After Four Weeks, New Drug Shows Promising Results In Boosting Life Skills For Down Syndrome Patients

French biotech company Aelis Farma, specializing in the treatment of brain disorders announced positive results Monday from a Phase 1/2 trial of its drug candidate AEF0217 in young adults with Down syndrome. The study, conducted in Spain, assessed the safety, tolerability and potential efficacy of AEF0217, a selective CB1 receptor inhibitor targeting cognitive and behavioral impairments.

The study successfully met its safety (primary), pharmacokinetics (secondary) and efficacy (secondary and exploratory) objectives. AEF0217 was well tolerated and no safety concerns were observed. The drug improved adaptive behavior in areas such as communication, daily living skills and socialization.

"The promising and impressive results of this study generate a real hope to develop an efficacious and safe treatment for the cognitive disabilities of people with Down syndrome," stated Prof. Rafael de la Torre Fornell, the trial's principal investigator. "Particularly striking are the efficacy data, which addresses crucial domains of adaptation, such as expression and writing abilities as well as certain daily living skills and societal interactions. These types of effects, especially because obtained after only four weeks of treatment, are certainly a first in the field of Down syndrome and represent a major step towards the development of a treatment that could significantly improve the autonomy and adaptation of people with Down syndrome. I would like to take this occasion to also send my warmest thanks to the participants, their families and family associations who supported this innovative project and helped us to complete this pioneering clinical trial."

It is noteworthy that AEF0217 seemed to induce larger improvements in participants with the greater impairments before starting the treatment.

Moreover, adaptive behavior and cognition were assessed after just four weeks of treatment, whereas experts generally believe several months are needed to modify these functions in neurodevelopmental disabilities.

Read Also: Can THC Turn Back The Clock? Cannabis Reverses Brain Aging, Boosts Mental Capacity, New Study Suggests

Get Benzinga's exclusive analysis and the top news about the cannabis industry and markets daily in your inbox for free. Subscribe to our newsletter here. If you're serious about the business, you can't afford to miss out.

Paving The Way For Treating Other Cognitive Disorders

Pier Vincenzo Piazza, CEO of Aelis Farma, concluded, "We see this accomplishment as a milestone in the development of a promising therapy that could significantly improve the autonomy and quality of life of people with Down syndrome. It is also, more generally, the first evidence of the positive impact of AEF0217 on cognition, which could pave the way for the treatment of other cognitive disorders."

Piazza also highlighted that these results offer a supplementary validation of the safety of the new pharmacological class developed by the company – Signaling Specific inhibitors of the CB₁ receptor (CB₁-Ssi). "We believe that the CB₁-SSi represent a major innovation in pharmacology that will allow to generate inhibitors not only efficacious but also safe and well tolerated, opening the way to new treatments of conditions without treatment today," he concluded.

Following these results, Aelis Farma plans a Phase 2 international study in 2025 to further evaluate its efficacy and adaptative behavior improvements.

The clinical development of AEF0217, the phase 1 program in healthy volunteers, the phase 1/2 just completed and the next phase 2 in people with Down syndrome are part of the European H2020 ICOD project (Improving COgnition in Down syndrome, Grant Agreement ID 899986). The ICOD project received €6 million ($6.33 million) of fundings from the European Commission and involves partners in Spain, France and Italy.

Read Next: French Biotech Candidate For Cannabis Use Disorder Fails Phase 2B Clinical Trial, Indivior Will Not Exercise Option

Photo generated with AI