Devon, PA, May 30, 2019 — Zynerba Pharmaceuticals, Inc.(NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that Ray Mannion, Zynerba’s Vice President of Manufacturing, will provide a formal presentation during the U.S. Food and Drug Administration’s (FDA) Public Hearing on Scientific Data and Information about Products Containing Cannabis or Cannabis-Derived Compounds. As noted in the Federal Register, the public hearing is scheduled from 8:00 a.m. to 6:00 p.m. ET tomorrow, May 31, 2019.
Slides from Mr. Mannion’s presentation, entitled ‘Cannabinoid Manufacturing and Product Quality’, are now available on Zynerba’s corporate website at https://zynerba.com/publications/.
“The FDA has a well-established history of protecting public health and safety through a variety of essential strategies, including existing regulations and processes that govern the manufacture of pharmaceutical products and establish controls to ensure necessary quality and safety standards are met,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “This existing robust framework should be leveraged in the regulation of all cannabinoid products, as less stringent manufacturing and quality standards would create an unnecessary public health risk.”
Ray Mannion has served as Zynerba’s Vice President of Manufacturing since April 2017. He has more than 35 years of international manufacturing, operations and engineering experience in the pharmaceutical, medical devices and electrical connection systems industries. Prior to Zynerba, he was Senior Director, Third Party Operations for Teva Pharmaceuticals Inc.where he successfully managed the production scale up for the commercial launch of the Zecuity® migraine patch while also managing all technical aspects of the project. Prior to Teva, Mr. Mannion held roles of increasing responsibility at NuPathe, Puricore, Kensey Nash Corporation, AMP Incorporated and others. Mr. Mannion received a B.S. in Industrial Engineering from Rutgers University and an M.B.A from Shippensburg University.
About Zynerba Pharmaceuticals, Inc. Zynerba Pharmaceuticals is the leader in pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X Syndrome, Autism Spectrum Disorder, 22q11.2 Deletion Syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us 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
Topline Data from the INSPIRE Trial Expected in the First Half 2020
Devon, PA., May 29, 2019 — Zynerba Pharmaceuticals, Inc.(NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that it has initiated the Phase 2 INSPIRE (Assessing the Impact of Zygel [Transdermal CBD Gel] on Pediatric Behavioral and Emotional Symptoms of 22q11.2 Deletion Syndrome) trial. The INSPIRE trial will assess the safety, tolerability and efficacy of Zygel (ZYN002 CBD gel) for the treatment of behavioral symptoms of 22q11.2 Deletion Syndrome (22q). The Company expects to present topline data from this study in the first half of 2020.
“Children born with 22q often require surgeries to rectify acute physical concerns, like anomalies of the heart and palate; once corrected, there are a myriad of behavioral symptoms that need to be addressed,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “Parents of children with 22q report significantly higher rates of withdrawn behavior, affective disorders, pervasive developmental problems and anxiety in their children compared to non-affected children. There is also an increased risk of developing psychoses such as schizophrenia later in life, compared to the general population. We are very excited to initiate the INSPIRE trial to assess the potential impact of Zygel in these children and adolescents, and look forward to presenting topline data in the first half of 2020.”
The 14-week INSPIRE trial is an open-label multi-dose Phase 2 clinical trial designed to evaluate the efficacy and safety of Zygel in approximately 20 children and adolescents (ages six through 17) with genetically-confirmed 22q. Enrolled patients will receive weight-based doses of 250 mg daily or 500 mg daily of Zygel. The efficacy assessments include the Aberrant Behavior Checklist-Community (ABC-C), the Anxiety, Depression and Mood Scale (ADAMS), the Columbia Suicide Severity Rating scale (C-SSRS), the Qualitative Caregiver Reported Behavioral Problem Survey, and Clinical Global Impression – Severity and Improvement.
