Neuroscience Today—April 1, 2026

This week’s Neuroscience update highlights regulatory progress in rare diseases, advances in care delivery, practical treatment data, and continued investment in earlier-stage CNS programs.

In Today’s Newsletter

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🧠 Neurocrine Biosciences publishes TD guidance for long-term care [1] [US • 26 Mar 2026]

https://neurocrine.gcs-web.com/news-releases/news-release-details/neurocrine-biosciences-presents-first-expert-consensus
Context: The company also reported a post-hoc KINECT-PRO analysis in adults aged 65 years and older showing clinically meaningful patient-reported improvement with INGREZZA (valbenazine).
Key point: Neurocrine Biosciences said a Delphi panel issued the first expert consensus recommendations for tardive dyskinesia screening, diagnosis and treatment in long-term care, including VMAT2 inhibitor use.
Implication: May influence prescriber choice and payer reviews pending full data.

🧠 Anavex withdraws EU filing for blarcamesine in early Alzheimer’s disease [2] [EU • 25 Mar 2026]

https://anavex.com/news/anavex-blarcamesine-eu-regulatory-update-alzheimers/
Context: The company said it will consider CHMP feedback, gather additional data, and conduct further analyses; the release does not claim efficacy or safety conclusions.
Key point: Anavex Life Sciences withdrew its EU marketing authorization application for blarcamesine as add-on therapy in early Alzheimer’s disease after CHMP feedback indicated no positive opinion at this time.
Implication: May influence prescriber choice and payer reviews pending full data.

🤝 Cognito Therapeutics and Ochsner Health launch an Alzheimer’s care collaboratory [3] [US • 25 Mar 2026]

https://www.businesswire.com/news/home/20260325653373/en/Cognito-Therapeutics-and-Ochsner-Health-Launch-Brain-Health-Collaboratory-to-Advance-New-Care-Models-for-Alzheimers-Disease
Context: The partners plan to build a Brain Health Index, study real-world delivery, and explore clinical and health economics evidence, including in dual-eligible Medicare and Medicaid populations.
Key point: Cognito Therapeutics and Ochsner Health launched a Brain Health Collaboratory to test new care models for cognitive decline and Alzheimer’s disease using Cognito’s investigational Spectris platform.
Implication: Access programs may expand screening, initiation, and follow-up at scale.

🧬 Denali wins FDA approval for AVLAYAH in Hunter syndrome [4] [US • 25 Mar 2026]

https://www.globenewswire.com/news-release/2026/03/25/3262412/0/en/Denali-Therapeutics-Announces-U-S-FDA-Approval-of-AVLAYAH-tividenofusp-alfa-eknm-for-Treatment-of-Hunter-Syndrome-MPS-II.html
Context: Approval was based on reduction in cerebrospinal fluid heparan sulfate as a surrogate endpoint; the company said confirmatory evidence is being generated in the ongoing Phase 2/3 COMPASS study.
Key point: Denali Therapeutics announced FDA accelerated approval of AVLAYAH (tividenofusp alfa-eknm) for neurologic manifestations of Hunter syndrome (MPS II) in eligible pediatric patients before advanced neurologic impairment.
Implication: May influence prescriber choice and payer reviews pending full data.

💊 Bristol Myers Squibb reports outpatient switch data for Cobenfy in schizophrenia [5] [EU • 28 Mar 2026]

https://news.bms.com/news/details/2026/Open-Label-Outpatient-Switch-Study-Demonstrates-Symptom-Stability-During-Transition-from-Oral-Atypical-Antipsychotics-to-Cobenfy-xanomeline-and-trospium-chloride/default.aspx
Context: The trial enrolled 105 outpatients and compared faster 2-week versus slower 4-week cross-titration; no discontinuations were attributed to lack of efficacy.
Key point: Bristol Myers Squibb reported that adults with schizophrenia remained symptomatically stable through 8 weeks when switched from oral atypical antipsychotics to Cobenfy monotherapy in an open-label Phase 4 study.
Implication: May influence prescriber choice and payer reviews pending full data.

