Mission gains clearance from US FDA for Phase II trial of MTX562 after it receives official approval of its Investigational New Drug (IND) application

在MTX562的研究性新药（IND）申请获得正式批准后，特派团获得了美国FDA对MTX562 II期试验的批准

Phase II trial expected to begin Q1 2024 in up to 160 patients with acute kidney injury (AKI) following cardiac surgery

第二阶段试验预计于2024年第1季度开始，对160名心脏手术后急性肾损伤（AKI）患者进行治疗

CAMBRIDGE, England, Dec. 14, 2023 /PRNewswire/ -- Mission Therapeutics ('Mission' or the 'Company'), a clinical-stage biotech developing first-in-class therapeutics targeting mitophagy, today announces that the US Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for its lead asset MTX652, allowing Mission to proceed with its planned Phase II clinical trial in the US..

英国剑桥，2023年12月14日/PRNewswire/--Mission Therapeutics（“Mission”或“公司”）是一家临床阶段的生物技术公司，正在开发针对线粒体自噬的一流疗法，今天宣布，美国食品和药物管理局（FDA）已批准其主要资产MTX652的研究性新药（IND）申请，允许Mission在美国进行计划的II期临床试验。。

Up to 160 adults with increased risk for Acute Kidney Injury (AKI) following cardiac surgery are planned to be recruited for the trial, which will take place at multiple sites in North America and Europe. The double-blind, placebo-controlled trial is expected to begin early next year and is intended to show that MTX652 protects this high-risk group of patients from AKI by assessing standard markers of renal function and renal injury over time..

计划招募多达160名心脏手术后急性肾损伤（AKI）风险增加的成年人参加该试验，该试验将在北美和欧洲的多个地点进行。这项双盲安慰剂对照试验预计将于明年初开始，旨在表明MTX652通过评估肾功能和肾损伤的标准标志物来保护这一高危人群免受AKI的影响。。

Dr Anker Lundemose, Chief Executive Officer of Mission Therapeutics, said: 'The FDA's approval of our Phase II clinical study for our lead asset MTX652 marks a major milestone for Mission. We now have two USP30 inhibitors advancing through clinical trials, MTX652 for acute kidney injury and MTX325 for Parkinson's Disease, validating our unique approach and the breadth of our assets.'.

Mission Therapeutics首席执行官Anker Lundemose博士说：“FDA批准我们的主要资产MTX652的II期临床研究，标志着Mission的一个重要里程碑。我们现在有两种USP30抑制剂正在进行临床试验，MTX652用于急性肾损伤，MTX325用于帕金森病，验证了我们独特的方法和我们资产的广度。”。

Dr Suhail Nurbhai, Chief Medical Officer of Mission Therapeutics, said: 'Recent reports suggest that up to 50% of high-risk patients suffer acute kidney injury following heart surgery[i] [ii]. There are no approved drug treatments and the immediate and longer-term consequences can be very serious, including continued decline of renal function and/or the requirement for renal replacement therapy.

Mission Therapeutics首席医疗官Suhail Nurbhai博士说：“最近的报告表明，高达50%的高危患者在心脏手术后遭受急性肾损伤。目前还没有批准的药物治疗方法，其直接和长期后果可能非常严重，包括肾功能持续下降和/或需要肾脏替代治疗。

We believe MTX562 has the potential to alleviate these outcomes and to meet this serious and important unmet medical need. We are delighted to have received this approval and look forward to starting this trial in 2024.'.

我们相信MTX562有可能缓解这些结果，并满足这一严重而重要的未满足医疗需求。我们很高兴获得这一批准，并期待着在2024年开始这项试验。”。

Dr Paul Thompson, Chief Scientific Officer of Mission Therapeutics, commented: 'In preclinical experiments, MTX652 demonstrated significant protective effects in multiple models of kidney injury, and this breadth of effect is very encouraging in indicating potential clinical benefit. We are very pleased with this strong preclinical foundation, which supports our progress into clinical efficacy trials.'.

Mission Therapeutics首席科学官保罗·汤普森博士评论道：“在临床前实验中，MTX652在多种肾损伤模型中表现出显着的保护作用，这种广泛的作用在表明潜在的临床益处方面非常令人鼓舞。我们对这一强大的临床前基础感到非常高兴，这支持了我们进入临床疗效试验的进展。”。

The FDA's decision follows completion of a Phase I First Time in Human (FTIH) study of MTX652 earlier in 2023, in which over 80 healthy volunteers received MTX652. The trial successfully demonstrated MTX652 was safe and well tolerated up to a single dose of 200mg a day and multiple doses of 100mg daily for 14 days, with an excellent pharmacokinetic profile..

