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Zynerba Pharmaceuticals Reports Third Quarter 2017 Financial Results and 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
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
FDAhas 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
FDAguidelines 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
FDAin 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
- 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
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
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
Recent Equity Financing
The Company believes that the cash and cash equivalent position 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. For example, there can be no guarantee that the Company will obtain approval for ZYN002 or ZYN001 from the
|ZYNERBA PHARMACEUTICALS, INC.
|CONSOLIDATED STATEMENTS OF OPERATIONS
|Three months ended||Nine months ended|
|September 30, 2017||September 30, 2016||September 30, 2017||September 30, 2016|
|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):|
|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 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|
| ZYNERBA PHARMACEUTICALS, INC.
|CONSOLIDATED BALANCE SHEETS
|September 30, 2017||December 31, 2016|
|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|
|Liabilities and Stockholders' Equity|
|Deferred grant revenue||833,975||833,975|
|Total current liabilities||8,429,367||6,966,966|
|Additional paid-in capital||134,489,599||75,545,875|
|Total stockholders' equity||64,640,589||29,587,308|
|Total liabilities and stockholders' equity||$||73,069,956||$||36,554,274|