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, 2020, and provided an overview of recent operational
highlights.
“We made good clinical, operational and regulatory progress
during the third quarter of 2020 including presenting new data from
the pivotal CONNECT-FX and Phase 2 BRIGHT trials, and completing
our discussions with the FDA to clarify our clinical path forward
to late stage clinical trials in patients with certain
developmental and epileptic encephalopathies,” said Armando Anido,
Chairman and Chief Executive Officer of Zynerba. “The fourth
quarter of this year is another important period for Zynerba. In
particular, we look forward to announcing the results of our fourth
quarter meeting with the FDA to discuss our pivotal CONNECT-FX
results in patients with a fully methylated FMR1 gene and to
understand the regulatory path forward.”
Third quarter 2020 and
Recent Highlights
Zygel in Fragile X Syndrome
(FXS)
Zynerba Expects to Announce the Outcome of its Fourth Quarter
Meeting with the U.S. Food and Drug Administration (FDA) to Discuss
the CONNECT-FX Trial and the Regulatory Path Forward for Zygel™ in
Pediatric and Adolescent Patients with a Fully Methylated FMR1 Gene
(FMet) in the Fourth Quarter of 2020 (Press release)
Presented New CONNECT-FX Data Supporting FMR1 Methylation Status
as a Correlate to Fragile X Syndrome Severity at the Virtual Joint
16th International Child Neurology Congress (ICNC) & 49th
Annual Child Neurology Society (CNS) Meeting
Zynerba utilized psychometric analyses to determine what
constitutes a clinically meaningful change from baseline as
measured by subscales of the ABC-CFXS. The results of these
analyses defined a clinically meaningful treatment response over 12
weeks of treatment as an improvement of three points or greater for
the Social Avoidance subscale, nine points or greater for the
Irritability subscale, and five points or greater for the Socially
Unresponsive / Lethargic subscale. Through this analysis, the
Company determined that 58.2% of FMet patients receiving Zygel
achieved a clinically meaningful change in their socially avoidant
behavior compared to 40.6% of patients receiving placebo (p=0.031;
statistically significant), and 40.3% of patients receiving Zygel
achieved a clinically meaningful change in Irritability compared to
23.8% of patients receiving placebo (p=0.036; statistically
significant). (Press release)
Presented CONNECT-FX Data Showing Statistically Significant
Caregiver-Reported Improvements in Most Impactful FXS Behaviors at
the 17th NFXF International Conference Research Roundup; Results
Support Statistically Significant Results of Pre-planned Ad Hoc
Analysis in FMet Patients
Consistent with guidance from the FDA on capturing the voice of
the patient in drug development, the Company collected qualitative
data on the clinical relevance of various FXS behaviors to
caregivers during CONNECT-FX. The results of the Qualitative
Caregiver Reported Behavioral Survey indicate that caregivers found
anxiety, socially avoidant behaviors, and disruptive behaviors to
be the most challenging. The results of the Caregiver Global
Impression – Change survey show a broad shift toward global
improvement from baseline to week 12 in FMet patients, with three
of the four behavioral domains (social avoidance and isolation,
irritable and disruptive behaviors, and social interactions)
showing statistically significant improvements in favor of patients
on Zygel compared to placebo and the fourth domain (overall
behavior) trending toward significance. (Press release)
Received New U.S. Patent for Treatment of Fragile X Syndrome
with Cannabidiol
The U.S. Patent and Trademark Office issued US Patent No.
