Neuroscience Today—March 4, 2026

LucidQuest

2 minutes ago

This week’s Neuroscience update covers key developments across late-stage clinical progress and data readouts, regulatory and reimbursement pathway updates, first-patient-dosed trial starts, partnerships, and patient advocacy initiatives.

In Today’s Newsletter

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In Today’s Newsletter

🧾 Plus Therapeutics gets Category III CPT code for CED with REYOBIQ [1] [US • 25 Feb 2026]

https://ir.plustherapeutics.com/news-releases/news-release-details/plus-therapeutics-announces-new-category-iii-cpt-code-convection
Context: Code tracks convection-enhanced delivery (CED) used to administer REYOBIQ in recurrent glioblastoma and pediatric brain cancer.
Key point: AMA CPT Editorial Panel approved Category III code X566T, publication 01 Jul 2026, effective for reporting 01 Jan 2027.
Implication: May influence prescriber choice and payer reviews pending full data.

😴 Axsome doses first patient in CLARITY Phase 3 (solriamfetol) for MDD with EDS symptoms [2] [US • 24 Feb 2026]

https://axsometherapeuticsinc.gcs-web.com/news-releases/news-release-details/axsome-therapeutics-initiates-clarity-phase-3-trial-solriamfetol
Context: Phase 3, double-blind, placebo-controlled, multicenter randomized withdrawal design with open-label period.
Key point: First patient dosed, primary endpoint is time from randomization to relapse of depressive symptoms.
Implication: May influence prescriber choice and payer reviews pending full data.

🧩 IRLAB doses first patient in IRL757 Phase Ib (Parkinson’s disease apathy), triggers milestone [3] [Sweden • 26 Feb 2026]

https://irlab.se/mfn_news/irlab-secures-milestone-payment-for-first-patient-dosed-in-phase-ib-study-with-irl757-in-parkinsons-disease/
Context: LIFT-PD Phase Ib (signal-finding) plans 75 patients across 16 sites (Germany, Bulgaria, Poland, Spain).
Key point: First patient dosed, triggers USD 3m milestone payment to IRLAB, study funded by MSRD (Otsuka affiliate).
Implication: Signals pipeline investment and modality expansion.

🧪 Evox partners with Rett Syndrome Research Trust to assess ExoEdit feasibility in Rett syndrome [4] [UK • 26 Feb 2026]

https://www.evoxtherapeutics.com/evox-therapeutics-partners-with-rett-syndrome-research-trust/
Context: Collaboration to assess exosome-based delivery for gene editing in the CNS, targeting Rett syndrome (MECP2 mutations referenced).
Key point: Evox and RSRT will evaluate feasibility of using ExoEdit to enable a gene editing treatment approach (pre-clinical feasibility stage).
Implication: Signals pipeline investment and modality expansion.

🧬 GEMMABio doses first patient in CHARISMA Phase 1/2 of GB221 gene therapy for SMA1 [5] [US • 26 Feb 2026]

https://www.prnewswire.com/news-releases/gemmabio-announces-first-patient-dosed-in-charisma-trial-of-gb221-investigational-nextgeneration-gene-therapy-for-spinal-muscular-atrophy-type-1-sma1-302698146.html
Context: CHARISMA (NCT07070999) evaluates GB221 delivered to CSF via intracisterna-magna injection in infants (two weeks to <12 months), symptomatic and presymptomatic cohorts.
Key point: First patient dosed, company notes FDA rare pediatric disease designation for GB221.
Implication: May influence prescriber choice and payer reviews pending full data.

🧬 uniQure: FDA says AMT-130 Phase I/II plus external control not sufficient for effectiveness evidence [6] [US • 02 Mar 2026]

https://www.globenewswire.com/news-release/2026/03/02/3247236/0/en/uniQure-Provides-Regulatory-Update-on-AMT-130-for-Huntington-s-Disease.html
Context: Final minutes from FDA Type A meeting (held 30 Jan 2026) on AMT-130 for Huntington’s disease.
Key point: FDA cannot agree that Phase I/II data versus an external control are sufficient for a marketing application, FDA strongly recommends a prospective randomized, double-blind, sham surgery-controlled study.
Implication: May influence prescriber choice and payer reviews pending full data.

🧠 Roche: Fenebrutinib met Phase 3 FENhance 1 primary endpoint in RMS, company plans submissions [7] [Switzerland • 02 Mar 2026]

https://www.roche.com/media/releases/med-cor-2026-03-02
Context: Pivotal Phase 3 FENhance 1 in relapsing MS compared fenebrutinib vs teriflunomide over at least 96 weeks, part of a three-study Phase 3 program (FENhance 1, FENhance 2, FENtrepid).
Key point: Roche reports significant relapse reduction versus teriflunomide and plans to submit totality of Phase 3 data to regulators, with full data to be presented at AAN 2026.
Implication: May influence prescriber choice and payer reviews pending full data.

