Macular telangiectasia type 2

▎药明康德内容团队编辑 预防糖尿病，口服疗法达到2期临床主要终点 Aphaia Pharma公司日前宣布，其用于治疗糖尿病前期的在研疗法APHD-012在2期临床试验中达到了主要终点。数据显示，与安慰剂相比，APHD-012在6周给药后在口服葡萄糖耐受测试（OGTT）异常的个体中显著改善了葡萄糖耐受，并且具有良好的安全性。 在一组包含健康人、糖尿病前期和糖尿病患者的志愿者中，6周APHD-0126治疗在OGTT中将血糖水平从9.0±0.6 mmol/L降低到7.9±0.6 mmol/L（n=23，p=0.01）；接受安慰剂治疗无显著效果（从8.8±0.4 mmol/L降至8.5±0.6 mmol/L；p=0.33）。 亚组分析显示，APHD-012在糖尿病前期患者中的效果更显著，将血糖水平从8.7±0.4 mmol/L降低到6.8±0.5 mmol/L；n=12，p=0.003）；在该亚组中，安慰剂无显著效果。 APHD-012是一种专有的口服葡萄糖配方，设计在小肠的特定部位释放，以恢复内源性营养感知信号通路，并刺激控制多种稳态功能的广谱肠激素的释放，包括胰高血糖素样肽1（GLP-1）、胰多糖素和氧促胰多糖素（OXM）等。 创新细胞疗法获FDA优先审评资格，治疗视网膜退化疾病 Neurotech Pharmaceuticals公司日前宣布，为细胞疗法revakinagene taroretcel（NT-501）递交的生物制品许可申请（BLA）获得FDA授予的优先审评资格，用于治疗2型黄斑毛细血管扩张症（MacTel）。 Revakinagene taroretcel基于该公司独特的封装细胞疗法（encapsulated cell therapy，ECT）平台。它将表达睫状神经营养因子（CNTF）的同种异体视网膜色素上皮（RPE）细胞封装在微小的移植物中，这些移植物在植入到眼睛中后可以持续释放CNTF，从而延缓视网膜退化，潜在改善患者的长期视力。移植物具有通透性的外膜在允许营养物质进入和CNTF释放的同时，保护移植细胞不受到宿主免疫系统的攻击，确保它们的长期存活和功能。 Revakinagene taroretcel在两项3期临床试验中已经获得积极结果，显著降低了2型MacTel患者的疾病进展速度。 亨廷顿病疗法2期临床结果积极 PTC Therapeutics日前分享了在研疗法PTC518治疗亨廷顿病（HD）患者的2期临床试验PIVOT-HD的中期结果。在第12个月时，接受PTC518治疗患者队列中显示出剂量依赖性突变亨廷顿蛋白（mHTT）的降低。此外，多项与HD相关的临床评估都表现出有利趋势。而且经过12个月的治疗，PTC518继续表现良好的安全性和耐受性。 数据显示，在第12个月时，5 mg和10 mg剂量的PTC518分别将血液中的mHTT水平降低了22%和43%。在脑脊液中，5 mg和 10 mg剂量分别降低mHTT水平21%和43%。此外，在第12个月时，PTC518在缓解运动症状进展上表现出显著效果，根据总运动评分（TMS）评估，5 mg和10 mg剂量组的症状加重分别为2.0和1.3分，而安慰剂组为4.9分。 开发潜在“first-in-class”PI3Kδ别构调节剂，新锐完成8000万欧元融资 iOnctura日前宣布完成8000万欧元的B轮融资。iOnctur正在开发一系列精准口服小分子抗癌药物。该公司已将两种候选疗法推进到中期临床开发阶段。主打在研药物roginolisib是一款PI3Kδ别构调节剂，具有独特的化学结构和结合模式。新闻稿指出，roginolisib有潜力成为首个成功靶向PI3Kδ关键癌症通路的治疗方法。 此次融资将用于加速roginolisib用于治疗葡萄膜黑色素瘤（UM）的临床试验，这是一种眼部罕见癌症，目前可用的治疗方法很少。在一项1b期临床试验中，roginolisib在UM中表现出长期安全性和可喜的疗效，且在数月内持续保持临床活性。 iOnctura的第二个临床开发项目cambritaxestat是一款autotaxin抑制剂，用于治疗autotaxin过表达的高度纤维化肿瘤。目前，cambritaxestat联合化疗治疗转移性胰腺癌的1b期临床试验正在进行中。 2.75亿美元合作！潜在“best-in-class”疗法即将进入临床 Alivexis和Melodia Therapeutics今日宣布，双方已签署了一项独家许可协议，在全球范围内开发、制造和商业化Alivexis的MDI-0151，这是一种具有“best-in-class”潜力的组织蛋白酶C（cathepsin C）抑制剂。Melodia将迅速启动MDI-0151的IND申请，为针对多种由中性粒细胞过度激活引起的难治性炎症性疾病的1/2a期临床试验做准备。 根据协议条款，Alivexis授予Melodia在全球范围内开发、制造和商业化MDI-0151的权利，并将从Melodia获得高达约2.75亿美元的预付款和开发及销售里程碑付款。MDI-0151抑制的组织蛋白酶C是一种负责激活在中性粒细胞破坏组织中起重要作用的蛋白酶的酶。由于抑制组织蛋白酶C对中性粒细胞的免疫功能影响较小，MDI-0151可能在有效治疗各种免疫和炎症性疾病同时，不会削弱免疫系统。 ▲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： [1] Neurotech Receives Priority Review of Biologics License Application (BLA) for NT-501 (revakinagene taroretcel) as a Treatment for Macular Telangiectasia Type 2 (MacTel). Retrieved June 20, 2024, from https://www.neurotechpharmaceuticals.com/receives-priority-review-for-bla-for-nt-501/ [2] iOnctura announces EUR80 million Series B financing to progress pipeline through Phase II trials. Retrieved June 20, 2024, from https://wp-ionctura-2024.s3.eu-west-2.amazonaws.com/media/2024/06/iOnctura-Financing-PR-19-6-FINAL.pdf [3] Interim PIVOT-HD Results Demonstrate Evidence of Favorable CNS Biomarker and Clinical Effects at Month 12 in Huntington's Disease Patients. Retrieved June 20, 2024, from https://ir.ptcbio.com/news-releases/news-release-details/interim-pivot-hd-results-demonstrate-evidence-favorable-cns [4] APHAIA PHARMA ANNOUNCES POSITIVE RESULTS FROM PHASE 2 TRIAL EVALUATING ITS LEAD DRUG FORMULATION. Retrieved June 20, 2024, from https://aphaiapharma.com/news/aphaia-pharma-announces-positive-results-from-phase-2-trial-evaluating-its-lead-drug-formulation/ [5] Melodia Therapeutics and Alivexis Sign Exclusive License Agreement for Cathepsin C Inhibitor Program MOD-A. Retrieved June 21, 2024, from https://www.businesswire.com/news/home/20240620110656/en/Melodia-Therapeutics-and-Alivexis-Sign-Exclusive-License-Agreement-for-Cathepsin-C-Inhibitor-Program-MOD-A 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

