DeFloria, a joint venture between Charlotte’s Web Holdings and Ajna BioSciences, is advancing toward the start of its Phase 2 clinical trial for AJA001, a cannabinoid-based (FDA) botanical drug developed to treat behavioural symptoms associated with autism spectrum disorder (ASD).
In February 2025, the company announced that its Investigational New Drug (IND) application for Phase 2 clinical trials had been cleared by the FDA, marking the first full-spectrum CBD-based drug in history to do so.
With approval granted, the company is now preparing to begin enrolling 60 participants, aged 13-29, for its 12-week study to establish a titration regimen and efficacy signals to inform dosing levels for eventual Phase 3 studies.
In a recently published interview in Clinical Leader Joel Stanley, CEO of Ajna BioSciences and Chairman of DeFloria, said that the trial is set to begin imminently.
“So, as soon as we close this round, we’ll move into those Phase 2 trials, and then it’ll probably be 14 to 16 months for results. We’ll start looking at future commercialization partners from that point and look at the appropriate ways to finance the larger Phase 3 trials.”
According to its specified timeline, DeFloria expects Phase 3 trials to begin next year, ahead of a final FDA approval in 2030.
It would become the first full-spectrum and only fifth botanical drug to be approved by the FDA, taking its place alongside Jazz Pharmaceutical’s $1bn Epidiolex, which was approved in 2018.
However, according to Stanley, being a US-based company has meant the process has been far harder than its UK-based pharmaceutical counterpart.
“Since 2018 (when the US legalized hemp), one CBD drug, Epidiolex, developed by GW Pharmaceuticals, has been approved for very rare epilepsy diagnoses,” he explained.
“A pure CBD drug — very different than our drug AJA001 — made by a UK company meant they didn’t need a Schedule 1 manufacturing license. Yet for American companies, the DEA considered CBD a Schedule 1 drug up until 2019. So, the UK company was able to race through the process.”
The market need is substantial. ASD affects approximately 1 in 36 children in the US, and currently only two FDA-approved drug treatments exist, both atypical antipsychotics known for serious side effects and poor long-term tolerability.
