Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
ZYN002 · 2 trials · 3 indications
The ABC-C FXS is a standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials
An adverse event (AE) is an undesirable medical occurrence or worsening of a pre-existing medical condition that occurs at any time after signing of the informed consent form whether or not it is considered to be related to treatment. Any AE that results in one or more of the following is considered a SAE: death, life threatening, in-patient hospitalization, persistent or significant disability/incapacity, congenital abnormality or birth defect, and other medically important events. TEAEs are defined as AEs with onset dates on or after the start of study drug. Adverse event data were collected as a single arm because, based on pharmacokinetics modeling, exposure is consistent between doses.
| Arm | Type | Description |
|---|---|---|
| ZYN002 | EXPERIMENTAL | Pharmaceutically manufactured cannabidiol, which is formulated as a clear gel for transdermal delivery. |
| Placebo | PLACEBO_COMPARATOR | Placebo is formulated as a clear gel for transdermal delivery. |
| Open-label | EXPERIMENTAL | Open-label |
| Name | Type | Description |
|---|---|---|
| ZYN002 | DRUG | Pharmaceutically manufactured cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery) |
| Placebo | DRUG | Placebo formulated as a clear gel that can be applied to the skin (transdermal delivery) Other Names: Placebo Comparator Matching Placebo |
Inclusion Criteria: * Male or female children and adolescents aged 3 to \< 30 years, at the time of Screening. * Patient resides with caregiver who will continue to provide consistent care throughout the study. * Judged by the Investigator to be in generally good health at Screening based upon the ...
ZYN002 is an investigational small molecule being studied for Fragile X Syndrome and 22Q11.2 Deletion Syndrome, both rare diseases. It is administered as a transdermal gel. The drug is not approved and remains in clinical development.
ZYN002 is being developed by Harmony Biosciences Holdings, Inc., traded on the NASDAQ under the ticker HRMY. The company is conducting clinical trials of the drug in Fragile X Syndrome and 22Q11.2 Deletion Syndrome.
ZYN002 is in Phase 2 clinical development for 22Q11.2 Deletion Syndrome. A Phase 3 trial in Fragile X Syndrome has been completed. The drug is investigational and not yet approved by regulatory authorities.
ZYN002 has been studied in two completed trials. NCT04977986 was a Phase 3 study in Fragile X Syndrome with 257 participants. NCT05149898, called INSPIRE, was a Phase 2 open-label study in 22Q11.2 Deletion Syndrome with 20 participants.
ZYN002 is a transdermal gel formulation of cannabidiol. The Phase 3 trial NCT04977986 is titled 'Clinical Study of Cannabidiol in Children, Adolescents, and Young Adults With Fragile X Syndrome,' indicating the drug's active ingredient is cannabidiol.