Preclinical Data Demonstrating the Efficacy of AEF0217 in a Genetic Mouse Model of Autism Spectrum Disorder Has Been Presented at the 2nd European Conference on Phelan-McDermid Syndrome

Regulatory News:

Aelis Farma (ISIN: FR0014007ZB4 – Ticker: AELIS), a clinical-stage biopharmaceutical company specialized in the development of treatments for brain diseases, today announced the presentation of new preclinical data on its drug candidate AEF0217 at the 2^nd European Conference on Phelan-McDermid Syndrome, which was held from June 9 to 11, 2023 at CEU San Pablo University in Madrid¹.

Phelan-McDermid 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 2^nd European 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.

Pier Vincenzo Piazza, CEO of Aelis Farma, said: “The preclinical data obtained by Dr. Betancur’s group have generated considerable excitement in the scientific community present at the meeting and encourage us to evaluate, in addition to the ongoing program in trisomy 21, the feasibility of developing AEF0217 in this new indication. More generally, the data suggest that AEF0217 could also help patients with autism spectrum disorder, which would significantly expand the fields of application of our second drug candidate.”