PhenoNet has received approval from the US Food and Drug Administration (FDA) to proceed with a Phase III clinical trial evaluating its investigational treatment, PHENOGENE-1A, in individuals with early Alzheimer’s disease.
The FDA issued a “May Proceed” notification to the company activating its investigational new drug (IND) application for this study.
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PHENOGENE-1A, an inhaled version of cromolyn, utilises PhenoNet’s targeted delivery technology with the intention of delivering therapeutic levels of the drug to the brain.
It is designed based on three hypothesised mechanisms related to Alzheimer’s: inhibiting amyloid-β aggregation, moderating pro-inflammatory cytokines and chemokines, and encouraging microglial removal of amyloid-β.
Cromolyn has been used clinically for several decades in other indications.
The upcoming trial, PHENOAD-002, will be a Phase III, randomised, double-blind, placebo-controlled study.
It aims to assess the safety and efficacy of inhaled PHENOGENE-1A in people with early Alzheimer’s. They will be stratified by their APOE4 genotype. Both APOE4 carriers and non-carriers will be included.
PhenoNet is basing this stratification on exploratory data from a previous Phase III trial, where a numerical difference was noted according to APOE4 status.
Patient screening is expected to begin in January 2027. The study will take place at approximately 90 sites across Europe and North America, aiming to recruit around 648 participants.
The primary endpoint will be measured over a period of 72 weeks.
The company also plans to seek a Special Protocol Assessment from the FDA to determine whether, if successful, the PHENOAD-002 trial could support a subsequent 505(b)(2) new drug application.
PhenoNet founder and chairman Dr David Elmaleh said: “Addressing the triggers of Alzheimer’s progression in diagnosed genotypes with a multifunctional therapeutic approach offers a new strategy to modify the disease course that could potentially benefit a large Alzheimer’s population.”
PhenoNet is focused on developing medicines for neurodegenerative diseases, including Alzheimer’s disease and amyotrophic lateral sclerosis.
