Full Press Release Details
Third Quarter 2023 Financial Results
Recent Company Progress
clearance of Investigational New Drug (IND) application for AMT-260 in
temporal lobe epilepsy ~
provide clinical update from U.S. and European Phase I/II trials of AMT-130 in
disease later in the fourth quarter of 2023 ~
CSL win 2023 Prix Galien USA award for HEMGENIX ~
~ Announced strategic
reorganization to focus on advancing multiple clinical-stage programs and expect
million of cost savings over the next three years ~
position of approximately $660 million as of September 30, 2023 expected to
into the second quarter of 2027 ~
MA and Amsterdam, the Netherlands, November 7, 2023 - 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 2023 and highlighted recent progress across its business.
quarter we made significant progress towards our goal of advancing multiple clinical-stage programs and focusing resources to generate
value from these important investments," stated Matt Kapusta, chief executive officer of uniQure. "We are very pleased
to have achieved FDA clearance of the IND for AMT-260 in refractory mesial temporal lobe epilepsy and look forward to initiating patient
enrollment in the Phase I/IIa study as soon as possible. In addition, we are making good progress toward initiating patient screening
for a Phase I/II trial of AMT-162 in SOD1-ALS and the submission of an IND for AMT-191 in Fabry disease. We also look forward to providing
another clinical update later this quarter from our ongoing Phase I/II studies of AMT-130 in Huntington's disease, which will include
up to 2.5 years of patient follow-up."
for 2024, we remain laser focused on execution across our portfolio of gene therapy product candidates with the goal of achieving clinical
proof-of-concept as rapidly as possible. We also remain committed to carefully managing costs, prudently
allocating capital, and thoughtfully evaluating strategies that can enhance value for shareholders in an increasingly challenging market
Upcoming Investor Events
Financial Highlights
position: As of September 30, 2023, the Company held cash and cash equivalents and investment securities of $658.9 million, compared
to $392.8 million as of December 31, 2022. The Company entered into a royalty agreement in May 2023 and received an upfront payment of
$375.0 million, and collected $100.0 million in July 2023 for a milestone due from CSL following the first sale of HEMGENIX
Revenues for the three months ended September 30, 2023 were $1.4 million, compared to $1.4 million in the same period in 2022. The
current period included an increase in license revenues of $0.5 million and contract manufacturing revenues of $0.4 million related to
contract manufacturing HEMGENIX for CSL, and a decrease
of $0.9 million in collaboration revenues.
Research and development expenses were $65.4 million for the three months ended September 30, 2023, compared to $48.1 million in
the same period in 2022. The change was primarily related to a non-cash, $8.8 million increase in the fair value of contingent consideration
associated with the Company's acquisition of Corlieve Therapeutics in 2021.
Selling, general and administrative expenses were $18.1 million for the three months ended September 30, 2023, compared to $13.3
million in the same period in 2022. The increase was primarily related to an increase in personnel and contractor-related expenses, and
in legal fees associated with various corporate initiatives.
items, net was an expense of $7.8 million for the three months ended September 30, 2023, compared to net income of $11.3 million for
the same period in 2022. The decrease in other non-operating items, net was primarily related to a decrease in foreign currency gains,
net of $14.2 million and an increase in interest expense of $12.4 million primarily related to the royalty agreement that the Company
entered into in May 2023, which partially was offset by an increase of $7.5 million in interest income earned on investment securities
the three months ended September 30, 2023, was $89.6 million, or $1.88 basic and diluted loss per ordinary share, compared to $47.9 million
net loss for the same period in 2022, or $1.02 basic and diluted loss per ordinary share.
mission is to reimagine the future of medicine by delivering innovative cures that transform lives. The recent approvals of our gene
therapy for hemophilia B - a historic achievement based on more than a decade of research and clinical development - represent
a major milestone in the field of genomic medicine and ushers in a new treatment approach for patients living with hemophilia. We are
now leveraging our modular and validated technology and manufacturing platform to advance a pipeline
of proprietary gene therapies for the treatment of patients with Huntington's disease, refractory mesial temporal lobe epilepsy,
amyotrophic lateral sclerosis (ALS), Fabry disease, and other severe diseases. www.uniQure.com
uniQure Forward-Looking
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," "establish," "estimate,"
"expect," "goal," "intend," "look forward to", "may," "plan," "potential,"
"predict," "project," "seek," "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 Company's statements about its
cash runway and its ability to fund operations into 2027, the Company's cost savings related to the recently-announced strategic
organization, whether there will be continued progress on the commercialization of HEMGENIX , the Company's plans
to announce follow up data from the U.S. and European Phase I/II clinical study of AMT-130 in 2023, whether that data will help to further
guide our ongoing clinical development of AMT-130, the Company's expectation to submit an investigational new drug application
for AMT-191 in Fabry disease in 2023 and begin patient dosing in 2024, the Company's expectation to initiate patient screening
in AMT-162 and AMT-260 in the fourth quarter of 2023 and begin patient dosing in each in the first quarter of 2024, and whether the Company
will begin interactions with regulatory agencies in the first quarter of 2024 with respect to AMT-130. The Company's actual results
could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks
associated with the regulatory approval and commercial launch of HEMGENIX , material changes to our interim or preliminary
data, our clinical trial for Huntington's disease, the impact of financial and geopolitical events on our Company and the wider
economy and health care system, the Company's ability to raise additional capital to support late stage development of the Company's
clinical program(s) if supported by future data, our Commercialization and License Agreement with CSL Behring, our clinical development
activities, clinical results, collaboration arrangements, regulatory oversight, product commercialization and intellectual property claims
and ongoing litigation, as well as the risks, uncertainties and other factors described under
the heading "Risk Factors" in the Company's periodic securities filings, including its Annual Report on Form 10-K filed
February 27, 2023 and the Quarterly Report on Form 10-Q filed November 7, 2023. Given these risks, uncertainties and other factors, you
should not place undue reliance on these forward-looking statements, and the Company assumes no obligation to update these forward-looking
statements, even if new information becomes available in the future.
