What's next in neurology — October 13, 2026

This edition covers what is coming in neurology between mid-November 2026 and the first half of 2027: a phase 3 readout in early Alzheimer disease, three FDA decisions with published dates (Duchenne muscular dystrophy, SCN2A/SCN8A epileptic encephalopathies and MOGAD), and a priority review in multiple sclerosis that has no announced date yet.

1. Oral AR1001 (mirodenafil) in early Alzheimer disease: Polaris-AD results

[Ongoing trial] [Global (13 countries, including the US, Europe, South Korea and China)] [Estimated date: November 2026, per AriBio (presentation announced for CTAD, Boston, November 16–19, 2026)]
What it is: Randomized, double-blind phase 3 trial (NCT05531526) that enrolled 1,535 patients with MCI due to AD or mild AD dementia and confirmed amyloid (MMSE 20–30), AR1001 30 mg daily versus placebo for 52 weeks; primary outcome: change in CDR-SB at week 52.
What problem it solves: Today the only disease-modifying option is anti-amyloid antibodies, which require infusions, serial MRI for ARIA and specialized centers; an oral drug without imaging surveillance would broaden access.
Critical take: Manufacturer-funded trial of a mechanism (PDE5 inhibition) that does not act directly on amyloid; the result may be negative and, even if positive, the size of the CDR-SB effect will matter before any claim of clinical benefit.
https://clinicaltrials.gov/study/NCT05531526
https://www.alzint.org/news-events/events/clinical-trials-on-alzheimers-disease-ctad-2026/
https://view.asiae.co.kr/en/article/2026072808394224049

2. Deramiocel in Duchenne muscular dystrophy: FDA decision

[Pending regulatory decision] [US] [Confirmed date: November 22, 2026]
What it is: Intravenous cell therapy (cardiosphere-derived cells, 150 million every 3 months); in the phase 3 HOPE-3 trial (randomized, double-blind, 106 patients randomized, 105 in the primary analysis, 12 months) it slowed progression on the PUL 2.0 upper-limb scale by 54% versus placebo (p = 0.029), according to the manufacturer. After the advisory committee, Capricor submitted 24-month open-label extension data, additional analyses and an indication narrowed to upper-limb function; the FDA classified this as a major amendment and moved the date from August 22 to November 22, 2026.
What problem it solves: Loss of arm function and cardiomyopathy in non-ambulant Duchenne have few options beyond corticosteroids, exon-skipping therapies for specific mutations and standard cardiac care.
Critical take: On July 29, 2026 the FDA advisory committee voted 9 to 3 that there is no substantial evidence of effectiveness in Duchenne cardiomyopathy. The FDA questioned changes to the statistical analysis plan made after the randomized phase ended (the latest version was finalized the same day as unblinding) and the possibility that blinding was compromised; under the original plan, its analysis found no significant difference on PUL 2.0. Approval is far from assured.
https://www.sec.gov/Archives/edgar/data/0001133869/000110465926100071/capr-20260824x8k.htm
https://www.capricor.com/investors/sec-filings/all-sec-filings/content/0001104659-25-117947/capr-20251203xex99d1.htm
https://www.neurologylive.com/view/fda-advisory-committee-votes-against-deramiocel-dmd-cardiomyopathy

3. Relutrigine in SCN2A and SCN8A epileptic encephalopathies: FDA decision

[Pending regulatory decision] [US] [Confirmed date: December 27, 2026]
What it is: Preferential blocker of the persistent sodium current; in the phase 2 EMBOLD study (NCT05818553, randomized, double-blind, placebo-controlled, ages 2 to 18) the confirmatory cohort reduced motor seizures by 53% placebo-adjusted at 16 weeks (cohort 1: 46%), according to Praxis. The FDA moved the date from September 27 after a "major amendment" (additional sensitivity analyses), without requesting new studies.
What problem it solves: DEEs caused by gain-of-function SCN2A/SCN8A variants present with early-onset refractory seizures; today clinicians use antiseizure combinations (including classic sodium-channel blockers) with inconsistent response and no specifically approved treatment.
Critical take: Few patients, short follow-up and Praxis sponsorship; it requires genetic diagnosis, which is not always available or covered in our settings.
https://ir.praxismedicines.com/news-releases/news-release-details/praxis-precision-medicines-announces-extension-period
https://aesnet.org/abstractslisting/relutrigine-demonstrates-robust-seizure-reduction-and-seizure-freedom-in-dees-results-from-the-embold-study
https://praxismedicines.com/wp-content/uploads/2025/12/Gil-Nagel_AES2025_EMBOLD-Topline.pdf

