Medtronic DBS Approved for Asleep Surgery, BHV-2100 Shows Early Promise, AOC 1044 Continues Dystrophin Production in Latest Update

WATCH TIME: 4 minutes

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

According to a new announcement, the FDA has granted approval to Medtronic’s deep brain stimulation (DBS) technology to be used while a patient is under general anesthesia, or asleep, in the context of treating Parkinson disease (PD) or essential tremor. Typically, the standard established DBS procedure is based on awake surgery with intraoperative microelectrode recording associated with clinical testing to ensure good positioning of the lead; however, this procedure is sometimes perceived as uncomfortable for patients who are unable to tolerate awake surgery with extensive time off their medication. Ultimately, the FDA’s decision to approve Medtronic’s DBS for asleep purposes may provide greater treatment opportunities to a significant portion of potential candidates for surgery.

New data from a randomized, placebo-controlled sequential single-ascending dose (SAD) study showed that treatment with BHV-2100 (Biohaven), a TRPM3 antagonist, was safe and well tolerated at single doses up to 500 mg. All told, the therapy was rapidly absorbed with a highly favorable pharmacokinetic profile, further supporting its future development into clinical trials for pain and migraine as a novel non-opioid treatment. Presented at the 2024 World Congress on Pain, held August 5-9 in Amsterdam, the Netherlands, the SAD portion of the trial included 39 healthy individuals who were randomly assigned 3:1 a single dose of to BHV-2100 (25, 75, 150, 250, or 500 mg) or placebo. At the conclusion of the trial period, there were no serious or severe adverse events (AEs) reported, and most AEs were mild and resolved spontaneously without treatment. In addition, there were no dose-limiting toxicities observed as well.

Avidity Biosciences recently announced new positive data from EXPLORE44, its phase 1/2 trial assessing AOC 1044, an antisense oligonucleotide, as a potential therapy for patients with Duchenne muscular dystrophy (DMD). Treatment with the therapy, otherwise known as delpacibart zotadirsen (or del-zota), resulted in statistically significant increases in dystrophin production and reductions in creatine kinase levels along with a favorable safety and tolerability profile. EXPLORE44, a randomized, placebo-controlled study, includes 25 patients with DMD across the US and Canada who are randomly assigned 3:1 to investigational AOC 1044 or placebo for a treatment period lasting approximately 12 or 16 weeks (dependent upon 6 or 8 week infusion schedule). In the study, AOC 1044 at 5 mg/kg demonstrated unsurpassed delivery of PMO concentrations of 200 nM in skeletal muscle. This group also showed a statistically significant increase of 25% of normal in dystophin production and a statistically significant increase of 37% in exon 44 skipping in studied patients, which had DMD mutations amenable to exon 44 skipping.

For more direct access to expert insight, head to NeurologyLive.com. This has been Neurology News Network. Thanks for watching.