells and increases immune effector cell composition

Article

作者: Joanna Kopecka ; Karolina Niewola-Staszkowska ; Francesca Finotello ; Roger Falkenstern-Ge ; German Ott ; Michael Lahn ; Lars van der Veen ; Julia Schüler ; Chiara Riganti ; Giusy Di Conza ; Claudia Kalla

Targeting aberrantly expressed kinases in malignant pleural mesothelioma (MPM) is a promising therapeutic strategy. We here investigated the effect of the novel and highly selective Phosphoinositide 3-kinase delta (PI3K-δ) inhibitor roginolisib (IOA-244) on MPM cells and on the immune cells in MPM microenvironment. To this aim, we analyzed the expression of PI3K-δ by immunohistochemistry in specimens from primary MPM, cell viability and death in three different MPM cell lines treated with roginolisib alone and in combination with ipatasertib (AKT inhibitor) and sapanisertib (mTOR inhibitor). In a co-culture model of patient-derived MPM cells, autologous peripheral blood mononuclear cells and fibroblasts, the tumor cell viability and changes in immune cell composition were investigated after treatment of roginolisib with nivolumab and cisplatin. PI3K-δ was detected in 66/89 (74%) MPM tumors and was associated with reduced overall survival (12 vs. 25 months, P=0.0452). Roginolisib induced apoptosis in MPM cells and enhanced the anti-tumor efficacy of AKT and mTOR kinase inhibitors by suppressing PI3K-δ/AKT/mTOR and ERK1/2 signaling. Furthermore, the combination of roginolisib with chemotherapy and immunotherapy re-balanced the immune cell composition, increasing effector T-cells and reducing immune suppressive cells. Overall, roginolisib induces apoptosis in MPM cells and increases the antitumor immune cell effector function when combined with nivolumab and cisplatin. These results provide first insights on the potential of roginolisib as a therapeutic agent in patients with MPM and its potential in combination with established immunotherapy regimen.

2023-12-01·International journal of toxicology

Non-Clinical Toxicology Evaluation of the Novel Non-ATP Competitive Oral PI3 Kinase Delta Inhibitor Roginolisib

Article

作者: Deken, Marcel A. ; Lahn, Michael ; van der Veen, Lars ; Schmitt, Michael

Roginolisib (IOA-244) is a novel, non-ATP competitive phosphoinositide-3-kinase (PI3K) delta inhibitor that regulates Akt/mTOR signaling. Roginolisib was administered once daily to rats and dogs in dose-range finding (DRF) and 4-week GLP toxicology studies. Free plasma levels of roginolisib exceeded the cellular target engagement IC90 for PI3Kδ for ≥12 hours at doses of 5 mg/kg, the IC90 for PI3Kβ for ≥2 hours at doses ≥15 mg/kg, and the IC50 for PI3Kα for ≥2 hours at dose levels ≥45 mg/kg. Toxicity in rats occurred at doses ≥100 mg/kg. In dogs, we observed dose-dependent skin and gastrointestinal toxicity and doses ≥30 mg/kg had a greater incidence of mortality. Lymphoid tissue toxicity occurred in both species. Toxicities in dogs observed at the ≥15 mg/kg dose, affecting the digestive mucosa, liver, and skin, cleared after treatment cessation. Doses ≤75 mg/kg were tolerated in rats and the no-observed-adverse-effect-level (NOAEL) in rats was 15 mg/kg. Due to mainly epithelial lesions of the skin at 5 mg/kg and necrotizing damage of the intestinal epithelia at ≥15 mg/kg, no NOAEL was determined in dogs. However, the adverse effects observed in dogs at 5 mg/kg were considered monitorable and reversible in patients with advanced malignancies. Furthermore, the PK profile subsequently proved to be a decisive factor for achieving selective PI3Kδ inhibition without the toxicities observed in dogs. As the result of the unique PK profile of roginolisib, patients were able to take daily roginolisib without dose modification and showed pharmacodynamic PI3Kδ inhibition over several months without gastrointestinal or dermatologic toxicities.

