The U.S. Food and Drug Administration (FDA) has authorized Phase 2 clinical trials for an oral CBD solution intended to address autism spectrum disorder (ASD).
The initiative to develop the drug is under DeFloria, Inc., a collaboration formed in 2023 among three entities – Charlotte’s Web Holdings, Inc., Ajna BioSciences PBC, both of Colorado, and British American Tobacco (BAT) PLC.
“A safe and effective botanical treatment for autism spectrum disorder would be a medical asset that could provide immense value to millions of families and Charlotte’s Web,” said Bill Morachnick, that company’s CEO.
The Phase 2 trials, scheduled to start this summer, are designed as an open-label, 12-week study involving 60 patients aged 13 to 29. The primary objectives are to assess the safety, tolerability, and efficacy of the drug, “AJA001,” in adolescents and adults with ASD, and to obtain data on which to base larger Phase 3 trials.
DeFloria brings together Charlotte’s Web’s expertise in hemp cultivation, Ajna BioSciences’ focus on botanical drug development, and strategic investment from British American Tobacco.
Beyond Phase I
The group hopes to develop AJA001 as a treatment for behavioral symptoms associated with ASD, addressing a critical need for more effective drugs to improve social communication and interaction, and quell restricted and repetitive behaviors. Irritability and impulsivity are common symptoms that can impact the quality of life for those who suffer the condition, and their families.
Phase 1 trials showed that AJA001 was well-tolerated across a range of doses, data that went into determining appropriate dosing protocols for the forthcoming Phase 2 study.
“We feel this research strongly supports cannabinoids as a viable therapy for irritability associated with autism spectrum disorder, which currently lacks adequate treatment options,” said Marcel Bonn-Miller, Chief Scientific Officer at Charlotte’s Web and a DeFloria Board Member.
Years away
The drug could still be as long as eight years away. Phase 2 trials, which assess efficacy, take two to three years; Phase 3, where the drug will be tested in a larger population, typically lasts 3 to 5 years. Once Phase 3 trials are complete, the company submits a New Drug Application to the FDA, which can take another year to approve.
AJA001 represents BAT’s first entry into clinical-stage cannabis research, as the tobacco giant has steadily expanded into the cannabis sector in a strategy that goes beyond tobacco, picking up assets in next-generation cannabis products that include both THC and CBD, vaping, and CBD-infused oral nicotine pouches.
CBD drugs
If it ever does reach regulatory approval, AJA001 would be the first botanical CBD drug specifically for ASD-related symptoms, and only the second FDA-approved prescription drug derived from CBD. The first, Epidiolex, was approved by the FDA in 2018 as a treatment for seizures associated with Lennox-Gastaut syndrome (LGS) and Dravet syndrome, two rare and severe forms of epilepsy. In 2020, its approval was expanded to include tuberous sclerosis complex.
Manufactured by GW Pharmaceuticals, a part of Ireland-based Jazz Pharmaceuticals, Epidiolex is a purified, plant-derived CBD oral solution that has demonstrated effectiveness in reducing seizure frequency in patients who have limited treatment options.