Zogenix Provides Corporate Update and Reports First Quarter Financial Results
- Following receipt of Breakthrough Therapy Designation, conducted successful Type B meeting with
FDAconfirming adequacy of Phase 3 program for ZX008 NDA in Dravet syndrome
- Study 1504 fully enrolled with 87 randomized patients; top-line results expected late June/early July
- On-track to submit U.S. and EU regulatory approval filings for ZX008 in Dravet syndrome in fourth quarter of 2018
“We continue to advance towards multiple key catalysts in our late-stage lead program, ZX008 for the treatment of Dravet syndrome, including results from our second Phase 3 trial, Study 1504, and U.S. and EU regulatory filings targeted for the fourth quarter of 2018,” said
“In addition, we continued to add to the positive efficacy data set for ZX008 in Dravet syndrome through the presentation of further analyses of Study 1, our first Phase 3 trial, in two poster presentations at the 2018
“We also continued to enroll patients in our ongoing global Phase 3 trial of ZX008 in Lennox-Gastaut syndrome (LGS) in children and adults up to age 35, and had 16 open sites in the U.S. and
- Conducted a successful Breakthrough Therapy Type B meeting with the
FDAregarding the ZX008 clinical development program and planned NDA submission in Dravet syndrome. Key outcomes included:
- Reaffirmation of Study 1 and Study 1504 as the clinical basis for the NDA submission;
- Concurrence that the anticipated ZX008 exposures in the Dravet syndrome program at the time of the NDA submission are likely to be sufficient to support the filing; and
- Agreement on the schedule for a rolling NDA submission.
- Enrolled and randomized a total of 87 patients into the efficacy cohort of Study 1504, with last patient scheduled to exit the treatment period at the end of May and top-line results expected end of June/early July.
- Presented new efficacy and safety results from Study 1 at the AAN 2018 Annual Meeting:
- Positive results of a post-hoc analysis evaluating the effect of ZX008 on controlling convulsive seizures in patients in Study 1 who had previously failed therapy with stiripentol. The data demonstrated that patients taking ZX008 0.8 mg/kg/day (max. 30 mg/day) achieved a 60.8% greater reduction in mean monthly convulsive seizures compared to placebo (p=0.002). Additionally, in the 0.8 mg/kg/day group, 72.7% of patients achieved an equal or greater than 50% reduction in convulsive seizures (p=0.006), and 50.0% achieved an equal or greater than 75% reduction (p=0.036). Across these and other efficacy measures, the results from the subset analysis of stiripentol non-responders were comparable to the full Study 1 population, indicating that a history of non-responsiveness to stiripentol is not a predictor of negative response to treatment with ZX008 in Dravet syndrome.
- Positive results of a prespecified secondary analysis evaluating the effect of ZX008 on total seizure frequency, showing that treatment with ZX008 at doses of 0.2 mg/kg/day and 0.8 mg/kg/day (max. 30 mg/day) resulted in significantly greater reductions in median monthly total seizure frequency compared to placebo (p= 0.031 and p<0.001, respectively).
- Encouraging preliminary quality of life and cognitive function data showing that patients treated with ZX008 over the 14-week double-blind treatment period experienced significant improvements on select measures of quality of life and executive function compared to those on placebo.
- Continued enrollment in global Phase 3 trial of ZX008 for treatment of LGS, Study 1601. At the end of April, 16 sites in the U.S. and
Canadawere open to recruit patients and Ethics Committee submissions to several European countries have been completed.
First Quarter 2018 Financial Results
- Due to the wind-down of Sumavel DosePro manufacturing operations in
September 2017, the Company recorded no revenue for the three months ended March 31, 2018. This compares with total revenue of $2.7 millionin the first quarter ended March 31, 2017, consisting entirely of contract manufacturing revenue for Sumavel DosePro.
- Research and development expenses for the first quarter ended Mach 31, 2018, totaled
$23.0 million, up from $13.3 millionin the first quarter ended March 31, 2017, as the Company continued enrollment and expanded the scope of its Phase 3 clinical trials for ZX008 in Dravet syndrome and LGS.
- Selling, general and administrative expenses for the first quarter ended March 31, 2018, totaled
$8.1 million, compared with $6.6 millionin the first quarter ended March 31, 2017.
- Net loss from continuing operations for the first quarter ended
March 31, 2018, was $30.2 million, compared with a net loss from continuing operations of $21.1 millionin the first quarter ended March 31, 2017.
- Total net loss for the first quarter ended March 31, 2018, was
$30.2 million, or a net loss of $0.87per share, compared with a total net loss of $21.3 million, or a net loss of $0.86per share, in the first quarter ended March 31, 2017.
- As of
March 31, 2018, the Company had cash and cash equivalents of $272.0 million, compared to $293.5 millionat December 31, 2017.
Conference Call Details
|Wednesday, May 9th @ 4:30pm Eastern Time/1:30pm Pacific Time
|Replays, available through May 23rd
For more information, visit www.zogenix.com.
646-597-6987 | Andrew@lifesciadvisors.com
Forward Looking Statements
Condensed Consolidated Balance Sheets (Unaudited)
(in thousands, except par value)
|Cash and cash equivalents||$||271,953||$||293,503|
|Other current assets||827||5,206|
|Total current assets||281,327||304,703|
|Property and equipment, net||287||245|
|Liabilities and stockholders’ equity|
|Common stock warrant liabilities||495||512|
|Total current liabilities||23,076||20,983|
|Deferred income taxes||17,425||17,425|
|Other long-term liabilities||684||784|
|Additional paid-in capital||875,957||873,526|
|Total stockholders’ equity||273,772||301,521|
|Total liabilities and stockholders’ equity||$||391,857||$||417,613|
Condensed Consolidated Statements of Operations (Unaudited)
(in thousands, except per share amounts)
|Three Months Ended March 31,|
|Contract manufacturing revenue||$||—||$||2,696|
|Costs and expenses:|
|Cost of contract manufacturing||—||2,487|
|Research and development||22,980||13,341|
|Selling, general and administrative||8,070||6,554|
|Asset impairment charges||—||813|
|Change in fair value of contingent consideration||—||600|
|Total costs and expenses||31,050||23,795|
|Loss from operations||(31,050||)||(21,099||)|
|Other income (expense):|
|Change in fair value of common stock warrant liabilities||17||587|
|Other income (expense)||26||(20||)|
|Total other income (expense)||870||(10||)|
|Loss from continuing operations before income taxes||(30,180||)||(21,109||)|
|Income tax expense||—||(17||)|
|Net loss from continuing operations||(30,180||)||(21,126||)|
|Loss from discontinued operations, net of tax||—||(181||)|
|Net loss per share, basic and diluted:|
|Weighted-average shares used in computing net loss per share, basic and diluted||34,841||24,813|
Source: Zogenix, Inc.