🧠This Week in Neuroscience: RNAi Tau Therapy CTA, MS Patent Win, Generic Ozanimod, AI Brain MRI, ECTRIMS 2025 & More
Stay informed with the latest neuroscience breakthroughs! In this week’s roundup, we highlight key updates across Alzheimer’s disease, multiple sclerosis, and neuropsychiatry—from regulatory milestones and IP protection to AI-powered imaging and major congress spotlights.
In Today’s Newsletter
🧬 Arrowhead files CTA for ARO-MAPT (RNAi tau) in AD/tauopathies [1]
Context: CTA submitted to New Zealand regulator to start a placebo-controlled, dose-escalating Phase 1/2a in up to 64 healthy volunteers and up to 48 early Alzheimer’s patients; subcutaneous dosing.
Key point: ARO-MAPT uses a new TRiM™ delivery system reporting BBB penetration and CNS knockdown in preclinical studies (endpoint not specified).
Implication: Signals pipeline investment and modality expansion.
đź“ś USPTO allows vidofludimus calcium dose-strength patent in progressive MS [2]
Context: Notice of Allowance covers daily 10–45 mg dose strengths for PMS (PPMS, SPMS) and other forms; protection expected into 2041 (potential extension).
Key point: Strengthens Immunic’s IP around vidofludimus calcium (IMU-838); company references positive Phase 2 CALLIPER results (details not specified here).
Implication: Signals pipeline investment and modality expansion.
💊 Tentative FDA nod for generic ozanimod (Zeposia) in MS [3] [US • 12 Sep 2025]
Context: Synthon to manufacture/supply generic for Zydus; first-filer status could enable shared 180-day exclusivity at launch.
Key point: Tentative approval indicates standards met, but marketing waits on expiry/resolution of brand protections.
Implication: Introduces competition that may affect pricing and formulary access.
🧠GE HealthCare to acquire icometrix (AI brain MRI) [4]
Context: Planned integration of icobrain with GE MRI systems; icobrain aria is FDA-cleared to detect/quantify ARIA (E/H) tied to anti-amyloid therapies.
Key point: Aims for seamless workflow, longitudinal ARIA tracking, and broader neuro MRI quantification (MS, dementia, epilepsy, stroke, TBI).
Implication: Signals pipeline investment and modality expansion.
🧪 IASO Bio to present BCMA CAR-T (Eque-cel) data in progressive MS at ANA & ECTRIMS [5]
Context: Independently developed fully human anti-BCMA CAR-T (equecabtagene autoleucel) for PMS; data via poster (ANA) and oral (ECTRIMS).
Key point: Company characterizes findings as “promising” (endpoint not specified; n not stated).
Implication: May influence prescriber choice and payer reviews pending full data.
🧷 Sanofi previews ECTRIMS 2025 (tolebrutinib, frexalimab) [6] [EU • 10 Sep 2025]
https://www.sanofi.com/fr/media-room/communiques-de-presse/2025/2025-09-10-06-00-00-3147483
Context: 14 abstracts incl. 3 orals; tolebrutinib subgroup analyses in nrSPMS and RMS (PIRA); frexalimab Phase 2 OLE plus ongoing Phase 3 FREXALT/FREVIVA.
Key point: Focus on disability accumulation and non-depleting anti-CD40L pathway biology (specific efficacy metrics not in release).
Implication: May influence prescriber choice and payer reviews pending full data.
🧩 BRIUMVI® (ublituximab) MS data agenda at ECTRIMS 2025 [7] [US • 10 Sep 2025]
Context: Oral: 6-year efficacy/safety from ULTIMATE I/II OLE; ePosters on modified dosing (ENHANCE) and real-world ENABLE Phase 4.
Key point: Abstracts posted; full data to be presented at ECTRIMS (specific outcomes not detailed here).
Implication: May influence prescriber choice and payer reviews pending full data.
🧠J&J to showcase neuropsychiatry data at Psych Congress 2025 [8]
Context: 21 abstracts across MDD, TRD, schizophrenia; includes seltorexant H2H vs quetiapine XR in MDD with insomnia, CAPLYTA® post-hoc in anxious distress, SPRAVATO® monotherapy post-hocs.
Key point: Portfolio-wide clinical and real-world updates (endpoints not specified here).
Implication: May influence prescriber choice and payer reviews pending full data.
Why it matters
- Ozanimod’s tentative generic approval foreshadows price pressure and broader access in relapsing MS once patents clear.
- AI-enabled ARIA detection embedded in MRI workflow could streamline monitoring for anti-amyloid therapies and MS imaging quantification.
- Early CNS RNAi (ARO-MAPT) signals modality expansion toward subcutaneous, BBB-penetrant gene silencing for neurodegeneration.
- ECTRIMS 2025 is shaping up as a dense MS data moment across BTKis, anti-CD40L, anti-CD20, CAR-T, and real-world evidence.
