Our development pipeline includes two lead product candidates, ZYN002 and ZYN001, which are currently being evaluated in a number of rare (affecting fewer than 200K patients in the U.S.) and near-rare (affecting fewer than one million people) neuropsychiatric disorders.

ZYN002 is the first and only pharmaceutically-produced CBD, a non-psychoactive cannabinoid, formulated as a patent-protected permeation-enhanced gel for transdermal delivery through the skin and into the circulatory system. ZYN002 being developed for patients suffering from Fragile X syndrome (FXS) and certain refractory epilepsies. The FDA has granted Zynerba Orphan Drug designation for the use of CBD as treatment of patients with FXS.

ZYN001, a pro-drug of THC that enables transdermal delivery through the skin and into the circulatory system via a patch, is currently in Phase 1 clinical studies.

Development Status and Upcoming Milestones

These timelines are subject to change due to regulatory, clinical and other considerations.

 Upcoming Milestones

Product Study  Key Milestone Timing
ZYN002 Fragile X syndrome FDA meeting to discuss pivotal program Complete
Initiation of pivotal program Mid-year 2018
Presentation/publication of additional Phase 2 FAB-C and open label data 2018
 Other neuropsychiatric indications Assessment of other rare and near-rare indications 2018
Refractory epilepsies Initiation of Phase 2 open-label study in DEE 1H 2018
Initiation of new Phase 2B study in adult refractory focal epilepsy 2H 2018
Presentation/publication of additional data from STAR 2 extension 2018
ZYN001 Safety and PK Completion of Phase 1 formulation studies 1H 2018
Tourette Syndrome Initiation of Phase 2 study 2H 2018
Other neuropsychiatric indications Assessment of other rare and near-rare indications 2018


Cannabinoids are a class of compounds derived from cannabis plants. The two primary cannabinoids contain in cannabis are cannabidiol (CBD) and Δ9-tetrahydrocannabinol (THC).

Cannabinoid  Cannabidiol  Δ9-Tetrahydrocannabinol
 Activity Non-psychoactive, multiple mechanisms Psychoactive, antinociceptive,
agonist of CB1 and CB2 receptors
Indications Being Developed Fragile X Syndrome
Refractory epilepsies:
– Adult refractory focal epilepsy
– Developmental and Epileptic Encephalopathies (DEE)
Tourette Syndrome
Potential Future Indications Autism spectrum disorders
Cognitive disorders
Memory disorders
Rare and near-rare neuralgias
Zynerba Asset CBD Gel – ZYN002 THC Pro-Drug Patch – ZYN001
Patent Protection 2030 2031

THC and CBD Safety

Clinical data suggest that THC and CBD have a high therapeutic index with low toxicity.1 In the nabiximols clinical program, dizziness and fatigue were considered very common (> 1/10) adverse events. These occurred in the first four weeks of exposure were mild to moderate and resolved within a few days with continued treatment.2  Nabiximols are not recommended for use in children or adolescents below 18 years of age due to lack of safety and efficacy data. In non-clinical studies, effects were observed at exposures considered in excess of human exposure suggesting little relevance to clinical use.2


Fine, PG, Rosenfeld, MJ. The Endocannabinoid System, Cannabinoids, and Pain. Rambam Maimodies Med J 2013;4 (4)E0022.

2. Sativex Summary of Product Characteristics, UK 2014.