About 22q11.2 Deletion Syndrome (22q) As the second most common chromosomal disorder after Down syndrome, 22q is caused by a small missing piece of the 22nd chromosome. The deletion occurs near the middle of the chromosome at a location designated q11.2. It is considered a mid-line condition, with physical symptoms including characteristic palate abnormalities, heart defects, immune dysfunction, and esophageal / GI issues, as well as debilitating neuropsychiatric and behavioral challenges. Anxiety is among the most common neuropsychiatric symptoms of 22q and researchers have found that for children with 22q, anxiety is linked to poorer adaptive behaviors such as self-care and communication skills that affect daily life. Children with 22q also experience withdrawn behavior, ADHD, cognitive impairment, and autism spectrum disorder that affect communication and social interaction. Later in life, they are at an increased risk of developing mental illnesses such as schizophrenia. It is estimated that 22q occurs in between one in 3,000 and one in 6,000 live births, suggesting that there are approximately 81,000 people living with 22q in the U.S.
About Zygel™ Zygel (CBD gel) is the first and only pharmaceutically-manufactured CBD formulated as a patent-protected permeation-enhanced clear gel, designed to provide controlled drug delivery into the bloodstream transdermally (i.e. through the skin). Recent studies suggest that Fragile X Syndrome (FXS) and other neuropsychiatric conditions may be associated with a disruption in the endocannabinoid (EC) system. Clinical and anecdotal data suggest that CBD may modulate the EC system and improve certain core social and behavioral symptoms, including social avoidance (prefers isolation from others, prefers solitary activities, avoids new social activities), irritability (aggressive to others, tantrums/outbursts, and stubbornness), social unresponsiveness/lethargy (lack of attention/interaction, inactive/lack of movement and can resist physical contact), and anxiety.
Zygel has been designated a Fast Track development program by the U.S. Food and Drug Administration for treatment of behavioral symptoms of FXS. Enrollment is ongoing in CONNECT-FX, a multi-national, randomized, double blind placebo controlled pivotal clinical trial of Zygel in FXS (https://www.connectfxtrial.com/); topline data are expected in the second half of 2019. Additionally, Zynerba expects topline data from its Phase 2 open label BELIEVE 1 trial of Zygel in developmental and epileptic encephalopathies (DEE) in the third quarter of 2019. Zynerba has also initiated the Phase 2 BRIGHT trial in Autism Spectrum Disorder and the Phase 2 INSPIRE trial in 22q11.2 Deletion Syndrome, with data expected from both studies in the first half of 2020.
About Zynerba Pharmaceuticals, Inc. Zynerba Pharmaceuticals is the leader in pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X Syndrome, Autism Spectrum Disorder, 22q11.2 Deletion Syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us 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 Zygel from the U.S. Food and Drug Administration (FDA) or foreign regulatory authorities; even if Zygel is approved, the Company may not be able to obtain the label claims that it is seeking from the FDA. 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 Company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated; the Company’s ability to obtain additional funding to support its clinical development programs; the results, cost and timing of the Company’s clinical development programs, including any delays to such clinical trials relating to enrollment or site initiation; clinical results for the Company’s product candidates may not be replicated or continue to occur in additional trials and may not otherwise support further development in a specified indication or at all; actions or advice of the FDA and foreign regulatory agencies may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional clinical trials; the Company’s ability to obtain and maintain regulatory approval for its product candidates, and the labeling under any such approval; the Company’s reliance on third parties to assist in conducting pre-clinical and clinical trials for its product candidates; delays, interruptions or failures in the manufacture and supply of the Company’s product candidates 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 Contact William Roberts, Vice President, Investor Relations and Corporate Communications Zynerba Pharmaceuticals 484.581.7489 robertsw@zynerba.com
Devon, PA, May 28, 2019 — Zynerba Pharmaceuticals, Inc.(NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that Zynerba’s Chief Executive Officer, Armando Anido will present a company overview at the Jefferies 2019 Global Healthcare Conference. The presentation will take place on Thursday June 6, 2019 at 8:30AM EDT at the Grand Hyatt in New York City. 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 is the leader in pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X Syndrome, Autism Spectrum Disorder, 22q11.2 Deletion Syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us 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
Devon, PA, May 8, 2019 — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today reported financial results for the first quarter ended March 31, 2019 and provided an overview of recent operational highlights.
