/PRNewswire/ -- Kaerus Bioscience ('Kaerus' or 'the Company'), a clinical stage biopharmaceutical company created by Medicxi for the development of therapeutics for rare genetic syndromes of neurodevelopment, today announces that its lead candidate KER-0193 has been granted both Orphan Drug Designation and Rare Pediatric Drug Designations for the treatment of Fragile X syndrome (FXS) by the U.S.

/PRNewswire/ -- Medicxi创立的临床阶段生物制药公司Kaerus Bioscience（“Kaerus”或“公司”），专注于开发治疗神经发育罕见遗传综合征的药物，今日宣布其主导候选药物KER-0193已获得美国授予的孤儿药资格和罕见儿科疾病资格，用于治疗脆性X综合征（FXS）。

Food and Drugs Administration (FDA)..

食品药品监督管理局 (FDA)。

It follows the recent

它遵循了最近的

successful completion of a Phase 1 trial of KER-0193

KER-0193的1期试验成功完成

in healthy volunteers, which confirmed the drug to be safe, well tolerated and exhibit excellent pharmacokinetics.

在健康志愿者中，这证实了该药物安全、耐受性良好且药代动力学表现优异。

FXS is an inherited condition, affecting about 1 in 7,000 males and 1 in 11,000 females, for which there are currently no approved treatments. It is the most common cause of inherited autism and intellectual disability, accounting for approximately 1 per cent of such cases globally.

脆性X综合症是一种遗传性疾病，大约每7000名男性和11000名女性中就有一人受到影响，目前尚无获批的治疗方法。它是遗传性自闭症和智力障碍最常见的原因，约占全球此类病例的1%。

Robert Ring

罗伯特·林格

, CEO of Kaerus Bioscience, said:

Kaerus Bioscience首席执行官表示：

'The FDA's granting of Orphan Drug Designation and Rare Pediatric Drug Designation for KER-0193 is an important step towards our objective of delivering an effective treatment for people with Fragile X syndrome.

“FDA授予KER-0193孤儿药资格和罕见儿科疾病药物资格是我们为脆性X综合征患者提供有效治疗的目标迈出的重要一步。”

'Not only do these two FDA designations affirm the therapeutic potential of KER-0193 in Fragile X syndrome, they also provide Kaerus with access to significant regulatory and financial incentives as we look to progress the drug through clinical trials and ultimately take it to market.'

“不仅这两个FDA指定肯定了KER-0193在脆性X综合症中的治疗潜力，它们还为Kaerus提供了重要的监管和财务激励，因为我们正计划推动该药物通过临床试验并最终将其推向市场。”

Paul Sekhri

保罗·塞赫里

, Chairman of Kaerus Bioscience, said:

凯罗斯生物科学公司董事长说：

'These important FDA designations are more excellent news for Kaerus, coming after the impressive Phase 1 results showing KER-0193 to be safe and well-tolerated. These developments put the Company in a superb position as we  look to move into Phase 2 trials.'

“这些重要的FDA指定对Kaerus来说是更棒的消息，继令人印象深刻的1期试验结果表明KER-0193安全且耐受性良好之后。这些进展使公司处于极佳的位置，因为我们正准备进入2期试验。”

Both FDA programmes are aimed at encouraging the development of treatments for rare diseases.

这两个FDA项目都旨在鼓励罕见病治疗药物的研发。

Discovered by Kaerus, KER-0193 is a novel, orally-bioavailable small molecule designed to specifically address hyper-excitability of brain function commonly associated with Fragile X syndrome. KER-0193 does this by targeting BK channels (calcium-activated potassium channels), which have been described as master regulators of excitability across the nervous system.

Kaerus发现的KER-0193是一种新型的、口服生物可利用的小分子，旨在专门应对与脆性X综合征常见的脑功能过度兴奋问题。KER-0193通过靶向BK通道（钙激活钾通道）来实现这一点，这些通道被认为是神经系统兴奋性的主要调节因子。

The genetic cause of FXS is directly linked to a reduction in BK channel function..

脆性X综合征的遗传原因直接与BK通道功能的降低有关。

Based on its broad efficacy profile in preclinical genetic models of FXS, Kaerus is excited by the therapeutic potential of KER-0193 to address the diversity of behavioural, sensory and cognitive challenges that adversely impact the lives of individuals with FXS.