10,758,497, titled “Treatment of Fragile X Syndrome with
Cannabidiol” which includes claims directed to a method of treating
FXS comprising administering 250mg or 500mg of synthetic or
purified cannabidiol in a pharmaceutically acceptable carrier to a
person in need thereof. This new patent, which expires in 2038, is
part of an expanding intellectual property portfolio covering
Zygel. (Press release)
Zygel in Autism
Spectrum Disorder (ASD)
Zynerba Intends to Discuss the Results of the Phase 2 BRIGHT
Trial in Children and Adolescents with Moderate to Severe ASD and
the Path Forward with the FDA in the First Half of 2021
Presented New Data Describing Statistically Significant Results
from the Phase 2 BRIGHT Trial in Patients with Autism Spectrum
Disorder (ASD) at the Virtual Joint 16th International Child
Neurology Congress (ICNC) & 49th Annual Child Neurology Society
(CNS) Meeting
Patients receiving Zygel in this study achieved statistically
significant caregiver-reported improvements compared to baseline
across all subscales of the Autism Impact Measure, which was
designed to measure change in frequency and impact of core ASD
symptoms: Atypical behavior (p<0.001), Communication
(p<0.001), Peer Interaction (p<0.001), Repetitive Behavior
(p<0.001), and Social Reciprocity (p=0.0053). In addition,
statistically significant improvements compared to baseline were
observed at week 14 of treatment with Zygel in the Autism Parenting
Stress Index (p<0.0001). Zynerba also measured notable
improvements in behaviors utilizing the Qualitative Caregiver
Behavioral Problems Survey after 14 weeks of study drug. Clinically
meaningful improvements were observed by a majority of surveyed
caregivers in behavioral, social and emotional behavioral problems.
(Press release)
Zygel in 22q11.2
Deletion Syndrome (22q)
Received Orphan Drug Designation for Cannabidiol for the
Treatment of 22q11.2 Deletion Syndrome
The FDA granted orphan drug designation for cannabidiol for use
in treating 22q, a rare midline condition featuring physical
abnormalities and debilitating neuropsychiatric and behavioral
symptoms including anxiety, withdrawn behavior, and social
interaction problems. Companies receiving orphan drug designation
may be entitled to various incentives, including tax credits for
qualified clinical studies, waiver of new drug application (NDA) /
biologics license application (BLA) user fees, and eligibility for
a seven-year exclusive marketing period for that drug and use upon
marketing approval. (Press release)
Zygel in Developmental and Epileptic
Encephalopathies (DEE)
Concluded Successful Discussions with FDA Regarding the DEE
Program and Path Forward; Evaluation of Initial Targets for Late
Stage Clinical Evaluation Progressing
Zynerba concluded its iterative discussions with the FDA
utilizing their ‘Written Response Only’ (WRO) meeting format
regarding the clinical pathway for Zygel in DEE during which the
FDA expressed support for a development program which would
evaluate the treatment of focal-impaired awareness and convulsive
seizures. Due to the heterogeneity of patients who fall under the
DEE umbrella, Zynerba will pursue individual syndromes rather than
considering DEE as a single disorder or condition. The Company is
in the process of finalizing its evaluation of which epileptic
syndromes it may pursue with Zygel, and expects to disclose its
first clinical target around year end 2020. (Press release)
Third quarter 2020
Financial Results
As of September 30, 2020, cash and cash equivalents were $64.3
million, compared to $70.1 million as of December 31, 2019.
Research and development expenses for the third quarter of 2020
were $5.8 million, including stock-based compensation of $0.5
million. General and administrative expenses for the third quarter
of 2020 were $3.4 million, including stock-based compensation
expense of $0.7 million. The net loss for the third quarter of 2020
was $9.0 million with basic and diluted net loss per share of
$(0.31).
Financial Outlook
Management believes that the current cash and cash equivalents
is sufficient to fund operations and capital requirements until
late in the fourth quarter of 2021.
