AEF0217 AEF0217 has successfully completed the Phase 1 clinical program and will enter Phase 1/2 study in subjects with Down syndrome before end of 2022 in Europe.
AEF0217 is the second drug candidate developed by Aelis Farma. It is specifically designed for the treatment of cognitive deficits.
AEF0217's major characteristics:
High potency in reversing cognitive deficits in validated translational models of Down syndrome, Fragile X syndrome and aging related cognitive impairments without identifiable behavioral side effects even in fragile trisomic and aged mice.
Very favorable ADMET profile, with good oral absorption, brain access, long half-life and a good tolerability (therapeutic index = 640).
Good pharmacokinetic, safety and efficacy profile in healthy volunteers
Clinical development of AEF0217, for which its first therapeutic indication is the cognitive deficits in Down syndrome, is supported by a grant from the EU (Horizon Programme H2020; Improving Cognition in Down syndrome, Grant N° 899986).