Author: Jennifer Guinan

Provides 10-year market exclusivity in EU upon regulatory approval

Devon, PA, February 28, 2022 — 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 European Commission (EC) has granted orphan drug designation to cannabidiol, the active ingredient in its transdermal gel, Zygel™, for the treatment of Fragile X syndrome (FXS).

“Orphan drug designation by the EC is another important milestone for Zygel,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “It underscores the urgent, unmet medical need for patients diagnosed with Fragile X syndrome, and will benefit us as we continue to advance our clinical programs and prepare to bring Zygel to market.”

Zynerba is currently enrolling patients in RECONNECT, a confirmatory pivotal Phase 3 trial of Zygel in children and adolescents with FXS, and continues to expect topline results from this trial in the second half of 2023. As previously announced, the Company believes, based on the EMA’s scientific advice, that the successful completion of the current development program for Zygel in FXS will satisfy the requirements of a marketing authorization application in the European Union (EU).

Orphan drug designation is granted to medicines that treat, prevent or diagnose a life-threatening or chronically debilitating rare disease, with a prevalence in the EU of not more than 5 in 10,000, and with either no currently approved method of diagnosis, prevention or treatment or with significant benefit to those affected by the disease. The designation potentially provides certain benefits to Zynerba, including 10-year EU market exclusivity upon regulatory approval, if received, reductions in EMA application fees, and access to protocol assistance.

The Company believes that there are approximately 105,000 patients with FXS in the EU and approximately 121,000 in Europe if the United Kingdom (UK) is included. Approximately 60% of all patients are believed to be completely methylated, which indicates that there are approximately 73,000 patients with FXS who are completely methylated in the EU and UK.

As previously disclosed, Zygel has also been granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the treatment of FXS and 22q11.2 deletion syndrome. Additionally, the FDA has granted Fast Track designation for Zygel for the treatment of behavioral symptoms associated with FXS. Based on latest census data available, we believe there are approximately 78,000 patients with FXS in the U.S., and approximately 47,000 of those patients are completely methylated.

About Zygel™
Zygel is the first and only pharmaceutically-manufactured cannabidiol 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 cannabidiol may modulate the endocannabinois system and improve certain behavioral symptoms associated with neuropsychiatric conditions. Zygel is an investigational drug product in development for the potential treatment of behavioral symptoms associated with Fragile X syndrome (FXS), autism spectrum disorder (ASD), and 22q11.2 deletion syndrome (22q). Zygel has been granted orphan drug designation by the FDA and the EC in the treatment of FXS and by the FDA for the treatment of 22q. Additionally, Zygel has been designated a Fast Track development program for treatment of behavioral symptoms of FXS.

About 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, 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, and the leading genetic cause of autism spectrum disorder (ASD). 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 U.S., there are approximately 78,000 people suffering with FXS, and approximately 121,000 in the EU and UK. We believe that approximately 60% of all patients with FXS have complete methylation of their FMR1 gene.

About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative 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, and 22q11.2 deletion syndrome. 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 expectations, projections and estimates regarding expenses, future revenue, capital requirements, incentive and other tax credit eligibility, collectability and timing, and availability of and the need for additional financing; 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 Administration, the European Medicines Agency and other 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; the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates; and the extent to which health epidemics and other outbreaks of communicable diseases, including COVID-19, could disrupt our operations or adversely affect our business and financial conditions. 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
Peter Vozzo
ICR Westwicke
Office: 443.213.0505
Cell: 443.377.4767
Peter.Vozzo@Westwicke.com

Topline data from Phase 2 INSPIRE trial expected mid-year 2022

Devon, PA, Feb. 28, 2022 — Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced the completion of enrollment for the 14-week Phase 2 INSPIRE (Assessing the Impact of Zygel [Transdermal CBD Gel] on Pediatric Behavioral and Emotional Symptoms of 22q11.2 Deletion Syndrome) trial of Zygel in the treatment of behavioral symptoms of 22q11.2 deletion syndrome (22q) in children and adolescents. The Company continues to expect topline data from this trial mid-year 2022. The Company has previously received orphan drug designation for Zygel in 22q from the FDA.

