BORDEAUX, France – Aelis Farma (Euronext Paris: AELIS) has officially commenced recruitment for its Phase 2B clinical trial evaluating AEF0217 in patients diagnosed with Down syndrome. This initiative spans France, Italy, and Spain, as detailed in a recent press release.
With a market capitalisation of $19.33 million, Aelis Farma has experienced a year-to-date stock increase of 9.33%, although it currently trades 52% below its 52-week high.
The clinical trial aims to recruit 188 participants aged between 16 and 32 years across 10 specialised centres. The first patient visit was recorded in December 2025, following the initial regulatory approval via the EU Clinical Trials Information System.
AEF0217 is characterised as a signalling-specific inhibitor of the CB1 receptor. Notably, the company has indicated that there are currently no approved pharmacological treatments available that address adaptive behaviour and cognitive impairments associated with Down syndrome.
This randomised, double-blind, placebo-controlled study will allocate participants to receive once-daily oral AEF0217 at doses of 0.1 mg, 0.2 mg, or 0.6 mg, or a placebo over a period of 24 weeks, followed by an eight-week treatment-free phase.
The primary endpoint of the trial is to assess changes in adaptive behaviours using the Vineland Adaptive Behaviour Scales. Secondary endpoints encompass cognition, quality of life, and sleep efficiency. An independent data monitoring committee will conduct an interim safety analysis after 40 participants have completed 12 weeks of treatment.
In a preceding Phase 1/2 study involving 29 participants aged 18 to 32 years, Aelis Farma reported that AEF0217 exhibited a favourable safety profile and resulted in statistically significant improvements in adaptive behaviours as measured by the Vineland scales.
Recruitment for this trial is anticipated to conclude by the end of 2026, with preliminary results expected to be available in the second half of 2027. Aelis Farma has disclosed that its financial runway extends to the first quarter of 2028. Analysis from InvestingPro indicates that Aelis Farma possesses more cash than debt on its balance sheet, maintaining a current ratio of 3.18, although the company is rapidly utilising its cash reserves.
The trial has received the necessary approvals from the European Medical Agency and the relevant national competent authorities in the three participating countries.
For more information regarding funding opportunities in the realm of clinical trials and research, please refer to pertinent grants and funding bodies that support innovative health initiatives.