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 year ended
December 31, 2018 and provided an overview of recent operational
highlights. The Company also announced that its current cash and
cash equivalent position is expected to fund operations and capital
requirements into the first quarter of 2021.
“The fourth quarter of 2018 was a period of strong
clinical and corporate momentum, as we achieved our remaining 2018
milestones and positioned ourselves well for a watershed year in
2019,” said Armando Anido, Chairman and Chief Executive Officer of
Zynerba. “We continued enrollment into CONNECT-FX, our pivotal
trial of Zygel™ in Fragile X Syndrome, and completed enrollment in
BELIEVE 1, our Phase 2 trial in Developmental and Epileptic
Encephalopathies. We also initiated the BRIGHT trial, which will
evaluate Zygel in Autism Spectrum Disorder and are on track to
initiate a Phase 2 trial in 22q11.2 Deletion Syndrome in the second
quarter of 2019. Finally, we have a cash position that we expect to
take us through our expected NDA submission and potential approval
for Zygel in Fragile X Syndrome.”
Fourth Quarter 2018 and Recent Highlights
Zygel in Fragile X Syndrome (FXS)
Fragile X Syndrome Pivotal Data Expected in the Second Half of
2019
Enrollment is progressing in CONNECT-FX, a pivotal,
multi-national, randomized, double blind, placebo-controlled trial
evaluating the efficacy and safety of Zygel (formerly referred to
as ZYN002) in three through 17-year old patients with 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. Clinical
investigative sites are enrolling patients in the United States,
Australia, and New Zealand. Patients who have completed the
double-blind phase are now enrolling into the 12-month open label
extension phase. The Company expects to report top line data in the
second half of 2019. If the data are positive, the Company expects
to submit its New Drug Application (NDA) for Zygel in FXS to the
U.S. Food and Drug Administration in the first half of 2020, with
potential approval by year-end 2020. There are currently no
approved products indicated for FXS.
Presented New Data at the 57th Annual Meeting of
the American College of Neuropsychopharmacology (ACNP) from
the Ongoing Open Label FAB-C Phase 2 Trial of Zygel
Zynerba presented new data demonstrating that treatment with
Zygel improved core emotional and behavioral symptoms of FXS with
statistical significance versus baseline across multiple measures
of efficacy at month three, and that these improvements were
sustained through 12 months of treatment. For example,
significant improvements vs. baseline in social avoidance as
measured by the ABC-CFXS were demonstrated at three months (58%
improvement; p=0.0040) and 12 months (77% improvement; p=0.0013) of
treatment with Zygel. Announced Receipt of New U.S. Patent for
Treatment of Fragile X Syndrome with Cannabidiol (CBD)
The U.S. Patent and Trademark Office issued U.S. Patent No.
10,213,390 titled “Treatment of Fragile X Syndrome with
Cannabidiol” which includes claims directed to methods of treating
Fragile X Syndrome by administering a therapeutically effective
amount of synthetic or purified CBD. This new patent expires in
2038 and is part of an expanding intellectual property portfolio
covering Zygel.
FAB-C Data Accepted for Presentation at the Annual Meeting of
the American Psychiatric Association (APA), May 18–22, 2019 in San
Francisco, CA
The presentation will describe data from the FAB-C (Treatment of
Fragile X Syndrome Anxiety and
Behavioral Challenges with CBD)
trial that highlight the short- and long-term positive impact of
ZYN002 on children and adolescents with FXS. The poster entitled
“Transdermal Cannabidiol (CBD) Gel for the Treatment of Fragile X
Syndrome” (poster #P5-092) will be presented on Monday, May 20,
2019 from 10:00AM to 12:00PM PDT during Poster Session 5.
Zygel in Developmental and Epileptic Encephalopathies
(DEE)
Enrollment Complete in Phase 2 BELIEVE 1 Trial; Topline Results
Expected in the Third Quarter of 2019
The Company has completed enrollment in BELIEVE 1, an open label
multi-dose Phase 2 clinical trial evaluating the efficacy and
safety of Zygel in children and adolescents (three through 17
years) with DEE. The primary efficacy assessment is reduction in
seizure frequency at week 26 compared to baseline. Patients
successfully completing the 26 weeks of the trial may elect to
enter a 6-month extension of the trial. The Company expects to
announce topline data through week 26 in the third quarter of
2019.
