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What was once thought impossible is now on the horizon as the medical cannabis and pharmaceutical industries continue to overlap. Joel Stanley, former CEO of Charlotte’s Web, is part of a joint effort to achieve approval from the FDA for a novel treatment for the symptoms of autism spectrum disorder (ASD). Stanley is the founder and CEO of Ajna BioSciences, a botanical drug development startup.
DeFloria, a joint venture between Ajna BioSciences, Charlotte’s Web, and British American Tobacco, is running trials of a drug candidate of the same name—AJA001 by its FDA code—which is being developed to treat the symptoms of ASD. It’s a multi-cannabinoid botanical drug product (BDP) designed to provide a broad therapeutic effect, delivered orally.
Last month, the venture announced positive results from a Phase 1 trial of the treatment, demonstrating its safety. The findings were presented last month at the 63rd Annual Meeting of the American College of Neuropsychopharmacology held in Phoenix, Arizona.
“The regulatory pathways for us to move a full-spectrum hemp-derived cannabinoid drug through the FDA has only been available to us fairly recently,” Stanley tells Forbes in a video call. “It’s taking a product similar to Charlotte’s Web, which I’m very proud of, and standardizing it to a pharmaceutical level—taking it to a higher level of quality control and consistency.” His brother Jared, former COO of Charlotte’s Web, is CEO of DeFloria. Patented genetics from Charlotte’s Web were used in the process.
There are very rare instances when cannabis-based drug candidates have achieved FDA approval, with only one containing cannabis: Epidiolex, which contains purified CBD, is a single-molecule drug, while others like dronabinolor nabilone, are synthetic and only mimic CBD and THC. But this would be the first time the first time a drug candidate of this kind is moving through the FDA process.
“It’s not only the first full-spectrum cannabinoid drug moving through the FDA, it’s the first oral systemic full-spectrum botanical drug moving through the FDA,” says Stanley. Stanley explained that Phase I trials were completed in Australia, finding positive results about the drug candidate’s bioavailability, pharmacokinetics, pharmacodynamics, and so on.
“We are extremely encouraged by these results from our Phase 1 single and multiple ascending dose trial, which demonstrate that AJA001 is well-tolerated across a range of doses and provides a favorable pharmacokinetic profile for both cannabidiol and tetrahydrocannabinol. Importantly, the results from this trial inform the doses to be evaluated in upcoming Phase 2 clinical trials of AJA001 in children, adolescents, and adults with ASD,” Marcel O. Bonn-Miller, Ph.D., chief scientific officer at Charlotte’s Web and board member at DeFloria, said in the announcement.
The team plans to launch two Phase 2 trials of the treatment in the second quarter of 2025.
The Unique Situation of a Botanical Drug Candidate Approval Process, and Autism
As a botanical drug candidate, the road to approval is less linear. “A lot of people don’t even realize that the FDA has a different regulatory regime for botanical drugs,” Stanley says. “And only four botanical drugs have been approved. And none of those are oral system. Three of them are topical and one of them coats the GI tract, so they’re not absorbed by the body.”
He explained that one barrier to the process has been what’s called in the FDA chemistry manufacturing controls, stringent rules that aren’t always straightforward when it comes to botanical drugs. Many drug candidates involve single molecules, often synthetic, but full-spectrum cannabis involves thousands of molecules.
They have the right team for the job. Dr. Orrin Devinski, who was the lead principal investigator for Epidiolex, is Ajna’s chief medical officer and oversees the clinical trial design. Epidiolex is the only non-synthetic cannabis drug to get through the FDA approval process. Devinski is also director at the Comprehensive Epilepsy Center at NYU Langone.
Stanley’s brother Jesse suggested the Sanskrit word “Ajna” for the drug candidate, which translates to the authority of consciousness or a doorway, allowing your conscious mind to communicate with your subconscious mind. The brothers have been working on this ambitious goal for over a decade.
AJA001 is developed from Charlotte’s Web proprietary hemp genetics. Joel and his brothers, known colloquially as the Stanley brothers, gained notoriety after naming a particularly useful strain of hemp after Charlotte Figi, who passed away in 2020 due to Dravet syndrome, but whose life was transformed by the magic of a delicate balance of cannabinoids. She became an icon in the medical cannabis industry. The story was propelled by appearances on places like CNN, with Dr. Sanjay Gupta, and a prominent presence in conversations in the medical cannabis community.
Autism is another critical disorder being treated with cannabis. About one in 36 children may have ASD, characterized by impaired communication, and social interactions, as well as impulsive behaviors. Today, for people with ASD, the only two approved drugs for autism symptoms are both atypical antipsychotics. Ajna Biosciences, Charlotte’s Web, and the Stanley brothers aim to change that.
“}]] Joel Stanley, the founder and former CEO of Charlotte’s Web, is leading an effort to achieve approval from the FDA for a novel treatment to treat autism spectrum disor… Read More
