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Zynerba Pharmaceuticals Provides 2018 Clinical and Corporate Update
- Focusing Strategy on Rare and Near-Rare Neurological and Psychiatric Disorders -
- Pediatric Neurologist Liza Squires, M.D. Joins as Chief Medical Officer -
- Conference Call to be Held at
“The decision to concentrate on rare and near-rare neurological and psychiatric disorders is driven by a number of important factors, including the compelling ZYN002 clinical data seen to date in Fragile X syndrome and refractory focal epilepsy and by the opportunities for a more efficient and rapid development and regulatory approval process,” said
2018 Anticipated Milestones
“As we head into 2018, we look forward to delivering on a number of significant corporate and clinical milestones that are important to our investors and the patients we seek to treat,” said Anido. “We view 2018 as a year of corporate evolution for Zynerba. We plan to initiate a pivotal study for ZYN002 in FXS that should read out in 2019, initiate two new Phase 2 studies in refractory epilepsies, and initiate a Phase 2 program with ZYN001 in Tourette Syndrome. We also will continue to collect a significant amount of important data from our ongoing open-label extension studies in Fragile X syndrome and adult refractory focal epilepsy that we expect to present and/or publish during the year.”
ZYN002 Development Plans
Zynerba is currently developing ZYN002, the first and only patent-protected, pharmaceutically-produced CBD that is formulated as a permeation-enhanced gel for transdermal delivery.
- The Company has a meeting scheduled with the
U.S. Food and Drug Administrationduring the first quarter of 2018 to discuss development strategy including a pivotal clinical program for ZYN002 in FXS, an inherited autism spectrum disorder affecting approximately 71,000 patients in the U.S. The Companyexpects to initiate the pivotal, double-blind placebo controlled clinical trial mid-year 2018. Zynerba has received U.S. Orphan Drug designation for the use of CBD as treatment of FXS;
- The Company expects to initiate an open-label study of ZYN002 in developmental and epileptic encephalopathies (DEE), a category of rare and ultra-rare, severe brain disorders manifesting with seizures or EEG abnormalities that can directly worsen cognition or behavior. The study will initiate in the first half of 2018 and will enroll approximately 48 pediatric and adolescent patients. It is designed to identify new indications to take into blinded, placebo-controlled studies;
- Zynerba intends to initiate a Phase 2B double-blind placebo controlled clinical trial of ZYN002 in adult refractory focal epilepsy, a subcategory of epilepsy impacting approximately 500,000 patients who are refractory to other anti-epileptic drugs (AED), in the second half of 2018. The design of this study will be modified from the STAR 1 trial design based on the data presented at the 2017
American Epilepsy Societymeeting and additional analysis of the STAR 1 and STAR 2 data. Changes to the trial design based on these data are expected to include an increase in the number of patients enrolled, changes in randomization methodologies utilized, an increase in baseline seizure frequency and an increase in the length of the study;
- The Company will continue to generate important open label data in the ongoing 12-month FAB-C extension study in pediatric and adolescent patients with FXS and the ongoing STAR 2 18-month extension study in adult refractory focal epilepsy and plans on presenting/publishing updates in 2018;
- Zynerba is assessing the potential for ZYN002 in other rare and near-rare neuropsychiatric indications;
- Zynerba is discontinuing its investment into the capital-intensive pain space, due to inconsistency with its rare and near-rare neuropsychiatric focus. As a result, although the initial Phase 2 STOP data were encouraging, the Company has discontinued investment into the ZYN002 osteoarthritis program.
ZYN001 Development Plans
Zynerba is currently in Phase 1 development of ZYN001, the Company’s patent-protected, pro-drug of tetrahydrocannabinol (THC) delivered via a transdermal patch.
- The Company expects to complete its Phase 1 evaluation of multiple formulations of ZYN001 in the first half of 2018;
- The Company expects to take ZYN001 into a Phase 2 clinical trial in Tourette Syndrome (TS), a neurodevelopmental disorder characterized by motor and vocal tics, late in the second half of 2018.
The National Institute of Neurological Disorders and Strokeestimate that 200,000 Americans have the most severe form of TS, and as many as one in 100 exhibit milder and less complex symptoms such as chronic motor or vocal tics. Two third-party controlled Phase 2 trials of THC in Tourette Syndrome have shown significant reduction in tics compared to placebo;
- Zynerba is assessing the potential for ZYN001 in other rare and near-rare neuropsychiatric indications;
- The Company is discontinuing its investment into the capital-intensive pain space, including its pursuit of fibromyalgia and general peripheral neuropathic pain (PNP) indications for ZYN001, due to inconsistency with its rare and near-rare neuropsychiatric focus.
Appointment of new Chief Medical Officer
To complement this concentration in rare and near-rare neuropsychiatric disorders,
“I’m pleased to announce the addition of Dr.
Zynerba believes it can fund operations and capital requirements well into 2019. As of
Conference call information
Zynerba management will host a live conference call and multimedia webcast tomorrow,
Zynerba’s ZYN002 CBD gel is the first and only pharmaceutically-produced CBD formulated as a patent-protected permeation-enhanced gel and is being studied in children with Fragile X syndrome and in refractory epilepsies. ZYN002 is a clear, permeation-enhanced gel that is designed to provide controlled drug delivery transdermally with once- or twice-daily dosing.
ZYN001 is a synthetic pro‑drug of THC in a state-of-the-art drug-adhesive matrix transdermal patch, currently in Phase 1 clinical development. THC is a CB1 agonist which acts at many sites along the central cannabinoid receptor system, and has been shown to be effective in the treatment of motor and vocal tics. A pro‑drug is a drug administered in an inactive or less active form and designed to enable more effective delivery, which is then converted into an active form through a normal metabolic process.
About Our Technology
Cannabinoids are a class of chemical compounds found in the Cannabis plant. The two primary cannabinoids contained in Cannabis are cannabidiol, or CBD, and ∆9-tetrahydrocannabinol, or THC. Clinical and preclinical data support the potential for CBD in treating epilepsy, Fragile X Syndrome and other neuropsychiatric disorders, and THC has positive effects on treating tics. Zynerba is developing therapeutic medicines that utilize innovative transdermal technologies that, if successful, may allow for sustained and controlled delivery of therapeutic levels of CBD and THC. Transdermal delivery of cannabinoids may have benefits over oral dosing because it allows the drug to be absorbed through the skin directly into the bloodstream. This avoids first-pass liver metabolism, potentially enabling lower dosage levels of active pharmaceutical ingredients with a higher bioavailability and improved safety profile. Transdermal delivery also avoids the gastrointestinal tract, lessening the opportunity for GI related adverse events and the potential degradation of CBD by gastric acid into THC, which may be associated with unwanted psychoactive effects. Using an established chemical pharmaceutical process for manufacturing, Zynerba replicates the CBD and THC found in the Cannabis plant. We believe that this will allow us to meet stringent global regulatory agencies’ standards while ensuring that we can efficiently supply the amount of product required to meet the demand of the markets that we are targeting.
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