DeFloria Reports Positive Phase 1 Trial Results for Autism Treatment AJA001

DeFloria, a collaborative effort between Ajna BioSciences and Charlotte's Web, announced promising outcomes from a Phase 1 clinical trial of AJA001, a botanical drug aimed at alleviating symptoms of autism spectrum disorder (ASD). The findings were disclosed through a poster presentation at the 63rd Annual Meeting of the American College of Neuropsychopharmacology in Phoenix, Arizona.

AJA001 is an orally administered drug comprising multiple cannabinoids, designed to offer a broad therapeutic impact. The trial involved a single ascending and multiple ascending dose study which evaluated the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of AJA001. Marcel O. Bonn-Miller, Ph.D., Chief Scientific Officer at Charlotte's Web and a Board Member at DeFloria, unveiled the results of this trial that included 70 healthy participants aged between 19 and 55 years.

The study demonstrated that AJA001 was well-tolerated at doses reaching 680 mg per day as a single dose and 660 mg per day when administered twice daily. Both the single and multiple dosage phases showed dose-proportional PK for cannabidiol (CBD) and tetrahydrocannabinol (THC), with plasma concentrations comparable to or surpassing those of existing cannabinoid treatments such as dronabinol, nabiximols, and Epidiolex®. Minimal accumulation of CBD or THC was observed after seven days of multiple dose administration.

The PD effects of AJA001 were dose-dependent, correlating with the peak plasma concentrations of THC and its active metabolite 11-OH-THC. Participant feedback on the Drug Effects Questionnaire (DEQ) indicated that AJA001’s subjective effects were moderate and short-lived. The results support the use of 100 mg to 660 mg of AJA001 per day in future Phase 2 trials, which will target children, adolescents, and adults with ASD. This dosage range equals a maximum of 395 mg of CBD and 15 mg of THC daily. Common adverse effects reported included somnolence, anxiety, dizziness, and headache. Notably, one serious adverse event involving anxiety was resolved by the following day without further issues.

Dr. Bonn-Miller expressed optimism about these findings, highlighting the favorable tolerability and pharmacokinetic profile of AJA001 across various doses. The data from this trial will guide the dosage levels to be explored in upcoming Phase 2 trials scheduled for the second quarter of 2025. Jared Stanley, CEO of DeFloria, emphasized the challenges in treating ASD and the need for better-tolerated therapies, affirming DeFloria's commitment to addressing the behavioral symptoms associated with ASD through the development of AJA001.

ASD affects approximately 1 in 36 children and is characterized by deficits in social communication, impaired social interactions, and repetitive behaviors or interests. These symptoms often lead to significant caregiver stress. AJA001, with its full-spectrum cannabinoid formulation derived from Cannabis sativa L, aims to offer broad therapeutic benefits, potentially applicable to various medical conditions.

DeFloria, formed through a partnership between Charlotte's Web, AJNA BioSciences, and British American Tobacco, seeks to innovate in healthcare by developing multi-compound drugs sourced from nature. The company is working towards securing FDA approval for this new botanical drug, targeting neurological conditions as identified by their research team.