FDA Authorizes Pivotal Phase 3 Clinical Trial For Alzheimer Treatment

The Food and Drug Administration (FDA) has permitted the initiation of a pivotal phase 3 clinical trial (NCT04669028) for the anti-inflammatory 17α-ethynyl-androst-5-ene-3,7,17-triol (HE3286)(NE3107; NeurMedix, San Diego, CA) for potential Alzheimer disease (AD) treatment. Increasing evidence links both inflammation and insulin resistance to AD dementia and progression. 

Approximately 316 participants will be randomly assigned 1:1 ratio to receive HE3286 or placebo. Inclusion/exclusion criteria are structured to help select only individuals with AD and exclude those with cognitive impairment secondary to other medical conditions. Stable regime of approved AD comedication is permitted and continued use of glycemic control medications that were at a stable dose for 3 months prior to randomization is permitted. Change from baseline to week 30 in Alzheimer's Disease Assessment Scale-Cognitive Subscale (ADAS-Cog) and mean change from baseline to week 30 in Alzheimer’s Disease Cooperative Study-Clinical Global Impression of Change (ADCS-CGIC) will be evaluated. 
Terren Peizer, chairman and chief executive officer, and majority shareholder of NeurMedix and BioVie, stated, “It is well-known that insulin resistance predicts neuroinflammation, and cognitive decline, and that up to 81% of AD patients have impaired glucose tolerance or type 2 diabetes. We believe NE3107 reduces neuroinflammation and restores insulin sensitivity, and thereby may halt the progression of Alzheimer disease. Of the 4 indications that we hope to pursue in the coming year, we believe that the application of NE3107 to AD has a very high probability of success as it uniquely addresses all of the known pathways of AD. The FDA’s authorization of this pivotal phase 3 clinical trial is an important milestone in our pursuit of a life changing, safe, and efficacious intervention for this largest unmet medical need.”

HE3286 is an anti-inflammatory metabolite of the adrenal steroid, dehydroepiandrosterone (DHEA). The phase 3 randomized double-blind placebo-controlled parallel group clinical trial will evaluate twice daily 20 mg oral HE3286 versus placebo for 30 weeks with a starting dose of 5 mg twice daily, uptitrated by doubling every 2 weeks to a dose of 20 mg twice daily in weeks 5-30.