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DeFloria's AJA001: A Novel Botanical Drug for Autism Spectrum Disorder Advances to Phase 2 Trials

• DeFloria, a joint venture involving Charlotte's Web, is developing AJA001, a multi-cannabinoid botanical drug, for treating Autism Spectrum Disorder (ASD). • AJA001 has completed Phase 1 trials, demonstrating safety and a favorable pharmacokinetic profile, paving the way for Phase 2 studies. • The Phase 2 trials, planned for the second quarter of 2025, will evaluate AJA001's efficacy in children, adolescents, and adults with ASD. • AJA001 represents a unique approach as the first oral systemic full-spectrum botanical drug to navigate the FDA approval process.

DeFloria, a joint venture between Ajna BioSciences, Charlotte's Web, and British American Tobacco, is advancing AJA001, a novel multi-cannabinoid botanical drug product (BDP), towards FDA approval for the treatment of Autism Spectrum Disorder (ASD). This initiative, spearheaded by Joel Stanley, former CEO of Charlotte's Web and current CEO of Ajna BioSciences, aims to provide a standardized pharmaceutical-grade treatment option derived from full-spectrum hemp.

Positive Phase 1 Results

Last month, DeFloria announced positive results from a Phase 1 trial of AJA001, demonstrating its safety and tolerability. The findings were presented at the 63rd Annual Meeting of the American College of Neuropsychopharmacology in Phoenix, Arizona. According to Marcel O. Bonn-Miller, Ph.D., chief scientific officer at Charlotte's Web and board member at DeFloria, the trial results indicate that AJA001 exhibits a favorable pharmacokinetic profile for both cannabidiol (CBD) and tetrahydrocannabinol (THC). These results have informed the doses to be evaluated in upcoming Phase 2 clinical trials of AJA001 in children, adolescents, and adults with ASD.

AJA001: A Unique Botanical Drug Candidate

AJA001 is distinguished as the first oral systemic full-spectrum botanical drug moving through the FDA approval process. Joel Stanley emphasized the novelty of this approach, noting that while the FDA has a regulatory pathway for botanical drugs, only four such drugs have been approved to date, none of which are oral systemics. The standardization of a full-spectrum hemp-derived cannabinoid drug to a pharmaceutical level involves overcoming challenges related to chemistry manufacturing controls, which are stringent rules that are not always straightforward when it comes to botanical drugs with thousands of molecules.

Upcoming Phase 2 Trials

The team plans to launch two Phase 2 trials of AJA001 in the second quarter of 2025. These trials will assess the efficacy of AJA001 in treating the symptoms of ASD in a broader patient population. The development of AJA001 leverages patented genetics from Charlotte's Web, building upon the legacy of the Stanley brothers' work with medical cannabis.

Expertise Driving the Approval Process

To navigate the complexities of the FDA approval process for a botanical drug, Ajna BioSciences has enlisted the expertise of Dr. Orrin Devinski, chief medical officer of Ajna and director at the Comprehensive Epilepsy Center at NYU Langone. Dr. Devinski previously served as the lead principal investigator for Epidiolex, the only non-synthetic cannabis drug to receive FDA approval, bringing invaluable experience to the AJA001 development program.
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Reference News

[1]
Charlotte's Web Founder Aims For FDA Approval Of Autism Spectrum Disorder Drug
forbes.com · Jan 24, 2025

Joel Stanley, founder of Ajna BioSciences, is leading efforts for FDA approval of AJA001, a multi-cannabinoid drug for a...

[2]
DeFloria Achieves Ground-Breaking Milestone in Botanical Drug Development
cannabisbusinesstimes.com · Mar 18, 2025
[3]
How Close Is The First FDA-Approved Full-Spectrum Botanical Drug For Autism
clinicalleader.com · May 6, 2025
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