“The completion of enrollment in our INSPIRE trial is an important step forward in advancing Zygel as a potential new treatment option for patients suffering from 22q,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “Pending results from the INSPIRE trial and subsequent discussion with the FDA on the regulatory path forward, we plan to advance our clinical development program in 22q with Zygel. We are thankful for the combined efforts of our researchers, investigators and the patient and caregiver community.”

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 children and adolescents (ages six through 17) with genetically-confirmed 22q. Enrolled patients 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 Qualitative Caregiver Reported Behavioral Problem Survey, and Clinical Global Impression – Severity and Improvement. A total of 20 patients have been enrolled in the INSPIRE trial.

About Zygel™
Zygel is the first and only pharmaceutically-manufactured cannabidiol 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 cannabidiol may modulate the endocannabinoid system and improve certain behavioral symptoms associated with neuropsychiatric conditions. Zygel is an investigational drug product in development for the potential treatment of behavioral symptoms associated with Fragile X syndrome (FXS), autism spectrum disorder (ASD), and 22q11.2 deletion syndrome (22q). Zygel has been granted orphan drug designation by the FDA in the treatment of FXS and 22q. Additionally, the FDA has designated Zygel a Fast Track development program for treatment of behavioral symptoms of FXS.

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 has a prevalence rate of approximately one in 4,000 people in the U.S.^1,2, suggesting that there are approximately 83,000 people living with 22q in the U.S.

¹The 22q Family Foundation
²The International 22q11.2 Foundation

About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative 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, and 22q11.2 deletion syndrome. 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 expectations, projections and estimates regarding expenses, future revenue, capital requirements, incentive and other tax credit eligibility, collectability and timing, and availability of and the need for additional financing; 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 Administration 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; the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates; and the extent to which health epidemics and other outbreaks of communicable diseases, including COVID-19, could disrupt our operations or adversely affect our business and financial conditions. 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
Peter Vozzo
ICR Westwicke
Office: 443.213.0505
Cell: 443.377.4767
Peter.Vozzo@Westwicke.com

Devon, PA, February 15, 2022 — Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced the appointment of Albert P. Parker, an accomplished industry executive with over 25 years of pharmaceutical, biotech and healthcare experience, as Chief Legal Officer and Corporate Secretary. He will assume the duties of Suzanne Hanlon who will retire from her position at the end of February 2022.

“We are excited to have Al join our senior leadership team. He is an experienced legal and business executive who brings significant strategic and hands-on legal and compliance expertise, coupled with experience developing corporate strategy and maximizing commercial opportunities at life science companies of varying sizes,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “I want to personally thank Suzanne for her significant contributions throughout her eight years at Zynerba as she successfully developed and led our legal organization and was a great partner to me, the board of directors and the management team. While Suzanne’s leadership, experience and friendship will be missed, I sincerely hope that she enjoys the fruits of a very successful career in her retirement.”

Mr. Parker served most recently as Chief Operating Officer and Corporate Secretary of Oncocyte Corporation, an oncology focused precision diagnostics and monitoring company. Before joining Oncocyte, Mr. Parker was the Managing Shareholder of GC Legal Advisors, where he represented and advised public and privately held companies primarily in the life sciences industry. Among his prior roles, Mr. Parker has served as Executive Vice President, General Counsel and Corporate Secretary at Sunovion Pharmaceuticals, Senior Vice President & Chief Counsel for Wyeth Pharmaceuticals, and Partner at Schnader Harrison Segal & Lewis, L.L.P. He earned his Juris Doctorate from the University of Pennsylvania Carey Law School, and a Bachelor of Arts in Economics from Penn State University.

