This week’s Neuroscience update highlights partnership activity, clinical trial progress, new long-term data, financing momentum, and emerging device evidence across neurological disorders.
In Today’s Newsletter
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In Today’s Newsletter
🤝 Rapport Therapeutics, Tenacia team up on RAP-219 in Greater China [1] [China • 09 Mar 2026]
https://investors.rapportrx.com/news-releases/news-release-details/rapport-therapeutics-and-tenacia-biotechnology-announce
Context: RAP-219 (Rapport Therapeutics; TARPγ8-specific AMPA receptor modulator) is in development for focal onset seizures and bipolar mania, with Phase 3 in focal onset seizures expected to begin in Q2 2026.
Key point: Tenacia Biotechnology received exclusive Greater China rights to develop and commercialize RAP-219, while Rapport keeps ex-Greater China rights and disclosed a $20 million upfront, up to $308 million in milestones, and tiered royalties.
Implication: Signals pipeline investment and modality expansion.
🧬 Therna, Charles River launch single-patient RNA collaboration [2] [US • 05 Mar 2026]
https://sg.finance.yahoo.com/news/therna-announces-collaboration-charles-river-130000083.html
Context: The first two programs target an adult with ultra-rare lung fibrosis and a newborn with Lamb-Shaffer syndrome, using mRNA and ASO approaches respectively.
Key point: Therna Biosciences and Charles River said they will advance personalized RNA medicines for severe ultra-rare disorders, with Charles River leading preclinical development support.
Implication: Signals pipeline investment and modality expansion.
💪 Dyne reports new DELIVER cardiopulmonary data for z-rostudirsen in DMD [3] [US • 08 Mar 2026]
https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-new-positive-cardiopulmonary-results
Context: The ongoing Phase 1/2 DELIVER trial evaluates z-rostudirsen in exon 51 skip-amenable Duchenne muscular dystrophy, with prior safety follow-up reported in 86 participants.
Key point: Dyne Therapeutics said new 24-month analyses showed improvement from baseline in lung and cardiac function measures versus expected natural history declines.
Implication: May influence prescriber choice and payer reviews pending full data.
🏃 Dyne starts Phase 3 HARMONIA for z-basivarsen in DM1 [4] [US • 08 Mar 2026]
https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-initiation-phase-3-harmonia-trial-z
Context: HARMONIA is a global, randomized, placebo-controlled, double-blind Phase 3 study in about 150 people with myotonic dystrophy type 1, with a 48-week treatment period.
Key point: Dyne Therapeutics initiated HARMONIA, designed with FDA alignment, with the five times sit to stand test as the primary endpoint and broader functional, CNS, and reported-outcome measures as secondary or exploratory endpoints.
Implication: May influence prescriber choice and payer reviews pending full data.
🧠 TG Therapeutics publishes BRIUMVI data in highly active RMS [5] [US • 09 Mar 2026]
https://ir.tgtherapeutics.com/news-releases/news-release-details/data-published-neurology-and-therapy-demonstrate-significant
Context: The post hoc pooled analysis drew from Phase 3 ULTIMATE I and II data in 168 participants with highly active disease at baseline, followed for 96 weeks.
Key point: TG Therapeutics reported that BRIUMVI (ublituximab-xiiy) significantly reduced relapse rates and MRI lesion activity, and increased NEDA-3 rates versus teriflunomide in highly active relapsing MS.
Implication: May influence prescriber choice and payer reviews pending full data.
💰 Cognito raises $105 million to advance Spectris in Alzheimer’s disease [6] [US • 05 Mar 2026]
https://www.businesswire.com/news/home/20260305121811/en/Cognito-Therapeutics-Announces-Oversubscribed-%24105-Million-Series-C-Financing-to-Advance-Spectris-in-Alzheimers-Disease
Context: Cognito Therapeutics said the HOPE pivotal study of Spectris is fully enrolled, with top-line data anticipated later in 2026; Spectris remains an investigational device.
Key point: Cognito Therapeutics closed an oversubscribed $105 million Series C to support pivotal readout, regulatory submission, launch readiness, and broader platform development.
Implication: Signals pipeline investment and modality expansion.
📘 SPR reports retrospective data on 60-day PNS for chronic shoulder pain [7] [US • 05 Mar 2026]
https://www.manilatimes.net/2026/03/05/tmt-newswire/globenewswire/predictors-of-success-for-60-day-peripheral-nerve-stimulation-pns-used-for-treatment-of-chronic-shoulder-pain-a-multi-center-retrospective-chart-review/2293457
Context: The multicenter retrospective chart review included 221 patients across five sites and was published in the American Journal of Physical Medicine & Rehabilitation.
