The Independent Data Monitoring Committee (IDMC) identified no safety concern and recommended that the current Phase 2b continue according to the current protocol.
The findings further support the favorable safety and tolerability profile previously observed with AEF0217 in the previous Phase 1/2 study in people with Down syndrome.
Recruitment is progressing as planned, with 65 participants randomized and 5 currently being randomized out of the 188 participants planned.
BORDEAUX, France, July 16, 2026--(BUSINESS WIRE)--Regulatory News:
Aelis Farma (ISIN: FR0014007ZB4 – Ticker: AELIS), a clinical-stage biopharmaceutical company specializing in the development of treatments for brain and peripheral diseases involving the CB1 receptor, announces that the Independent Data Monitoring Committee ("IDMC") has recommended the continuation, without modification, of the ongoing Phase 2b clinical trial evaluating AEF0217 in adults and older adolescents with Down syndrome.
The IDMC is an independent committee composed of experts in clinical pharmacology, clinical research, pediatrics and Down syndrome. Its role is to periodically review the safety and tolerability data generated during the study and provide independent recommendations regarding its continuation.
The committee conducted the first scheduled safety review of the Phase 2b study after more than 30 participants had completed at least four weeks of treatment, in accordance with the study's IDMC Charter. The review was based on blind safety data from 42 randomized participants, including 32 participants who had completed at least four weeks of treatment as of the June 4, 2026 data cut-off.
The review showed that no severe or serious adverse events were reported, that all reported adverse events were mild and that no new safety concern was identified. In addition, none of the adverse events reported were considered by the investigators to be related to the investigational treatment. Finally, no participant discontinued treatment or withdrew from the study following randomization because of an adverse event.
Following its review, the IDMC recommended that the trial proceed according to the current version of the protocol, without modifications.
Safety findings consistent with the previous Phase 1/2 study in Down syndrome
The findings from this first Phase 2b safety review are consistent with and further support the favourable safety and tolerability profile previously observed with AEF0217.
In the previous randomized, double-blind, placebo-controlled Phase 1/2 study, 29 young adults with Down syndrome received either AEF0217 at 0.2 mg once daily or placebo for four weeks, according to a 2:1 randomization ratio. Safety and tolerability were the primary objectives of the study.