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Apr 9, 2021

The European Commission grants €6 million to the ICOD project for the development of AEF0217 (AELIS FARMA) to treat cognitive deficits in Down syndrome

** • Selected as part of the Horizon 2020 EC program, ICOD supports the development of AEF0217 as the first pharmacological therapy of cognitive deficits in Down syndrome.
• AEF0217, developed by AELIS FARMA, represents a new pharmacological class of compounds with a novel mechanism of action that offers a unique opportunity to treat cognitive deficits. **

Bordeaux, France, April 9th, 2021 – AELIS FARMA, a biotechnology company specialized in the treatment of brain disorders, announced today that the ICOD project (Improving Cognition in Down Syndrome) has received a €6 million grant from the European Commission as part of the H2020 program. The ICOD project will finance the clinical development of AELIS FARMA’s AEF0217 for the treatment of cognitive deficits in Down syndrome.

Down syndrome (also known as trisomy 21) is a genetic condition affecting one million individuals in the EU and US. The prevalence of Down syndrome is on the rise because there has been an increase in both the average age of pregnant women (+50% from the early 1990s to 2015) and the life expectancy of individuals with Down syndrome (from 19 to 66 years old over the last 30 years). The cognitive disabilities associated with Down syndrome have no approved pharmacological treatment and impose a significant burden on the individuals, their families and the healthcare system.

The ICOD project, developed in collaboration with the Institut Hospital del Mar d'Investigacions Mèdiques (IMIM) in Barcelona and Prof. Rafael de la Torre, will involve five clinical centers in three countries: France, Spain and Italy1. The ICOD project will support the initial clinical development of AEF0217, from phase I studies in healthy volunteers (in 2021) to a phase II proof of concept study of clinical efficacy in individuals with Down syndrome (in 2023). The overarching goal of the project is to make AEF0217 available to the Down syndrome community 6 to 7 years from now.

AEF0217 provides a new approach to treat cognitive deficits. AEF0217 is a signaling specific inhibitor of the CB1 receptor (CB1-SSi), the primary receptor of the endocannabinoid system, responsible for the regulation of a large number of behavioral and cognitive processes. Recent studies2 have shown that cognitive disorders related to Down Syndrome involve hyperactivation of the CB1 receptor.

CB1-SSi are a new pharmacological class, discovered by Dr. Piazza, AELIS FARMA’s CEO, during his tenure as director of the Neurocenter Magendie of INSERM in Bordeaux. These innovative compounds selectively inhibit a pathological hyperactivation of the CB1 receptor without modifying its normal physiological activity. AEF0217 has received one of the most thorough evaluations of preclinical efficacy in the field of Down syndrome. Using a recently validated translational approach, these studies have shown that AEF0217 completely reverses cognitive deficits in adult trisomic animals. Because of its unique mechanism of action and preclinical efficacy AEF0217 has the potential to significantly improve the cognitive abilities of individuals with Down Syndrome, without producing significant side effects.

"We are very proud to have been awarded this grant and very pleased by the outstanding positive evaluation it has received. After the Seal of Excellence received last year, the award of the ICOD project provides a full validation by the European Commission of the innovative therapeutic approach developed by AELIS FARMA" said Pier Vincenzo Piazza, co-founder and CEO of AELIS FARMA. "Down syndrome, like many neuro-developmental disorders, is a real burden for the individuals and their families. At AELIS FARMA we are very excited to try and contribute to alleviate this suffering by increasing the autonomy of subjects with Down syndrome and their social insertion. “AEF0217 offers the promise of a high therapeutic potential conjugated with a very good tolerability, a crucial characteristic in vulnerable subjects with Down syndrome. We are very excited to have been awarded the ICOD project that will allow to accelerate the development of this innovative therapeutic approach” concludes Prof. Rafael de la Torre, Principal Investigator of the clinical studies and coordinator of the ICOD project.

About AELIS FARMA Founded in 2014, initially as a spin-off of INSERM (the French National Institute of Health and Medical Research), AELIS FARMA is a clinical-stage biotechnology company developing a new class of drugs, the Signaling Specific Inhibitors of the CB1 receptor (CB1-SSi), the major receptor of the endocannabinoid system. The discovery of these drugs by Pier Vincenzo Piazza, during his tenure as Director of the INSERM’s Neurocentre Magendie in Bordeaux, has been awarded the Grand Prix of Inserm and of the French National Academy of Science.

CB1-SSi have a strong potential for the treatment of numerous brain diseases. The Company has developed two first-in-class drug candidates, AEF0117 and AEF0217, and has a pipeline of new innovative CB1-SSi for other diseases associated with CB1 receptor anomalies.

AEF0117 targets disorders associated with cannabis use (addiction and psychosis) and is scheduled to enter a Phase IIb clinical trial in the US in 2021.

AEF0217, which targets cognitive impairment such as those observed in Down syndrome, has successfully completed preclinical development, and will enter Phase I in Europe in 2021.

Based in Bordeaux, at the INSERM’s Neurocentre Magendie, AELIS FARMA has 12 members of staff and counts among its investors the Nouvelle-Aquitaine Region, Inserm Transfert Initiative, Bpifrance, ACI and NACO regional funds and IRDI SORIDEC Gestion. Its research programs have received grants from the Deeptech program of Bpifrance, from the FEDER and H2020 programs of the EU and from the National Institute on Drug Abuse (NIH-NIDA) in the US.

1 Clinical center involved in the ICOD project: Institut Hospital del Mar d'Investigacions Mèdiques (IMIM), Barcelona (Prof. R. de la Torre, PI and coordinator of the project) and Hospital Universitario de La Princesa, Madrid (Dr. F. Moldenhauer) in Spain; Research Institute for Mental Retardation and Brain Aging, Oasi Maria SS. Onlus, Troina (Prof. F. Caraci) in Italy; University Hospital of Saint-Etienne (CHUSE) (Dr. R. Touraine) and the Jérôme Lejeune Institute (Dr. S. Durand) in France.
2 Navarro-Romero et al. Cannabinoid type-1 receptor blockade restores neurological phenotypes in two models for Down syndrome. Neurobiol Dis. 2019; 125:92-106.