About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative 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, and 22q11.2 deletion syndrome. 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 expectations, projections and estimates regarding expenses, future revenue, capital requirements, incentive and other tax credit eligibility, collectability and timing, and availability of and the need for additional financing; 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 Administration 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; the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates; and the extent to which health epidemics and other outbreaks of communicable diseases, including COVID-19, could disrupt our operations or adversely affect our business and financial conditions. 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
Peter Vozzo
ICR Westwicke
Office: 443.213.0505
Cell: 443.377.4767
Peter.Vozzo@Westwicke.com

Devon, PA, February 10, 2022  — Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that Armando Anido, Chairman and Chief Executive Officer of Zynerba, will virtually present a company overview during the 11^th Annual SVB Leerink Global Healthcare Conference on Friday, February 18, 2022 at 8:40 a.m. ET.

Investors interested in arranging a virtual meeting with the Company’s management during the conference should contact the SVB Leerink conference coordinator. A webcast of the presentation will be available under the Events & Webcasts tab of the Investors section of the Zynerba website at www.zynerba.com. An archived replay will be available on the Company’s website following the event for 60 days.

About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative 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, and 22q11.2 deletion syndrome. 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.

Zynerba Contact
Peter Vozzo
ICR Westwicke
Office: 443.213.0505
Cell: 443.377.4767
Peter.Vozzo@Westwicke.com

Devon, PA, January 6, 2022 — Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that Armando Anido, Chairman and Chief Executive Officer of Zynerba Pharmaceuticals, will present during the H.C. Wainwright BioConnect Conference being held virtually January 10-13, 2022.

A webcast of the presentation will be available on-demand beginning January 10, 2022, at 7:00 a.m. ET under the Events & Webcasts tab of the Investors section of the Zynerba website at www.zynerba.com. An archived replay will be available on the Company’s website following the event for 60 days.

About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative 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, and 22q11.2 deletion syndrome. 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.

Zynerba Contacts
Peter Vozzo
ICR Westwicke
Office: 443.213.0505
Cell: 443.377.4767
Peter.Vozzo@Westwicke.com

– Company plans to focus development of Zygel™ in FXS, ASD and 22q –

– Company plans to initiate Phase 3 trial with Zygel in ASD in the second half of 2022 –

– Zynerba to hold conference call tomorrow, January 5, 2022 at 9:00 a.m. ET –

Devon, PA, January 4, 2022 — Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today provided program updates for Fragile X syndrome (FXS), autism spectrum disorder (ASD), 22q11.2 deletion syndrome (22q) and developmental and epileptic encephalopathies (DEE). In addition to the continued clinical development of Zygel in FXS, the Company plans to focus on the development of Zygel in ASD and 22q. Based on Company research and strategic prioritization, the decision has been made to not move forward in DEE at this time.

“Committing to a Phase 3 development program for patients with autism spectrum disorder is a key step in the advancement of a new treatment option for patients who have high unmet medical needs and limited FDA approved treatment options,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “Data from our ASD clinical development program to date are compelling and, with a clear regulatory path toward potential approval, we are excited to move forward with this program.”

Anido added, “In addition, we are making good progress towards completing enrollment in the INSPIRE trial for patients with 22q11.2 deletion syndrome and expect to have topline results mid-year 2022. Putting resources behind a second indication beyond FXS and completing the INSPIRE trial is consistent with our mission of being a leader in rare and near-rare neuropsychiatric disorders, including FXS, ASD and 22q.”

