~ Six patient procedures now completed in Phase I/II for Huntington’s disease, including two procedures conducted in February 2021, with full enrollment of first cohort expected mid-2021 ~
~ Last patient in HOPE-B pivotal study to complete 52-week follow-up visit before end of the first quarter of 2021, with top-line data to be presented in the second quarter of 2021 ~
~ Analysis of the HOPE-B safety event expected to be completed and submitted to the FDA before end of the first quarter of 2021 ~
~ Research & Development Day to be held mid-2021 to announce expanded pipeline and platform advancements ~
LEXINGTON, Mass. and AMSTERDAM, March 01, 2021 (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 2020 and highlighted recent progress across its business.
“Despite unprecedented challenges in 2020, the uniQure team demonstrated extraordinary resiliency and dedication to the patients we serve, qualities which have defined our company for more than two decades,” stated Matt Kapusta, chief executive officer at uniQure. “I’m very proud of uniQure’s accomplishments, including completing patient dosing in the HOPE-B pivotal trial, presenting positive 26-week data on 54 patients that met the study’s first primary endpoint, and announcing a landmark transaction with CSL Behring for the global commercialization of etranacogene dezaparvovec, which is expected to close in the second quarter of 2021 following the U.S. antitrust review. We also became the first company to initiate the clinical investigation of an AAV gene therapy for Huntington’s disease and have now enrolled six patients in the study.”
“Looking at 2021, we are focused on important catalysts as we prepare the filings for regulatory approval of etranacogene dezaparvovec and advance our Phase I/II study of AMT-130 in Huntington’s disease, with early biomarker data on the initial patients expected as early as the end of this year. We are also making investments to expand our pipeline of promising gene therapies and further strengthen our AAV manufacturing platform. We look forward to hosting a Research and Development Day mid-year during which we will unveil new research programs and other technology initiatives.”
Recent Company Progress
Upcoming Investor Events (each to be conducted virtually)
Financial Highlights
Cash Position: As of December 31, 2020, the Company held cash and cash equivalents of $244.9 million, compared to $377.8 million as of December 31, 2019.
Revenues: Revenue for the year ended December 31, 2020 was $37.5 million, compared to $7.3 million during the same period in 2019. The change was primarily related to an increase in non-cash license revenue that we recognized as of the December 1, 2020 effective date of the amended Bristol-Myers Squibb collaboration and license agreement, as well as from revenue that we recognized in December 2020 following achievement of a research milestone for one of the four Collaboration Targets.
R&D Expenses: Research and development expenses were $122.4 million for the year ended December 31, 2020, compared to $94.7 million during the same period in 2019. The change was primarily related to increased activities associated with our ongoing clinical studies of etranacogene dezaparvovec and AMT-130, increased share-based compensation, increased license expenses and the recruitment of personnel to support the development of our product candidates.
SG&A Expenses: Selling, general and administrative expenses were $42.6 million for the year ended December 31, 2020, compared to $33.5 million during the same period in 2019. The change was primarily related to increases in personnel and consulting expenses to support our growth, as well as increases in professional fees.
Other non-operating items, net: Other expenses were $16.0 million for the year ended December 31, 2020, compared to other expenses of $3.1 million during the same period in 2019 primarily related to foreign exchange results related to changes in the exchange rates between the Euro and the U.S. Dollar.
Net Loss: The net loss for the year ended December 31, 2020 was $125.0 million, or $2.81 loss per share, compared to $124.2 million, or $3.11 loss per share during the same period in 2019.
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, 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, uniQure’s expectations about its cash runway, the timing of submitting data to the FDA or providing an update related to the investigation of any safety event related to the HOPE-B study, the enrollment of patients in, or Data Safety Monitoring Board review of, our Phase I/II gene therapy clinical trial of AMT-130 in Huntington’s disease, the timing of the announcement of data in our Phase I/II gene therapy clinical trial of AMT-130, the timing of the announcement of data in our HOPE-B pivotal trial of etranacogene dezaparvovec, the timing of filing for regulatory approval of etranacogene dezaparvovec, the timing of regulatory approval of our agreement with CSL Behring, the potential financial compensation we may be paid pursuant to our agreement with CSL Behring, our plans to advance or expand our pipeline, accelerate research, identify business development opportunities, invest in technology, or scale our manufacturing capabilities. uniQure’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with the impact of the ongoing COVID-19 pandemic on our Company and the wider economy and health care system, our Commercialization and License Agreement with CSL Behring, the regulatory approval of that transaction, 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 periodic securities filings, including its Annual Report on Form 10-K filed March 1, 2021. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and uniQure assumes 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 | Chiara Russo | Tom Malone |
Direct: 339-970-7536 | Direct: 617-306-9137 | Direct: 339-970-7558 |
Mobile: 617-680-9452 | Mobile: 617-306-9137 | Mobile:339-223-8541 |
m.cantor@uniQure.com | c.russo@uniQure.com | t.malone@uniQure.com |
uniQure N.V.
