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 fourth quarter and full year
ended December 31, 2019 and provided an overview of recent
operational highlights.
“The fourth quarter of 2019 capped off a year of strong
execution by Zynerba,” said Armando Anido, Chairman and Chief
Executive Officer of Zynerba. “With a number of shots-on-goal in
our clinical pipeline, each with near term milestones, our outlook
is promising for the remainder of 2020 and beyond. We are
positioned for major news events throughout this year and next,
including the topline results from our pivotal CONNECT-FX trial of
Zygel™ in patients with Fragile X syndrome which are expected late
next quarter.”
Fourth Quarter 2019 and Recent Highlights
Zygel in Fragile X Syndrome (FXS)
Enrollment Complete in Pivotal FXS Trial; Topline Results
Expected in the Second Quarter of 2020
Enrollment is complete with 212 patients randomized into
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. 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 and the ABC-CFXS Socially
Unresponsive/Lethargic subscale score, and Clinical Global
Impression - Improvement (CGI-I) at the end of the treatment
period. The Company expects to report topline results late in the
second quarter of 2020. If the results are positive, Zynerba
intends to request a meeting with the FDA to determine the
acceptability of the data as a basis for a New Drug Application
(NDA) and to seek advice on preparation of the marketing
authorization. The Company expects to submit its 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. (Press
release)
Robust Enrollment Continues into Open Label Extension Study
During the screening phase of CONNECT-FX, caregivers of patients
in the trial were informed that their participating child may have
the opportunity to receive Zygel in an open label extension trial
following the child’s compliant completion of CONNECT-FX,
regardless of their child’s perceived response or actual blinded
drug assignment at randomization in CONNECT-FX. As of March 9,
2020, 97% of the 163 patients who have completed the 14-week
blinded portion of the CONNECT-FX trial have enrolled in the open
label extension trial.
New U.S. Patent Received for Treatment of FXS with Transdermal
Cannabidiol (CBD)
The U.S. Patent and Trademark Office issued U.S. 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 CBD daily via a gel or cream. This new patent expires
in 2038 and is part of an expanding intellectual property portfolio
covering Zygel. (Press release)
Poster Describing Health State Utility Indices (HUI) that
Estimate the Severity of FXS and Other Pediatric Disorders
Presented at the American Society for Experimental
Neurotherapeutics (ASENT) 2020 Meeting
An HUI specific to FXS, known as the ABC-UI, was derived from
the ABC-CFXS to measure the health-related quality of life (HRQoL)
benefit of treatments for FXS (Kerr C et al. Qual Life
Res.2015;24(2):305-314); HUI are measured on a scale of 0 to 1 and
used in clinical and economic analyses of therapies with potential
impact on HRQoL. This poster described the evaluation of the
potential benefit of Zygel on the ABC-UI in FXS through post hoc
analysis of data from the FAB-C trial. The mean ABC-UI for FXS
patients was calculated to be 0.57 at baseline, estimating a
significant disease-related impact on HRQoL in FXS despite the
children and adolescents in the study being maintained on standard
of care for FXS, and suggesting an impact similar or worse than
other debilitating pediatric conditions as described in the
published literature in measures of HUI. Additionally, compared to
baseline, patients receiving Zygel experienced significant (P <
0.01) and sustained improvement in their mean ABC-UI from week 4 to
12. (Press release)
Poster Describing Caregivers’ Perspectives on FXS Diagnosis and
Patient Journey Perspective Presented at the American Society for
Experimental Neurotherapeutics (ASENT) 2020 Meeting
The poster described the results of a caregiver survey that
found an average age of 3 years at initial diagnosis, a high
prevalence of comorbid conditions including ASD and
attention-deficit/hyperactivity disorder, and standard of care
consisting primarily of counseling/therapy and prescription
medications that are not indicated for FXS. While caregivers of
children with FXS often notice a variety of initial symptoms early
and seek help from a health care professional, it is not until
subsequent physician visits, often involving a specialist, that a
formal diagnosis is made. (Press release)
Zygel in Autism Spectrum Disorder (ASD)
Completed Enrollment in Phase 2 Open Label Trial of Zygel in
ASD; Topline Results Expected in the Second Quarter of 2020
Enrollment is complete in the Phase 2 BRIGHT trial assessing the
safety, tolerability and efficacy of Zygel for the treatment of
pediatric and adolescent patients with ASD. The 14-week trial is
evaluating the efficacy and safety of Zygel in 37 children and
adolescents (ages four through 17) with moderate-to-severe 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. The mean age of the 37 patients enrolled in the BRIGHT
trial is 9.2 years. Ninety-two (92) percent of the enrolled
patients are male, accurately reflecting the overall prevalence and
gender ratio of moderate-to-severe ASD in the United States and in
other studies. Zynerba expects to report topline results from this
study in the second quarter of 2020. (Press release)
New U.S. Patent Received for Treatment of ASD with Transdermal
Cannabidiol
The U.S. Patent and Trademark Office has issued U.S. Patent No.