Pipeline Updates

Zygel in Fragile X Syndrome (FXS)

• The Company continues to expect topline results from RECONNECT, a confirmatory pivotal Phase 3 trial of Zygel in patients with FXS, in the second half of 2023.
• The Company received written scientific advice from the European Medicines Agency (EMA) providing clarity and guidance on the clinical and regulatory requirements for the submission of a marketing authorization application (MAA) in the European Union (EU) for Zygel for the treatment of behavioral symptoms associated with FXS. Based on the EMA’s scientific advice, the Company believes that the successful completion of the current development program for Zygel in FXS will satisfy the requirements of an MAA in the EU.     
• Presented data at the BRAIN Foundation Synchrony 2021 Symposium describing how Zygel may provide therapeutic benefit in FXS through its effects on the endocannabinoid system. The presentation included results from the Phase 3 CONNECT-FX trial demonstrating that Zygel was superior to placebo in multiple analyses in the groups of patients with either ≥90% methylation or complete methylation (100%) of their FMR1 gene. Data also showed that in the Company’s ongoing open label extension study, Zygel continued to be well-tolerated, without reports of clinically significant changes in vital signs, ECGs or laboratories (including liver function) with a median length of treatment of 21 months. (Presentation)

Zygel in Autism Spectrum Disorder (ASD)

• The Company plans to advance its program in ASD with two Phase 3 trials, the first of which is expected to start in the second half of 2022. The Company is finalizing the Phase 3 study protocol and will submit an Investigational New Drug application to the U.S. Food and Drug Administration (FDA) prior to commencing the pivotal program.
• Previous discussions with the FDA included agreement on utilizing the irritability subscale of the Aberrant Behavior Checklist – Community (ABC-C) as the primary endpoint to support an indication for the treatment of irritability in ASD. This is the same primary endpoint utilized in the pivotal trials for the two existing FDA approved treatments for ASD.
• In the Company’s exploratory, open-label Phase 2 BRIGHT trial in 37 children and adolescents with ASD, all five subscales of the ABC-C showed both statistically significant (p<0.0002) and clinically meaningful improvements at 14 weeks of treatment from baseline, including the irritability subscale which showed a 39.1% improvement (p<0.0001). Furthermore, as previously reported, the 18 patients who continued through 38 weeks of treatment in the extension portion of the BRIGHT trial saw a 56.1% improvement in the ABC-C irritability subscale compared to baseline (p<0.0001).
• In the United States, new estimates published in the U.S. Centers for Disease Control and Prevention’s Morbidity and Mortality Weekly Report confirm an increasing trend in the prevalence of ASD, with approximately one in 44 eight year old children (or approximately 1.4 million children in total, if extrapolated to include children ages 3 to 17 years) identified with ASD. This increasing prevalence underscores the significant unmet medical need, as well as opportunity, to develop effective and well-tolerated treatment alternatives for these patients, especially considering that there are currently only two FDA approved treatments available for patients with ASD, each of which are antipsychotics.

Zygel in 22q11.2 Deletion Syndrome (22q)

• The Company continues to screen and enroll patients for the 14-week open-label Phase 2 INSPIRE trial, and expects topline data mid-year 2022.
• In the fourth quarter of 2021, the Company initiated an additional clinical site in the United States to assist completion of enrollment.
• The Company plans to move forward in 22q as an orphan indication pending results from the ongoing Phase 2 INSPIRE trial in children and adolescents with genetically confirmed 22q, and subsequent discussion with the FDA on the regulatory path forward.

Zygel in Developmental and Epileptic Encephalopathies (DEE)

• Feedback received from the FDA indicated that selecting a specific DEE syndrome will be required for a pediatric indication in epilepsy rather than evaluating improvements in certain seizure types across all DEEs. During 2021, the Company evaluated individual syndromes and conducted a feasibility study. Based on Company research and strategic prioritization, the decision has been made to maintain focus on other important neuropsychiatric behavioral conditions and not to pursue further development in DEE at this time.

Financial Outlook
As a result of the pipeline update and anticipated additional clinical studies in ASD, management now believes the Company’s $75.6 million of cash and cash equivalents as of September 30, 2021 are sufficient to fund operations and capital requirements into the second half of 2023.

