NEW YORK, March 28, 2024 /PRNewswire/ -- ProJenX, a clinical-stage biotechnology company developing novel, brain-penetrant therapies targeting biologically-defined pathways for the treatment of amyotrophic lateral sclerosis (ALS) and other debilitating brain diseases, today announced that the United States Food and Drug Administration (FDA) has removed a partial clinical hold on Study PRO-101, a hybrid Phase 1 clinical trial evaluating prosetin—a first-in-class MAP4 kinase (MAP4K) inhibitor—in healthy volunteers and participants with ALS.

纽约，2024年3月28日/PRNewswire/-ProJenX，一家临床阶段生物技术公司，开发了针对生物学定义的治疗肌萎缩侧索硬化症（ALS）和其他衰弱性脑部疾病的途径的新型脑渗透疗法，今天宣布，美国食品和药物管理局（FDA）取消了部分临床搁置研究PRO-101，这是一项混合1期临床试验，评估健康志愿者和ALS参与者中的第一类MAP4激酶（MAP4K）抑制剂prosettin。

The healthy volunteer portions of the trial have been completed, and the company is initiating Part 1c of PRO-101, designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in participants with ALS.

该试验的健康志愿者部分已经完成，该公司正在启动PRO-101的1c部分，旨在评估脯西汀在ALS参与者中的安全性，耐受性，药代动力学和药效学。

Stan Abel, President and Chief Executive Officer at ProJenX, commented, 'Recent clinical trial results in ALS have further demonstrated the urgent need for meaningful therapies that can alter the course of this devastating disease. Our Columbia University and Project ALS co-founders have invested over two decades in scientific research that shows a critical role for MAP4K inhibition on ALS motor neuron survival, and we are thrilled to be advancing prosetin to people living with ALS in the coming months.'

ProJenX总裁兼首席执行官斯坦·阿贝尔（StanAbel）评论道：“最近ALS的临床试验结果进一步表明，迫切需要有意义的治疗方法来改变这种毁灭性疾病的进程。我们的哥伦比亚大学和ALS项目联合创始人已经投资了20多年的科学研究，这些研究表明MAP4K抑制对ALS运动神经元存活具有关键作用，我们很高兴在未来几个月将prosettin推广给ALS患者。”

'Following recent Clinical Trial Application authorizations in Canada and Europe, we are very pleased that FDA has lifted this partial clinical hold, which previously limited prosetin dose levels in Study PRO-101 in the United States,' added Erin Fleming, Co-Founder and Chief Operating Officer at ProJenX.

ProJenX联合创始人兼首席运营官艾琳·弗莱明（ErinFleming）补充道：“继最近在加拿大和欧洲获得临床试验申请授权后，我们非常高兴FDA解除了这一部分临床禁令，此前该禁令限制了美国PRO-101研究中的prosetin剂量水平。”

'The FDA's decision allows us to fully pursue a global strategy to bring this promising investigational treatment to people with ALS and other neurodegenerative diseases.'

“FDA的决定使我们能够全面推行全球战略，为ALS和其他神经退行性疾病患者带来这种有前途的研究性治疗。”

Prosetin is a selective, oral, brain-penetrant, MAP4K inhibitor developed by ProJenX co- founders at Columbia University for the treatment of ALS. Following the discovery that MAP4K inhibition confers potent motor neuron protection across multiple patient stem cell-derived models of ALS, prosetin was optimized for potency against MAP4Ks, efficacy in motor neuron rescue, and preferential distribution to the CNS.

Prosettin是哥伦比亚大学ProJenX联合创始人开发的一种选择性口服脑渗透MAP4K抑制剂，用于治疗ALS。在发现MAP4K抑制在多个患者干细胞衍生的ALS模型中赋予有效的运动神经元保护作用后，Prosettin针对MAP4K的效力，运动神经元拯救的功效以及对中枢神经系统的优先分布进行了优化。

'There is an immense unmet need for safe and effective treatment options for ALS, and prosetin is an exciting investigational therapy option in the current clinical trial landscape,' said Jinsy Andrews, MD, MSc, Associate Professor of Neurology and Director of Neuromuscular Clinical Trials at Columbia University.

哥伦比亚大学神经病学副教授兼神经肌肉临床试验主任、医学博士、理学硕士金西·安德鲁斯（JinsyAndrews）说：“对于ALS的安全有效的治疗选择，还有巨大的需求尚未得到满足，在目前的临床试验环境中，普罗西汀是一种令人兴奋的研究性治疗选择。”

'I am encouraged by prosetin's compelling preclinical efficacy data and by its consistent safety and tolerability profile in healthy volunteers and chronic toxicology studies, and look forward to working with the ProJenX team to bring Study PRO-101 to people living with ALS in the United States.'

“我对Prosettin令人信服的临床前疗效数据及其在健康志愿者和慢性毒理学研究中的一致安全性和耐受性感到鼓舞，并期待着与ProJenX团队合作，将研究PRO-101带给美国ALS患者。”

About PRO-101

关于PRO-101

PRO-101 is a three-part Phase 1 clinical trial designed to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in healthy volunteers and patients with ALS. Parts 1a and 1b, which have been completed, consisted of a randomized, double-blind, placebo-controlled, dose-escalating study to evaluate the safety, tolerability, and pharmacokinetics of single ascending and multiple doses of prosetin in healthy volunteers.

PRO-101是一项由三部分组成的1期临床试验，旨在评估健康志愿者和ALS患者中prosettin的安全性，耐受性，药代动力学和药效学。已经完成的第1a和1b部分包括一项随机，双盲，安慰剂对照，剂量递增的研究，以评估健康志愿者单次递增和多次递增剂量prosettin的安全性，耐受性和药代动力学。

Based on favorable safety, tolerability, and pharmacokinetic data from this portion of the study, Part 1c will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in ALS patients. For more information about PRO-101, contact trials@projenx.com..

基于这部分研究的良好安全性，耐受性和药代动力学数据，第1c部分将评估脯西汀在ALS患者中的安全性，耐受性，药代动力学和药效学。有关PRO-101的更多信息，请联系trials@projenx.com..

About ProJenX

关于ProJenX

ProJenX is a clinical-stage biotechnology company developing novel, brain-penetrant, targeted therapies to address untreatable brain diseases, with an initial focus on ALS. ProJenX was created out of a long-term research collaboration between Project ALS and researchers at Columbia University to rapidly develop and commercialize its lead therapy candidate, prosetin, for people living with ALS.

ProJenX是一家临床阶段生物技术公司，开发新型脑渗透靶向疗法，以解决无法治疗的脑部疾病，最初专注于ALS。ProJenX是在ALS项目和哥伦比亚大学研究人员的长期研究合作下创建的，旨在为ALS患者快速开发其主要治疗候选药物prosettin并将其商业化。

At the heart of ProJenX's approach is an innovative, patient-specific, cell-based drug discovery platform that can be leveraged for research and drug development for ALS and other debilitating brain diseases. For more information, visit projenx.com..

ProJenX方法的核心是一个创新的，针对患者的，基于细胞的药物发现平台，可用于ALS和其他使人衰弱的脑部疾病的研究和药物开发。有关更多信息，请访问projenx.com。。