Zynerba Pharmaceuticals Announces Publication of Preclinical Data in Neuropsychopharmacology Demonstrating Effect of Cannabidiol Gel Treatment in Reduction of Relapse in an Addiction Model

 

Results show effect of transdermally-delivered cannabidiol (ZYN002) in an animal model of drug relapse and extend its therapeutic potential to relapse prevention in substance use disorders

Devon, PA, March 26, 2018  — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), a clinical-stage specialty neuropsychiatric pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare and near-rare neurological and psychiatric disorders with high unmet medical needs, today announced that researchers at The Scripps Research Institute and the University of Maryland have generated preclinical data from a study funded by NIH grants that suggest potential value in using transdermal cannabidiol (CBD) (ZYN002) to reduce the risk of relapse among recovering drug and alcohol addicts. ZYN002, which was provided by the Company for use in the study, is the first and only pharmaceutically-produced CBD formulated as a patent-protected permeation-enhanced transdermal gel. The results have been published in a paper entitled, ‘Unique Treatment Potential of Cannabidiol for the Prevention of Relapse to Drug Use: Preclinical Proof of Principle’ (Gonzalez-Cuevas, G et al.) in Neuropsychopharmacology, the official publication of the American College of Neuropsychopharmacology (ACNP).

“Remaining drug-free is a constant battle for former substance abusers, and their susceptibility to stress and anxiety can increase their vulnerability for relapse,” said Dr. Friedbert Weiss, investigator for this study and Professor, Department of Neuroscience at The Scripps Research Institute in La Jolla, California. “These preclinical data are interesting as they show that we may be able to use transdermally-delivered CBD to impact multiple dimensions of relapse, namely benefit across a number of vulnerability states and effects that are maintained beyond the brief treatment period. CBD is broadly active and these data provide continued evidence that transdermal CBD may have therapeutic potential in a number of neurological disorders and conditions, including prevention of relapse.”

In summary, according to the authors, the findings in this publication provide proof of principle supporting the potential of transdermal CBD for relapse prevention along two dimensions: beneficial actions across several vulnerability states, and long-lasting effects with only brief treatment.

The publication noted that the clinical potential of CBD when administered orally is constrained to some extent by low bioavailability and potential for conversion into psychoactive cannabinoids in gastric fluid. The authors identified the transdermal route of administration as an effective delivery method that eliminates both of these limitations and produces stable and sustained plasma CBD levels. The researchers utilized a rat model of drug addiction (alcohol, cocaine) and addiction-like behavior to test ZYN002 administered once daily for one week. In this study, ZYN002 reduced relapse provoked by stress and drug cues, and reduced anxiety and impulsivity in the drug-experienced rats without sedative and nonspecific effects on motivation. Five months after therapy ended, treated animals still showed reduced relapse induced by stress or drug cues. According to the authors of the publication, the mechanisms by which CBD impacts these behaviors is not yet understood, but may be related to its ability to modulate the endocannabinoid system or its proneurogenic activity, both of which may explain the long-lasting attenuation of drug seeking and relapse and experimental anxiety.

About ZYN002
Zynerba’s ZYN002 CBD gel is the first and only pharmaceutically-produced CBD formulated as a patent-protected permeation-enhanced transdermal gel and is being studied in children and adolescents with Fragile X syndrome and developmental and epileptic encephalopathies, and in adult epilepsy patients with focal seizures. ZYN002 is a clear, permeation-enhanced gel that is designed to provide controlled drug delivery transdermally with once- or twice-daily dosing.

About Zynerba Pharmaceuticals, Inc. 
Zynerba Pharmaceuticals (NASDAQ:ZYNE) is a clinical-stage specialty pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare or near-rare neuropsychiatric diseases with high unmet medical needs. We are dedicated to improving the lives of people with severe health conditions by developing cannabinoid medicines designed to meet the rigorous efficacy and safety standards established by global regulatory agencies. Through the discovery and development of these potentially life-changing medicines, Zynerba seeks to improve the lives of patients battling severe, chronic health conditions including Fragile X syndrome, refractory epilepsies, Tourette Syndrome, and other neuropsychiatric disorders. Learn more at www.zynerba.com and follow the Company on Twitter at @ZynerbaPharma.

