Devon, PA, November 12, 2019 — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that the U.S. Patent and Trademark Office has issued US Patent No. 10,471,022, titled “Treatment of Fragile X Syndrome with Cannabidiol” which includes claims directed to a method of treating Fragile X syndrome, comprising transdermally administering 250 mg or 500 mg of cannabidiol (CBD) daily via a gel or cream.
This new patent, which expires in 2038, is part of an expanding intellectual property portfolio covering the Company’s transdermal CBD product candidate, Zygel™ (ZYN002 CBD gel).
Zygel is currently being studied in CONNECT-FX, a pivotal, multi-national, randomized, double blind, placebo-controlled study evaluating the efficacy and safety of Zygel in 204 three through 17-year old FXS patients with full mutation of the FMR1 gene. The primary endpoint is the change from baseline to the end of the treatment period in the Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS) Social Avoidance subscale. Patients who have completed the double-blind phase are now enrolling into the 12-month open label phase. The Company expects to report top line data in the first half of 2020.
About Zynerba Pharmaceuticals, Inc. Zynerba Pharmaceuticals is the leader in pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are committed to improving the lives of patients and their families living with severe, chronic health conditions including Fragile X syndrome, autism spectrum disorder, 22q11.2 deletion syndrome, and a heterogeneous group of rare and ultra-rare epilepsies known as developmental and epileptic encephalopathies. Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
Cautionary Note on Forward-Looking Statements This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Such statements are subject to numerous important factors, risks and uncertainties that may cause actual events or results to differ materially from the Company’s current expectations. For example, there can be no guarantee that the Company will obtain approval for ZYN002 from the U.S. Food and Drug Administration (FDA) or foreign regulatory authorities; even if ZYN002 are approved, the Company may not be able to obtain the label claims that it is seeking from the FDA. In addition, the Company’s cash and cash equivalents may not be sufficient to support its operating plan for as long as anticipated. Management’s expectations and, therefore, any forward-looking statements in this press release could also be affected by risks and uncertainties relating to a number of other factors, including the success and timing of the Company’s product development activities, studies and clinical trials and the Company’s expectations regarding its ability to obtain and adequately maintain sufficient intellectual property protection for its product candidates. This list is not exhaustive and these and other risks are described in the Company’s periodic reports, including the annual report on Form 10-K, quarterly reports on Form 10-Q and current reports on Form 8-K, filed with or furnished to the Securities and Exchange Commission and available at www.sec.gov. Any forward-looking statements that the Company makes in this press release speak only as of the date of this press release. The Company assumes no obligation to update forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.
Investor Contact William Roberts, Vice President, Investor Relations and Corporate Communications Zynerba Pharmaceuticals 484.581.7489 robertsw@zynerba.com
– Topline Results from Pivotal Trial in Fragile X Syndrome On Track for 1H2020 –
– Positive Topline Results from BELIEVE 1 Phase 2 Trial of Zygel™ in
Children and Adolescents with Developmental and Epileptic Encephalopathies Suggest
Compelling Seizure Reductions and Excellent Tolerability –
– Phase 2 Topline Results in Autism Spectrum Disorder and 22q11.2 Deletion
Syndrome On Track for 1H2020 –
Devon, PA, November 6, 2019 — Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today reported financial results for the third quarter ended September 30, 2019 and provided an overview of recent operational highlights.
“The third quarter of 2019 was a remarkable period of progress and execution for Zynerba,” said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We announced compelling topline safety and efficacy results from our six month BELIEVE Phase 2 trial of Zygel™ in childhood epilepsies. In this study, patients experienced median reductions in their most common and debilitating seizures of 44% or more starting at month two and continuing through month six. In this medically fragile patient population, and consistent with our prior trials, Zygel was very well tolerated. Caregivers also reported important improvements in seizure intensity and duration in their children, and in socio-behavioral and cognitive impairments that are common in this population. Finally, we continued to progress towards full enrollment in our pivotal CONNECT-FX trial in children and adolescents with Fragile X syndrome, and expect to announce topline results in the first half of next year.”
Presented Positive Topline Efficacy and Tolerability Results from BELIEVE
1 Open Label Phase 2 DEE Study
The topline results of this six month Phase 2 evaluation of Zygel in 48
children and adolescents with various DEEs showed meaningful reductions in
seizures and excellent tolerability. Patients experienced 44% to 58% monthly median
reductions in focal impaired-awareness seizures (FIAS; previously known as
complex partial seizures) and/or convulsive seizures (CS; focal to bilateral
tonic-clonic seizures and generalized tonic-clonic seizures), the
most common and debilitating seizure types, starting at month two and
continuing through month six. In addition, 42% to 63% of patients experienced a
≥50% monthly reduction in these seizures. Children with DEE are medically fragile,
and as such, adverse events (all events, whether unrelated or related to study drug,
that occur during the trial period) are common and expected. Only ten patients
experienced a serious adverse event (SAE). Of those ten, eight were deemed to
be unrelated to drug and only two were deemed possibly related to study drug,
including one case of lower respiratory tract infection and one case of status epilepticus,
both of which are common events in this patient population. There were no
drug-related hepatic, gastrointestinal, or lethargy-related SAEs observed
during this study.