“We are entering a transformational period for Zynerba during which we expect top line data from four neuropsychiatric disorder trials with Zygel™, our patent protected CBD gel,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We’ve made great progress with Zygel which was recently designated as a Fast Track development program by the FDA for treating behavioral symptoms of Fragile X Syndrome. Over the next 14 months, we expect to announce data from the CONNECT-FX pivotal trial in Fragile X, our Phase 2 BELIEVE 1 trial in developmental and epileptic encephalopathies, our Phase 2 BRIGHT trial in Autism Spectrum Disorder, and the Phase 2 trial in 22q11.2 Deletion Syndrome that we intend to initiate this quarter. We expect that our cash position will take us through all of these data readouts, through our anticipated NDA submission and potential approval for Zygel in FXS, and into the first quarter of 2021.”
First Quarter 2019 and Recent Highlights
Zygel in Fragile X Syndrome (FXS)
Fragile X Syndrome Pivotal Data Expected in the Second Half of 2019
Enrollment is progressing in CONNECT-FX, a pivotal, multi-national, randomized, double blind, placebo-controlled trial evaluating the efficacy and safety of Zygel (ZYN002 CBD gel) in three through 17-year old patients with FXS. The primary endpoint is the change from baseline to the end of the treatment period in the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance subscale. Clinical investigative sites are enrolling patients in the United States, Australia, and New Zealand. Patients who have completed the double-blind phase are now enrolling into the 12-month open label extension phase. The Company expects to report top line data in the second half of 2019. If the data are positive, the Company expects to submit its New Drug Application (NDA) for Zygel in FXS to the U.S. Food and Drug Administration(FDA) in the first half of 2020, with potential approval by year-end 2020.
Received Fast Track Designation for Zygel for Treatment of Behavioral Symptoms Associated with Fragile X Syndrome
FDA’s Fast Track program is designed to facilitate the development of drugs intended to treat serious conditions and fill unmet medical needs and can lead to expedited review by FDA in order to get new important drugs to the patient earlier.
Announced Receipt of New U.S. Patent for Treatment of Fragile X Syndrome with Cannabidiol (CBD)
The U.S. Patent and Trademark Office issued U.S. Patent No. 10,213,390 titled “Treatment of Fragile X Syndrome with Cannabidiol” which includes claims directed to methods of treating FXS by administering a therapeutically effective amount of synthetic or purified CBD. This new patent expires in 2038 and is part of an expanding intellectual property portfolio covering Zygel.
Zygel in Developmental and Epileptic Encephalopathies (DEE)
Topline Results Expected in the Third Quarter of 2019
Enrollment is complete in BELIEVE 1, an open label multi-dose Phase 2 clinical trial evaluating the efficacy and safety of Zygel in children and adolescents (three through 17 years) with DEE. The primary efficacy assessment is reduction in seizure frequency at week 26 compared to baseline. The Company expects to announce topline data through week 26 in the third quarter of 2019.
Zygel in Autism Spectrum Disorder (ASD)
Announced Initiation of Phase 2 Open Label Study in Zygel in ASD; Data Expected in the First Half of 2020
The Company initiated the Phase 2 BRIGHT trial in March 2019 to assess the safety, tolerability and efficacy of Zygel for the treatment of child and adolescent patients with ASD. The 14-week trial is designed to evaluate the efficacy and safety of Zygel in approximately 36 children and adolescents (ages four through 17) with ASD as confirmed by DSM-5 diagnostic criteria for ASD. The efficacy assessments include the Aberrant Behavior Checklist, Parent Rated Anxiety Scale – Autism Spectrum Disorder, Autism Impact Measure, and Clinical Global Impression – Severity and Improvement. Zynerba expects to present topline data from this study in the first half of 2020.
