Nature Medicine Publishes Discovery of New Pharmacological Class by Aelis Farma, Phase 2a Study of First Drug Candidate for Cannabis Use Disorder - Seite 2

Because of its unique mechanism of action, AEF0117 significantly reduced phase 2a study participants’ self-reported ratings of the positive subjective effects of cannabis, the primary outcome measure, by a mean of 38% (p<0.04), while also reducing cannabis use as measured by self-administration (p<0.05), the key secondary endpoint. AEF0117 produced no treatment-related serious adverse events or treatment-emergent adverse events distinct from placebo. These reductions in cannabis effects occurred without precipitating cannabis withdrawal, even for volunteers who smoked several grams of cannabis per day.

“No other medication has been shown to safely reduce the direct effects of smoked cannabis in daily cannabis smokers,” said Margaret (Meg) Haney, PhD, supervisor of the phase 1 studies and principal investigator of the 2a proof-of-concept study, and Professor of Neurobiology in the Department of Psychiatry at Columbia University Irving Medical Center, where she is the Director of the Cannabis Research Laboratory and Co-Director of the Substance Use Research Center. “These novel findings clearly suggest that AEF0117 may be an effective approach for patients seeking treatment for CUD.”

Aelis Farma thanks the volunteer study participants, Dr. Haney and her team at Columbia, the National Institute of Drug Abuse, and all the participating study centers and staff for their assistance.

About Cannabis Use Disorder
Cannabis use is widespread and often leads to CUD, the current definition of problematic cannabis use, which encompasses addiction.² CUD affects about 14.2 million individuals in the U.S,³ and its prevalence is increasing worldwide.⁴ Currently, CUD is treated using evidence-based behavioral treatments, which often have poor adherence and limited success.⁵ No medications are approved by the FDA to treat CUD.

About AEF0117
AEF0117 is the first drug candidate of the new pharmacological class of CB₁-SSi, a rationally designed analog of pregnenolone, the naturally occurring steroid hormone that binds to a specific site on the CB₁ receptor. Pregnenolone and AEF0117 do not modify the binding of agonists to the CB₁ but only block certain signaling pathways activated by cannabinoid agonists, such as THC, on the CB₁. Through this selective mechanism of action, AEF0117 potently inhibits THC’s behavioral effects without altering normal behavior. Unlike pregnenolone, AEF0117, is highly bioavailable when taken orally, exhibits favorable pharmacokinetic properties for once-daily administration, and is not converted into other steroids. AEF0117 has a 13,000-fold therapeutic index (i.e., the ratio between the toxic and active dose).