- Strengthened IP (vidofludimus) may shape competitive positioning ahead of potential late-stage designs in progressive MS.
📢 Stay Ahead in Neuroscience Research!
✅ Contact LucidQuest at info@lqventures.com for strategic guidance on neuroscience and breakthrough therapies
FAQ
What is ARO-MAPT and what’s novel about it?
ARO-MAPT is an RNAi therapeutic targeting MAPT (tau) with a proprietary delivery system reporting BBB penetration and CNS knockdown in preclinical studies; Arrowhead filed a CTA in New Zealand for a Phase 1/2a with subcutaneous dosing [1].
What protection does Immunic’s new patent add?
A USPTO Notice of Allowance covers vidofludimus calcium (and other forms) dose strengths (about 10–45 mg daily) for progressive MS, with expected US protection into 2041 (potentially extendable) [2].
When could a generic ozanimod actually reach patients?
The FDA granted tentative approval, meaning it meets requirements but can’t be marketed until brand patents/exclusivity expire or are resolved; first-filer status could allow shared 180-day exclusivity at launch [3].
How does icometrix fit into GE HealthCare’s neuro strategy?
GE intends to integrate icobrain (incl. FDA-cleared ARIA detection/quantification) with its MRI systems to streamline assessment/monitoring in Alzheimer’s and other neuro conditions, and broaden quantitative neuro imaging tools (e.g., MS) [4].
What MS programs is Sanofi highlighting at ECTRIMS?
Subgroup analyses for tolebrutinib in nrSPMS and RMS (PIRA focus), and Phase 2 OLE plus ongoing Phase 3 trials (FREXALT, FREVIVA) for frexalimab; specific efficacy numbers aren’t in the release [6].
What BRIUMVI data will TG Therapeutics show?
An oral presentation on 6-year ULTIMATE I/II OLE results, plus ePosters on modified dosing (ENHANCE) and real-world ENABLE Phase 4 experience; outcomes will be detailed at the meeting [7].
Entities / Keywords
Arrowhead Pharmaceuticals; ARO-MAPT; MAPT/tau; TRiM™; Alzheimer’s disease; tauopathies • Immunic; vidofludimus calcium; IMU-838; progressive MS; PPMS; SPMS • Zydus Lifesciences; Synthon; ozanimod; Zeposia; tentative approval; 180-day exclusivity • GE HealthCare; icometrix; icobrain; ARIA-E; ARIA-H; MRI; Alzheimer’s • IASO Biotherapeutics; equecabtagene autoleucel; Eque-cel; anti-BCMA; CAR-T; PMS; ANA; ECTRIMS • Sanofi; tolebrutinib; HERCULES; GEMINI 1/2; PIRA; frexalimab; FREXALT; FREVIVA • TG Therapeutics; BRIUMVI; ublituximab; ULTIMATE I/II OLE; ENHANCE; ENABLE • Johnson & Johnson; seltorexant; quetiapine XR; CAPLYTA® (lumateperone); SPRAVATO® (esketamine); MDD; TRD; schizophrenia.
References
- https://www.biospace.com/press-releases/arrowhead-pharmaceuticals-requests-regulatory-clearance-to-initiate-phase-1-2a-study-of-aro-mapt-for-the-treatment-of-alzheimers-disease-and-other-tauopathies
- https://www.prnewswire.com/news-releases/immunic-receives-notice-of-allowance-for-united-states-patent-protecting-vidofludimus-calciums-dose-strengths-in-progressive-multiple-sclerosis-302550163.html
- https://multiplesclerosisnewstoday.com/news-posts/2025/09/12/new-generic-version-zeposia-ms-wins-tentative-fda-backing/
- https://www.gehealthcare.com/about/newsroom/press-releases/ge-healthcare-announces-intent-to-acquire-icometrix-to-strengthen-neurology-portfolio-with-brain-mri-assessment-solutions
- https://www.prnewswire.com/news-releases/iaso-bio-to-present-promising-findings-on-equecabtagene-autoleucel-for-multiple-sclerosis-at-both-ana-and-ectrims-2025-302554857.html
- https://www.sanofi.com/fr/media-room/communiques-de-presse/2025/2025-09-10-06-00-00-3147483
- https://www.globenewswire.com/news-release/2025/09/10/3147668/8790/en/TG-Therapeutics-Announces-Schedule-of-Data-Presentations-for-BRIUMVI-in-Multiple-Sclerosis-at-the-2025-European-Committee-for-Treatment-and-Research-in-Multiple-Sclerosis-Annual-Me.html
- https://innovativemedicine.jnj.com/us/news-center/neuroscience/johnson-johnson-to-showcase-industry-leading-neuropsychiatry-innovations-at-the-2025-psych-congress-annual-meeting