🗣️ MSAA launches “Your MS, Your Voice” self-advocacy campaign for MS Awareness Month [8] [US • 02 Mar 2026]

https://www.prnewswire.com/news-releases/the-multiple-sclerosis-association-of-america-msaa-launches-campaign-focused-on-self-advocacy-strategies-in-recognition-of-ms-awareness-month-302701467.html
Context: Month-long education push with planned podcast episodes and a self-advocacy video (release dates stated by MSAA).
Key point: MSAA announced resources focused on self-advocacy strategies for people living with MS, care partners, and clinicians.
Implication: May expand screening, initiation, and follow-up at scale.

🤝 Novartis completes acquisition of Avidity Biosciences, adds AOC platform and late-stage programs [9] [Switzerland • 27 Feb 2026]

https://www.novartis.com/news/media-releases/novartis-successfully-completes-acquisition-avidity-biosciences-strengthening-late-stage-neuroscience-pipeline-and-advancing-xrna-strategy
Context: Novartis says Avidity brings Antibody Oligonucleotide Conjugates (AOC) and three late-stage programs, transaction originally announced 26 Oct 2025 (per Novartis).
Key point: Novartis closed the deal, Avidity becomes a wholly owned subsidiary, shareholders receive USD 72.00 per share in cash (per release).
Implication: Signals pipeline investment and modality expansion.

Why it Matters

MS is seeing continued competition between oral agents and high-efficacy mechanisms, Roche is positioning BTK inhibition across relapsing and progressive biology. [7]
FDA feedback for neurosurgical gene therapy programs reinforces the bar for controlled evidence, even with durability claims and external comparators. [6]
Reimbursement mechanics (CPT tracking codes) can materially change adoption timelines for procedure-dependent oncology delivery. [1]
Multiple first-patient-dosed updates underscore near-term clinical execution risk and catalyst cadence across CNS and neuromuscular pipelines. [2], [3], [5]
Large-cap BD continues to concentrate differentiated delivery and RNA platforms inside scaled commercial organizations. [9]

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FAQ

What is fenebrutinib (Roche) and what did FENhance 1 show?

Fenebrutinib is an investigational BTK inhibitor, Roche says Phase 3 FENhance 1 met its primary endpoint in relapsing MS versus teriflunomide, with broader Phase 3 submissions planned. [7]

What did FDA tell uniQure about AMT-130 for Huntington’s disease?

FDA meeting minutes state Phase I/II data compared to an external control are not sufficient as primary evidence of effectiveness for a marketing application, and FDA strongly recommended a prospective randomized, double-blind, sham-controlled study. [6]

What is the new CPT code Plus Therapeutics announced, and why does it matter?

Plus says AMA approved a Category III CPT code (X566T) to track CED procedures used to deliver REYOBIQ, with publication 01 Jul 2026 and effective reporting 01 Jan 2027, supporting utilization tracking relevant to coverage and reimbursement discussions. [1]

What is Axsome’s CLARITY Phase 3 trial design for solriamfetol?

CLARITY is a randomized withdrawal Phase 3 study in adults with MDD with excessive daytime sleepiness symptoms, with an open-label period followed by 1:1 randomization to continue solriamfetol or switch to placebo, primary endpoint is time to relapse of depressive symptoms. [2]

What is GEMMABio’s GB221 program for SMA1?

GEMMABio reports first patient dosed in CHARISMA Phase 1/2 of GB221, a gene therapy delivered to CSF via intracisterna-magna injection in infants, and notes FDA rare pediatric disease designation for GB221. [5]

Why did Novartis acquire Avidity Biosciences?

Novartis says the acquisition adds Avidity’s muscle-directed AOC platform and three late-stage neuromuscular programs, strengthening its neuroscience and RNA therapeutics strategy. [9]

Entities / Keywords

Plus Therapeutics (PSTV), REYOBIQ, convection-enhanced delivery (CED), Category III CPT, X566T, recurrent glioblastoma (rGBM), pediatric brain cancer
Axsome Therapeutics (AXSM), solriamfetol, CLARITY, Phase 3, major depressive disorder (MDD), excessive daytime sleepiness (EDS), randomized withdrawal
IRLAB Therapeutics (IRLAB A), IRL757, LIFT-PD, Parkinson’s disease apathy, MSRD, Otsuka
Evox Therapeutics, Rett Syndrome Research Trust (RSRT), ExoEdit, exosome delivery, gene editing, MECP2, Rett syndrome
GEMMABio (GEMMA Biotherapeutics), GB221, CHARISMA, SMA1, intracisterna-magna (ICM), rare pediatric disease designation (RPDD), Fiocruz
uniQure (QURE), AMT-130, Huntington’s disease, FDA Type A meeting minutes, sham-controlled study
Roche, fenebrutinib, BTK inhibitor, FENhance 1, FENhance 2, FENtrepid, RMS, PPMS, teriflunomide
MSAA, MS Awareness Month, self-advocacy, patient education
Novartis (NVS), Avidity Biosciences, Antibody Oligonucleotide Conjugates (AOC), acquisition