Aelis Farma: AEF0217 favored in 16 of 19 subdomains in daily life, according to parents
Aelis Farma published on November 6, 2026 the results of a retrospective observational study, conducted via an online questionnaire among parents and caregivers of participants in its previous Phase 1/2 trial with AEF0217.
The objective was to verify whether the improvements in adaptive behaviors measured at the time by the Vineland scale (VABS-2) translate into noticeable changes in the daily lives of people with Down syndrome.
Sixteen of nineteen subdomains in favor of AEF0217 according to caregivers
Of the 19 subdomains assessed (daily life, autonomy, sociability, communication), 16 show a difference in favor of AEF0217, i.e. 84%, and none in favor of the placebo. The magnitude of the differences, measured by the absolute standardized mean difference (ASMD), is described as large in five cases, notable in six and relevant in five.
The highest value (ASMD of 1.00) concerns the frequency of writing and the use of grammar and spelling. All five skills in the sociability domain favor the treatment, including adaptation to new situations (0.92) and development of interpersonal relationships (0.83).
Regarding overall perception, 37.5% of caregivers in the treated group consider that the effects meet their expectations, compared with 10% in the placebo group.
Furthermore, 27% of caregivers in the AEF0217 group perceived lasting effects well beyond the end of treatment, whereas all caregivers in the placebo group describe effects that decline rapidly after discontinuation.
A sample of 22 caregivers, ahead of a Phase 2b trial with 188 participants
According to the company, this study “was not intended to independently demonstrate the efficacy of AEF0217”. Of the 26 caregivers who agreed to participate, 22 provided fully analyzable responses (14 in the treated group, 8 in the placebo group), for a 28-day treatment at a dose of 0.2 mg during the Phase 1/2.
Given the small size of the sample, the differences were analyzed using the ASMD, presented by the company as the most appropriate approach. In communication, the smaller differences are, according to the company, due to improvements also reported in the placebo group, an area where the placebo effect could be most significant.
The next stage of development relies on an international, randomized, double-blind, placebo-controlled Phase 2b trial, which plans to recruit 188 participants aged 16 to 32 in France, Spain and Italy. They will receive one of three doses (0.1, 0.2 or 0.6 mg) or a placebo for six months.
In July 2026, an independent data monitoring committee identified no safety issues and recommended continuing the study without modification of the protocol. For Pier Vincenzo Piazza, chief executive officer, these observations constitute “an important milestone”; the trial is progressing according to the planned timeline, with results expected by the end of 2027.
