Issued by the U.S. Patent and Trademark Office under the title “Treatment of Fragile X Syndrome with Cannabidiol,” the patent includes claims to treat fragile X syndrome by administering a therapeutic dose of the synthetic or purified cannabidiol. ZYN002 remains under patent law until 2038.
The patent comes while enrollment for the pivotal Phase 2/3 clinical trial, called CONNECT-FX continues. This trial will assess the safety and effectiveness of ZYN002 as a potential therapy to treat behavioral symptoms in people with fragile X syndrome.
Cannabis contains more than 100 pharmacologically active compounds (cannabinoids), with the most studied compounds being tetrahydrocannabinol (THC) and cannabidiol.
ZYN002 is a cannabidiol administered as a gel through the skin once or twice daily, allowing its direct absorption into the blood. This direct formulation allows patients to use lower doses than a pill, and reduces the risk of side effects.
The CONNECT-FX trial (NCT03614663) is looking to recruit 204 children ages 3-17, who have been diagnosed with fragile X and carry a full mutation of the FMR1 gene, from approximately 20 locations in Australia, New Zealand, and the United States.
Participants will be randomized to treatment with ZYN002 (one of two doses) or a placebo, after classification by gender, weight, and investigator geographic region.
At the end of dosing, a 12-month extension study will be conducted, where all patients will receive the treatment.
The study’s primary objective is to assess changes from the beginning of the trial, or baseline, up to the end of the treatment period using the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance subscale. The ABC-C is a 58-item rating scale used to evaluate maladaptive behaviors, including irritability, hyperactivity, lethargy/withdrawal, repetitive acts, and inappropriate speech.
Additional objectives include measuring changes from baseline to the end of the treatment period in the ABC-CFXS Irritability subscale score, the ABC-CFXS Socially Unresponsive/Lethargic subscale score, and improvement in Clinical Global Impression — Improvement (CGI-I) at the end of the treatment period.
Topline results are expected during the second half of 2019.
Previous data from the Phase 2 trial, called FAB-C (“Treatment of Fragile X Syndrome Anxiety and Behavioral Challenges with CBD”), showed that one-year treatment with ZYN002 led to significant improvements in core emotional and behavioral symptoms.
Zynerba aims to extend the clinical development of ZYN002 transdermal gel to patients with autism spectrum disorder and 22q deletion syndrome in addition to its fragile X and developmental and epileptic encephalopathy programs.