Aelis Farma's Drug Candidate For the Treatment Of Cognitive Disorders Authorized For The First Trial In People With Down Syndrome

Aelis Farma AELIS, a clinical stage biopharmaceutical company specializing in the development of treatments for brain diseases, announced positive results from its phase 1 clinical trials in healthy volunteers with its drug candidate AEF0217. 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). Acute 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). The 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. AEF0217 is positioned as the first treatment for cognitive deficits caused by hyperactivity of the CB₁ receptor. AEF0217 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.

The 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 (7 days) ascending doses trial, and a pharmacokinetic trial analyzing the impact of food consumption on drug absorption. This 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 3 adverse events related to AEF0217, manifesting as low intensity diarrhea, were observed during the full program. In 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. 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.

It is thanks to these positive data that the AEMPS has authorized the advancement of 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.

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