DEVON, Pa., March 11, 2019 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (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 Zygelâ„¢ 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.â€
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.
Corporate
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,â€
“estimates,†“anticipates,†“expects,†“plans,†“intends,†“may,â€
“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. | |||||||||||||||