This week’s Neuroscience news brings you the most important developments, regulatory updates, and industry breakthroughs across biopharma.
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✅ Koselugo FDA approval for adult NF1 PN (selumetinib) [1] [US • 20 Nov 2025] 🎯
Context: KOMET Phase 3, largest placebo-controlled NF1 PN adult study; ORR primary, safety consistent with pediatric use.
Key point: FDA approved selumetinib for symptomatic, inoperable NF1 PN in adults, supported by KOMET ORR advantage vs placebo (endpoint and n reported by sponsor).
Implication: May influence prescriber choice and payer reviews pending full data.
🧠 J&J ends Auτonomy PoC in early AD (posdinemab) [2] [US • 21 Nov 2025]
https://www.jnj.com/media-center/press-releases/johnson-johnson-statement-on-the-au%CF%84onomy-study
Context: Precision approach PoC in early Alzheimer’s; scheduled review.
Key point: Posdinemab did not achieve statistical significance in slowing clinical decline; study discontinued.
Implication: May influence prescriber choice and payer reviews pending full data.
🧪 Novo Nordisk oral semaglutide misses in AD (evoke/evoke+) [3] [DK • 24 Nov 2025]
Context: Two 2-year Phase 3 trials, randomized double-blind, n=3,808 adults with early symptomatic AD.
Key point: No superiority vs placebo on CDR-SB; biomarker improvements did not translate to clinical delay; extensions halted.
Implication: May influence prescriber choice and payer reviews pending full data.
🧪 Rakovina AI-discovered CNS-penetrant ATR/mTOR inhibitors (preclinical) [4] [23 Nov 2025]
Context: SNO poster; Enki AI platform; mouse PK and BBB exposure; comparisons to clinical ATR inhibitors.
Key point: Lead compounds show dual ATR/mTOR inhibition and CNS penetration in preclinical studies; rationale for PTEN-deficient tumors.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.
🧠 1910’s CANDID-CNS AI BBB model published in JCIM [5] [US • 24 Nov 2025]
Context: Attentive GNN predicts BBB penetration, including bRo5 and stereochemistry; benchmarks vs Pfizer CNS MPO.
Key point: Reported performance gains on bRo5 and stereoisomer tasks; contributed to internal program selection per company.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.
☢️ Plus Therapeutics completes FDA Type B meeting on Reyobiq in LM [6] [US • 24 Nov 2025]
Context: FDA meeting on pivotal/registrational design for LM; amendments planned per feedback.
Key point: Company will align LM program and share next-step guidance in early 2026 per minutes.
Implication: May influence prescriber choice and payer reviews pending full data.
🧫 Plus Therapeutics’ CNSide gets Humana national coverage [7] [US • 20 Nov 2025]
Context: Agreement effective 29 Oct 2025; now 67M covered lives; assay supports LM diagnosis and monitoring.
Key point: Humana national coverage for CNSide CSF TCE LDT, with reported sensitivity/specificity and RWE utilization.
Implication: Access programs: May expand screening, initiation, and follow-up at scale.
🧬 Sarepta DM1 siRNA SRP-1003 dose escalation and $200M milestone [8] [US • 24 Nov 2025]
Context: Phase 1/2 MAD: cohorts 1–2 complete, 3 ongoing, 4 dosing, 5 planned early 2026; Arrowhead collaboration.
Key point: Enrollment target met, progression to higher doses, and Arrowhead to receive $200M milestone per agreement.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.
🧯 SciSparc/NeuroThera/Clearmind file Israeli patent for non-hallucinogenic neuroplastogen combo [9] [IL • 20 Nov 2025]
Context: Patent on MEAI plus N-acylethanolamines such as PEA for depression; part of 13-patent collaboration portfolio.
Key point: Filing advances IP around non-hallucinogenic neuroplastogen approach; preclinical data cited by companies.
Implication: Partnerships/BD: Signals pipeline investment and modality expansion.
🧩 Contineum PIPE-307 misses endpoints in RRMS Phase 2 VISTA [10] [US • 20 Nov 2025]
Context: Randomized, double-blind, placebo-controlled PoC; M1 antagonist; LCLA primary endpoint; imaging included.
Key point: No significant changes on primary or secondary measures; safety acceptable; full data planned for meeting/journal.
Implication: May influence prescriber choice and payer reviews pending full data.
Why it Matters
- Two high-profile Alzheimer’s programs reported negatives, underscoring trial-level hurdles in early AD [2][3].
- First adult NF1 PN approval for selumetinib expands treatment continuity from pediatrics to adults [1].
- AI-enabled BBB prediction and CNS-penetrant design are advancing toolkits for neuro drug discovery [4][5].