项与 Roginolisib 相关的新闻（医药）

2026-04-20

·Derek工作室

【信号通路】《Nat Rev Drug Discov》——PI3K/AKT/mTOR靶向药涅槃之路

在现代肿瘤药物研发史上，很少有一条信号通路能像PI3K/AKT/mTOR这样，掌管细胞生长、增殖、代谢与存活，在癌症、免疫缺陷、遗传疾病中全面失控，是学界公认的“黄金靶点”。它横跨肿瘤、免疫、遗传疾病、组织再生四大领域，调控细胞增殖、存活、代谢、迁移与蛋白翻译的全部核心过程，是人类疾病中异常激活频率最高的通路之一。过去二十年，这条通路的药物研发走得磕磕绊绊：毒性难控、耐药频发、多款药物黯然撤市，一度让整个领域陷入低谷。 2026年3月30日，Bart Vanhaesebroeck教授在《Nature Reviews Drug Discovery》发表重磅综述《A renaissance in targeting the PI3K/AKT/mTOR pathway》，以完整的临床证据、药物结构创新与生物学机制突破，正式宣告：靶向PI3K/AKT/mTOR通路的药物研发，迎来了真正意义上的科学复兴。这不是简单的新药上市，而是一场从靶点理解、分子设计、临床策略到适应症拓展的全面革命，它将重新定义精准医疗的边界，也为无数耐药、罕见病与晚期肿瘤患者带来全新希望。【信号通路与免疫】 PI3K/AKT/mTOR通路调控线粒体能量代谢调节T细胞功能【信号通路与疾病】PI3K - Akt 信号通路：癌症的 “隐形推手” 与治疗新曙光【信号通路】PI3K-Akt信号通路一、PI3K/AKT/mTOR：被称为“全能靶点”的底层逻辑 PI3K/AKT/mTOR通路的核心逻辑并不复杂，却在生命活动中占据不可替代的地位。ClassIPI3K作为通路起点，在受体酪氨酸激酶、G蛋白偶联受体或RAS激活后，催化细胞膜上的PI(4,5)P2生成第二信使PI(3,4,5)P3，后者招募并激活AKT，AKT进一步磷酸化激活mTORC1与mTORC2两大复合物，最终驱动细胞生长、蛋白合成与代谢重编程。而PTEN作为通路最关键的负向调控因子，通过去磷酸化阻断信号传递，如同系统的“刹车装置”。 1.1通路异常：几乎所有重大疾病的共同根源在正常细胞中，这套系统精密平衡；但在疾病状态下，PIK3CA激活突变、AKT1E17K突变、PTEN功能缺失、TSC1/TSC2失活等遗传改变，会让通路持续处于“油门踩死”的状态。数据显示，约8%的实体瘤存在PIK3CA突变，超过30%的肿瘤伴随PTEN失活，在HR+/HER2−乳腺癌中这一比例更是高达40%。同时在慢性淋巴细胞白血病、滤泡淋巴瘤、活化PI3Kδ综合征（APDS）、PIK3CA相关过度增生综合征（PROS）、结节性硬化症等疾病中，通路异常同样是致病根源。这意味着，只要能安全、精准、持久地调控这条通路，就能同时攻克肿瘤、免疫缺陷与罕见遗传疾病，这也是它被称为“全能靶点”的根本原因。超40%的HR+乳腺癌携带PIK3CA突变，是内分泌耐药的核心元凶；超30%的肿瘤存在PTEN缺失，直接松开通路的“刹车”；白血病、淋巴瘤高度依赖PI3Kδ信号存活；结节性硬化、PIK3CA 相关过度增生综合征（PROS）等罕见病，根源就是通路先天异常激活。 