PhenoNet has received FDA "May Proceed" clearance to activate its IND and begin PHENOAD-002, a Phase III trial of inhaled PHENOGENE-1A, an aerosolised cromolyn formulation designed to reach the brain. The randomised, double-blind, placebo-controlled study will enrol about 648 participants across ~90 sites in Europe and North America and will stratify participants by APOE4 status. Screening is expected to begin in January 2027, and the primary endpoint will be assessed over 72 weeks. PhenoNet also intends to seek an SPA to determine whether the trial could support a 505(b)(2) filing.
FDA Clears PhenoNet To Launch Phase III Trial Of Inhaled PHENOGENE-1A In Early Alzheimer’s
PHENOGENE-1A uses PhenoNet’s technology to deliver therapeutic drug levels to the brain. Credit: superbeststock / Shutterstock.com.
PhenoNet announced that the US Food and Drug Administration (FDA) has issued a "May Proceed" notification, activating the company’s investigational new drug (IND) application for a Phase III study of inhaled PHENOGENE-1A in people with early Alzheimer’s disease.
PHENOGENE-1A is an inhaled formulation of cromolyn delivered using PhenoNet’s targeted delivery technology designed to achieve therapeutic levels in the brain. The candidate was engineered to act on three hypothesised pathways in Alzheimer’s disease: inhibition of amyloid-β aggregation, moderation of pro-inflammatory cytokines and chemokines, and promotion of microglial clearance of amyloid-β. Cromolyn itself has a long clinical history in other indications as a mast cell stabilizer.
PHENOAD-002: Trial Design And Goals
The upcoming PHENOAD-002 trial will be a Phase III, randomised, double-blind, placebo-controlled study evaluating the safety and efficacy of inhaled PHENOGENE-1A in people with early Alzheimer’s disease. Participants will be stratified by APOE4 genotype so that both APOE4 carriers and non-carriers are enrolled — an approach informed by exploratory findings from an earlier Phase III study that observed numerical differences by APOE4 status.
Screening is expected to begin in January 2027. The trial will be conducted at approximately 90 sites across Europe and North America and aims to recruit about 648 participants. The primary endpoint will be assessed over a 72-week period.
Regulatory Pathway And Company Focus
PhenoNet plans to request a Special Protocol Assessment (SPA) from the FDA to clarify whether a successful PHENOAD-002 could support a subsequent 505(b)(2) new drug application — a regulatory pathway that can allow reliance in part on existing data.
Dr David Elmaleh, PhenoNet Founder and Chairman, 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 therapies for neurodegenerative diseases, including Alzheimer’s disease and amyotrophic lateral sclerosis (ALS).
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