Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), a clinical-stage
specialty pharmaceutical company dedicated to developing and
commercializing innovative pharmaceutically-produced transdermal
cannabinoid treatments, today reported financial results for the
third quarter ended September 30, 2017 and provided an overview of
recent operational highlights.
“We announced important data during the third
quarter, including remarkable Phase 2 data from our FAB-C study of
ZYN002 in children and adolescents with Fragile X syndrome,” said
Armando Anido, Chairman and Chief Executive Officer. “We achieved
the primary endpoint and numerous secondary endpoints with
statistical significance compared to baseline, and anticipate
moving into a pivotal program in Fragile X in the first half of
2018. Additionally, we look forward to outlining our path forward
in Epilepsy, and meeting with the FDA to discuss our OA pivotal
program in the first quarter of 2018.”
Third Quarter 2017 and Recent
Highlights
ZYN002 in Fragile X Syndrome
(FXS)
Announced Positive Results from FAB-C Open Label
Exploratory Phase 2 Clinical Trial of ZYN002 Cannabidiol (CBD) Gel
in Pediatric and Adolescent Fragile X Syndrome Patients; Company
Expects to Initiate Pivotal Program in the First Half of 2018
A total of 20 patients were enrolled into the
12-week open label Phase 2 FAB-C trial to evaluate ZYN002
cannabidiol (CBD) gel in pediatric and adolescent patients with
FXS.
- The study successfully met its primary endpoint, achieving a
46% improvement (p<0.0001) in the total score of Anxiety,
Depression, and Mood Scale (ADAMS) at week twelve compared to
baseline;
- The results of the secondary endpoints reinforce those
demonstrated in the ADAMS. For example, ZYN002 also achieved
clinically meaningful and statistically significant improvements
compared to baseline in all measures of the Aberrant Behavior
Checklist for Fragile X (ABC-FXS), which address the key symptoms
of FXS including social avoidance, temper tantrums, repetitive
movements, and hyperactivity;
- ZYN002 was shown to be extremely well tolerated, and the safety
profile was consistent with previously released data from clinical
trials;
- Thirteen (13) of the 18 patients completing the study have
enrolled into the 52-week open label extension and remain in the
trial;
- The Company anticipates that it will meet with the U.S. Food
and Drug Administration (FDA) in the first quarter of 2018 to
discuss the results of the study and next steps, including a
pivotal program expected to initiate in the first half of 2018;
and
- The FDA has granted orphan-drug designation to Zynerba for the
use of CBD for the treatment of FXS.
ZYN002 in Osteoarthritis
Announced Important Results from Phase 2 STOP Clinical Trial for
ZYN002 CBD Gel in Adult Osteoarthritis Patients; Study Achieved Key
Secondary Endpoints with Statistical Significance
Three hundred and twenty (320) patients were randomized into the
double-blind, placebo-controlled Phase 2 STOP trial in
osteoarthritis of the knee.
- Statistically significant results were achieved for a number of
secondary endpoints, though the study did not achieve its primary
endpoint of change from baseline in the weekly mean of the 24-hour
average worst pain score at week 12;
- Importantly, the composite responder analysis (defined as a ≥30
percent reduction in worst average daily pain scores and a ≥20
percent improvement in the WOMAC physical function score) for 250
mg daily of ZYN002 4.2% CBD gel achieved statistical significance
(p=0.016). This composite responder endpoint is consistent with the
FDA guidelines requiring the use of both pain and function
endpoints in pivotal osteoarthritis programs;
- ZYN002 was shown to be extremely well tolerated and the safety
profile was consistent with previously released data from clinical
trials; and
- Zynerba anticipates that it will meet with the FDA in the first
quarter of 2018 to discuss the results of the study. The Company
expects to initiate a pivotal Phase 2/3 program in 2018.
ZYN002 in Focal Seizures
Announced Results from Phase 2 STAR 1 Clinical Trial for ZYN002
CBD Gel in Adult Epilepsy Patients with Focal Seizures
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.
