Experts say we can thank an improved understanding of the biology behind central nervous system (CNS) diseases for the resurgence of the sector, facilitating heightened confidence among investors to invest in earlier, more innovative therapies.
A panel comprised of pharma, biotech, and investor representatives agreed a more robust understanding of CNS pathology has been pivotal in revitalizing therapeutic innovation, speaking at the 2026 Nordic Life Science Days meeting in Stockholm, Sweden, 8 September. A shift in focus from symptoms to biomarkers in assessing efficacy has been especially key, they said, with investors relying on renewed data to invest earlier in the development of new therapies.
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Following a decade-long slump, the CNS therapeutic market now appears to have made a turnaround. The Alzheimer’s disease space offers a clear example; after few approvals between 2010 and 2020, Eisai’s Leqembi (lecanemab) launched in 2023, then Eli Lilly’s Kisunla (donanemab) in 2024, both potential blockbusters in coming years. There has been a notable uptick in M&A within the therapeutic area too – Johnson & Johnson’s (J&J) $14.6bn takeover of neuroscience specialist Intra-Cellular Therapies was the largest pharma deal in 2025.
This advancement has been marked, for one, by a shift from basing diagnoses on apparent symptoms in patients to a greater focus on the molecular pathology underlying disease, notes Maha Radhakrishnan, executive partner at US investment firm Sofinnova Investments. Specifically, she says increasing attention on biomarkers like neurofilament light chain has changed the way trials are run.
Biomarker signals can be seen far sooner than changes to symptoms, Radhakrishnan says. So too can they be reliably observed in smaller cohorts than are needed to assess functional disease changes, adds Kevin Dalgaard, senior principal at the Copenhagen, Denmark-based venture capital fund Lundbeckfonden BioCapital.
This means signals can be shown through biomarkers in earlier stage trials. In turn, investors are now able to make confident investments in new therapies far earlier in their development, says Radhakrishnan.
A better understanding of disease pathology has also changed how CNS candidates can transition from preclinical to clinical study, according to Antti Vuolanto, CEO of Finnish biotech Herantis Pharma. Tenuous links between symptomatic expression of disease in animal and human models can be supplanted by a more objective appreciation of how translatable the molecular biology between species is.
A focus on molecular biology could yield further benefits across indications, Radhakrishnan adds. As many CNS disease share aspects of pathology, she notes a candidate or mechanism validated in one may prove effective in another.