Zygel in 22q11.2 Deletion Syndrome (22q)
Initiation of Phase 2 Trial in 22q Anticipated Later this Quarter; Data Expected in the First Half of 2020
Zynerba has finalized the protocol to evaluate the efficacy and safety of Zygel in approximately 20 children and adolescents (ages six through 17) with genetically-confirmed 22q. The Company expects to initiate this study during the second quarter of 2019, and to present topline data in the first half of 2020.
Corporate
Dr. Thomas Harrison resigned from the Company’s Board of Directors to focus on his role with Merida Capital Partners as Senior Operating Partner. Dr. Harrison served on Zynerba’s Board of Directors since April 2015.
“On behalf of Zynerba’s Board of Directors and management team, I would like to thank Tom for his outstanding service and leadership during his term as a member of our board,” continued Mr. Anido. “His expertise and counsel have been invaluable to Zynerba and we wish him well in his new endeavor.”
First Quarter 2019 Financial Results
As of March 31, 2019, cash and cash equivalents were $68.3 million, compared to $59.8 million as of December 31, 2018. Included in this cash and cash equivalents position is $18.1 million in net proceeds from the 3,439,523 shares sold and issued at a weighted average selling price of $5.44 per share during the first quarter of 2019 under an Open Market Sales Agreement, or “at-the-market” (ATM) offering program, with Jefferies LLC. Research and development expenses for the first quarter of 2019 were $6.3 million, including stock-based compensation of $0.7 million. General and administrative expenses for the first quarter of 2019 were $3.2 million, including stock-based compensation expense of $0.8 million. The net loss for the first quarter of 2019 was $9.1 million with basic and diluted net loss per share of $(0.47).
Financial Outlook
The Company’s cash and cash equivalent position as of March 31, 2019 was $68.3 million. Management believes that the cash and cash equivalent position is sufficient to fund operations and capital requirements beyond the expected NDA submission and potential approval in FXS and into the first quarter of 2021.
About Zynerba Pharmaceuticals, Inc. Zynerba Pharmaceuticals is the leader in pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X Syndrome, Autism Spectrum Disorder, 22q11.2 Deletion Syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us 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. 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 Company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated; the Company’s ability to obtain additional funding to support its clinical development programs; the results, cost and timing of the Company’s clinical development programs, including any delays to such clinical trials relating to enrollment or site initiation; clinical results for the Company’s product candidates may not be replicated or continue to occur in additional trials and may not otherwise support further development in a specified indication or at all; actions or advice of the U.S. Food and Drug Administrationand foreign regulatory agencies may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional clinical trials; the Company’s ability to obtain and maintain regulatory approval for its product candidates, and the labeling under any such approval; the Company’s reliance on third parties to assist in conducting pre-clinical and clinical trials for its product candidates; delays, interruptions or failures in the manufacture and supply of the Company’s product candidates 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 Commissionand 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)
ZYNERBA PHARMACEUTICALS, INC. CONSOLIDATED BALANCE SHEETS
Zynerba 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
– Designation Facilitates and Expedites Development of Drugs for Patients with Serious Unmet Medical Needs –
– Enrollment Progressing in Pivotal CONNECT-FX Trial of Zygel in FXS, with Data Expected in the Second Half of 2019 –
Devon, PA, May 6, 2019 — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the Company’s lead development candidate Zygel™ (ZYN002 CBD gel) for treatment of behavioral symptoms associated with Fragile X Syndrome (FXS). FDA’s Fast Track program is designed to facilitate the development of drugs intended to treat serious conditions and fill unmet medical needs, and can lead to expedited review by FDA in order to get new important drugs to the patient earlier.
“The FDA’s decision to grant Fast Track Designation for Zygel underscores the significance and severity of the unmet medical need that exists for patients living with Fragile X Syndrome and their caregivers,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We believe that Zygel has the potential to be the first treatment indicated to directly address the core behavioral symptoms of this syndrome, and we look forward to working closely with the FDA to obtain approval to market Zygel as soon as possible.”