Conference Call Information
Zynerba management will host a live conference call and webcast tomorrow, January 5, 2022, at 9:00 a.m. Eastern Time to discuss updates to its clinical development plans. The call can be accessed by dialing (866) 573-0180 (U.S. and Canada) or (430) 775-1345 (international) and referencing conference ID 4181504. To access the live webcast or the replay, visit the investor page of the Company’s website at http://ir.zynerba.com/. The webcast will be recorded and available on the Company’s website for 30 days.

About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative 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, and 22q11.2 deletion syndrome. 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 expectations, projections and estimates regarding expenses, future revenue, capital requirements, incentive and other tax credit eligibility, collectability and timing, and availability of and the need for additional financing; 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 Administration 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; the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates; the timing and outcome of current and future legal proceedings; and the extent to which health epidemics and other outbreaks of communicable diseases, including COVID-19, could disrupt our operations or adversely affect our business and financial conditions. 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 Contacts
Peter Vozzo
ICR Westwicke
Office: 443.213.0505
Cell: 443.377.4767
Peter.Vozzo@Westwicke.com

Devon, PA, December 6, 2021  — Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced presentation details for The BRAIN Foundation Synchrony 2021 Symposium, being held virtually December 11-12, 2021. The presentation can be accessed by registering for free at https://synchronysymposium.com/. A copy of the presentation slides will be made available on the Zynerba corporate website following the presentation at http://zynerba.com/publications/.

Oral Presentation Details
Title:	ZYN002 Cannabidiol Transdermal Gel Efficacy and Safety: Recent Clinical Research Advances in the Treatment of Autism and Fragile X Syndrome
Date:	December 11, 2021
Time:	5:00 p.m. ET

About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative 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 expectations, projections and estimates regarding expenses, future revenue, capital requirements, incentive and other tax credit eligibility, collectability and timing, and availability of and the need for additional financing; 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 Administration 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; the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates; the timing and outcome of current and future legal proceedings; and the extent to which health epidemics and other outbreaks of communicable diseases, including COVID-19, could disrupt our operations or adversely affect our business and financial conditions. 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 Contacts
Peter Vozzo
ICR Westwicke
Office: 443.213.0505
Cell: 443.377.4767
Peter.Vozzo@Westwicke.com

Devon, PA, November 22, 2021 — 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 European Patent Office has issued Patent No. 3687513, titled “Treatment of Fragile X Syndrome and Autism with Cannabidiol,” which includes claims directed to methods of treating one or more behavioral symptoms of Fragile X syndrome and methods of treating one or more behavioral symptoms of autism spectrum disorder by transdermally administering a therapeutically effective amount of cannabidiol (CBD).

This new patent, which expires in 2038, is part of an expanding international intellectual property portfolio covering the Company’s transdermal cannabidiol product candidate, Zygel™ CBD gel. There are currently five corresponding U.S. patents related to methods of treating Fragile X syndrome or autism spectrum disorder.

About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative 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 expectations, projections and estimates regarding expenses, future revenue, capital requirements, incentive and other tax credit eligibility, collectability and timing, and availability of and the need for additional financing; 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 Administration 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; the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates; the timing and outcome of current and future legal proceedings; and the extent to which health epidemics and other outbreaks of communicable diseases, including COVID-19, could disrupt our operations or adversely affect our business and financial conditions. 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
Peter Vozzo
ICR Westwicke
Office: 443.213.0505
Cell: 443.377.4767
Peter.Vozzo@Westwicke.com

– Initiated RECONNECT, a confirmatory pivotal Phase 3 trial of Zygel™ in patients with Fragile X Syndrome (FXS); topline results expected second-half 2023 –

– Cash runway well into the first half of 2024; $75.6 million at September 30, 2021 –

Devon, PA November 15, 2021 — 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 third quarter ended September 30, 2021, and provided an overview of recent operational highlights and a pipeline update.