Zygel in Autism Spectrum Disorder (ASD) and 22q11.2
Deletion Syndrome (22q)
Expanded Clinical Pipeline into Two New Neuropsychiatric
Indications
The Company completed an extensive review of the
neuropsychiatric disorder landscape for additional priority
indications for clinical development, and announced that it would
evaluate Zygel in ASD and 22q.
- Zynerba recently initiated the Phase 2 BRIGHT (An
Open-Label
Tolerability and Efficacy Trial
of ZYN002 Administered as a Transdermal Gel to
Children and Adolescents with
Autism Spectrum Disorder) clinical trial. The trial will assess the
safety, tolerability and efficacy of Zygel for the treatment of
child and adolescent patients with ASD. The Company expects to
release top line data from this trial in the first half of
2020.
- 22q is the most common gene deletion syndrome affecting as many
as 81,000 patients in the U.S. 22q is associated with increased
anxiety, withdrawn behavior and social interaction problems. The
Company expects to initiate an open label Phase 2 trial of Zygel in
22q in the second quarter of 2019, and to present top line data in
the first half of 2020.
Corporate
Selected Zygel as New Brand Name for ZYN002 CBD Transdermal
Gel
Announced Addition of Pamela Stephenson to Board of
Directors
Pamela Stephenson brings 24 years of commercialization expertise
including health economics, market access, and commercial planning
to Zynerba’s Board of Directors. Ms. Stephenson’s experience
includes nearly 11 years at Vertex, including in her tenure as Vice
President for Global Market Access and Value, and 10 years with
Pfizer in various positions of increasing strategic importance.
Fourth Quarter 2018 Financial Results
As of December 31, 2018, cash and cash equivalents were $59.8
million, compared to $62.5 million as of December 31, 2017.
Research and development expenses for the fourth quarter of 2018
were $4.9 million, including stock-based compensation of $0.8
million. General and administrative expenses for the fourth quarter
of 2018 were $3.3 million, including stock-based compensation
expense of $0.8 million. Net loss for the fourth quarter of 2018
was $7.8 million with basic and diluted net loss per share of
$(0.44).
On June 9, 2017, we entered into an Open Market Sales Agreement,
or “at-the-market” (ATM) offering program, with Jefferies LLC,
pursuant to which we may sell, from time to time, up to $50 million
of our common stock. From January 29, 2019 through March 6, 2019,
the Company has sold and issued 3,439,523 shares under its ATM
program, at a weighted average selling price of $5.44 per share,
for gross proceeds of $18.7 million. Net proceeds after deducting
commissions and offering expenses were $18.1 million, which will be
recorded in the first quarter of 2019 and included in the March 31,
2019 cash and cash equivalents position.
Financial Outlook
The Company’s cash and cash equivalent position as of December
31, 2018 was $59.8 million. Including the $18.1 million in net
proceeds from the shares sold and issued under its ATM program in
the first quarter of 2019, 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 in FXS and into the first quarter 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) |
|
|
|
|
|
Three months ended December
31, |
|
Year ended December
