Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), a clinical-stage
specialty pharmaceutical company dedicated to developing and
commercializing innovative transdermal synthetic cannabinoid
treatments, today reported financial results for the second quarter
ended June 30, 2017 and provided an overview of recent operational
highlights.
“We expect to announce top-line results from the
STAR 1 trial soon, followed by top-line data from the STOP trial
later this month; and we remain on track to report top-line results
from the FAB-C Fragile X study in September,” said Armando Anido,
Chairman and Chief Executive Officer. “We also met a significant
milestone during the quarter in initiating the Phase 1 program for
ZYN001, a pro-drug of THC delivered via patch, and expect to
initiate our Phase 2 program by the end of this year. With two
clinical stage assets, Zynerba is well-positioned to address a
number of serious unmet medical needs.”
Second Quarter 2017 and Recent
Highlights
Completed Dosing in Phase 2 STAR 1 Clinical Trial
for ZYN002 Cannabidiol (CBD) Gel in Adult Epilepsy Patients; Top
Line Data Expected Soon
A total of 188 patients were randomized in the
Phase 2 STAR 1 double-blind, placebo-controlled clinical trial in
adult patients with refractory epilepsy, exceeding the 180-patient
enrollment target. Following randomization, patients were dosed
with either 195 mg or 390 mg of CBD in ZYN002 4.2% gel or placebo
daily for 12 weeks. The primary endpoint of the trial is the median
reduction in seizure frequency per 28-day period compared to
baseline.
Dosing Continues in Phase 2 STAR 2 Open-Label
Extension Clinical Trial for ZYN002 CBD Gel in Adult Epilepsy
Patients
Ninety-eight percent of the patients who
completed the STAR 1 trial elected to enroll into the STAR 2 trial,
designed to evaluate long-term safety and tolerability of ZYN002
CBD gel across a range of doses. In the open-label extension study,
patients receive a high or low-dose of ZYN002 (390 mg or 195 mg of
CBD in ZYN002 4.2% gel daily, respectively) for up to 52 weeks.
Completed Dosing of Phase 2 STOP Clinical Trial for
ZYN002 CBD Gel in Adult Osteoarthritis Patients; Top Line Data
Expected this Month
Dosing is complete in the randomized, double-blind,
placebo-controlled Phase 2 STOP trial in osteoarthritis of the
knee. We exceeded the initial target enrollment of 300 patients
with 320 patients randomized into one of three dosing groups.
Patients received either 250 mg or 500 mg of CBD in ZYN002 4.2% gel
or placebo daily for 12 weeks. The primary endpoint of the trial is
the change from baseline in the weekly mean of the 24-hour average
worst pain score at week 12.
Completed Enrollment in FAB-C Exploratory Phase 2
Clinical Trial of ZYN002 CBD Gel in Pediatric Fragile X Syndrome
Patients; Top Line Data Expected in September 2017
Enrollment is complete and dosing is ongoing in the
Phase 2 exploratory FAB-C clinical trial designed to evaluate the
safety and efficacy of ZYN002 CBD gel administered over a 12-week
period in pediatric patients with Fragile X syndrome (FXS). We
exceeded the initial target enrollment of 16 patients with 20
patients enrolled. The study is evaluating 50 mg of CBD in ZYN002
4.2% gel once daily, which may be titrated up to 125 mg two times
per day during the six-week titration period. Between weeks six and
twelve, patients receive a maintenance dose of 50 mg, 100 mg or 250
mg daily of CBD in ZYN002 4.2% gel. The primary objective is to
assess intra-patient changes in anxiety, depression and mood (as
measured by the ADAMS scale) versus baseline. The U.S. Food
and Drug Association has granted orphan-drug designation to
ZYN002 CBD gel for the treatment of Fragile X syndrome.
Initiated Phase 1 Program for ZYN001 Pro-drug of
Tetrahydrocannabinol (THC) Delivered via Transdermal Patch
The company has initiated a Phase 1 program to
assess ZYN001, a patent-protected, pro-drug of THC delivered via a
patch. This first in man study is a randomized, double-blind,
placebo-controlled Phase 1 trial. First, the safety, tolerability
and pharmacokinetic profile of a single dose of ZYN001 versus
placebo will be evaluated. Several formulations and patch wear
times ranging from 24 hours to 7 days will be assessed in up to 48
healthy subjects. Based on results from the single dose portion of
this trial, two formulations will be evaluated in multiple patch
applications for 14 days in up to 32 healthy subjects who will be
randomized 3:1 to ZYN001 or placebo. Results from this study will
inform the planned Phase 2 program for ZYN001 in fibromyalgia and
neuropathic pain, expected to initiate in the fourth quarter of
2017.