“We remain focused on enrolling patients into our confirmatory pivotal Phase 3 RECONNECT trial in children and adolescents with Fragile X Syndrome,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “If the results from RECONNECT are positive, Zygel has the potential to become the first FDA approved treatment option for the significant unmet medical need that affects FXS patients and their families.”

Third Quarter 2021 and Recent Highlights and Zygel Pipeline Update

Zygel in Fragile X Syndrome (FXS)

• Zynerba initiated a pivotal, multinational randomized, double-blind, placebo-controlled, multiple-center, efficacy and safety (RECONNECT) Phase 3 trial during the third quarter of 2021. The RECONNECT trial is designed to evaluate the efficacy and safety of Zygel (cannabidiol formulated in a transdermal gel) in children and adolescents with FXS. The trial is planned to confirm the positive results observed in a population of responders in the Company’s CONNECT-FX trial. (Press Release)
• RECONNECT is an 18-week trial that is expected to enroll approximately 200 children and adolescents, aged three through 17 years, at approximately 25 clinical sites in the United States, Australia, the UK and Ireland. Approximately 160 of the patients enrolled will have complete (100%) methylation of their FMR1 gene and approximately 40 patients will have partial methylation of their FMR1 gene. Patients will be randomized 1:1 to either Zygel or placebo. Randomization will be stratified by gender, methylation status and weight.
• The primary endpoint for the trial will be the change from baseline to the end of the treatment period in the Aberrant Behavior Checklist-Community FXS Specific (ABC-C_FXS) Social Avoidance subscale in patients who have complete methylation of their FMR1 gene. The ABC-C_FXS Social Avoidance subscale is the same primary endpoint used in the CONNECT-FX trial. The Company believes that the results from RECONNECT, if positive, will be sufficient to support the submission of a New Drug Application for Zygel in patients with FXS.
• Presented data at the American Academy of Child and Adolescent Psychiatry (AACAP) Annual Meeting demonstrating that more patients on Zygel attained and maintained clinically meaningful improvement in social avoidance as compared to placebo-treated patients during the double-blind treatment phase of CONNECT-FX. In addition, patients who received blinded Zygel in CONNECT-FX continued to improve on open-label Zygel with approximately two-thirds of patients achieving sustained meaningful improvement in social avoidance at 16 weeks and 24 weeks on Zygel.
• Presented data at the 2021 National Organization for Rare Disorders (NORD) Rare Diseases and Orphan Products Breakthrough Summit describing the design of the Phase 3 RECONNECT trial of Zygel in children and adolescents with FXS based on learnings from CONNECT-FX.

Zygel in Autism Spectrum Disorder (ASD)

• Presented data at The Society for the Study of Behavioural Phenotypes (SSBP) Conference 2021 demonstrating that through 38 weeks of treatment, the Phase 2 BRIGHT trial suggests initial evidence of a positive benefit-risk profile for Zygel when administered in addition to stable standard of care in children and adolescents with moderate-to-severe ASD. Furthermore, in patients who completed the 38-week treatment period in BRIGHT, statistically significant improvements compared to baseline were sustained in all efficacy measures of ASD compared to baseline. (Press Release)

Zygel in 22q11.2 Deletion Syndrome (22q)

• The Company continues to screen patients for the 14-week open-label Phase 2 INSPIRE trial in children and adolescents with genetically confirmed 22q.

Zygel in Developmental and Epileptic Encephalopathies (DEE)

• Published results from the Company’s open-label Phase 2 BELIEVE study in the Journal of the American Medical Association (JAMA) Network Open. The article describes the positive results from BELIEVE, the first trial of a non-oral formulation of cannabidiol in children and adolescents with DEEs, and concludes that Zygel was safe, well tolerated, and was associated with reductions in focal impaired awareness seizures (FIAS) and tonic-clonic seizure (TCS) frequency and disease burden. (Press Release)

Zynerba will provide an update on its development path forward in ASD, 22q and DEE by the end of 2021.

