The defining idea of 2025 was that CNS innovation became an execution game—pairing disease-modifying ambition with deployable pathways: home dosing, safer monitoring, and diagnostics that can scale.
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The year’s storyline: Three Neuroscience Shifts that kept repeating in 2025
Disease modification moved from “frontier” to “framework”
Alzheimer’s decisions increasingly revolved around eligibility, monitoring, and stopping rules—turning treatment into a pathway rather than a single prescribing event.
Multiple sclerosis development became disability-first
MS momentum increasingly focused on relapse-independent disability progression and progressive phenotypes, supported by BTK inhibitor programs and longer-horizon durability datasets in established classes.
Diagnostics became infrastructure
Blood-based Alzheimer’s biomarkers, MS decision-support assays, digital biomarkers, and workflow-integrated tools increasingly shaped who gets treated, how risk is managed, and how outcomes are tracked.
Practice-Shaping Clinical Readouts in Neuroscience That Defined 2025
1) Tolebrutinib in nrSPMS: a disability progression signal in a limited-option setting
Tolebrutinib was reported to delay six-month confirmed disability progression by 31 percent in non-relapsing secondary progressive MS (nrSPMS). A pooled analysis across GEMINI 1 and 2 (relapsing MS) reported disability worsening delayed by 29 percent versus Aubagio, despite the trials missing their primary endpoint.
2) BRIUMVI long-term durability: six-year outcomes as differentiators
In six-year follow-up for relapsing MS, 89.9 percent of patients were reported to remain free from disability progression, with annualized relapse rate declining to 0.012 in year six and safety described as consistent.
3) Real-world comparative safety: ocrelizumab vs rituximab in practice-adjacent evidence
An observational multicenter EHR analysis of more than 1,400 adults across six California sites, using a simulated target trial approach, reported lower rates of hospitalization, infections, and hypogammaglobulinemia with ocrelizumab versus rituximab.
4) Migraine: atogepant’s acute positioning signal (ECLIPSE)
In AbbVie’s Phase 3 ECLIPSE study for acute migraine treatment, 24.3 percent achieved pain freedom at two hours versus 13.1 percent on placebo. EMA approval was pending.
5) Imaging-driven MS efficacy: obexelimab (MoonStone) lesion reduction
In the Phase 2 MoonStone study, weeks 8–12 imaging showed a 95 percent relative reduction in new gadolinium-enhancing T1 lesions versus placebo (p=0.0009), alongside reduced T2 lesion burden; safety was described largely as mild injection-site reactions.
Neuroscience Approvals, guidelines, and access decisions that moved in 2025
Europe: Alzheimer’s access expands with defined guardrails
The European Union approved Leqembi (lecanemab) for early-stage Alzheimer’s disease across EU member states plus Iceland, Liechtenstein, and Norway.
The European Commission also authorized donanemab (Kisunla) with eligibility defined by ApoE4 status and with treatment cessation once plaques are minimal.
United States: home dosing expands the Alzheimer’s delivery model
The FDA approved LEQEMBI IQLIK for weekly at-home subcutaneous maintenance dosing in early Alzheimer’s disease.
Multiple sclerosis: regulatory movement and guideline actions across key markets
The UAE was described as the first country to approve tolebrutinib for nrSPMS, and the FDA review timeline was tracked through 2025, including a target action date of December 28, 2025.
In the UK, NICE recommended cladribine as an MS option, including relevance for patients considering pregnancy via a two-course annual regimen.
US Migraine and neurology tools: drugs, digital therapies, and pediatric expansion
The FDA approved Atzumi (dihydroergotamine nasal powder) for acute migraine in adults and cleared CT-132, described as the first digital therapy to prevent episodic migraine as an adjunct to standard medications. The FDA also approved AJOVY for episodic migraine prevention in pediatric patients aged 6 to 17 meeting weight criteria.
Broader neurology: Selected US and EU approvals
The FDA approved Koselugo (selumetinib) for symptomatic, inoperable NF1 plexiform neurofibromas in adults.
Europe saw an approval for VYVGART in CIDP.
Safety and Delivery Activity in Neuroscience in 2025: Keeping Patients on Therapy
Biosimilar switching stress-tested real-world implementation
In the UK, MS patients reported severe side effects after switching from Tysabri to the biosimilar Tyruko under an NHS cost-saving initiative, with more than 170 patients raising concerns and an ongoing regulator review noted.
High-sensitivity safety signals changed program trajectories
Sarepta paused shipments of Elevidys after safety concerns following a patient death due to liver failure in a Duchenne program.
Long-term medication risk signals widened the monitoring lens
A study cited an association between gabapentin exposure (six or more prescriptions) and higher dementia risk, alongside increased mild cognitive impairment.
Convenience upgrades were positioned as adherence interventions
Weekly at-home Alzheimer’s maintenance dosing for Leqembi and efforts to shorten administration for MS therapies (including a referenced path toward a 10-minute subcutaneous formulation in South Korea) reflect the growing linkage between delivery format, persistence, and real-world outcomes.
Diagnostics, stratification, and measurement in Neuroscience in 2025: better matching and better endpoints
Alzheimer’s biomarker testing accelerated into routine workflows
Quest launched AD-Detect, measuring amyloid beta and p-tau217 and generating a proprietary likelihood score.
The FDA approved the Elecsys blood test (Roche and Eli Lilly) measuring pTau181 for people aged fifty-five and older with cognitive decline signs, reporting a 97.9% negative predictive value for ruling out Alzheimer’s disease.
C2N Diagnostics submitted an Alzheimer’s blood test for FDA review using high-resolution mass spectrometry to quantify plasma amyloid and tau peptides with proprietary algorithms.
MS decision tools became practice-relevant
Octave’s MSDA biomarker test influenced clinical decisions in nearly sixty percent of cases and led to treatment modification in one in five.
Digital biomarkers and CNS diagnostics broadened the measurement stack
Cumulus reported NeuLogiq digital biomarkers outperforming ADAS-Cog for detecting Alzheimer’s disease and highlighted scalable remote EEG monitoring. Plus Therapeutics launched CNSide, a CSF diagnostic assay for CNS metastases, reporting high sensitivity and specificity and influencing treatment decisions in early rollout sites.
Neuroscience 2025 Catalyst calendar (only timelines explicitly stated)
Late 2025
- FDA target action date for tolebrutinib: December 28, 2025.
January 2026
- Denali’s tividenofusp alfa (Hunter syndrome with neurological symptoms): PDUFA date January 5, 2026.
Q1 2026
- uniQure AMT-130 (Huntington’s disease): BLA planned Q1 2026 (with a 36-month disease-slowing signal versus external controls cited).
First half of 2026
- REGENXBIO RGX-202 (Duchenne muscular dystrophy): topline data expected first half of 2026.
Key Neuroscience Takeaways From 2025
Neuroscience in 2025 wasn’t defined by a single molecule—it was defined by a systems shift. Alzheimer’s moved toward pathway-based care (including Europe-wide decisions and at-home maintenance dosing), MS development leaned harder into disability biology and long-horizon outcomes, and diagnostics became deployable infrastructure (blood tests, MS activity assays, digital biomarkers). The next winners will pair credible efficacy with scalable delivery, monitorable safety, and a clear biomarker-to-decision pathway.
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