Dr. Pier Vincenzo Piazza,
Co-founder and CEO of Aelis Farma
The year 2025 was marked by further progress in the clinical development of our two drug candidates, AEF0117 and AEF0217.
For AEF0117, the final results of the Phase 2b study in cannabis use disorder (CUD) demonstrated that AEF0117 is capable of statistically significantly reducing cannabis use in patients with a strong motivation to quit and confirmed the excellent safety and tolerability of AEF0117.
Regarding AEF0217, developed for the treatment of behavioral and cognitive impairments, the positive results in terms of safety, tolerability, and efficacy obtained in a Phase 1/2 study in people with Down syndrome enabled us to obtain regulatory authorizations and start the recruitment of an international multicenter Phase 2b study in France, Spain, and Italy.
The first participant first visit took place in December 2025. This double-blind, placebo-controlled study will recruit 188 participants with Down syndrome, aged 16 to 32, who will be treated with either placebo or one of three doses of AEF0217.
The study aims to confirm the positive effects of AEF0217 on adaptive behaviors and the favorable safety profile observed in the Phase 1/2 trial.
Finally, our proprietary research platform has enabled us to select new CB1-SSi with novel functional properties for a wider range of diseases involving the CB1 receptor.
For the year 2026, our main objective is to continue the Phase 2b clinical study with AEF0217 and initial preclinical proof-of-concept studies of our new compounds, all of which are already fully funded.
We also aim to develop industrial partnerships in order to further the development of AEF0117 and to fully exploit AEF0217 numerous therapeutic indications, which extend well beyond the cognitive deficits associated with Down syndrome.
Backed by the skills of our teams, we are confident of achieving these objectives, and of becoming a leading player in the development of innovative treatments for cerebral and peripheral diseases.