The New Gold Rush in Brain Science

For a long time, brain science has been considered one of the most uncertain fields in terms of return on investment in pharmaceutical research and development. Whether it is Alzheimer's disease or mental illnesses such as schizophrenia, the complex pathogenic mechanisms have made this area a hotspot for R&D failures.

According to previous data from the Tufts Center for the Study of Drug Development, the research and development time for central nervous system drugs is 20% longer than that for non-central nervous system drugs, and the approval time is 38% longer. High risk and high failure rates have also once made capital and pharmaceutical companies hesitant to enter this field.

But in the past two years, an obvious change is taking place: the brain science track is heating up. The reason is that individual markets are reaching a turning point.

On the one hand, therapeutic drugs for diseases like AD are beginning to deliver commercial returns, with sales achieving exponential growth.

On the other hand, breakthroughs in frontier technologies such as small molecules, RNAi, cell therapy, and AI have revealed new possibilities to the market.

When technology and market certainty overlap, MNCs also start to act intensively, with frequent mergers, acquisitions, and cooperation activities, restarting investor confidence.

Brain science may usher in a new "gold rush."

/ 01 / The Track Gradually Heats Up

The most intuitive signal of the warming trend is the simultaneous increase in both research and development and transaction volumes.

Taking Alzheimer's disease (AD) as an example, the number of clinical trials has increased significantly. According to the latest article "2025 Alzheimer's Disease Drug Development Pipeline," 138 new drugs are currently being evaluated in 182 clinical trials, marking a 9% increase compared to 2024. Among these, 70% are potential therapies targeting entirely new mechanisms.

Moreover, areas related to mental illnesses such as schizophrenia, depression, and bipolar disorder are also gaining attention. According to IQVIA's "2025 Global R&D Trends," the three major disease areas of Alzheimer's disease, depression, and Parkinson's disease have each initiated over 200 new clinical trials in the past five years.

The trading that took place at the time further highlighted the level of activity.

In January 2025, Johnson & Johnson acquired Intra-Cellular Therapies for $14.6 billion. Its core asset, Caplyta, covers bipolar depression, schizophrenia, and major depressive disorder. On November 3, 2025, Roche and Manifold Bio reached a collaboration with total payments exceeding $2 billion, pointing to another path. By utilizing brain shuttle technology and a next-generation delivery platform, they aim to systematically address the blood-brain barrier bottleneck.

Although some people are leaving, there are still quite a few participants coming in.

/ 02 / Multiple Inflection Points Approaching

The trigger for the升温 is "confidence."

Although failure remains the norm, breakthroughs are starting to emerge in some areas, particularly in Alzheimer's disease.

Biogen's Leqembi achieved sales of $214 million in 2024, with the latest Q3 2025 sales reaching $121 million, representing an 82% year-over-year increase.

Not only are drugs being commercialized, but diagnostics are also driving industry growth.

In October 2025, the AD blood test method Elecsys® pTau181 jointly developed by Roche and Eli Lilly received FDA approval, which permits its use in primary healthcare institutions. Biogen stated that this product not only significantly improves the diagnostic efficiency of memory decline diseases but also promotes the popularity of Leqembi.

For the same AD indication, Lantheus announced in October 2025 that the NDA for its tracer MK-6240, used to evaluate Tau protein in the brains of AD patients, has been accepted by the FDA. The "visualization" of Tau protein will provide more precise disease management for AD diagnosis and treatment.

At the same time, emerging technical means such as brain-computer interfaces and brain stimulation devices have also been incorporated into the investment narrative of brain science.

Neuralink and Synchron brain-computer interfaces have successively entered human clinical trials, with Musk's Neuralink project further sparking interest in the field of neurotechnology.

Of course, scientific progress also provides a more solid foundation for this round of enthusiasm.

At this year's JPM conference, BioSpace interviewed investors who unanimously agreed that current advancements in pathology and disease understanding have enabled scientists and biotechnology companies to understand the brain better than ever before.

Among them, Guldan Bhutani, Managing Partner of MBX Capital, pointed out that the rapid maturation of plasma and cerebrospinal fluid biomarkers, as well as imaging agents, has enabled more refined patient stratification in brain science drug development, significantly improving the predictability of efficacy.

The Allen Brain Cell Atlas released by the Allen Institute for Brain Science has become the "common language" of global brain research. This "language" of brain gene expression is crucial for understanding the physiological and pathological processes of the brain, and has significantly accelerated the discovery of drug targets.

As Taylor Molenkov, Deputy Director of Data at the Allen Institute, said: "Single-cell genomics has opened up new avenues for us to understand cell types in the brain and to use these cells for targeted therapies."

It is worth noting that policies are also driving the progress of brain science. The brain initiatives launched by the United States, the European Union, and China demonstrate a high level of attention to brain science. During the period from 2014 to 2023, the U.S. government's investment in the "Brain Initiative" has exceeded 4 billion U.S. dollars. China's Brain Project officially started in 2021, with the first phase lasting five years and a planned budget of 5 billion yuan, with total funding expected to exceed 10 billion yuan over two phases.

Driven by capital, science, and policy, brain science is now encountering an unprecedented investment opportunity.

/ 03 / Embracing a New Era of Brain Science

Against this backdrop, brain science is entering a new treatment landscape.

The most typical change also comes from AD. The approval of two Aβ monoclonal antibodies, Lecanemab and Donanemab, has enabled AD treatment to achieve "from 0 to 1." Nowadays, the Tau target is gradually replacing the position of Aβ. The pathological accumulation of Tau protein is more strongly correlated with cognitive loss than Aβ, making it a key target for halting the deterioration of AD.

Currently, this target revolution has progressed to the second stage. The second-generation Tau antibodies significantly reduced the whole-brain accumulation of Tau in clinical trials by targeting the microtubule-binding region (MTBR). Among them, Eisai's second-generation Tau antibody E2814 has received FDA Fast Track designation, and recently, a clinical trial of E2814 in combination with Lecanemab has been initiated.

In the field of mental illness, multi-target systemic regulation is gradually replacing the traditional monoamine hypothesis strategy.

Lumateperone, acquired by Johnson & Johnson through the purchase of Intra-Cellular Therapies, is a once-daily oral antipsychotic medication approved by the FDA for the treatment of schizophrenia in adults. It exerts its therapeutic effects by acting on D2 receptors, 5-HT receptors, and the glutamatergic neurotransmitter system simultaneously. Compared to traditional antipsychotics, it is well-tolerated and more conducive to long-term treatment.

Another layer of structural change comes from technological updates. Drug forms such as ASO, RNAi, and brain-targeting antibodies are redefining the boundaries of drug treatment in brain science.

In the field of antibodies, a new generation of brain carrier technology is addressing the issue of the blood-brain barrier, with Alector Therapeutics' AL137 being a product of this approach. BIIB080 from Biogen represents an important attempt for ASO in the central nervous system, directly reducing Tau protein expression through ASO and intervening in the disease process at its source.

Alnylam's Mivelsiran has demonstrated the potential of RNAi technology to cross the blood-brain barrier, and ALN-HTT02, a drug from the same delivery platform, has further validated its scalability in the Huntington's disease program.

These "effective" attempts are not focused on a single product, but rather on platform capabilities that are replicable and scalable.

After decades of exploration, brain science remains a "new field," but the research enthusiasm and commercialization process are accelerating at an unprecedented speed, marking the arrival of a new "gold rush" era for the brain science industry.

       Title: The New Gold Rush in "Brain Science"