LITTLETON, Colo. — U.S. Food and Drug Administration (FDA) has completed its review of the Investigational New Drug (IND) application for AJA001, an orally delivered, multi-cannabinoid botanical drug, concluding DeFloria Inc., a collaboration between Ajna BioSciences PBC and Charlotte’s Web Holdings Inc., may proceed with its planned Phase 2 clinical trial. DeFloria is developing AJA001 as a treatment for symptoms of autism spectrum disorder (ASD) and plans to initiate the Phase 2 study by mid-year 2025.
With as many as 1 in 36 children diagnosed, ASD represents a growing and underserved market valued at nearly $4 billion in the U.S. with a greater than 4% compounded annual growth rate (CAGR). Safe and effective treatment options are severely limited for the autism community, with only two approved treatments – atypical antipsychotics that exhibit frequent adverse side effects, poor tolerance, and a lack of compliance from patients.
“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,” said Jared Stanley, CEO of DeFloria. “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.”
AJA001 was developed by DeFloria, Ajna BioSciences, and Charlotte’s Web to leverage a decade of Charlotte’s Web’s observational data, research, and innovative cultivation processes that are the foundation of the proprietary hemp genetics of AJA001. The unique collaboration also brings compliance with current good manufacturing practices (cGMP) and FDA quality requirements to advance a pharmaceutical botanical drug through development and ultimately to support a registrational review and approval process.
“It is exciting to see AJA001 advance through the botanical drug pathway following the guidance established by the FDA,” stated Jinhui Dou, Ph.D., former FDA expert pharmacologist and botanical review team lead. “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.”
The Phase 2 clinical trial is designed as an open-label, 12-week study to investigate AJA001 as a potential treatment for the behavioral symptoms associated with ASD. The study will enroll 60 adolescent and young adult patients (ages 13-29 years old). The primary goal of the Phase 2 study will be to establish a titration regimen and efficacy signals to inform dosing levels for Phase 3 studies.
“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,” stated Orrin Devinsky, MD and chief medical advisor. “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.”
In addition to the US Phase 2 open-label IND study, DeFloria also plans to initiate a Phase 2 open-label pediatric study of AJA001 in Australia in 2025.
“For more than 15 years, my family and our team have been laying the foundation for this botanical drug,” said Joel Stanley, CEO, Ajna BioSciences and Chairman of the Board, DeFloria. “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.”
About ASD
Approximately one in thirty-six children may have autism spectrum disorder (ASD) (Maenner et al 2023). ASD is characterized by core deficits in social communication, impaired reciprocal social interactions, and restricted, repetitive, and stereotyped patterns of behaviors or interests. Irritability, impulsivity, and “temper tantrums” are common symptoms and contribute to high caregiver burden (Lecavalier et al 2006).
About DeFloria
DeFloria Inc., a collaboration between Charlotte’s Web Inc., AJNA BioSciences PBC, and British American Tobacco PLC (BAT), is on a mission to address crucial healthcare needs through groundbreaking innovations from multi-compound drugs originating in nature. DeFloria merges the experience of its partners with other drug development experts, for a combined century of experience in regulatory, research, and quality-controlled standardized manufacturing. DeFloria’s ambitious endeavor aims to secure FDA approval for a novel botanical drug developed from Charlotte’s Web proprietary hemp genetics that targets autism spectrum disorder (ASD).
About Ajna BioSciences
Ajna BioSciences PBC is a botanical drug development company based in Denver, Colorado, focused on natural pharmaceuticals for Central Nervous System disorders. With specialized laboratory infrastructure and industry-leading Chemistry, Manufacturing, and Controls (CMC) capabilities, Ajna accelerates the development of FDA-approved botanical medicines. Ajna leads CMC in the partnership, regulatory filings, commercialization strategies, and interfaces with DeFloria’s experts to guide the clinical trial programs.
About Charlotte’s Web
Charlotte’s Web Holdings Inc., a Certified B Corporation and a market leader in hemp wellness products, pioneered the CBD industry with its groundbreaking full-spectrum hemp extracts. Founded by the seven Stanley brothers, Charlotte’s Web set the standard for quality and efficacy in plant-based health solutions, earning global recognition as the world’s most trusted hemp extract. As a partner in DeFloria, Charlotte’s Web contributes years of intellectual property and over $50 million in GMP manufacturing infrastructure, ensuring scalable production for clinical trials and future market supply.
