MISSION Therapeutics Ltd.

根据公开信息，上周（10月13日~10月19日），全球范围内有至少13家致力于创新药研发的新锐公司宣布完成新一轮融资。通过梳理，这些获得资本青睐的新锐公司正在开发的产品包括小分子、多肽、抗体偶联药物（ADC）、反义寡核苷酸（ASO）、细胞疗法等等。 BridGene Biosciences 融资轮次：B+轮 融资金额：2800万美元 10月16日，BridGene宣布已于近期完成2800万美元B+轮融资。本轮融资由康君资本领投，高特佳投资、比邻星创投以及现有投资方龙磐投资和磐谷创投共同参与。BridGene是一家处于临床阶段的小分子创新药生物科技公司，专注于开发针对传统“不可成药”靶点的新型疗法。该公司核心候选药物BGC-515是一款针对实体瘤的共价TEAD抑制剂，目前在1期临床，并计划于近期启动2期临床研究。此外，资金还将用于推动公司第二个管线项目于2027年进入临床阶段，并加速公司在肿瘤及自身免疫疾病领域的后续管线布局。 Veradermics 融资轮次：C轮 融资金额：1.5亿美元 10月16日，Veradermics宣布完成超额认购的1.5亿美元C轮融资。此次融资将用于推进其核心在研产品VDPHL01的注册性试验及预定的新药申请（NDA）提交。VDPHL01是一款专为男女脱发患者设计、潜在首款缓释口服minoxidil候选药物，目前正在进行3期试验。该公司同时公布了进行中2期临床研究的初步结果，该研究评估VDPHL01用于治疗男女模式性脱发（pattern hair loss）的疗效与安全性。主观评价方面，在4个月结束时，95%的受试者表示对头发覆盖度的满意度提高。 Peptilogics 融资轮次：7800万美元 融资金额：B2轮 10月16日，Peptilogics宣布完成一轮超额认购的7800万美元B2轮融资。Peptilogics是一家临床阶段生物技术公司，致力于开发变革性外科治疗药物，以有效治疗和预防严重的医疗器械感染。此笔资金将用于支持其研究性药物zaloganan (PLG0206) 治疗假体关节感染 (PJI) 的关键性2/3期试验。这是一款广谱、抗菌和抗生物膜肽治疗药物，通过独特的作用机制靶向并破坏细菌膜，可快速局部渗透生物膜并杀死隐藏的细菌。在1b期研究中，在清创、抗生素治疗和假体保留 (DAIR）手术期间使用zaloganan冲洗液，14 名患者中的13名 (93%) 在12个月时保持无感染状态。 Adcytherix 融资轮次：A轮 融资金额：1.05亿欧元 10月16日，Adcytherix宣布成功完成1.05亿欧元（约合1.22亿美元）A轮融资。该公司致力于设计和开发具有新型有效载荷的专有抗体偶联药物（ADC），本轮融资资金将用于将其主导候选药物ADCX-020推进至临床阶段，并计划于2025年底前提交IND申请。与此同时，Adcytherix将扩展其使用新型有效载荷的专有ADC管线。 Step Pharma 融资轮次：C轮 融资金额：3800万欧元 10月15日，Step Pharma宣布完成3800万欧元C轮融资。本轮融资将支持该公司就其主导资产 dencatistat在多个适应症中进行的持续临床开发。这是一种高选择性、具有口服生物利用度的CTPS1抑制剂，正在开发用于治疗癌症和血液疾病，目前已经启动了针对T或B细胞淋巴瘤、实体瘤、原发性血小板增多症等适应症的临床研究。研究表明，多种都高度依赖CTPS1（嘧啶合成途径的关键组成部分）进行DNA合成和细胞增殖。 Pelage Pharmaceuticals 融资轮次：B轮 融资金额：1.2亿美元 10月15日，Pelage Pharmaceuticals宣布完成1.2亿美元B轮融资。所获资金将用于推进公司核心项目PP405的持续开发。基于之前在雄激素性脱发研究中获得的2a期试验积极结果，公司计划于2026年推进PP405进入3期临床。PP405是一种新型的外用小分子，设计用于激活休眠毛囊干细胞，重启毛发生长。此前一项2期临床研究结果显示，接受PP405治疗且脱发程度更高的男性中，有31%达成发密度较基线提升超过20%的应答，而此数值在安慰剂组为0%。 Tubulis 融资轮次：C轮 融资金额：3.61亿美元 10月15日，Tubulis宣布成功完成一笔3.08亿欧元（约合3.61亿美元）的C轮融资。本轮融资所得将用于将Tubulis的主导ADC候选产品TUB-040的临床开发扩展到更早的治疗线和更多的肿瘤适应症。TUB-040靶向NaPi2b，这是一种在卵巢癌和肺腺癌中过度表达的抗原。TUB-040目前正在一项针对铂耐药卵巢癌以及复发或难治性非小细胞肺癌患者的1/2a期研究中进行评估，并于2024年6月获得美国FDA的快速通道资格。该笔资金还将推进Tubulis的其它管线，包括临床阶段靶向5T4的ADC候选药物TUB-030、多个临床前项目，并扩展其专有的ADC平台技术。 