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Zynerba Pharmaceuticals Reports Fourth Quarter and Year End 2019 Financial Results and Operational Highlights
- Key Recent Clinical Milestones Include Completion of Enrollment in Pivotal CONNECT-FX Trial in Fragile X Syndrome and Phase 2 BRIGHT Trial in Autism Spectrum Disorder -
“The fourth quarter of 2019 capped off a year of strong execution by Zynerba,” said
Fourth Quarter 2019 and Recent Highlights
Zygel in Fragile X Syndrome (FXS)
Enrollment Complete in Pivotal FXS Trial; Topline Results Expected in the Second Quarter of 2020
Enrollment is complete with 212 patients randomized into CONNECT-FX, a pivotal, multi-national, randomized, double blind, placebo-controlled trial evaluating the efficacy and safety of Zygel in treating common behavioral symptoms of 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. Key secondary endpoints are the change from baseline to the end of the treatment period in the ABC-CFXS Irritability subscale score and the ABC-CFXS Socially Unresponsive/Lethargic subscale score, and Clinical Global Impression - Improvement (CGI-I) at the end of the treatment period. The Company expects to report topline results late in the second quarter of 2020. If the results are positive, Zynerba intends to request a meeting with the FDA to determine the acceptability of the data as a basis for a New Drug Application (NDA) and to seek advice on preparation of the marketing authorization. The Company expects to submit its NDA for Zygel in FXS to the
Robust Enrollment Continues into Open Label Extension Study
During the screening phase of CONNECT-FX, caregivers of patients in the trial were informed that their participating child may have the opportunity to receive Zygel in an open label extension trial following the child’s compliant completion of CONNECT-FX, regardless of their child’s perceived response or actual blinded drug assignment at randomization in CONNECT-FX. As of
New
The
Poster Describing Health State Utility Indices (HUI) that Estimate the Severity of FXS and Other Pediatric Disorders Presented at the
An HUI specific to FXS, known as the ABC-UI, was derived from the ABC-CFXS to measure the health-related quality of life (HRQoL) benefit of treatments for FXS (Kerr C et al.
Poster Describing Caregivers’ Perspectives on FXS Diagnosis and Patient Journey Perspective Presented at the
The poster described the results of a caregiver survey that found an average age of 3 years at initial diagnosis, a high prevalence of comorbid conditions including ASD and attention-deficit/hyperactivity disorder, and standard of care consisting primarily of counseling/therapy and prescription medications that are not indicated for FXS. While caregivers of children with FXS often notice a variety of initial symptoms early and seek help from a health care professional, it is not until subsequent physician visits, often involving a specialist, that a formal diagnosis is made. (Press release)
Zygel in Autism Spectrum Disorder (ASD)
Completed Enrollment in Phase 2 Open Label Trial of Zygel in ASD; Topline Results Expected in the Second Quarter of 2020
Enrollment is complete in the Phase 2 BRIGHT trial assessing the safety, tolerability and efficacy of Zygel for the treatment of pediatric and adolescent patients with ASD. The 14-week trial is evaluating the efficacy and safety of Zygel in 37 children and adolescents (ages four through 17) with moderate-to-severe ASD. The efficacy assessments include the Aberrant Behavior Checklist, Parent Rated Anxiety Scale – Autism Spectrum Disorder, Autism Impact Measure, and Clinical Global Impression – Severity and Improvement. The mean age of the 37 patients enrolled in the BRIGHT trial is 9.2 years. Ninety-two (92) percent of the enrolled patients are male, accurately reflecting the overall prevalence and gender ratio of moderate-to-severe ASD in
New
The
Poster Describing the Baseline Characteristics of Patients in Phase 2 BRIGHT Trial in ASD Presented at the
The poster further describes the baseline characteristics of the pediatric and adolescent patients in the fully-enrolled Phase 2 BRIGHT trial, indicating that the trial enrolled a broad patient population and was enriched for disease severity to avoid floor effects on outcome measures. At baseline, at least 92% of patients have moderate to severe symptoms of ASD as measured by the Autism Diagnostic Observation Schedule (ADOS®-2) comparison score and the Diagnostic and Statistical Manual of Mental Disorders 5th edition (DSM-5) severity level score; this severity is further confirmed by an ABC-C Irritability subscale score of 30. In addition, 24% of patients enrolled in the BRIGHT trial had a PRAS-ASD score of >52, indicating possible clinical anxiety. (Press release)
Zygel in 22q11.2 Deletion Syndrome (22q)
Phase 2 Open Label Trial of Zygel in 22q Ongoing; Data Now Expected in the Third Quarter of 2020
The Company is conducting the 14-week Phase 2 INSPIRE trial to evaluate the safety, tolerability and efficacy of Zygel in approximately 20 children and adolescents (ages six through 17) with genetically-confirmed 22q. The efficacy assessments include the Aberrant Behavior Checklist-Community (ABC-C), the Anxiety, Depression and Mood Scale (
Zygel in Developmental and Epileptic Encephalopathies (DEE)
Meeting with
Zynerba expects to meet with the FDA to discuss the clinical path forward in DEE. Based on the Phase 2 trial design and positive efficacy and safety results, Zynerba anticipates that it will discuss the pursuit of an indication that includes all syndromes and encephalopathies in the DEE category that present with focal impaired-awareness seizures (FIAS; previously known as complex partial seizures) and/or convulsive seizures (CS), the most common and debilitating seizure types representing 75% to 80% of all seizures.
