Aelis Farma S.A. announced the recruitment of the first patient for its phase 1/2 trial of AEF0217 in participants with Down syndrome. AEF0217 is Aelis Farma's second drug candidate. It belongs to a new class of drugs discovered by the company, signaling-specific inhibitors of the CB1 receptor of the endocannabinoid system a receptor implicated in many brain disorders.

AEF0217 is being developed as the first treatment of cognitive impairments caused by a hyperactivity of the CB1 receptor and, as a first indication, of those associated with Down syndrome. AEF0217, like other CB1-SSis, is able to inhibit only certain components of CB1's activity, which seems to enable this compound to counteract a pathological hyperactivity of the receptor without altering its normal physiological functions and, as a consequence, without causing major side effects. Aelis Farma recently published the positive results of the phase 1 clinical trials analyzing the safety and pharmacokinetics of AEF0217 in healthy volunteers.

These studies have confirmed the particularly favorable safety profile of CB1-SSi, a particularly important characteristic for fragile populations such as those with Down syndrome. The results of these trials showed that all doses of AEF0217 were well tolerated without any serious adverse effects and that AEF0217 has a favorable pharmacokinetic profile. These positive results allow now to administer AEF0217 to people with Down syndrome.

The phase 1/2 clinical trial with AEF0217 is a monocentric, double-blind study comparing one dose of AEF0217 to placebo, after 28 days of treatment once a day in approximately 45 participants with Down syndrome. The main objective of the trial is to assess the safety and absorption of AEF0217 and it could also provide the first indications of activity as a treatment for the cognitive impairments of Down syndrome. The first patient has been recruited by the teams of Prof. Rafael de la Torre Fornell at the Hospital del Mar Medical Research Institute (IMIM) in Barcelona (Spain).

Depending on the rate of recruitment, the study should be completed in the second quarter of 2023.