Omnix Medical 融资轮次：C轮 融资金额：2500万美元 10月15日，Omnix Medical宣布获得2500万美元C轮融资。Omnix是一家致力于开发用于治疗危及生命感染的下一代抗感染药物的生物医药公司，此次融资将使该公司能够完成其先导化合物OMN6的2期概念验证研究。这些研究将侧重于治疗由革兰氏阴性菌（特别是鲍曼不动杆菌）引起的危及生命的感染，包括对碳青霉烯类和粘菌素等最后防线治疗耐药的菌株。OMN6是一种潜在“first-in-class”抗菌肽，其设计旨在对细菌膜具有高选择性，其特异性地结合细菌膜独特的脂质成分，并迅速形成孔洞，破坏其稳定性并摧毁膜结构，导致细菌迅速死亡。该产品速的作用机制、工程化的稳定性以及避免酶降解的能力使得治疗周期更短，并最大限度地降低了耐药性发展的风险。 Mission Therapeutics 融资轮次：未披露 融资金额：1330万美元 10月15日，Mission Therapeutics宣布已筹集1330万美元，用于推进一项主导候选药物MTX325在帕金森病患者中进行的1b期机制验证研究。Mission是一家临床阶段生物技术公司，致力于开发通过增强线粒体自噬来促进细胞和器官健康的“first-in-class”疗法。MTX325是一种具有口服生物利用度和脑渗透性的USP30抑制剂，旨在改善线粒体质量，通过抑制USP30（一种线粒体去泛素化酶）发挥作用，从而增加线粒体泛素化并促进适当的线粒体自噬——这是细胞用来清除自身功能失调线粒体的基本过程。线粒体功能障碍是帕金森病病理生理学的一个主要假说。抑制USP30是一种经过充分验证的方法，可通过恢复线粒体自噬来实现帕金森病的神经保护。 Kardigan 融资轮次：B轮 融资金额：2.54亿美元 10月15日，Kardigan宣布完成2.54亿美元B轮融资。Kardigan致力于新型心血管药物的开发，以提供能使患者超越症状管理、实现功能性治愈的药物。本轮融资将用于持续推动针对特定类型扩张型心肌病、急性严重高血压和钙化性主动脉瓣狭窄的三个后期研发项目取得进展，包括：danicamtiv，一种针对由肌节变异驱动的遗传性扩张型心肌病的心脏肌球蛋白激活剂；tonlamarsen，一种血管紧张素原靶向反义寡核苷酸（ASO）疗法，拟用于中断急性严重高血压的危险循环；以及ataciguat，一种每日一次的口服可溶性鸟苷酸环化酶激活剂，拟开发作为钙化性主动脉瓣狭窄的潜在"积极监测"的替代方案。 Tr1X Bio 融资轮次：未披露 融资金额：5000万美元 10月14日，Tr1X Bio宣布美国FDA批准其治疗进展型多发性硬化的同种异体CAR-Tr1 Treg细胞疗法TRX319的IND申请，并获得额外5000万美元融资。Tr1X 是一家临床阶段生物医药公司，致力于开发具有治愈自身免疫和炎症性疾病潜力的同种异体工程化1型调节性T细胞（Tr1 Treg）和CAR-Tr1 Treg细胞疗法。TRX319旨在将靶向B细胞控制与主动抗炎信号传导和致病性T细胞调节相结合，并具有中枢神经系统渗透性。本轮融资还将助力Tr1X Bio另一款在研新药TRX103开展针对难治性克罗恩病患者的1/2a期临床试验。TRX103是一种在研同种异体即用型工程化T细胞产品，可以模拟1型调节性T细胞（Tr1）功能的细胞，称为TRX细胞。 Kailera Therapeutics 融资轮次：B轮 融资金额：6亿美元 10月14日，Kailera Therapeutics宣布完成6亿美元B轮融资。本轮融资由Bain Capital Private Equity领投，所获资金将用于推进其差异化的肥胖症治疗产品管线的开发。融资款项将主要用于支持其核心候选药物KAI-9531（一款注射用GLP-1/GIP双受体激动剂，具有潜在“best-in-category”的减重效果）在年底前启动全球3期临床试验。同时，资金也将用于推进其口服小分子GLP-1受体激动剂KAI-7535开展全球临床研究。该药在中国的2期临床试验中已表现出具有竞争力的减肥效果。该公司也将继续推进其早期项目，包括注射用GLP-1/GIP/胰高血糖素受体三激动剂KAI-4729，以及每日一次口服型KAI-9531。 Excellergy 融资轮次：A轮 融资金额：7000万美元 10月13日，Excellergy宣布正式启动并完成7000万美元的A轮融资，以推进其潜在“first-in-class”效应细胞反应抑制剂（ECRIs）产品组合。Excellergy是一家旨在开发新型过敏治疗药物的生物技术公司，其通过设计新型三功能ECRIs重新定义过敏治疗，旨在通过靶向效应细胞实现强效、快速缓解并完全控制过敏反应。这类新的ECRIs采用三功能作用机制：1）快速移除肥大细胞和嗜碱性粒细胞上结合FceRI的IgE，且不触发这些细胞的激活；2）提供强效、高亲和力的游离IgE中和作用；3）加速 FceRI表达的下调。这三个功能同步作用，与现有已批准疗法相比，有潜力在更广泛的患者群体中实现更快速的起效和更完全的过敏控制。该公司计划在2026年初将其主导资产推进至临床阶段。 期待在资本的助力下，更多前沿疗法和技术能够更快惠及患者。 参考资料： [1]各公司官网及公开资料 版权说明：本文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。转载授权或其他合作需求，请联系wuxi_media@wuxiapptec.com。 免责声明：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。