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, |
|
|
2020 |
|
|
|
2019 |
|
|
|
2020 |
|
|
|
2019 |
|
Operating
expenses: |
|
|
|
|
|
|
|
Research and development |
$ |
5,805,948 |
|
|
$ |
(1,604,399 |
) |
|
$ |
30,038,582 |
|
|
$ |
12,926,096 |
|
General and administrative |
|
3,425,831 |
|
|
|
3,530,617 |
|
|
|
11,834,434 |
|
|
|
9,977,550 |
|
Total operating expenses |
|
9,231,779 |
|
|
|
1,926,218 |
|
|
|
41,873,016 |
|
|
|
22,903,646 |
|
Loss from operations |
|
(9,231,779 |
) |
|
|
(1,926,218 |
) |
|
|
(41,873,016 |
) |
|
|
(22,903,646 |
) |
Other income
(expense): |
|
|
|
|
|
|
|
Interest income |
|
10,781 |
|
|
|
436,846 |
|
|
|
239,066 |
|
|
|
1,226,998 |
|
Foreign exchange gain (loss) |
|
172,467 |
|
|
|
(457,018 |
) |
|
|
(85,171 |
) |
|
|
(551,944 |
) |
Total other income (expense) |
|
183,248 |
|
|
|
(20,172 |
) |
|
|
153,895 |
|
|
|
675,054 |
|
Net loss |
$ |
(9,048,531 |
) |
|
$ |
(1,946,390 |
) |
|
$ |
(41,719,121 |
) |
|
$ |
(22,228,592 |
) |
|
|
|
|
|
|
|
|
Net loss per
share - basic and diluted |
$ |
(0.31 |
) |
|
$ |
(0.08 |
) |
|
$ |
(1.59 |
) |
|
$ |
(1.03 |
) |
|
|
|
|
|
|
|
|
Basic and
diluted weighted average shares outstanding |
|
29,243,375 |
|
|
|
23,186,410 |
|
|
|
26,258,626 |
|
|
|
21,598,764 |
|
|
|
|
|
|
|
|
|
Non-cash
stock-based compensation included above: |
|
|
|
|
|
|
|
Research and development |
$ |
544,909 |
|
|
$ |
573,446 |
|
|
$ |
1,590,285 |
|
|
$ |
1,915,578 |
|
General and administrative |
|
717,716 |
|
|
|
802,779 |
|
|
|
2,343,125 |
|
|
|
2,438,644 |
|
Total |
$ |
1,262,625 |
|
|
$ |
1,376,225 |
|
|
$ |
3,933,410 |
|
|
$ |
4,354,222 |
|
|
|
|
|
|
|
|
|
ZYNERBA PHARMACEUTICALS,
INC.CONSOLIDATED BALANCE SHEETS
|
(unaudited) |
|
|
|
September 30, 2020 |
|
December 31, 2019 |
Assets |
|
|
|
Current
assets: |
|
|
|
Cash and cash equivalents |
$ |
64,311,205 |
|
|
$ |
70,063,242 |
|
Incentive and tax receivables |
|
6,533,079 |
|
|
|
14,613,969 |
|
Prepaid expenses and other current assets |
|
3,913,112 |
|
|
|
2,378,812 |
|
Total current assets |
|
74,757,396 |
|
|
|
87,056,023 |
|
Property and
equipment, net |
|
596,512 |
|
|
|
362,724 |
|
Incentive
and tax receivables |
|
1,684,616 |
|
|
|
— |
|
Right-of-use
assets |
|
166,889 |
|
|
|
345,849 |
|
Total assets |
$ |
77,205,413 |
|
|
$ |
87,764,596 |
|
Liabilities and Stockholders' Equity |
|
|
|
Current
liabilities: |
|
|
|
Accounts payable |
$ |
3,404,129 |
|
|
$ |
4,740,981 |
|
Accrued expenses |
|
6,987,653 |
|
|
|
7,073,506 |
|
Lease liabilities |
|
174,073 |
|
|
|
243,677 |
|
Total current liabilities |
|
10,565,855 |
|
|
|
12,058,164 |
|
Lease
liabilities, long-term |
|
— |
|
|
|
109,689 |
|
Total liabilities |
|
10,565,855 |
|
|
|
12,167,853 |
|
|
|
|
|
Stockholders' equity: |
|
|
|
Common stock |
|
29,449 |
|
|
|
23,211 |
|
Additional paid-in capital |
|
259,164,854 |
|
|
|
226,409,156 |
|
Accumulated deficit |
|
(192,554,745 |
) |
|
|
(150,835,624 |
) |
Total stockholders' equity |
|
66,639,558 |
|
|
|
75,596,743 |
|
Total liabilities and stockholders' equity |
$ |
77,205,413 |
|
|
$ |
87,764,596 |
|
|
|
|
|
Zynerba
Contacts
Jim Fickenscher, CFO and VP Corporate DevelopmentZynerba
Pharmaceuticals484.581.7483fickenscherj@zynerba.com
Will Roberts, VP Investor Relations and Corporate
CommunicationsZynerba
Pharmaceuticals484.581.7489robertsw@zynerba.com
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