Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), a
clinical-stage specialty neuropsychiatric pharmaceutical company
dedicated to developing and commercializing innovative
pharmaceutically-produced transdermal cannabinoid treatments for
rare and near-rare neurological and psychiatric disorders with high
unmet medical needs, today reported financial results for the
fourth quarter and year ended December 31, 2017 and provided
an overview of recent operational highlights.
“We made significant corporate and clinical
progress throughout 2017, and achieved our goal of utilizing data
from three Phase 2 studies of ZYN002 to determine the direction and
strategic focus of the Company,” said Armando Anido, Chairman and
Chief Executive Officer of Zynerba. “We enter 2018 with strong
momentum including having completed a positive meeting with the FDA
to discuss our clinical path for ZYN002 in Fragile X syndrome. We
have a refined development and commercialization strategy focused
on rare and near-rare neuropsychiatric conditions, and the
expectation of achieving numerous milestones in the coming year
including initiations of the pivotal Fragile X syndrome study and
Phase 2 studies in developmental and epileptic encephalopathies,
adult refractory focal seizures, and Tourette Syndrome.”
Fourth Quarter 2017 and Recent
Highlights
ZYN002 in Fragile X Syndrome
(FXS)
Announced Positive Meeting with U.S. Food and Drug
Administration and Plans to Conduct a Single Pivotal Study of
ZYN002 in Fragile X Syndrome to Support a New Drug Application
(NDA) Filing
The Company expects to initiate a pivotal study mid-year 2018 in
approximately 200 pediatric and adolescent patients in the U.S.,
Australia and New Zealand to support an NDA for ZYN002 in FXS. The
FDA and the Company are in agreement that the primary and key
secondary endpoints for the study should assess observable
behaviors in patients with FXS as reported by the caregiver using
the validated Aberrant Behavior Checklist in Fragile X syndrome
(ABC-FXS). If the pivotal trial meets its endpoints, approval for
an indication encompassing the treatment of behavioral symptoms
associated with Fragile X syndrome may be granted.
ZYN002 in Developmental and Epileptic Encephalopathies
(DEE)
Announced Intent to Study ZYN002 in DEE; Initiation of Phase 2
Trial Expected in the First Half of 2018
DEE is a category of rare and ultra-rare, severe brain disorders
manifesting with seizures or EEG abnormalities that can directly
worsen cognition or behavior. The category affects ~45,000 patients
in the U.S. and includes a number of syndromes, including Doose,
Dravet, Lennox-Gastaut, and West, among others. The Phase 2 open
label DEE study will enroll approximately 48 pediatric and
adolescent patients and will help identify new indications to take
into blinded placebo-controlled studies. The primary endpoints are
expected to be reduction in seizures at 12 and 24 weeks. Results
from the study are expected in 2019.
ZYN002 in Focal Epilepsy
Clinical Data from the STAR 1 and STAR 2 Studies of ZYN002 in
Patients with Focal Seizures Presented at the 2017 Annual Meeting
of the American Epilepsy Society (AES) in Washington, D.C.
Data suggest clinically meaningful responses to ZYN002, as
measured by reductions in focal seizures from the baseline period
of STAR 1, are correlated with longer-term use of ZYN002.
- Patients taking ZYN002 for six months experienced a
>30% median reduction in seizures from baseline;
- Patients taking ZYN002 for nine months experienced a >65%
(195 mg in STAR 1 and 390 mg in STAR 2) and >48% (390 mg in STAR
1 and STAR 2) median reduction in seizures from baseline;
- In STAR 1, patients with more severe epilepsy (defined as a
baseline seizure frequency of ≥15 per month) taking ZYN002 had a
greater percent reduction in seizures compared to patients with
severe epilepsy receiving placebo;
- ZYN002 was very well tolerated with an incidence of adverse
events comparable to placebo and no clinically significant
differences between the active treatment groups; and
- There were no clinically significant changes in ECGs or
laboratory results in patients receiving ZYN002.
Initiation of Double-Blind, Placebo Controlled Phase 2b Clinical
Trial of ZYN002 in Approximately 300 Adult Patients with Refractory
Focal Epilepsy Expected in the Second Half of 2018
Learnings from the STAR 1 and STAR 2 trials provide insight into
a revised Phase 2b clinical trial design. Anticipated changes to
the trial include increases in (1) baseline seizure frequency, (2)
patient count and (3) trial duration. In addition, the Company will
stratify randomization by baseline seizure rate and gender. Zynerba
also anticipates testing a higher daily dose of ZYN002 than was
used in the STAR trials. The study will be conducted in sites in
the U.S., Australia and New Zealand.
