Month: September 2019

Devon, PA, September 26, 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 2019 Cantor Global Healthcare Conference. The presentation will take place on Thursday October 3^rd at 8:55 AM EDT at the InterContinental New York Barclay. A live webcast of the presentation will be accessible on the Investor Relations page of http://www.zynerba.com.

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 

– Study Achieves a 44% Median Seizure Reduction in Focal Impaired Awareness (Complex Partial) and Convulsive Seizures in DEE Patients by Month Two; Reductions were Sustained through Month Six of Treatment with Zygel –

– At Least 42% of these Patients Experienced a ≥50% Improvement from Month Two through Month Six –

– Qualitative Assessments Demonstrate Improvements in Seizure Intensity and Duration, and Socio-behavioral and Cognitive Impairments –

Devon, PA, September 18, 2019  — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced positive top line results from the open label Phase 2 BELIEVE 1 (Open Label Study to Assess the Safety and Efficacy of Zygel™ (ZYN002) Administered as a Transdermal Gel to Children and Adolescents with Developmental and Epileptic Encephalopathy) clinical trial. The trial assessed the safety and efficacy of Zygel in developmental and epileptic encephalopathies (DEE), a heterogeneous group of rare pediatric epilepsy syndromes, including Dravet syndrome (DS) and Lennox-Gastaut syndrome (LGS). DEE is characterized by the presence of multiple focal and generalized seizure types and severe cognitive and behavioral impairment. The most common and debilitating seizure types in people with epilepsy are focal impaired-awareness and convulsive seizures. Patients who experienced these seizure types achieved 44% to 58% monthly median reductions in seizures compared to baseline from month two to month six of treatment with Zygel. Further, qualitative assessments by caregivers in the study demonstrate that use of Zygel may result in improved socio-behavioral and cognitive symptoms of DEE. Zygel was also well tolerated in this study.

“The data from the BELIEVE 1 clinical trial are promising and suggest that Zygel may reduce seizure frequency in many types of difficult to treat developmental and epileptic encephalopathies and improve important behavior deficits, alertness, social interactions, and enable the child to be well enough to attend school more consistently,” said Ingrid Scheffer, AO, MBBS, PhD, FRACP, Professor and Chair, Paediatric Neurology Research, The University of Melbourne, and an investigator in the BELIEVE 1 trial. “DEE are the most challenging and poorly controlled epilepsy disorders with many symptoms that adversely affect patient and family function. I believe that this drug holds promise as a potential treatment for DEE.”

“We are encouraged by the positive top line results of the BELIEVE 1 trial of Zygel in children and adolescents with DEE, and we believe these data represent an important step forward for these patients and their families,” said Armando Anido, Zynerba’s Chairman and Chief Executive Officer. “These results suggest that Zygel may produce clinically meaningful reductions in seizures and may improve many of the difficult behaviors and symptoms, such as seizure intensity, fatigue, social isolation, poor cognition, and language deficits. Once we complete our analyses of the data, we intend to seek a meeting with the FDA, likely in the first half of next year, to discuss the clinical pathway to approval.”

Study Design

The six-month BELIEVE 1 clinical trial is an exploratory open label multi-dose Phase 2 clinical trial designed to evaluate the safety and efficacy of Zygel in children and adolescents (three to <18 years) with DEE as classified by the International League Against Epilepsy (ILAE) (Scheffer et al. 2017). Forty-eight patients with confirmed DEE were enrolled in the clinical trial and are included in the safety data for the trial. Forty-six patients are included in the modified intent-to-treat population (mITT). The two patients excluded from the intent-to-treat population included one patient who did not complete 80% of their seizure diaries and a second patient who did not complete a minimum of eighty days of treatment. Enrolled patients received weight-based initial doses of 250 mg or 500 mg daily of Zygel. Patients could be titrated up to 1,000 mg daily.

Baseline Patient Demographics

The BELIEVE 1 trial enrolled 48 patients between the ages of three and 16 (mean=10.5; median=10.0). Fifty-four percent of patients were male, and 46% were female. Patients weighed between 14.3 and 110 kilograms (mean=39.3; median=36.1). Patient BMI ranged between 12.5 and 35.4 (mean=19.2; median=18.6).

Top-line Efficacy Results

Of the 46 patients in the mITT population, 33 (72%) had focal impaired-awareness seizures (FIAS; previously known as complex partial seizures) and/or convulsive seizures (focal to bilateral tonic-clonic seizures and generalized tonic-clonic seizures) at baseline. These patients experienced a mean baseline seizure count of 64 FIAS and/or convulsive seizures, and a median baseline seizure count of 8.2 FIAS and/or convulsive seizures. Compared to baseline seizure frequency, these patients experienced a ≥44% median reduction in seizures from month two onwards using monthly seizure frequency normalized to 28 days (SF28).