UNAUDITED CONSOLIDATED BALANCE SHEETS
December 31, | December 31, | |||||
2020 | 2019 | |||||
(in thousands, except share and per share amounts) | ||||||
Current assets | ||||||
Cash and cash equivalents | $ | 244,932 | $ | 377,793 | ||
Accounts receivables | 6,618 | - | ||||
Accounts receivable from related party | - | 947 | ||||
Prepaid expenses | 4,337 | 4,718 | ||||
Other current assets | 3,024 | 748 | ||||
Total current assets | 258,911 | 384,206 | ||||
Non-current assets | ||||||
Property, plant and equipment, net | 32,328 | 28,771 | ||||
Operating lease right-of-use assets | 26,086 | 26,797 | ||||
Intangible assets, net | 3,361 | 5,427 | ||||
Goodwill | 542 | 496 | ||||
Restricted cash | 2,748 | 2,933 | ||||
Deferred tax asset | 16,419 | - | ||||
Total non-current assets | 81,484 | 64,424 | ||||
Total assets | $ | 340,395 | $ | 448,630 | ||
Current liabilities | ||||||
Accounts payable | $ | 3,772 | $ | 5,681 | ||
Accrued expenses and other current liabilities | 18,038 | 12,457 | ||||
Current portion of operating lease liabilities | 5,524 | 5,865 | ||||
Current portion of deferred revenue | - | 7,627 | ||||
Total current liabilities | 27,334 | 31,630 | ||||
Non-current liabilities | ||||||
Long-term debt | 35,617 | 36,062 | ||||
Operating lease liabilities, net of current portion | 30,403 | 31,133 | ||||
Deferred revenue, net of current portion | - | 23,138 | ||||
Derivative financial instruments related party | - | 3,075 | ||||
Other non-current liabilities | 3,136 | 534 | ||||
Total non-current liabilities | 69,156 | 93,942 | ||||
Total liabilities | $ | 96,490 | $ | 125,572 | ||
Shareholders' equity | ||||||
Total shareholders' equity | 243,905 | 323,058 | ||||
Total liabilities and shareholders' equity | $ | 340,395 | $ | 448,630 | ||
uniQure N.V.
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS
Years ended December 31, | |||||||||||||
2020 | 2019 | 2018 | |||||||||||
(in thousands, except share and per share amounts) | |||||||||||||
Total revenues | $ | 37,514 | $ | 7,281 | $ | 11,284 | |||||||
Operating expenses: | |||||||||||||
Research and development expenses | (122,400 | ) | (94,737 | ) | (74,809 | ) | |||||||
Selling, general and administrative expenses | (42,580 | ) | (33,544 | ) | (25,305 | ) | |||||||
Total operating expenses | (164,980 | ) | (128,281 | ) | (100,114 | ) | |||||||
Other income | 3,342 | 1,888 | 2,146 | ||||||||||
Other expense | (1,302 | ) | (2,028 | ) | (1,548 | ) | |||||||
Loss from operations | (125,426 | ) | (121,140 | ) | (88,232 | ) | |||||||
Non-operating items, net | (16,017 | ) | (3,061 | ) | 5,159 | ||||||||
Loss before income tax income / (expense) | (141,443 | ) | (124,201 | ) | (83,073 | ) | |||||||
Income tax income / (expense) | 16,419 | - | (231 | ) | |||||||||
Net loss | $ | (125,024 | ) | $ | (124,201 | ) | $ | (83,304 | ) | ||||
Basic and diluted net loss per ordinary share | $ | (2.81 | ) | $ | (3.11 | ) | $ | (2.35 | ) | ||||
Weighted average shares used in computing basic and diluted net loss per ordinary share | 44,466,365 | 39,999,450 | 35,639,745 | ||||||||||