Topline Data on NE3017 Announced, Pitolisant Improves EDS in DM1, Nerivio Reduces Migraine Treatment Days


Neurology News Network for the week ending December 16, 2023. [WATCH TIME: 3 minutes]

WATCH TIME: 3 minutes

Welcome to this special edition of Neurology News Network. I’m Marco Meglio.

Newly announced topline data from a phase 3 study of BioVie’s Alzheimer agent NE3107 showed beneficial treatment effects over placebo that were potentially equal to or greater than data reported from previously approved antiamyloid therapies. In addition, patients on the permeable anti-inflammatory insulin sensitizer saw an average of more than 5 years of age deceleration in comparison with placebo, becoming the first such agent to demonstrate this impact on DNA methylation, according to the company. At the conclusion of the analysis, NE3017-treated patients demonstrated improved performance compared with placebo on several cognitive and functional assessments, which included the Clinical Dementia Rating-Sum of Boxes (CDR-SB), Alzheimer’s Disease Assessment Scale-Cognitive subscale (ADAS-Cog12), Mini-Mental State Examination (MMSE), Alzheimer’s Disease Cooperative Study-Activities of Daily Living (ADCS-ADL), ADCS-Clinical Global Impression of Change (GCIC), and AD Composite Score.

Harmony Biosciences has announced topline data from its phase 2 signal detection study of adults with myotonic dystrophy type 1, with results showing that treatment with pitolisant (Wakix) improved excessive daytime sleepiness and fatigue, 2 common symptoms of DM1. Although not powered for statistical significance, the results suggest that pitolisant, a selective histamine 3 receptor antagonist/inverse agonist, could be a potentially effective therapy for patients with DM1 with these symptoms. From baseline to the end of the 11-week double-blind treatment period, patients on high- and low-dose pitolisant reported mean changes of –2.5 and –1.0 in Daytime Sleepiness Scale the primary efficacy end point, in comparison with changes of –0.2 for those on placebo. On Epworth Sleepiness Scale, investigators reported mean changes of –4.88 for the higher dose pitolisant group vs –0.10 for placebo. Above all, the safety profile of the agent was consistent with previous studies, with no new safety signals or serious adverse events reported.

A real-world study (NCT05769322) published in Frontiers in Pain Research demonstrated that frequent use of Theranica’s remote electrical device (REN; Nerivio) as an acute treatment for migraine in adolescents resulted in reductions in mean monthly treatment days in the subsequent months, suggesting a preventive benefit in this subpopulation. All told, results showed a significant month-to-month decrease in the primary end point of mean monthly migraine treatment days (MMTD), reducing from 12.6 MMTD in the first month to 9.0 in the second month of consecutive use. The number of MMTD further decreased to 7.4 MMTD in the third month, reflecting an additional reduction of 1.6 MMTD in the third month of treatment.

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