Patient & Caregivers

Angelman syndrome (AS) is a complex, heterogeneous, and severe life-long neurodevelopmental disorder which includes developmental delay, sleep disorders, speech and motor impairment, anxiety, as well as electroencephalography (EEG) abnormalities and seizures.
The frequency, severity, and pharmacological intractability of the seizures exact a heavy toll on individuals with AS and their caregivers. It is estimated that the US currently has around 30,000-33,000 AS patients. Despite the considerable impact on individuals and caregivers, no Therefore, there is urgent clinical need for better control of seizures while simultaneously improving other symptoms in AS.

Cannabidiol (CBD), a major phytocannabinoid constituent of cannabis, is gaining attention for its antiepileptic, anxiolytic, and antipsychotic properties. Recent anecdotal reports, accumulating animal model data, and mechanistic insights suggest that CBD provides a viable treatment opportunity for core pathologies of AS, which include but are not limited to behavioral deficits, EEG abnormalities, and seizures.

The potential medicinal effects of CBD hold promise for the simultaneous amelioration of behavioral deficits, EEG abnormalities, and seizures in AS. Maternal Ube3a deficient mice (Ube3am–/p+, AS model mice) have abnormal phenotypes that resemble many of the clinical symptoms observed in AS, thereby providing us an opportunity to evaluate possible therapeutic strategies.

Biom Therapeutics has developed BIO017 as treatment for AS, and in a well-defined preclinical study published in reputed Journal of Clinical Investigation we show that BIO017 (100 mg/kg) treatment attenuated hyperthermia- and acoustically induced seizures in a mouse model of AS. Further, BIO017 has shown positive human safety data in phase 2 studies as a treatment against epileptic patients. Biom is planning to initiate for the pivotal randomized controlled study by Q2 of 2021.

Cannabidiol (CBD), a major phytocannabinoid constituent of cannabis, is gaining attention for its antiepileptic, anxiolytic, and antipsychotic properties. Recent anecdotal reports, accumulating animal model data, and mechanistic insights suggest that CBD provides a viable treatment opportunity for core pathologies of AS, which include but are not limited to behavioral deficits, EEG abnormalities, and seizures.

The potential medicinal effects of CBD hold promise for the simultaneous amelioration of behavioral deficits, EEG abnormalities, and seizures in AS. Maternal Ube3a deficient mice (Ube3am–/p+, AS model mice) have abnormal phenotypes that resemble many of the clinical symptoms observed in AS, thereby providing us an opportunity to evaluate possible therapeutic strategies.

Biom Therapeutics has developed BIO017 as treatment for AS, and in a well-defined preclinical study published in reputed Journal of Clinical Investigation we show that BIO017 (100 mg/kg) treatment attenuated hyperthermia- and acoustically induced seizures in a mouse model of AS. Further, BIO017 has shown positive human safety data in phase 2 studies as a treatment against epileptic patients. Biom is planning to initiate for the pivotal randomized controlled study by Q2 of 2021.