SAN DIEGO, May 21, 2024 /PRNewswire/ -- Transposon Therapeutics, a biotechnology company developing a platform of novel, orally administered therapies for the treatment of neurodegenerative and aging-related diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to TPN-101 for progressive supranuclear palsy (PSP)..

2024年5月21日，圣地亚哥，转座子治疗公司，一家开发用于治疗神经退行性疾病和衰老相关疾病的新型口服治疗平台的生物技术公司，今天宣布，美国食品和药物管理局（FDA）已授予TPN-101进行性核上性麻痹（PSP）的快速通道指定。。

'Fast Track designation for TPN-101 is an important acknowledgement by the FDA of the critical need to find an effective treatment for PSP, a rare and devastating neurological disorder with no approved treatment options,' said Dennis Podlesak, Chairman and Chief Executive Officer of Transposon. 'We look forward to working collaboratively with the FDA to advance the development of TPN-101 as rapidly as possible for the treatment of PSP and other neurodegenerative diseases including ALS and Alzheimer's disease.'.

转座子董事长兼首席执行官丹尼斯·波德莱萨克（DennisPodlesak）说：“TPN-101的快速命名是FDA对寻找有效治疗PSP的关键需求的重要认可，PSP是一种罕见且毁灭性的神经系统疾病，目前尚无经批准的治疗选择。”我们期待着与FDA合作，尽快推进TPN-101的开发，以治疗PSP和其他神经退行性疾病，包括ALS和阿尔茨海默氏病。”。

The designation of TPN-101 as a Fast Track product for PSP is supported by data from a Phase 2, randomized, double-blind, placebo-controlled study of TPN-101 in patients with PSP. In that study, TPN-101 was the first treatment for PSP to reduce levels of neurofilament light chain (NfL), a key biomarker of neurodegeneration in tauopathies such as PSP and Alzheimer's disease.

TPN-101作为PSP快速通道产品的指定得到了PSP患者TPN-101第二阶段随机双盲安慰剂对照研究数据的支持。在该研究中，TPN-101是PSP降低神经丝轻链（NfL）水平的第一种治疗方法，NfL是PSP和阿尔茨海默氏病等tau蛋白病中神经变性的关键生物标志物。

TPN-101 also showed dose-related reductions in interleukin 6 (IL-6) cytokine levels, a biomarker of neuroinflammation that is elevated in PSP and correlates with disease progression and severity. Participants treated with TPN-101 for the entire 48-week trial duration showed a stabilization of their clinical symptoms as measured by the PSP Rating Scale (PSPRS) between weeks 24 and 48.

TPN-101还显示白细胞介素6（IL-6）细胞因子水平的剂量相关降低，白细胞介素6（IL-6）细胞因子水平是神经炎症的生物标志物，在PSP中升高，并与疾病进展和严重程度相关。在整个48周的试验期间，用TPN-101治疗的参与者在第24周和第48周之间通过PSP评定量表（PSPRS）测量，其临床症状稳定。

Further information on the study can be accessed at ClinicalTrials.gov..

有关该研究的更多信息，请访问ClinicalTrials.gov。。

About Fast Track Designation

关于快速通道指定

The FDA grants Fast Track designation to facilitate the development and expedite the review of medicines to treat serious conditions and fill an unmet medical need. Fast Track status allows for enhanced communication and collaboration between the FDA and drug developers, potentially speeding up the delivery of life-saving treatments to patients..

FDA授予快速通道指定，以促进药物的开发和加速审查，以治疗严重疾病并满足未满足的医疗需求。快速通道状态可以增强FDA与药物开发人员之间的沟通与合作，从而有可能加速向患者提供挽救生命的治疗。。

About PSP

关于PSP

PSP is a rare neurodegenerative disorder that causes slowing of movement, loss of balance leading to falls, impaired eye movements, and disturbances in cognition and behavior. The disease typically affects people in their mid- to late-60s, and the mean survival for individuals with PSP is 6 to 7 years.

PSP是一种罕见的神经退行性疾病，会导致运动减慢，失去平衡导致跌倒，眼球运动受损以及认知和行为障碍。该疾病通常影响60多岁中后期的人群，PSP患者的平均生存期为6至7年。

There are currently no treatments capable of delaying the progression of the disease..

目前还没有能够延缓疾病进展的治疗方法。。

About Transposon

关于转座子

Transposon Therapeutics, Inc. is a clinical-stage biopharmaceutical company developing a platform of novel therapies for the treatment of neurodegenerative and aging-related diseases, including progressive supranuclear palsy, amyotrophic lateral sclerosis, frontotemporal dementia, and Alzheimer's disease.

Transposon Therapeutics，Inc.是一家临床阶段的生物制药公司，开发了一个新疗法平台，用于治疗神经退行性疾病和衰老相关疾病，包括进行性核上性麻痹，肌萎缩侧索硬化症，额颞叶痴呆和阿尔茨海默氏病。

The company's lead clinical compound, TPN-101, is first-in-class to address LINE-1 reverse transcriptase for treating neurodegenerative and autoimmune diseases. The company also has a discovery platform supporting a deep pipeline of novel therapies to address additional indications..

该公司的主要临床化合物TPN-101是第一个用于治疗神经退行性疾病和自身免疫性疾病的LINE-1逆转录酶。该公司还拥有一个发现平台，支持新疗法的深层管道，以解决其他适应症。。

About TPN-101

关于TPN-101

TPN-101 specifically inhibits the LINE-1 reverse transcriptase that promotes LINE-1 replication. LINE-1 elements are a class of retrotransposable elements that in humans are uniquely capable of replicating and moving to new locations within the genome. When this process becomes dysregulated, LINE-1 reverse transcriptase drives overproduction of LINE-1 DNA, triggering innate immune responses that contribute to neurodegenerative, autoimmune and aging-related disease pathology..

TPN-101特异性抑制促进LINE-1复制的LINE-1逆转录酶。LINE-1元件是一类逆转录转座因子，在人类中独特地能够复制并移动到基因组内的新位置。当这个过程失调时，LINE-1逆转录酶会驱动LINE-1 DNA的过量产生，引发先天性免疫反应，从而导致神经退行性疾病，自身免疫疾病和衰老相关疾病的病理学。。