Pharmaceutical-grade cannabidiol (CBD) oral solution made by INSYS Therapeutics, Inc. (INSY), a leader in the development, manufacture and commercialization of pharmaceutical cannabinoids and spray technology, was generally well-tolerated—even at high doses—as an investigational treatment for refractory pediatric epilepsy, according to new clinical data from a long-term safety study presented yesterday at the American Epilepsy Society (AES) 2018 annual meeting.

“The results of this study support the promising safety profile of CBD oral solution from INSYS in the context of treating pediatric patients with intractable seizures due to epilepsy,” said James W. Wheless, M.D., professor and chief of pediatric neurology at the University of Tennessee Health Science Center in Memphis, where he directs the Neuroscience Institute & Le Bonheur Comprehensive Epilepsy at Le Bonheur Children’s Hospital and holds the Le Bonheur Chair in Pediatric Neurology. “In addition, these long-term safety data are consistent with those from other studies of CBD in refractory pediatric epilepsy.”

Dr. Wheless presented the results of this multicenter, open-label, flexible-dose study (INS011-14-030) during AES Poster Session 3. The presentation (Abstract 3.294) reported safety data from 52 patients (nine infants, 26 children and 17 adolescents) from 11 U.S. clinical sites.

A total of 45 patients completed the study, which was designed to assess the long-term safety and tolerability of the company’s proprietary formulation of CBD oral solution as adjunctive treatment for pediatric patients (ages 1–17 years old) with treatment-resistant epilepsy. Patients received doses of CBD oral solution at 10, 20, or 40 mg/kg/day (in two divided doses) for approximately 48 weeks, with dose changes at the investigators’ discretion. Defined as the dose with the longest duration, the mean modal dose was 24.42 mg/kg/day (higher in infants than in children and adolescents); and the mean total duration of study drug was 311.1 days (with a maximum exposure of 384 days).

Safety assessments included treatment-emergent adverse events (TEAEs), electrocardiograms, physical and neurologic exams and lab tests. Additional assessments included Clinical Global Impression of Severity (CGI-S) and Clinical Global Impression of Improvement (CGI-I) by investigator and caregiver at baseline (CGI-S only), Week 4, 8, 12, 24 and 36, final visit and follow-up visit.

Through 48 weeks of follow up, 47 patients (90.4 percent) experienced one or more TEAEs during the study; and 24 patients (46.2 percent) had TEAEs that were possibly or probably related to the study drug, most being mild or moderate in intensity. A total of 278 TEAEs were reported, with the following reported in at least five subjects: seizure, upper respiratory tract infection, somnolence, anemia, diarrhea and pyrexia. Of the 37 serious adverse events reported, three were considered possibly related to the study drug: moderate change in seizure presentation; moderate ataxia; and worsening seizure exacerbation.

Eligibility criteria included participation in a previous CBD study (specifically, INS011-14-029 or INS011-15-054). Of the 61 patients that enrolled in one of these studies, 25 continue to receive CBD from INSYS as part of an expanded access program that ensures continued supply of this investigational drug.

The company’s clinical research on CBD, a non-psychoactive component of cannabis, includes three ongoing trials: childhood absence epilepsy (Phase 2), infantile spasms(Phase 3) and Prader-Willi syndrome (Phase 2). Results from the Phase 2 trial in childhood absence epilepsy are expected soon, pending completion of enrollment. All three trials are actively enrolling patients.

In addition, INSYS has formed collaborative research partnerships with leading academic institutions to advance the medical science of pharmaceutical cannabinoids in other disease states, including autism, early psychosis and anxiety in anorexia nervosa with the University of California San Diego’s Center for Medicinal Cannabis Research, and cocaine addiction with the University of Montreal and the Canadian Institutes of Health Research.

About INSYS

INSYS Therapeutics is a specialty pharmaceutical company that develops and commercializes innovative drugs and novel drug delivery systems of therapeutic molecules that improve patients’ quality of life. Using proprietary spray technology and capabilities to develop pharmaceutical cannabinoids, INSYS is developing a pipeline of products intended to address unmet medical needs and the clinical shortcomings of existing commercial products. INSYS is committed to developing medications for potentially treating anaphylaxis, epilepsy, Prader-Willi syndrome, opioid addiction and overdose, and other disease areas with a significant unmet need.