Regulatory momentum, precision diagnostics, and late-stage CNS execution accelerate across neurology
Today’s NeuroPulse reflects a neurology sector translating scientific validation into regulatory action, late-stage execution, and scalable infrastructure. Across Alzheimer’s disease, stroke, autism, ALS, epilepsy, and rare neurodevelopmental conditions, the common thread is clear: platforms enabling earlier diagnosis, biologically targeted intervention, and real-world evidence generation are moving from promise to deployment.
CAMP4 and GSK Partner on RNA Therapies
Pulse:
• CAMP4 entered a strategic research and licensing partnership with GSK to develop antisense oligonucleotide therapies for neurodegenerative and renal diseases, highlighting pharma’s expanding interest in RNA amplification beyond traditional gene-silencing approaches.
• The collaboration leverages CAMP4’s RAP Platform®, which maps regulatory RNAs to selectively increase gene expression in diseases driven by protein under-expression.
• CAMP4 will receive $17.5 million upfront, with eligibility for development and commercial milestones, along with tiered royalties on future product sales.
• GSK will lead downstream development and commercialization, reinforcing its long-term commitment to RNA-based therapeutic pipelines in neurology
CranioSense Secures $5.5M to Advance Noninvasive Brain Pressure Monitoring
Pulse:
• CranioSense was awarded $5.5 million in non-dilutive funding from the NIH and U.S. Department of Defense to advance its noninvasive intracranial pressure monitoring technology.
• The device aims to deliver real-time brain pressure assessment using optical sensors and proprietary algorithms, addressing a major unmet need in acute care.
• Funding will support pivotal validation studies and regulatory milestones ahead of FDA submission.
• The platform has the potential to transform emergency and military neurotrauma care by making brain pressure monitoring as routine as blood pressure measurement
Health Canada Authorizes TNKase for Acute Ischemic Stroke
Pulse:
• Health Canada granted market authorization for TNKase® (tenecteplase) for the treatment of acute ischemic stroke, marking the first new stroke therapy approved in Canada in nearly 27 years.
• The approval is based on the AcT trial, which demonstrated non-inferiority to alteplase while offering a simpler, faster single-bolus administration.
• TNKase has already been incorporated into Canadian Stroke Best Practices recommendations as an alternative thrombolytic option.
• The decision reflects growing regulatory acceptance of streamlined acute stroke workflows that improve speed, access, and system-level efficiency
CorestemChemon Advances ALS Commercialization Strategy
Pulse:
• CorestemChemon advanced regulatory review in Korea for its ALS stem cell therapy Neuronata-R, refining its approval strategy to a slow-progressor ALS subgroup supported by post-hoc Phase 3 analyses.
• Subgroup data showed stabilization of functional scores, preserved respiratory capacity, and favorable neurofilament biomarker trends, strengthening engagement with the MFDS.
• In parallel, the company entered an NSF-backed U.S. regenerative medicine ecosystem, establishing infrastructure for FDA engagement and targeting a BLA submission in 2026.
• Commercial readiness is supported by KRW 26.1 billion in new capital and a significant CEO share purchase, signaling confidence at a late-stage regulatory inflection point
Anavex Requests EMA Re-Examination for Alzheimer’s Drug
Pulse:
• Anavex submitted a formal request for the EMA to re-examine its opinion on blarcamesine for early Alzheimer’s disease.
• The re-examination will be conducted by a new rapporteur team, with Anavex also requesting input from an independent Scientific Advisory Group.
• The move reflects continued regulatory engagement rather than program termination following a negative initial opinion.
• Anavex is positioning blarcamesine as an oral therapy targeting upstream disease mechanisms via restoration of cellular homeostasis
Actinogen Completes Enrollment in Pivotal Alzheimer’s Trial
Pulse:
• Actinogen randomized its final participant in the XanaMIA Phase 2b/3 Alzheimer’s trial, closing enrollment ahead of schedule with 247 patients.
• The study evaluates once-daily oral Xanamem over 36 weeks in mild-to-moderate Alzheimer’s disease, with disease confirmation using blood-based pTau biomarkers.
• An independent interim safety and futility analysis is planned for January 2026, with topline final results expected in November 2026.
• Completion of enrollment positions Actinogen to generate late-stage data for a differentiated, non-antibody Alzheimer’s mechanism targeting brain cortisol regulation
Brainomix AI Reveals Efficacy Signal in Stroke Phase 2 Trial
Pulse:
• Brainomix applied its FDA-cleared AI imaging platform to analyze Argenica’s Phase 2 trial of neuroprotective drug ARG-007 in acute ischemic stroke.