1.2已上市药物全景：从拓荒到挣扎截至2026年1月，FDA正式批准的PI3K/AKT/mTOR通路抑制剂已形成清晰谱系，包括PI3Kα抑制剂、PI3Kδ/γ抑制剂、泛PI3K抑制剂、AKT抑制剂、mTORC1抑制剂五大类。其中，alpelisib、inavolisib开启了PI3Kα靶向乳腺癌的时代，capivasertib成为首个上市AKT抑制剂，leniolisib填补了APDS罕见病治疗空白，而雷帕霉素、依维莫司、替西罗莫司则作为经典mTOR抑制剂，在移植免疫与肿瘤治疗中长期占据一席之地。但与之相伴的是，多款药物因安全性问题撤回惰性淋巴瘤适应症，甚至全面撤市，让这条通路的临床价值长期被低估。二、二十年血泪教训：第一代药物为何集体折戟？尽管生物学价值明确，但PI3K/AKT/mTOR抑制剂的研发之路却充满坎坷。早在1999年，mTORC1抑制剂雷帕霉素就以免疫抑制剂身份获批上市，开启了通路靶向的序幕；随后十几年间，一系列ATP竞争性抑制剂陆续进入临床，却集体暴露致命短板，最终构成了靶点研发史上最深刻的一课。 2.1靶点毒性不可规避：正常细胞同样高度依赖通路调控正常生理功能，抑制必然带来连锁反应：PI3Kα与AKT参与胰岛素信号调控，抑制后直接引发高血糖、腹泻、皮疹；PI3Kδ/γ主导免疫细胞稳态，抑制后导致免疫失衡、结肠炎、机会性感染；mTOR抑制剂则常引发严重口腔黏膜炎，让患者无法长期耐受。简单来说，不抑制不足以抗癌，抑制过度则无法耐受，这是早期药物最难以突破的死结。抑制PI3Kα/AKT→高血糖、腹泻、皮疹，直接逼停治疗；抑制PI3Kδ/γ→免疫紊乱、结肠炎、严重感染，多款药物因此撤市； mTOR抑制剂→口腔黏膜炎，慢性疾病患者根本无法长期耐受。 2.2反馈环路与代偿激活：耐药与生俱来通路内部存在强大的负反馈环路，单一节点抑制会触发代偿性复活：抑制mTORC1会解除对IRS1的抑制，导致PI3K/AKT重新活化；抑制PI3Kα会引发PI3Kβ等其他亚型代偿，让肿瘤快速耐药。临床治疗中，PTEN缺失、AKT突变、PIK3CA二次突变都能快速让药物失效，而早期广谱抑制剂进一步加速了这一过程。 2.3认知误区：将细胞稳态误判为细胞毒杀早期研究因工具化合物非特异性，错误认为PI3K/AKT/mTOR抑制剂可直接杀伤肿瘤细胞。后续精准研究证实，这类药物主要为细胞稳态效应（cytostatic），而非细胞毒杀（cytotoxic），单药难以实现肿瘤消退，必须依赖合理联合，而这一点在早期临床设计中被普遍忽视。 2.4临床与监管困境：血液肿瘤领域全面受挫在慢性淋巴细胞白血病与淋巴瘤领域，PI3Kδ抑制剂曾迎来爆发式上市，但伴随严重免疫毒性与黑框警告，叠加BTK抑制剂、BCL-2抑制剂等更优方案出现，最终idelalisib、duvelisib、umbralisib、copanlisib等先后撤回惰性淋巴瘤适应症。监管端也提出更高要求，以总生存作为主要终点，进一步压缩了这类药物的临床空间。三、复兴核心：六大颠覆性技术，彻底改写药物规则真正的破局，来自基础生物学的深度突破与药物化学的颠覆性创新。综述明确指出，新一代PI3K/AKT/mTOR调控剂已经完全跳出传统ATP竞争性抑制的框架，形成了突变选择性抑制、共价不可逆抑制、蛋白降解、变构调控、蛋白互作干扰、通路激活六大创新技术体系，每一种策略都直击老药的核心缺陷。 