VENT-02

▎药明康德内容团队编辑本期看点1. 再生细胞疗法NRTX-1001用于治疗癫痫能够持久减少患者的癫痫发作，2名患者分别在接受治疗后的16个月和21个月内，癫痫发作较基线减少＞95。2. 新型口服脑渗透性NLRP3抑制剂VENT-02的早期临床数据积极，对血液中导致炎症反应的关键因素IL-1ß有100%的抑制作用。3. 治疗天使综合征（Angelman syndrome）的反义寡核苷酸疗法GTX-102在早期临床试验中表现积极，在第170天显示出快速且具有临床意义的多领域功能改善。图片来源：123RFNRTX-1001：公布1/2期临床试验的新数据Neurona Therapeutics公司公布了其候选再生细胞疗法NRTX-1001的1/2期临床试验的新数据。NRTX-1001是一种来源于人类多能干细胞的再生神经细胞治疗候选药物，用于治疗癫痫。这些完全分化的中间神经元分泌抑制性神经递质γ-氨基丁酸（GABA）。一次给药之后，这些人类中间神经元可在大脑的癫痫区域实现靶向整合并持久地抑制癫痫活动。接受NRTX-1001一次性治疗后患者需在第一年接受免疫抑制，目的是促进NRTX-1001的整合和长期持久性。此次公布的结果显示，队列1接受低剂量NRTX-1001治疗的5名受试者癫痫发作的中位数比基线减少了75%，在治疗后的六个月内，5名受试者中有4名报告癫痫发作比基线减少>50%。两名受试者在接受NRTX-1001治疗后分别接受了16个月和21个月的随访，结果显示，癫痫发作较基线持续减少＞95%，生活质量测试评分也有所提高。截至目前，所有接受治疗的受试者对NRTX-1001的耐受性都很好，神经心理学测试表明没有发现神经认知障碍。该试验中队列2的前两名受试者接受了更高剂量的NRTX-1001，目前尚未报告任何严重不良事件。VENT-02：公布1期临床试验数据Ventus Therapeutics公司公布了其新型口服脑渗透性NLRP3抑制剂VENT-02的1期临床试验结果。该1期临床试验评估了VENT-02在成年健康受试者中的药效学、药代动力学、安全性和耐受性。NLRP3炎症小体的异常激活与纤维化、皮肤病和风湿病等全身性疾病有关，也是帕金森病、阿尔茨海默病和难治性癫痫等多种神经系统疾病病理变化的关键驱动因素。VENT-02对NLRP3的治疗性抑制可阻止NLRP3炎症小体的形成，进而抑制IL-1β和IL-18的产生和细胞焦亡。此次公布的结果显示，VENT-02对血液中的IL-1ß有100%的抑制作用，24小时内脑脊液中的药物含量显著降低，炎症生物标志物（如高敏C反应蛋白[hsCRP]）也明显减少，从而证明了VENT-02对靶点的全面作用。根据试验中观察到的半衰期和靶点覆盖率，VENT-02已被证明具有每日给药一次的潜力。在这项1期临床试验中，VENT-02的耐受性良好，没有报告剂量限制性毒性或严重不良反应，仅观察到轻度或中度与治疗相关的不良反应。中度不良反应包括头痛和恶心，只有在剂量高于预期治疗剂量数倍时才会出现。该公司预计将于2024年下半年开展针对中枢神经系统疾病的临床试验。GTX-102：公布1/2期临床试验的中期数据Ultragenyx Pharmaceutical公司公布了其用于治疗天使综合征的在研疗法GTX-102在1/2期临床试验中获得的最新数据。天使综合征是一种由UBE3A基因缺失或突变所造成的神经系统发育障碍疾病，主要疾病特征包括发育迟缓、智力残疾、言语及平衡障碍。GTX-102是一种反义寡核苷酸疗法，可以促进神经元细胞中父系UBE3A的等位基因表达，产生患者体内所缺失的关键蛋白产物。此次公布的数据分析显示，接受GTX-102治疗的扩展队列A和B的患者，在第170天显示出快速且具有临床意义的多领域功能改善。此外，部分剂量递增队列患者在第758天显示出长期和持续的临床益处，远超过自然历史数据。基于这些数据，该公司计划启动关键性3期临床试验。AB-1005（AAV2-GDNF）：公布1b期临床试验的新数据拜耳（Bayer）公司旗下Asklepios BioPharmaceutical（AskBio）公司公布了其治疗帕金森病的在研基因疗法AB-1005的1b期临床试验结果。AB-1005是一种基于腺相关病毒血清型2（AAV2）载体的在研基因疗法，含有编码人胶质细胞源性神经营养因子（GDNF）的转基因。通过磁共振成像（MRI）监测的对流增强给药方式直接进行神经外科注射后，GDNF可以在大脑局部区域实现稳定和连续的表达。此次公布的结果显示，接受AB-1005治疗后的第18个月时，所有11名患者均耐受良好，未发生严重不良事件，达到试验的主要目标。基于这一结果，两家公司已经启动了一项2期临床试验，预计将于今年晚些时候开始在美国、欧盟和英国招募患者。BB-301：公布1b/2a期临床试验中首位患者的数据Benitec Biopharma公司公布了其开发的用于治疗眼咽肌营养不良（Oculopharyngeal Muscular Dystrophy）的沉默和取代基因疗法BB-301在早期临床试验中的首位患者的数据。该疗法利用一种新型的、经过改良的AAV9载体，表达一种独特的、单一的双功能构建体，从而能够促进密码子优化的PABPN1基因和两个针对PABPN1突变体的siRNA的共同表达。这两个siRNA被塑造成microRNA骨架，以抑制有缺陷的PABPN1突变体的表达，同时允许表达经过密码子优化的PABPN1基因，用PABPN1蛋白的功能性版本取代突变体。此次公布的数据显示，与该研究在自然史观察导入期进行的治疗前评估相比，接受BB-301治疗后第90天时，该研究中首个患者的多种吞咽功能指标均获得了改善。安全性方面，未在接受BB-301治疗的两名受试者中观察到严重不良事件。两名受试者出现了短暂的2级胃食管反流病（即“反酸”或“烧心”）。这两名受试者在短期服用了经批准用于治疗胃食管反流病的普通处方药后，胃食管反流病都得到了缓解。▲接受BB-301治疗后第90天时，患者在所有临床结局上都有改善（图片来源：参考资料[16]）Botensilimab：公布1期临床试验的新数据Agenus公司公布了其在微卫星稳定性（MSS）或错配修复缺陷型（dMMR）转移性结直肠癌（CRC）患者中开展的1期临床试验的积极结果。Botensilimab是一种在研新型多功能CTLA-4抗体，旨在将临床获益扩展到既往对标准治疗或在研疗法无反应的“冷”肿瘤。除与CTLA-4受体结合外，其Fc增强的结构能够诱导记忆免疫反应，下调调节性T细胞，并使T细胞更好地启动和活化，从而放大免疫反应。在一项超过300例患者的1b期临床研究中，botensilimab已经在9种实体肿瘤中证明了其临床应答，无论是单独使用还是与巴替利单抗联合使用。截至2024年3月1日的数据，在对77例无活动性肝转移的难治性MSS-CRC患者进行的1期试验中，中位随访时长为13.6个月，观察到的总缓解率为23%。18例获得缓解患者的中位缓解持续时间尚未达到。预计12个月和18个月的总生存率分别为71%和62%，中位总生存期为21.2个月。目前，botensilimab的2期试验已完成注册，该公司计划于2024年下半年展示详细的2期临床试验的疗效结果。WAL0921：公布1+期临床试验数据Walden Biosciences公司公布了其人源化单克隆抗体WAL0921针对健康受试者的1+期临床试验的结果。WAL0921能够结合循环游离可溶性尿激酶纤溶酶原激活受体（suPAR）及其膜结合形式uPAR，并抑制其导致肾脏疾病的病理活性。此次公布的结果显示，在40名健康受试者中，WAL0921的安全性和耐受性良好。此外，WAL0921能够快速、剂量依赖性地降低循环游离的suPAR水平。