Aelis Farma, a clinical-stage biopharmaceutical company specializing in the development of treatments for brain diseases, has announced the enrollment of the first patient in its Phase 1/2 study with AEF0217 in participants with trisomy 21 (Down’s syndrome). ).
AEF0217 is Aelis Farma’s second drug candidate. It is part of a new class of drugs discovered by the company, specific inhibitors of CB1 receptor signaling of the endocannabinoid system (CB1-SSi), a receptor implicated in numerous brain diseases. AEF0217 is being developed as the first treatment for cognitive deficits caused by CB1 receptor hyperactivity, and as a first indication those associated with trisomy 21 (Down syndrome). AEF0217, like other CB1-SSi, is able to inhibit only certain components of CB1 activity, which seems to allow it to counter the pathological hyperactivity of the receptor without altering its normal physiological functions and, therefore, without inducing noticeable side effects.
Aelis Farma recently published the positive results of safety and pharmacokinetic studies in healthy volunteers (phase 1) with AEF0217, which confirmed the particularly favorable safety profile of CB1-SSi, a particularly important characteristic for fragile populations such as those carrying trisomy 21. Indeed, these studies showed that all doses of AEF0217 were well tolerated without any serious adverse effects being identified. In addition, AEF0217 showed a favorable pharmacokinetic profile. These positive results now make it possible to administer AEF0217 to people with trisomy 21.
This phase 1/2 with AEF0217 is a single-center, double-blind clinical study comparing one dose of AEF0217 to placebo, after 28 days of once-daily treatment, in approximately 45 participants with trisomy 21. Its main objective is to evaluate the safety and uptake of AEF0217 in people with Down syndrome and the study could potentially provide the first indications on the activity of AEF0217 as a treatment for the cognitive deficits of Down syndrome.
The recruitment of the first patient was carried out by the teams of Prof. Rafael de la Torre Fornell at the Medical Research Institute of Hospital del Mar (IMIM) in Barcelona (Spain). Depending on the rate of recruitment, the study could be finalized in the 2nd quarter of 2023.
Teacher. Rafael de la Torre Fornell, principal investigator of the study, declares: “The administration of AEF0217 in a first person with Down syndrome is an emotional moment for me and my teams. This is a further step towards the development of a treatment to improve the cognitive abilities of these endearing and unique people who desperately need it”.
Pier Vincenzo Piazza, Managing Director of Aelis Farma, concludes: “I would like to congratulate the teams of Prof. Rafael de la Torre Fornell and those of Aelis Farma for this important step forward in the development of AEF0217, a drug candidate based on our innovative approach of selective inhibition of part of the activity of the CB1 receptor. In the event of favorable results, we will be even closer to a promising therapy that could significantly improve the quality of life of people with Down syndrome, and even pave the way for the treatment of other cognitive deficits”.
About the clinical program of AEF0217 for the treatment of trisomy 21 cognitive impairment: the European ICOD project
The phase 1/2 study of AEF0217 is part of the European project H2020 ICOD (Improving COgnition in Down syndrome, Grant N° 899986), and is carried out in collaboration with the Hospital del Mar Institute of Medical Research (IMIM) of Barcelona (Spain) and Prof. Rafael de la Torre Fornell, project coordinator and principal investigator of the study. In February 2021, the ICOD project received funding of €6 million from the European Commission to finance the clinical development of AEF0217 for the treatment of cognitive deficits linked to trisomy 21.
Source and visual: Aelis Farma
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» Aelis Farma: recruitment of the 1st patient in the phase 1/2 study with AEF0217 in cognitive disorders of trisomy 21 MyPharma Editions
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