Shanghai, China and Boston, MA., – January 2, 2025 – NeuShen Therapeutics (“NeuShen” or the “Company”), a clinical-stage global biotechnology company focused on developing innovative therapies for neurological and psychiatric disorders, today announces that the U.S. Food and Drug Administration (“FDA”) has granted the Company with an Investigational New Drug (“IND”) clearance to proceed in the U.S. with the clinical development of NS-136, a selective M4 muscarinic acetylcholine receptor positive allosteric modulator (M4 PAM), for the treatment of agitation in Alzheimer’s disease (AAD). This marks a pivotal step forward in the global development of one of the Company’s lead assets and underscores a major milestone in its globalized strategy.

NS-136 is a novel selective M4 muscarinic acetylcholine receptor positive allosteric modulator (M4 PAM) with the potential to treat a broad range of psychiatric disorders, including agitation in Alzheimer’s disease (AAD), schizophrenia, etc. NS-136 has demonstrated a favorable safety and pharmacokinetic profile in preclinical and Phase 1 studies, as well as therapeutic efficacy in behavioral models for various psychiatric symptoms, positioning itself as a potential first-in-class and best-in-class next-generation antipsychotic.

NS-136 is currently undergoing a Phase 2 clinical trial in China for the treatment of schizophrenia. The newly approved Phase 2 clinical trial by the FDA is designed to evaluate the efficacy and safety of NS-136 in agitation in Alzheimer’s disease (AAD), which will adopt a randomized, double-blind, placebo-controlled, multicenter design. 

“FDA’s clearance to initiate Phase 2 study of NS-136 is a significant milestone for NeuShen and a testament to our R&D capabilities.” commented Dr. Joan Huaqiong Shen, founder and Chief Executive Officer of the Company. “As an innovative drug candidate targeting a novel mechanism of action, NS-136 has potential as a differentiated treatment option across multiple psychiatric indications. We look forward to initiating the international multicenter Phase 2 clinical trial of NS-136 in agitation in Alzheimer's disease soon and bringing this innovative therapy to patients worldwide as soon as possible.”

Alzheimer's disease is a severe neurodegenerative disorder that affects memory, thinking, and behavior, and is the leading cause of dementia in the elderly. As one of its most common neuropsychiatric symptoms, agitation affects up to 50% of patients with moderate to severe Alzheimer's disease. Manifestations include excessive motor activity, verbal aggression, irritability, and resistance to care. These symptoms substantially increase caregiver burden, accelerate institutionalization, and impair patient quality of life. Despite its high prevalence, treatment options for agitation in Alzheimer's disease remain limited, indicating high unmet clinical need.

Existing preclinical and clinical evidence supports the muscarinic acetylcholine receptor subtype 4 (M4) as a promising therapeutic target for neuropsychiatric symptoms, including agitation. M4 receptors play a pivotal role in modulating dopaminergic and glutamatergic neurotransmission within brain circuits governing behavioral regulation. Positive allosteric modulators (PAMs) of M4 selectively potentiate receptor activity in the presence of endogenous acetylcholine, restoring synaptic balance while minimizing cholinergic side effects that often accompany non-selective muscarinic agonists.

About NeuShen Therapeutics

NeuShen Therapeutics is a clinical-stage global biotechnology company focused on developing innovative therapies for neurological and psychiatric disorders. With R&D centers in Shanghai and Boston, the Company brings together an international team of R&D experts and is advised by a group seasoned leaders in academia and industry. NeuShen Therapeutics has established a globally competitive CNS drug pipeline including multiple programs with first- or best-in-class potentials, and its lead assets have advanced into Phase 2 clinical development.