3.1突变选择性变构抑制剂：只杀突变，保留野生型以STX-478（tersolisib）、RLY-2608（zovegalisib）为代表，这类药物不结合ATP口袋，而是识别PI3Kα突变体特有的变构口袋，对H1047R、E542K、E545K等热点突变具有高度选择性，完全保留野生型PI3Kα功能。临床数据显示，这类药物在最大耐受剂量下不引发高血糖、无3/4级皮疹与黏膜炎，耐受性显著优于alpelisib与inavolisib，同时能克服老药的继发耐药，为经治患者提供全新选择。 3.2共价抑制剂：长效强效，一击锁死激酶通过亲电弹头与靶点蛋白形成不可逆结合，实现长效、强效、高选择性抑制。代表性药物TOS-358与Compound9靶向PI3Kα非催化半胱氨酸，不仅抑制活性还能诱导蛋白降解，在动物模型中展现出更强的抗肿瘤效果且不影响血糖稳态。相比于可逆药物，共价抑制剂能突破结合动力学限制，实现更稳定的通路控制。 3.3蛋白降解剂PROTAC：不只抑制，直接清除蛋白不同于传统抑制剂仅阻断激酶活性，降解剂能将PI3K、AKT、mTOR蛋白直接送入蛋白酶体降解，同时消除激酶功能与支架功能。针对PI3Kα、PI3Kγ、PI3Kδ、AKT、mTOR的降解剂均已进入临床前或临床早期，在低剂量下即可实现完全通路抑制，大幅降低毒性风险，被视为攻克耐药的终极武器之一。 3.4构象选择性抑制剂：以roginolisib重新定义PI3Kδ roginolisib不采用传统“螺旋桨”结构，而是通过稳定PI3Kδ的非活性构象实现超高选择性，不影响其他亚型，从而避免结肠炎、免疫紊乱等毒性。临床中可连续长期给药，无需间断，在葡萄膜黑色素瘤、淋巴瘤中展现单药疗效，成为新一代安全型PI3Kδ抑制剂标杆。 3.5RAS–PI3Kα breaker：不抑激酶，只断互作全新机制药物，不影响PI3Kα激酶活性，仅通过共价结合阻断PI3Kα与RAS的相互作用，专门针对RAS突变与HER2扩增肿瘤，具备不升高血糖、不干扰免疫的独特优势。BBO-1020、VVD-699等已进入临床I期，开辟RAS靶向全新路径。 3.6通路激活剂：从抗癌到修复，颠覆认知最具突破性的是，通路不再只有“抑制”一条路。UCL-TRO-1938作为直接变构PI3Kα激活剂，可用于心肌缺血再灌注损伤保护与神经再生；NV-5138作为mTORC1选择性激活剂，进入难治性抑郁症临床II期。这条通路由此从“抗癌靶点”拓展至“组织修复与神经调控”平台。四、临床全面爆发：从乳腺癌到罕见病，适应症全线突破随着创新药物不断落地，PI3K/AKT/mTOR靶向治疗已经从“小众备选”升级为多疾病领域的标准方案，临床价值全面爆发，覆盖实体瘤、血液肿瘤、原发性免疫缺陷、过度增生综合征、血管畸形等一系列疾病。 4.1HR+/HER2−乳腺癌：通路抑制剂成为核心支柱在乳腺癌领域，PI3Kα与AKT抑制剂已经构建起完整的一线、二线治疗梯队。alpelisib联合氟维司群获批二线治疗，inavolisib联合哌柏西利与氟维司群成为一线首选，将总生存期提升至34个月，首次在PI3K通路抑制剂中实现总生存获益里程碑；capivasertib采用“用4天停3天”给药方案，覆盖PIK3CA、AKT1、PTEN三类突变患者，高血糖风险显著降低，目前已向前列腺癌等实体瘤拓展。 