FDA Approves Medtronic's Adaptive DBS, Hold Lifted on ELEVATE-44-102 Trial, Ecopipam Meets Primary and Secondary End Points in Tourette

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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 Adaptive deep brain stimulation (aDBS) and BrainSense Electrode Identifier (EI), marking a significant advancement in personalized care for patients with Parkinson disease (PD). The decision comes less than 2 months after the European Union gave CE Mark approval for the pair of digital modalities to be used in PD care. Each technology is a bit different: aDBS dynamically adjusts DBS in real-time based on patient-specific brain activity whereas the EI optimizes initial DBS programming by precisely identifying the strongest signal location. Research has shown that through EI, clinicians can conduct more accurate and precise initial programming that is 85% faster than traditional electrode selection.

The FDA has lifted its clinical hold on Entrada Therapeutics’ investigational new drug (IND) application for ENTR-601-44 in Duchenne muscular dystrophy (DMD) and provided authorization to initiate ELEVATE-44-102, a randomized, double-blind placebo-controlled, multiple ascending dose (MAD) phase 1b study assessing the investigational therapy in adult patients with a confirmed mutation in the DMD gene amenable to exon 44 skipping.The phase 1b trial is investigating the safety and tolerability of ENTR-601-44 in approximately 32 nonambulatory and ambulatory adult patients with DMD. Entrada noted that MAD study is also designed to assess target engagement as measured by exon skipping and dystrophin production, and pharmacokinetics. Dosing for the trial will be administered every 6 weeks, with the planned doses across 4 cohorts anticipated to range from 0.16 mg/kg up to 1.28 mg/kg.

New topline data from the phase 3 D1AMOND study (NCT05615220) showed that Emalex Biosciences’ ecopipam, an investigational dopamine-1 receptor antagonist, met its primary and secondary end points among patients with Tourette syndrome (TS). The company noted that it plans to meet with the FDA and other global health authorities to discuss a potential new drug application submission for ecopipam in TS later this year. For the primary efficacy end point, measured by the time to relapse among pediatric patients, results indicated that 41.9% of those randomized to ecopipam experienced a relapse, compared with 68.1% of those on placebo (hazard ratio, 0.5 [0.3-0.8]; P = .0084). As for the secondary efficacy end point, which assessed time to relapse in both pediatric and adult participants after randomization, findings showed that 41.2% of patients in the ecopipam group relapsed, compared with 67.9% in the placebo group (hazard ratio, 0.5 [0.3-0.8]; P = .0050).

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