Corporate
Enhanced Senior Management Team
Fourth quarter and full year 2019 Financial Results
As of
Research and development expenses for the year ended
Financial Outlook
The Company’s cash and cash equivalents as of
About
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
(unaudited) | ||||||||||||||||
Three months ended |
Year ended |
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2019 | 2018 | 2019 | 2018 | |||||||||||||
Revenue | $ | — | $ | 86,000 | $ | — | $ | 86,000 | ||||||||
Operating expenses: | ||||||||||||||||
Research and development | 7,457,953 | 4,876,162 | 20,384,049 | 27,245,043 | ||||||||||||
General and administrative | 3,958,211 | 3,256,044 | 13,935,761 | 13,238,787 | ||||||||||||
Total operating expenses | 11,416,164 | 8,132,206 | 34,319,810 | 40,483,830 | ||||||||||||
Loss from operations | (11,416,164 | ) | (8,046,206 | ) | (34,319,810 | ) | (40,397,830 | ) | ||||||||
Other income (expense): | ||||||||||||||||
Interest income | 295,140 | 321,621 | 1,522,138 | 961,323 | ||||||||||||
Foreign exchange gain (loss) | 406,033 | (65,658 | ) | (145,911 | ) | (474,668 | ) | |||||||||
Total other income | 701,173 | 255,963 | 1,376,227 | 486,655 | ||||||||||||
Net loss | $ | (10,714,991 | ) | $ | (7,790,243 | ) | $ | (32,943,583 | ) | $ | (39,911,175 | ) | ||||
Net loss per share - basic and diluted | $ | (0.46 | ) | $ | (0.44 | ) | $ | (1.50 | ) | $ | (2.61 | ) | ||||
Basic and diluted weighted average shares outstanding | 23,191,428 | 17,616,373 | 22,000,203 | 15,308,886 | ||||||||||||
Non-cash stock-based compensation included above: | ||||||||||||||||
Research and development | $ | 456,420 | $ | 819,715 | $ | 2,371,998 | $ | 3,087,498 | ||||||||
General and administrative | 751,253 | 778,915 | 3,189,897 | 3,538,245 | ||||||||||||
Total | $ | 1,207,673 | $ | 1,598,630 | $ | 5,561,895 | $ | 6,625,743 | ||||||||
CONSOLIDATED BALANCE SHEETS
Assets | ||||||||
Current assets: | ||||||||
Cash and cash equivalents | $ | 70,063,242 | $ | 59,763,773 | ||||
Incentive and tax receivables | 14,613,969 | 3,444,620 | ||||||
Prepaid expenses and other current assets | 2,378,812 | 3,747,087 | ||||||
Total current assets | 87,056,023 | 66,955,480 | ||||||
Property and equipment, net | 362,724 | 371,963 | ||||||
Right-of-use assets | 345,849 | — | ||||||
Total assets | $ | 87,764,596 | $ | 67,327,443 | ||||
Liabilities and Stockholders' Equity | ||||||||
Current liabilities: | ||||||||
Accounts payable | $ | 4,740,981 | $ | 4,461,567 | ||||
Accrued expenses | 7,073,506 | 5,264,215 | ||||||
Lease liabilities | 243,677 | — | ||||||
Total current liabilities | 12,058,164 | 9,725,782 | ||||||
Lease liabilities, long-term | 109,689 | — | ||||||
Total liabilities | 12,167,853 | 9,725,782 | ||||||
Stockholders' equity: | ||||||||
Common stock | 23,211 | 17,627 | ||||||
Additional paid-in capital | 226,409,156 | 175,476,075 | ||||||
Accumulated deficit | (150,835,624 | ) | (117,892,041 | ) | ||||
Total stockholders' equity | 75,596,743 | 57,601,661 | ||||||
Total liabilities and stockholders' equity | $ | 87,764,596 | $ | 67,327,443 | ||||
Zynerba Contacts
484.581.7483
fickenscherj@zynerba.com
484.581.7489
robertsw@zynerba.com
Source: Zynerba Pharmaceuticals, Inc.