4.2血液肿瘤与免疫疾病：PI3Kδ安全重生曾经因毒性撤市的PI3Kδ抑制剂迎来全面重生。leniolisib获批治疗APDS原发性免疫缺陷，实现长期安全有效，显著改善感染与生活质量；roginolisib在慢性淋巴细胞白血病、骨髓纤维化、葡萄膜黑色素瘤中展现稳定疗效；同时，PI3Kγ抑制剂在急性髓系白血病中重新崛起，从免疫靶点转变为白血病细胞内在依赖靶点。 4.3罕见病：无药可治局面被彻底打破 alpelisib获批PIK3CA相关过度增生综合征（PROS），miransertib、engasertib等AKT抑制剂在血管畸形、Proteus综合征、遗传性出血性毛细血管扩张症中展现明确疗效；雷帕霉素类药物长期用于结节性硬化症，而PI3K/AKT/mTOR通路靶向药正在成为一系列“TORopathies”类罕见病的标准解决方案。 4.4免疫肿瘤与联合策略：打开更大空间 PI3Kδ抑制剂可选择性下调Treg细胞，增强抗肿瘤免疫；PI3Kγ抑制剂重编程肿瘤相关巨噬细胞；PI3Kβ抑制剂则在PTEN缺失肿瘤中逆转免疫逃逸。这些发现让通路抑制剂从单纯“杀伤肿瘤”转变为“免疫调控+肿瘤抑制”的双重武器，与PD-1/PD-L1抑制剂的联合正在成为新的研究热点。五、从失败到复兴：靶点研发的教科书级启示回顾PI3K/AKT/mTOR通路二十年研发历程，它不仅是一部药物创新史，更是一部人类认知不断深化的科学史。从最初认为“通路抑制即可杀伤肿瘤”，到发现抑制剂仅为细胞稳态而非细胞毒性；从广谱抑制导致毒性失控，到突变选择性实现精准打击；从单一靶点阻断引发耐药，到多节点联合与蛋白降解彻底阻断通路；从仅关注肿瘤细胞内在调控，到重视免疫微环境与代谢协同；从只追求通路抑制，到开发激活剂用于组织再生——每一次认知升级，都带来一次技术突破。从盲目广谱抑制，到亚型选择性、突变选择性、构象选择性；从单纯阻断激酶，到蛋白降解、蛋白互作干扰、通路激活；从只盯肿瘤细胞，到兼顾免疫、代谢、耐受。这场复兴告诉我们：没有失败的靶点，只有不够精准的药物。这场复兴的核心启示在于：没有失败的靶点，只有不够深刻的机制理解与不够精准的分子设计。PI3K/AKT/mTOR通路从未失去价值，只是需要更科学、更精细、更个性化的调控方式。它告诉整个医药行业，真正的突破永远来自基础研究的深度，而非临床试错的速度。六、未来十年：PI3K/AKT/mTOR将走向何方？站在2026年的时间节点，我们已经清晰看到这条通路的未来图景：突变选择性抑制剂将成为实体瘤主流，共价药物与降解剂攻克耐药难题，RAS–PI3Kbreaker打开RAS肿瘤治疗新窗口，PI3Kδ抑制剂重塑免疫肿瘤治疗格局，激活剂开启再生医学新赛道。从乳腺癌到白血病，从罕见病到抑郁症，从晚期肿瘤到组织修复，PI3K/AKT/mTOR调控剂正在构建一个横跨疾病治疗与健康维护的完整生态。它的复兴，不仅让一个沉寂多年的靶点重新焕发生机，更证明了基础研究与药物创新的深度融合，终将突破临床困境，为人类健康带来持续而深远的改变。在精准医疗的下一个十年，PI3K/AKT/mTOR通路注定会成为最耀眼的核心赛道之一，而这场始于科学、归于临床的复兴，才刚刚拉开序幕。【信号通路】肿瘤经典十二大信号通路，你关注哪个？