Mission Therapeutics, a clinical-stage biotechnology company, has raised $13.3m to advance the clinical development of its lead Parkinson’s disease (PD) candidate, MTX325, into a phase 1b proof-of-mechanism study. The clinical development of MTX 325 is further supported by a $5.2m grant from the Michael J. Fox Foundation and Parkinson’s UK. MTX325 is a first-in-class USP30 inhibitor that targets mitochondrial dysfunction, which has been linked to PD pathophysiology. Supporting preclinical data, published in Nature Communications in November 2023, and authored by Mission Therapeutics scientists alongside researchers from Cambridge University, Harvard University and the University of Dundee, demonstrated that USP30 inhibition reduced biomarkers associated with PD. Recently completed phase 1a studies in healthy volunteers confirmed that MTX325 achieves sufficient penetration into functional brain tissue. The additional funding will enable Mission Therapeutics to initiate phase 1b proof-of-mechanism studies in patients with PD. The study is due to start in early 2026, as the company has already received regulatory approval from the UK’s Medicines and Healthcare products Regulatory Agency (MHRA). Results are expected in 2027, with intellectual property exclusivity continuing until 2041. Anker Lundemose, executive director at Mission Therapeutics, said: “Thanks to this additional $13.3m from our investors, we can now make the next vital steps progressing MTX325 into PD patients with this essential phase 1b clinical trial. “This will enable us to build upon the compelling preclinical data package for MTX325, published in Nature Communications in 2023, and the results from the phase 1a studies that we have obtained.” The financing round was led by current investors, including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.

