FDA clears DeFloria’s cannabis-based autism treatment for phase 2 trial

DeFloria said Monday that the Federal Drug Administration has stamped the firm’s application to begin a Phase 2 clinical trial for AJA001, an oral cannabinoid drug being made to treat symptoms of autism spectrum disorder.

The company, which was formed in 2023 as a joint-venture among Charlotte’s Web (OTCQX: CWBHF), Ajna BioSciences and British American Tobacco (NYSE: BTI), plans to start the trial by mid-year 2025. BAT holds an equity stake through 200,000 preferred units following an initial $10 million investment, with Charlotte’s Web and Ajna each holding 400,000 voting common units.

“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,” DeFloria CEO Jared Stanley said in a statement.

The planned 12-week study will test the drug on 60 adolescents and young adults with autism, ages 13-29, to establish dosing levels for future larger studies.

AJA001 was made using Charlotte’s Web’s hemp genetics and contains full spectrum hemp extract with cannabidiol (CBD) as the primary ingredient.

According to DeFloria, autism affects as many as 1 in 36 children and represents a $4 billion market in the U.S. that’s growing more than 4% annually. Current treatment options are limited to just two approved drugs – antipsychotics that often cause side effects and compliance problems.

“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,” said Orrin Devinsky, MD and Chief Medical Advisor for DeFloria.

The drug’s development builds on the foundation of Charlotte’s Web’s origin story.

“For more than 15 years, my family and our team have been laying the foundation for this botanical drug,” said Joel Stanley, CEO of Ajna BioSciences and Chairman of DeFloria’s Board. “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.”

Charlotte’s Web CEO Bill Morachnick emphasized the significance of this milestone in extending the company’s leadership “from the consumer sector into the regulated medical sector.”

DeFloria also plans to conduct a separate pediatric study of AJA001 in Australia in 2025.