2026-02-04

·动脉网

瑞士生物技术公司获得两项出版物和FDA快速通道指定Two publications and an FDA fast track designation for Swiss biotechs

MoonLake Immunotherapeutics has received FDA Fast Track designation for the treatment of palmoplantar pustulosis with its novel IL-17 inhibitor, speeding up drug development. Meanwhile, Alys Pharmaceuticals and iOnctura both published high-impact papers confirming the potential of their lead assets.. MoonLake Immunotherapeutics公司已获得FDA快速通道资格，用于其新型IL-17抑制剂治疗掌跖脓疱病，加速了药物开发。同时，Alys Pharmaceuticals和iOnctura均发表了高影响力论文，证实了各自核心资产的潜力。MoonLake Immunotherapeutics 月湖免疫治疗公司, a clinical-stage biopharmaceutical company focused on developing advanced therapies for inflammatory diseases, announced that its lead asset, sonelokimab, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for the treatment of moderate to severe palmoplantar pustulosis (PPP), a chronic inflammatory skin disease. ，一家专注于开发炎症性疾病先进疗法的临床阶段生物制药公司，宣布其主要资产 sonelokimab 已获得美国食品药品监督管理局（FDA）快速通道资格，用于治疗中度至重度掌跖脓疱病（PPP），一种慢性炎症性皮肤病。The designation was granted following positive results from the Phase 2 LEDA trial. FDA Fast Track is intended to facilitate the development and expedite the review of new therapies for serious conditions where there is an unmet medical need.. 该指定是在第二阶段LEDA试验取得积极结果后授予的。FDA快速通道旨在促进严重疾病新疗法的开发，并加速对未满足医疗需求的疗法的审查。Sonelokimab (M1095) is an investigational 40 kDa humanized Nanobody® consisting of three heavy-chain-only antibodies covalently linked by flexible glycine-serine spacers, able to inhibit interleukin-17 (IL-17), a major inflammatory driver. Nanobodies® represent a new generation of antibody-derived targeted therapies. Sonelokimab（M1095）是一种研究中的40 kDa人源化纳米抗体®，由三个仅含重链的抗体通过柔性的甘氨酸-丝氨酸间隔区共价连接而成，能够抑制白细胞介素-17（IL-17），这是一种主要的炎症驱动因子。纳米抗体®代表了新一代的抗体衍生靶向疗法。They have several advantages over traditional antibodies, including their small size, enhanced tissue penetration, resistance to temperature changes, ease of manufacturing, and their ability to be designed into multivalent therapeutic molecules with bespoke target combinations.. 它们相比传统抗体有几个优势，包括体积小、组织穿透力强、抗温度变化、易于制造，以及能够被设计成具有定制靶点组合的多价治疗分子。Two high-impact papers published 两篇高影响力的论文发表Alys Pharmaceuticals 艾丽斯制药公司, which is focused on immuno-dermatology, has announced a recent publication in ，该公司专注于免疫皮肤病学，宣布了最近在某个刊物上的发表。Nature Communications 自然通讯featuring encouraging data from their lead asset ALY-101, a small interfering RNA (siRNA)-lipid conjugate. The paper demonstrates that the siRNA remains durable in the skin for over two months, highlighting its strong therapeutic potential. ALY-101 is currently in Phase 2a clinical trials for the treatment of patchy alopecia areata, a chronic autoimmune disease that causes hair to fall out.. 主打产品ALY-101（一种小干扰RNA（siRNA）-脂质结合物）的鼓舞人心的数据。本文展示了该siRNA在皮肤中能持续两个月以上，突显了其强大的治疗潜力。ALY-101目前正处于治疗斑块型斑秃的2a期临床试验阶段，斑秃是一种导致脱发的慢性自身免疫性疾病。Dedicated to combating neglected and hard-to-treat cancers, 致力于对抗被忽视和难以治疗的癌症，iOnctura 离子肿瘤学is developing new treatments with precision oral small molecules that target cancers in novel ways. The company recently published encouraging results on their lead asset roginolisib, a novel allosteric modulator of PI3Kδ in 正在开发以精确口腔小分子治疗癌症的新方法。该公司最近发布了其主要资产 roginolisib（一种新型的 PI3Kδ 别构调节剂）的鼓舞人心的结果。Molecular Oncology 分子肿瘤学. The paper provides an in‑depth look at the unique biology and differentiated mechanism of roginolisib, reinforcing its potential to deliver meaningful anti‑tumor activity without the immune‑related toxicities traditionally associated with PI3Kδ inhibition. 本文深入探讨了roginolisib的独特生物学特性及其差异化作用机制，进一步证实其在不产生与PI3Kδ抑制相关的免疫毒性的情况下，具有显著抗肿瘤活性的潜力。Roginolisib is now being evaluated across a broad clinical program, including a randomized Phase II study in uveal melanoma (UM), randomized Phase I/II trials in non‑small cell lung cancer (NSCLC) and CLL, and additional Phase I/II studies in myelofibrosis and peripheral T‑cell lymphoma (PTCL). More than 170 patients have been treated so far.. Roginolisib 目前正在广泛的临床项目中进行评估，包括在葡萄膜黑色素瘤 (UM) 中的随机 II 期研究、在非小细胞肺癌 (NSCLC) 和慢性淋巴细胞白血病 (CLL) 中的随机 I/II 期试验，以及在骨髓纤维化和外周 T 细胞淋巴瘤 (PTCL) 中的其他 I/II 期研究。迄今为止，已有超过 170 名患者接受了治疗。(Press release/AJ) （新闻稿/AJ）0 0Comments 评论Back to all news 返回所有新闻 Please 请login 登录or 或sign up 注册to comment. 评论。Commenting guidelines 评论指南 Send 发送