Corporate

• In November 2021, Stephen O’Quinn, Pharm.D. was appointed as the Vice President, Medical Affairs. Dr. O’Quinn is a senior pharmaceutical executive with more than 30 years of experience in clinical development, medical affairs and commercialization of medicines in multiple therapy areas, including neurology and psychiatry. Dr. O’Quinn spent over 20 years with GlaxoSmithKline in senior leadership roles. He most recently served as a consultant to pharmaceutical clients, including Zynerba Pharmaceuticals, supporting clinical development and medical affairs activities. Dr. O’Quinn earned a Doctor of Pharmacy, with highest honors, from the University of North Carolina at Chapel Hill. He completed a post-doctoral fellowship in Cardiovascular Pharmacotherapy with the UNC School of Pharmacy and Division of Cardiology. He also completed marketing and executive leadership training at The Wharton School of the University of Pennsylvania.

Third Quarter 2021 Financial Results

Research and development expenses were $6.3 million for the third quarter of 2021, including stock-based compensation of $0.8 million. General and administrative expenses were $3.9 million in the third quarter of 2021, including stock-based compensation expense of $0.8 million. The net loss for the third quarter of 2021 was $10.6 million, with basic and diluted loss per share of $(0.26).  

Financial Outlook

As of September 30, 2021, cash and cash equivalents were $75.6 million, compared to $59.2 million as of December 31, 2020. Management believes that the Company’s cash and cash equivalents as of September 30, 2021 are sufficient to fund operations and capital requirements well into the first half of 2024.

About Zynerba Pharmaceuticals, Inc.

Zynerba Pharmaceuticals is the leader in innovative 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 expectations, projections and estimates regarding expenses, future revenue, capital requirements, incentive and other tax credit eligibility, collectability and timing, and availability of and the need for additional financing; 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 Administration 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; the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates; the timing and outcome of current and future legal proceedings; and the extent to which health epidemics and other outbreaks of communicable diseases, including COVID-19, could disrupt our operations or adversely affect our business and financial conditions. 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 September 30,								Nine months ended September 30,
		2021				2020				2021				2020
Operating expenses:
Research and development	$	6,341,171			$	5,805,948			$	16,402,129			$	30,038,582
General and administrative		3,869,481				3,425,831				11,531,824				11,834,434
Total operating expenses		10,210,652				9,231,779				27,933,953				41,873,016
Loss from operations		(10,210,652	)			(9,231,779	)			(27,933,953	)			(41,873,016	)
Other income (expense):
Interest income		5,038				10,781				16,614				239,066
Foreign exchange (loss) gain		(376,637	)			172,467				(576,619	)			(85,171	)
Total other expense		(371,599	)			183,248				(560,005	)			153,895
Net loss	$	(10,582,251	)		$	(9,048,531	)		$	(28,493,958	)		$	(41,719,121	)
Net loss per share – basic and diluted	$	(0.26	)		$	(0.31	)		$	(0.73	)		$	(1.59	)
Basic and diluted weighted average shares outstanding		40,092,128				29,243,375				38,933,209				26,258,626
Non-cash stock-based compensation included above:
Research and development	$	818,390			$	544,909			$	2,443,667			$	1,590,285
General and administrative		751,603				717,716				2,325,512				2,343,125
Total	$	1,569,993			$	1,262,625			$	4,769,179			$	3,933,410

