NeuShen Therapeutics Emerges as a CNS Innovator with Dual-Platform Pipeline as the $100B+ CNS Market Heats Up

In the final month of 2023 alone, the CNS field witnessed two consecutive blockbuster deals:BMS acquired Karuna for $14 billion, gaining access to its schizophrenia drug KarXT, which is currently under FDA review and is projected by analysts to reach peak annual sales of up to $6 billion. AbbVie acquired Cerevel and its neuroscience pipeline for $8.7 billion; this pipeline includes multiple clinical-stage and preclinical candidates, most notably the M4 receptor agonist emraclidine, which targets negative symptoms of schizophrenia and cognitive impairment.

Furthermore, a social anxiety drug named Fasedienol (PH94B) also “broke out of the circle” in 2023.The reason is that Vistagen, the company developing the product, announced positive top-line results from its Phase 3 PALISADE-2 clinical trial of fasedienol nasal spray for the treatment of social anxiety disorder (SAD). The data released represent the “first positive Phase 3 study” of a therapeutic approach for SAD in the United States in 15 years, driving the company’s stock price to surge consecutively last August.

Neuroscience, the jewel in the crown of science and the final frontier in humanity’s exploration of nature, is on the verge of an explosion, driven by years of accumulated progress in basic research and industrial translation. As a biotech company dedicated to developing innovative CNS drugs, NeuShen Therapeutics has rapidly delivered its answer, leveraging over 20 years of CNS pharmaceutical experience among its team members: within less than two years of its establishment, two projects in its pipeline have advanced to the IND/clinical trial application stage.

NeuShen Therapeutics is a CNS-focused innovative drug company founded in 2022, with Dr. Huaqiong Shen as its founder and CEO.

When it comes to Dr. Shen, public records are replete with labels and highlights associated with her: “academic prodigy who entered university in her first year of high school,” “an accidental journey into medicine,” “2020 PharmaBoardroom Women Leader of the Year,” “one of Endpoints News’ 20 Most Influential Women in Global Biopharma R&D in 2021,” “Forbes China’s Top 50 Women in Science and Technology 2021,” and “the sole female recipient of the BIO China Association’s Annual Leadership Award in 2022.”

For over two decades, Dr. Shen has focused on the CNS field, serving as an executive at multinational pharmaceutical companies such as Johnson & Johnson, Pfizer, Wyeth, and Eli Lilly. He has acted as the global project lead for multiple CNS compounds, including olanzapine for pediatric indications, bipolar disorder, duloxetine for anxiety disorders, and desvenlafaxine. His portfolio also includes first-in-class candidates that did not reach the market, such as vabicaserin for the treatment of psychosis and mood disorders, and the global clinical development of babineurumab, a monoclonal antibody for Alzheimer’s disease. In addition, Dr. Shen holds a board certification in psychiatry and neurology issued by the American Board of Psychiatry and Neurology and holds academic appointments at both Indiana University and Peking University.

Throughout his extensive career in scientific research and industry, the two experiences that left the deepest impression on Dr. Shen were, in fact, less “prominent” compared to the aforementioned achievements.

The first incident occurred while Dr. Shen was working as a psychiatrist in the United States.At that time, Dr. Shen encountered a patient with severe depression.Prior to consulting Dr. Shen, the patient had undergone psychological and pharmacological treatments with multiple psychiatrists, but with minimal efficacy. The primary reasons were the prolonged latency period for drug onset (approximately 4–6 weeks) or the emergence of intolerable side effects.

Dr. Shen recalled, “Unlike malignant diseases such as tumors, CNS disorders are not immediately life-threatening, but they impose a heavy burden on families and society. This young woman lost both her family and her job as a result. For this patient, I could only adjust her medication slowly and subtly, while providing psychological counseling to treat her gradually. At that time, I truly wished there were medications on the market with faster onset of action and fewer side effects. After three months of active cooperation with the treatment, the young woman excitedly brought her boyfriend to see me, saying, ‘Dr. Shen, you have changed my life!’”

A year later, the young woman had returned to her nursing position and happened to run into Dr. Shen at the hospital. “From a distance, she loudly greeted me and proudly told everyone around, ‘This is my doctor; she saved my life!’”“Although most people did not know her or me at the time, I truly experienced the sense of responsibility and fulfillment that comes with being a physician. I came to realize that healing is not limited to treating physical ailments; psychiatrists, too, can help patients who feel their lives are worse than death regain their vitality and radiance.”

The second event occurred during Dr. Shen’s tenure at Pfizer. At that time, she proactively initiated an effort to engage China in global clinical research and development.At the Global Alzheimer’s Disease Conference held in Hawaii, she led her team in organizing a satellite symposium on Asian clinical development. Amidst a crowded conference schedule, she meticulously managed every detail—from the design of the invitations and venue selection to the careful curation of content and communication with participating experts—to ensure international attendance. As a result, the symposium was highly successful.

The experts joked, “We were moved by your indomitable spirit to attend this conference, and only after arriving did we realize how excellent it was and how much we had gained.” Many of these experts became lifelong friends, and Pfizer’s senior management consequently gave Dr. Shen greater recognition and support. Later, they worked together with these experts to facilitate the clinical trial for the validation of the Asian Alzheimer’s Disease Scale.

It was precisely these experiences and capabilities, coupled with a character marked by a willingness to embrace challenges and a commitment to excellence, that made the founding of NeuShen Therapeutics a natural choice for Dr. Shen.

In addition to Dr. Shen, every core member of NeuShen Therapeutics boasts extensive experience in scientific research and industry development. The team members have previously led the project initiation, IND filing, and NDA submission for dozens of CNS programs.

