May 19, 2025 – 4B Technologies(4B) announced that its independently developed, first-in-class (FIC) drug candidate 4B02-01 injection has been approved for entering clinical trial by the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA). The drug will be developed for the treatment of amyotrophic lateral sclerosis (ALS). The phase I trial will be conducted at the China-Japan Friendship Hospital, led by Principal Investigator (PI) Dr. Jintong Li.
4B Technologies is dedicated to developing innovative medicines for major unmet clinical needs in neurological diseases, with a particular focus on ALS, one of the world’s five major incurable diseases. Guided by a “patient-centric” R&D philosophy and a "multi-target, comprehensive coverage" strategy, 4B has built a differentiated pipeline with diverse mechanisms of actions. As the third ALS drug candidate to enter clinical development, 4B02-01 employs a unique TrkB activation mechanism for neuroprotection, complementing 4B’s other clinical-stage programs, 4B71-01 (a CSF1R inhibitor, modulating neuroinflammation) and 4B06-01 (AI-aided development of a modulator for neural transmission) to offer additional possibilities in tackling this tremendous medical challenge.
ALS is a life-threatening degenerative motor neuron disease characterized by damage to upper and lower motor neurons, progressive loss of motor function, muscle atrophy and weakness, ultimately leading to respiratory failure and death. From the onset of initial symptoms to the loss of speech, swallowing, and breathing, patients typically survive only 2-5 years. Globally, there are approximately 450,000 ALS patients, including 42,000 in China. The pathogenesis of about 90% of ALS cases remains unknown, and current drug treatments and comprehensive therapies can only extend survival by a few months or temporarily improve quality of life, highlighting an urgent unmet medical need.
Dr. Bai Lu, Founder of 4B，commented:
"Translating scientific discoveries into clinical applications is a challenging process, particularly for innovative neurological drugs. Applying original research on neurotrophic factors to clinical practice and ultimately benefiting patients has been the ultimate goal of my decades-long scientific career. Encouragingly, after years of in-depth research on TrkB, the receptor for brain-derived neurotrophic factor (BDNF), 4B has developed a TrkB-activating antibody, realizing the journey from basic research to translational medicine and clinical application—a vivid embodiment of the 'bench-to-bedside' concept. 4B02-01 overcomes the limitations of BDNF as a drug candidate, fully mimicking TrkB-mediated biological functions to provide neuroprotective and reparative effects by specifically activating downstream signaling pathways. We look forward to its clinical proof-of-concept (PoC) and hope it will bring tangible benefits to patients, potentially becoming a blockbuster therapy for major neurological disorders."
Dr. Ping Liu, CMO of 4B, said:
" In the field of ALS treatment, there is an urgent need to rapidly advance innovative drugs and therapies into the clinical stage for verification of safety and efficacy in order to break through the limitations of existing therapies and deliver significant clinical benefits to patients. 4B02-01 is an original drug developed based on over 20 years of research by Professor Bai Lu in neurotrophic factors, successfully translated into clinical development. With its novel target and mechanism, it holds great promise in achieving this goal. We are pleased that 4B02-01 has obtained CDE approval based on its promising preclinical efficacy and safety profile. We will spare no effort to initiate and advance Phase I trials promptly, striving to bring meaningful clinical benefits to ALS patients as soon as possible."