uniQure Announces First Quarter 2023 Financial Results and Highlights Recent Company Progress
~ Clinical update from
~ 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 ~
“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
Recent Updates
- Commercial launch of HEMGENIX® (etranacogene dezaparvovec-drbl) for the treatment of hemophilia B in the
United States andEuropean Union - In
February 2023 , theEuropean Commission granted conditional marketing approval for HEMGENIX®, the first and only one-time gene therapy for the treatment of adults 18 years of age and older living with severe and moderately severe hemophilia B. This follows theNovember 2022 U.S. marketing approval of HEMGENIX® in adults living with hemophilia B. CSL Behring licensed the exclusive global rights to HEMGENIX® from uniQure in May 2021 and is solely responsible for its commercialization. Following the first commercial sale of HEMGENIX® inthe United States , uniQure is entitled to receive a$100.0 million payment fromCSL Behring . The Company is also eligible to receive a$75.0 million payment fromCSL Behring following the first product sale in one of five major European countries if achieved prior toJuly 2, 2023 .
- In
- Advancing AMT-130 for the treatment of Huntington’s disease
- The Company plans to announce one to two-years of follow up data from its 26 patient, randomized, controlled and double-blinded
U.S. Phase I/II clinical study in the second quarter of 2023. The data update is expected to include safety and tolerability, biomarker, imaging and functional data across both high and low dose cohorts. - Three of the six control patients in the higher-dose cohort have now crossed over to treatment. Two of these three patients have received the higher dose of AMT-130. Control patients in the higher-dose cohort have the option to cross over to treatment if they meet the study’s eligibility criteria after unblinding. The company expects to complete any additional crossover procedures by the end of the second quarter of 2023.
- All six patients have been enrolled in the lower-dose cohort of the European, open-label Phase I/II clinical trial of AMT-130. The Company continues to enroll the second, higher-dose cohort of nine patients. Patient enrollment in the higher-dose cohort is expected to complete in mid- 2023 with one-year of clinical data from the lower-dose cohort expected in the second half of 2023.
- A third cohort of patients is expected to begin enrollment in the
U.S. in the second half of 2023 to explore the feasibility of certain surgical adaptations aimed at enhancing procedure efficiency.
- The Company plans to announce one to two-years of follow up data from its 26 patient, randomized, controlled and double-blinded
- Expanding the Pipeline and Progress Towards Investigational New Drug (IND) Applications
American Society of Gene andCell Therapy (ASGCT) 2023 – The company will have a major presence at the annual ASGCT meeting being held inLos Angeles, CA fromMay 16-20, 2023 . Twelve abstracts have been accepted for presentation, including two oral presentations, that will feature innovation in expanding the Company’s research pipeline and advancements in AAV gene-therapy manufacturing.- AMT-162 for the treatment of SOD1 amyotrophic lateral sclerosis (ALS) - In
January 2023 , the Company entered into a global licensing agreement with Apic Bio for ABP-102, now AMT-162 for the treatment of superoxide dismutase 1 (SOD1) amyotrophic lateral sclerosis (ALS), a rare, genetic form of ALS. The Company expects to initiate a Phase I/II clinical study of AMT-162 in the second half of 2023. - AMT-260 for the treatment of refractory temporal lobe epilepsy (rTLE) – In the third quarter of 2022, the Company initiated a GLP toxicology study of AMT-260 in non-human primates. The Company expects to submit an investigational new drug (IND) application and initiate a Phase I/II clinical study of AMT-260 in the second half of 2023.
- AMT-191 for the treatment of Fabry disease – In the third quarter of 2022, the Company initiated a GLP toxicology study of AMT-191 in non-human primates which is expected to support an IND submission in 2023.
- Strong cash position to advance the Company’s programs
- As of
March 31, 2023 , the Company had cash and cash equivalents and investment securities of$315.3 million . The Company expects cash and cash equivalents and investment securities will fund operations into 2025 assuming the achievement of$100.0 million of first commercial sale milestone inthe United States , and into the first half of 2025 if the$75.0 million first commercial sale milestone for HEMGENIX® in any of the five contractually defined European countries is achieved prior toJuly 2, 2023 .