基于其在FXS临床前遗传模型中的广泛疗效，Kaerus对KER-0193的治疗潜力感到兴奋，该药物有望应对FXS患者所面临的各种行为、感官和认知挑战，从而改善他们的生活。

Kaerus's conviction has been strengthened by a pre-planned sub-study, conducted during the Phase 1 trial, in which the effects of KER-0193 on brain activity in healthy volunteers were investigated using electroencephalography (EEG). The study convincingly demonstrated that KER-0193 enters the brain and produces pharmcodynamic effects on brain activity in regions often implicated in patients with FXS.

在第一阶段试验期间进行的一项预先计划的子研究进一步坚定了Kaerus的信心。该子研究使用脑电图（EEG）调查了KER-0193对健康志愿者大脑活动的影响。研究有力地证明，KER-0193能够进入大脑，并在通常与FXS患者相关的大脑区域对脑活动产生药效学影响。

These results replicated observations from EEG profiling of KER-0193 in preclinical animal studies, demonstrating proof of mechanism..

这些结果复制了临床前动物研究中 KER-0193 的 EEG 分析观察结果，证明了其作用机制。

Beyond FXS, reduced BK channel activity has been implicated in a number of other neurological conditions, including epilepsy and several rare genetic epileptic encephalopathies.

除了FXS，BK通道活性降低还与许多其他神经系统疾病有关，包括癫痫和几种罕见的遗传性癫痫性脑病。

Dr Ring said

林格博士说

: 'We are actively exploring opportunities to expand the therapeutic potential of our BK modulator platform to these other indications, where patient populations struggle with significant unmet treatment needs.'

“我们正在积极探索将我们的BK调节剂平台的治疗潜力扩展到其他适应症的机会，这些患者群体在治疗需求上存在显著的未满足情况。”

The company is finalising preparations for a Phase 2 proof-of-concept study in FXS patients.

公司正在为FXS患者进行第二阶段概念验证研究做最后的准备。

About Kaerus

关于Kaerus

Backed by Medicxi, Kaerus Bioscience Ltd is a clinical-stage biotechnology company committed to turning scientific advances into treatment realities for patients with rare genetic syndromes of neurodevelopment. Kaerus has developed a pipeline of targeted, small molecule therapeutics that address an underlying ion channel dysfunction in Fragile X syndrome, which is the most common inherited cause of intellectual disability and autism globally..

Kaerus Bioscience Ltd是一家临床阶段的生物技术公司，致力于将科学进展转化为治疗罕见神经发育遗传综合征患者的现实疗法，受到Medicxi的支持。Kaerus已经开发出一条针对性的小分子治疗管线，旨在解决脆性X综合征中潜在的离子通道功能障碍，该综合征是全球最常见的遗传性智力障碍和自闭症病因。

For more information about Kaerus, please visit

有关 Kaerus 的更多信息，请访问

www.kaerusbio.com

www.kaerusbio.com

About KER-0193

关于KER-0193

KER-0193 is a novel, proprietary, and orally-bioavailable small molecule modulator of BK channels discovered by Kaerus. KER-0193 specifically addresses abnormal function of BK channels linked to the genetic cause of Fragile X syndrome. KER-0193 has already demonstrated broad effects on improving syndrome-relevant behavioural, sensory and cognitive deficits observed in genetic animal models of Fragile X.

KER-0193是一种新型的、专有的、口服生物可利用的小分子BK通道调节剂，由Kaerus发现。KER-0193专门针对与脆性X综合征遗传原因相关的BK通道功能异常。KER-0193已经在脆性X综合征的基因动物模型中显示出对改善相关行为、感官和认知缺陷的广泛效果。

Fragile X syndrome, which affects about 1 in 7,000 males and 1 in 11,000 females, is the most common inherited cause of autism and intellectual disability globally. There are currently no approved treatments for Fragile X..

脆性X综合征是一种最常见的遗传性自闭症和智力障碍疾病，全球约每7000名男性中有1人，每11000名女性中有1人受到影响。目前尚无获批的脆性X综合征治疗方法。

About Medicxi

关于Medicxi

Medicxi is a healthcare-focused investment firm with the mission to create and invest in companies across the full drug development continuum. Leveraging deep expertise in drug development and company creation spanning over two decades, Medicxi invests in early and late-stage therapeutics with a product vision that can fulfil a clear unmet medical need..

Medicxi是一家专注于医疗保健的投资公司，其使命是创建并投资于覆盖整个药物开发领域的公司。凭借在药物开发和公司创立方面超过二十年的深厚专业知识，Medicxi投资于早期和晚期治疗领域，拥有能够满足明确未满足医疗需求的产品愿景。