LX2006 Improves Cardiac Biomarkers of FA, ATH434 Positive Results in MSA, Brivaracetam Effective Regardless of Intellectual Disabilities

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

Newly announced interim data from the phase 1/2 SUNRISE-FA clinical trial (NCT05445323) an investigation-initiated phase 1a trial (NCT05302271) showed that LX2006 (Lexeo Therapeutics), an investigational gene therapy, was safe and well tolerated, with meaningful improvements in cardiac biomarkers among patients with Friedreich ataxia (FA) cardiomyopathy. An independent data and safety monitoring board has ruled that the drug may proceed to an additional higher dose cohort expected to include at least 3 patients with the disease. SUNRISE-FA, an ongoing, dose-ascending, open-label trial, includes approximately 9 individuals with FA-associated cardiomyopathy who are followed for a 52-week period.

Newly announced interim data from a phase 2 open-label study (NCT05864365) showed that treatment with investigational ATH434 (Alterity Therapeutucs) was safe, with noted improvements in disability and biomarker assessments after 6 months in patients with multiple system atrophy (MSA). Final, 12-month data from the trial are expected in the first half of 2025. Within the small-scale trial, which included 10 participants with MSA, interim data comprised of 7 patients who completed 6 months of treatment with ATH434, 3 of whom who also completed 12 months of treatment. ATH434, an oral agent designed to inhibit the aggregation of pathological proteins implicated in neurodegeneration, was considered well tolerated by patients, with most adverse events (AEs) mild to moderate in severity.

In a recently published study, brivaracetam (Briviact; UCB), an FDA-approved antiseizure medication (ASM), demonstrated similar efficacy, tolerability, and physical and mental health adverse effects among patients with epilepsy regardless of whether they had intellectual disability (ID). These data, along with previously published real-world studies, indicate that brivaracetam may be an appropriate ASM for patients with ID. Pooled data from 12 UK NHS Trusts from 2016–2022 were analyzed, comprising 102 patients with epilepsy without ID and 37 with ID. Of those with ID, 17 were mild and 20 were moderate to profound. Those with ID were more likely to be younger (P <.001), male (P <.005), and to have existing neurodevelopment conditions (P <.001). Between the 2 groups, those with no ID had slightly higher mean starting doses of brivaracetam (57 mg vs 49 mg) for the 12-month study.

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