10,568,848, titled “Treatment of Autism with Cannabidiol” which
includes claims directed to methods of treating ASD by
transdermally administering, via a gel or cream, a therapeutically
effective amount of purified CBD. The patent expires in 2038.
(Press release)
Poster Describing the Baseline Characteristics of Patients in
Phase 2 BRIGHT Trial in ASD Presented at the American Society for
Experimental Neurotherapeutics (ASENT) 2020 Meeting
The poster further describes the baseline characteristics of the
pediatric and adolescent patients in the fully-enrolled Phase 2
BRIGHT trial, indicating that the trial enrolled a broad patient
population and was enriched for disease severity to avoid floor
effects on outcome measures. At baseline, at least 92% of patients
have moderate to severe symptoms of ASD as measured by the Autism
Diagnostic Observation Schedule (ADOS®-2) comparison score and the
Diagnostic and Statistical Manual of Mental Disorders 5th edition
(DSM-5) severity level score; this severity is further confirmed by
an ABC-C Irritability subscale score of 30. In addition, 24% of
patients enrolled in the BRIGHT trial had a PRAS-ASD score of
>52, indicating possible clinical anxiety. (Press release)
Zygel in 22q11.2 Deletion Syndrome (22q)
Phase 2 Open Label Trial of Zygel in 22q Ongoing; Data Now
Expected in the Third Quarter 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 now expects to report
topline results from this study in the third quarter of 2020.
Zygel in Developmental and Epileptic Encephalopathies
(DEE)
Meeting with U.S. Food and Drug Administration (FDA) to Discuss
Pathway for Zygel in DEE Expected in the First Half of 2020
Zynerba expects to meet with the FDA to discuss the clinical
path forward in DEE. Based on the Phase 2 trial design and positive
efficacy and safety results, Zynerba anticipates that it will
discuss the pursuit of an indication that includes all syndromes
and encephalopathies in the DEE category that present with focal
impaired-awareness seizures (FIAS; previously known as complex
partial seizures) and/or convulsive seizures (CS), the most common
and debilitating seizure types representing 75% to 80% of all
seizures.
Corporate
Enhanced Senior Management Team
Paul M. Kirsch joined Zynerba as Vice President of Regulatory
Affairs and Quality Assurance, bringing 30 years of regulatory
affairs management experience with companies including Trevena,
Inc., Iroko Pharmaceuticals, LLC, Teva Pharmaceuticals, and
Cephalon, Inc. He has extensive regulatory experience with orphan
and neuroscience products, and has led five successful NDAs into
commercialization in multiple indications.
Fourth quarter and full year 2019 Financial
Results
As of December 31, 2019, cash and cash equivalents were $70.1
million, compared to $59.8 million as of December 31, 2018.
Research and development expenses for the fourth quarter of 2019
were $7.5 million, including stock-based compensation of $0.5
million. General and administrative expenses for the fourth quarter
of 2019 were $4.0 million, including stock-based compensation
expense of $0.8 million. The net loss for the fourth quarter of
2019 was $10.7 million with basic and diluted net loss per share of
$(0.46).
Research and development expenses for the year ended December
31, 2019 were $20.4 million, including stock-based compensation of
$2.4 million. General and administrative expenses for the year
ended December 31, 2019 were $13.9 million, including stock-based
compensation expense of $3.2 million. The net loss for the full
year of 2019 was $32.9 million with basic and diluted net loss per
share of $(1.50).