Fifty-five percent (55%) of patients with FIAS and/or convulsive seizures experienced a ≥50% median reduction in seizures at month six of treatment with Zygel.

FIAS and convulsive seizures	Month 1 (n=33)	Month 2 (n=33)	Month 3 (n=33)	Month 4 (n=32)	Month 5 (n=32)	Month 6 (n=29)
Median % reduction in seizure frequency	16%	44%	44%	47%	58%	51%
≥50% responder rate	30%	42%	46%	47%	63%	55%

Thirteen of the 46 patients had a variety of non-FIAS and non-convulsive seizure types at baseline. The number of individual seizure types in these patients was too small to draw definitive conclusions and further analyses are warranted. 

Patients with either DS or LGS who experienced FIAS and/or convulsive seizures (n=11) experienced a 51% median reduction in FIAS and/or convulsive seizures at month six of treatment compared to baseline. Sixty percent (60%) of patients with DS or LGS experienced a ≥50% median reduction in FIAS or convulsive seizures at month six of treatment with Zygel.

Lennox-Gastaut and Dravet syndromes	Month 1 (n=11)	Month 2 (n=11)	Month 3 (n=11)	Month 4 (n=11)	Month 5 (n=11)	Month 6 (n=10)
Median % reduction in seizure frequency	18%	6%	46%	23%	63%	51%
≥50% responder rate	36%	36%	46%	40%	64%	60%

Safety data

Zygel was well tolerated, and the safety profile was consistent with previously released data from Zygel clinical trials. Eight patients discontinued the study; one discontinued as a result of an application site reaction, and seven discontinued as a result of withdrawal of consent or perceived lack of efficacy. Through six months of therapy, ninety-six percent (96%) of patients experienced a treatment emergent adverse event (TEAE) and 60% of patients experienced a treatment related adverse event. Most were mild to moderate. The most common treatment related adverse events (in >5% of patients) are application site dryness (8.3%), application site pain (8.3%), and somnolence (8.3%). Ten patients reported a serious adverse event (SAE); most were infection-related. Two SAEs (lower respiratory tract infection and status epilepticus) were determined to be possibly related to treatment. There were no patient deaths during the study.

Qualitative analysis of the impact of Zygel on behavioral and cognitive symptoms

Parents and caregivers were asked to provide a qualitative assessment regarding their child’s overall experiences during treatment with Zygel. The experiences of 43 patients are summarized below.

• 58% reported improved vitality (e.g. alertness / awareness, energy)
• 51% reported improvement in seizures
• 47% reported improved cognition and concentration
• 44% reported improved socially avoidant behaviors
• 28% reported that their child attended school on time / more often
• 26% reported difficulty in application of the gel to their child (e.g. time it takes for gel to dry)

About Developmental and Epileptic Encephalopathies (DEE)
DEE is a heterogeneous group of epilepsy syndromes that may be associated with severe cognitive impairment and behavioral disturbances. These disorders are often progressive, and are highly resistant to treatment. DEE includes a number of rare and ultra-rare epilepsy syndromes including early myoclonic encephalopathy, epileptic encephalopathy with continuous spike and wave during sleep, and certain syndromes including Ohtahara, West, Landau-Kleffner, Lennox-Gastaut, Doose and Dravet. Improved seizure control may have a positive impact on development and quality of life.

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

Media contact
Molly Devlin
Evoke KYNE
215.928.2199
Molly.Devlin@evokegroup.com

– Preliminary Evidence Shows that CBD May Prove to be Effective in Managing the Spectrum of Behavioral Symptoms Associated with these Conditions –

– New Data Presented Today at the Society for the Study of Behavioural Phenotypes (SSBP) Research Symposium –

Devon, PA, September 5, 2019  — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, is presenting data today describing the results of a retrospective literature review on patients with autism spectrum disorder (ASD), Fragile X syndrome (FXS), and 22q11.2 deletion syndrome (22qDS) conducted to determine symptomatic overlap between these disorders. The data indicate that patients with ASD, FXS, and 22qDS share a constellation of sociobehavioral symptoms and that the pharmacology of CBD is broad, continues to be defined, and may prove to be beneficial in addressing important behavioral symptoms of these conditions.

