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 - Seite 2

Dr. Catalina Betancur of the Neuroscience Paris Seine laboratory (INSERM, CNRS, Sorbonne University, Paris), who coordinated the studies presented, commented: “We are delighted to have had the opportunity to present the results of the effects of AEF0217 on a mouse model of Phelan-McDermid Syndrome at this conference, which brings together the most prominent international experts in this rare disease for which there is no effective treatment. Our preclinical data have been very favorably received by scientists and families attending the conference and encourage us to push forward the evaluation of AEF0217 in autism spectrum disorder. We are delighted to be participating in the development of a potential new treatment for a population of patients in dire need.”

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.

About Phelan-McDermid Syndrome²

Phelan-McDermid Syndrome (PMS) is one of the most frequent genetic causes of autism spectrum disorder. It is an orphan disease resulting from the loss of genetic material at the terminal end of chromosome 22 (22q13 deletion) or from a mutation in the SHANK3 gene. The genetic characteristic that affected people have in common is the absence or the mutation of the SHANK3 gene. The absence or the mutation of a copy of this gene results in a general developmental delay, and in particular in a delayed or absent speech, intellectual disability, autism spectrum disorder and motor skill deficits. Some affected people may also suffer from epilepsy. This mutation generally occurs spontaneously and it is not hereditary.

About the European Conference on Phelan-McDermid Syndrome

This conference, organized by the Spanish Phelan-McDermid Syndrome Association, aims to inform professionals and families about the latest advances in the scientific research and care of Phelan-McDermid Syndrome (PMS). It brings together international experts in various disciplines and provides the families of people with this syndrome an opportunity to discuss the difficulties and challenges they face.