FDA的决定是在2023年早些时候完成MTX652的第一阶段首次人体（FTIH）研究之后做出的，其中80多名健康志愿者接受了MTX652。该试验成功证明MTX652安全且耐受性良好，每天单剂量200mg，每天多剂量100mg，持续14天，具有良好的药代动力学特征。。

About MTX652 and USP30

关于MTX652和USP30

MTX652 is a potent and selective compound designed to improve mitochondrial quality and function by enhancing mitophagy through inhibition of USP30, a deubiquitylating enzyme localised to mitochondria which is a negative regulator of mitophagy. It thereby promotes cellular health by enhancing this key mechanism, which cells use to clear dysfunctional mitochondria.

MTX652是一种有效且选择性的化合物，旨在通过抑制USP30（一种定位于线粒体的去泛素化酶，是线粒体自噬的负调节剂）来增强线粒体自噬，从而改善线粒体的质量和功能。因此，它通过增强细胞用于清除功能失调的线粒体的关键机制来促进细胞健康。

MTX652 has the potential to reduce the impact of impaired mitochondria associated with Ischaemic Reperfusion Injury (IRI) in the heart and kidneys in patients who have undergone heart surgery..

MTX652有可能减少心脏手术患者心脏和肾脏缺血再灌注损伤（IRI）相关线粒体受损的影响。。

About Mission Therapeutics

关于Mission Therapeutics

Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria, promoting cell health and function. Mitochondria are energy producing organelles which require lifetime quality control through a ubiquitin-mediated clearance mechanism known as mitophagy.

Mission Therapeutics是发现和开发新疗法的世界领先者，该疗法可促进去除功能失调的线粒体，促进细胞健康和功能。线粒体是产生能量的细胞器，需要通过泛素介导的清除机制（称为线粒体自噬）进行终身质量控制。

In certain situations, such as cellular stress, cell injury, and/or defects of the mitophagy process, the mitochondria can become dysfunctional and damaging to the cell, leading to reduced energy production, oxidative stress, inflammation and potentially cell death. Dysfunctional mitochondria are significant drivers of disease pathophysiology in acute kidney injury (AKI), Parkinson's Disease (PD), heart failure, Duchenne's Muscular Dystrophy, IPF, mitochondrial diseases and Alzheimer's..

在某些情况下，例如细胞应激，细胞损伤和/或线粒体自噬过程的缺陷，线粒体可能功能失调并对细胞造成损害，导致能量产生减少，氧化应激，炎症和潜在的细胞死亡。线粒体功能异常是急性肾损伤（AKI），帕金森病（PD），心力衰竭，杜兴氏肌营养不良症，IPF，线粒体疾病和阿尔茨海默氏病的疾病病理生理学的重要驱动因素。。

USP30 is a deubiquitylating enzyme that constantly removes ubiquitin from mitochondria, providing a potential brake on clearance of dysfunctional mitochondria. Mission is currently developing two small molecule drugs, MTX652 (peripheral) and MTX325 (targeting the CNS) which, through inhibition of the mitochondrial DUB enzyme USP30, will promote clearance of dysfunctional mitochondria – consequently improving overall cellular health.

USP30是一种去泛素化酶，可不断从线粒体中去除泛素，为清除功能异常的线粒体提供潜在的制动作用。Mission目前正在开发两种小分子药物MTX652（外周）和MTX325（靶向中枢神经系统），通过抑制线粒体DUB酶USP30，将促进功能失调的线粒体的清除，从而改善整体细胞健康。

Mission's USP30 inhibitors MTX652 and MTX325 could potentially be used to treat any disease or condition driven by mitochondrial dysfunction..

Mission的USP30抑制剂MTX652和MTX325可能用于治疗由线粒体功能障碍引起的任何疾病或病症。。

Mission is backed by blue chip investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.

这项使命得到了包括辉瑞风险投资公司、索芬诺娃合作伙伴、罗氏风险基金、SR One、IP Group和Rosetta Capital在内的蓝筹股投资者的支持。

[i] https://guardtherapeutics.com/en/press-releases/guard-therapeutics-reports-robust-efficacy-of-rmc-035-in-phase-2-akita-and-advances-clinical-development-program

[i] （笑声）https://guardtherapeutics.com/en/press-releases/guard-therapeutics-reports-robust-efficacy-of-rmc-035-in-phase-2-akita-and-advances-clinical-development-program

[ii] https://pubmed.ncbi.nlm.nih.gov/34474590/

[ii]https://pubmed.ncbi.nlm.nih.gov/34474590/

SOURCE Mission Therapeutics

SOURCE Mission Therapeutics公司