Aelis Farma announced the presentation of new preclinical data on its drug candidate AEF0217 at the 2nd European Conference on Phelan-McDermid Syndrome, which was held from June 9 to 11, 2023 at CEU San Pablo University in Madrid. Phelan-McD Germid Syndrome (PMS), which is caused by the deletion of chromosome 22q13 including the SHANK3 gene or by a sequence variation in this gene, is one of the most frequently observed genetic mutations in autism. It is an orphan disease for which there is currently no treatment.

In affected people, these mutations can lead to development delays in multiple areas, in particular delayed speech, intellectual disability and often autism spectrum disorder. Dr. Pier Vincenzo Piazza, CEO of Aelis Farma, alongside Flavio Tomasi, PhD student in Dr. Catalina Betancur's INSERM/CNRS laboratory at the Sorbonne university, gave an oral communication entitled: “Inhibition of the cannabinoid CB1 receptor rescues deficits in a mouse model of Phelan-McDermid syndrome” at the 2ndEuropean Conference on Phelan-McDermid Syndrome, organized by the Spanish Phelan-McDermid Syndrome Association. The presented data was obtained within the context of a collaboration between several laboratories coordinated by Dr. Betancur.

The results showed the ability of AEF0217 to statistically significantly reverse behavioral, cognitive and motor deficits observed in a genetic mouse model of Phelan-McDermid Syndrome. In these mice, ARF0217 also reversed a neurological alteration (cortical hyperactivity), considered as a neurobiological marker of autism. Based on these promising results Aelis will now analyze the feasibility to develop of AEF0217 in this indication and more generally in autism spectrum disorder.

AEF0217 is the second drug candidate developed by Aelis Farma. It belongs to a new generation of drugs discovered by the Company, the Signaling-Specific inhibitors of the CB1 receptor of the endocannabinoid system (CB1-SSi). The CB1 is one of the most expressed neurotransmitter receptors in the brain implicated in many diseases.

AEF0217 is currently being developed for the treatment of cognitive disorders in people with Down syndrome (trisomy 21) and evaluated in a phase 1/2 study in this population.