🌡️ Tonix doses first participant in TNX-1900 migraine pharmacodynamic study [6] [26 Mar 2026]

https://ir.tonixpharma.com/news-events/press-releases/detail/1620/tonix-pharmaceuticals-announces-first-participant-dosed-in
Context: The study measures forehead skin blood flow responses using Laser Speckle Contrast Imaging in healthy female volunteers; the company positions oxytocin as distinct from CGRP-pathway treatments.
Key point: Tonix Pharmaceuticals dosed the first participant in an investigator-initiated Phase 1 study of TNX-1900, intranasal potentiated oxytocin, in a trigeminal neurovascular reactivity model relevant to migraine and craniofacial pain.
Implication: Signals pipeline investment and modality expansion.

🧪 Quantum BioPharma names Phase 2 investigator for Lucid-MS [7] [US • 26 Mar 2026]

https://www.globenewswire.com/news-release/2026/03/26/3262941/0/en/Quantum-Biopharma-Announces-Appointment-of-Principal-Investigator-for-Planned-Phase-2-Clinical-Trial-of-Lucid-21-302-Lucid-MS-in-Multiple-Sclerosis.html
Context: The company said it plans to submit an IND in the coming weeks; Lucid-MS is described as a first-in-class, non-immunomodulatory neuroprotective candidate targeting demyelination.
Key point: Quantum BioPharma appointed Salvatore Napoli, MD, as principal investigator for a planned Phase 2 trial of Lucid-21-302 (Lucid-MS) in multiple sclerosis.
Implication: Signals pipeline investment and modality expansion.

🧠 Lunai Bioworks buys BBB platform for Alzheimer’s and broader CNS delivery [8] [US • 26 Mar 2026]

https://www.biospace.com/press-releases/lunai-bioworks-executes-20m-strategic-transaction-at-fixed-1-50-conversion-acquiring-bbb-delivery-platform-for-cns-alzheimers-therapies-with-broad-cns-delivery-applications
Context: The company said the platform is designed to enable brain penetration and brain-specific activation, and that the deal is structured as Series B Convertible Preferred at a fixed conversion price.
Key point: Lunai Bioworks announced a $20 million strategic transaction to acquire blood-brain barrier delivery technology and CNS Alzheimer’s assets from Clemann Group, SAS or its assignee.
Implication: Signals pipeline investment and modality expansion.

🎗️ Lantern Pharma and Starlight win IND clearance for STAR-001 pediatric CNS cancer study [9] [US • 27 Mar 2026]

https://ir.lanternpharma.com/news-1/news/news-details/2026/Lantern-Pharma-and-Starlight-Therapeutics-Announce-FDA-Clearance-of-IND-for-a-Planned-Phase-1-Pediatric-CNS-Cancer-Trial-of-STAR-001/default.aspx
Context: The study is planned as a multicenter, open-label dose-escalation trial of STAR-001 alone and with spironolactone, including ATRT, DIPG, GBM, medulloblastoma, and ependymoma.
Key point: Lantern Pharma and Starlight Therapeutics received FDA clearance of the IND for a planned Phase 1 trial of STAR-001 in children with relapsed or refractory CNS malignancies.
Implication: Signals pipeline investment and modality expansion.

⚖️ Praxis wins FDA filing acceptance and priority review for relutrigine [10] [US • 31 Mar 2026]

https://www.manilatimes.net/2026/03/31/tmt-newswire/globenewswire/praxis-precision-medicines-announces-fda-acceptance-and-priority-review-of-new-drug-application-for-relutrigine-in-patients-with-scn2a-and-scn8a-dees/2310882
Context: The company said the FDA assigned a PDUFA target action date of 27 Sep 2026; the NDA is supported by the EMBOLD study, which was stopped early for efficacy after interim analysis.
Key point: Praxis Precision Medicines said FDA accepted and granted priority review to the NDA for relutrigine in SCN2A and SCN8A developmental and epileptic encephalopathies.
Implication: May influence prescriber choice and payer reviews pending full data.

