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 has received FDA clearance for its Investigational New Drug (IND) application for AJA001, an oral cannabinoid drug targeting Autism Spectrum Disorder (ASD). The company plans to initiate a Phase 2 clinical trial by mid-2025, enrolling 60 patients aged 13-29 years for a 12-week study.
The ASD market is valued at nearly $4 billion in the U.S., with a >4% CAGR. Currently, only two approved treatments exist, both being atypical antipsychotics with significant side effects and poor patient compliance.
AJA001 was developed through collaboration between Ajna BioSciences and Charlotte's Web, leveraging Charlotte's Web's decade of observational data and proprietary hemp genetics. The company also plans to initiate a Phase 2 open-label pediatric study in Australia in 2025.
DeFloria ha ricevuto l'approvazione della FDA per la sua domanda di Nuovo Farmaco Investigativo (IND) per AJA001, un farmaco cannabinoide orale mirato al Disturbo dello Spettro Autistico (ASD). L'azienda prevede di avviare un trial clinico di Fase 2 entro metà del 2025, arruolando 60 pazienti di età compresa tra i 13 e i 29 anni per uno studio della durata di 12 settimane.
Il mercato dell'ASD è valutato a quasi 4 miliardi di dollari negli Stati Uniti, con un CAGR superiore al 4%. Attualmente, esistono solo due trattamenti approvati, entrambi antipsicotici atipici con effetti collaterali significativi e scarsa aderenza da parte dei pazienti.
AJA001 è stato sviluppato attraverso una collaborazione tra Ajna BioSciences e Charlotte's Web, sfruttando un decennio di dati osservazionali di Charlotte's Web e genetiche esclusive di canapa. L'azienda prevede anche di avviare uno studio pediatrico di Fase 2 a etichetta aperta in Australia nel 2025.
DeFloria ha recibido la aprobación de la FDA para su solicitud de Nuevo Medicamento en Investigación (IND) para AJA001, un medicamento cannabinoide oral dirigido al Trastorno del Espectro Autista (ASD). La compañía planea iniciar un ensayo clínico de Fase 2 para mediados de 2025, inscribiendo a 60 pacientes de entre 13 y 29 años para un estudio de 12 semanas.
El mercado del ASD está valorado en casi 4 mil millones de dólares en EE.UU., con un CAGR superior al 4%. Actualmente, solo existen dos tratamientos aprobados, ambos antipsicóticos atípicos con efectos secundarios significativos y pobre adherencia por parte de los pacientes.
AJA001 fue desarrollado a través de una colaboración entre Ajna BioSciences y Charlotte's Web, aprovechando una década de datos observacionales de Charlotte's Web y genética de cáñamo patentada. La compañía también planea iniciar un estudio pediátrico de Fase 2 en Australia en 2025.
DeFloria는 자폐 스펙트럼 장애(ASD)를 목표로 하는 경구용 칸나비노이드 약물 AJA001에 대한 임상 신약 신청(IND)이 FDA 승인을 받았습니다. 이 회사는 2025년 중반까지 2상 임상 시험을 시작할 계획이며, 13세에서 29세 사이의 60명의 환자를 모집하여 12주간 연구를 진행할 예정입니다.
ASD 시장은 미국에서 거의 40억 달러로 평가되며, 연평균 성장률(CAGR)은 4% 이상입니다. 현재 승인된 치료법은 두 가지뿐이며, 둘 다 상당한 부작용과 낮은 환자 순응도를 가진 비정형 항정신병 약물입니다.
AJA001은 Ajna BioSciences와 Charlotte's Web 간의 협력을 통해 개발되었으며, Charlotte's Web의 10년간의 관찰 데이터와 독점적인 헴프 유전자를 활용했습니다. 이 회사는 또한 2025년에 호주에서 2상 공개 소아 연구를 시작할 계획입니다.
DeFloria a reçu l'approbation de la FDA pour sa demande de Nouveau Médicament Investigational (IND) pour AJA001, un médicament cannabinoïde oral ciblant le Trouble du Spectre Autistique (ASD). L'entreprise prévoit de lancer un essai clinique de Phase 2 d'ici mi-2025, en recrutant 60 patients âgés de 13 à 29 ans pour une étude de 12 semaines.
Le marché de l'ASD est évalué à près de 4 milliards de dollars aux États-Unis, avec un taux de croissance annuel composé (CAGR) supérieur à 4%. Actuellement, seuls deux traitements approuvés existent, tous deux étant des antipsychotiques atypiques avec des effets secondaires significatifs et une faible adhésion des patients.
AJA001 a été développé grâce à une collaboration entre Ajna BioSciences et Charlotte's Web, tirant parti d'une décennie de données d'observation de Charlotte's Web et de génétique de chanvre propriétaire. L'entreprise prévoit également de lancer une étude pédiatrique ouverte de Phase 2 en Australie en 2025.
DeFloria hat die Genehmigung der FDA für seinen Antrag auf einen neuen investigativen Arzneimittel (IND) für AJA001 erhalten, ein orales Cannabinoid-Medikament, das auf Autismus-Spektrum-Störungen (ASD) abzielt. Das Unternehmen plant, bis Mitte 2025 eine Phase-2-Studie zu starten, in der 60 Patienten im Alter von 13 bis 29 Jahren für eine 12-wöchige Studie rekrutiert werden.
Der ASD-Markt wird in den USA auf fast 4 Milliarden Dollar geschätzt, mit einer über 4% CAGR. Derzeit gibt es nur zwei zugelassene Behandlungen, beide atypische Antipsychotika mit erheblichen Nebenwirkungen und geringer Patientenadhärenz.
AJA001 wurde in Zusammenarbeit zwischen Ajna BioSciences und Charlotte's Web entwickelt, wobei auf ein Jahrzehnt an Beobachtungsdaten von Charlotte's Web und proprietären Hanfgenetik zurückgegriffen wurde. Das Unternehmen plant auch, 2025 eine offene Phase-2-Studie bei Kindern in Australien zu starten.
- FDA clearance received for Phase 2 clinical trial
- Targeting $4 billion U.S. market with >4% CAGR
- Addressing underserved market with only two current treatment options
- Planned expansion with additional pediatric study in Australia
- Phase 2 trial won't start until mid-2025
- initial trial size of 60 patients
- Early-stage drug development with no guaranteed success
DeFloria plans to initiate a Phase 2 clinical trial by mid-year 2025
With as many as 1 in 36 children diagnosed, ASD represents a growing and underserved market valued at nearly
"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
"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
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
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Investor Contact:
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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,
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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SOURCE DeFloria, LLC
FAQ
When will DeFloria start the Phase 2 trial for AJA001 (CWBHF)?
How many patients will be enrolled in AJA001's Phase 2 trial (CWBHF)?
What is the market size for Autism Spectrum Disorder treatment that CWBHF is targeting?
How long will the Phase 2 trial for AJA001 (CWBHF) last?
Will CWBHF conduct additional trials for AJA001 outside the US?