- Priority review granted with a PDUFA goal date set for December 17, 2024. CUMBERLAND, R.I.--(BUSINESS WIRE)-- Neurotech Pharmaceuticals, Inc., an innovator in sustained drug delivery for chronic retinal diseases, announces that the U.S. Food and Drug Administration (FDA), has determined that the Biologic License Application (BLA) for NT-501, an investigational encapsulated cell therapy for the treatment of MacTel, is sufficiently complete to permit a substantive review. The application has a prescription drug user fee act (PDUFA) goal date of December 17, 2024. MacTel is a progressive, neurodegenerative disease of the retina that results in the deterioration of central vision, significantly impacting patients' quality of life. NT-501 is an ocular implant designed to deliver sustained therapeutic doses of ciliary neurotrophic factor (CNTF) directly to the retina to slow the progression of the disease. “This is a significant achievement for Neurotech,” said Richard Small, Chief Executive Officer. “I would like to express my gratitude to our employees for reaching this important milestone.” About Macular Telangiectasia (MacTel) MacTel, or idiopathic juxtafoveal macular telangiectasia, is a rare neurodegenerative disease with characteristic localized retinal degeneration with secondary alterations of the retinal vasculature.1 MacTel typically affects both eyes and causes deterioration in central vision impacting patients’ quality of life. About Encapsulated Cell Therapy (ECT) The ECT platform is a cell-based delivery system that was designed to provide long-term, sustained delivery of therapeutic proteins for the treatment of chronic retinal diseases. This versatile platform consists of a small, semi-permeable capsule containing proprietary allogeneic RPE cells genetically engineered to produce specific therapeutic proteins for targeted disease treatment. The capsule is surgically inserted into the patient’s vitreous and sutured to the sclera during an outpatient procedure. Once in place, the capsule's semi-permeable membrane allows essential nutrients to enter, while also permitting the therapeutic proteins to exit into the vitreous, providing targeted and continuous treatment. Simultaneously, the membrane protects the encapsulated RPE cells from the host's immune system, ensuring their long-term survival and functionality. About NT-501 Implant NT-501 leverages our ECT platform to deliver CNTF for the treatment of chronic retinal diseases, such as MacTel. CNTF is a potent neuroprotective protein that promotes the survival and maintenance of photoreceptors. This targeted therapy provides sustained delivery of CNTF, aiming to slow retinal degeneration and potentially improve long-term visual outcomes for patients. About Neurotech Pharmaceuticals, Inc. Neurotech Pharmaceuticals, Inc. is a private, clinical stage biotechnology company and an innovator in sustained drug delivery for chronic retinal diseases. The core platform technology, ECT, enables continuous production of therapeutic proteins to the back of the eye. For more information about us, please visit . 1. Charbel Issa P, Gillies MC, Chew EY, Heeren TFC, Bird AC, Peto T, Holz FG, Scholl HPN (2013) Macular Telangiectasia Type 2.

