NG-350A

NG-350A is a next generation, systemically administered immunotherapy designed to drive intratumoral expression of a CD40 agonist monoclonal antibody in both primary and metastatic tumor sites Proof-of-concept FORTRESS trial combines NG-350A with standard-of-care chemoradiotherapy in adult patients with locally advanced rectal cancer and at least one risk factor for local or distant recurrence CAMBRIDGE, MA, USA I April 03, 2025 I Akamis Bio, a clinical-stage oncology company using a proprietary Tumor-Specific Immuno-Gene Therapy (T-SIGn®) platform to deliver novel immunotherapeutic payloads to solid tumors, today announced enrollment of the first patient in the proof-of-concept FORTRESS study of NG-350A in locally advanced rectal cancer (LARC). FORTRESS is a multi-center, open-label, non-randomized, Phase 1b study designed to measure clinical complete response (cCR) rates in patients with LARC. The study builds upon the Akamis Bio-supported, CEDAR study, which showed a significantly greater complete response rate in LARC patients treated with a combination of Akamis Bio’s first generation immunotherapy and chemoradiotherapy (CRT), relative to expected outcomes using standard-of-care CRT alone. Prior clinical studies of NG-350A have demonstrated a favorable safety profile for the therapy, with no observed transgene-related or off-target toxicities. “We have previously demonstrated that intravenously administered T-SIGn® therapeutics can reach both primary and metastatic tumor sites to drive local expression of immunotherapeutic payloads,” said Oliver Rosen, MD, Akamis Bio’s chief medical officer. “The results from prior clinical studies have provided what we believe is a clear roadmap for the design of the FORTRESS trial, where our aim is to demonstrate the safety and efficacy of NG-350A in LARC in order to advance a new therapeutic approach that can improve the current standard of care for patients living with this disease.” About the FORTRESS Trial The Phase 1b FORTRESS trial ( NCT06459869 ) is an open-label, single-arm, and multicenter trial of NG-350A in combination with chemoradiotherapy (CRT) in adult patients with locally advanced rectal cancer (LARC) and at least one risk factor for local or distant recurrence or with oligometastatic disease. The study is planning to enroll approximately 30 patients aged eighteen and older with histologically confirmed adenocarcinoma of the rectum which is locally advanced (clinical stage II-III based on pelvic MRI). During the 12-week active study treatment period, patients will receive NG-350A plus CRT (oral capecitabine plus long-course intensity-modulated radiotherapy). The primary endpoint for the study will be the proportion of patients achieving a clinical complete response (cCR) at week 12. Key secondary endpoints will include the incidence and severity of adverse events, clinical response (CR) outcome, and MRI-based tumor regression grade (mrTRG). About NG-350A NG-350A is a clinical-stage, intravenously delivered T-SIGn® therapeutic designed to drive intratumoral expression of a CD40 agonist monoclonal antibody triggering the activation of antigen-presenting cells (APCs) resident in solid tumors and their draining lymph nodes. Once activated, APCs recruit T cells into the vicinity of the tumor to deliver a potent anti-tumor immune response. Akamis Bio has evaluated NG-350A’s safety, tolerability, and preliminary efficacy as a monotherapy (FORTITUDE study) and in combination with pembrolizumab (FORTIFY study) in patients with metastatic or advanced epithelial tumors. Across these studies, NG-350A has demonstrated a consistent safety and tolerability profile, as well as strong evidence of tumor-selective delivery, replication and transgene expression. About LARC Colorectal cancer is the third most common cancer diagnosed in both men and women in the United States with about 145,000 people newly diagnosed each year. Amongst the incident colorectal cancer population, about 45,000 people are diagnosed specifically with rectal cancer of which approximately 60 percent have locally advanced rectal cancer (LARC). LARC is defined by the spread of the rectal cancer