 botanical drug developed to treat behavioral symptoms associated with autism spectrum disorder (ASD).){kind=link}
DeFloria is moving towards the beginning of a Phase 2 Clinical Trial for AJA001. AJA001 is cannabinoid based botanical (FDA approved) drug that was developed to treat behavioral symptoms related to autism spectrum disorder.
The FDA approved the FDA-approved Investigational New Drug Application (IND) for Phase 2 clinical studies in February 2025. It was the FDA-approved full-spectrum CBD drug.
The company has now received approval to enroll 60 participants aged 13 to 29 for a 12-week study. This will establish a dosing regimen, a titration schedule, and efficacy indicators to guide future Phase 3 trials.
A recent interview published in Medical Leader Joel Stanley of Ajna BioSciences & Chairman of DeFloria said the trial would begin very soon.
The results will be available in 14-16 months after we have completed this trial. Then we will start to look for commercialization partners, and then the most appropriate way to fund larger Phase 3 trial.
DeFloria has specified a timeline for Phase 3 tests to start next year. This will be followed by a FDA approval final in 2030.
This would be the fifth and first botanical medicine to receive FDA approval, replacing Jazz Pharmaceuticals’ $1bn Epidiolex approved in 2018.
Stanley says that being an American-based firm has made the process much more difficult than for its UK-based counterpart.
Epidiolex (created by GW Pharmaceuticals), a CBD-based drug approved in 2018 for rare cases of epilepsy, was developed.
“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. The DEA still considered CBD a schedule 1 drug for American companies until 2019. The UK firm was therefore able to speed through the entire process.”
There is a substantial market demand. ASD affects about 1 out 36 US-born children. Currently, only two FDA-approved drugs are available, which both suffer from serious side-effects and have poor tolerability over the long term.