Presented Qualitative Data Evaluating the Impact of Zygel on Quality of
Life of Children with DEE
As part of the BELIEVE 1 study, caregivers were asked to provide a
qualitative assessment regarding their child’s overall experiences during
treatment with Zygel. Caregiver feedback to a series of open-ended questions was
collected and coded by two independent reviewers. These qualitative assessments
indicated improvements in alertness, awareness, or energy (58% of caregivers);
seizures (51% of caregivers); cognition/concentration (47% of caregivers);
socially-avoidant behaviors (44% of caregivers); and school attendance (28% of caregivers).
Preparations Underway for First Half 2020 Meeting with U.S. Food and
Drug Administration (FDA) to Discuss Pathway for Zygel in DEE
Zynerba intends to meet with the FDA in the first half of 2020 to
discuss the clinical path forward in DEE. Based on the Phase 2 trial design and
results, the Company anticipates that it will discuss the pursuit of an
indication that includes all syndromes and encephalopathies in the DEE category
that present with FIAS and/or CS, the most common and debilitating seizure
types representing 75% to 80% of all seizures.
Zygel in Fragile X Syndrome (FXS)
Fragile X Syndrome Pivotal
Data Expected in the First Half of 2020
Enrollment is progressing in CONNECT-FX, a pivotal, multi-national,
randomized, double blind, placebo-controlled trial evaluating the efficacy and
safety of Zygel in treating common behavioral symptoms of FXS in three through
17-year old patients with FXS. The Company expects to report top line results
in the first half of 2020. The primary endpoint is
the change from baseline to the end of the treatment period in the Aberrant
Behavior Checklist-Community FXS Specific (ABC-CFXS) Social
Avoidance subscale. Key secondary endpoints are the change from baseline to the
end of the treatment period in the ABC-CFXS Irritability subscale
score, the ABC-CFXS Socially Unresponsive/Lethargic subscale score,
and improvement in Clinical Global Impression – Improvement (CGI-I) at the end
of the treatment period. If the results
are positive, the Company expects to submit its New Drug Application (NDA) for Zygel
in FXS to the U.S. Food and Drug Administration (FDA) in the second half of
2020, with potential approval by mid-year 2021.
Poster Further Validating
the Use of the ABC-CFXS Presented at the 22nd Society for
the Study of Behavioural Phenotypes (SSBP) Symposium
The poster described data collected via web-based journals and in-depth
interviews of caregivers of children with FXS. The data indicate that nine of
ten caregivers (90%) reported that their children had behaviors representative
of social avoidance, socially unresponsiveness/lethargic, and irritability and that
the described behaviors had strong concordance with individual domains of the
ABC-CFXS. We believe that these data help elucidate the most common
core behaviors of FXS, and further validate the appropriateness of the ABC-CFXS as
an effective tool for use in clinical studies as a means to measure
improvements in these core and common FXS behaviors.
Zygel 12-week Open Label Phase
2 FXS Data Published in the Journal of Neurodevelopmental Disorders
The results of the Phase 2 FAB-C clinical trial have been published in
the peer-reviewed Journal of
Neurodevelopmental Disorders in a paper entitled, ‘A Phase 1/2, Open Label
Assessment of the Safety, Tolerability, and Efficacy of Transdermal Cannabidiol
(ZYN002) for the Treatment of Pediatric Fragile X Syndrome’ (Heussler,
Helen; Cohen, Jonathan; Silove, Natalie, et al.).
Zygel in Autism Spectrum Disorder (ASD)
Phase 2 Open Label Trial
of Zygel in ASD Ongoing; Data Expected in the First Half of 2020
The Company is conducting the Phase 2 BRIGHT trial
to assess the safety, tolerability and efficacy of Zygel for the treatment of
child and adolescent patients with ASD. The 14-week trial is designed to
evaluate the efficacy and safety of Zygel in approximately 36 children and
adolescents (ages four through 17) with ASD as confirmed by DSM-5 diagnostic
criteria for ASD. The efficacy assessments include the Aberrant Behavior
Checklist, Parent Rated Anxiety Scale – Autism Spectrum Disorder, Autism Impact
Measure, and Clinical Global Impression – Severity and Improvement. Zynerba
expects to report topline results from this study in the first half of 2020.
Poster Describing the Shared Sociobehavioral
Symptoms in ASD, FXS, and 22q11.2 Deletion Syndrome Presented at the 22nd
SSBP Symposium
The poster described the results of a
retrospective literature review on patients with ASD, FXS, and 22q11.2 deletion
syndrome (22q) conducted to determine symptomatic overlap between these
disorders. The data indicate that patients with ASD, FXS, and 22q 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.
Zygel in 22q11.2 Deletion Syndrome (22q)
Phase 2 Open Label Trial
of Zygel in 22q Ongoing; Data Expected in the First Half of 2020
The Company is conducting the 14-week Phase 2
INSPIRE trial to evaluate the safety, tolerability and efficacy of Zygel in
approximately 20 children and adolescents (ages six through 17) with
genetically-confirmed 22q. 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. Zynerba expects to report
topline results from this study in the first half of 2020.
