Full Press Release Details
Announces Third Quarter 2020 Financial Results
Highlights Recent Company Progress
Enrolled First Four Patients in Phase I/II Clinical Trial of AMT-130 in Huntington's Disease ~
Top-Line Data from HOPE-B Pivotal Trial in Hemophilia B Expected Before Year End ~
Initiated IND-enabling Studies for AMT-150 in Spinocerebellar Ataxia Type 3 (SCA3) ~
MA and Amsterdam, the Netherlands, October 27, 2020 - 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 2020 and highlighted recent progress across its business.
made excellent progress during the third quarter in our Phase I/II gene therapy clinical trial of AMT-130 in Huntington's
disease as the first four patients have now been enrolled in the study, and we look forward to completing enrollment of our first
dose cohort by mid-2021," stated Matt Kapusta, chief executive officer at uniQure. "Regarding our hemophilia B gene
therapy program, all 54 patients in the HOPE-B pivotal trial of etranacogene dezaparvovec have now completed their 26-week follow-up
visits, and we remain on track to announce top-line data from this registration-enabling study before the end of this year. This
is expected to be the first data from a pivotal study of a hemophilia B gene therapy, as well as the first clinical data from
a large-scale study evaluating gene therapy in patients irrespective of their preexisting AAV neutralizing antibodies. We also
continue to work toward the regulatory approval and closing of our landmark licensing agreement for etranacogene dezaparvovec
like to specially thank all our employees for their tireless focus and dedication during these challenging times," he added.
"We've made very strong progress thus far in 2020, and I look forward to ending the year on a strong note."
Investor Events (each to be conducted virtually)
Position: As of September 30, 2020, the Company held cash and cash equivalents of $279.5 million, compared to $314.3 million
as of June 30, 2020.
Revenue for the three months ended September 30, 2020 was $1.8 million, compared to $1.0 million during the same period in
Expenses: Research and development expenses were $36.3 million for the three months ended September 30, 2020, compared to
$23.6 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.
Expenses: Selling, general and administrative expenses were $10.8 million for the three months ended September 30, 2020, compared
to $8.9 million during the same period in 2019. The change was primarily related to increases in personnel and consulting expenses
and share-based compensation expenses.
non-operating items, net: Other expense was $9.3 million for the three months ended September 30, 2020, compared to other
income of $7.7 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.
Loss: The net loss for the three months ended September 30, 2020 was $53.8 million, or $1.21 loss per share, compared to $23.6
million, or $0.58 loss per share during the same period in 2019.
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
Forward-Looking Statements
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 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 HOPE-B pivotal trial of etranacogene
dezaparvovec, the regulatory approval of our agreement with CSL Behring, the potential financial compensation we may be paid pursuant
to our agreement with CSL Behring, the potential filing of an IND application for our AMT-150 product candidate for Spinocerebellar
Ataxia Type 3 (SCA3), our plans to advance or expand our pipeline, accelerate research, identify business development opportunities,
invest in technology, or scale our manufacturing capabilities, our plans to nominate and appoint Robert Gut to our Board. 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 2, 2020 and
Quarterly Report on Form 10-Q filed on October 27, 2020. 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.
| 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 |
CONSOLIDATED BALANCE SHEETS
| September 30, | December 31, | |||||||
| 2020 | 2019 | |||||||
| (in thousands, except share and per share amounts) | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 279,493 | $ | 377,793 | ||||
| Accounts receivable and accrued income from related party | 223 | 947 | ||||||
| Prepaid expenses | 4,168 | 4,718 | ||||||
| Other current assets | 3,496 | 748 | ||||||
| Total current assets | 287,380 | 384,206 | ||||||
| Non-current assets | ||||||||
| Property, plant and equipment, net | 29,636 | 28,771 | ||||||
| Operating lease right-of-use assets | 26,207 | 26,797 | ||||||
| Intangible assets, net | 4,052 | 5,427 | ||||||
| Goodwill | 518 | 496 | ||||||
| Restricted cash | 2,714 | 2,933 | ||||||
| Total non-current assets | 63,127 | 64,424 | ||||||
| Total assets | $ | 350,507 | $ | 448,630 | ||||
| Current liabilities | ||||||||
| Accounts payable | $ | 2,348 | $ | 5,681 | ||||
| Accrued expenses and other current liabilities | 17,784 | 12,457 | ||||||
| Current portion of operating lease liabilities | 5,552 | 5,865 | ||||||
| Current portion of deferred revenue | 7,666 | 7,627 | ||||||
| Total current liabilities | 33,350 | 31,630 | ||||||
| Non-current liabilities | ||||||||
| Long-term debt, net of current portion | 35,494 | 36,062 | ||||||
| Operating lease liabilities, net of current portion | 30,333 | 31,133 | ||||||
| Deferred revenue, net of current portion | 22,413 | 23,138 | ||||||
| Derivative financial instruments related party | 547 | 3,075 | ||||||
| Other non-current liabilities | 477 | 534 | ||||||
| Total non-current liabilities | 89,264 | 93,942 | ||||||
| Total liabilities | $ | 122,614 | $ | 125,572 | ||||
| Shareholders' equity | ||||||||
| Total shareholders' equity | 227,893 | 323,058 | ||||||
| Total liabilities and shareholders' equity | $ | 350,507 | $ | 448,630 |
CONSOLIDATED STATEMENTS OF OPERATIONS
| Three months ended September 30, | ||||||||
| 2020 | 2019 | |||||||
| (in thousands, except share and per share amounts) | ||||||||
| Total revenues | $ | 1,789 | $ | 1,046 | ||||
| Operating expenses: | ||||||||
| Research and development expenses | (36,302 | ) | (23,554 | ) | ||||
| Selling, general and administrative expenses | (10,789 | ) | (8,929 | ) | ||||
| Total operating expenses | (47,091 | ) | (32,483 | ) | ||||
| Other income | 1,032 | 453 | ||||||
| Other expense | (220 | ) | (342 | ) | ||||
| Loss from operations | (44,490 | ) | (31,326 | ) | ||||
| Non operating items, net | (9,285 | ) | 7,722 | |||||
| Net loss | $ | (53,775 | ) | $ | (23,604 | ) | ||
| Basic and diluted net loss per ordinary share | $ | (1.21 | ) | $ | (0.58 | ) | ||
| Weighted average shares used in computing basic and diluted net loss per ordinary share | 44,471,844 | 40,738,938 |