ZYN001 in Tourette Syndrome
Dosing Continues in the Phase 1 Program for ZYN001 Pro-drug of
Tetrahydrocannabinol (THC) Delivered via Transdermal Patch;
Initiation of Phase 2 Study in Patients with Tourette Syndrome (TS)
Expected in the Second Half of 2018
The Company is executing on 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 trial designed to assess the safety,
tolerability and pharmacokinetic profile of multiple formulations
of ZYN001. The Company expects to complete its Phase 1 evaluation
in the first half of 2018, and then move into a Phase 2 clinical
trial in Tourette Syndrome late in the second half of
2018.
Corporate
Corporate Strategy Focused on Rare and Near-Rare
Neuropsychiatric Disorders with High Unmet Medical
Needs
Zynerba believes that its strategic focus provides opportunities
for an efficient development and commercialization strategies that
may include (1) orphan drug designation for some indications; (2)
access to other available regulatory designations, which, if
granted, can accelerate commercial approval; (3) a targeted
physician audience enabling modest commercial investments; and (4)
consistent pricing across all indications. The Company also
discontinued its development programs in the capital-intensive pain
spaces.
Enhanced Senior Management Team
In January 2018, Liza Squires, M.D. was named Chief Medical
Officer. She has over 25 years of experience in rare and
neuropsychiatric disorders with companies including Aevi Genomics
Medicine, Lumos Pharma, and Shire Pharmaceuticals. She also served
as the Director of Pediatric Neurology for DeVos Children’s
Hospital.
In March 2018, Joe Apostolico joined Zynerba as Vice President,
Human Resources. Mr. Apostolico brings the Company over 30 years of
broad global healthcare and pharmaceutical human resource
leadership experience from his tenures with companies including
Adaptimmune Therapeutics and GSK.
Fourth Quarter 2017 Financial Results
As of December 31, 2017, cash and cash equivalents were $62.5
million, compared to $31.0 million as of December 31, 2016.
Research and development expenses for the fourth quarter of 2017
were $5.8 million, including stock-based compensation of $0.6
million. General and administrative expenses for the fourth quarter
of 2017 were $2.4 million, including stock-based compensation
expense of $0.8 million. Net loss for the fourth quarter of 2017
was $8.1 million with basic and diluted net loss per share of
$(0.60).
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 September 28, 2017 through October 26,
2017, the Company has sold and issued 296,594 shares under its ATM
program, at a weighted average selling price of $10.74 per share,
for gross proceeds of $3.2 million. Net proceeds after deducting
underwriting and commissions and offering expenses were $3.0
million, which were recorded in the fourth quarter and are included
in the December 31, 2017 cash and cash equivalents position.
Financial Outlook
The Company believes that the cash and cash equivalent position
of $62.5 million as of December 31, 2017 is sufficient to fund
operations and capital requirements well into 2019.
About Zynerba Pharmaceuticals, Inc. Zynerba
Pharmaceuticals (NASDAQ:ZYNE) is a clinical-stage specialty
neuropsychiatric pharmaceutical company dedicated to developing and
commercializing innovative pharmaceutically-produced transdermal
cannabinoid treatments for rare or near-rare neuropsychiatric
disorders with high unmet medical needs. We are dedicated to
improving the lives of people with severe health conditions by
developing cannabinoid medicines designed to meet the rigorous
efficacy and safety standards established by global regulatory
agencies. Through the discovery and development of these
potentially life-changing medicines, Zynerba seeks to improve the
lives of patients battling severe, chronic health conditions
including Fragile X syndrome, refractory epilepsies, Tourette
