FDA Approves DaxibotulinumtoxinA for Cervical Dystonia, Genetic Parkinson Trial Announced, DBS Improves Poststroke Mobility


Neurology News Network for the week ending August 19, 2023. [WATCH TIME: 4 minutes]

WATCH TIME: 4 minutes

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

The FDA has approved Revance’s daxibotulinumtoxinA-Ianm (Daxxify) for the treatment of cervical dystonia in adults, expanding its previous label as a therapy for the temporary improvement of moderate to severe glabellar lines. Powered by Peptide Exchange Technology, it becomes the first and only peptide-formulated, long-lasting neuromodulator for patients with cervical dystonia. The expanded indication was based on results from the phase 3 ASPEN trial and its open-label study, which included 382 patients and 1240 treatments over an 88-week time span. In ASPEN-1 both the 125- and 250-unit dose groups of daxibotulinumtoxinA met the primary end point of clinically meaningful improvement in the signs and symptoms of cervical dystonia, on average, by weeks 4 and 6.

BIAL R&D announced the dosing of the first patient in its phase 2 clinical trial, ACTIVATE, to investigate BIA 28-6156, an allosteric activator of the enzyme beta-glucocerebrosidase, as a treatment of patients with genetically-mutated Parkinson disease.. The trial, which includes those with a mutation in the GBA1 gene (GBA-PD), otherwise the most common genetic risk factor of the disease, is screening patients across sites in North America and with a Europe-based trial planned to initiate in the third quarter of 2023. Investigators will recruit and enroll patients who were diagnosed with PD between 1 to 7 years prior to genetic screening, have a modified Hoehn and a Yahr score at most of 2.5, as well as a score of at least 22 on the Montreal Cognitive Assessment. Also, to be eligible, participants must be on stable dose of PD medication and continue the treatment throughout the duration of the study.

Investigators at Cleveland Clinic published promising findings from a phase 1 trial assessing deep brain stimulation (DBS) of the dentate nucleus in individuals with chronic poststroke hemiparesis. All told, patients demonstrated rehabilitative effects and associated neurophysiological gains, further supporting the use of this intervention for recovery of function, even in those in late phases of disability. Published in Nature, the trial featured 12 stroke survivors with persistent, moderate-to-severe upper extremity impairment who underwent DN-DBS combined with renewed physical rehabilitation. Over 168 participant-months of DBS implant experience and 72 months of DN stimulation experience, treatment resulted in no device failures and no study-related, serious adverse events (AEs). Overall, there was a median decrease of 0.5 points on the upper extremity Fugl-Meyer (FM-UE) Assessment between the pre- and post-surgery time points (month 1 vs 0) for the full sample of participants, further supporting the safety of the surgical implant procedure.

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