to nearby tissues or lymph nodes. In patients with LARC, tumors have either grown through muscle and into the outermost layers of the rectum, or in more severe cases, through the wall of the rectum where they may attach to other organs or structures and/or into the lymph nodes. About T-SIGn ® Akamis Bio’s T-SIGn® therapeutics are based on a replication competent, chimeric group B adenovirus backbone which has been adapted via directed evolution to home specifically to both primary and metastatic epithelial-derived solid tumor tissue following intravenous delivery. Once at the tumor site, T-SIGn® therapeutics can drive the intratumoral expression of multiple transgene payloads, turning solid tumor cells into “drug factories” while leaving healthy tissue unaltered and intact. The intratumoral expression of immunologically active biomolecules and therapeutic proteins can result in the remodeling of the solid tumor microenvironment, triggering robust antitumor immune responses. T-SIGn® therapeutics have the potential to be used in the monotherapy setting, as well as in combination with other immuno-oncology agents to target the key mechanisms that tumors use to evade the immune system. About Akamis Bio Headquartered in Cambridge, Massachusetts, Akamis Bio is a clinical-stage oncology company developing systemically administered immunotherapies to treat solid tumors, initially in patients with locally advanced rectal cancer (LARC). Its proprietary Tumor-Specific Immuno-Gene Therapy (T-SIGn®) platform is designed to deliver novel immunotherapeutic proteins, biomolecules and transgene combinations to treat solid tumors, with its lead clinical-stage program, NG-350A, driving intratumoral expression of a CD40 agonist monoclonal antibody. To learn more, please visit www.akamisbio.com . SOURCE: Akamis Bio

▎药明康德内容团队编辑 据创鉴汇不完全统计，上周（12月16日至12月22日）全球大健康领域共披露融资事件27起，总额近48亿元。按照金额划分，亿元及以上融资11起。按照已披露的融资轮次划分，早期融资（B轮以前）13起，中后期融资（B轮及以后）7起。生物医药领域共12家公司获融资，涉及药物类型包括创新抗体、细胞基因疗法、RNA药物等。 #01 Ottimo Pharma完成超1.4亿美元A轮融资 关键词：双功能抗体药物 最新融资：超1.4亿美元A轮 本轮投资机构：Avoro Capital Advisors、Invus、J.P. Morgan Life Sciences Private Capital、Janus Henderson Investors等 12月20日，Ottimo Pharma公司今日宣布完成超过1.4亿美元的A轮融资。本次融资将加速其主打候选药物jankistomig的开发进程。Jankistomig是一款潜在“first-in-class”的PD-1/VEGFR2双功能性抗体，适用于多种实体瘤适应症，提供静脉输注（IV）和皮下注射（SC）两种形式。同时，融资获得资金还将用于推进后续双功能产品管线的开发。Jankistomig的设计不同于传统的双特异性抗体，其双臂中的任何一臂均可与PD-1或VEGFR2结合。它通过靶向免疫检查点蛋白和血管生成，为癌症治疗提供解决方案。这一创新方法旨在提供更广泛的治疗窗口，不受肿瘤VEGF水平的限制，有潜力改善多种实体瘤适应症的治疗结果。目前，jankistomig正在进行支持IND的临床前研究，计划于2025年底提交IND申请。 #02 Akamis Bio完成6000万美元融资 关键词：抗肿瘤基因疗法 最新融资：6000万美元A轮 本轮投资机构：Sedgwick Yard 近日，Akamis Bio完成6000万美元A轮融资，将推动Akamis Bio的NG-350A项目，该项目是针对晚期直肠癌患者的一种免疫肿瘤基因治疗，目前正在进行1期临床试验。NG-350A是一种静脉内递送的转基因武装肿瘤基因疗法，旨在推动CD40激动剂单克隆抗体在原发性和转移性上皮源性实体瘤的瘤内表达。Akamis Bio利用其专有的T-SIGn治疗平台，递送免疫治疗蛋白及生物分子，使患者自身的免疫系统能够识别并清除癌症。  #03 Tessera Therapeutics获得5000万美元投资 关键词：基因疗法 最新融资：5000万美元未知轮 本轮投资机构：比尔及梅琳达·盖茨基金会 12月18日消息，Tessera Therapeutics公司宣布与比尔及梅琳达·盖茨基金会达成协议，共同资助Tessera的镰状细胞病（SCD）体内治疗项目。盖茨基金会的资助包括初期投资，并有可能追加投资至最高5000万美元，旨在支持Tessera的SCD项目进入临床阶段，并帮助其实现开发全球可及的变革性基因药物的目标。Tessera正在开发用于SCD的基因书写疗法，旨在通过单次静脉注射在体内直接纠正导致SCD的基因突变，而无需进行复杂的干细胞动员或有毒的化疗预处理。此外，Tessera还利用其专有的非病毒递送平台开发了脂质纳米颗粒，以确保基因书写疗法能够精准递送到长期造血干细胞中。 #04 Borealis Biosciences获得3000万美元A轮融资 关键词：肾脏疾病RNA药物 最新融资：3000万美元A轮 本轮投资机构：Westlake Village BioPartners 12月19日，Borealis Biosciences宣布额外获得了3000万美元A轮融资。Borealis旨在通过开发RNA疗法促进科学和转化上的突破，以满足肾病患者的主要未满足需求。该公司的目标包括更好地了解患者分层、遗传学定义的治疗靶标，并致力将治疗有效载荷递送到特定肾细胞类型以及推进RNA化学的进展。该公司建立于Chinook Therapeutics成功的基础上。Chinook是一家肾病公司，由Versant于2019年创立，去年被诺华以高达35亿美元的款项收购。Borealis研究的核心包含来自Chinook的科学与学术团队，此外还有RNA治疗领域的其他专家。 #05 Indapta Therapeutics完成2250万美元融资 关键词：NK细胞疗法 最新融资：2250万美元未知轮 本轮投资机构：Leaps by Bayer、Myeloma Investment Fund、Pontifax Venture Capital、RA Capital Management、祥峰投资 近日，Indapta Therapeutics公司宣布筹集了2250万美元以加速其差异化的同种异体自然杀伤（NK）细胞疗法的临床开发。该公司于近期公布了其同种异体NK细胞疗法IDP-023治疗晚期多发性骨髓瘤或非霍奇金淋巴瘤患者的1/2期临床试验结果。患者接受了1到3剂IDP-023、伴或不伴IL-2治疗。结果显示，在9名接受治疗的患者中，有5名患者观察到了客观缓解。此外，IDP-023联合CD20或CD38靶向单克隆抗体的队列招募正在进行中。 读者们请星标⭐创鉴汇，第一时间收到推送 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：本文由药明康德内容团队根据公开资料整理编辑，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发/复制至其他平台。转发授权请在「创鉴汇」微信公众号留言联系我们。 更多数据内容推荐 点击“在看”，分享创鉴汇健康新动态