快速通道siRNA

2026-01-09

·PRNewswire

iOnctura CEO to present at the 2026 J.P. Morgan Healthcare Conference

Participation underscores growing excitement around company's momentum and potential in precision oncology GENEVA, AMSTERDAM and CAMBRIDGE, Mass., Jan. 9, 2026 /PRNewswire/ -- iOnctura, a clinical-stage biopharmaceutical company developing precision oral small molecules for neglected and hard-to-treat cancers, today announces that its Chief Executive Officer, Catherine Pickering, will present at the 44th Annual J.P. Morgan Healthcare Conference, taking place January 12–15, 2026, in San Francisco, California. Dr. Pickering will present on Thursday, January 15, 2026 at 11:00 PST. The presentation will highlight iOnctura's clinical progress with a focus on its lead program, roginolisib, the first allosteric modulator of PI3Kδ. Recognized as the world's largest and most influential healthcare investment conference, the J.P. Morgan Healthcare Conference is an invite-only event that serves as a vital forum for innovation and capital formation across the pharmaceutical and biotechnology sectors. Participation reflects iOnctura's growing visibility within the global oncology ecosystem and underscores the company's progress towards advancing transformative targeted therapies for patients with cancer. "We are honored to be invited to present at this year's J.P. Morgan Healthcare Conference," said Catherine Pickering, CEO and co-founder of iOnctura. "This recognition reflects the increasing interest in our science-driven approach and the meaningful clinical advances we have achieved with roginolisib. We look forward to sharing our progress and engaging with partners committed to transforming cancer care." Members of the management team will also be available for one-on-one meetings with investors and partners during the conference. For more information contact: Corporate Press Office: [email protected] About iOnctura iOnctura is a clinical-stage precision oncology company combating neglected and hard-to-treat cancers with a pipeline of first-in-class small molecules. The bold new treatments extend lives and improve healthspans, changing the outlook for patients and their families. Lead asset, roginolisib, is a non-ATP competitive, allosteric modulator of PI3Kδ with a unique chemical structure and binding mode. Allosteric modulation is a new archetype for precise inhibition of PI3Kδ, promising clinical activity without the detrimental tolerability seen with previous generations of inhibitors. Roginolisib is being investigated in multiple randomized Phase II studies in solid and hematological malignancies. iOnctura is headquartered in Amsterdam, The Netherlands with subsidiaries located in Geneva, Switzerland and Cambridge, MA, USA. iOnctura is backed by specialist institutional investors including Syncona, M Ventures, Inkef Capital, EIC Fund, VI Partners, Schroders Capital and XGEN Venture. About roginolisib Roginolisib is a first-in-class, non-ATP competitive, allosteric modulator of PI3Kδ with a unique chemical structure and binding mode. Allosteric modulation is a new archetype for precise inhibition of PI3Kδ, promising clinical activity without the detrimental tolerability seen with previous generations of inhibitors. The PI3K signaling pathway is one of the most commonly dysregulated pathways across multiple cancer types. The potential of roginolisib has been validated by positive clinical signals in Phase I in solid tumor and hematological malignancies, including a doubling of overall survival compared to historical controls in rare eye cancer, uveal melanoma. The company has carefully designed its clinical program to allow full development in uveal melanoma, while in parallel validating the program in larger market indications. The Phase II OCULE-01 study in uveal melanoma started in March 2025, the PULMO-01 study in non-small cell lung cancer (NSCLC) started in May 2025 and the HEMA-MED Phase I/II study in myelofibrosis began in November 2025. Logo : SOURCE iOnctura 21% more press release views with Request a Demo

临床2期临床1期

100 项与 Roginolisib 相关的药物交易

登录后查看更多信息

研发状态

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
原发性血小板增多症后骨髓纤维化	临床2期	意大利	iOnctura SA	2025-11-17
原发性血小板增多症后骨髓纤维化	临床2期	西班牙	iOnctura SA	2025-11-17
原发性血小板增多症后骨髓纤维化	临床2期	英国	iOnctura SA	2025-11-17
真性红细胞增多症后骨髓纤维化	临床2期	意大利	iOnctura SA	2025-11-17
真性红细胞增多症后骨髓纤维化	临床2期	西班牙	iOnctura SA	2025-11-17
真性红细胞增多症后骨髓纤维化	临床2期	英国	iOnctura SA	2025-11-17
外周T细胞淋巴瘤	临床2期	法国	iOnctura SA	2025-08-20
晚期鳞状非小细胞肺癌	临床2期	比利时	iOnctura SA	2025-04-22
晚期鳞状非小细胞肺癌	临床2期	意大利	iOnctura SA	2025-04-22
晚期鳞状非小细胞肺癌	临床2期	西班牙	iOnctura SA	2025-04-22

