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DeFloria advances cannabis-based autism drug through early trial

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DeFloria announced Thursday that its experimental cannabis-based drug for autism spectrum disorder showed positive early results, with patients tolerating the treatment well across multiple dose levels.

The drug, AJA001, being developed as a multi-compound hemp extract with a full spectrum of cannabinoids, demonstrated safety at daily doses up to 660 mg when given twice per day, according to results presented at a neuropsychopharmacology conference in Phoenix. The Phase 1 trial included 70 healthy volunteers ranging from 19 to 55 years old.

“We are extremely encouraged by these results,” Marcel O. Bonn-Miller, Ph.D., chief scientific officer at Charlotte’s Web Holdings Inc. and DeFloria board member, said in a statement. He noted that the drug achieved blood concentration levels matching or exceeding existing cannabis-based medicines like Epidiolex.

The Colorado-based company, formed in April as a joint venture between Charlotte’s Web (OTCQX: CWBHF), Ajna BioSciences and British American Tobacco (NYSE: BTI), aims to help address a significant unmet need. About 1 in 36 children may have autism spectrum disorder, according to recent CDC data cited by the company. BAT holds an equity stake through 200,000 preferred units following an initial $10 million investment, with Charlotte’s Web and Ajna each holding 400,000 voting common units.

Common side effects included drowsiness, anxiety, dizziness and headaches, DeFloria reported. One participant receiving a higher 906.4 mg dose experienced anxiety that resolved within a day.

The drug combines multiple cannabinoids from hemp plants, including carefully controlled amounts of CBD and THC. The trial results support testing doses containing up to 395 mg CBD and 15 mg THC daily in future studies.

“Currently available treatments for ASD are not well tolerated, making patient compliance a challenge,” said Jared Stanley, DeFloria’s CEO.

The company is preparing to submit the Phase 1 trial data as part of its Investigational New Drug application to the FDA. If approved, DeFloria plans to explore the drug’s potential for addressing behavioral symptoms and improving quality of life for individuals with autism spectrum disorder in two Phase 2 trials starting in the second quarter of 2025.

The treatment uses hemp extract from Charlotte’s Web’s proprietary CW1AS1 cannabis strain. If successful in later trials, AJA001 could become one of the first FDA-approved botanical drugs derived from hemp for autism spectrum disorder.

“DeFloria’s Phase 1 results validate our strategic vision of advancing botanical science through pharmaceutical pathways,” said Bill Morachnick, CEO of Charlotte’s Web. “This milestone demonstrates how our proprietary genetics and expertise in hemp cultivation can create significant value beyond our core wellness business.”



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autism

Study: Early cannabis use in pregnancy doesn’t predict autism

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Cannabis use during pregnancy has been increasing in the US, and many people may be wondering what impacts this trend could have on children. Some previous research has pointed towards the possibility that prenatal cannabis exposure could increase the likelihood of having autistic children. But a recent study—one of the largest ever conducted on cannabis and autism—found that cannabis exposure in early pregnancy did not lead to higher odds of an autism diagnosis.

What is autism?

Autism is classified as a developmental disorder. It impacts how individuals interact with others, communicate, learn, and experience life. One in 36 Americans are diagnosed with autism; symptoms usually appear during the first two years of a person’s life. Autism is also a spectrum condition—it encompasses a wide variety of different symptoms that an individual might experience, and different levels of health care support may be needed.

Some of the challenges that autistic people might face include having a style of communication that non-autistic people have trouble understanding, having sensory issues because of heightened or reduced sensory perception, and having more repetitive or restricted patterns of behavior than non-autistic peers.

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While medical literature classifies autism as a disorder, many autistic advocates point out that many of the “symptoms” of autism are not inherently problematic or disordered; they simply clash with non-autistic expectations, leaving autistic people marginalized. 

Previous research on cannabis exposure and autism

The recent study, conducted by researchers from Kaiser Permanente and UC San Francisco, evaluated whether cannabis use in early pregnancy could increase the likelihood of a woman giving birth to an autistic child. Autism has been linked to a variety of potential environmental causes, but most research points towards genetics as the primary cause.  

One study, published in 2020, did find an association between prenatal cannabis use and autism, leading to questions about whether cannabis use could cause autism. However, otherstudies found no associations. Unfortunately, those studies utilized self-reporting methods, which may have skewed the results due to a potential underreporting of cannabis use. 

Researchers in the recent Kaiser / UCSF used more rigorous methods to determine whether prenatal cannabis use caused higher rates of autism in children.

Studying prenatal cannabis and autism

This recent study was one of the largest ever done on the topic (it included 178,948 pregnancies), but it also utilized a more robust data set for cannabis use and autism.

This recent study was one of the largest ever done on the topic (it included 178,948 pregnancies), but it also utilized a more robust data set for cannabis use and autism. The study incorporated information from Kaiser Permanente Northern California’s integrated health-care system, which universally screens pregnant individuals for cannabis use—via both self-reporting and a urine toxicology test. Kaiser also routinely screens and assesses children for autism.

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The researchers also included related factors in their analysis like sociodemographic data, the additional medications and substances that a mother consumes, and general health conditions.

Prenatal cannabis use not associated with autism

While an initial analysis appeared to indicate an association between autism and prenatal cannabis use, the association disappeared after adjusting for factors like sociodemographic characteristics and the medical conditions of the mother.

This lack of association suggests that mothers who use cannabis during pregnancy are more likely to have autistic children—not because of the cannabis itself, but because of other factors that also increase likelihood of cannabis use. 

While the researchers don’t identify those specific factors, these results shouldn’t be that surprising: For one, Autism has high hereditary rates. Furthermore, cannabis is sometimes used as an effective treatment for symptoms of autism. Separate studies have noted that autistic individuals tend to have lower levels of endocannabinoids in their bodies, which could suggest an impairment of the endocannabinoid system. 

Mothers with autism—or other conditions tied to the same genes as autism—are more likely to both use cannabis and have autistic children; this correlation may help explain the initial results of the study. Still, only more research will offer conclusive answers to these questions. 

Limitations and future research

This study used a strong methodology, with a large and diverse data set. Still, it was limited by the fact that cannabis use was only measured in early pregnancy. Future studies should measure cannabis use throughout pregnancy, and ideally include information like the methods and dosing of the cannabis itself.

While this study suggests prenatal cannabis use is not associated with autism, the authors nonetheless caution that there is evidence that cannabis can impact development in other ways and should be avoided during pregnancy. 



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