AEF0217

AEF0217 is a first-in-class small molecule drug developed by Aelis Farma for the treatment of cognitive impairments, with an initial focus on behavioral and cognitive deficits associated with Down syndrome. It belongs to a novel pharmacological class called Signaling Specific inhibitors of the CB1 receptor (CB1-SSi). Unlike traditional CB1 antagonists, which block all activity at the cannabinoid receptor type 1 (CB1), CB1-SSi like AEF0217 selectively inhibit only certain pathological signaling pathways of the CB1 receptor while sparing its normal physiological functions. This selectivity aims to provide therapeutic benefits without the side effects typically seen with earlier generation CB1 antagonists. Preclinical and clinical studies have shown that hyperactivity of the CB1 receptor is implicated in cognitive disorders linked to Down syndrome; by modulating this hyperactivity, AEF0217 has demonstrated improvements in communication, daily living skills, social interactions, and cognitive flexibility in young adults with Down syndrome. The drug has shown favorable safety and pharmacokinetic profiles in both healthy volunteers and individuals with Down syndrome[3][5][9][10].

Other names

1-((3s,8s,9s,10r,13s,14s,17s)3-benzyloxy-10,13,17-trimethyl-2,3,4,7,8,9,10,11,12,13,14,15,16,17-tetradecahydro-1h-cyclopenta[a]phenanthrene-17-yl)ethan-1-one3-benzyloxypregnenolone3-benzyloxy-pregn-5-en-20-one