VTP-200：公布1b/2期临床试验数据Barinthus Biotherapeutics公司公布了其在研免疫治疗联合方案VTP-200用于治疗持续性高危HPV感染和产生相关癌前病变的患者的早期临床试验数据。VTP-200是一种需要接种两次的HPV疫苗，首次接种使用ChAdOx载体，第二次接种使用MVA载体，两者都编码相同的HPV抗原，旨在激发针对HPV的抗原特异性T细胞免疫反应。此次公布的结果显示，该研究达到了主要安全性终点。VTP-200总体耐受性良好，没有发生治疗相关的≥3级的不良事件或严重不良事件。在最高剂量的ChAdOx-HPV给药组（第2组）中观察到，高危（hr）HPV清除率为60%，宫颈病变清除率为67%，呈积极趋势。不过，来自五个活性剂量组的汇总数据显示，治疗组在hrHPV清除率或宫颈病变清除率上与安慰剂组相比，差异无统计学意义。SLATEv1：公布1/2期临床试验的中期数据Gritstone bio公司公布了其“现货型”新抗原疫苗SLATEv1联合免疫检查点抑制剂晚期转移性实体瘤的1期临床试验的中期结果。SLATEv1能够提供一组癌症患者共享的20种新抗原，包括多种癌基因（KRAS、TP53、BRAF和CTNNB1）中的多个复发突变，有望驱动T细胞反应。此次公布的结果显示，该联合疗法具有良好的耐受性，观察到的治疗相关不良事件大多数为1/2级，与基于病毒载体的疫苗和免疫检查点抑制剂的预期急性炎症一致。2例患者出现3/4级严重治疗相关不良事件，这些不良事件也是剂量限制性毒性。患者的总缓解率为0%，中位无进展生存期和总生存期分别为1.9个月和7.9个月。相对于患者肿瘤表达的KRAS新抗原，T细胞反应偏向于疫苗中编码的人类白细胞抗原匹配的TP53新抗原，这表明以前未知的新抗原免疫显性层次结构可能会影响多表位共享新抗原疫苗的治疗效果。基于这些结果，Gritstone bio公司开发了一种专门针对KRAS衍生的新抗原的优化疫苗，该疫苗正在2期临床研究中进行评估。大家都在看药明康德为全球生物医药行业提供一体化、端到端的新药研发和生产服务，服务范围涵盖化学药研发和生产、生物学研究、临床前测试和临床试验研发、细胞及基因疗法研发、测试和生产等领域。如您有相关业务需求，欢迎点击下方图片填写具体信息。▲如您有任何业务需求，请长按扫描上方二维码，或点击文末“阅读原文/Read more”，即可访问业务对接平台，填写业务需求信息▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放参考资料（可上下滑动查看）[1] AskBio presents 18-month Phase Ib trial results of AB-1005 gene therapy for patients with Parkinson’s disease. 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Retrieved April 19, 2024, from https://www.prnewswire.com/news-releases/aspen-neuroscience-announces-first-patient-dosed-in-first-in-human-phase-12a-clinical-trial-of-autologous-neuronal-cell-replacement-therapy-for-parkinsons-disease-302119216.html[24] AviadoBio Announces First Patient Treated in ASPIRE-FTD Clinical Trial Evaluating AVB-101 for Frontotemporal Dementia with GRN Mutations. Retrieved April 19, 2024, from https://www.businesswire.com/news/home/20240415509017/en免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。分享，点赞，在看，聚焦全球生物医药健康创新

VENT-02 demonstrated robust pharmacodynamic modulation, a broad therapeutic index, and potential for once-daily dosing VENT-02 provided full target coverage with a single dose No dose-limiting toxicities or serious adverse events observed Initiation of clinical trials in CNS diseases expected beginning in the second half of 2024 WALTHAM, Mass. & MONTREAL--(BUSINESS WIRE)-- Ventus Therapeutics, a clinical-stage biopharmaceutical company utilizing its proprietary structural biology and computational chemistry platform, ReSOLVE™, to develop differentiated small molecule therapeutics, today announced results from its Phase 1 clinical trial of VENT-02, a novel, oral, brain-penetrant NLRP3 inhibitor. The Phase 1 trial evaluated the pharmacodynamics, pharmacokinetics, safety, and tolerability of VENT-02 across a broad range of single and multiple ascending doses in adult healthy volunteers. “We are pleased with the results of this trial in which VENT-02 demonstrated a broad therapeutic index and complete target coverage, underscoring its potential to treat diseases of the CNS as well as peripheral diseases with high unmet needs,” said Xavier Valencia, M.D., Head of Clinical Development at Ventus. “Our next trial for VENT-02, a Phase 1b trial in patients with Parkinson’s disease, will evaluate multiple doses and dose regimens against a placebo control and assess a collection of markers that map multiple components of inflammation to disease activity. Based on the promising