The cannabinoid pharmaceutical company DeFloria, jointly established by the renowned CBD enterprise Charlotte's Web, Ajna BioSciences, and the international tobacco giant British American Tobacco (BAT), recently announced that its new cannabis-based medication AJA001, designed for the treatment of autism, has received FDA approval and is set to commence Phase II clinical trials.

It has been reported that the FDA has completed its review of the Investigational New Drug (IND) clinical trial for AJA001, an oral phytocannabinoid medicine developed by DeFloria Company. The conclusion is that the medication can continue to proceed to Phase II of the clinical trials as planned. AJA001 is a phytocannabinoid medicine designed to treat Autism Spectrum Disorder (ASD), with the second phase of clinical research expected to commence in mid-2025.

Translation: About Autism

The symptomatic expression of Autism Spectrum Disorder includes: impaired social communication and reciprocal social interaction, along with restricted, repetitive, and stereotyped patterns of behavior or interests. Irritability, impulsivity, and "temper tantrums" are common symptoms that can increase the burden on caregivers.

Approximately 1 in 36 children is diagnosed with autism spectrum disorder (ASD), which represents a growing market that is underserved. In the United States, this market is valued at nearly $4 billion, with a compound annual growth rate (CAGR) of over 4%.

According to GlobalData, in 2023 the number of diagnosed autism cases in 16 major pharmaceutical markets worldwide reached 9.81 million, with a prevalence of generic drugs flooding the market. Currently, only two drugs have received FDA approval for the treatment of autism-related irritability: Risperdal (risperidone) from Janssen Pharmaceuticals, a division of Johnson & Johnson, and Abilify (aripiprazole) from Otsuka Pharmaceutical Co., Ltd.

In fact, for individuals with autism, there are very limited options for safe and effective treatment. Currently, the two approved therapeutic drugs are atypical antipsychotics, which often come with adverse side effects, and both tolerance and patient compliance are poor.

DeFloria's CEO, Jared Stanley, stated: "The submission of the New Drug Application for AJA001 and the launch of Phase 2 clinical trials in the United States mark a historic moment in our development of plant-based medicinal therapies with systemic absorption, in compliance with the FDA guidelines set for advanced clinical trials." "DeFloria is committed to supporting the ASD community and developing medications with broad therapeutic effects to treat behavioral symptoms associated with ASD."

AJA001 was developed through a collaboration between DeFloria, Ajna BioSciences, and the Charlotte Web, utilizing the latter's decade of observational data, research, and innovative cultivation processes, which form the foundation of AJA001's unique cannabis genetics. This distinctive partnership is compliant with current Good Manufacturing Practices (cGMP) and FDA quality requirements, aiming to advance the development of medicinal plant-based drugs and ultimately support the registration review and approval process.

Dr. Dou Jinhui, a former FDA pharmacologist and head of the botanical drug review panel, expressed his excitement: "Seeing AJA001 make progress through the botanical drug pathway in accordance with FDA guidelines is very exciting. As the first oral botanical medicine to meet cGMP and quality standards, AJA001 has set a benchmark for future drugs of this kind."

It is reported that the outcomes of the prior Phase I single ascending dose (SAD) and multiple ascending dose (MAD) trials (NCT06019065) demonstrate that AJA001 is well-tolerated at single-dose levels up to 680 mg/day and twice-daily dosing levels up to 660 mg/day. The results indicate that the effects are dose-dependent, and correlate with the peak plasma concentrations of Tetrahydrocannabinol (THC) and its active metabolite 11-OH-THC.

The upcoming Phase 2 clinical trial is a 12-week open-label study designed to investigate AJA001 as a potential treatment method for ASD-related behavioral symptoms. The trial will recruit 60 adolescent and young adult patients (ages 13-29). The primary objective of the study is to establish a titration scheme and efficacy signals to guide dosing for Phase 3 clinical trials.

"There is already a substantial amount of data supporting the potential therapeutic efficacy of cannabinoids in treating autism spectrum disorder, and we hope this new treatment will be safer and more effective than currently available therapies," stated Dr. Orrin Devinsky, MD and Chief Medical Advisor. "DeFloria has taken the lead in developing a promising drug for this indication through the FDA's rigorous evaluation process, with the potential to benefit millions of patients with autism."

In addition to launching a Phase 2 open-label clinical trial in the United States, DeFloria also plans to initiate a Phase 2 open-label pediatric study of AJA001 in Australia in 2025.

"Ajna BioSciences CEO and Chairman of the DeFloria Board, Joel Stanley, stated, 'For over 15 years, we have been laying the groundwork for this botanical medicine. Now, with the achievement of this Phase II milestone, we are one step closer to providing a potential treatment option for Autism Spectrum Disorder.'"

About AJA001

AJA001 is a botanical medicine made from a full-spectrum hemp extract (FSHE) derived from the proprietary cannabis variety CW1AS1 of Cannabis sativa L. The primary cannabinoid component is cannabidiol (CBD), and it also includes other active cannabinoids. As an innovative multi-cannabinoid botanical medicine, AJA001 is capable of providing a wide range of therapeutic effects due to its diverse mechanisms of action, thus holding potential therapeutic applications under various medical conditions.

About DeFloria

DeFloria, Inc. is a collaborative project involving Charlotte's Web, AJNA BioSciences PBC, and the international tobacco giant British American Tobacco (BAT). Its mission is to meet critical healthcare needs through breakthrough innovations derived from natural botanical medicines. DeFloria combines the expertise of its partners with that of other pharmaceutical development experts, boasting nearly a century of combined experience in regulatory compliance, research, and quality control of standardized production. DeFloria has set an ambitious goal to develop a new botanical drug using the proprietary hemp genetics of Charlotte's Web, aiming to gain FDA approval for the treatment of Autism Spectrum Disorder (ASD).

About Ajna BioSciences

Ajna BioSciences PBC is a plant-based pharmaceutical development company headquartered in Denver, Colorado, focusing on natural medications for central nervous system disorders. With specialized laboratory infrastructure and industry-leading Chemistry, Manufacturing, and Controls (CMC) capabilities, Ajna accelerates the development of plant-based medications towards FDA approval. Ajna leads CMC collaborations, overseeing regulatory filings, commercialization strategies, and interfaces with experts at DeFloria to guide clinical trial programs.

About Charlotte's Web

Charlotte's Web is a B Corp Certified company and a market leader in hemp health products, having pioneered the CBD industry with its groundbreaking full-spectrum hemp extract. It has set the standard for the quality and efficacy of plant health solutions, gaining global recognition and becoming the world's most trusted hemp extract. As a partner of DeFloria, Charlotte's Web has contributed years of intellectual property and over $50 million in GMP manufacturing infrastructure, ensuring scalable production for clinical trials and future market supply.

It has been reported that elsewhere in the field of ASD (Autism Spectrum Disorder) treatment, the Israeli Ministry of Health's Medical Cannabis Agency (IMCA) has restarted the clinical trial of SciSparc's SCI-210 drug, which is aimed at treating pediatric patients.

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