Aelis Farma has announced positive results from its safety trials in healthy volunteers and the authorization to start the first trial in people with Down syndrome, with AEF0217, its drug candidate for the treatment of cognitive disorders.\n\n\n\nAelis Farma is a clinical stage biopharmaceutical company specializing in the development of treatments for brain diseases, made the announcement today (November 15). AEF0217 is being developed for the treatment of cognitive deficits caused by a hyperactivity of the CB1 receptor, and as a first indication those associated with Trisomy 21 (Down syndrome).\n\n\n\nAcute and chronic administration of AEF0217, up to 30 times the anticipated therapeutic range, is well tolerated and presents favorable safety and plasma exposure profiles, which allow the next stage of clinical development. AEF0217 is the second drug candidate of the new class of drugs developed by Aelis Farma: the Signaling Specific inhibitors of the CB1 receptor (CB1-SSi).\n\n\n\nNew class of drugs developed by Aelis Farma\n\n\n\nThe CB1 is the main receptor of the brain’s endocannabinoid system that is responsible for regulating several physiological and cognitive processes. Recent research has suggested that cognitive disorders linked to Trisomy 21 involve a hyperactivity of the CB1 receptor.\n\n\n\nAEF0217 is positioned as the first treatment for cognitive deficits caused by hyperactivity of the CB1 receptor. It aims to capitalize on the distinctive characteristics of CB1-SSi which allow to inhibit a hyperactivity of the CB1 receptor without affecting normal physiological functions and without causing significant side effects, two criteria that are particularly important for vulnerable populations such as those with Down syndrome.\n\n\n\nPier Vincenzo Piazza, CEO of Aelis Farma, commented: “The very positive results of the phase 1 program achieved with AEF0217 constitute a significant step in the development of this drug candidate to treat cognitive deficits and in particular those linked to Trisomy 21, also known as Down syndrome. They also confirm the strong potential of the therapeutic class developed by Aelis Farma, the CB1-SSi, showing again an excellent safety profile as well as favorable pharmacokinetic characteristics.\n\n\n\nNo serious adverse reactions \n\n\n\n“AEF0217 can now move forward and enter a phase 1/2 trial in people with Trisomy 21. This significant progress enables us to swiftly move towards the confirmation of AEF0217’s therapeutic potential to improve the major pharmacological innovation for brain diseases 2 / 4 cognitive deficits of people with Trisomy 21, for which there are no effective therapeutic solutions today.”\n\n\n\nThe phase 1 clinical program of AEF0217 combines three clinical trials authorized by the Spanish health authority, the AEMPS, in September 2021: a single ascending doses trial, a multiple (seven days) ascending doses trial, and a pharmacokinetic trial analyzing the impact of food consumption on drug absorption.\n\n\n\nThis overall clinical program was designed to assess the safety, tolerability and absorption of AEF0217, in the range between 0.2 mg and 6 mg, in comparison with a placebo, in 68 healthy volunteers, aged between 18 and 55 years old. The results obtained have shown that all the doses of AEF0217 were well tolerated by healthy volunteers with no serious adverse reaction. Only three adverse events related to AEF0217, manifesting as low intensity diarrhea, were observed during the full program.\n\n\n\nAdvancement authorized for Aelis Farma \n\n\n\nIn addition, no relevant clinical changes in routine biological tests, electrocardiograms or vital signs were observed. Similarly, psychometric tests assessing main psychological dimensions, such as depression, anxiety, psychosis and suicidal tendencies did not show significant difference between AEF0217 and placebo. Aelis Farma added that the pharmacokinetic profile of AEF0117 has also proven favorable and, as expected, is characterized by very good plasma absorption, linearity between doses and a long half-life.\n\n\n\n“These positive results for AEF0217 are all the more encouraging as the safety of the compound is a particularly important criterion for the treatment of the Down syndrome population and for acceptance by the families of a treatment for cognitive deficits for these vulnerable people. We are delighted to be part of this revolutionary and exciting project to assess this drug candidate, which represents real hope for many people affected by Down syndrome and their families,” added Rafael de la Torre Fornell, principal investigator of the clinical trials and coordinator of the ICOD project.\n\n\n\nIt is thanks to these positive data that the AEMPS has authorized the advancement of Aelis Farma’s AEF0217 to a first clinical trial in participants with Down syndrome. The main objective of this phase 1/2 study will be to demonstrate the safety, tolerability and the pharmacokinetic profile of AEF0217 in people with Down syndrome. The trial could also provide initial evidence of the activity of this drug candidate. The first enrollments are expected before the end of 2022 and the first clinical results should be available in mid-2023.