Full Press Release Details
uniQure Announces First Quarter 2023 Financial
and Highlights Recent Company Progress
~ Clinical update from U.S. Phase I/II trial
in Huntington's disease expected in second quarter 2023 ~
~ Continued progress towards initiating two
new Phase I/II clinical studies for AMT-260 in refractory temporal lobe epilepsy and AMT-162 in SOD1-ALS ~
Lexington, MA and Amsterdam, the Netherlands,
May 9, 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 first quarter of 2023 and highlighted recent progress
across its business.
"uniQure continued its momentum from a
historic 2022 into the first quarter of 2023 as we make progress across all of our corporate objectives," stated Matt Kapusta,
chief executive officer of uniQure. "Our priorities for 2023 include supplying CSL's launch of HEMGENIX ,
advancing the development of AMT-130 in Huntington's disease, and preparing for the initiation of two new clinical studies in SOD1-ALS
and refractory temporal lobe epilepsy. Later in this second quarter of 2023, we look forward to providing a clinical update from our
ongoing U.S. Phase I/II study of AMT-130, the first investigational AAV gene therapy in clinical development for Huntington's disease.
This update will help to further guide our clinical development of AMT-130 as we work diligently to bring a disease-modifying treatment
option to patients with this devastating neurodegenerative disease."
Financial Highlights
Cash position: As of March 31, 2023,
the Company held cash and cash equivalents and investment securities of $315.3 million, compared to $392.8 million as of December 31,
Revenues: Revenue for the three months
ended March 31, 2023 was $5.3 million, compared to $1.8 million in the same period in 2022. The increase is primarily a result of
contract manufacturing revenues of $4.9 million recognized in the current period related to contract manufacturing HEMGENIX
for CSL Behring partially offset by a decrease in collaboration revenues of $1.4 million.
R&D expenses: Research and development
expenses were $60.8 million for the three months ended March 31, 2023, compared to $45.0 million in the same period in 2022. The
increase was primarily related to the $10.0 million payment made to Apic Bio to acquire ABP-102, now AMT-162, the preclinical development
of the temporal lobe epilepsy program (AMT-260), an increase in personnel and contractor related expenses to support the growth of the
Company, and contractual payments owed to a licensor upon EMA approval of HEMGENIX .
SG&A expenses: Selling, general and
administrative expenses were $17.8 million for the three months ended March 31, 2023, compared to $11.0 million in the same period
in 2022. The increase was primarily related to an increase in personnel and contractor related expenses to support the growth of the Company
and an increase in professional fees incurred in the current period, primarily driven by an increase in professional fees related to the
Apic Bio transaction.
Other non-operating items, net:
Other non-operating items net was an expense of
$4.3 million for the three months ended March 31, 2023, compared to net income of $6.8 million for the same period in 2022. The decrease
in other non-operating items, net was primarily related to recognizing foreign currency losses, net of $2.4 million in the current period
compared to recognizing foreign currency gains, net of $8.6 million in the prior period.
The net loss for the three months ended March 31,
2023, was $77.2 million, or $1.63 basic and diluted loss per ordinary share, compared to $46.7 million net loss for the same period in
2022, or $1.00 basic and diluted loss per ordinary share.
HEMGENIX is a gene therapy that
reduces the rate of abnormal bleeding in eligible people with hemophilia B by enabling the body to continuously produce factor IX, the
deficient protein in hemophilia B. It uses AAV5, a non-infectious viral vector, called an adeno-associated virus (AAV). The AAV5 vector
carries the Padua gene variant of Factor IX (FIX-Padua) to the target cells in the liver, generating factor IX proteins that are 5x-8x
more active than normal. These genetic instructions remain in the target cells, but generally do not become a part of a person's
own DNA. Once delivered, the new genetic instructions allow the cellular machinery to produce stable levels of factor IX.
HEMGENIX is a registered trademark
uniQure is delivering on the promise of gene
therapy - single treatments with potentially curative results. The recent approvals of our gene therapy for hemophilia B -
an historic achievement based on more than a decade of research and clinical development - represents 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 platform to advance a pipeline of proprietary gene therapies for the treatment of patients with Huntington's
disease, refractory temporal lobe epilepsy, ALS, Fabry disease, and other severe 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," "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, whether there will be continued progress on the commercialization
of HEMGENIX , statements about whether a third cohort of patients in our AMT-130 clinical trial for Huntington's
Disease will begin enrollment in the U.S. in the second half of 2023, whether the Company will announce one to two-years of follow up
data from the U.S. Phase I/II clinical study of AMT-130 in the second quarter of 2023, whether that data will help to further guide our
ongoing clinical development of AMT-130, whether clinical data from the lower-dose cohort for AMT-130 is expected to be presented in the
second half of 2023, whether the Company will submit an investigational new drug application for Fabry disease in 2023, whether the company
will initiate a Phase I/II clinical study of AMT-162 for the treatment of ALS in the second half of 2023, and whether the Company will
submit an investigational new drug application or initiate a Phase I/II clinical study of AMT-260 for rTLE in the second half of 2023.
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 , 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, our Commercialization and License Agreement with CSL Behring, 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 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 May 9, 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.
| 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 |