Full Press Release Details
Second Quarter 2022 Financial Results
Recent Company Progress
~ Announced 12-month
data on the lower-dose cohort of AMT-130 in Huntington's disease showed the investigative gene therapy was generally well tolerated
at this dose with a mean reduction of 53.8% of mutant Huntingtin protein (mHTT) observed in cerebral spinal fluid (CSF) ~
~ Announced postponement
of AMT-130 higher-dose procedures due to recent suspected unexpected severe adverse reactions at this dose; Lower-dose procedures are
expected on anticipated data readouts in 2023 ~
Advancing regulatory reviews of the U.S. and European marketing applications for etranacogene
dezaparvovec in hemophilia B ~
conference call and webcast today at 8:30 a.m. ET ~
MA and Amsterdam, the Netherlands, August 8, 2022 - 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 second quarter of 2022 and highlighted recent progress across its business.
continued to make strong progress across all of our programs highlighted by encouraging data from the lower-dose cohort in our Phase
I/II study of AMT-130 and the advancement of the U.S. and European regulatory reviews for the marketing applications for etranacogene
dezaparvovec in hemophilia B," stated Matt Kapusta, chief executive officer at uniQure. "Our
manufacturing, CMC and Quality teams are working tirelessly to produce commercial launch supplies and also to ensure successful pre-approval
site inspections, which are expected to be completed in the third quarter of this year. We also are very pleased to have recently received
notification that GMP certification will be provided to our Lexington manufacturing facility by the European authorities."
July, we reported to the health authorities suspected unexpected severe adverse reactions in three of
the 14 patients treated with the higher dose of AMT-130,"
he continued. "While these patients have fully or substantially recovered, and no clear root cause has yet been identified, we
believe it's prudent to temporarily delay additional higher-dose procedures until we put additional monitoring and treatment plans
in place and complete our safety review early in the fourth quarter of 2022. The delay is not expected to impact any future lower-dose
procedures, as no serious adverse events related to AMT-130 have been reported in any of the additional 12 patients treated at the lower
dose, and no impact is expected on the timing of previously guided data read-outs in 2023, including the presentation of one to
two-year follow-up on both low and high-dose cohorts in the second quarter of 2023."
| o | At this time, the DSMB does not view these findings as a dose-limiting toxicity. The DSMB has recommended temporarily delaying higher-dose enrollment pending a safety review, which is expected to take place early in the fourth quarter of 2022. The DSMB is permitting continued enrollment at the lower-dose of AMT-130, and the Company expects to begin crossing over control patients from the U.S. study at this dose in the third quarter of 2022. | |
| o | No impact is expected on the timing of previously guided data read-outs in 2023. In the U.S. Phase I/II study, all 26 patients in the first two dose cohorts have been enrolled, and the Company continues to expect to present one to two-year follow up data in the second quarter of 2023. In the European Phase Ib/II study, the six-patient lower-dose cohort is fully enrolled and the Company continues to expect to present one-year follow-up data in 2023. Four of nine patients in the European study have been enrolled in the higher-dose cohort. To date, a total of 36 patients have been enrolled across the two clinical trials including 10 control patients and 26 patients treated with AMT-130, of which 14 patients received the higher-dose and 12 patients received the lower-dose. |
| o | AMT-260 for the treatment of refractory temporal lobe epilepsy (rTLE) - In July 2022, the Company initiated an IND-enabling GLP toxicology study in non-human primates to support an IND submission expected in 2023. | |
| o | AMT-191 for the treatment of Fabry disease - The Company expects to initiate a GLP toxicology study of AMT-191 in non-human primates in the second half of 2022, which is expected to support an IND submission in 2023. |
Financial Highlights
As of June 30, 2022, the Company held cash and cash equivalents of $500.5 million, compared to $556.3 million as of December 31,
Collaboration revenue for the three months ended June 30, 2022, was $0.5 million, compared to
collaboration revenue of $1.5 million for the same period in 2021. Revenue for 2021 during this period also included license revenue
of $462.4 million from selling the exclusive global rights to etranacogene dezaparvovec to CSL Behring and nil in the same period 2022.
revenues: Cost of contract revenues for the three months ended June 30, 2022 was nil compared to $23.2 million for the same
period in 2021. Costs incurred in 2021 are associated with recognized license revenue.
manufacturing: Cost of contract manufacturing for the three months ended June 30, 2022, was $0.8 million compared to nil for the
same period in 2021. Costs incurred in 2022 related to the manufacture of etranacogene dezaparvovec for CSL Behring.
Research and development expenses were $46.2 million for the three months ended June 30, 2022, compared to $32.8 million during the
same period in 2021. The increase was related to the preclinical development of temporal lobe epilepsy (AMT-260) advancing the clinical
development of the Company's Huntington's disease gene therapy program, and recruitment of personnel to support the development
of product candidates.
Selling, general and administrative expenses were $12.5 million for the three months ended June 30, 2022, compared to $17.3 million
during the same period in 2021. The reduction was primarily related to incurring financial advisory fees to close the CSL Behring transaction
in 2021 with no such fees in 2022.
income, net was income of $16.7 million for the three months ended June 30, 2022, compared to other non-operating income, net of $4.7
million for the same period in 2021. The increase in other non-operating income, net was primarily related to an increase in net foreign
currency gains of $12.8 million ($19.4 million recorded in the current period compared to $6.6 million for the same period in 2021).
the three months ended June 30, 2022, was $39.1 million, or $0.84 basic and diluted loss per ordinary share, compared to $399.5 million
net income for the same period in 2021, or $8.68 basic net income per ordinary share and $8.51 diluted net income per ordinary share.
