Hangzhou, September 13, 2025 – The national clinical study coordination and initiation meeting for the investigational new drug "CB03-154," developed by Shanghai Zhimeng Biopharma, Inc. (hereinafter referred to as "Zhimeng Biopharma"), was successfully held in Hangzhou. Thirty-six investigators and industry experts from 15 research centers across China participated in this national investigator meeting both on-site and online. This clinical study is led by Principal Investigator Professor Fan Dongsheng, a renowned ALS expert and Director of the Department of Neurology at Peking University Third Hospital. It is a multicenter, randomized, double-blind, placebo-controlled Phase II/III adaptive clinical study, followed by an open-label extension study, evaluating the efficacy and safety of CB03-154 in adult patients with Amyotrophic Lateral Sclerosis.
CB03-154 is a novel, next-generation KCNQ2/3 potassium channel opener for central nervous system diseases, independently developed by Zhimeng Biopharma. In July this year, CB03-154 received clinical trial approval from the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) of China to conduct Phase II/III studies for ALS in China. Previously, CB03-154 tablets had been granted Orphan Drug Designation (ODD) by the U.S. FDA for the treatment of ALS patients.
Present at the meeting were Principal Investigators and department experts from 15 research units nationwide, including Professor Fan Dongsheng from Peking University Third Hospital, Professor Shang Huifang from West China Hospital of Sichuan University, Professor Jiang Haishan from Nanfang Hospital of Southern Medical University, Professor Feng Honglin from the First Affiliated Hospital of Harbin Medical University, Professor Liu Yaling from the Second Hospital of Hebei Medical University, Professor Wang Junling from Xiangya Hospital of Central South University, Professor Zou Zhangyu from Union Hospital affiliated with Fujian Medical University, Professor Niu Qi from Jiangsu Provincial People's Hospital, Professor Zhang Min from Tongji Hospital affiliated with Huazhong University of Science and Technology / Shanxi Bethune Hospital, Professor Cao Lili from Qilu Hospital of Shandong University, and Professor Yu Xuefan from the First Hospital of Jilin University. Also attending were Professor Mi Weifeng from Peking University Sixth Hospital, sponsor representatives including Zhimeng Biopharma's Chairman Mr. Chen Huanming, CMO Dr. Lu Zhihong, Medical Director Dr. Zhou Yanling, and Clinical Operations Director Mr. Ma Chaoming, as well as Boji Pharmaceutical's CMO Dr. Zhu Quan, Clinical Department III Directors Mr. Wang Yinguang and Mr. Niu Shen, and Clinical Department III Deputy Director Ms. Hu Wanping.
The meeting commenced with an opening address by Dr. Chen Huanming, Chairman and CEO of Zhimeng Biopharma. Dr. Chen introduced Zhimeng Biopharma's current status and its robust R&D pipeline. Zhimeng has consistently focused on research in liver diseases and central nervous system disorders, with the company's goal being the development of First-in-class/Best-in-class new drugs for major diseases. These drugs aim to address urgent clinical needs and possess international leadership and global competitiveness. Dr. Chen stated: "This meeting will allow us to listen to the valuable insights of clinical experts. We believe such discussions will undoubtedly facilitate the efficient execution of the Phase II trial, accelerate the new drug approval process, and bring new hope to ALS patients."
Professor Fan Dongsheng from Peking University Third Hospital expressed: "There remains a significant unmet clinical need for ALS treatments, and we have high expectations for CB03-154. Biomedicine, as a new quality productive force, requires strong collaboration between clinical research institutions, enterprises, and scientists to translate scientific achievements into practical applications. All participating research units in this study will undoubtedly work together in unity and with concerted effort. We look forward to the generation of compelling data from this project, hoping to bring innovative treatment options to patients as soon as possible."
Dr. Lu Zhihong, Chief Medical Officer of Zhimeng Biopharma, presented the research background and mechanism of action of CB03-154. She stated that KCNQ2/3 is a clinically valid target for efficacy in epilepsy and neuropathic pain, and has shown positive efficacy signals for ALS in preclinical in vitro and animal studies. CB03-154 is a next-generation KCNQ2/3 potassium channel opener. Previously, Phase I clinical trials in healthy subjects have been conducted in the US and Australia, along with a bridging trial in healthy subjects in China. Preliminary results from the Phase I clinical trials have demonstrated favorable tolerability and safety profiles, potentially overcoming the safety issues associated with first and second-generation drugs.
Dr. Zhou Yanling, Medical Director of Zhimeng Biopharma, provided a detailed introduction to the trial protocol. Addressing the current treatment landscape for ALS, Dr. Zhou pointed out: "As a severe neurodegenerative disease, ALS presents a huge unmet clinical need. The primary goals of ALS drug development are to delay disease progression, improve patients' quality of life, and extend survival." Attendees engaged in in-depth communication and exchange regarding the trial design, inclusion criteria, assessment procedures, and other aspects.
Investigators Chen Lu and Sun Can from Peking University Third Hospital, psychiatric expert Professor Mi Weifeng from Peking University Sixth Hospital, and Liu Jia from Boji Pharmaceutical conducted scale training for all participants. The training covered the ALS Functional Rating Scale-Revised (ALSFRS-R), the Rasch-built Overall Amyotrophic Lateral Sclerosis Disability Scale (ROADS), the Columbia-Suicide Severity Rating Scale (C-SSRS), the Hamilton Depression Rating Scale-17 items (HAMD-17), and guidelines for filling out the ALSAQ-40 patient self-assessment form, among others.
Subsequently, Mr. Ma Chaoming, Clinical Operations Director of Zhimeng Biopharma, provided an overview of the project's overall progress. He emphasized the importance of risk management and proposed specific risk management plans focusing on enrollment management and scale assessments, aiming to minimize risks for the clinical trial project.
As the meeting ended, the national lead investigator, Professor Fan Dongsheng from Peking University Third Hospital, delivered a vote of thanks on behalf of the investigators. He expressed gratitude for Zhimeng Biopharma's trust and the support of clinical research institutions. He noted that the experts had engaged in profound discussions on the clinical trial protocol and thanked everyone for their active participation and valuable suggestions. The research institutions are committed to advancing this project with high standards and efficiency, each fulfilling their responsibilities while maintaining close collaboration. "We look forward to the successful completion of the CB03-154 clinical project," Professor Fan said.
Finally, Dr. Chen Huanming, Chairman of Zhimeng Biopharma, summarized the seminar. He stated that thorough discussions had been held regarding the product's research background, protocol design, project progress, and risk control. "The research on this project has spanned nearly 20 years. Bringing this drug to market approval early and providing an effective treatment method for ALS patients is the shared aspiration of everyone who has contributed to this effort," Dr. Chen said. He sincerely thanked all the experts and collaborating units involved in the project. Zhimeng Biopharma will work together with all partners to advance the clinical project, hoping their efforts will benefit more ALS patients, offering them hope and courage for life.