• Standardized AI-based baseline imaging reduced variability in stroke severity assessment, revealing statistically significant efficacy in patients with severe strokes.
• Treated patients showed improved neurological outcomes at 24 hours and 90 days, along with smaller final infarct volumes.
• The findings support precision patient selection and highlight how AI imaging can unlock hidden drug signals in complex neuro trials
MediciNova Completes Enrollment in Phase 2 Neuropathy Trial
Pulse:
• MediciNova completed patient enrollment in its randomized Phase 2 OXTOX trial evaluating MN-166 (ibudilast) for the prevention of chemotherapy-induced peripheral neuropathy.
• The study enrolled 100 metastatic colorectal cancer patients across 11 Australian sites and is investigator-initiated.
• Participants will continue treatment through chemotherapy, with topline data expected in 2026 once the six-month follow-up is completed.
• The trial expands MN-166’s clinical footprint beyond neurodegeneration into supportive oncology care, where effective neuropathy prevention remains an unmet need
ProMIS Exceeds Target Enrollment in Alzheimer’s Phase 1b Trial
Pulse:
• ProMIS Neurosciences exceeded enrollment targets in its PRECISE-AD Phase 1b trial of PMN310, enrolling 144 patients with early Alzheimer’s disease.
• PMN310 is designed to selectively target toxic soluble amyloid-beta oligomers while avoiding plaque binding, to reduce ARIA risk.
• The trial has shown a favorable safety profile to date, with no treatment-related serious adverse events reported.
• Blinded interim biomarker data are expected in Q2 2026, with final unblinded topline results anticipated in Q4 2026
Gain Therapeutics Reports CNS Target Engagement in Parkinson’s Phase 1b Study
Pulse:
• Gain Therapeutics reported a reduction of glucosylsphingosine in cerebrospinal fluid in its Phase 1b study of GT-02287 in Parkinson’s disease.
• The biomarker change provides first-in-class evidence of increased GCase activity in the brain following treatment with a GCase modulator.
• The study showed favorable tolerability and supported continued dosing in a nine-month extension phase.
• The data strengthen the case for GT-02287 as a potential disease-modifying approach in Parkinson’s linked to lysosomal dysfunction
ALZpath Blood Test Enables Landmark Alzheimer’s Prevalence Study
Pulse:
• ALZpath’s pTau217 blood test was used in a large population-based study published in Nature to assess Alzheimer’s disease prevalence at scale.
• The study analyzed more than 11,000 samples, showing that over one in ten adults above age 70 may be eligible for disease-modifying treatment.
• Results demonstrated that blood-based biomarkers can reliably detect Alzheimer’s neuropathology across asymptomatic and symptomatic populations.
• The findings support broader adoption of blood diagnostics to enable earlier diagnosis and scalable screening strategies
MARAbio Launches First Blood Test for Maternal Autoantibody–Related Autism
Pulse:
• MARAbio commercially launched the MAR-Autism™ Test, the first blood test designed to identify maternal autoantibodies that cause a biologically defined subtype of autism known as MARA.
• Research indicates MARA may account for up to one in five autism cases, with autoantibodies crossing the placenta and disrupting fetal brain development.
• Clinical validation showed ≥99% specificity for pathogenic autoantibody patterns, enabling early risk identification in mothers before or after pregnancy.
• The launch positions blood-based immunodiagnostics as a new entry point for autism risk stratification and early intervention strategies
Brain-Based Stroke and Neurotech Infrastructure Expands
Pulse:
• Across today’s updates, regulators, sponsors, and developers are converging on faster stroke intervention, precision neurology trials, and scalable diagnostics.
• AI imaging, noninvasive monitoring, and biomarker-driven trial design are increasingly shaping regulatory and clinical decision-making.
• Late-stage trials and approvals signal maturation across stroke and neurodegeneration pipelines.
• Together, these developments reflect neurology’s shift from exploratory innovation to operational execution.
Thermo Fisher Launches Global Alzheimer’s Real-World Evidence Registry
Pulse:
• Thermo Fisher enrolled the first patient in its PPD™ CorEvitas™ Alzheimer’s Disease Registry, a global initiative designed to generate regulator-grade real-world evidence.
• The longitudinal registry will track treatment patterns, long-term safety, imaging findings, and clinical outcomes across routine care settings.
• MRI-based monitoring of brain bleeding, swelling, and plaque clearance addresses regulatory scrutiny around disease-modifying Alzheimer’s therapies.
• The launch strengthens Thermo Fisher’s role as a data infrastructure partner supporting post-authorization studies and payer confidence
Yesterday’s NeuroPulse focused on early signals, biomarkers, discovery platforms, and trial setup.