pharmacodynamic and safety data from this Phase 1 trial and our clinical development plans, we believe VENT-02 is poised to demonstrate best-in-class potential.” In this Phase 1 clinical trial, VENT-02 was well-tolerated, with no dose-limiting toxicities or serious adverse events reported and only mild or moderate treatment-related adverse events observed. The moderate adverse events included headache and nausea and were only observed at a dose multiple times higher than the intended therapeutic doses. VENT-02 demonstrated full target engagement through 100% inhibition of IL-1ß in the blood in an ex vivo whole blood challenge assay, significant drug levels in the CSF for 24 hours, and robust reduction of inflammatory biomarkers such as hsCRP. Based on the half-life and target coverage observed in the trial, VENT-02 has demonstrated the potential for once-daily dosing. “The excellent properties of VENT-02 are a reflection of Ventus’ industry-leading exploration of NLRP3, including demonstrating proof-of-concept in preclinical studies in multiple disease-relevant models, validating biomarkers, and collaborating with academic institutions and the Michael J. Fox Foundation,” said Ventus President and CEO, Marcelo Bigal, M.D., Ph.D. “As a result, we are ready to bring VENT-02 into Parkinson’s disease and other pre-defined diseases where the CNS plays an important role, capitalizing on innovative trials that we are crafting to be informative in reducing risk in the future steps of clinical development. In addition, we expect that our planned trials will allow us to explore the potential impact of NLRP3 inhibition across multiple patient populations suffering from serious diseases.” Ventus expects to initiate a Phase 1b trial of VENT-02 in patients with Parkinson’s disease in the second half of 2024 and a Phase 2 trial in patients with treatment-refractory epilepsy in 2025. About NLRP3 NLRP3 is the best understood member of a family of proteins known as inflammasomes. Inflammasomes are multiprotein complexes that regulate the innate immune system and are involved in intracellular surveillance of danger and pathogen signals that trigger an intense inflammatory response, including the release of IL-1β and IL-18 and the induction of pyroptosis, an inflammatory form of cell death. Therapeutic inhibition of NLRP3 can prevent the formation of the NLRP3 inflammasome, which in turn inhibits the production of IL-1β and IL-18 as well as pyroptosis. Aberrant activation of the NLRP3 inflammasome has been associated with systemic conditions, including fibrotic, dermatological, and rheumatological diseases, and is a key driver of disease pathology in several neurological disorders, including Parkinson’s disease, Alzheimer’s disease, and treatment-refractory epilepsy. About Ventus Therapeutics Ventus Therapeutics is a clinical-stage biopharmaceutical company deploying deep protein science expertise and a proprietary computational chemistry platform to develop novel small molecule therapeutics for immunology, inflammation, and neurology disorders. Using its proprietary drug discovery platform, ReSOLVE™, the company screened its first target in 2020, selected three development candidates in 2022, and advanced its two wholly-owned product candidates