Zynerba is conducting a pivotal trial to assess the efficacy and safety of Zygel as treatment for the behavioral symptoms of FXS in pediatric and adolescent patients (three through 17 years of age). Zygel is a pharmaceutically-manufactured CBD formulated as a patent-protected permeation-enhanced clear gel, designed to provide controlled transdermal drug delivery into the bloodstream.
About Fast Track Designation Fast Track Designation is intended to facilitate development and expedite review of drugs to treat serious or life-threatening conditions so that a product can reach the market expeditiously. A drug that is intended to treat a serious or life-threatening condition that demonstrates the potential to address an unmet medical need may qualify for Fast Track designation. Features of this designation include opportunities for frequent interactions with the review team. These include meetings with the FDA to discuss items such as study design, extent of safety data required to support approval, dose-response concerns, accelerated approval, the structure and content of an NDA, and other critical issues. In addition, such a product could be eligible for priority review if supported by clinical data at the time of NDA.
About Fragile X Syndrome (FXS) 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. FXS is caused by a mutation in the Fragile X Mental Retardation gene (FMR1) 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 and behavioral symptoms, such as social avoidance and irritability. In the US, there are about 71,000 patients suffering with FXS.
About Zygel™ Zygel (CBD gel) is the first and only pharmaceutically-manufactured CBD formulated as a patent-protected permeation-enhanced clear gel, designed to provide controlled drug delivery into the bloodstream transdermally (i.e. through the skin). Recent studies suggest that FXS and other neuropsychiatric conditions may be associated with a disruption in the endocannabinoid (EC) system. Clinical and anecdotal data suggest that CBD may modulate the EC system and improve certain core social and behavioral symptoms, including social avoidance (prefers isolation from others, prefers solitary activities, avoids new social activities), irritability (aggressive to others, tantrums/outbursts, and stubbornness), and social unresponsiveness/lethargy (lack of attention/interaction, inactive/lack of movement and can resist physical contact).
Enrollment is ongoing in the multi-national, randomized, double blind placebo controlled Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X (CONNECT-FX), a pivotal clinical trial of ZYN002 in FXS (https://www.connectfxtrial.com/); topline data from CONNECT-FX are expected in the second half of 2019. Additionally, Zynerba expects topline data from its Phase 2 Open Label Study to Assess the Safety and Efficacy of ZYN002 Administered as a TransdermalGel to Children and Adolescents with Developmental and Epileptic Encephalopathy (BELIEVE 1) clinical trial in the third quarter of 2019. Zynerba has also initiated a Phase 2 study of Zygel in Autism Spectrum Disorder, with data expected in the first half of 2020.
About Zynerba Pharmaceuticals, Inc. Zynerba Pharmaceuticals is the leader in pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X Syndrome, Autism Spectrum Disorder, 22q11.2 Deletion Syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us 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 Zygel from the U.S. Food and Drug Administration (FDA) or foreign regulatory authorities; even if Zygel is approved, the Company may not be able to obtain the label claims that it is seeking from the FDA. 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 Company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated; the Company’s ability to obtain additional funding to support its clinical development programs; the results, cost and timing of the Company’s clinical development programs, including any delays to such clinical trials relating to enrollment or site initiation; clinical results for the Company’s product candidates may not be replicated or continue to occur in additional trials and may not otherwise support further development in a specified indication or at all; actions or advice of the FDA and foreign regulatory agencies may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional clinical trials; the Company’s ability to obtain and maintain regulatory approval for its product candidates, and the labeling under any such approval; the Company’s reliance on third parties to assist in conducting pre-clinical and clinical trials for its product candidates; delays, interruptions or failures in the manufacture and supply of the Company’s product candidates 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 Contact William Roberts, Vice President, Investor Relations and Corporate Communications Zynerba Pharmaceuticals 484.581.7489 robertsw@zynerba.com
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