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Zynerba Pharmaceuticals Announces Positive Top Line Results from ZYN002 CBD Gel Phase 1 Multiple Rising Dose Trial and Initiation of Phase 2 Study in Adult Epilepsy Patients with Refractory Focal Seizures
Top line results from the 24 healthy volunteers ranging from 25 to 53 years old and 12 epilepsy patients from 19 to 65 years old demonstrated that ZYN002 CBD gel was safe and well-tolerated at all dose levels. The PK findings are being used to establish the high and low doses for the Phase 2 studies. The twice daily dosing provided a more favorable PK profile with comparable results between healthy volunteers and epilepsy patients.
Transdermal application of ZYN002 was very well tolerated with minimal skin erythema. Skin dryness at the application site was common for both ZYN002 and placebo gel. Overall, the incidence of adverse events associated with ZYN002 CBD gel was similar to placebo in both healthy volunteers and adult epilepsy patients. There were no reports of somnolence or fatigue and a very low incidence of gastrointestinal events was observed. There were no serious adverse events or discontinuations for healthy volunteers and epilepsy patients receiving ZYN002 CBD gel. One healthy volunteer receiving placebo gel developed a serious adverse event suspected to be a catheter infection and was discontinued from the study.
In addition, healthy volunteers and epilepsy patients had no drug related changes in performance on the Trail Making Test, a test of visual attention, psychomotor ability, and task switching; a divided attention task; and the Paced Auditory Serial Addition Task (PASAT), a test that measures working memory and focused attention. These results indicate that ZYN002 did not produce impairment in critical areas of cognitive functioning often impacted by CNS drugs. No changes in mood symptoms as accessed by the Inventory of Depression and Anxiety Symptoms (IDAS) and the Positive and Negative Affect Schedule (PANAS) were observed for ZYN002 indicating that ZYN002 is not associated with declines in psychological health.
Zynerba also announced the results in epilepsy patients in the single rising dose trial. The results demonstrated that ZYN002 is safe and well tolerated in patients with epilepsy. The incidence of adverse events associated with ZYN002 was similar to placebo and similar to the healthy volunteers.
“We are extremely encouraged by these results from our Phase 1 multiple rising dose trial, which are consistent with the results of the single rising dose trial, and reinforce that ZYN002 CBD gel is well-tolerated across a range of doses,” said
Mr. Anido continued, “In addition to STAR 1 in refractory epilepsy, we also expect to initiate two Phase 2 trials for ZYN002 CBD gel in osteoarthritis and Fragile X Syndrome during the second half of 2016. We expect to report top line results from all three studies in the first half of 2017.”
More About the STAR 1 Study
The STAR 1 (Synthetic Transdermal Cannabidiol for the Treatment of Epilepsy) clinical study is a Phase 2 multi-center, double-blind, placebo-controlled, multi-dose clinical trial designed to evaluate the efficacy and safety of ZYN002 in patients with refractory focal seizures. Approximately 180 patients will be enrolled in the trial and will be followed for 8 weeks during the baseline phase. After the baseline phase, patients will be randomized (1:1:1) to receive one of two doses of ZYN002 or placebo for 12 weeks. The primary endpoint of the study is median percentage change in seizure frequency at 12 weeks. The Company expects to commence dosing in STAR1 during the third quarter of 2016, with preliminary results expected in the first half of 2017.
About ZYN002 CBD Gel
Zynerba’s ZYN002 CBD gel is the first and only synthetic CBD formulated as a patent-protected permeation-enhanced gel and is being studied in refractory epilepsy, Fragile X Syndrome and osteoarthritis. ZYN002 is a clear, permeation-enhanced gel that is designed to provide consistent, controlled drug delivery transdermally with convenient twice-daily dosing. Transdermal therapeutics are absorbed through the skin directly into the systemic circulation, avoiding first-pass liver metabolism and potentially enabling lower dosage levels of active pharmaceutical ingredients and rapid and reliable absorption with high bioavailability. In addition, transdermal delivery avoids the gastrointestinal tract and potential stomach acid degradation of CBD into THC (associated with psychoactive effects), as demonstrated in a Zynerba in vitro study.
Epilepsy is a disease characterized by an enduring predisposition to generate epileptic seizures (transient symptoms due to abnormal neuronal activity in the brain) and by the neurobiological, cognitive, psychological and social consequences of the condition. Focal seizures usually start in a small area of the temporal lobe or frontal lobe of the brain and quickly involve other areas of the brain that affect alertness and awareness. Approximately 2.2 million patients in
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the Company’s current expectations. For example, there can be no guarantee that the Company will obtain approval for ZYN002 or ZYN001 from the U.S. Food and Drug Administration (
Richard Baron, CFO Zynerba Pharmaceuticals484.581.7505 firstname.lastname@example.org Kimberly Minarovich Argot Partners212.600.1902 email@example.com Media Contact Eliza Schleifstein Argot Partners917-763-8106 firstname.lastname@example.org