- ZYN002 did not demonstrate a statistically significant
reduction of focal seizures during the treatment period compared to
the baseline period for either the high or low dose cohorts
compared to placebo;
- The Company believes that the study failed to achieve its
endpoints primarily due to a bimodal distribution of patient
responses on placebo; approximately 24% of patients receiving
placebo achieved >50% reductions in focal seizures, while the
other approximately 76% of placebo patients showed no improvement
or worsening of focal seizures; and
- ZYN002 was shown to be extremely well tolerated and the safety
profile was consistent with previously released data from the Phase
1 trials.
Dosing Continues in the Phase 2 STAR 2 Open-Label Extension
Trial for ZYN002 CBD Gel in Adult Epilepsy Patients; Clinically
Meaningful Responses Achieved in Patients on ZYN002 for at least 6
Months
As of November 13, 2017, one hundred (100) patients remain in
the STAR 2 open label 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 are receiving ZYN002 for up to
52 weeks.
- Eighty-nine (89) patients have reached six months of drug
exposure in STAR 2; and
- Clinically meaningful reductions in seizures (>50%) have
been observed in patients on ZYN002 for at least six months.
Data from the ZYN002 epilepsy program have been accepted for
poster presentation at the 2017 American Epilepsy Society (AES)
Meeting.
Further analysis of data from STAR 1 and STAR 2 will clarify the
study design and inclusion / exclusion criteria ahead of a new
Phase 2 clinical study in epilepsy. The Company expects to outline
its path forward in epilepsy in the first quarter of 2018.
ZYN001 Phase 1 Evaluation
Dosing Continues in the Phase 1 Program for ZYN001 Pro-drug of
Tetrahydrocannabinol (THC) Delivered via Transdermal Patch
Dosing is underway in 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. The safety, tolerability and pharmacokinetic profile
of a single dose of ZYN001 versus placebo is initially being
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 are now expected in the
first half of 2018. These data will inform the planned Phase 2
program for ZYN001, now expected to initiate in 2018.
Third Quarter 2017 Financial Results
As of September 30, 2017, cash and cash
equivalents were $66.3 million, compared to $70.2 million as of
June 30, 2017. Research and development expenses for the third
quarter of 2017 were $5.8 million, including stock-based
compensation of $0.6 million. General and administrative expenses
for the third quarter of 2017 were $2.8 million, including
stock-based compensation expense of $1.1 million. Net loss for the
third quarter of 2017 was $8.3 million with basic and diluted net
loss per share of $0.63.
Recent Equity Financing
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. None of the proceeds were settled prior to September 30,
2017, and therefore the cash for the sale of these common shares
will be recorded in the fourth quarter.
Financial Outlook
The Company believes that the cash and cash equivalent position
of $66.3 million as of September 30, 2017 is sufficient to
develop five Phase 3-ready programs and initiate at least one
pivotal program and fund operations and capital requirements into
2019.
About Zynerba Pharmaceuticals, Inc. Zynerba
Pharmaceuticals (NASDAQ:ZYNE) is dedicated to improving the lives
of people with severe health conditions where there is a high unmet
medical need by developing and commercializing
pharmaceutically-produced transdermal cannabinoid medicines
designed to meet the rigorous efficacy and safety standards
established by global regulatory agencies. Through the discovery
