The Billion-Dollar CNS Track That Big Pharma Couldn't Crack: This Chinese Pharmaceutical Company Breaks Through from the Source Mechanism

The World Health Organization released the "Global Status Report on Neurology" showing that neurological diseases have affected more than 3 billion people worldwide, causing over 11 million deaths annually, and have become one of the top threats to global health.

Among them, the number of patients with CNS diseases such as depression and Alzheimer's disease is rapidly increasing, with hundreds of millions of patients in China alone. The "2022 National Depression Blue Paper" shows that the lifetime prevalence rate of adult depressive disorders in China is 6.8%, with a depression prevalence rate of 3.4%. Based on this, it is estimated that there are approximately 95 million people with depressive disorders in China, including about 40 million with depression. Each year, around 280,000 people die by suicide, of which 40% may suffer from depression. According to the "China Alzheimer's Disease Report 2025," the number of Alzheimer's disease and related dementia patients in China exceeded 13 million in 2019, with existing cases reaching 16.9908 million in 2021. By 2025, the scale of Alzheimer's disease patients aged 60 and above in China is expected to reach between 12 million and 14 million.

Obviously, due to the huge unmet clinical needs, the rigidity of demand, and the extremely high scientific barriers, CNS has become a strategic battleground for global MNCs and Biotechs. In the clinical field,According to the IQVIA report, by 2025, the total number of global CNS disease trials will approach 3,400, with China's share in global CNS clinical trials increasing from 6% during 2015–2019 to 13% during 2020–2024.In the market,According to a Precedence Research report, the global CNS drug market size is expected to reach USD 127.29 billion by 2025 and is projected to reach approximately USD 267.62 billion by 2034, with a compound annual growth rate (CAGR) of 8.64% from 2025 to 2034.

It is not difficult to see that the market prospect of CNS is no less than that of oncology and cardiovascular diseases. However, in contrast, the success rate of CNS drug development is only 6%, less than half of the success rate of other drug developments. Large pharmaceutical companies such as AstraZeneca (AZ), GlaxoSmithKline (GSK), AbbVie, Eli Lilly, Sanofi, Novo Nordisk have all encountered clinical failures or strategic adjustments in this field. To address the pain points facing this billion-dollar market, a Chinese Biotech company based in Shanghai —

Synphatec Biotech, is attempting to solve the industry's long-standing problems from the source with its unique "biological mechanism-driven" R&D philosophy.

In the CNS field, breakthroughs in "entirely new targets" mostly stem from the refined regulation of known biological mechanisms—while the basic frameworks, such as neurotransmitter systems, are already well-defined, precise fine-tuning of receptor subtypes and molecular structures can still give rise to innovative drugs with disruptive therapeutic effects. This research and development logic is becoming the core driving force behind innovation in the CNS field today. The story of Synphatec Biotech also begins with fundamental research into biological mechanisms.

In March 2019, the U.S. FDA approved the world's first fast-acting antidepressant drug — Janssen’s esketamine hydrochloride nasal spray (Spravato). This medication can alleviate depressive symptoms within hours, completely overturning the traditional treatment model where antidepressants take 2-4 weeks to take effect. However, the clinical application of esketamine has always been limited: as the S-enantiomer of ketamine, it carries inherent "original sins" — hallucinogenic effects, addiction potential, anesthetic side effects, and cardiovascular risks. These side effects have led to its classification as a Schedule I controlled substance, allowing patients to use it only in hospitals and clinics under strict supervision by doctors and nurses.

Dr. Geng Yang, co-founder and CEO of Synphatec Biotech, told VCBeat, "Although it is a nasal spray and sounds convenient for treatment, in fact, patients are strictly controlled during the process of using it. Patients need to use it under the supervision and guidance of doctors and nurses, and they have to lie down for at least two hours before leaving after spraying."

Dr. Geng Yang pointed out, "The more fundamental issue is that the antidepressant effect of esketamine was clinically discovered by accident, and its molecular mechanism has remained unclear for a long time. Due to the lack of clarity in its mechanism, optimizing it becomes very difficult—it’s like not having a target and not knowing what to do."

The mechanism of Esketamine's antidepressant effect is unclear, which is precisely the unexplored area that the founding team of Sleptai has chosen to focus on.Starting from the question "Why does ketamine have antidepressant effects," it took Silept's founding team 8 years just to identify the antidepressant target of ketamine and its mechanism of action.

The eight-year perseverance has finally yielded breakthrough results.

In September 2023, the research group of Chen Yelin and Geng Yang published a research paper titled "GluN2A mediates ketamine-induced rapid antidepressant-like responses" in the journal *Nature Neuroscience*. This study reveals for the first time internationally that GluN2A—the most important subtype of NMDA receptor—is the direct target of esketamine's antidepressant effects, rather than GluN2B, which has received more attention from the academic community. More importantly, the study found that the hallucinogenic effects of esketamine do not depend on GluN2A—this suggests the possibility of designing an ideal next-generation rapid-acting antidepressant molecule.

This research lays the theoretical foundation for the development of fast-acting antidepressant drugs without hallucinogenic side effects, and also becomes the core logic of Synphatec Biotech's antidepressant pipeline. In 2022, based on the scientific research achievements of Chen Yelin and Geng Yang's research groups, Synphatec Biotech was officially established, starting the transformation journey from laboratory to industrialization.

Unlike most Biotechs, SlipTech is not a platform-based company that is currently the most talked-about by the general public. Instead, it is an enterprise founded on biological innovation, which then adapts and builds a suitable platform through innovative mechanisms.