- As of
Financial Highlights
Cash position: As of
Revenues: Revenue for the three months ended
R&D expenses: Research and development expenses were
SG&A expenses: Selling, general and administrative expenses were
Other non-operating items, net:
Other non-operating items net was an expense of
Net loss:
The net loss for the three months ended
About HEMGENIX®
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 of
About uniQure
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
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
uniQure Contacts:
FOR INVESTORS: | FOR MEDIA: | |
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 |
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2023 | 2022 | |||||
(in thousands, except share and per share amounts) | ||||||
Current assets | ||||||
Cash and cash equivalents | $ | 153,851 | $ | 228,012 | ||
Current investment securities | 161,487 | 124,831 | ||||
Accounts receivable and contract asset | 104,793 | 102,376 | ||||
Inventories | 7,477 | 6,924 | ||||
Prepaid expenses | 12,567 | 11,817 | ||||
Other current assets and receivables | 2,468 | 2,814 | ||||
Total current assets | 442,643 | 476,774 | ||||
Non-current assets | ||||||
Property, plant and equipment, net | 50,072 | 50,532 | ||||
Non-current investment securities | - | 39,984 | ||||
Operating lease right-of-use assets | 32,135 | 32,726 | ||||
Intangible assets, net | 59,704 | 58,778 | ||||
25,998 | 25,581 | |||||
Deferred tax assets, net | 14,331 | 14,528 | ||||
Other non-current assets | 6,121 | 6,061 | ||||
Total non-current assets | 188,361 | 228,190 | ||||
Total assets | $ | 631,004 | $ | 704,964 | ||
Current liabilities | ||||||
Accounts payable | $ | 8,885 | $ | 10,984 | ||
Accrued expenses and other current liabilities | $ | 22,529 | $ | 30,571 | ||
Current portion of contingent consideration | 27,253 | 25,982 | ||||
Current portion of operating lease liabilities | 7,669 | 8,382 | ||||
Total current liabilities | 66,336 | 75,919 | ||||
Non-current liabilities | ||||||
Long-term debt | 103,253 | 102,791 | ||||
Operating lease liabilities, net of current portion | 31,075 | 31,719 | ||||
Contingent consideration, net of current portion | 9,641 | 9,334 | ||||
Deferred tax liability, net | 6,970 | 8,257 | ||||
Other non-current liabilities | 958 | 935 | ||||
Total non-current liabilities | 151,897 | 153,036 | ||||
Total liabilities | 218,233 | 228,955 | ||||
Shareholders' equity | ||||||
Total shareholders' equity | 412,771 | 476,009 | ||||
Total liabilities and shareholders' equity | $ | 631,004 | $ | 704,964 | ||
uniQure N.V. UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS |
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Three months ended |
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2023 | 2022 | ||||||
(in thousands, except share and per share amounts) | |||||||
Total revenues | $ | 5,325 | $ | 1,792 | |||
Operating expenses: | |||||||
Cost of contract manufacturing revenues | (2,435 | ) | - | ||||
Research and development expenses | (60,809 | ) | (45,003 | ) | |||
Selling, general and administrative expenses | (17,848 | ) | (10,987 | ) | |||
Total operating expenses | (81,092 | ) | (55,990 | ) | |||
Other income | 1,811 | 311 | |||||
Other expense | (216 | ) | (193 | ) | |||
Loss from operations | (74,172 | ) | (54,080 | ) | |||
Non-operating items, net | (4,262 | ) | 6,786 | ||||
Loss before income tax benefit / (expense) | $ | (78,434 | ) | $ | (47,294 | ) | |
Income tax benefit / (expense) | 1,207 | 616 |
|||||
Net loss | $ | (77,227 | ) | $ | (46,678 | ) | |
Basic and diluted net loss per ordinary share | $ | (1.63 | ) | $ | (1.00 | ) | |
Weighted average shares used in computing basic and diluted net loss per ordinary share | 47,436,335 | 46,599,114 | |||||