Latest Neuroscience News - Sept 16th to Sept 30th

Watch Our Video Summary Capturing Top Neuroscience News from the Last Two Weeks

From ocrelizumab’s favorable MS outcomes, FDA’s tolebrutinib review delay, and Kisunla’s EU approval in early Alzheimer’s—this week’s neuroscience signals are robust. We span multiple sclerosis, Alzheimer’s, Huntington’s, Parkinson’s, and neurodegenerative therapy development, with regulatory updates and clinical trial advances across areas, underscoring long-term efficacy and innovation in treatment modalities.

🧪 Ocrelizumab vs rituximab outcomes in MS [1] [US •22 Sep 2025]

https://www.rarediseaseadvisor.com/news/ocrelizumab-offer-better-outcomes-rituximab-ms/
Context: Retrospective, multicenter EHR study (>1,400 adults) across six California sites; simulated target trial approach.
Key point: Observational analysis (Annals of Neurology) found lower hospitalization and hypogammaglobulinemia, plus lower infections, with ocrelizumab vs rituximab.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.

⏳ FDA delays Sanofi’s tolebrutinib (nrSPMS) review [2] [US • 23 Sep 2025]

https://finance.yahoo.com/news/fda-delays-key-sanofi-multiple-160706027.html
Context: Application seeks label for non-relapsing SPMS; extension triggered by additional information.
Key point: FDA extended NDA review for tolebrutinib in nrSPMS/relapse-independent disability by 3 months to 28 Dec 2025 (major amendment).
Implication: May influence prescriber choice and payer reviews pending full data.

🤝 JCR & Alexion hit milestone in neurodegenerative program [3] [JP • 22 Sep 2025]

https://www.biospace.com/press-releases/jcr-pharmaceuticals-and-alexion-achieve-milestone-in-collaborative-neurodegenerative-disease-program
Context: Broader collaboration includes oligonucleotide and JUST-AAV genomic medicine agreements (2023–2025).
Key point: Milestone achieved for a BBB-penetrant therapeutic protein using J-Brain Cargo®; payment from Alexion triggered.
Implication: Signals pipeline investment and modality expansion.

🧠 Kesimpta switch/long-term data at ECTRIMS [4] [CH • 24 Sep 2025]

https://www.novartis.com/news/media-releases/new-novartis-data-further-support-benefits-kesimpta-relapsing-ms-following-switch-from-oral-disease-modifying-therapies
Context: No new safety concerns reported post-switch; early use supported by OLE findings.
Key point: ARTIOS (Ph3b, open-label) reported low ARR (0.06/96 wks) and >90% NEDA-3 after switching from fingolimod/fumarates; ALITHIOS OLE showed >90% progression-free up to 7 years.
Implication: May influence prescriber choice and payer reviews pending full data.

🛡️ BRIUMVI 6-year durability in RMS [5] [US • 24 Sep 2025]

https://www.globenewswire.com/news-release/2025/09/24/3155543/8790/en/New-Data-for-BRIUMVI-Demonstrate-89-9-of-Patients-with-Relapsing-Multiple-Sclerosis-Were-Free-from-Disability-Progression-After-6-Years-of-Continuous-BRIUMVI-Treatment.html
Context: ULTIMATE I/II open-label extension; observational ENABLE showed very low on-treatment relapses.
Key point: 89.9% progression-free at 6 years; ARR declined to 0.012 in year 6; safety consistent; real-world ENABLE and dosing ENHANCE updates supportive.
Implication: May influence prescriber choice and payer reviews pending full data.

🧷 EC authorizes Kisunla (donanemab) for early AD [6] [EU • 25 Sep 2025]

https://investor.lilly.com/news-releases/news-release-details/lillys-kisunla-donanemab-receives-marketing-authorization-0
Context: Based on TRAILBLAZER-ALZ 2/6; gradual titration lowered ARIA-E vs earlier schedule.
Key point: EU approval for early symptomatic AD with amyloid confirmation in ApoE4 non-carriers/heterozygotes; supports stopping once plaques minimal.
Implication: May influence prescriber choice and payer reviews pending full data.

🧬 AMT-130 gene therapy positive in Huntington’s [7] [24 Sep 2025]

https://www.uniqure.com/investors-media/press-releases
Context: 29 treated (high/low dose); external controls from Enroll-HD; BLA planned Q1 2026; safety manageable.
Key point: Pivotal Ph1/2 met primary endpoint: 75% slowing on cUHDRS at 36 months vs propensity-matched control; TFC slowing significant; CSF NfL below baseline.
Implication: May influence prescriber choice and payer reviews pending full data.

🧩 AbbVie files NDA for tavapadon in Parkinson’s [8] [US • 26 Sep 2025]

https://news.abbvie.com/2025-09-26-AbbVie-Submits-New-Drug-Application-to-U-S-FDA-for-Tavapadon-for-the-Treatment-of-Parkinsons-Disease
Context: Once-daily selective D1/D5 partial agonist; adverse events generally mild/moderate in studies.
Key point: NDA supported by three positive Ph3 TEMPO trials (early PD monotherapy and adjunct to levodopa), improving MDS-UPDRS and “on” time.
Implication: May influence prescriber choice and payer reviews pending full data.

Why it matters

Anti-CD20 choices in MS may shift with head-to-head-style RWE and long-term data [1][4][5]
Regulatory moves shape near-term options in progressive MS and early Alzheimer’s care pathways [2][6].
Gene and cell-targeted modalities continue to edge into neurodegeneration with measurable functional signals [7].
BBB-penetrant delivery platforms remain a strategic focus for big-rare alliances [3].

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FAQ

Does ocrelizumab outperform rituximab in safety for MS?

The observational study reported lower hospitalization, hypogammaglobulinemia, and infections with ocrelizumab vs rituximab; as RWE, findings require cautious interpretation [1].

What changed for Sanofi’s tolebrutinib timeline?

FDA extended the tolebrutinib nrSPMS review by 3 months to 28 Dec 2025 due to a major amendment submitted during review [2].

How strong are Kesimpta’s switch and long-term data?

ARTIOS suggested low ARR and >90% NEDA-3 post-switch from orals, and ALITHIOS showed >90% progression-free up to 7 years; both open-label with no new safety issues reported [4].

What’s new about BRIUMVI’s durability?

Six-year extension data showed ~90% progression-free and very low relapse rates, with a safety profile consistent over time; supportive real-world findings were also presented [5].

What exactly did the EU approve for donanemab?

Kisunla was authorized for early symptomatic AD with confirmed amyloid in ApoE4 non-carriers/heterozygotes, with evidence supporting treatment completion once plaques are minimal [6].

What did uniQure show in Huntington’s?

High-dose AMT-130 achieved significant slowing on cUHDRS and TFC at 36 months vs external controls; safety was manageable; BLA submission targeted for Q1 2026 [7].

Entities / Keywords

Ocrelizumab (Genentech/Roche); Rituximab; Sanofi; Tolebrutinib (BTK inhibitor); JCR Pharmaceuticals; Alexion (AstraZeneca Rare Disease); J-Brain Cargo®; JUST-AAV; Novartis; Kesimpta (ofatumumab); TG Therapeutics; BRIUMVI (ublituximab-xiiy); Eli Lilly; Kisunla (donanemab); uniQure; AMT-130; AbbVie; Tavapadon (D1/D5 partial agonist).