Call and Webcast Information
will host an investor conference call and webcast today, Monday, August 8, 2022, at 8:30 a.m. ET. The conference call may be accessed
via pre-registering here. Participants will then receive a dial-in number and personal PIN. Research analysts who wish to ask
a question should do so through the pre-registered conference call number. If you are joining the conference call, please dial-in 15
minutes before the start time. The webcast of the conference call also may be accessed here, or through the Investors &
Newsroom section of the uniQure website. Following the live webcast, a replay of the call will be available on our website for several
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, refractory temporal lobe epilepsy, Fabry disease, and other 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," "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, whether our announced delay of the AMT-130 high-dose procedures will be temporary,
whether the delay will impact any future low-dose procedures, whether any control patients in the low-dose cohort of the AMT-130 clinical
trial study will receive treatment in the third quarter of 2022 or ever, whether we will announce any data readouts for our AMT-130 clinical
trial in 2023, whether our pre-approval site inspections for AMT-061 will be completed in the third quarter of this year or will be successful,
whether we will ultimately receive GMP certification for our Lexington manufacturing facility by the European authorities or other regulatory
authorities, whether we will submit an IND in for AMT-260 for the treatment of refractory temporal lobe epilepsy (rTLE) in 2023 or ever,
whether we will initiate a GLP toxicology study of AMT-191 for Fabry disease in non-human primates in the second half of 2022 or submit
an IND in 2023, and whether our cash and cash equivalents will fund operations into the first half of 2025. 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 impact of the postponement in our clinical trial for Huntington's disease and the pending safety review,
the ongoing COVID-19 pandemic 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 25, 2022. 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.
| 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 |
UNAUDITED CONSOLIDATED
| June 30, 2022 | December 31, 2021 | |||||||
| (in thousands, except share and per share amounts) | ||||||||
| Current assets | ||||||||
| Cash and cash equivalents | $ | 500,524 | $ | 556,256 | ||||
| Accounts receivable and contract asset | 3,119 | 58,768 | ||||||
| Inventories | 2,949 | - | ||||||
| Prepaid expenses | 15,126 | 10,540 | ||||||
| Other current assets and receivables | 1,581 | 2,675 | ||||||
| Total current assets | 523,299 | 628,239 | ||||||
| Non-current assets | ||||||||
| Property, plant and equipment, net | 45,984 | 43,505 | ||||||
| Operating lease right-of-use assets | 28,482 | 25,573 | ||||||
| Intangible assets, net | 57,450 | 62,686 | ||||||
| Goodwill | 24,976 | 27,633 | ||||||
| Deferred tax assets, net | 15,046 | 15,647 | ||||||
| Other non-current assets | 5,974 | 5,897 | ||||||
| Total non-current assets | 177,912 | 180,941 | ||||||
| Total assets | $ | 701,211 | $ | 809,180 | ||||
| Current liabilities | ||||||||
| Accounts payable | $ | 10,028 | $ | 2,502 | ||||
| Accrued expenses and other current liabilities | 23,047 | 28,487 | ||||||
| Current portion of contingent consideration | 8,681 | - | ||||||
| Current portion of operating lease liabilities | 6,505 | 5,774 | ||||||
| Total current liabilities | 48,261 | 36,763 | ||||||
| Non-current liabilities | ||||||||
| Long-term debt | 101,890 | 100,963 | ||||||
| Contingent consideration, net of current portion | 20,405 | 29,542 | ||||||
| Operating lease liabilities, net of current portion | 30,721 | 28,987 | ||||||
| Deferred tax liability, net | 9,953 | 12,913 | ||||||
| Other non-current liabilities | 3,493 | 4,236 | ||||||
| Total non-current liabilities | 166,462 | 176,641 | ||||||
| Total liabilities | 214,723 | 213,404 | ||||||
| Shareholders' equity | ||||||||
| Total shareholders' equity | 486,488 | 595,776 | ||||||
| Total liabilities and shareholders' equity | $ | 701,211 | $ | 809,180 |
UNAUDITED CONSOLIDATED
STATEMENTS OF OPERATIONS
| Three months ended June 30, | ||||||||
| 2022 | 2021 | |||||||
| (in thousands, except share and per share amounts) | ||||||||
| Total revenues | $ | 497 | $ | 463,868 | ||||
| Operating expenses: | ||||||||
| Cost of contract revenues | - | (23,178 | ) | |||||
| Cost of contract manufacturing | (832 | ) | - | |||||
| Research and development expenses | (46,192 | ) | (32,747 | ) | ||||
| Selling, general and administrative expenses | (12,491 | ) | (17,299 | ) | ||||
| Total operating expenses | (59,515 | ) | (73,224 | ) | ||||
| Other income | 3,186 | 7,590 | ||||||
| Other expense | (229 | ) | (226 | ) | ||||
| (Loss) / income from operations | (56,061 | ) | 398,008 | |||||
| Non-operating items, net | 16,682 | 4,718 | ||||||
| (Loss) / income before income tax benefit / (expense) | $ | (39,379 | ) | $ | 402,726 | |||
| Income tax benefit / (expense) | 318 | (3,258 | ) | |||||
| Net (loss) / income | $ | (39,061 | ) | $ | 399,468 | |||
| Earnings per ordinary share - basic | ||||||||
| Basic net (loss) / income per ordinary share | $ | (0.84 | ) | $ | 8.68 | |||
| Earnings per ordinary share - diluted | ||||||||
| Diluted net (loss) / income per ordinary share | $ | (0.84 | ) | $ | 8.51 | |||
| Weighted average shares - basic | 46,668,554 | 46,037,900 | ||||||
| Weighted average shares - diluted | 46,668,554 | 46,929,870 |