~ Announced Completion of Full Patient Enrollment in HOPE-B Pivotal Study of Etranacogene Dezaparvovec ~
~ Achieved Site Initiation and Began Patient Screening for Phase I/II Clinical Study of AMT-130 in Huntington’s Disease ~
~Strong Balance Sheet with September 30, 2019 Cash and Cash Equivalents of $403 Million Expected to Fund Operations into Mid-2022 ~
LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Oct. 28, 2019 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today reported its financial results for the third quarter of 2019 and highlighted recent progress across its business.
“We achieved a major company milestone for 2019 with the earlier than expected completion of full patient enrollment in the HOPE-B pivotal study of etranacogene dezaparvovec in hemophilia B, as well as the initiation of clinical sites and patient screening for the Phase I/II clinical study of AMT-130 in Huntington’s disease,” stated Matt Kapusta, chief executive officer of uniQure. “We also strengthened our balance sheet with new capital that we expect will fund operations into mid-2022 and allows us to deliver on key corporate goals across the business. We look forward to continuing our momentum through the remainder of the year and sharing our progress during what we believe will be a very productive 2020.”
Recent Company Progress
Upcoming Investor and Industry Events
Financial Highlights
Cash Position: As of September 30, 2019, the Company held cash and cash equivalents of $403.2 million, compared to $184.1 million as of June 30, 2019. The Company currently expects cash and cash equivalents will be sufficient to fund operations into 2022.
Revenues: Revenue for the three months ended September 30, 2019 was $1.0 million, compared to $3.1 million during the same period 2018. The decrease reflects the October 2018 termination of the development of S100A1 under our collaboration with Bristol-Myers Squibb.
R&D Expenses: Research and development expenses were $23.6 million for the three months ended September 30, 2019, compared to $20.5 million during the same period 2018. The change was primarily related to increased activities associated with our ongoing clinical studies of etranacogene dezaparvovec, the planned Phase I/II study of AMT-130, increased share-based compensation and the hiring of additional clinical and operations staff at our Lexington site.
SG&A Expenses: Selling, general and administrative expenses were $8.9 million for three months ended September 30, 2019, compared to $5.9 million during the same period 2018. The change was primarily related to increases in personnel and consulting expenses, professional fees and share-based compensation expenses.
Net Loss: The net loss for the three months ended September 30, 2019 was $23.6 million, or $0.58 per share, compared to $22.0 million, or $0.59 per share during the same period 2018.
About uniQure
uniQure is delivering on the promise of gene therapy – single treatments with potentially curative results. We are leveraging our modular and validated technology platform to rapidly advance a pipeline of proprietary gene therapies to treat patients with hemophilia B, hemophilia A, Huntington's disease, Fabry disease, spinocerebellar ataxia Type 3 and other diseases. www.uniQure.com
uniQure Forward-Looking Statements
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as "anticipate," "believe," "could," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "should," "will," "would" and similar expressions. Forward-looking statements are based on management's beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, our ability to provide further clinical updates and data with respect to our pivotal Phase III study of etranacogene dezaparvovec before the end of 2020 or ever, the ability to submit a BLA for marketing authorization of etranacogene dezaparvovec in 2021, or ever, the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies, our ability to enroll the first sentinel patient in our AMT-130 Phase I/II clinical study in early 2020 or ever, and our ability to fund our operations into mid-2020. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure’s Quarterly Report on Form 10-Q filed on October 28, 2019. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.
uniQure Contacts:
FOR INVESTORS: | FOR MEDIA: | |
Maria E. Cantor Direct: 339-970-7536 Mobile: 617-680-9452 m.canto@uniQure.com | Eva M. Mulder Direct: +31 20 240 6103 Mobile: +31 6 52 33 15 79 e.mulder@uniQure.com | Tom Malone Direct: 339-970-7558 Mobile:339-223-8541 t.malone@uniQure.com |
uniQure N.V.
UNAUDITED CONSOLIDATED BALANCE SHEETS
September 30, | December 31, | |||||
2019 | 2018 | |||||
(in thousands, except share and per share amounts) | ||||||
Current assets | ||||||
Cash and cash equivalents | $ | 403,163 | $ | 234,898 | ||
Accounts receivable and accrued income from related party | 101 | 233 | ||||
Prepaid expenses | 3,820 | 1,116 | ||||
Other current assets | 558 | 329 | ||||
Total current assets | 407,642 | 236,576 | ||||
Non-current assets | ||||||
Property, plant and equipment, net of accumulated depreciation of $26.9 million as of September 30, 2019, and $22.9 million as of December 31, 2018, respectively. | 28,278 | 29,179 | ||||
Operating lease right-of-use assets | 26,852 | - | ||||
Intangible assets, net | 5,424 | 5,201 | ||||
Goodwill | 482 | 506 | ||||
Restricted cash | 2,914 | 2,444 | ||||
Total non-current assets | 63,950 | 37,330 | ||||
Total assets | $ | 471,592 | $ | 273,906 | ||
Current liabilities | ||||||
Accounts payable | $ | 3,782 | $ | 3,792 | ||
Accrued expenses and other current liabilities | 12,702 | 8,232 | ||||
Current portion of operating lease liabilities | 4,436 | - | ||||
Current portion of deferred rent | - | 311 | ||||
Current portion of deferred revenue | 7,527 | 7,634 | ||||
Total current liabilities | 28,447 | 19,969 | ||||
Non-current liabilities | ||||||
Long-term debt | 35,928 | 35,471 | ||||
Operating lease liabilities, net of current portion | 31,242 | - | ||||
Deferred rent, net of current portion | - | 8,761 | ||||
Deferred revenue, net of current portion | 23,882 | 28,861 | ||||
Derivative financial instruments related party | 1,354 | 803 | ||||
Other non-current liabilities | 507 | 435 | ||||
Total non-current liabilities | 92,913 | 74,331 | ||||
Total liabilities | 121,360 | $ | 94,300 | |||
Total shareholders' equity | 350,232 | 179,606 | ||||
Total liabilities and shareholders' equity | $ | 471,592 | $ | 273,906 | ||
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS
Three months ended September 30, | |||||||
2019 | 2018 | ||||||
in thousands, except share and per share amounts | |||||||
Total revenues | $ | 1,046 | $ | 3,148 | |||
Operating expenses: | |||||||
Research and development expenses | (23,554 | ) | (20,541 | ) | |||
Selling, general and administrative expenses | (8,929 | ) | (5,898 | ) | |||
Total operating expenses | (32,483 | ) | (26,439 | ) | |||
Other income | 453 | 557 | |||||
Other expense | (342 | ) | (490 | ) | |||
Loss from operations | (31,326 | ) | (23,224 | ) | |||
Non operating items, net | 7,722 | 1,157 | |||||
Loss before income tax expense | (23,604 | ) | (22,067 | ) | |||
Income tax expense | - | 32 | |||||
Net loss | $ | (23,604 | ) | $ | (22,035 | ) | |
Basic and diluted net loss per ordinary share | $ | (0.58 | ) | $ | (0.59 | ) | |
Weighted average shares used in computing basic and diluted net loss per ordinary share | 40,738,938 | 37,247,193 | |||||