Why it Matters

Rare neurogenetic and lysosomal disorders moved closer to commercialization this week, with Denali’s AVLAYAH approval and Praxis’s relutrigine priority review highlighting FDA momentum in high-unmet-need CNS diseases.[4][10]
Alzheimer’s disease remained split between regulatory friction and care-model innovation, with Anavex withdrawing its EU filing while Cognito and Ochsner expanded real-world delivery infrastructure.[2][3]
Psychiatry coverage deepened beyond efficacy headlines, as Neurocrine focused on implementation guidance in long-term care and Bristol Myers Squibb addressed practical switching data for Cobenfy.[1][5]
Early-stage CNS platform building continued across migraine, multiple sclerosis, Alzheimer’s delivery, and pediatric brain tumors, suggesting sustained investor and developer appetite for differentiated mechanisms and delivery approaches.[6][7][8][9]
Blood-brain barrier access remains a central strategic theme, appearing in Denali’s approved TransportVehicle platform and Lunai’s acquired BBB delivery technology.[4][8]

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FAQ

What changed for AVLAYAH in Hunter syndrome this week?

Denali Therapeutics said FDA granted accelerated approval to AVLAYAH for neurologic manifestations of Hunter syndrome in eligible pediatric patients. The approval was based on biomarker evidence, with confirmatory study work continuing in COMPASS.[4]

What does Anavex’s EU withdrawal mean for blarcamesine?

Anavex said it withdrew the EU marketing application after CHMP feedback indicated no positive opinion at this time. The company said it will gather more data and analyses, so this is a regulatory setback rather than an end to development.[2]

How clinically useful are the new Cobenfy switch data?

Bristol Myers Squibb reported symptom stability through 8 weeks when patients switched from oral atypical antipsychotics to Cobenfy, with no discontinuations due to lack of efficacy in the study. The evidence is open-label, but it addresses a practical treatment-transition question.[5]

What did Neurocrine add beyond INGREZZA efficacy messaging?

Neurocrine paired a post-hoc KINECT-PRO analysis in older adults with expert consensus recommendations for long-term care. The practical emphasis was on routine screening, diagnosis, and treatment workflows for tardive dyskinesia in a high-risk setting.[1]

Why does Praxis’s relutrigine filing matter in SCN2A and SCN8A DEEs?

Praxis said FDA accepted the NDA and granted priority review, with a PDUFA date of 27 Sep 2026. The company also said there are no currently approved targeted therapies for SCN2A and SCN8A DEEs.[10]

What is distinct about the STAR-001 pediatric CNS cancer program?

Lantern Pharma and Starlight Therapeutics said the planned Phase 1 study will test STAR-001 alone and with spironolactone in relapsed or refractory pediatric CNS tumors. The company frames the combination around ERCC3-related DNA repair vulnerability identified with its RADR platform.[9]

Entities / Keywords

Neurocrine Biosciences, INGREZZA, valbenazine, tardive dyskinesia, TD, VMAT2 inhibitor, KINECT-PRO, long-term care
Anavex Life Sciences, blarcamesine, early Alzheimer’s disease, EMA, CHMP, EU marketing authorization
Cognito Therapeutics, Ochsner Health, Spectris, Brain Health Collaboratory, Brain Health Index, Alzheimer’s disease
Denali Therapeutics, AVLAYAH, tividenofusp alfa-eknm, Hunter syndrome, MPS II, TransportVehicle, transferrin receptor, blood-brain barrier
Bristol Myers Squibb, Cobenfy, xanomeline and trospium chloride, schizophrenia, PANSS, cross-titration
Tonix Pharmaceuticals, TNX-1900, intranasal potentiated oxytocin, migraine, craniofacial pain, trigeminal neurovascular reactivity, CGRP
Quantum BioPharma, Lucid-21-302, Lucid-MS, multiple sclerosis, demyelination, neuroprotection
Lunai Bioworks, blood-brain barrier delivery, BBB platform, CNS Alzheimer’s therapies
Lantern Pharma, Starlight Therapeutics, STAR-001, LP-184, pediatric CNS cancer, ATRT, DIPG, GBM, medulloblastoma, ependymoma, spironolactone, ERCC3
Praxis Precision Medicines, relutrigine, SCN2A, SCN8A, developmental and epileptic encephalopathies, DEEs, NDA, priority review