Aducanumab Reversal Sends Drug to FDA, Cala Trio Reduces Hand Tremor, Ganaxolone Promising in Refractory Status Epilepticus


Neurology News Network for the week ending October 26, 2019.

This week, Neurology News Network covered the stunning decision reversal for aducanumab as Biogen announced a new analysis showed positive results for the early Alzheimer drug, results of the PROSPECT trial showing significant improvement in hand tremor with the wrist-worn Cala Trio device, and results of a phase 2 trial of ganaxolone in refractory status epilepticus.

Marco: Welcome to Neurology News Network. I’m Marco Meglio. Let’s get into the news from this week.

In a surprising reversal, Biogen has announced that it will bring its anti-amyloid drug aducanumab to the FDA for approval despite failing a previous futility analysis earlier this year. The decision to proceed with the regulatory process was made after Biogen analyzed an expanded dataset from the phase 3 EMERGE trial that included an additional 3 months of data on patients receiving the high-dose drug. They reported observing statistically significant changes on the Clinical Dementia Rating-Sum of Boxes score. Notably, data from the identical phase 3 ENGAGE study did not show the same results, with the study drug performing worse than placebo on the primary end point. Still, Biogen said that data from patients in the ENGAGE trial who achieved sufficient exposure to a high-dose of the drug support the findings from the EMERGE trial.

Results of the landmark PROSPECT trial of the Cala Trio, a wrist-worn wearable neuromodulation device for essential tremor, suggest that the device is safe and effective for the reduction of hand tremor. PROSPECT, the largest trial ever conducted for essential tremor, included 263 patients at 26 sites. Treatment with the device was associated with significant improvements in symptomatic relief at 3 months compared with baseline. Patients utilized the device for 40-minute sessions, twice daily, over the 3-month study period, with each device calibrated for the patient using it. At month 3, 62% of patients improved from severe/moderate to mild/slight on the TETRAS scale and 68 percent of patients improved from severe/moderate on the ADLs scale, with an average symptom relief of 96.7 minutes after each session with the device.

Marinus Pharmaceuticals presented positive results from its phase 2 trial of ganaxolone at the Neurocritical Care Society‘s 17th Annual Meeting. The top-line findings showed that no patients progressed to intravenous anesthetics within 24 hours of treatment initiation. Treatment with ganaxolone also reduced median time to status cessation to 5 minutes. Notably, 16 of 17 patients were status-free 24 hours from infusion initiation. During the follow-up period, 3 patients in the 500 mg group ultimately escalated to additional IV AEDs or IV anesthetics due to status relapse during that time. The positive results support a potential pivotal phase 3 study of the drug.

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