and development of these life-changing medicines, Zynerba seeks to
improve the lives of patients battling severe, chronic health
conditions including Fragile X syndrome, epilepsy, osteoarthritis,
fibromyalgia and peripheral neuropathic pain. 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 |
|
Nine months ended |
|
|
|
September 30, 2017 |
|
September 30, 2016 |
|
September 30, 2017 |
|
September 30, 2016 |
|
Revenue |
|
$ |
— |
|
|
$ |
— |
|
|
$ |
— |
|
|
$ |
7,250 |
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
Research
and development |
|
|
5,753,764 |
|
|
|
4,504,097 |
|
|
|
16,978,016 |
|
|
|
11,880,264 |
|
|
General
and administrative |
|
|
2,795,839 |
|
|
|
1,493,461 |
|
|
|
7,640,489 |
|
|
|
4,649,948 |
|
|
Total
operating expenses |
|
|
8,549,603 |
|
|
|
5,997,558 |
|
|
|
24,618,505 |
|
|
|
16,530,212 |
|
|
Loss from
operations |
|
|
(8,549,603 |
) |
|
|
(5,997,558 |
) |
|
|
(24,618,505 |
) |
|
|
(16,522,962 |
) |
|
Other
income (expense): |
|
|
|
|
|
|
|
|
|
Interest
income |
|
|
161,930 |
|
|
|
22,747 |
|
|
|
363,350 |
|
|
|
53,243 |
|
|
Foreign
exchange gain (loss) |
|
|
76,468 |
|
|
|
(6,270 |
) |
|
|
361,450 |
|
|
|
(49,668 |
) |
|
Total
other income (expense) |
|
|
238,398 |
|
|
|
16,477 |
|
|
|
724,800 |
|
|
|
3,575 |
|
|
Loss
before income taxes |
|
|
(8,311,205 |
) |
|
|
(5,981,081 |
) |
|
|
(23,893,705 |
) |
|
|
(16,519,387 |
) |
|
Income
tax benefit |
|
|
— |
|
|
|
— |
|
|
|
— |
|
|
|
(27,543 |
) |
|
Net
loss |
|
$ |
(8,311,205 |
) |
|
$ |
(5,981,081 |
) |
|
$ |
(23,893,705 |
) |
|
$ |
(16,491,844 |
) |
|
|
|
|
|
|
|
|
|
|
|
Net loss per share -
basic and diluted |
|
$ |
(0.63 |
) |
|
$ |
(0.67 |
) |
|
$ |
(1.87 |
) |
|
$ |
(1.86 |
) |
|
|
|
|
|
|
|
|
|
|
|
Basic and
diluted weighted average shares outstanding |
|
13,098,914 |
|
|
|
8,912,508 |
|
|
|
12,743,332 |
|
|
|
8,865,854 |
|
|
|
|
|
|
|
|
|
|
|
|
Non-cash stock-based
compensation included above: |
|
|
|
|
|
|
|
|
|
Research
and development |
|
$ |
591,898 |
|
|
$ |
292,385 |
|
|
$ |
1,722,456 |
|
|
$ |
916,036 |
|
|
General
and administrative |
|
|
1,130,745 |
|
|
|
513,019 |
|
|
|
2,544,260 |
|
|
|
1,465,906 |
|
|
Total |
|
$ |
1,722,643 |
|
|
$ |
805,404 |
|
|
$ |
4,266,716 |
|
|
$ |
2,381,942 |
|
|
|
|
|
|
|
|
|
|
|
|
|
ZYNERBA PHARMACEUTICALS,
INC. |
CONSOLIDATED BALANCE SHEETS |
(Unaudited) |
|
|
|
|
|
|
|
September 30, 2017 |
|
December 31, 2016 |
|
Assets |
|
|
|
|
Current
assets: |
|
|
|
|
Cash and
cash equivalents |
$ |
66,251,286 |
|
|
$ |
30,965,791 |
|
|
Incentive
and tax receivables |
|
3,617,956 |
|
|
|
3,613,943 |
|
|
Prepaid
expenses and other current assets |
|
3,010,144 |
|
|
|
1,830,958 |
|
|
Total
current assets |
|
72,879,386 |
|
|
|
36,410,692 |
|
|
Property
and equipment, net |
|
190,370 |
|
|
|
143,382 |
|
|
Other
assets |
|
200 |
|
|
|
200 |
|
|
Total
assets |
$ |
73,069,956 |
|
|
$ |
36,554,274 |
|
|
Liabilities and Stockholders' Equity |
|
|
|
|
Current
liabilities: |
|
|
|
|
Accounts
payable |
$ |
3,116,100 |
|
|
$ |
1,848,084 |
|
|
Accrued
expenses |
|
4,479,292 |
|
|
|
4,284,907 |
|
|
Deferred
grant revenue |
|
833,975 |
|
|
|
833,975 |
|
|
Total
current liabilities |
|
8,429,367 |
|
|
|
6,966,966 |
|
|
Stockholders' equity: |
|
|
|
|
Common
stock |
|
13,257 |
|
|
|
9,995 |
|
|
Additional paid-in capital |
|
134,489,599 |
|
|
|
75,545,875 |
|
|
Accumulated deficit |
|
(69,862,267 |
) |
|
|
(45,968,562 |
) |
|
Total
stockholders' equity |
|
64,640,589 |
|
|
|
29,587,308 |
|
|
Total
liabilities and stockholders' equity |
$ |
73,069,956 |
|
|
$ |
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
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