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 from the U.S. Food and Drug Administration (FDA) or foreign regulatory authorities; even if ZYN002 are approved, the Company may not be able to obtain the label claims that it is seeking from the FDA. In addition, the Company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated.  Management’s expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: the success, cost and timing of the Company’s product development activities, studies and clinical trials; the success of competing products that are or become available; the Company’s ability to commercialize its product candidates; the size and growth potential of the markets for the Company’s product candidates, and the Company’s ability to service those markets; the Company’s ability to develop sales and marketing capabilities, whether alone or with potential future collaborators; the rate and degree of market acceptance of the Company’s product candidates; and the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates. This list is not exhaustive and these and other risks are described in the Company’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at www.sec.gov. Any forward-looking statements that the Company makes in this press release speak only as of the date of this press release. The Company assumes no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Investor Contact
Will Roberts, VP Investor Relations and Corporate Communications
Zynerba Pharmaceuticals
484.581.7489
robertsw@zynerba.com

Zynerba Pharmaceuticals Announces Poster Presentation at the 2018 Annual Meeting of the American Academy of Neurology (AAN)

Devon, PA, March 22, 2018  — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), a clinical-stage specialty neuropsychiatric pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare and near-rare neurological and psychiatric disorders with high unmet medical needs, today announced the acceptance and presentation details of a late breaking poster at the 2018 Annual Meeting of the American Academy of Neurology (AAN) in Los Angeles, CA from April 21st through 27th, 2018.  The poster has been accepted to the Emerging Science session at the AAN.

Wednesday April 25, 2018
Poster title: “Transdermal Cannabidiol (CBD) Gel for the Treatment of Focal Epilepsy in Adults”
Session: Emerging Science – poster presentations
Poster session: IV
Poster number:  P4.468
Author presentation time: 5:30PM – 7:30PM EDT
Display time: 11:30AM – 7:00PM EDT

About Zynerba Pharmaceuticals, Inc. 
Zynerba Pharmaceuticals (NASDAQ:ZYNE) is a clinical-stage specialty pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare or near-rare neuropsychiatric diseases with high unmet medical needs. We are dedicated to improving the lives of people with severe health conditions by developing cannabinoid medicines designed to meet the rigorous efficacy and safety standards established by global regulatory agencies. Through the discovery and development of these potentially life-changing medicines, Zynerba seeks to improve the lives of patients battling severe, chronic health conditions including Fragile X syndrome, refractory epilepsies, Tourette Syndrome, and other neuropsychiatric disorders. Learn more at www.zynerba.com and follow the Company on Twitter at @ZynerbaPharma.

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.  These and other risks are described in the Company’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at www.sec.gov. Any forward-looking statements that the Company makes in this press release speak only as of the date of this press release. The Company assumes no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Zynerba Contact
Will Roberts, VP Investor Relations and Corporate Communications
484.581.7489
robertsw@zynerba.com

Media contact
Theresa Dolge
Tonic Life Communications
Office: 215-928-2748
Theresa.Dolge@toniclc.com

Zynerba Pharmaceuticals to Present at the Oppenheimer 28th Annual Healthcare Conference

Devon, PA, March 14, 2018 — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), a clinical-stage specialty neuropsychiatric pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare and near-rare neurological and psychiatric disorders with high unmet medical needs, today announced that Zynerba’s Chief Executive Officer, Armando Anidowill present a company overview at the Oppenheimer 28th Annual Healthcare Conference on Wednesday March 21, 2018 at 8:35 AM ET at the Westin New York Grand Central hotel in New York, NY.

A live webcast of the presentation will be accessible on the Investor Relations page of http://www.zynerba.com. A replay of the webcast will be available for 90 days following the conclusion of the event.