4. Satralizumab in MOGAD: first possible approval for the disease

[Pending regulatory decision] [US; EU in 2027] [Confirmed date: FDA decision expected by January 10, 2027, per Roche]
What it is: Anti-IL-6 receptor antibody already approved for NMOSD; in the phase 3 METEOROID trial (double-blind, placebo-controlled) it reduced the risk of first relapse by 68% (p = 0.0025); at 48 weeks, 87% were relapse-free versus 67% on placebo. Priority review in the US; the EMA has validated the application, with a European decision expected in the third quarter of 2027.
What problem it solves: MOGAD has no approved treatment; relapses are currently prevented off-label with corticosteroids, IVIG, rituximab or tocilizumab, based on observational evidence.
Critical take: Roche-sponsored trial; a substantial share of MOGAD patients have a monophasic course, so the key decision will be whom to treat and for how long.
https://www.roche.com/media/releases/med-cor-2026-09-10

5. Fenebrutinib in relapsing and primary progressive multiple sclerosis

[Pending regulatory decision] [US] [Estimated date: first half of 2027, per the FDA's six-month priority-review timeline (BioPharma Dive); Roche has not published a date]
What it is: Oral reversible BTK inhibitor; versus teriflunomide (FENhance 1 and 2, 96 weeks) it reduced annualized relapse rate by 51.1% and 58.5%; versus ocrelizumab in PPMS (FENtrepid) the HR for 12-week composite confirmed disability progression was 0.88 (95% CI 0.75–1.03), without superiority. Roche announced on September 30, 2026 that the FDA accepted the application under priority review.
What problem it solves: It would offer a high-efficacy oral alternative to intravenous or subcutaneous anti-CD20 therapy, and a second option in PPMS, where only ocrelizumab is approved today.
Critical take: In PPMS there were more liver enzyme elevations than with ocrelizumab (versus teriflunomide they were comparable), and Roche acknowledges an imbalance in deaths across the three pivotal studies; in relapsing MS Roche describes disability progression only as "consistent positive trends".
https://www.roche.com/media/releases/med-cor-2026-09-30
https://biopharmadive.com/news/roche-fenebrutinib-ms-fda-accept-application-btk/831746

*What's next in neurology — October 13, 2026*
1. Early Alzheimer: phase 3 results for oral AR1001 (Polaris-AD), announced for CTAD, Nov 16–19, 2026.
2. Duchenne: FDA decides on deramiocel on Nov 22, 2026 (its advisory committee voted 9-3 against in July).
3. SCN2A/SCN8A encephalopathies: FDA decides on relutrigine on Dec 27, 2026.
4. MOGAD: satralizumab, FDA decision by Jan 10, 2027; if approved, it would be the first approved treatment for the disease.
5. Multiple sclerosis: fenebrutinib under priority review; decision estimated in the first half of 2027.
Full text and audio: https://boletinesmedicos.com/en/neurology/2026-10-13-viene.html
MedBulletins · boletinesmedicos.com/en/

What's next in neurology (November 2026 – first half of 2027), with sources:

• AR1001, an oral PDE5 inhibitor, will present phase 3 results in early Alzheimer disease (1,535 patients) at CTAD, November 16–19.
• Deramiocel in Duchenne: FDA decision on November 22, after an advisory committee voted 9-3 against in July.
• Relutrigine in SCN2A/SCN8A epileptic encephalopathies: decision on December 27.
• Satralizumab in MOGAD: decision by January 10, 2027; nothing is approved today.
• Fenebrutinib in relapsing and primary progressive MS: priority review, no published date.

For each item: the problem it solves, current practice and a critical take.
https://boletinesmedicos.com/en/neurology/2026-10-13-viene.html

#Neurology #Alzheimers #Epilepsy #MultipleSclerosis #MedicalBulletins

What's next in neurology: AR1001 in Alzheimer (CTAD, Nov 16-19), deramiocel in Duchenne (FDA Nov 22), relutrigine in SCN2A/SCN8A (Dec 27), satralizumab in MOGAD (Jan 10) and fenebrutinib in MS. https://boletinesmedicos.com/en/neurology/2026-10-13-viene.html