Our development efforts are targeting multiple neuropsychiatric conditions with our lead product candidate, ZYN002, which is 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 is being developed for patients suffering from Fragile X syndrome (FXS) and certain refractory epilepsies including developmental and epileptic encephalopathies (DEE). The FDA has granted Zynerba Orphan Drug designation for the use of CBD as treatment of patients with FXS.

Development Status and Expected 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 Initiation of CONNECT-FX, a pivotal trial in patients three to 17 years of age Complete
Presentation/publication of additional Phase 2 FAB-C and open label data Presented at 16th NFXF International Fragile X Conference; additional opportunities in 2018 and 2019
Present top-line pivotal CONNECT-FX data 2H 2019
Developmental and Epileptic Encephalopathies (DEE) Initiation of Phase 2 open-label study in DEE Complete
Present top-line Phase 2 BELIEVE-1 data 2019
Adult Refractory Focal Epilepsy Initiation of new Phase 2B study in adult refractory focal epilepsy 2H 2018
Presentation/publication of additional data from STAR 2 extension Presented at AAN; additional opportunities in 2018
Other neuropsychiatric indications Assessment of other rare and near-rare indications 2018 and 2019


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

CBD Safety

Clinical data suggest that CBD has a high therapeutic index with low toxicity.1 Zynerba’s ZYN002 safety database across all studies is comprised of data from approximately 600 volunteers and patients. Across these studies, ZYN002 has been well tolerated, consistent with previously reported clinical data.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.