Single Ascending and Multiple Ascending Dose Trial
Demonstrated AJA001 to be Safe and Well-tolerated, with Formulation
Optimized for ASD
Presentation at the 63rd Annual Meeting of the
American College of Neuropsychopharmacology
LITTLETON, Colo., Dec. 12,
2024 /PRNewswire/ -- DeFloria, a joint venture
between Ajna BioSciences and Charlotte's Web (TSX: CWEB, OTCQX:
CWBHF), reported positive results from a Phase 1 single ascending
and multiple ascending dose trial of AJA001 in development for the
treatment of symptoms of autism spectrum disorder (ASD) in a poster
presentation at the 63rd Annual Meeting of the American
College of Neuropsychopharmacology in Phoenix, Arizona. AJA001 is an orally
delivered, multi-cannabinoid botanical drug product (BDP) designed
to provide a broad therapeutic effect.
In a poster entitled, "A Single and Multiple Ascending Dose and
Food Effect Study to Evaluate the Safety, Tolerability,
Pharmacokinetics, and Pharmacodynamics of AJA001, a Full-Spectrum
Hemp-Derived Botanical Drug for Autism Spectrum Disorder, "
Marcel O. Bonn-Miller, Ph.D., Chief
Scientific Officer at Charlotte's Web and Board Member at DeFloria,
presented the results from the recently completed Phase 1
randomized, double-blind, placebo-controlled trial that evaluated
the tolerability and pharmacokinetic (PK) / pharmacodynamic (PD)
profile of several dose levels of AJA001 in 70 healthy volunteers
ranging from 19 to 55 years.
Results demonstrated that AJA001 was well-tolerated at doses up
to 680 mg per day administered as a single dose and up to 660 mg
per day administered twice a day. In both the single and multiple
ascending dose portions of the trial, AJA001 displayed
dose-proportional PK for CBD and THC that matched or exceeded the
plasma concentrations of the comparable cannabinoid therapeutics
dronabinol, nabiximols, and Epidiolex®. PK data
following multiple dose administration of AJA001 showed minimal
accumulation of CBD or THC after 7 days.
AJA001's PD effects were shown to be dose-dependent and
correlated with peak plasma concentrations of THC and its active
metabolite 11-OH-THC after administration of single doses. An
analysis of participant responses on the Drug Effects Questionnaire
(DEQ) demonstrated that the magnitude and time course of AJA001's
subjective drug effects were moderate and transient in
nature.
The safety, PK, and PD findings support doses from 100 mg to 660
mg of AJA001 per day for Phase 2 trials in children,
adolescents, and adults with ASD. This is equivalent to an upper
limit of 395 mg CBD and 15 mg THC per day. The most common
treatment emergent adverse events in the AJA001 treated groups were
somnolence, anxiety, dizziness, and headache. One serious adverse
event was reported by a participant receiving 906.4 mg of AJA001
who reported anxiety that resolved by the next day with no
sequalae.
A copy of the poster is available on the DeFloria website at
http://defloria.bio.
"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," said Dr. Bonn-Miller. "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."
"We are excited to present this important data and look
forward to initiating two Phase 2 trials of AJA001 in ASD in the
second quarter of 2025," said Jared
Stanley, CEO of DeFloria. "Despite its prevalence, ASD
remains a difficult condition to treat. Moreover, currently
available treatments for ASD are not well tolerated, making patient
compliance a challenge. DeFloria is committed to supporting
the ASD community and developing AJA001 to treat the behavioral
symptoms associated with ASD with a drug product that is designed
to provide broad therapeutic efficacy."
About ASD
Approximately 1 in 36 children may have 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 AJA-001
AJA001 Oral Solution is a botanical drug product (BDP)
containing full spectrum hemp extract (FSHE) botanical drug
substance (BDS) derived from Cannabis sativa L proprietary
CW1AS1 hemp cultivar. The primary cannabinoid is cannabidiol
(CBD). As an innovative multi-cannabinoid formulation, it may
provide broad therapeutic efficacy. Its full-spectrum composition
has potential therapeutic applications across various medical
conditions due to its varied mechanisms of action.
About DeFloria
DeFloria LLC, 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. With 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 cutting-edge botanical drug developed from Charlotte's Web
proprietary hemp genetics that targets a neurological condition
identified by the DeFloria leadership and research teams.
Cautionary Note of Forward-Looking Statement
This news release contains "forward-looking statements" within
the meaning of the United States Private Securities Litigation
Reform Act of 1995. Often, but not always, forward-looking
statements can be identified by the use of words such as "plans",
"expects" or "does not expect", "is expected", "estimates",
"intends", "anticipates" or "does not anticipate", or "believes",
or variations of such words and phrases or state that certain
actions, events or results "may", "could", "would", "might" or
"will" be taken, occur or be achieved. Forward-looking statements
involve known and unknown risks, uncertainties and other factors
which may cause the actual results, performance or achievements of
DeFloria, LLC to be materially different from any future results,
performance or achievements expressed or implied by the
forward-looking statements contained in this news release. Examples
of such statements and uncertainties include statements with
respect to future dose trial results, therapeutic efficacy, or
approvals of AJA001 Oral Solution.
Risks, uncertainties and other factors involved with
forward-looking statements could cause actual events, results,
performance, prospects and opportunities to differ materially from
those expressed or implied by such forward-looking statements,
including negative outcomes in future dose trials, therapeutic
efficacy, or delays in approvals of AJA001 Oral Solution.
In respect of the forward-looking statements, DeFloria, LLC has
provided such statements in reliance on certain assumptions that it
believes are reasonable at this time. Although DeFloria, LLC
and its collaborators and partners believe that the assumptions and
factors used in preparing the forward-looking statements in this
news release are reasonable, undue reliance should not be placed on
such information or statements and no assurance can be given that
such events will occur in the disclosed time frames or at all.
Should one or more of the foregoing risks or uncertainties
materialize, or should assumptions underlying the forward-looking
statements prove incorrect, actual results may vary materially from
those described herein as intended, planned, anticipated, believed,
estimated or expected. Although DeFloria, LLC has attempted to
identify important risks, uncertainties and factors which could
cause actual results to differ materially, there may be others that
cause results not to be as anticipated, estimated or intended. The
forward-looking statements included in this news release are made
as of the date of this news release and DeFloria, LLCs does not
undertake any obligation to publicly update such forward-looking
statements to reflect new information, subsequent events or
otherwise unless required by applicable securities laws.
Investor/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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