About Zynerba Pharmaceuticals, Inc. 
Zynerba Pharmaceuticals (NASDAQ:ZYNE) is a clinical-stage specialty neuropsychiatric pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare or near-rare neuropsychiatric disorders with high unmet medical needs. We are dedicated to improving the lives of people with severe health conditions by developing cannabinoid medicines designed to meet the rigorous efficacy and safety standards established by global regulatory agencies. Through the discovery and development of these potentially life-changing medicines, Zynerba seeks to improve the lives of patients battling severe, chronic health conditions including Fragile X syndrome, refractory epilepsies, Tourette Syndrome, and other neuropsychiatric disorders. Learn more at www.zynerba.com and follow the Company on Twitter at @ZynerbaPharma.

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. These and other risks are described in the Company’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at www.sec.gov. Any forward-looking statements that the Company makes in this press release speak only as of the date of this press release. The Company assumes no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Investor Contact
William Roberts, Vice President, Investor Relations and Corporate Communications
Zynerba Pharmaceuticals
484.581.7489
robertsw@zynerba.com

Zynerba Pharmaceuticals Reports Fourth Quarter and Year End 2017 Financial Results and Operational Highlights

Devon, PA, March 12, 2018 —Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), a clinical-stage specialty neuropsychiatric pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare and near-rare neurological and psychiatric disorders with high unmet medical needs, today reported financial results for the fourth quarter and year ended December 31, 2017 and provided an overview of recent operational highlights.

“We made significant corporate and clinical progress throughout 2017, and achieved our goal of utilizing data from three Phase 2 studies of ZYN002 to determine the direction and strategic focus of the Company,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We enter 2018 with strong momentum including having completed a positive meeting with the FDA to discuss our clinical path for ZYN002 in Fragile X syndrome. We have a refined development and commercialization strategy focused on rare and near-rare neuropsychiatric conditions, and the expectation of achieving numerous milestones in the coming year including initiations of the pivotal Fragile X syndrome study and Phase 2 studies in developmental and epileptic encephalopathies, adult refractory focal seizures, and Tourette Syndrome.”

Fourth Quarter 2017 and Recent Highlights

ZYN002 in Fragile X Syndrome (FXS)

Announced Positive Meeting with U.S. Food and Drug Administration and Plans to Conduct a Single Pivotal Study of ZYN002 in Fragile X Syndrome to Support a New Drug Application (NDA) Filing

The Company expects to initiate a pivotal study mid-year 2018 in approximately 200 pediatric and adolescent patients in the U.S., Australia and New Zealand to support an NDA for ZYN002 in FXS. The FDA and the Company are in agreement that the primary and key secondary endpoints for the study should assess observable behaviors in patients with FXS as reported by the caregiver using the validated Aberrant Behavior Checklist in Fragile X syndrome (ABC-FXS). If the pivotal trial meets its endpoints, approval for an indication encompassing the treatment of behavioral symptoms associated with Fragile X syndrome may be granted.

ZYN002 in Developmental and Epileptic Encephalopathies (DEE)

Announced Intent to Study ZYN002 in DEE; Initiation of Phase 2 Trial Expected in the First Half of 2018

DEE is a category of rare and ultra-rare, severe brain disorders manifesting with seizures or EEG abnormalities that can directly worsen cognition or behavior. The category affects ~45,000 patients in the U.S. and includes a number of syndromes, including Doose, Dravet, Lennox-Gastaut, and West, among others. The Phase 2 open label DEE study will enroll approximately 48 pediatric and adolescent patients and will help identify new indications to take into blinded placebo-controlled studies. The primary endpoints are expected to be reduction in seizures at 12 and 24 weeks. Results from the study are expected in 2019.

ZYN002 in Focal Epilepsy

Clinical Data from the STAR 1 and STAR 2 Studies of ZYN002 in Patients with Focal Seizures Presented at the 2017 Annual Meeting of the American Epilepsy Society (AES) in Washington, D.C.

