Nizubaglustat

潜在首款！创新基因疗法获FDA优先审评资格REGENXBIO公司今日宣布，美国FDA已受理其为在研基因疗法clemidsogene lanparvovec（RGX-121）提交的生物制品许可申请（BLA），用于治疗II型黏多糖贮积症（MPS II，亦称亨特综合征）。FDA已授予该申请优先审评资格，预计在今年11月9日之前完成审评。新闻稿指出，RGX-121有望成为治疗MPS II的首款一次性基因疗法。RGX-121旨在使用AAV9载体将编码人艾杜糖酸-2-硫酸酯酶（IDS）的转基因递送至中枢神经系统（CNS）。向CNS细胞内递送编码IDS的转基因可以为患者提供一个永久性的IDS分泌源，并能穿越血脑屏障，实现对CNS中其他细胞遗传缺陷或功能异常的长期修复。RGX-121已获得美国FDA授予的孤儿药资格、罕见儿科疾病认定、快速通道资格和再生医学先进疗法认定。此前公布的关键性临床试验结果显示，接受RGX-121治疗的MPS II患者的脑脊液D2S6水平平均降低86%，接近正常水平，D2S6是大脑疾病活动的关键生物标志物之一。此外，对在临床试验的剂量探索阶段接受治疗患者的长期随访数据显示，在接受关键性试验阶段推荐剂量治疗的患者中，80%的患者在最近一次随访时不需要接受标准的静脉输注酶替代疗法。降低死亡风险17%，HER2抗体疗法组合展现长期疗效罗氏（Roche）旗下基因泰克（Genentech）及其合作伙伴今日宣布，针对人类表皮生长因子受体2（HER2）阳性早期乳腺癌患者开展的3期临床试验APHINITY公布了具有统计学显著性的最终总生存期（OS）结果。研究显示，在手术后接受为期一年的Perjeta（pertuzumab）、Herceptin（trastuzumab）联合化疗辅助治疗的患者，与仅接受Herceptin、化疗及安慰剂治疗的患者相比，死亡风险在十年随访时降低了17%。十年期结果显示：接受Perjeta组合治疗的患者中，有91.6%在十年时仍然生存，而接受Herceptin、化疗及安慰剂治疗的患者中，该比例为89.8%（HR=0.83，95% CI：0.69-1.00，p=0.044）。在预设的淋巴结阳性亚组中，死亡风险降低了21%（HR=0.79，95% CI：0.64-0.97）。Perjeta是一种靶向HER2的单克隆抗体疗法，已获批与Herceptin和化疗联用，作为完整早期乳腺癌治疗方案的一部分，用于HER2阳性、局部晚期、炎性或早期乳腺癌成人患者的术前（新辅助）治疗；以及用于术后（辅助）治疗HER2阳性、复发风险高的早期乳腺癌成人患者。穿越血脑屏障，1.32亿欧元助力创新小分子进入3期临床开发Azafaros公司今日宣布，已完成超额认购的1.32亿欧元B轮融资。本轮融资将助力Azafaros加速其核心在研产品nizubaglustat的开发，预计该药物将在今年晚些时候进入治疗C型尼曼皮克病（NPC）以及GM1/GM2神经节苷脂贮积症的3期临床研究。该公司同时推动管线拓展至其他适应症。Nizubaglustat是一种口服小分子，具有独特的双重作用机制，被开发用于治疗伴有神经系统症状的罕见溶酶体贮积症，包括GM1和GM2神经节苷脂贮积症以及NPC。该药物已获得美国FDA授予治疗GM1、GM2神经节苷脂贮积症和NPC的罕见儿科疾病认定和孤儿药资格。参考资料：[1] Ten-Year APHINITY Data Show Genentech’s Perjeta-based Regimen Reduced the Risk of Death by 17% in HER2-Positive Early-Stage Breast Cancer. Retrieved May 13, 2025, from https://www.businesswire.com/news/home/20250512791224/en/Ten-Year-APHINITY-Data-Show-Genentechs-Perjeta-based-Regimen-Reduced-the-Risk-of-Death-by-17-in-HER2-Positive-Early-Stage-Breast-Cancer[2] REGENXBIO Announces FDA Acceptance and Priority Review of the BLA for RGX-121 for MPS II. Retrieved May 13, 2025, from https://www.prnewswire.com/news-releases/regenxbio-announces-fda-acceptance-and-priority-review-of-the-bla-for-rgx-121-for-mps-ii-302453436.html[3] Azafaros Secures € 132M in Oversubscribed Series B Financing to Advance Phase 3 Clinical Programs of Innovative Therapies in Lysosomal Storage Disorders. Retrieved May 13, 2025, from https://www.businesswire.com/news/home/20250512102563/en/Azafaros-Secures-%E2%82%AC-132M-in-Oversubscribed-Series-B-Financing-to-Advance-Phase-3-Clinical-Programs-of-Innovative-Therapies-in-Lysosomal-Storage-Disorders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。版权说明：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

LEIDEN, Netherlands--(BUSINESS WIRE)--Azafaros, a clinical-stage company focused on developing disease-modifying therapeutics to offer new treatment options to patients with rare lysosomal storage disorders, announces the completion of an oversubscribed €132M Series B financing led by Jeito Capital, co-led by Forbion Growth and with additional participation from Seroba, Pictet Group and existing investors Forbion Ventures, Schroders Capital and BioGeneration Ventures (BGV). This financing enables Azafaros to accelerate the development of its lead product nizubaglustat, scheduled to enter Phase 3 studies for Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses later this year, as well as expanding the Azafaros pipeline to other indications. Rachel Mears, Partner at Jeito Capital; Julien Elric, Senior Principal at Jeito Capital; and Audrey Cacaly, Principal at Forbion Growth will also join Azafaros’s Board of Directors as Board members. Founded in 2018 by BGV, leveraging science from Leiden University and Amsterdam UMC, Azafaros is led by a team of highly experienced industry experts in rare disease drug development and commercialization. The company is developing a first-in-class dual-acting drug candidate to offer new treatment options to patients with lysosomal storage disorders, a group of severe rare genetic diseases that often cause progressive neurodegeneration and, in many cases, fatal outcomes. “This successful Series B round marks a significant milestone for Azafaros, allowing us to accelerate the development of nizubaglustat and leverage our scientific understanding and competencies to bring additional candidates into development,” said Stefano Portolano, Chief Executive Officer at Azafaros. “The fact that we have been able to attract leading life sciences investors to join our existing, strong group of specialist investors is a testament to the impressive accomplishments of the team and the large unmet medical need that currently exists for patients with these hugely debilitating neurological diseases. We look forward to bringing nizubaglustat to patients.” “Azafaros has been impressive in its execution, with nizubaglustat poised to begin Phase 3 clinical development and the potential to significantly improve the lives of NPC and GM1/GM2 patients. We are excited to support and accelerate the Azafaros team in this important next step in the Company’s clinical development