DeFloria Announces Positive Results from Phase 1 Trial of AJA001 Being Developed for the Treatment of Autism Spectrum Disorder (ASD)

Single Ascending and Multiple Ascending Dose Trial Demonstrated AJA001 to be Safe and Well-tolerated, with Formulation Optimized for ASD

Presentation at the 63^rd 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 63^rd 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
[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