| 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 |
UNAUDITED CONSOLIDATED
| September 30, | December 31, | |||||||
| 2023 | 2022 | |||||||
| (in thousands, except share and per share amounts) | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 229,484 | $ | 228,012 | ||||
| Current investment securities | 429,428 | 124,831 | ||||||
| Accounts receivable and contract asset | 1,644 | 102,376 | ||||||
| Inventories | 15,654 | 6,924 | ||||||
| Prepaid expenses | 14,884 | 11,817 | ||||||
| Other current assets and receivables | 2,532 | 2,814 | ||||||
| Total current assets | 693,626 | 476,774 | ||||||
| Non-current assets | ||||||||
| Property, plant and equipment, net | 45,946 | 50,532 | ||||||
| Non-current investment securities | - | 39,984 | ||||||
| Operating lease right-of-use assets | 30,360 | 32,726 | ||||||
| Intangible assets, net | 57,976 | 58,778 | ||||||
| Goodwill | 25,273 | 25,581 | ||||||
| Deferred tax assets, net | 12,351 | 14,528 | ||||||
| Other non-current assets | 6,018 | 6,061 | ||||||
| Total non-current assets | 177,924 | 228,190 | ||||||
| Total assets | $ | 871,550 | $ | 704,964 | ||||
| Current liabilities | ||||||||
| Accounts payable | $ | 5,584 | $ | 10,984 | ||||
| Accrued expenses and other current liabilities | 28,427 | 30,571 | ||||||
| Current portion of contingent consideration | 26,708 | 25,982 | ||||||
| Current portion of operating lease liabilities | 7,888 | 8,382 | ||||||
| Total current liabilities | 68,607 | 75,919 | ||||||
| Non-current liabilities | ||||||||
| Long-term debt | 101,431 | 102,791 | ||||||
| Liability from royalty financing agreement | 383,711 | - | ||||||
| Operating lease liabilities, net of current portion | 28,977 | 31,719 | ||||||
| Contingent consideration, net of current portion | 14,030 | 9,334 | ||||||
| Deferred tax liability, net | 4,917 | 8,257 | ||||||
| Other non-current liabilities | 1,093 | 935 | ||||||
| Total non-current liabilities | 534,159 | 153,036 | ||||||
| Total liabilities | 602,766 | 228,955 | ||||||
| Shareholders' equity | ||||||||
| Total shareholders' equity | 268,784 | 476,009 | ||||||
| Total liabilities and shareholders' equity | $ | 871,550 | $ | 704,964 |
UNAUDITED CONSOLIDATED
STATEMENTS OF OPERATIONS
| Three months ended September 30, | ||||||||
| 2023 | 2022 | |||||||
| (in thousands, except share and per share amounts) | ||||||||
| Total revenues | $ | 1,407 | $ | 1,449 | ||||
| Operating expenses: | ||||||||
| Cost of contract manufacturing revenues | (1,006 | ) | (861 | ) | ||||
| Research and development expenses | (65,400 | ) | (48,068 | ) | ||||
| Selling, general and administrative expenses | (18,074 | ) | (13,324 | ) | ||||
| Total operating expenses | (84,480 | ) | (62,253 | ) | ||||
| Other income | 1,424 | 1,485 | ||||||
| Other expense | (228 | ) | (199 | ) | ||||
| Loss from operations | (81,877 | ) | (59,518 | ) | ||||
| Non-operating items, net | (7,763 | ) | 11,332 | |||||
| Loss before income tax (expense) / benefit | $ | (89,640 | ) | $ | (48,186 | ) | ||
| Income tax (expense) / benefit | 69 | 329 | ||||||
| Net loss | $ | (89,571 | ) | $ | (47,857 | ) | ||
| Basic and diluted net loss per ordinary share | $ | (1.88 | ) | $ | (1.02 | ) | ||
| Weighted average shares used in computing basic and diluted net loss per ordinary share | 47,770,101 | 46,772,430 |