journey. Leading this round further demonstrates Jeito’s commitment to making a meaningful difference in patients’ lives by pursuing much needed benefits for those suffering from rare diseases,” said Rachel Mears, Partner at Jeito Capital. “Azafaros exemplifies the kind of science-driven, mission-focused company we seek to back. Nizubaglustat has the potential to fundamentally change the treatment landscape for rare genetic diseases, and we are proud to support their journey as they move closer to delivering real hope and this much needed treatment option to patients and families,” noted Audrey Cacaly, Principal at Forbion Growth. About nizubaglustat Nizubaglustat is a small molecule, orally available and brain penetrant azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease Type C (NPC). Nizubaglustat has received Rare Pediatric Disease Designations (RPDD) for the treatment of GM1 and GM2 gangliosidoses and NPC, Orphan Drug Designations (ODD) for GM1 and GM2 gangliosidosis (Sandhoff and Tay-Sachs Diseases) and NPC, as well as Fast Track Designation and IND clearance for GM1/GM2 gangliosidoses and NPC from the US Food and Drug Administration (FDA). Additionally, nizubaglustat has been awarded Orphan Medicinal Product Designation (OMPD) for the treatment of GM1 and GM2 gangliosidoses by the European Medicines Agency (EMA) and Innovation Passport for the treatment of GM1 and GM2 gangliosidoses from the UK Medicines and Healthcare Products Regulatory Agency (MHRA). About GM1 and GM2 gangliosidoses GM1 gangliosidosis and GM2 gangliosidosis (Tay-Sachs and Sandhoff diseases) are lysosomal storage disorders caused by the accumulation of GM1 or GM2 gangliosides respectively, in the central nervous system (CNS), resulting in progressive and severe neurological impairment and premature death. These diseases mostly affect infants and children, and no disease-modifying treatments are currently available. About Niemann-Pick disease Type C (NPC) Niemann-Pick disease Type C is a progressive, life-limiting neurological lysosomal storage disorder caused by mutations in the NPC1 or NPC2 gene and aberrant endosomal-lysosomal trafficking, leading to the accumulation of various lipids, including gangliosides in the CNS. The onset of disease can happen throughout the lifespan of an affected individual, from prenatal life through adulthood. About Azafaros Azafaros is a clinical-stage company founded in 2018 with a deep understanding of rare genetic disease mechanisms using compound discoveries made by scientists at Leiden University and Amsterdam UMC and is led by a team of highly experienced industry experts. Azafaros aims to build a pipeline of disease-modifying therapeutics to offer new treatment options to patients and their families. By applying its knowledge, network and courage, the Azafaros team challenges traditional development pathways to rapidly bring new drugs to the rare disease patients who need them. Azafaros is supported by leading healthcare investors, including Jeito Capital, Forbion Growth, Seroba, Pictet Group and a syndicate of leading Dutch and Swiss existing investors including Forbion Ventures, BioGeneration Ventures (BGV), BioMedPartners, Asahi Kasei Pharma Ventures, and Schroders Capital. About Jeito Capital Jeito Capital is a global leading Private Equity fund with a patient benefit driven approach that finances and accelerates the development and growth of ground-breaking medical innovation. Jeito empowers and supports managers through its expert, integrated, multi-talented team and through the investment of significant capital to ensure the growth of companies, building market leaders in their respective therapeutic areas with accelerated patients’ access globally, especially in Europe and the United States. Jeito has built a diversified portfolio of clinical biopharmas with cutting-edge innovations addressing high unmet needs. Jeito Capital is based in Paris with a presence in Europe and the United States. For more information, please visit www.jeito.life or follow us on LinkedIn. About Forbion Forbion is a leading global venture capital firm with deep expertise in Europe and offices in Naarden, The Netherlands, Munich, Germany and Boston, USA. Forbion invests in innovative biotech companies, managing approximately €5 billion across multiple fund strategies that cover all stages of (bio-) pharmaceutical drug development. In addition, Forbion’s team consists of over 30 investment professionals who have built an impressive performance track record since the late nineties with 128 investments across 11 funds. Forbion typically selects impactful investments that will positively affect the health and well-being of people and the planet, as well as meet its financial return objectives. The firm is a signatory to the United Nations Principles for Responsible Investment. Forbion operates a joint venture with BGV, the manager of seed and early-stage funds, especially focused on Benelux and Germany.