T21RS Webinar – March 5, 2026

T21RS is pleased to invite you to the free Webinar hosted by the Education and Training Committee.

Prof. Rafael De La Torre, Integrative Pharmacology and Systems Neuroscience Research Group, Hospital del Mar Medical Research Institute, Barcelona, Spain

Prof. Filippo Caraci, Department of Drug and Health Sciences, University of Catania, Catania; Oasi Research Institute-IRCCS, Unit of Neuropharmacology and Translational Neurosciences, Troina, Italy

will present the topic:

“The European ICOD project: preliminary evidence of AEF0217 efficacy on adaptive functionality and cognition from Phase I/II studies in adults with Down syndrome”

DS causes lifelong cognitive dysfunction, which results in important negative consequences for these individuals, their families and society. Despite the heavier social burden and the greater medical needs linked to this intellectual disability, there isn’t an approved therapy for DS-related cognitive dysfunction yet. The final goal of the ICOD project (Improving COgnition in Down syndrome), funded in the framework of the H2020 research program, is to make available an innovative first-in-class drug for DS individuals 7 years after its clinical development, offering people with DS and their families a novel approach for the treatment of DS-related functional and cognitive impairments.

The ICOD project is accelerating the clinical development of the molecule AEF0217. This first-in-class new drug belongs to a new pharmacological class the “signaling specific inhibitors of the CB1 receptor (CB1-SSi)” developed by the biotech Aelis Farma. AEF0217 targets the CB1 receptor, whose hyperactivity has been linked to cognitive deficits in DS. AEF0217 can reverse cognitive impairments in surrogate mouse models using an innovative translational medicine approach.

The safety and tolerability of AEF0217 were assessed by the ICOD project in Phase I clinical trials including healthy neurotypical subjects. The molecule is quite safe and well tolerated, according to the results. Additional research in young people with DS (Phase I/II trial) has shown first clinical evidence of AEF0217’s effectiveness, particularly in the domains of cognition and adaptive functionality. The trial’s findings will be examined and presented.

Phase II safety and efficacy studies in a multicenter international clinical study involving ten clinical sites and three countries (Spain, France, and Italy) are about to begin. Results are anticipated for the third quarter of 2027.

Date: Thursday, March 5, 2026

Time: 6:00 PM Central Europe Time (12 PM USA Eastern Time)

 To attend, please preregister [HERE].

We look forward to you joining our Webinar!