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Zynerba Pharmaceuticals Reports Fourth Quarter and Year End 2018 Financial Results and Operational Highlights
“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
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
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
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
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.
Announced Addition of
Fourth Quarter 2018 Financial Results
The Company’s cash and cash equivalent position as of
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
CONSOLIDATED STATEMENTS OF OPERATIONS
|Three months ended December 31,||Year ended December 31,|
|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):|
|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 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|
CONSOLIDATED BALANCE SHEETS
|December 31, 2018||December 31, 2017|
|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|
|Liabilities and Stockholders' Equity|
|Deferred grant revenue||—||171,975|
|Total current liabilities||9,725,782||7,442,721|
|Deferred grant revenue, long-term||—||662,000|
|Additional paid-in capital||175,476,075||138,916,900|
|Total stockholders' equity||57,601,661||60,949,588|
|Total liabilities and stockholders' equity||$||67,327,443||$||69,054,309|
Source: Zynerba Pharmaceuticals, Inc.