Financial Outlook
The Company’s cash and cash equivalents as of December 31, 2019
was $70.1 million. Management believes that the cash runway 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;
the Company’s expectations regarding its ability to obtain and
adequately maintain sufficient intellectual property protection for
its product candidates; and the timing and outcome of current and
future legal proceedings. 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 December 31, |
|
Year ended December 31, |
|
|
|
2019 |
|
|
|
2018 |
|
|
|
2019 |
|
|
|
2018 |
|
Revenue |
|
$ |
— |
|
|
$ |
86,000 |
|
|
$ |
— |
|
|
$ |
86,000 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
|
7,457,953 |
|
|
|
4,876,162 |
|
|
|
20,384,049 |
|
|
|
27,245,043 |
|
General and administrative |
|
|
3,958,211 |
|
|
|
3,256,044 |
|
|
|
13,935,761 |
|
|
|
13,238,787 |
|
Total operating expenses |
|
|
11,416,164 |
|
|
|
8,132,206 |
|
|
|
34,319,810 |
|
|
|
40,483,830 |
|
Loss from operations |
|
|
(11,416,164 |
) |
|
|
(8,046,206 |
) |
|
|
(34,319,810 |
) |
|
|
(40,397,830 |
) |
Other income
(expense): |
|
|
|
|
|
|
|
|
Interest income |
|
|
295,140 |
|
|
|
321,621 |
|
|
|
1,522,138 |
|
|
|
961,323 |
|
Foreign exchange gain (loss) |
|
|
406,033 |
|
|
|
(65,658 |
) |
|
|
(145,911 |
) |
|
|
(474,668 |
) |
Total other income |
|
|
701,173 |
|
|
|
255,963 |
|
|
|
1,376,227 |
|
|
|
486,655 |
|
Net loss |
|
$ |
(10,714,991 |
) |
|
$ |
(7,790,243 |
) |
|
$ |
(32,943,583 |
) |
|
$ |
(39,911,175 |
) |
|
|
|
|
|
|
|
|
|
Net loss per
share - basic and diluted |
|
$ |
(0.46 |
) |
|
$ |
(0.44 |
) |
|
$ |
(1.50 |
) |
|
$ |
(2.61 |
) |
|
|
|
|
|
|
|
|
|
Basic and
diluted weighted average shares outstanding |
|
|
23,191,428 |
|
|
|
17,616,373 |
|
|
|
22,000,203 |
|
|
|
15,308,886 |
|
|
|
|
|
|
|
|
|
|
Non-cash
stock-based compensation included above: |
|
|
|
|
|
|
|
|
Research and development |
|
$ |
456,420 |
|
|
$ |
819,715 |
|
|
$ |
2,371,998 |
|
|
$ |
3,087,498 |
|
General and administrative |
|
|
751,253 |
|
|
|
778,915 |
|
|
|
3,189,897 |
|
|
|
3,538,245 |
|
Total |
|
$ |
1,207,673 |
|
|
$ |
1,598,630 |
|
|
$ |
5,561,895 |
|
|
$ |
6,625,743 |
|
|
|
|
|
|
|
|
|
|
ZYNERBA PHARMACEUTICALS,
INC.CONSOLIDATED BALANCE SHEETS
|
|
December 31, 2019 |
|
December 31, 2018 |
Assets |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
70,063,242 |
|
|
$ |
59,763,773 |
|
Incentive and tax receivables |
|
|
14,613,969 |
|
|
|
3,444,620 |
|
Prepaid expenses and other current assets |
|
|
2,378,812 |
|
|
|
3,747,087 |
|
Total current assets |
|
|
87,056,023 |
|
|
|
66,955,480 |
|
Property and
equipment, net |
|
|
362,724 |
|
|
|
371,963 |
|
Right-of-use
assets |
|
|
345,849 |
|
|
|
— |
|
Total assets |
|
$ |
87,764,596 |
|
|
$ |
67,327,443 |
|
Liabilities and Stockholders' Equity |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts payable |
|
$ |
4,740,981 |
|
|
$ |
4,461,567 |
|
Accrued expenses |
|
|
7,073,506 |
|
|
|
5,264,215 |
|
Lease liabilities |
|
|
243,677 |
|
|
|
— |
|
Total current liabilities |
|
|
12,058,164 |
|
|
|
9,725,782 |
|
Lease
liabilities, long-term |
|
|
109,689 |
|
|
|
— |
|
Total liabilities |
|
|
12,167,853 |
|
|
|
9,725,782 |
|
|
|
|
|
|
Stockholders' equity: |
|
|
|
|
Common stock |
|
|
23,211 |
|
|
|
17,627 |
|
Additional paid-in capital |
|
|
226,409,156 |
|
|
|
175,476,075 |
|
Accumulated deficit |
|
|
(150,835,624 |
) |
|
|
(117,892,041 |
) |
Total stockholders' equity |
|
|
75,596,743 |
|
|
|
57,601,661 |
|
Total liabilities and stockholders' equity |
|
$ |
87,764,596 |
|
|
$ |
67,327,443 |
|
|
|
|
|
|
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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