Plus, news about Scynexis, Step Pharma, Eradivir, J&J, Omnix Medical and Mission Therapeutics: 💼 Boehringer Ingelheim does another cancer deal: The German drugmaker will pay up to $991 million to Seoul-based AimedBio for access to an antibody-drug conjugate that is slated to enter Phase 1 next year. The companies didn’t disclose any details about the ADC, other than saying it’s for cancer. Boehringer has done a series of deals in recent years to boost its oncology footprint, including with Nerio Therapeutics , Tessellate Bio and Accent Therapeutics. — Kyle LaHucik 👁️ EyePoint Pharmaceuticals’ $150M offering: The company is selling 11 million shares for $12.00 apiece to help progress Duravyu as a treatment for wet age-related macular degeneration and diabetic macular edema. — Jaimy Lee 💰 Scynexis gets $22M from GSK, to terminate Phase 3 trial: The biotech said the payment resolves a dispute with the British drugmaker, and it will now wind down the late-stage study testing SCY-247 in invasive candidiasis. The companies will continue to work together on vaginal infection drug Brexafemme, which was pulled from the market in 2023 over contamination concerns. The payment extends Scynexis’ cash runway for two years. Its stock price $SCYX was up 6% at market open on Wednesday. — Jaimy Lee 💶 Step Pharma’s €38M Series C: The money will help the French biotech continue work on its early-phase CTPS1 inhibitor called dencatistat in certain blood cancers and solid tumors. The raise was led by new investor V-Bio Ventures, with existing investors Bpifrance, Hadean Ventures, Inserm Transfert Initiative, Kurma Partners, Pontifax Venture Capital and Sunstone Life Science Ventures also pitching in. The biotech raised €35 million in 2021. — Reynald Castaneda Hello Victoria Ward , Endpoints is fielding its next Pulse Poll — a one-minute check-in on how artificial intelligence is reshaping biopharma. Results are confidential and will feed into our coverage of AI's real-world impact. Hello Victoria Ward , Endpoints is fielding its next Pulse Poll — a one-minute check-in on how artificial intelligence is reshaping biopharma. Results are confidential and will feed into our coverage of AI's real-world impact. Hello Victoria Ward , Endpoints is fielding its next Pulse Poll — a one-minute check-in on how artificial intelligence is reshaping biopharma. Results are confidential and will feed into our coverage of AI's real-world impact. Hello Victoria Ward , Endpoints is fielding its next Pulse Poll — a one-minute check-in on how artificial intelligence is reshaping biopharma. Results are confidential and will feed into our coverage of AI's real-world impact. Hello Victoria Ward , Endpoints is fielding its next Pulse Poll — a one-minute check-in on how artificial intelligence is reshaping biopharma. Results are confidential and will feed into our coverage of AI's real-world impact. Hello Victoria Ward , Endpoints is fielding its next Pulse Poll — a one-minute check-in on how artificial intelligence is reshaping biopharma. Results are confidential and will feed into our coverage of AI's real-world impact. Hello Victoria Ward , Endpoints is fielding its next Pulse Poll — a one-minute check-in on how artificial intelligence is reshaping biopharma. Results are confidential and will feed into our coverage of AI's real-world impact. Hello Victoria Ward , Endpoints is fielding its next Pulse Poll — a one-minute check-in on how artificial intelligence is reshaping biopharma. Results are confidential and will feed into our coverage of AI's real-world impact. 🌡 Eradivir’s flu candidate clears Phase 2a trial: Influenza patients treated with a 300 mg dose of the biotech’s EV25 demonstrated a “significant reduction” in symptom duration and severity compared with placebo. Furthermore, more than 80% of placebo patients went on to develop lower respiratory tract infection versus about 35% of treated patients. Eradivir said it is planning to start a large Phase 2b study in the US and Europe during next year’s flu season. — Ayisha Sharma ⚠️ Johnson & Johnson’s Carvykti gets black box warning for intestinal inflammation: The FDA added the warning for immune effector cell-associated enterocolitis following cases reported in clinical trials and post-marketing data. The agency noted that some cases resulted in death from gut perforation and sepsis. The multiple myeloma CAR-T therapy’s label was also updated with overall survival benefit. J&J and Legend Biotech developed Carvykti. — Lei Lei Wu 💰 Omnix Medical’s $25M Series C: The Israeli company said the round was co-led by Harel Insurance & Finance and the EIC Fund. It’s developing a drug called OMN6 to treat infections caused by Acinetobacter baumannii and other Gram-negative bacteria. — Jaimy Lee 💸 Mission Therapeutics corrals $13.3M: The Cambridge, UK-based drugmaker will use the proceeds to run a Phase 1b study to investigate its lead asset, codenamed MTX325, for Parkinson’s disease. The study is set to start in the first half of 2026. Its latest raise is from existing investors. — Reynald Castaneda