- Coverage expansion for CNS diagnostics can accelerate LM care pathways while trials mature [7].
- Early clinical RNAi progress and BD milestones show capital flow into neuromuscular RNA therapeutics [8].
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FAQ
What did FDA approve for NF1 PN in adults?
Selumetinib (Koselugo) gained US approval for symptomatic, inoperable NF1 PN in adults, supported by the KOMET Phase 3 ORR advantage and consistent safety with pediatric experience [1].
Did Johnson & Johnson’s posdinemab help in early Alzheimer’s?
No. The Auτonomy PoC did not achieve statistical significance in slowing clinical decline, and the study will be discontinued. Further analyses are planned for disclosure [2].
Did oral semaglutide work for Alzheimer’s in evoke/evoke+?
Topline results did not show superiority vs placebo on CDR-SB over 2 years. Biomarker changes were observed but did not translate to clinical benefit. Extensions will be stopped [3].
What is 1910’s CANDID-CNS and why is it notable?
An AI model published in JCIM that predicts BBB penetration for bRo5 molecules and distinguishes stereoisomers, with reported performance gains over CNS MPO, enabling broader CNS design space [5].
How is Plus Therapeutics progressing in LM?
They completed a Type B FDA meeting on Reyobiq’s LM program to discuss pivotal design, and separately secured Humana national coverage for the CNSide CSF assay, expanding access [6][7].
What is Sarepta’s latest on SRP-1003 for DM1?
Phase 1/2 MAD has advanced through multiple cohorts with further dose escalation planned. Hitting the enrollment trigger activates a $200M milestone to Arrowhead under their collaboration [8].
Entities / Keywords
Koselugo, selumetinib; Alexion, AstraZeneca; NF1, plexiform neurofibromas.
Johnson & Johnson; posdinemab; Auτonomy; early Alzheimer’s.
Novo Nordisk; semaglutide; evoke, evoke+; CDR-SB.
Rakovina Therapeutics; ATR/mTOR dual inhibition; PTEN-deficient tumors; SNO.
1910; CANDID-CNS; BBB; bRo5; JCIM; CNS MPO.
Plus Therapeutics; Reyobiq; leptomeningeal metastases; CNSide; Humana.
Sarepta Therapeutics; SRP-1003; DM1; Arrowhead Pharmaceuticals.
SciSparc; NeuroThera Labs; Clearmind Medicine; MEAI; PEA; depression.
Contineum Therapeutics; PIPE-307; RRMS; VISTA; LCLA.
References
- https://www.astrazeneca.com/media-centre/press-releases/2025/koselugo-selumetinib-approved-in-the-us.html
- https://www.jnj.com/media-center/press-releases/johnson-johnson-statement-on-the-au%CF%84onomy-study
- https://www.novonordisk.com/content/nncorp/global/en/news-and-media/news-and-ir-materials/news-details.html?id=916462
- https://investingnews.com/rakovina-therapeutics-showcases-compelling-preclinical-data-on-ai-discovered-cns-penetrant-atr-mtor-inhibitors-at-the-2025-society-for-neuro-oncology-annual-meeting/
- https://www.businesswire.com/news/home/20251124252059/en/1910-Publishes-CANDID-CNS-an-AI-Model-That-Unlocks-Beyond-Rule-of-5-Chemical-Space-and-Stereochemistry-to-Predict-BloodBrain-Barrier-Penetration
- https://www.globenewswire.com/fr/news-release/2025/11/24/3193399/0/en/Plus-Therapeutics-Announces-Completion-of-U-S-FDA-Meeting-on-Future-Clinical-Development-Plans-for-REYOBIQ-in-Leptomeningeal-Metastases.html
- https://www.globenewswire.com/news-release/2025/11/20/3191797/0/en/Plus-Therapeutics-Secures-National-Coverage-Agreement-with-Humana-for-CNSide-Cerebrospinal-Fluid-Assay-for-Metastatic-CNS-Cancer.html
- https://investorrelations.sarepta.com/news-releases/news-release-details/sarepta-provides-progress-update-srp-1003-its-investigational
- https://www.manilatimes.net/2025/11/20/tmt-newswire/globenewswire/scisparc-neurothera-labs-announces-israeli-patent-application-in-collaboration-with-clearmind-medicine-for-novel-non-hallucinogenic-neuroplastogen-treatment-for-depression/2228257
- https://www.businesswire.com/news/home/20251120828592/en/Contineum-Therapeutics-Reports-Topline-Data-From-Its-Phase-2-PIPE-307-VISTA-Trial-for-the-Treatment-of-Relapsing-Remitting-Multiple-Sclerosis-RRMS