DeFloria plans to start a Phase II trial evaluating its multi-cannabinoid botanical autism spectrum disorder (ASD) drug, AJA001, by mid-2025 after receiving clearance from the US Food and Drug Administration (FDA).
DeFloria is a joint venture between Ajna BioSciences, Charlotte’s Web, and British American Tobacco PLC (BAT). It is developing AJA001 as a treatment for symptoms of ASD.
Jinhui Dou, PhD, former FDA expert pharmacologist and botanical review team lead said in a press release: “It is exciting to see AJA001 advance through the botanical drug pathway following the guidance established by the FDA. As the first orally ingested botanical drug designed to meet cGMP and quality standards, AJA001 is setting the bar for future pharmaceuticals in this class.”
Clinical trial design
The open-label Phase II trial plans to enroll 60 adolescent and young adult patients between the ages of 13 and 29 years. The company has designed the 12-week study to “establish a titration regimen and efficacy signals to inform dosing levels for Phase III studies”.
In addition to the US Phase II open-label trial, DeFloria also plans to initiate a Phase II open-label trial of AJA001 in the pediatric population in Australia this year.
“There is strong data supporting the potential efficacy of cannabinoids to treat autism spectrum disorder, offering what we hope will be a safer and more effective therapy than what is currently available,” Dr Orrin Devinsky, Chief Medical Advisor. “DeFloria is at the forefront of advancing a promising drug for this indication through the FDA’s rigorous assessment process to potentially help millions affected by autism spectrum disorder.”
DeFloria defines AJA001 as a botanical drug consisting of full spectrum hemp extract (FSHE) derived from Cannabis sativa L proprietary CW1AS1 hemp cultivar. The company boasts of its “broad therapeutic efficacy with potential therapeutic applications across various medical conditions due to its varied mechanisms of action”.
Ajna BioSciences’ product pipeline also consists of another multi-cannabinoid drug, AJA003, which is being developed as a treatment for post-traumatic stress disorder (PTSD). Other Ajna pipeline drugs include two tryptamine formulations, AJA002 and AJA004, that are being evaluated to treat generalized anxiety disorders and an undisclosed indication, respectively.