Data suggest clinically meaningful responses to ZYN002, as measured by reductions in focal seizures from the baseline period of STAR 1, are correlated with longer-term use of ZYN002.

  • Patients taking ZYN002 for six months  experienced a >30% median reduction in seizures from baseline;
  • Patients taking ZYN002 for nine months experienced a >65% (195 mg in STAR 1 and 390 mg in STAR 2) and >48% (390 mg in STAR 1 and STAR 2) median reduction in seizures from baseline;
  • In STAR 1, patients with more severe epilepsy (defined as a baseline seizure frequency of ≥15 per month) taking ZYN002 had a greater percent reduction in seizures compared to patients with severe epilepsy receiving placebo;
  • ZYN002 was very well tolerated with an incidence of adverse events comparable to placebo and no clinically significant differences between the active treatment groups; and
  • There were no clinically significant changes in ECGs or laboratory results in patients receiving ZYN002.

Initiation of Double-Blind, Placebo Controlled Phase 2b Clinical Trial of ZYN002 in Approximately 300 Adult Patients with Refractory Focal Epilepsy Expected in the Second Half of 2018

Learnings from the STAR 1 and STAR 2 trials provide insight into a revised Phase 2b clinical trial design. Anticipated changes to the trial include increases in (1) baseline seizure frequency, (2) patient count and (3) trial duration. In addition, the Company will stratify randomization by baseline seizure rate and gender. Zynerba also anticipates testing a higher daily dose of ZYN002 than was used in the STAR trials. The study will be conducted in sites in the U.S., Australia and New Zealand.

ZYN001 in Tourette Syndrome

Dosing Continues in the Phase 1 Program for ZYN001 Pro-drug of Tetrahydrocannabinol (THC) Delivered via Transdermal Patch; Initiation of Phase 2 Study in Patients with Tourette Syndrome (TS) Expected in the Second Half of 2018

The Company is executing on a Phase 1 program to assess ZYN001, a patent-protected, pro-drug of THC delivered via a patch. This first-in-man study is a randomized, double-blind, placebo-controlled trial designed to assess the safety, tolerability and pharmacokinetic profile of multiple formulations of ZYN001. The Company expects to complete its Phase 1 evaluation in the first half of 2018, and then move into a Phase 2 clinical trial in Tourette Syndrome late in the second half of 2018.

Corporate

Corporate Strategy Focused on Rare and Near-Rare Neuropsychiatric Disorders with High Unmet Medical Needs  

Zynerba believes that its strategic focus provides opportunities for an efficient development and commercialization strategies that may include (1) orphan drug designation for some indications; (2) access to other available regulatory designations, which, if granted, can accelerate commercial approval; (3) a targeted physician audience enabling modest commercial investments; and (4) consistent pricing across all indications. The Company also discontinued its development programs in the capital-intensive pain spaces.

Enhanced Senior Management Team

In January 2018, Liza Squires, M.D. was named Chief Medical Officer. She has over 25 years of experience in rare and neuropsychiatric disorders with companies including Aevi Genomics Medicine, Lumos Pharma, and Shire Pharmaceuticals. She also served as the Director of Pediatric Neurology for DeVos Children’s Hospital.

In March 2018, Joe Apostolico joined Zynerba as Vice President, Human Resources. Mr. Apostolico brings the Company over 30 years of broad global healthcare and pharmaceutical human resource leadership experience from his tenures with companies including Adaptimmune Therapeutics and GSK.

Fourth Quarter 2017 Financial Results

As of December 31, 2017, cash and cash equivalents were $62.5 million, compared to $31.0 million as of December 31, 2016. Research and development expenses for the fourth quarter of 2017 were $5.8 million, including stock-based compensation of $0.6 million. General and administrative expenses for the fourth quarter of 2017 were $2.4 million, including stock-based compensation expense of $0.8 million. Net loss for the fourth quarter of 2017 was $8.1 millionwith basic and diluted net loss per share of $(0.60).

