~ Patient Dosing Nearly Completed in Phase III HOPE-B Trial of Etranacogene Dezaparvovec
in Hemophilia B ~
~ First Patient Procedure in Phase I/II Clinical Trial of AMT-130 in Huntington’s Disease
Expected to Occur Around the End of the First Quarter of 2020 ~
~ Strong Balance Sheet Expected to Fund Operations into 2022 ~
LEXINGTON, Mass. and AMSTERDAM, March 02, 2020 (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 2019 and highlighted recent progress across its business.
“The past year was marked by strong execution in our pivotal, Phase III HOPE-B study of etranacogene dezaparvovec in hemophilia B for which we achieved full enrollment and expect to announce top-line data before the end of this year,” stated Matt Kapusta, chief executive officer of uniQure. “We believe etranacogene dezaparvovec has a potential best-in-class profile as a one-time treatment for patients with hemophilia B, and our primary goal is to advance this program for regulatory review and potential approval.”
“We have also successfully screened patients in the randomized Phase I/II clinical trial of AMT-130 in Huntington’s disease and expect the first procedure to occur around the end of this first quarter of 2020,” he added. “In addition, we have continued to advance our research pipeline of innovative AAV gene therapy candidates and further build on our proprietary, commercial-scale manufacturing capabilities. We expect this year to be a transformational one for uniQure in which we announce new clinical data and advance our internally discovered investigational gene therapies.”
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Financial Highlights
Cash Position: As of December 31, 2019, the Company held cash and cash equivalents of $377.8 million, compared to $234.9 million as of December 31, 2018. The Company currently expects cash and cash equivalents will be sufficient to fund operations into 2022.
Revenues: Revenue for the year ended December 31, 2019 was $7.3 million, compared to $11.3 million for the year ended December 31, 2018. The decrease in 2019 reflects the termination of the S100A1 program for the treatment of congestive heart failure under our collaboration with Bristol-Myers Squibb.
R&D Expenses: Research and development expenses were $94.7 million for the year ended December 31, 2019, compared to $74.8 million for the year ended December 31, 2018. The change was primarily related to increased activities associated with our ongoing clinical studies of etranacogene dezaparvovec, the recently initiated 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 $33.5 million for the year ended December 31, 2019, compared to $25.3 million for the year ended December 31, 2018. The change was primarily related to increases in personnel and consulting expenses, professional fees and share-based compensation expenses.
Other income, net: Other expense was $0.1 million for 2019, compared to other income of $0.6 million for 2018.
Net Loss: The net loss for the full years 2019 and 2018 was $124.2 million, or $3.11 per share and $83.3 million, or $2.34 per share, respectively.
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, the ability to provide top-line data or any further clinical updates and data with respect to our pivotal Phase III study of AMT-061 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 ongoing or planned clinical studies, the ability to dose the first sentinel patient in the AMT-130 Phase I/II clinical study by around the end of the first quarter of 2020, or ever, the ability to fund operations into 2022, whether AMT-061 proves to have a best-in-class profile as a one-time treatment for patients with hemophilia, whether 2020 will be a transformational year for uniQure in which new clinical data is announced or investigational gene therapies are advanced, whether we can provide a safety update for AMT-130 this year, whether the Data Safety Monitoring Board for AMT-130 will convene later in 2021, whether important imaging and biomarker data from the AMT-130 trial will be obtained in 2021, or ever, whether IND-enabling studies for Spinocerebellar Ataxia Type 3 (SCA3) are initiated in 2020, and whether an IND application for SCA3 will be filed in 2021, or ever. 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 Annual Report on Form 10-K filed on March 2, 2020. 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 | Eva M. Mulder | Tom Malone |
Direct: 339-970-7536 | Direct: +31 20 240 6103 | Direct: 339-970-7558 |
Mobile: 617-680-9452 | Mobile: +31 6 52 33 15 79 | Mobile:339-223-8541 |
m.canto@uniQure.com | e.mulder@uniQure.com | t.malone@uniQure.com |
uniQure N.V. | ||||||
UNAUDITED CONSOLIDATED BALANCE SHEETS | ||||||
December 31, | December 31, | |||||
2019 | 2018 | |||||
(in thousands, except share and per share amounts) | ||||||
Current assets | ||||||
Cash and cash equivalents | $ | 377,793 | $ | 234,898 | ||
Accounts receivable and accrued income from related party | 947 | 233 | ||||
Prepaid expenses | 4,718 | 1,116 | ||||
Other current assets | 748 | 329 | ||||
Total current assets | 384,206 | 236,576 | ||||
Non-current assets | ||||||
Property, plant and equipment, net | 28,771 | 29,179 | ||||
Operating lease right-of-use assets | 26,797 | - | ||||
Intangible assets, net | 5,427 | 5,201 | ||||
Goodwill | 496 | 506 | ||||
Restricted cash | 2,933 | 2,444 | ||||
Total non-current assets | 64,424 | 37,330 | ||||
Total assets | $ | 448,630 | $ | 273,906 | ||
Current liabilities | ||||||
Accounts payable | $ | 5,681 | $ | 3,792 | ||
Accrued expenses and other current liabilities | 12,457 | 8,232 | ||||
Current portion of operating lease liabilities | 5,865 | - | ||||
Current portion of deferred rent | - | 311 | ||||
Current portion of deferred revenue | 7,627 | 7,634 | ||||
Total current liabilities | 31,630 | 19,969 | ||||
Non-current liabilities | ||||||
Long-term debt | 36,062 | 35,471 | ||||
Operating lease liabilities, net of current portion | 31,133 | - | ||||
Deferred rent, net of current portion | - | 8,761 | ||||
Deferred revenue, net of current portion | 23,138 | 28,861 | ||||
Derivative financial instruments related party | 3,075 | 803 | ||||
Other non-current liabilities | 534 | 435 | ||||
Total non-current liabilities | 93,942 | 74,331 | ||||
Total liabilities | 125,572 | 94,300 | ||||
Total shareholders' equity | 323,058 | 179,606 | ||||
Total liabilities and shareholders' equity | $ | 448,630 | $ | 273,906 | ||
uniQure N.V. | ||||||||||||
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||||||
Years ended December 31, | ||||||||||||
2019 | 2018 | 2017 | ||||||||||
(in thousands, except share and per share amounts) | ||||||||||||
Total revenues | 7,281 | 11,284 | 13,107 | |||||||||
Operating expenses: | ||||||||||||
Research and development expenses | (94,737 | ) | (74,809 | ) | (72,172 | ) | ||||||
Selling, general and administrative expenses | (33,544 | ) | (25,305 | ) | (24,635 | ) | ||||||
Total operating expenses | (128,281 | ) | (100,114 | ) | (96,807 | ) | ||||||
Other income | 1,888 | 2,146 | 15,430 | |||||||||
Other expense | (2,028 | ) | (1,548 | ) | (3,073 | ) | ||||||
Loss from operations | (121,140 | ) | (88,232 | ) | (71,343 | ) | ||||||
Non operating items, net | (3,061 | ) | 5,159 | (8,116 | ) | |||||||
Loss before income tax expense | (124,201 | ) | (83,073 | ) | (79,459 | ) | ||||||
Income tax expense / (benefit) | - | (231 | ) | 199 | ||||||||
Net loss | $ | (124,201 | ) | $ | (83,304 | ) | $ | (79,260 | ) | |||
Basic and diluted net loss per ordinary share | $ | (3.11 | ) | $ | (2.34 | ) | $ | (2.94 | ) | |||
Weighted average shares used in computing basic and diluted net loss per ordinary share | 39,999,450 | 35,639,745 | 26,984,183 | |||||||||