Second Quarter 2017 Financial
Results
As of June 30, 2017, cash and cash equivalents
were $70.2 million, compared to $77.5 million as of March 31, 2017.
Research and development expenses for the second quarter of 2017
were $5.7 million, including stock-based compensation of $0.6
million. General and administrative expenses for the second quarter
of 2017 were $2.6 million, including stock-based compensation
expense of $0.8 million. Net loss for the second quarter of 2017
was $8.3 million with basic and diluted net loss per share of
$0.64.
Financial Outlook
The Company believes that the current cash and cash equivalent
position of $70.2 million is sufficient to develop five Phase
3-ready programs and, assuming support from the FDA to move
forward, initiate at least one Phase 3 program and fund operations
and capital requirements into 2019.
About Zynerba Pharmaceuticals, Inc. Zynerba
Pharmaceuticals (NASDAQ: ZYNE) is a clinical-stage specialty
pharmaceutical company focused on developing and commercializing
proprietary next-generation synthetic cannabinoid therapeutics
formulated for transdermal delivery. Zynerba is developing
therapeutic candidates based on proprietary transdermal
technologies that, if successfully developed, may allow sustained,
consistent and controlled delivery of therapeutic levels of two
cannabinoids: cannabidiol (CBD), a non-psychoactive cannabinoid,
and tetrahydrocannabinol (THC). Transdermal delivery has the
potential to reduce adverse effects associated with oral dosing.
ZYN002, the Company’s CBD gel, is the first and only synthetic CBD
formulated as a patent-protected permeation-enhanced gel. In March
2017, the Company completed enrollment in the Phase 2 STAR 1
(Synthetic Transdermal Cannabidiol for the Treatment of Epilepsy)
clinical trial of ZYN002 CBD gel in refractory epilepsy patients
with focal seizures, the most common form of epilepsy in adults.
Also in March 2017, the Phase 2 STOP (Synthetic Transdermal
Cannabidiol for the Treatment of Knee Pain due to Osteoarthritis)
clinical trial in patients with knee pain due to osteoarthritis was
fully enrolled. In June 2017, the Company completed enrollment in
its exploratory Phase 2 FAB-C (Treatment of Fragile X Syndrome
Anxiety and Behavioral Challenges with CBD) clinical trial in
children with Fragile X syndrome. Zynerba is also developing
ZYN001, which utilizes a synthetically manufactured pro-drug of
THC. A Phase 1 clinical program for ZYN001 was initiated in the
second quarter of 2017. Learn more at www.zynerba.com and
follow the Company 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 or ZYN001 from the U.S.
Food and Drug Administration (FDA) or foreign regulatory
authorities; even if ZYN002 or ZYN001 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 following: the success, cost and timing of
the Company’s product development activities, studies and clinical
trials; the success of competing products that are or become
available; 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 |
|
Six months ended |
|
|
|
June 30, 2017 |
|
June 30, 2016 |
|
June 30, 2017 |
|
June 30, 2016 |
|
Revenue |
|
$ |
— |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
7,250 |
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
5,732,797 |
|
|
|
4,807,177 |
|
|
|
11,224,252 |
|
|
|
7,376,167 |
|
|
General
