DeFloria, a joint venture between Ajna BioSciences and Charlotte's Web, has announced positive results from its Phase 1 trial of AJA001, a novel multi-cannabinoid botanical drug product (BDP) being developed for the treatment of Autism Spectrum Disorder (ASD). The single ascending and multiple ascending dose trial demonstrated that AJA001 was safe and well-tolerated, with a formulation optimized for individuals with ASD. The findings were presented at the 63rd Annual Meeting of the American College of Neuropsychopharmacology. Given the limitations of current ASD treatments and the high prevalence of the disorder—affecting approximately 1 in 36 children, according to the CDC—AJA001 represents a potentially significant advancement.
Phase 1 Trial Details
The Phase 1 randomized, double-blind, placebo-controlled trial evaluated the tolerability and pharmacokinetic (PK) / pharmacodynamic (PD) profile of AJA001 in 70 healthy volunteers aged 19 to 55 years. Participants received single doses up to 680 mg or multiple doses up to 660 mg twice daily. The primary endpoint was safety and tolerability, while secondary endpoints included PK and PD assessments.
Key Findings
The results indicated that AJA001 was well-tolerated across the tested dose range. Pharmacokinetic analysis revealed dose-proportional PK for CBD and THC, with plasma concentrations matching or exceeding those of comparable cannabinoid therapeutics like dronabinol, nabiximols, and Epidiolex. Minimal accumulation of CBD or THC was observed after 7 days of multiple dose administration. Dose-dependent PD effects correlated with peak plasma concentrations of THC and its active metabolite 11-OH-THC.
Marcel O. Bonn-Miller, Ph.D., Chief Scientific Officer at Charlotte's Web and Board Member at DeFloria, stated, "We are extremely encouraged by these results," highlighting that the drug achieved blood concentration levels matching or exceeding existing cannabis-based medicines like Epidiolex.
Safety Profile
The most common treatment-emergent adverse events in the AJA001-treated groups were somnolence, anxiety, dizziness, and headache. One serious adverse event, anxiety, was reported by a participant receiving 906.4 mg of AJA001, which resolved within a day without sequelae. This safety profile supports further investigation of AJA001 in Phase 2 trials.
Implications for Future Development
The safety, PK, and PD findings support doses ranging from 100 mg to 660 mg of AJA001 per day for Phase 2 trials in children, adolescents, and adults with ASD. This corresponds to an upper limit of 395 mg CBD and 15 mg THC per day. DeFloria is preparing to submit the Phase 1 trial data as part of its Investigational New Drug application to the FDA. Pending approval, the company plans to initiate two Phase 2 trials in the second quarter of 2025 to explore AJA001's potential for addressing behavioral symptoms and improving the quality of life for individuals with ASD.
Jared Stanley, DeFloria’s CEO, noted, "Currently available treatments for ASD are not well tolerated, making patient compliance a challenge," underscoring the need for new therapeutic options.
If successful in later trials, AJA001, which utilizes hemp extract from Charlotte's Web's proprietary CW1AS1 cannabis strain, could become one of the first FDA-approved botanical drugs derived from hemp for autism spectrum disorder.
Bill Morachnick, CEO of Charlotte’s Web, commented, "DeFloria’s Phase 1 results validate our strategic vision of advancing botanical science through pharmaceutical pathways," emphasizing the value of their proprietary genetics and expertise in hemp cultivation.