Third quarter 2019
Financial Results
Our Australian subsidiary, Zynerba
Pharmaceuticals Pty Ltd, or the Subsidiary, is incorporated in Australia and is
eligible to participate in an Australian research and development tax incentive
program. As part of this program, the
Subsidiary is eligible to receive a cash refund from the Australian Taxation
Office for a percentage of the research and development costs expended by the
Subsidiary in Australia. In July 2019, the Australian government’s Department
of Industry, Innovation and Science, or AusIndustry, responded to an Advance
Overseas Finding, or AOF, application submitted by Zynerba that will allow
certain research and development expenses incurred with respect to our product
candidate Zygel outside of Australia to be eligible for the Australian research
and development tax incentive program. As a result of this finding, we are
eligible to receive a cash refund from the Australian Taxation Office for the
qualifying research and development costs expended outside of Australia in
2018, 2019 and 2020. During the three months ending September 30, 2019, we
recorded an $8.3 million credit to research and development expenses for
amounts expected to be received through the AOF for the period from January 1,
2018 through September 30, 2019. Although the AOF approval extends into 2020,
management believes that substantially all qualifying amounts have been
recorded as of September 30, 2019.
The following table summarizes research and
development expenses for the three months ended September 30, 2019 and 2018.
Excluding the
$8.3 million reduction in research and development expenses for amounts
expected to be received through the AOF for the period from January 1, 2018
through September 30, 2019, research and development expenses increased by $1.8
million to $6.7 million for the three months ended September 30, 2019 from
$4.9 million for the three months ended September 30, 2018. The increase
was primarily related to an increase in clinical trial and manufacturing costs
related to our Zygel program. Stock based compensation included in the R&D
costs were $0.6 million.
General and administrative expenses for the third
quarter of 2019 were $3.5 million, including stock-based compensation expense
of $0.8 million.
The net loss for the third quarter of 2019
was $1.9 million with basic and diluted net loss per share of $(0.08).
Financial
Outlook
The Company’s cash and cash equivalent
position as of September 30, 2019 was $77.5 million. Management believes that
the cash and cash equivalent position is sufficient to fund operations and
capital requirements beyond the expected NDA submission and potential approval
of Zygel in FXS and into the second half of 2021.
About Zynerba
Pharmaceuticals, Inc.
Zynerba
Pharmaceuticals is the leader in pharmaceutically-produced transdermal
cannabinoid therapies for rare and near-rare neuropsychiatric disorders. We are
committed to improving the lives of patients and their families living with
severe, chronic health conditions including Fragile X syndrome, autism spectrum
disorder, 22q11.2 deletion syndrome, and a heterogeneous group of rare and
ultra-rare epilepsies known as developmental and epileptic encephalopathies.
Learn more at www.zynerba.com and follow us on Twitter at @ZynerbaPharma.
Cautionary Note on
Forward-Looking Statements
This press release contains forward-looking statements
within the meaning of The Private Securities Litigation Reform Act of 1995. We
may, in some cases, use terms such as “predicts,” “believes,” “potential,”
“proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,”
“intends,” “may,” “could,” “might,” “will,” “should” or other words that convey
uncertainty of future events or outcomes to identify these forward-looking statements.
Such statements are subject to numerous important factors, risks and
uncertainties that may cause actual events or results to differ materially from
the Company’s current expectations. Management’s expectations and, therefore,
any forward-looking statements in this press release could also be affected by
risks and uncertainties relating to a number of other factors, including the
following: the Company’s cash and cash equivalents may not be sufficient to
support its operating plan for as long as anticipated; the Company’s ability to
obtain additional funding to support its clinical development programs; the
results, cost and timing of the Company’s clinical development programs,
including any delays to such clinical trials relating to enrollment or site
initiation; clinical results for the Company’s product candidates may not be
replicated or continue to occur in additional trials and may not otherwise
support further development in a specified indication or at all; actions or
advice of the U.S. Food and Drug 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; and the
Company’s expectations regarding its ability to obtain and adequately maintain
sufficient intellectual property protection for its product candidates. This
list is not exhaustive and these and other risks are described in the Company’s
periodic reports, including the annual report on Form 10-K, quarterly reports
on Form 10-Q and current reports on Form 8-K, filed with or furnished to the
Securities and Exchange Commission and available at www.sec.gov. Any
forward-looking statements that the Company makes in this press release speak
only as of the date of this press release. The Company assumes no obligation to
update forward-looking statements whether as a result of new information,
future events or otherwise, after the date of this press release.
ZYNERBA PHARMACEUTICALS, INC.
CONSOLIDATED STATEMENTS OF
OPERATIONS
(unaudited)
ZYNERBA PHARMACEUTICALS, INC.
CONSOLIDATED BALANCE SHEETS
Zynerba Contacts Jim Fickenscher, CFO and VP Corporate Development Zynerba Pharmaceuticals 484.581.7483 fickenscherj@zynerba.com
Will Roberts, VP Investor Relations and Corporate CommunicationsZynerba Pharmaceuticals 484.581.7489 robertsw@zynerba.com
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