CUMBERLAND, R.I.--(BUSINESS WIRE)-- Neurotech Pharmaceuticals, Inc., a private clinical stage biotech company, announced today that Alex Gorsky has been elected to Neurotech’s board as Lead Director. Gorsky is the former Chairman and CEO of Johnson & Johnson. This press release features multimedia. View the full release here: Alex Gorsky (Photo: Business Wire) Under Gorsky’s leadership, Johnson & Johnson grew to become the world’s largest diversified healthcare company and one of the foremost innovators in research and development for emerging health technologies. His influence has shaped both the healthcare landscape and the greater business community through his work as a member of the Business Roundtable, the Business Council, and many industry groups. He currently sits on the boards of Apple, IBM, JPMorgan Chase, and the Travis Manion Foundation, as well as the Wharton School of the University of Pennsylvania Board of Advisors. “I am delighted to welcome Alex to Neurotech’s Board of Directors,” said Jim Mazzo, Neurotech’s Executive Chairman. “As a visionary in patient focused healthcare, his expertise and enthusiasm for innovative technology will greatly enrich Neurotech’s mission to revolutionize treatments for various ocular diseases using our encapsulated cell therapy.” Mr. Gorsky said, “I am honored to join Neurotech’s board and look forward to using my experience to help the company navigate in a rapidly evolving environment. I am inspired by the Lowy family’s long-term commitment. This unique scientific approach and novel technology for the treatment of chronic eye diseases has the potential to provide access to important health options for patients around the world.” About Macular telangiectasia type 2 Macular telangiectasia type 2 (MacTel), or idiopathic juxtafoveal macular telangiectasia type 2, is a rare neurodegenerative disease with characteristic alterations of the retinal vasculature and localized retinal degeneration.1 MacTel typically affects both eyes and causes a gradual deterioration in central vision. About NT-501 Implant Designed to be implanted into the vitreous cavity of the eye, the investigational NT-501 implant is a tiny hollow cylindrical membrane which encapsulates human epithelial cells genetically engineered to produce ciliary neurotropic factor (CNTF) continuously, a protein now clinically validated in Phase 3 clinical trials to slow the progression of MacTel. About Encapsulated Cell Therapy Encapsulated Cell Therapy (ECT) is an investigational first-in-class, platform technology that promotes continuous production of therapeutic proteins to the eye with the potential to treat a broad array of ocular diseases. It utilizes a proprietary, well-characterized retinal pigment epithelial cell line that is genetically engineered to produce therapeutically active biologics. The cells are encapsulated in a semi-permeable membrane that allows for selective passage of therapeutic proteins. The ECT platform is inserted during a single outpatient surgical procedure through a small scleral incision, and can also be removed through the same incision, if desired. ECT has the potential to address the current limitations of intraocular drug delivery by allowing for and ensuring patient compliance, while reducing the treatment burden with one surgical procedure that can deliver the drug for at least 2 years. About Neurotech Pharmaceuticals, Inc. Neurotech Pharmaceuticals, Inc. is a private clinical stage biotech company focused on developing transformative therapies for chronic eye diseases. The core platform technology, Encapsulated Cell Therapy (ECT), enables continuous production of therapeutic proteins to the eye. Neurotech is currently studying in the clinic ECT candidates to treat Macular telangiectasia type 2 and glaucoma. To learn more, visit . 1. Charbel Issa P, Gillies MC, Chew EY, Heeren TFC, Bird AC, Peto T, Holz FG, Scholl HPN (2013) Macular Telangiectasia Type 2. Prog. Retin. Eye Res. 34: 49-77.