Co-founder and Director, Dr. Mike PooleWith over 20 years of industry experience, he is the founder and Head of R&D at Neumora. Neumora, a highly anticipated company, successfully listed on the Nasdaq last September and currently has a market capitalization of approximately $2.8 billion. He previously served as Chief of Staff and Senior Advisor to the President of Global Health at the Bill & Melinda Gates Foundation, and held positions such as Vice President of Central Nervous System Drug Development and Therapeutic Area Head at AstraZeneca, Pfizer, and Wyeth.

Dr. Wu Lingyun, Senior Vice President of the Chemistry Department at NeuShen TherapeuticsHe is also an industry veteran with over 20 years of experience in CNS drug discovery. He graduated from the Department of Chemistry at Tsinghua University, earned his Ph.D. from Princeton University, and completed postdoctoral training at Yale University. He later held key R&D positions at Lundbeck, a renowned Danish CNS pharmaceutical company, and WuXi AppTec.Dr. Zhang Chao, Chief Operating OfficerGraduated from the Department of Biology at Fudan University and earned a Ph.D. from the Shanghai Institute of Materia Medica, Chinese Academy of Sciences. Possesses nearly 20 years of R&D experience in multinational pharmaceutical and biotechnology companies, along with extensive expertise in project management and corporate operations.

An excellent and complementary team naturally gains recognition from capital markets. In its founding year, NeuShen Therapeutics completed a Pre-A financing round of approximately USD 20 million, with investors including well-known firms such as Longpan Investment, Yisheng Capital, and Qiji Investment.

 Image source: NeuShen Therapeutics

With dual support from funding and its team, NeuShen Therapeutics rapidly establishedAdeno-associated virus (AAV) gene therapy platform NeuGem™ and small-molecule drug development platform NeuChime™.

It is worth noting that most of the currently commonly used capsid engineering platforms are characterized by long development cycles, low yields, and labor-intensive processes.NeuShen Therapeutics’ capsid engineering platform features rational design, artificial intelligence, and directed evolution, enabling innovative gene expression cassette design to develop gene therapies with enhanced safety and efficacy.Leveraging the NeuGem™ technology platform, researchers can conduct next-generation screening of AAV capsids for blood-brain barrier crossing in non-human primates to achieve enhanced central nervous system transduction and reduced hepatotoxicity, thereby developing AAV-compatible microRNA scaffolds for effective and precise gene silencing.

Currently, NeuShen Therapeutics’ AAV capsid engineering platform has yielded highly positive data in primate studies, with multiple abstracts submitted to the annual meeting of the American Society of Gene & Cell Therapy (ASGCT). Additionally, the company has achieved a significant milestone in its collaborative project on ALS with the University of Massachusetts, and one research finding has been presented at the annual meeting of the European Society of Gene & Cell Therapy (ESGCT).

NeuShen Therapeutics’ small-molecule drug platform R&D strategy integrates traditional small-molecule drug discovery and optimization with AI-assisted drug discovery technologies, rapidly addressing current therapeutic gaps by driving incremental innovation on validated targets to enhance efficiency and creativity.

Based on this platform,NeuShen Therapeutics has a schizophrenia pipeline candidate, NS-136.This pipeline candidate is a muscarinic acetylcholine receptor positive allosteric modulator (M4 Positive Allosteric Modulator). In various animal models, NS-136 has demonstrated efficacy superior to that of currently disclosed competitors, with advantages including improvement of both positive and negative symptoms, potential for enhancing cognitive function, and a favorable safety window.Currently, the clinical trial application for this pipeline in Australia has been submitted, and the Investigational New Drug (IND) application in China is expected to be submitted in Q1 of this year.

The IND application for NS-041, another core pipeline candidate of NeuShen Therapeutics, was accepted by the CDE in December 2023.NS-041 is a highly selective KCNQ2/3 activator with global independent intellectual property rights, indicated for epilepsy and major depressive disorder. Preclinical studies have demonstrated that NS-041 exhibits significant antiepileptic and antidepressant effects in various animal models. Furthermore, the differentiated efficacy and safety profile of NS-041 observed in preclinical studies highlights its potential as a best-in-class KCNQ2/3 activator.

Why pursue a dual strategy in both small-molecule and gene therapy? Dr. Shen explained, “Our development of novel drugs for the central nervous system (CNS) is driven by patient needs. Certain challenges cannot be fully addressed by small molecules alone at this stage. The AAV gene therapy platform has made it possible to achieve breakthroughs in treating diseases that have long remained intractable. Under the guidance of Professor Guangping Gao, we have successfully established our proprietary technology platform and rapidly achieved encouraging progress. The clinical trials for candidate drugs derived from these different technologies share similar characteristics, which represents one of our core strengths. By building R&D teams simultaneously in China and the United States, and adopting a strategic layout encompassing two therapeutic modalities, multiple pipelines, and multiple indications, we are better positioned to fully leverage domesticStrong“Leverage execution capabilities and the advantages of Boston’s cutting-edge innovation to advance the pipeline more rapidly and effectively, striking an optimal balance between R&D risk mitigation and accelerated value realization.”

It should be emphasized that all projects initiated by NeuShen Therapeutics undergo rigorous project evaluation, with the aim of achieving a top-three global ranking in R&D speed while demonstrating superior efficacy and safety margins compared to other publicly disclosed pipelines.

On the other hand, Dr. Shen told VCBeat, “To advance the CNS pharmaceutical sector, the efforts of a single company are certainly insufficient. We aim to collaborate as extensively as possible with tens of millions of patients in China, rapidly growing pharmaceutical companies, and regulatory authorities, so as to build an ecosystem for research and translational innovation in novel drugs in China, thereby driving industry development.”

We look forward to Dr. Shen and NeuShen Therapeutics successfully overcoming new challenges, and bringing safer, more effective, and more accessible CNS drugs to the over 240 million patients with mental disorders in China at an early date.