On June 9, 2017, we entered into an Open Market Sales Agreement, or “at-the-market” (ATM) offering program, with Jefferies LLC, pursuant to which we may sell, from time to time, up to $50 million of our common stock. From September 28, 2017 through October 26, 2017, the Company has sold and issued 296,594 shares under its ATM program, at a weighted average selling price of $10.74 per share, for gross proceeds of $3.2 million. Net proceeds after deducting underwriting and commissions and offering expenses were $3.0 million, which were recorded in the fourth quarter and are included in the December 31, 2017 cash and cash equivalents position.

Financial Outlook

The Company believes that the cash and cash equivalent position of $62.5 million as of December 31, 2017 is sufficient to fund operations and capital requirements well into 2019.

About Zynerba Pharmaceuticals, Inc. 
Zynerba Pharmaceuticals (NASDAQ:ZYNE) is a clinical-stage specialty neuropsychiatric pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare or near-rare neuropsychiatric disorders with high unmet medical needs. We are dedicated to improving the lives of people with severe health conditions by developing cannabinoid medicines designed to meet the rigorous efficacy and safety standards established by global regulatory agencies. Through the discovery and development of these potentially life-changing medicines, Zynerba seeks to improve the lives of patients battling severe, chronic health conditions including Fragile X syndrome, refractory epilepsies, Tourette Syndrome, and other neuropsychiatric disorders. Learn more at www.zynerba.com and follow the Company on Twitter at @ZynerbaPharma.

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 (FDA) or foreign regulatory authorities; even if ZYN002 or ZYN001 are approved, the Company may not be able to obtain the label claims that it is seeking from the FDA. In addition, the Company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated. Management’s expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: the success, cost and timing of the Company’s product development activities, studies and clinical trials; the success of competing products that are or become available; the Company’s ability to commercialize its product candidates; the size and growth potential of the markets for the Company’s product candidates, and the Company’s ability to service those markets; the Company’s ability to develop sales and marketing capabilities, whether alone or with potential future collaborators; the rate and degree of market acceptance of the Company’s product candidates; and the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates. This list is not exhaustive and these and other risks are described in the Company’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at www.sec.gov. Any forward-looking statements that the Company makes in this press release speak only as of the date of this press release. The Company assumes no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

ZYNERBA PHARMACEUTICALS, INC.
CONSOLIDATED STATEMENTS OF OPERATIONS
(unaudited)
Three months ended Year ended
December 31, 2017
  December 31, 2016
December 31, 2017
  December 31, 2016
Revenue $ $ $ $ 7,250
Operating expenses:
Research and development 5,828,091 4,904,363 22,806,107 16,784,626
General and administrative 2,376,413 1,780,304 10,016,902 6,430,252
Total operating expenses 8,204,504 6,684,667 32,823,009 23,214,878
Loss from operations (8,204,504 ) (6,684,667 ) (32,823,009 ) (23,207,628 )
Other income (expense):
Interest income (expense), net 156,204 26,980 519,554 80,222
Foreign exchange gain (loss) (70,299 ) (139,829 ) 291,151 (189,497 )
Loss on dosposal of equipment (99,147 ) (99,147 )
Total other income (expense) 85,905 (211,996 ) 810,705 (208,422 )
Loss before income taxes (8,118,599 ) (6,896,663 ) (32,012,304 ) (23,416,050 )
Income tax benefit (27,543 )
Net loss $ (8,118,599 ) $ (6,896,663 ) $ (32,012,304 ) $ (23,388,507 )
Net loss per share – basic and diluted $ (0.60 ) $ (0.71 ) $ (2.48 ) $ (2.58 )
Basic and diluted weighted average shares outstanding 13,423,669 9,678,924 12,914,814 9,070,232
Non-cash stock-based compensation included above:
Research and development $ 562,410 $ 365,072 $ 2,284,866 $ 1,281,108
General and administrative 817,726 522,352 3,361,986 1,988,258
Total $ 1,380,136 $ 887,424 $ 5,646,852 $ 3,269,366
ZYNERBA PHARMACEUTICALS, INC.
CONSOLIDATED BALANCE SHEETS
December 31, 2017   December 31, 2016
Assets
Current assets:
Cash and cash equivalents $ 62,510,277 $ 30,965,791
Incentive and tax receivables 3,983,604 3,613,943
Prepaid expenses and other current assets 1,733,701 1,830,958
Total current assets 68,227,582 36,410,692
Property and equipment, net 164,527 143,382
Other assets 662,200 200
Total assets $ 69,054,309 $ 36,554,274
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable $ 3,355,255 $ 1,848,084
Accrued expenses 3,915,491 4,284,907
Deferred grant revenue 171,975 833,975
Total current liabilities 7,442,721 6,966,966
Deferred grant revenue, long-term 662,000
Total liabilities 8,104,721 6,966,966
Stockholders’ equity:
Common stock 13,554 9,995
Additional paid-in capital 138,916,900 75,545,875
Accumulated deficit (77,980,866 ) (45,968,562 )
Total stockholders’ equity 60,949,588 29,587,308
Total liabilities and stockholders’ equity $ 69,054,309 $ 36,554,274