and administrative |
|
|
2,632,857 |
|
|
|
1,476,357 |
|
|
|
4,844,650 |
|
|
|
3,156,487 |
|
|
Total
operating expenses |
|
|
8,365,654 |
|
|
|
6,283,534 |
|
|
|
16,068,902 |
|
|
|
10,532,654 |
|
|
Loss from
operations |
|
|
(8,365,654 |
) |
|
|
(6,283,534 |
) |
|
|
(16,068,902 |
) |
|
|
(10,525,404 |
) |
|
Other
income (expense): |
|
|
|
|
|
|
|
|
|
Interest
income |
|
|
124,535 |
|
|
|
18,118 |
|
|
|
201,420 |
|
|
|
30,496 |
|
|
Foreign
exchange (loss) gain |
|
|
(82,360 |
) |
|
|
(20,250 |
) |
|
|
284,982 |
|
|
|
(43,398 |
) |
|
Total
other income (expense) |
|
|
42,175 |
|
|
|
(2,132 |
) |
|
|
486,402 |
|
|
|
(12,902 |
) |
|
Loss
before income taxes |
|
|
(8,323,479 |
) |
|
|
(6,285,666 |
) |
|
|
(15,582,500 |
) |
|
|
(10,538,306 |
) |
|
Income
tax expense |
|
|
— |
|
|
|
(56,277 |
) |
|
|
— |
|
|
|
(27,543 |
) |
|
Net
loss |
|
$ |
(8,323,479 |
) |
|
$ |
(6,229,389 |
) |
|
$ |
(15,582,500 |
) |
|
$ |
(10,510,763 |
) |
|
|
|
|
|
|
|
|
|
|
|
Net loss per share -
basic and diluted |
|
$ |
(0.64 |
) |
|
$ |
(0.70 |
) |
|
$ |
(1.24 |
) |
|
$ |
(1.19 |
) |
|
|
|
|
|
|
|
|
|
|
|
Basic and
diluted weighted average shares outstanding |
|
13,052,294 |
|
|
|
8,860,592 |
|
|
|
12,562,594 |
|
|
|
8,842,271 |
|
|
|
|
|
|
|
|
|
|
|
|
Non-cash stock-based
compensation included above: |
|
|
|
|
|
|
|
|
|
Research
and development |
|
$ |
588,713 |
|
|
$ |
374,919 |
|
|
$ |
1,130,558 |
|
|
$ |
623,651 |
|
|
General
and administrative |
|
|
766,661 |
|
|
|
437,820 |
|
|
|
1,413,515 |
|
|
|
952,887 |
|
|
Total |
|
$ |
1,355,374 |
|
|
$ |
812,739 |
|
|
$ |
2,544,073 |
|
|
$ |
1,576,538 |
|
|
|
|
|
|
|
|
|
|
|
|
ZYNERBA PHARMACEUTICALS,
INC.CONSOLIDATED BALANCE SHEETS
(Unaudited) |
|
|
|
|
|
|
|
|
June 30, 2017 |
|
December 31, 2016 |
|
Assets |
|
|
|
|
|
Current
assets: |
|
|
|
|
|
Cash and
cash equivalents |
|
$ |
70,179,199 |
|
|
$ |
30,965,791 |
|
|
Incentive
and tax receivables |
|
|
3,971,828 |
|
|
|
3,613,943 |
|
|
Prepaid
expenses and other current assets |
|
|
2,307,466 |
|
|
|
1,830,958 |
|
|
Total
current assets |
|
|
76,458,493 |
|
|
|
36,410,692 |
|
|
Property
and equipment, net |
|
|
177,994 |
|
|
|
143,382 |
|
|
Incentive and tax receivables |
|
|
2,456,286 |
|
|
|
— |
|
|
Other
assets |
|
|
200 |
|
|
|
200 |
|
|
Total
assets |
|
$ |
79,092,973 |
|
|
$ |
36,554,274 |
|
|
Liabilities and Stockholders' Equity |
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
Accounts
payable |
|
$ |
2,024,991 |
|
|
$ |
1,848,084 |
|
|
Accrued
expenses |
|
|
5,004,856 |
|
|
|
4,284,907 |
|
|
Deferred
grant revenue |
|
|
833,975 |
|
|
|
833,975 |
|
|
Total
current liabilities |
|
|
7,863,822 |
|
|
|
6,966,966 |
|
|
Stockholders' equity: |
|
|
|
|
|
Common
stock |
|
|
13,257 |
|
|
|
9,995 |
|
|
Additional paid-in capital |
|
|
132,766,956 |
|
|
|
75,545,875 |
|
|
Accumulated deficit |
|
|
(61,551,062 |
) |
|
|
(45,968,562 |
) |
|
Total
stockholders' equity |
|
|
71,229,151 |
|
|
|
29,587,308 |
|
|
Total
liabilities and stockholders' equity |
|
$ |
79,092,973 |
|
|
$ |
36,554,274 |
|
|
|
|
|
|
|
|
Investor Contacts
Jim Fickenscher, CFO and VP Corporate Development
Zynerba Pharmaceuticals
484.581.7483
fickenscherj@zynerba.com
Will Roberts, VP Investor Relations and Corporate Communications
Zynerba Pharmaceuticals
484.581.7489
robertsw@zynerba.com
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