Key point: The 60-day SPR SPRINT Peripheral Nerve Stimulation system was associated with substantial pain reduction in many patients, and bimodal stimulation showed higher response rates than motor-only or sensory-only paradigms.
Implication: Could inform practice and payer discussions; interpretation depends on study design and confounding control.
Why it Matters
- Neurology remained a clear theme this week, spanning epilepsy, bipolar mania, multiple sclerosis, Alzheimer’s disease, DMD, and DM1 [1][3][4][5][6].
- Partnership and financing news suggest continued investor and business-development interest in CNS and neurotechnology programs [1][6].
- Dyne added both near-term execution and longer-term evidence signals, combining new DELIVER analyses with a Phase 3 start in DM1 [3][4].
- Personalized medicine continues to move toward highly individualized development models, including single-patient RNA programs in ultra-rare disease [2].
- Real-world and retrospective evidence remains active in device-based pain care, though interpretation still depends on study design limits [7].
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FAQ
What is RAP-219, and what changed this week?
RAP-219 is Rapport Therapeutics’ investigational TARPγ8-specific AMPA receptor modulator for neurological and psychiatric disorders. This week, Tenacia obtained exclusive Greater China rights under a collaboration that includes upfront, milestone, and royalty economics for Rapport [1].
What does the Therna and Charles River collaboration cover?
The collaboration focuses on single-patient RNA therapeutics for severe ultra-rare disorders, starting with programs in rare lung fibrosis and Lamb-Shaffer syndrome. Charles River is supporting preclinical development, while Therna contributes its AI-enabled RNA design platform [2].
What did Dyne report for z-rostudirsen in Duchenne muscular dystrophy?
Dyne said new DELIVER analyses out to 24 months showed improvement in cardiopulmonary measures relative to expected natural history decline. The release also noted previously reported favorable safety and tolerability findings in the trial population [3].
What is the HARMONIA study testing in DM1?
HARMONIA is Dyne’s Phase 3 confirmatory trial of z-basivarsen in myotonic dystrophy type 1. It plans to enroll about 150 participants and uses five times sit to stand at week 49 as the primary endpoint [4].
What stood out in the BRIUMVI publication?
The published post hoc pooled analysis in highly active relapsing MS showed lower relapse and MRI activity, plus higher NEDA-3 rates, for BRIUMVI versus teriflunomide. The subgroup included 168 participants from ULTIMATE I and II [5].
Is Spectris already approved for Alzheimer’s disease?
No. Cognito described Spectris as an investigational device that is not approved by the U.S. Food and Drug Administration. The new Series C financing is intended to support pivotal readout, submission, and launch preparation [6].
Entities / Keywords
Rapport Therapeutics, Tenacia Biotechnology, RAP-219, focal onset seizures, bipolar mania, Greater China, Therna Biosciences, Charles River, personalized RNA therapeutics, mRNA, antisense oligonucleotides, ASO, Lamb-Shaffer syndrome, Dyne Therapeutics, z-rostudirsen, DYNE-251, DELIVER, Duchenne muscular dystrophy, DMD, exon 51 skipping, z-basivarsen, DYNE-101, HARMONIA, myotonic dystrophy type 1, DM1, TG Therapeutics, BRIUMVI, ublituximab-xiiy, ULTIMATE I, ULTIMATE II, relapsing multiple sclerosis, RMS, NEDA-3, Cognito Therapeutics, Spectris, HOPE study, Alzheimer’s disease, SPR, SPRINT Peripheral Nerve Stimulation, PNS, chronic shoulder pain, bimodal stimulation
References
- https://investors.rapportrx.com/news-releases/news-release-details/rapport-therapeutics-and-tenacia-biotechnology-announce
- https://sg.finance.yahoo.com/news/therna-announces-collaboration-charles-river-130000083.html
- https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-new-positive-cardiopulmonary-results
- https://investors.dyne-tx.com/news-releases/news-release-details/dyne-therapeutics-announces-initiation-phase-3-harmonia-trial-z
- https://ir.tgtherapeutics.com/news-releases/news-release-details/data-published-neurology-and-therapy-demonstrate-significant
- https://www.businesswire.com/news/home/20260305121811/en/Cognito-Therapeutics-Announces-Oversubscribed-%24105-Million-Series-C-Financing-to-Advance-Spectris-in-Alzheimers-Disease
- https://www.manilatimes.net/2026/03/05/tmt-newswire/globenewswire/predictors-of-success-for-60-day-peripheral-nerve-stimulation-pns-used-for-treatment-of-chronic-shoulder-pain-a-multi-center-retrospective-chart-review/2293457