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)

DeFloria plans to initiate a Phase 2 clinical trial by mid-year 2025

LITTLETON, Colo., Feb. 24, 2025 /PRNewswire/ -- DeFloria, Inc., a collaboration between Ajna BioSciences PBC and Charlotte's Web Holdings, Inc. (TSX: CWEB, OTCQX: CWBHF), announced that the 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 that the Company 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 1 in 36 children may have autism spectrum disorder (ASD) (Maenner et al 2023). ASD is characterized by core deficits in (1) social communication, (2) impaired reciprocal social interactions, and (3) 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 AJA001
AJA001 is a botanical drug consisting of full spectrum hemp extract (FSHE) derived from Cannabis sativa L proprietary CW1AS1 hemp cultivar. The primary cannabinoid is cannabidiol (CBD) and includes other cannabinoids that are expected to have activity. As an innovative multi-cannabinoid botanical drug, AJA001 may provide broad therapeutic efficacy with potential therapeutic applications across various medical conditions due to its varied mechanisms of action. 

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 the 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.

Cautionary Note of Forward-Looking Statement

This press release may contain forward-looking statements. Forward-looking statements include all statements that do not relate solely to historical or current facts and can be identified by the use of words such as "may," "will," "expect," "project," "estimate," "anticipate," "plan," "believe," "potential," "should," "continue" or the negative versions of those words or other comparable words. Forward-looking statements are not guarantees of future actions or performance. These forward-looking statements, include those related to drug development outcomes, and are based on information currently available to the Company and its current plans or expectations and are subject to a number of uncertainties and risks that could significantly affect current plans. The Company is under no obligation to, and expressly disclaims any such obligation to, update or alter its forward-looking statements, whether as a result of new information, future events or otherwise.

Investor Contact:
Tiberend Strategic Advisors, Inc.
Jon Nugent
(205) 566-3026
[email protected]

Media Contact:
Tiberend Strategic Advisors, Inc.
Casey McDonald
(646) 577-8520
[email protected]

References:

Maenner MJ, Warren Z, Williams AR, et al. Prevalence and Characteristics of Autism Spectrum Disorder Among Children Aged 8 Years — Autism and Developmental Disabilities Monitoring Network, 11 Sites, United States, 2020. MMWR Surveill Summ 2023;72(No. SS-2):1–14.

Lecavalier L, Leone S, Wiltz J. The impact of behaviour problems on caregiver stress in young people with autism spectrum disorders. J Intellect Disabil Res. 2006;50(Pt 3):172-83.

SOURCE DeFloria, LLC