FDA Grants Investigational New Drug Application to DeFloria for Phase 2 Oral Cannabinoid Autism Treatment

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A recent press release (1) announced that DeFloria, Inc., which is a a collaboration between Charlotte's Web, Inc., AJNA BioSciences PBC, and British American Tobacco PLC (BAT), received a Investigational New Drug (IND) application for AJA001 from the US Food and Drug administration (FDA) after they completed their review. AJA001 is a multi-cannabinoid botanical drug that is administered orally. With the IND application granted, DeFloria will now be able to move forward with their Phase 2 clinical trial, slated for mid-year 2025. DeFloria hopes that this multi-cannabinoid botanical drug can be utilized as a form of treatment for symptoms associated with autism spectrum disorder (ASD) (1).

The statistics behind ASD are that about 1 in 36 children receive a diagnosis (1). As mentioned in the press release (1), ASD has a market value of around $4 billion in the US and a 4% compounded annual growth rate (CAGR). Currently there are only two treatments for autism that are approved which are atypical antipsychotics which bring their own adverse side effects.

“IND submission for AJA001 and the initiation of the Phase 2 study in the US mark a historic moment in our mission to develop a systemically absorbed botanical drug therapy that meets guidelines established by the FDA for advanced clinical testing,” Jared Stanley, CEO of DeFloria, said (1). “DeFloria is committed to supporting the ASD community and developing AJA001 to treat the behavioral symptoms associated with ASD with a drug that is designed to provide broad therapeutic efficacy.”

“It is exciting to see AJA001 advance through the botanical drug pathway following the guidance established by the FDA,” Jinhui Dou, Ph.D., former FDA expert pharmacologist and botanical review team lead, explained (1). “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.”

According to the press release (1), the Phase 2 clinical trial willbe an “open-label” 12-week study that will seek to find how AJA001 could be used to help with behavioral symptoms attributed to ASD. To gather their data, the study will use 60 adolescent and young adult patients between the ages of 13-29 years old (1). The main focus of the Phase 2 clinical trial will be to decipher what regimen and efficacy signals are needed to be used so that dosage amounts can be used in the following Phase 3 clinical trial.

“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,” Orrin Devinsky, MD and Chief Medical Advisor, mentioned (1). “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.”

“For more than 15 years, my family and our team have been laying the foundation for this botanical drug,” Joel Stanley, CEO, Ajna BioSciences and Chairman of the Board, DeFloria, said (1). “Charlotte Figi's story, shared by Dr. Sanjay Gupta on CNN, put the benefits of cannabinoids on the world's radar and sparked a movement. Now, with this Phase 2 milestone, we're one step closer to delivering a potential treatment option for autism spectrum disorder, a condition for which few effective therapies exist despite impacting millions worldwide.”

DeFloria hopes to also implement a Phase 2 open-label pediatric study for AJA001 sometime in 2025 in Australia (1).

Reference

1. DeFloria LLC. Defloria receives Investigational New Drug Application Clearance from FDA for phase 2 clinical trial of AJA001, an oral cannabinoid drug being developed for the treatment of autism spectrum disorder (ASD) https://www.prnewswire.com/news-releases/defloria-receives-investigational-new-drug-application-clearance-from-fda-for-phase-2-clinical-trial-of-aja001-an-oral-cannabinoid-drug-being-developed-for-the-treatment-of-autism-spectrum-disorder-asd-302382573.html (accessed Feb 24, 2025).