After Four Weeks, New Drug Shows Promising Results In Boosting Life Skills For Down Syndrome Patients
French biotech company Aelis Farma, specializing in the treatment of brain disorders announced positive results Monday from a Phase 1/2 trial of its drug candidate AEF0217 in young adults with Down syndrome. The study, conducted in Spain, assessed the safety, tolerability and potential efficacy of AEF0217, a selective CB1 receptor inhibitor targeting cognitive and behavioral impairments.
The study successfully met its safety (primary), pharmacokinetics (secondary) and efficacy (secondary and exploratory) objectives. AEF0217 was well tolerated and no safety concerns were observed. The drug improved adaptive behavior in areas such as communication, daily living skills and socialization.
“The promising and impressive results of this study generate a real hope to develop an efficacious and safe treatment for the cognitive disabilities of people with Down syndrome,” stated Prof. Rafael de la Torre Fornell, the trial’s principal investigator. “Particularly striking are the efficacy data, which addresses crucial domains of adaptation, such as expression and writing abilities as well as certain daily living skills and societal interactions. These types of effects, especially because obtained after only four weeks of treatment, are certainly a first in the field of …