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


NCT07018752 (PlatΤform, ASH2025)	临床1/2期	外周T细胞淋巴瘤	49	Roginolisib	觸觸獵壓夢醖鬱範餘鹹(築網鏇淵壓衊鹹簾鹹範) = Golcadomide + Oral Azacitidine: neutropenia (67%, mainly grade 3) and thrombocytopenia (11%). Roginolisib: anemia (19%), thrombocytopenia (16%), and neutropenia (16%), with 1 grade 4 event (thrombocytopenia). 願鏇鏇淵廠繭壓醖願築 (積醖願蓋網觸繭壓膚窪 )	积极	2025-12-06
NCT06644183 (ASH2025)	临床1/2期	复发性慢性淋巴细胞白血病	28	Roginolisib + Venetoclax + Rituximab	製壓構艱簾夢鬱簾餘醖(艱願鑰顧糧窪願觸窪製) = 膚夢鏇窪醖齋壓窪膚繭築夢遞膚積積鏇壓醖獵 (鬱觸鬱鹽鏇製衊構淵製 )	积极	2025-12-06
NCT04328844 (DIONE-01, ESMO_IO2024) 人工标引	临床1期	葡萄膜黑色素瘤	29	Roginolisib	衊網遞積積鏇夢鹽夢淵(鏇鬱選繭觸獵夢壓遞鏇) = 構鏇簾築鹽壓鏇範構壓齋鹽築餘醖積蓋遞選網 (憲窪鹽鹹選膚選鹽網窪 ) 更多	积极	2024-12-12
NCT04328844 (Roginolisib (IOA-244), ESMO2024)	临床1期	葡萄膜黑色素瘤 CD63 expression on basophils \| blood-based proteins	29	Roginolisib 80 mg QD	鑰糧鑰夢憲齋築窪壓衊(積窪壓觸餘夢憲顧壓衊) = 願齋製觸窪糧鏇夢齋鹹憲鏇廠淵蓋憲獵遞遞鑰 (遞壓醖鹹齋獵衊遞築艱 )	积极	2024-09-14
NCT04328844 (FiH dose escalation study, ICML2023)	临床2期	滤泡性淋巴瘤	8	Roginolisib 80 mg QD	積襯醖膚範簾齋遞廠糧(餘餘顧觸憲鹽鹽廠繭網) = 獵繭範壓構淵膚齋窪選糧餘淵網選獵窪糧糧製 (醖製蓋鏇顧齋遞衊願鏇 ) 更多	积极	2023-06-09
EHA2023	N/A	难治性滤泡性淋巴瘤	8	Roginolisib 20 mg QD	顧艱遞構網廠構醖醖鏇(構製獵膚鬱製鹽衊積鬱) = AST/ALT elevation (G3) were observed in 2/8 pts 廠積糧觸範製壓襯淵選 (醖鬱願衊糧網鏇築築築 ) 更多	-	2023-06-08
EHA2023	N/A	难治性滤泡性淋巴瘤	8	Roginolisib 80 mg QD		-	2023-06-08
NCT04328844 (DIONE-01, ASCO 12023 \| ASCO 22023) 人工标引	临床1期	葡萄膜黑色素瘤 \| 晚期癌症	24	Roginolisib (Part A: pts with solid tumours and Follicular Lymphoma)	鏇觸襯窪襯範醖衊構壓(鏇夢壓夢鑰淵鹽窪艱繭) = roginolisib 80 mg QD 蓋齋觸顧蓋網築窪繭鹹 (餘觸膚衊窪築齋艱憲窪 ) 更多	积极	2023-05-26
NCT04328844 (DIONE-01, ASCO 12023 \| ASCO 22023) 人工标引	临床1期	葡萄膜黑色素瘤 \| 晚期癌症	24	Roginolisib (Part A(dose escalation): UM pts)		积极	2023-05-26
NCT04328844 (ASCO2022) 人工标引	临床1期	晚期癌症	23	IOA-244 (Part A Solid Tumor (completed))	淵蓋淵製鑰鏇觸醖鬱鹹(製衊窪憲憲鹽選襯遞觸) = 醖齋鬱醖鹽壓遞鬱簾獵夢築壓鏇鬱網衊艱膚獵 (壓選選顧襯襯鑰簾鬱製 )	积极	2022-06-02
NCT04328844 (ASCO2022) 人工标引	临床1期	晚期癌症	23	IOA-244 (Uveal Melanoma Pts (progressed ≥1 line prior therapy))	憲網製積鏇網壓獵壓構(壓艱構夢鹹醖鏇鬱壓積) = 網廠蓋齋夢鬱壓窪膚醖夢範願繭製製衊構憲構 (憲鹹餘網醖窪繭醖構鹽 ) 更多	积极	2022-06-02