SHANGHAI, Oct. 20, 2023 /PRNewswire/ -- Shanghai Zhimeng Biopharma, Inc. ('Zhimeng'), announced that one of its innovative small-molecule KCNQ2/3 selective opener compounds (CB03), developed for the treatment of amyotrophic lateral sclerosis (ALS), received orphan drug designation (ODD) from US FDA.

上海，2023年10月20日/PRNewswire/-上海志门生物制药有限公司（“志门”）宣布，其创新的小分子KCNQ2/3选择性开放剂化合物（CB03）之一，用于治疗肌萎缩侧索硬化症（ALS），获得美国FDA的孤儿药指定（ODD）。

CB03 is also being developed for other central nervous diseases, such as refractory epilepsy, major depressive disorders (MDD), etc..

CB03也正在开发用于其他中枢神经疾病，例如难治性癫痫，重度抑郁症（MDD）等。。

CB03 is a candidate drug for the treatment of ALS and other central nerve system (CNS) diseases independently developed by Zhimeng. A phase 1 study for the evaluation of the safety, tolerability, and pharmacokinetics of CB03 in healthy subjects is currently ongoing in Australia and US. The phase 1 study is expected to complete the dosing phase (both single dose and multiple dose) before the end of 2023..

CB03是由志孟独立开发的用于治疗ALS和其他中枢神经系统（CNS）疾病的候选药物。目前正在澳大利亚和美国进行评估CB03在健康受试者中的安全性，耐受性和药代动力学的1期研究。预计1期研究将在给药阶段（单剂量和多剂量）之前完成。2023年底。。

'The orphan drug designation by US FDA for CB03 is an important milestone in its global clinical development for ALS and other central nervous system diseases,' said Dr. Huanming Chen, President and CEO of Zhimeng. 'Since ALS is a very serious disease without adequate treatment options, we hope our dedicated efforts will allow us to bring a safer and more effective medicine to ALS patients worldwide as the first indication for CB03.'.

Zhimeng总裁兼首席执行官Huanming Chen博士说：“美国FDA为CB03指定的孤儿药是其在ALS和其他中枢神经系统疾病全球临床开发中的一个重要里程碑。“由于ALS是一种非常严重的疾病，没有足够的治疗选择，我们希望我们的辛勤努力将使我们能够为全球ALS患者带来更安全，更有效的药物，作为CB03的首要适应症。”。

About CB03

关于CB03

Studies on neurons indicate that the hyperexcitability of neurons is the one of the important underlying causes associated with neurodegenerative and neuropsychic CNS diseases, including ALS, epilepsy, MDD, neuropathic pain, among others. As the most widely distributed and the most diverse group of ion channels, potassium (K+) channels are mainly involved in the modulation of neuronal excitability and the frequency and amplitude of action potential discharges.

对神经元的研究表明，神经元的过度兴奋是与神经退行性和神经精神性CNS疾病相关的重要潜在原因之一，包括ALS，癫痫，MDD，神经性疼痛等。钾离子通道作为分布最广，种类最多的离子通道，主要参与神经元兴奋性的调节以及动作电位放电的频率和幅度。

CB03 as a KCNQ2/3 potassium ion channel opener thus has its potential for become a safe and effective treatment option for ALS and other CNS diseases..

因此，CB03作为KCNQ2/3钾离子通道开放剂，有可能成为ALS和其他CNS疾病的安全有效的治疗选择。。

As a new generation of selective KCNQ2/3 potassium channel opener, CB03 showed better chemical and metabolic stability, higher biological/pharmacological activities in in vitro and ALS animal disease models, and more druggable pharmacokinetic properties. Thus, CB03 is unlikely to present the same safety concerns as other channel openers and warrant further clinical development to demonstrate potential benefits in the ALS patient population..

作为新一代选择性KCNQ2/3钾通道开放剂，CB03显示出更好的化学和代谢稳定性，体外和ALS动物疾病模型中更高的生物/药理活性，以及更多的可药物药代动力学特性。因此，CB03不太可能提出与其他渠道开放者相同的安全问题，并需要进一步的临床开发来证明ALS患者人群的潜在益处。。

About Amyotrophic lateral sclerosis (ALS)

关于肌萎缩性嵴髓侧索硬化症

ALS is a neurodegenerative disease that affects nerve cells in the brain and spinal cord. ALS causes loss of muscle control and the disease gets worse over time. The exact causes of ALS are still not known, but neuron hyperexcitability causing neuron damage may play an important role in disease initiation and progression.

ALS是一种神经退行性疾病，会影响大脑和脊髓中的神经细胞。ALS导致肌肉控制丧失，疾病随着时间的推移变得更糟。ALS的确切原因尚不清楚，但引起神经元损伤的神经元过度兴奋可能在疾病的发生和发展中起重要作用。

A small number (about 10%) of ALS cases are inherited..

少数（约10%）ALS病例是遗传的。。

ALS often begins with muscle twitching and weakness in an arm or leg, trouble swallowing or slurred speech. Eventually ALS affects control of the muscles needed to move, speak, eat, and breathe. A large percentage of ALS patients may only survive for 3-5 years after diagnosis of the disease. There are a few drugs approved for treating the symptoms of ALS or slowing down the disease progression, but currently no cure for this fatal disease..

ALS通常始于肌肉抽搐和手臂或腿部无力，吞咽困难或言语不清。最终，ALS影响对移动，说话，进食和呼吸所需肌肉的控制。大部分ALS患者只能在诊断出疾病后存活3-5年。有几种药物被批准用于治疗ALS症状或减缓疾病进展，但目前无法治愈这种致命疾病。。

About Zhimeng

关于志门

Zhimeng is a clinical-stage biopharmaceutical company committed to developing innovative drugs for the treatment of chronic hepatitis B (CHB) and severe neurological diseases with significant unmet medical needs. The company has recently announced the successful completion of the phase Ib clinical trial on its novel HBV capsid inhibitor, Canocapavir (ZM-H1505R), and has started the phase II study in June 2022.  In addition, the clinical trial application of the company's TLR8 agonist (CB06) was approved by the FDA in December 2021, and the phase I study was launched in the United States in March 2022..

志孟是一家临床阶段的生物制药公司，致力于开发治疗慢性乙型肝炎（CHB）和严重神经系统疾病的创新药物，这些药物的医疗需求尚未得到满足。该公司最近宣布成功完成其新型HBV衣壳抑制剂Canocapavir（ZM-H1505R）的Ib期临床试验，并于2022年6月开始进行II期研究。此外，该公司的临床试验应用TLR8激动剂（CB06）于2021年12月获得FDA批准，I期研究于2022年3月在美国启动。。

Disclaimer

免责声明

This press release contains forward-looking statements. While Zhimeng considers the projections to be based on reasonable assumptions, these forward-looking statements may be called into question by a number of hazards and uncertainties, so that actual results may differ materially from those anticipated in such forward-looking statements..

本新闻稿包含前瞻性声明。虽然志梦认为这些预测是基于合理的假设，但这些前瞻性陈述可能会受到一些危害和不确定性的质疑，因此实际结果可能与这些前瞻性陈述中预期的结果大不相同。。

For more information, please visit: www.corebiopharma.com

欲了解更多信息，请访问：www.corebiopharma.com

SOURCE Shanghai Zhimeng Biopharma, Inc.

资料来源上海志门生物制药有限公司。