31, |
|
|
|
2018 |
|
2017 |
|
2018 |
|
2017 |
|
Revenue |
|
$ |
86,000 |
|
|
$ |
— |
|
|
$ |
86,000 |
|
|
$ |
— |
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
Research and development |
|
|
4,876,162 |
|
|
|
5,828,091 |
|
|
|
27,245,043 |
|
|
|
22,806,107 |
|
|
General and administrative |
|
|
3,256,044 |
|
|
|
2,376,413 |
|
|
|
13,238,787 |
|
|
|
10,016,902 |
|
|
Total operating expenses |
|
|
8,132,206 |
|
|
|
8,204,504 |
|
|
|
40,483,830 |
|
|
|
32,823,009 |
|
|
Loss from operations |
|
|
(8,046,206 |
) |
|
|
(8,204,504 |
) |
|
|
(40,397,830 |
) |
|
|
(32,823,009 |
) |
|
Other income (expense): |
|
|
|
|
|
|
|
|
|
Interest income |
|
|
321,621 |
|
|
|
156,204 |
|
|
|
961,323 |
|
|
|
519,554 |
|
|
Foreign exchange (loss) gain |
|
|
(65,658 |
) |
|
|
(70,299 |
) |
|
|
(474,668 |
) |
|
|
291,151 |
|
|
Total other income (expense) |
|
|
255,963 |
|
|
|
85,905 |
|
|
|
486,655 |
|
|
|
810,705 |
|
|
Net loss |
|
$ |
(7,790,243 |
) |
|
$ |
(8,118,599 |
) |
|
$ |
(39,911,175 |
) |
|
$ |
(32,012,304 |
) |
|
|
|
|
|
|
|
|
|
|
|
Net loss per share -
basic and diluted |
|
$ |
(0.44 |
) |
|
$ |
(0.60 |
) |
|
$ |
(2.61 |
) |
|
$ |
(2.48 |
) |
|
|
|
|
|
|
|
|
|
|
|
Basic and
diluted weighted average shares outstanding |
|
17,616,373 |
|
|
|
13,423,669 |
|
|
|
15,308,886 |
|
|
|
12,914,814 |
|
|
|
|
|
|
|
|
|
|
|
|
Non-cash stock-based
compensation included above: |
|
|
|
|
|
|
|
|
|
Research and development |
|
$ |
819,715 |
|
|
$ |
562,410 |
|
|
$ |
3,087,498 |
|
|
$ |
2,284,866 |
|
|
General and administrative |
|
|
778,915 |
|
|
|
817,726 |
|
|
|
3,538,245 |
|
|
|
3,361,986 |
|
|
Total |
|
$ |
1,598,630 |
|
|
$ |
1,380,136 |
|
|
$ |
6,625,743 |
|
|
$ |
5,646,852 |
|
|
|
|
|
|
|
|
|
|
|
|
ZYNERBA PHARMACEUTICALS,
INC.CONSOLIDATED BALANCE SHEETS
|
|
|
|
|
|
|
|
December 31, 2018 |
|
December 31, 2017 |
|
Assets |
|
|
|
|
|
Current assets: |
|
|
|
|
|
Cash and cash equivalents |
|
$ |
59,763,773 |
|
|
$ |
62,510,277 |
|
|
Incentive and tax receivables |
|
|
3,444,620 |
|
|
|
3,983,604 |
|
|
Prepaid expenses and other current assets |
|
|
3,747,087 |
|
|
|
1,733,701 |
|
|
Total current assets |
|
|
66,955,480 |
|
|
|
68,227,582 |
|
|
Property and equipment, net |
|
|
371,963 |
|
|
|
164,527 |
|
|
Other assets |
|
|
— |
|
|
|
662,200 |
|
|
Total assets |
|
$ |
67,327,443 |
|
|
$ |
69,054,309 |
|
|
Liabilities and Stockholders'
Equity |
|
|
|
|
|
Current liabilities: |
|
|
|
|
|
Accounts payable |
|
$ |
4,461,567 |
|
|
$ |
3,355,255 |
|
|
Accrued expenses |
|
|
5,264,215 |
|
|
|
3,915,491 |
|
|
Deferred grant revenue |
|
|
— |
|
|
|
171,975 |
|
|
Total current liabilities |
|
|
9,725,782 |
|
|
|
7,442,721 |
|
|
Deferred grant revenue, long-term |
|
|
— |
|
|
|
662,000 |
|
|
Total liabilities |
|
|
9,725,782 |
|
|
|
8,104,721 |
|
|
|
|
|
|
|
|
Stockholders' equity: |
|
|
|
|
|
Common stock |
|
|
17,627 |
|
|
|
13,554 |
|
|
Additional paid-in capital |
|
|
175,476,075 |
|
|
|
138,916,900 |
|
|
Accumulated deficit |
|
|
(117,892,041 |
) |
|
|
(77,980,866 |
) |
|
Total stockholders' equity |
|
|
57,601,661 |
|
|
|
60,949,588 |
|
|
Total liabilities and stockholders' equity |
|
$ |
67,327,443 |
|
|
$ |
69,054,309 |
|
|
|
|
|
|
|
|
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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