Syndrome, and other neuropsychiatric disorders. 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 |
|
Year ended |
|
|
December 31, 2017 |
|
|
December 31, 2016 |
|
December 31, 2017 |
|
|
December 31, 2016 |
|
Revenue |
|
$ |
— |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
7,250 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
5,828,091 |
|
|
|
4,904,363 |
|
|
|
22,806,107 |
|
|
|
16,784,626 |
|
General
and administrative |
|
|
2,376,413 |
|
|
|
1,780,304 |
|
|
|
10,016,902 |
|
|
|
6,430,252 |
|
Total
operating expenses |
|
|
8,204,504 |
|
|
|
6,684,667 |
|
|
|
32,823,009 |
|
|
|
23,214,878 |
|
Loss from
operations |
|
|
(8,204,504 |
) |
|
|
(6,684,667 |
) |
|
|
(32,823,009 |
) |
|
|
(23,207,628 |
) |
Other
income (expense): |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest
income (expense), net |
|
|
156,204 |
|
|
|
26,980 |
|
|
|
519,554 |
|
|
|
80,222 |
|
Foreign
exchange gain (loss) |
|
|
(70,299 |
) |
|
|
(139,829 |
) |
|
|
291,151 |
|
|
|
(189,497 |
) |
Loss on
dosposal of equipment |
|
|
— |
|
|
|
(99,147 |
) |
|
|
— |
|
|
|
(99,147 |
) |
Total
other income (expense) |
|
|
85,905 |
|
|
|
(211,996 |
) |
|
|
810,705 |
|
|
|
(208,422 |
) |
Loss
before income taxes |
|
|
(8,118,599 |
) |
|
|
(6,896,663 |
) |
|
|
(32,012,304 |
) |
|
|
(23,416,050 |
) |
Income
tax benefit |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(27,543 |
) |
Net
loss |
|
$ |
(8,118,599 |
) |
|
$ |
(6,896,663 |
) |
|
$ |
(32,012,304 |
) |
|
$ |
(23,388,507 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share -
basic and diluted |
|
$ |
(0.60 |
) |
|
$ |
(0.71 |
) |
|
$ |
(2.48 |
) |
|
$ |
(2.58 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Basic and diluted
weighted average shares outstanding |
|
|
13,423,669 |
|
|
|
9,678,924 |
|
|
|
12,914,814 |
|
|
|
9,070,232 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Non-cash stock-based
compensation included above: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research
and development |
|
$ |
562,410 |
|
|
$ |
365,072 |
|
|
$ |
2,284,866 |
|
|
$ |
1,281,108 |
|
General
and administrative |
|
|
817,726 |
|
|
|
522,352 |
|
|
|
3,361,986 |
|
|
|
1,988,258 |
|
Total |
|
$ |
1,380,136 |
|
|
$ |
887,424 |
|
|
$ |
5,646,852 |
|
|
$ |
3,269,366 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
ZYNERBA PHARMACEUTICALS, INC. |
CONSOLIDATED BALANCE SHEETS |
|
|
|
December 31, 2017 |
|
December 31, 2016 |
Assets |
|
|
|
|
|
|
|
|
Current
assets: |
|
|
|
|
|
|
|
|
Cash and
cash equivalents |
|
$ |
62,510,277 |
|
|
$ |
30,965,791 |
|
Incentive
and tax receivables |
|
|
3,983,604 |
|
|
|
3,613,943 |
|
Prepaid
expenses and other current assets |
|
|
1,733,701 |
|
|
|
1,830,958 |
|
Total
current assets |
|
|
68,227,582 |
|
|
|
36,410,692 |
|
Property
and equipment, net |
|
|
164,527 |
|
|
|
143,382 |
|
Other
assets |
|
|
662,200 |
|
|
|
200 |
|
Total
assets |
|
$ |
69,054,309 |
|
|
$ |
36,554,274 |
|
Liabilities and Stockholders' Equity |
|
|
|
|
|
|
|
|
Current
liabilities: |
|
|
|
|
|
|
|
|
Accounts
payable |
|
$ |
3,355,255 |
|
|
$ |
1,848,084 |
|
Accrued
expenses |
|
|
3,915,491 |
|
|
|
4,284,907 |
|
Deferred
grant revenue |
|
|
171,975 |
|
|
|
833,975 |
|
Total
current liabilities |
|
|
7,442,721 |
|
|
|
6,966,966 |
|
Deferred
grant revenue, long-term |
|
|
662,000 |
|
|
|
— |
|
Total
liabilities |
|
|
8,104,721 |
|
|
|
6,966,966 |
|
|
|
|
|
|
|
|
|
|
Stockholders' equity: |
|
|
|
|
|
|
|
|
Common
stock |
|
|
13,554 |
|
|
|
9,995 |
|
Additional paid-in capital |
|
|
138,916,900 |
|
|
|
75,545,875 |
|
Accumulated deficit |
|
|
(77,980,866 |
) |
|
|
(45,968,562 |
) |
Total
stockholders' equity |
|
|
60,949,588 |
|
|
|
29,587,308 |
|
Total
liabilities and stockholders' equity |
|
$ |
69,054,309 |
|
|
$ |
36,554,274 |
|
|
|
|
|
|
|
|
|
|
Investor 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
Zynerba Pharmaceuticals (NASDAQ:ZYNE)
Historical Stock Chart
From Aug 2024 to Sep 2024
Zynerba Pharmaceuticals (NASDAQ:ZYNE)
Historical Stock Chart
From Sep 2023 to Sep 2024