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
jnugent@tiberend.com
Media Contact:
Tiberend Strategic Advisors, Inc.
Casey McDonald
(646) 577-8520
cmcdonald@tiberend.com
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.
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