The poster, entitled Common Behavioral Features of Autism, Fragile X Syndrome, and 22q11.2 Deletion Syndrome, is being presented on September 5^th and 6^th at the 22^nd Society for the Study of Behavioural Phenotypes (SSBP) Research Symposium at Aston University in Birmingham, UK. A copy of the poster is available on the Zynerba corporate website at http://zynerba.com/publications/.

Honey Heussler, MBBS, FRACP, MRCPCH, PGCAP, DM, Associate Professor, University of Queensland and Medical Director Child Development, Children’s Health Queensland is presenting data showing that patients with ASD, FXS, and 22qDS share a constellation of sociobehavioral symptoms that includes anxiety, which may lead to social avoidant behavior, aggression, irritability, attention deficits, and poor communication. Dr. Heussler is presenting the poster from 12:40 – 1:45 British Summer Time (BST) on September 5^th and 6^th, 2019.

“Those of us who care for patients and families contending with certain neuropsychiatric dysfunction understand that there are significant shared sociobehavioral symptoms between such disorders, though until now no review has been conducted to examine or clarify the overlap,” said Dr. Heussler. “These data on the shared behaviors between ASD, FXS and 22qDS are important to understanding disease impact, patient care, and the development of potential treatments. One such potential treatment being studied in well-controlled clinical trials is a proprietary gel formulation of CBD, which has diverse pharmacologic effects and may prove to be important in these neuropsychiatric disorders.”

Based on insights from Company data, a search of the PubMed database was conducted using the terms “behavior,” “behavioral symptoms,” “autism spectrum disorder,” “ASD,” “Fragile X Syndrome,” “FXS,” “22q11.2 deletion syndrome,” “parents,” “caregivers,” and “CBD and treatment of anxiety” with no restriction on date or publication type. Records were then analyzed for relevance. The most common behavioral manifestations across all conditions are anxiety-related, such as social avoidance, aggression, irritability, attention deficits, stereotypy, poor communication, and social unresponsiveness.

ASD

Anxiety-related symptoms are common in patients with ASD with up to 84% of children experiencing some degree of debilitating anxiety. Rates of physician-diagnosed anxiety disorders in these patients range from 42% to 55% and may include simple phobias, generalized anxiety disorder, separation anxiety disorder, obsessive-compulsive disorder, and social phobias. Comorbid anxiety disorders can be associated with behaviors such as aggression/irritability and isolation from same-age peers (due to bullying/victimization in school). Inattention and hyperactivity are often present in Attention Deficit-Hyperactivity Disorder and ASD.

FXS

In FXS, severe cognitive and social impairments are more common in males than in females. FXS usually has profound effects on the life of patients (comorbid conditions, social impairment) as well as their caregivers and families (mental health, absence from work/school). Anxiety and social avoidance are considered core features of FXS; further, anxiety can be thought of as a foundational precipitant to social avoidance. Social avoidance encompasses behaviors that may include seeking isolation, lack of interaction, social escape, and gaze avoidance that distance the individual from his/her social counterparts.

22qDS

The most common behavioral/psychiatric diagnoses in children with 22qDS were ADHD, ASD, and anxiety. Up to one-third of patients with 22qDS will develop schizophrenia or schizo-affective disorder by late adolescent and early adulthood. The emergence of social deficits during adolescence can represent a major source of disability in some individuals with 22qDS. Cross-sectional studies (observational research that analyzes data collected at one given point of time across a sample population) show that children with 22qDS are withdrawn and shy, and have social impairments which may be less of a concern to the individual.

The Potential Role of CBD in Managing Behavioral Symptoms Associated with ASD, FXS, and 22qDS
The authors conclude that preliminary evidence, including findings from the Phase 2 open-label FAB-C study in children and adolescents with FXS and a retrospective literature review based on insights from that Company data, shows that CBD improves social anxiety and associated behavioral manifestations suggesting that it may prove to be effective in managing the spectrum of behavioral symptoms associated with these conditions.

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.

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

Media contact
Molly Devlin
Evoke KYNE
215.928.2199
Molly.Devlin@evokegroup.com

– Ninety Percent of Interviewed Caregivers Reported Social Avoidant, Irritability, and Socially Lethargic Behaviors in their Children with FXS –

– New Data Presented Today at the Society for the Study of Behavioural Phenotypes (SSBP) Research Symposium –

Devon, PA, September 5, 2019 — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, is presenting data today evaluating the Social Avoidance, Irritability, and Socially Unresponsive/Lethargic subscales of the Aberrant Behavior Checklist Community: FXS Specific (ABC-C_FXS) in relation to the experience of caregivers caring for a child with Fragile X syndrome. The poster describes data collected via web-based journals and in-depth interviews of caregivers of children with Fragile X syndrome (FXS). The data indicate that nine of ten caregivers reported their children had behaviors representative of social avoidance, socially unresponsiveness/lethargic, and irritability and the behaviors described had strong concordance with individual items of the ABC-C_FXS.

