Neuroscience is back. After a litany of divestments and scale backs in the 2010s, diseases of the brain are now the second-most-funded area in biotech, behind only cancer. However, this remains a complex and challenging field, leading neuroscience biotechs – three of which are profiled below – to develop very different strategies.
The resurgence of neuroscience investment over the past five years is partly down to a better technological toolset. Genetics and biomarkers have matured enough to de-risk programs – whether in terms of identifying patient subgroups or measuring disease progression more objectively – while technology that can break through the blood-brain barrier (a historical barrier to drug delivery) is now a reality according to L.E.K. Consulting’s analysis of neuroscience drug development trends.
Investors and scientists are also drawing confidence from the first new drug approvals in decades for schizophrenia and Alzheimer’s disease in 2023/2024.
And Big Pharma is pouring money into acquiring neuroscience biotechs: BMS acquiring Karuna for USD 14 billion, AbbVie buying Cerevel for 9 billion, and Biogen picking up Reata for 7 billion in 2023. In 2025, J&J spent 14.6 billion on Intra-Cellular Therapies, while Novartis splurged 12 billion to acquire Avidity Biosciences.
Finally, although the recent regulatory approvals offer hope, there remains a huge level of unmet need for patients with neurological diseases, leaving plenty of potential targets for drug developers. More than one in three people globally live with these conditions and around 90 percent of rare neurological diseases have no disease-modifying therapy available at all.
How can the next wave of neurology biotechs hope to capitalise on this momentum?
Acadia Pharmaceuticals: Scaling to One Billion Alone
San Diego-based Acadia Pharmaceuticals is more advanced than most and has chosen to commercialise its products itself, rather than via partnerships. Of its two marketed molecules, Nuplazid treats hallucinations and delusions in Parkinson’s disease psychosis while DAYBUE is the first-ever approved treatment for Rett syndrome, a severe genetic disorder affecting mostly young girls.
Both Nuplazid and DAYBUE were already on the market when BMS and J&J alumni Catherine Owen Adams was drafted in as CEO in 2024, but she has since overseen a dramatic scale up of the company’s commercial operations. Acadia’s full-year revenue guidance for 2026 is now between USD 1.24 and 1.3 billion while DAYBUE sales reached USD 125 million (up 30 percent from 2025) in Q2 2026. NUPLAZID generated USD 183 million (up 10 percent) over the same period.
The company is now targeting USD 1.7 billion in annual net sales by 2028, with Adams – a first-time biotech CEO – confident that there is still space in which to grow. “While it is difficult to precisely estimate market share, we believe that only around 20 percent of patients who could benefit from Nuplazid [in the US] are currently receiving it,” she told PharmaBoardroom earlier this year.
Adams is well placed to comment on the impact that this type of medication can have. A caregiver to two parents, one with Alzheimer’s disease and one with Lewy body disease, she describes psychosis as “a very real part of my life over the past several years”
While Nuplazid (launched in 2016) is approaching the end of its patent protection in the US, DAYBUE (2023) is a much younger product, and Adams has bold plans to take it global. The European Medicines Agency’s scientific committee gave a positive recommendation for DAYBUE earlier this year and, if launched, it would be the first approved Rett syndrome treatment in the EU.
Since our conversation, Acadia has added a new drug to its pipeline – remlifanserin, targeting psychosis in Alzheimer’s disease – with initial Phase II trial results due in September–October 2026 and an estimated peak sales potential of up to USD four billion if approved.
Denali Therapeutics: Breaking through the Blood-Brain Barrier
Also based in California, Denali Therapeutics has spent over a decade trying to get drugs across the blood-brain barrier, the body’s natural defence that normally keeps large therapeutic molecules out of the brain.
CEO Ryan Watts, a developmental neurobiologist by training, has been consumed with this challenge for over two decades, eventually leaving a senior role at Roche/Genentech to found his own firm 11 years ago. Watts and the Denali team then developed a proprietary ‘TransportVehicle’ technology, which hitches a ride into the brain using the body’s own iron-transport system, and were rewarded with an FDA approval earlier this year.
Sales for this first drug, which treats the rare genetic disease Hunter syndrome, have thus far been modest, sitting at around USD 3.6 million in its first full commercial quarter. However, Denali has said that patient interest exceeded its own internal projections and expects sales to roughly triple next quarter.
More significantly, with the technology now proven in a disease for which an enzyme therapy already existed, Watts is confident about Denali’s ability to go after more challenging (and prevalent) targets.
“Working first in rare diseases with neurologic manifestations like Hunter syndrome allowed us to take biology off the table, focus on the brain delivery and biomarker challenges, and then return to the harder biology questions in diseases like Alzheimer’s, Parkinson’s, and ALS with better tools and a clearer framework,” says Watts.
Neurvati Neurosciences: Trashed to Treasure
Neurvati Neurosciences represents another approach within neuroscience: acquiring molecules discarded by other firms and building companies around them.
“Neuroscience has always demanded significant capital, long development timelines, and a willingness to persist through uncertainty,” explains President and CEO Bruce Leuchter.
“Our approach is to identify mid- to late-stage product candidates, typically Phase II or III, from large- and mid-cap biopharma companies,” he continues. “These programmes require substantial resources to complete global trials and are often externalised for reasons ranging from portfolio reshaping to budget constraints.”
Leuchter explains that thanks to this strategy and the backing of Blackstone Life Sciences, Neurvati can avoid the pitfalls of traditional venture-backed outfits. While other firms generally focus on preclinical or early-stage assets and must repeatedly attract financing. Neurvati can “eliminate capital markets risk and move forward with both committed funding and operational readiness.”
The first proof of concept is GRIN Therapeutics, which is developing a drug called radiprodil for GRIN-related neurodevelopmental disorder, a rare and severe childhood epilepsy and developmental condition caused by a specific gene mutation. Blackstone has committed USD 200 million to fund the development of radiprodil, currently in Phase III trials.
If the GRIN project succeeds in bringing a product to market, Leuchter envisions Neurvati becoming “a genuine port of call for neuroscience companies developing promising therapeutics and seeking alternative pathways.”