Investor Contacts

Jim Fickenscher, CFO and VP Corporate Development
Zynerba Pharmaceuticals
484.581.7483
fickenscherj@zynerba.com

Will Roberts, VP Investor Relations and Corporate Communications
Zynerba Pharmaceuticals
484.581.7489
robertsw@zynerba.com

Zynerba Pharmaceuticals Announces Positive Meeting with U.S. Food and Drug Administration and Plans to Conduct a Single Pivotal Study of ZYN002 in Fragile X Syndrome to Support an NDA Filing

 

Company Expects to Initiate Pivotal Study Mid-Year 2018 and Deliver Top-line Data in 2019

Devon, PA, March 5, 2018  – Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), a clinical-stage specialty neuropsychiatric pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare and near-rare neurological and psychiatric disorders with high unmet medical needs, today announced the results of a positive meeting held with the U.S. Food and Drug Administration (FDA) regarding its planned development strategy for ZYN002 in Fragile X syndrome (FXS). FXS is a rare genetic developmental disability that is the leading known cause of both inherited intellectual disability and autism spectrum disorder.

ZYN002 is the first and only pharmaceutically-produced cannabidiol (CBD) formulated as a patent-protected permeation-enhanced transdermal gel. Zynerba has received U.S. Orphan Drug designation for the use of CBD as a treatment of FXS. Currently, there are no approved therapies to treat FXS or its most common symptoms.

Based on the Company’s dialogue with the FDA, the Company expects to initiate a single pivotal study mid-year 2018 to support a New Drug Application (NDA) for ZYN002 in FXS. The FDA and the Company are in agreement that the primary and key secondary endpoints for the study should assess observable behaviors in patients with FXS as reported by the caregiver using the validated Aberrant Behavior Checklist in Fragile X syndrome (ABC-FXS). If the pivotal trial meets its endpoints, approval for an indication encompassing the treatment of behavioral symptoms associated with Fragile X syndrome may be granted.

“The FDA meeting was an important milestone for us as we advance the development of ZYN002 for patients and their families suffering with the profound behavioral symptoms of Fragile X syndrome,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We are pleased with the outcome of the discussion and the guidance on trial design, and believe we now have a path forward to advance the development of ZYN002 to an NDA. We look forward to initiating the pivotal study mid-year, and potentially providing FXS patients and their families an effective and well tolerated therapy to treat the complex behavioral symptoms of Fragile X syndrome.”