The poster, entitled Content Validity of the ABC-C_FXS and Subscales in Fragile X Syndrome, is being presented on September 5^th and 6^th at the 22^nd Society for the Study of Behavioural Phenotypes (SSBP) Research Symposium at Aston University in Birmingham, UK. A copy of the poster is available on the Zynerba corporate website at http://zynerba.com/publications/.

Terri Browning Sebree, Zynerba’s President, is presenting data providing qualitative evidence of the appropriateness of Social Avoidance, Irritability, and Socially Unresponsive/Lethargic subscales of the ABC-C_FXS in assessing core symptoms of FXS. In addition, the data further validate social avoidance, irritability, and socially unresponsive/lethargic behaviors as core FXS behavioral symptoms from the perspective of caregivers of children with the disorder. The Company is utilizing the ABC-C_FXS to assess improvements in core behaviors of FXS in the ongoing 14-week pivotal CONNECT-FX study of Zygel™ (CBD gel). Ms. Sebree will present the poster from 12:40 to 1:45 British Summer Time (BST) on September 5^th and 6^th, 2019.

“Fragile X syndrome is a complex diagnosis for a child and his/her family, marked by a myriad of specific behavioral and emotional symptoms often manifesting as anxiety and social avoidant behaviors,” said Ms. Sebree. “Through studies like this one, we are able to not only elucidate the most common core behaviors of FXS, but also further validate the appropriateness of the ABC-C_FXS as an effective tool for use in clinical studies as a means to measure improvements in these core and common FXS behaviors.”

Ten caregivers of children formally diagnosed with FXS participated in this study with assistance from the National Fragile X Foundation (NFXF) via online invitation through the NFXF website. The study found that 90% of caregivers reported at least one behavior that was representative of social avoidance, socially unresponsive/lethargic, and irritability

Social Avoidance

It was common for their children with FXS to prefer the company of single family members to groups of people, even friends, and to seek isolation from others either via physical setting (staying in their room or in the car) or blocking out the world (using headphones). Social avoidance had a negative impact on important activities, such as travel, schooling, or visits to the doctor.

The social-avoidant behaviors reported by caregivers, including ‘seeks isolation from others’, ‘prefers to be alone’, and ‘prefers solitary activities’ corresponded to items on the Social Avoidance subscale, including seeks isolation from others and isolates himself/herself from other children or adults

Irritability

Irritability was associated with a broad spectrum of verbal (talking back, hollering, articulate yelling, non-verbal screams) and physical behaviors (hitting siblings, overturning furniture) at inappropriate times.

Frequently reported aspects of misbehavior and irritability mapped onto items of the Irritability subscale, specifically ‘aggressive to others’, ‘irritability’, ‘temper tantrums/outbursts’, ‘screaming/yelling inappropriately’, ‘harming of others’, and ‘stubbornness’.

Socially Unresponsive/Lethargic

Caregivers frequently reported of ‘lack of interaction’ and ‘lack of attention’ in their children with FXS, which mapped to subscale items such as ‘preoccupied’, ‘stares into space’, ‘unresponsive to structured activities (does not react)’, and ‘shows few social reactions to others’. Items on the subscale pertaining to communication were identified as important by caregivers, particularly with respect to how they may exacerbate other syndrome-related behaviors.

The authors conclude that these data further validate social avoidance, irritability, and socially unresponsive/lethargic as core phenotypic behaviors of children with FXS and show that caregiver experiences correspond with ABC-C_FXS subscales, further validating the appropriateness of the ABC-C_FXS as an effective assessment tool in clinical trials.  

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.

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

Media contact
Molly Devlin
Evoke KYNE
215.928.2199
Molly.Devlin@evokegroup.com

Devon, PA, September 3, 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 two upcoming conferences in New York City.

• The HC Wainwright 21^st Annual Healthcare conference on Monday September 9, 2019 at 9:10AM EDT at the Lotte New York Palace Hotel; and
• The Ladenburg Thalmann Healthcare Conference on Tuesday, September 24, 2019 at 2:30PM EDT at the Sofitel Hotel.

A live webcast of the presentations will be accessible on the Investor Relations page of http://www.zynerba.com. A replay of the webcasts 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