AJA001, a botanical drug for autism spectrum disorder (ASD), showed positive Phase 1 trial results. Phase 2 trials are set for mid-2025. AJA001, derived from Charlotte's Web’s hemp genetics, would be the first full-spectrum cannabinoid drug seeking FDA approval, offering a new treatment option for ASD.
A groundbreaking effort is underway to achieve FDA approval for a drug targeting autism spectrum disorder (ASD). Ajna BioSciences, a botanical drug development startup, is leading the initiative in partnership with Charlotte's Web and British American Tobacco. Together, they are developing AJA001, a multi-cannabinoid botanical drug designed to treat ASD symptoms with an oral treatment.
Last month, the venture announced promising results from Phase 1 trials, which showed the drug's safety and tolerability. The findings were shared at the 63rd Annual Meeting of the American College of Neuropsychopharmacology in Phoenix. The goal is to elevate cannabis-based treatments to a pharmaceutical standard with consistent quality control.
While cannabis-based drugs like Epidiolex have received FDA approval, AJA001 would be the first full-spectrum cannabinoid drug to do so. Phase 1 trials in Australia showed positive results in bioavailability and pharmacokinetics, setting the stage for Phase 2 trials, which are expected to begin in mid-2025.
Navigating the FDA’s botanical drug approval process is complex, as cannabis involves thousands of molecules compared to synthetic single-molecule drugs. The team behind AJA001 includes an expert who previously led the Epidiolex trials, bringing valuable experience to the process.
The name “Ajna” was chosen for its symbolic meaning of bridging the conscious and subconscious. This ambitious goal has been in the works for over a decade, with AJA001, developed from Charlotte's Web’s proprietary hemp genetics, potentially offering the first effective treatment for ASD beyond antipsychotic medications.