Zynerba plans to initiate a pivotal 14-week randomized, double blind, placebo controlled clinical trial in approximately 200 pediatric and adolescent patients in the U.S., Australia and New Zealand. Patients will be randomized 1:1 to receive either one of two weight based doses of ZYN002, or one of two matching administrations of placebo. Zynerba anticipates initiation of this pivotal clinical trial mid-year 2018. Additional protocol details will be shared at that point. All patients will be eligible to enroll in a 12-month open label extension after completing dosing in the pivotal study.

“We believe that ZYN002 may address core behavioral symptoms of FXS and improve the quality of life for patients and their families,” said Dr. Liza Squires, Zynerba’s Chief Medical Officer. “There are currently no drugs indicated to address behavioral symptoms in the FXS population. We believe we have designed an efficient pivotal program that includes endpoints that measure clinically relevant and observable behaviors in patients with FXS, and if successful, positions us to bring the FXS community its first targeted treatment designed with patients’ symptoms in mind.”

About Fragile X syndrome
Fragile X syndrome is a rare genetic developmental disability that is the leading known cause of both inherited intellectual disability and autism spectrum disorder, affecting 1 in 3,600 to 4,000 males and 1 in 4,000 to 6,000 females. It is the most common inherited intellectual disability in males and a significant cause of intellectual disability in females. It is caused by a mutation in the Fragile X Mental Retardation gene located on the X chromosome and leads to dysregulation of the endocannabinoid pathway including the reduction in endogenous cannabinoids (2-AG and anandamide). The disorder negatively affects synaptic function, plasticity and neuronal connections, and results in a spectrum of intellectual disabilities, social anxiety and memory problems. In the US, there are about 71,000 patients suffering with FXS.

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 and Fragile X syndrome, and THC has positive effects on treating symptoms of Tourette Syndrome. 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 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 large markets that we are targeting.

About ZYN002
Zynerba’s ZYN002 CBD gel is the first and only pharmaceutically-produced CBD formulated as a patent-protected permeation-enhanced transdermal gel and is being studied in children and adolescents with Fragile X syndrome and developmental and epileptic encephalopathies, and in adult epilepsy patients with focal seizures. ZYN002 is a clear, permeation-enhanced gel that is designed to provide controlled drug delivery transdermally with once- or twice-daily dosing.

About Zynerba Pharmaceuticals, Inc. 
Zynerba Pharmaceuticals (NASDAQ:ZYNE) is a clinical-stage specialty pharmaceutical company dedicated to developing and commercializing innovative pharmaceutically-produced transdermal cannabinoid treatments for rare or near-rare neuropsychiatric diseases with high unmet medical needs. We are dedicated to improving the lives of people with severe health conditions by developing cannabinoid medicines designed to meet the rigorous efficacy and safety standards established by global regulatory agencies. Through the discovery and development of these potentially life-changing medicines, Zynerba seeks to improve the lives of patients battling severe, chronic health conditions including Fragile X syndrome, refractory epilepsies, Tourette Syndrome, and other neuropsychiatric disorders. Learn more at www.zynerba.com and follow the Company on Twitter at @ZynerbaPharma.

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 from the U.S. Food and Drug Administration (FDA) or foreign regulatory authorities; even if ZYN002 are approved, the Company may not be able to obtain the label claims that it is seeking from the FDA. In addition, the Company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated.  Management’s expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the following: the success, cost and timing of the Company’s product development activities, studies and clinical trials; the success of competing products that are or become available; the Company’s ability to commercialize its product candidates; the size and growth potential of the markets for the Company’s product candidates, and the Company’s ability to service those markets; the Company’s ability to develop sales and marketing capabilities, whether alone or with potential future collaborators; the rate and degree of market acceptance of the Company’s product candidates; and the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates. This list is not exhaustive and these and other risks are described in the Company’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at www.sec.gov. Any forward-looking statements that the Company makes in this press release speak only as of the date of this press release. The Company assumes no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Investor Contact
Will Roberts, VP Investor Relations and Corporate Communications
Zynerba Pharmaceuticals
484.581.7489
robertsw@zynerba.com