AMX0035 Shows Safety in Alzheimer Disease, Outcomes of Urgent Prevention in TIA, FDA Grants IDE to NeuroPace's RNS System


Neurology News Network for the week ending November 20, 2021.

This week Neurology News Network covered the phase 2 results of AMX0035 in Alzheimer disease, the 10-year follow-up of the EXPRESS study evaluating urgent prevention of transient ischemic attack, and the investigational device exemption granted to NeuroPace's responsive neurostimulation system for idiopathic generalized epilepsy.

Welcome to this special edition of Neurology News Network. I’m Marco Meglio. Please excuse our appearance this week as a majority of the US workforce, including the NeurologyLive team, moves to working remote as we come together to help reduce the spread of the novel coronavirus.

Clinical data from the phase 2 AMX-8000 PEGASUS trial (NCT03533257) of AMX0035, a combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO), in patients with Alzheimer disease (AD) were presented at the 14th Clinical Trials on Alzheimer’s Disease Conference (CTAD), confirming the treatment met its primary end point of safety and tolerability.1 Similar to previous clinical trial findings in amyotrophic lateral sclerosis (ALS), treatment with AMX0035 was found to be associated with a higher incidence of gastrointestinal events when compared with placebo. A total of 133 patients with dementia or mild cognitive impairment (MCI) because of AD were screened, with a total of 95 patients eventually included in the double-blind, placebo-controlled, multicenter trial. Also noted was the possibility of AMX0035 activity improving certain biomarkers of AD pathology, namely amyloid beta species (Aß1-42 and Aß1-40), total tau (t-tau), and phospho-tau 181 (p-tau) in the cerebrospinal fluid (CSF).

Findings from a 10-year follow-up analysis of the EXPRESS study suggest that urgent assessment and treatment of transient ischemic attack (TIA) or minor stroke have long-term benefits, with reductions in recurrent strokes and improved outcomes irrespective of age. Additionally, patients demonstrated an increased disability-free and quality-adjusted life expectancy, making urgent acute prevention highly cost-effective.EXPRESS was a prospective population-based before versus after study that assessed the effects of early assessment and treatment of TIA or minor stroke through outpatient clinics. Previously reported results showed that this method of care reduced 90-day stroke risk by 80%.The 10-year risk of disabling/fatal recurrent stroke was significantly lower in those patients treated in the phase 2 clinic. Notably, after 90 days, neither phase group differed in terms of recurrent or disabling stroke.

According to a recent announcement, the FDA has granted an investigational device exemption (IDE) to NeuroPace for its responsive neurostimulation (RNS) system to be evaluated in patients with drug-resistant idiopathic generalized epilepsy. The news comes just a few months after the company announced it had been granted breakthrough device designation status from the FDA for the treatment of IGE. Dubbed NAUTILUS, this new, prospective, single-blind, multicenter, randomized pivotal study will be the first to evaluate the effects of brain-responsive neuromodulation in this patient population. Enrollment is expected to begin in 2022. NeuroPace has yet to announce further details of the study, including primary and secondary end points. The RNS System was originally approved in 2013 for the treatment of medical refractory epilepsy and remains the only such of its kind approved for this patient population. It acts on closed-looped technology that monitors and responds to a patient’s unique brain patterns to deliver therapy in real time prior to symptom onset.

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