ZYNERBA PHARMACEUTICALS, INC.
CONSOLIDATED BALANCE SHEETS

	(unaudited)
	September 30, 2021				December 31, 2020
Assets
Current assets:
Cash and cash equivalents	$	75,642,498			$	59,157,187
Incentive and tax receivables		8,535,607				9,042,586
Prepaid expenses and other current assets		3,880,014				5,166,401
Total current assets		88,058,119				73,366,174
Property and equipment, net		447,728				585,403
Incentive and tax receivables		868,083				—
Right-of-use assets		622,240				105,199
Total assets	$	89,996,170			$	74,056,776
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable	$	2,636,350			$	2,522,716
Accrued expenses		8,053,949				11,280,843
Lease liabilities		207,635				109,689
Total current liabilities		10,897,934				13,913,248
Lease liabilities, long-term		411,237				—
Total liabilities		11,309,171				13,913,248
Stockholders’ equity:
Common stock		41,282				29,975
Additional paid-in capital		309,312,130				262,286,008
Accumulated deficit		(230,666,413	)			(202,172,455	)
Total stockholders’ equity		78,686,999				60,143,528
Total liabilities and stockholders’ equity	$	89,996,170			$	74,056,776

 

Zynerba Contacts

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

Peter Vozzo
ICR Westwicke
Office: 443.213.0505
Cell: 443.377.4767
Peter.Vozzo@Westwicke.com

Devon, PA, October 21, 2021 — Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced oral and poster presentations at the Society for Developmental & Behavioral Pediatrics Annual Meeting, being held virtually October 21-25, 2021, and the 68^th American Academy of Child & Adolescent Psychiatry Annual Meeting, being held virtually October 18-30, 2021.

Presentation Details

Society for Developmental & Behavioral Pediatrics (SDBP) Annual Meeting

Title	A Pivotal Study of ZYN002 Cannabidiol (CBD) Transdermal Gel in Children and Adolescents With Fragile X Syndrome [CONNECT-FX (ZYN2-CL-016)]
Presentation Type	Oral
Session	Platform 2: NDD Phenotypes and Treatment
Date	Saturday, October 23, 2021
Time	4:00 – 5:30 p.m. ET

 

Title	Longer Term Tolerability and Efficacy of ZYN002 Cannabidiol Transdermal Gel in Children and Adolescents with Autism Spectrum Disorder (ASD): An Open-Label Phase 2 Study (BRIGHT [ZYN2-CL-030])
Presentation Type	Poster
Session	Poster Session 2: Neurodevelopmental Disabilities, COVID-19
Date	Monday, October 25, 2021
Time	1:45 – 2:45 p.m.

 

68^th American Academy of Child & Adolescent Psychiatry (AACAP) Annual Meeting

Title	RECONNECT (ZYN2-CL-033): Design of a Phase 3 Trial of ZYN002 Cannabidiol Transdermal Gel in Children and Adolescents with Fragile X Syndrome Based Upon Learnings from CONNECT-FX (ZYN2-CL-016)
Presentation Type	Oral
Session	Research Pipeline: New Findings on Diagnostic and Therapeutics
Date	Wednesday, October 27, 2021
Time	3:15 p.m. – 5:30 p.m. ET

 

Title	Longer-Term Tolerability and Efficacy of ZYN002 Cannabidiol Transdermal Gel in Children and Adolescents With Autism Spectrum Disorder: An Open-Label Phase 2 Study (BRIGHT [ZYN2-CL-030])
Presentation Type	Oral
Session	Research Pipeline: New Findings on Diagnostic and Therapeutics
Date	Wednesday, October 27, 2021
Time	3:15 p.m. – 5:30 p.m. ET

 

Copies of the presentations and posters will be available on the Zynerba corporate website following each presentation at http://zynerba.com/publications/.

About Zynerba Pharmaceuticals, Inc.
Zynerba Pharmaceuticals is the leader in innovative 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 expectations, projections and estimates regarding expenses, future revenue, capital requirements, incentive and other tax credit eligibility, collectability and timing, and availability of and the need for additional financing; 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 Administration 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; the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates; the timing and outcome of current and future legal proceedings; and the extent to which health epidemics and other outbreaks of communicable diseases, including COVID-19, could disrupt our operations or adversely affect our business and financial conditions. 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
Peter Vozzo
ICR Westwicke
Office: 443.213.0505
Cell: 443.377.4767
Peter.Vozzo@Westwicke.com