Dr. Geng Yang elaborated on this unique development strategy in an interview, "The currently more 'popular' platform companies actually have their own 'worries' — enterprises need to clearly define the细分领域 (specific segments) they want to layout, decide which targets to research, and determine what pipelines to layout based on those targets. But"The development of Silept doesn't rely on a single platform. We start from specific biological mechanisms, focusing on depression and Alzheimer's disease in the CNS field. Based on the unique mechanisms of these diseases, we adapt and establish suitable platforms to achieve the goal of disease intervention."

Based on this concept, Synphatec Biotech has successfully built two major technology platforms: the i-NaS small molecule platform and the SynX intracranial delivery platform.

The i-NaS platform is a small molecule drug screening platform developed by Synphatec Biotech specifically for psychiatric disorders such as depression. The core advantage of this platform lies in its clear target (NMDA receptor), defined mechanism, and a complete screening logic and process established accordingly. Based on this platform, Synphatec Biotech has laid out multiple pipelines for antidepressants and analgesics.

Its first pipeline product, SP-101 injection,It is a new rapid antidepressant drug developed by Silept,对标Johnson & Johnson's Spravato. The drug received implied clinical approval from the CDE in July 2025, with an approved indication for depression accompanied by acute suicidal ideation or behavior, and its safety has been preliminarily verified in Phase I clinical trials.Previous preclinical data showed, SP-101 takes effect in just 1 hour and has no side effects such as dissociation, anesthesia, or addiction. It is expected to become the safest rapid antidepressant drug globally, with Best-in-Disease potential.

Dr. Geng Yang introduced that, centered around the core target of NMDA receptors, Synphatec Biotech has subsequently laid out multiple indications, including pain and anxiety, "This is the logic behind the layout of our entire small molecule platform and pipeline."

Unlike depression, the treatment of Alzheimer's disease requires intervention in the pathological process of amyloid-beta (Aβ) in the brain, which involves how to effectively deliver large-molecule drugs into the cranium. To this end, Synphatec Biotech has built the SynX large-molecule intracranial delivery platform, covering two technical routes: viral platform and non-viral platform.

The core pipeline of this sector, SP-201,It is an innovative drug for Alzheimer's disease. Its mechanism is based on an important discovery published by Chen Yelin and Geng Yang's team in the journal *Neuron* in 2023: the C-terminal active fragment of ApoE is a substrate-selective inhibitor of γ-secretase, which can reduce the production of Aβ at its source. The difference in inhibitory potency, ApoE2 > ApoE3 > ApoE4, precisely explains the variation in Alzheimer's disease risk among populations with different ApoE genotypes.

SP-201 has preliminarily completed the verification of preclinical efficacy and safety. In AD mouse models and non-human primate models, SP-201 can rapidly and significantly reduce core biomarkers such as soluble Aβ, while the efficiency of plaque clearance is superior to marketed antibody drugs, demonstrating Best-in-Disease therapeutic potential.

Dr. Geng Yang emphasized that these two platforms were not "built for the sake of building platforms," but were developed based on clear disease mechanisms. This strategy of "establishing platforms based on mechanisms and expanding pipelines through platforms" allows Sleptai to maintain R&D focus while continuously expanding.The ability to generate innovative pipelines. Currently, SllepTech has successfully transformed from a single asset to a multi-pipeline + dual-platform model, with multiple pipelines nearing clinical Proof of Concept (POC), and its long-term value will be gradually unlocked.

Mentioning the development trend of the industry, Dr. Geng Yang said, "The CNS field is in a historic period of development opportunities."

From the perspective of clinical breakthroughs, the CNS field has recently entered a fruitful period of approvals: the successive approvals of Lecanemab and Donanemab mark the formal entry into the era of disease-modifying treatments for Alzheimer's disease; Zuranolone has been approved for marketing, becoming the first oral treatment for postpartum depression; KarXT has also been approved for marketing, becoming the first schizophrenia treatment drug with a new mechanism of action in over 70 years, providing a new option for schizophrenia treatment. These breakthroughs confirm that the "explosion after a long silence" in the CNS field is indeed happening.

From a capital perspective, CNS has become another hot track for China's Biotech going global, following oncology and autoimmune diseases. Since 2024, several Chinese CNS Biotechs have reached overseas licensing deals, with the involved amounts repeatedly setting new records. Behind this trend is the improvement in the original innovation capabilities of Chinese companies in CNS target discovery and mechanism research.

Facing this historical opportunity, Synphatec Biotech has formed a clear development strategy: in the R&D endAdhere to a differentiated route driven by bio-mechanism innovation, advancing with a strategy of dual filings in China and the U.S. and rapid Proof of Concept (POC);In the BD end of the pipelineMaintain an open attitude and actively expand cooperation with MNCs to leverage external experience to strengthen internal capabilities;On the team sideThe upgrade from a team of scientists to a "scientific research + industry composite team" has been completed, forming an efficient collaboration model of "university research team + enterprise transformation team."

Looking ahead, SlipTech's goals are clear and pragmatic: within the next one to two years, both core pipelines will advance to critical milestone nodes — SP-101 is expected to yield efficacy data from clinical patients, and SP-201 is anticipated to show key biomarker readings and potential disease progression impact data. In the long term, SlipTech aims to continuously iterate and expand its product pipeline around its two core platforms.

From an eight-year dedication to a single target point in academic research, to the industrialization layout of a dual-platform and multi-pipeline system, the growth trajectory of Sileptai reflects the typical path of China's CNS Biotech sector transitioning from following innovation to pioneering innovation. On the eve of the explosion of the billion-dollar CNS market, this company, labeled as "innovation biology-driven," is standing at a critical juncture for value realization.