into the clinic in 2023: VENT-03, a potent, selective, oral cGAS inhibitor in Phase 1, and VENT-02, a potent, brain-penetrant, oral NLRP3 inhibitor in Phase 1. In addition, Ventus has out-licensed VENT-01, a peripherally-restricted NLRP3 inhibitor, to Novo Nordisk A/S. These programs exemplify Ventus’ robust discovery capabilities and focus on differentiated programs for multi-indication immunology and neurology targets. For more information, please visit and engage with Ventus on LinkedIn. Forward-Looking Statements This press release contains forward-looking statements. These statements involve known and unknown risks, uncertainties, and other important factors that may cause Ventus’ actual results, performance, or achievements to be materially different from any future results, performance, or achievements expressed or implied by the forward-looking statements. In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “would,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “contemplates,” “believes,” “estimates,” “predicts,” “potential,” or “continue” or the negative of these terms or other similar expressions. All statements other than statements of historical facts contained in this press release are forward-looking statements, including statements regarding the timing, progress, and results of Ventus’ clinical trials, the anticipated timing of initiation and completion of trials, the period during which the results of the trials will become available, and Ventus’ development plans. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond Ventus’ control, you should not rely on these forward-looking statements as predictions of future events. Such risks include, but are not limited to, the risk that Ventus is unable to further advance VENT-02 and VENT-03 in clinical development, obtain regulatory approval, and ultimately commercialize VENT-02 and VENT-03, or experience significant delays in doing so; that Ventus will require substantial additional funding to finance its operations; that the development and commercialization of pharmaceutical products is subject to extensive regulation, and the regulatory approval processes of the U.S. Food and Drug Administration and comparable foreign authorities are lengthy, time-consuming, and inherently unpredictable; and that Ventus’ success depends on its, its current and future licensors’, and its exclusive licensees’ ability to obtain, maintain, enforce, and protect its intellectual property and its proprietary technologies. Forward-looking statements included herein are based upon information available to Ventus as of the date of this press release, and while Ventus believes such information forms a reasonable basis for such statements, such information may be limited or incomplete, and Ventus’ statements should not be read to indicate that it has conducted an exhaustive inquiry into, or review of, all potentially available relevant information. The events and circumstances reflected in the forward-looking statements may not be achieved or occur and actual results could differ materially from those projected in the forward-looking statements. Except as required by applicable law, Ventus does not plan to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances, or otherwise.