uniQure Announces 2023 Financial Results and Highlights Recent Company Progress
~ Presented promising clinical update from
~ Announced FDA clearance of two Investigational New Drug (IND) applications; Initiation of Phase I/II clinical trials in mesial temporal lobe epilepsy (mTLE) and Fabry disease, in addition to SOD1-ALS, are expected in the first half of 2024 ~
~ Announced strategic reorganization to advance multiple clinical-stage programs and deliver
~ Cash position of approximately
“We are pleased with the progress made across the company in 2023 and are now laser-focused on execution across multiple clinical programs,” stated
“While we enter 2024 with a strong balance sheet, we remain disciplined on the prudent and efficient allocation of our capital,” he continued. “Any decision to advance AMT-130 into late-stage development will necessitate a clear and timely approval pathway and financial feasibility, including through a partnership which we would secure before beginning any Phase III trial.”
Recent Updates
- Advancing AMT-130 for the treatment of Huntington’s disease
- In
December 2023 , the Company announced updated clinical data from the ongoingU.S. and European Phase I/II studies of AMT-130 for the treatment of early-stage Huntington’s disease.
- Patients treated with AMT-130 continued to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to a non-concurrent criteria-matched natural history of the disease. When compared to the expected rate of decline from an inclusion criteria-matched natural history data set, AMT-130 showed favorable trends in composite Unified Huntington’s Disease Rating Scale (cUHDRS), Total Functional Capacity (TFC) and Total Motor Score (TMS) key clinical rating scales designed to assess disease progression.
- Further declines in neurofilament light chain (NfL), an exploratory biomarker for the measurement of neuronal degradation and disease progression were observed among patients treated with AMT-130. Mean NfL in the cerebrospinal fluid (CSL) for low-dose patients remained below baseline at 30 months of follow-up and high-dose patients were near baseline at 18 months.
- AMT-130 continued to be generally well-tolerated with a manageable safety profile across both doses.
- Patients treated with AMT-130 continued to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to a non-concurrent criteria-matched natural history of the disease. When compared to the expected rate of decline from an inclusion criteria-matched natural history data set, AMT-130 showed favorable trends in composite Unified Huntington’s Disease Rating Scale (cUHDRS), Total Functional Capacity (TFC) and Total Motor Score (TMS) key clinical rating scales designed to assess disease progression.
- In the fourth quarter of 2023, the Company initiated patient dosing in a third cohort of up to 12 patients to further investigate both doses of AMT-130 in combination with perioperative immunosuppression, with a focus on evaluating near-term safety and tolerability. Enrollment in this cohort is expected to be completed in the second half of 2024.
- In the second quarter of 2024, the Company expects to initiate regulatory interactions with the
U.S. Food and Drug Administration (FDA) to discuss data from the ongoing Phase I/II studies and potential strategies for the further development of AMT-130. By the end of 2024, the Company expects to have greater clarity regarding a potential approval pathway for AMT-130. - In mid-2024, the Company expects to provide an interim update from the ongoing Phase I/II studies of AMT-130, including up to 24- and 36-month follow-up data from all treated patients in the
U.S. and European trials.
- In
- Advancing additional programs into the clinic
- AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (rMTLE) – In
September 2023 , the Company announced the clearance of an IND for the Phase I/IIa clinical study of AMT-260. Site initiation is underway, and patient enrollment is expected to begin in the first half of 2024. - 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 known as AMT-162, for the treatment of superoxide dismutase 1 (SOD1) ALS, a rare, genetic form of ALS. Patient enrollment in a Phase I/II clinical trial is expected to begin in the first half of 2024. - AMT-191 for the treatment of Fabry disease – In
November 2023 , the Company announced the clearance of an IND for the Phase I/IIa clinical study of AMT-191. Patient enrollment is expected to begin in the first half of 2024.
- AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (rMTLE) – In
- Generating value from the commercial launch of HEMGENIX®
- The Company continues to provide commercial supply of etranacogene dezaparvovec (HEMGENIX®) to its partner
CSL Behring and manufactures the product at its cGMP facility inLexington, MA. The Company is one of a select number of gene therapy companies with a qualified facility producing routine commercial manufacturing for the market.
- The Company continues to provide commercial supply of etranacogene dezaparvovec (HEMGENIX®) to its partner
Upcoming Investor Events
- 44th Annual
Cowen Health Care Conference ,March 5, 2024 –Boston, MA Leerink Global Biopharma Conference 2024,March 12, 2024 –Miami, FL - UBS Virtual CNS Day,
March 18, 2024
Financial Highlights
Cash position: As of
Revenues: Revenue for the year ended
Cost of contract manufacturing revenues: Cost of contract manufacturing revenues were
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 year ended
About uniQure
uniQure’s 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
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. Examples of these forward-looking statements include, but are not limited to, statements concerning the Company’s cash runway and its ability to fund its operations into the second quarter of 2027; the Company’s expected cost savings related to its strategic reorganization; the Company’s plans to announce additional follow up data from the ongoing
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 |
||||||
2023 | 2022 | |||||
(in thousands, except share and per share amounts) | ||||||
Current assets | ||||||
Cash and cash equivalents | $ | 241,360 | $ | 228,012 | ||
Current investment securities | 376,532 | 124,831 | ||||
Accounts receivable and contract asset | 4,193 | 102,376 | ||||
Inventories, net | 12,024 | 6,924 | ||||
Prepaid expenses | 15,089 | 11,817 | ||||
Other current assets and receivables | 2,655 | 2,814 | ||||
Total current assets | 651,853 | 476,774 | ||||
Non-current assets | ||||||
Property, plant and equipment, net | 46,548 | 50,532 | ||||
Non-current investment securities | — | 39,984 | ||||
Operating lease right-of-use assets | 28,789 | 32,726 | ||||
Intangible assets, net | 60,481 | 58,778 | ||||
26,379 | 25,581 | |||||
Deferred tax assets, net | 12,276 | 14,528 | ||||
Other non-current assets | 5,363 | 6,061 | ||||
Total non-current assets | 179,836 | 228,190 | ||||
Total assets | $ | 831,689 | $ | 704,964 | ||
Current liabilities | ||||||
Accounts payable | $ | 6,586 | $ | 10,984 | ||
Accrued expenses and other current liabilities | $ | 30,534 | $ | 30,571 | ||
Current portion of contingent consideration | 28,211 | 25,982 | ||||
Current portion of operating lease liabilities | 8,344 | 8,382 | ||||
Total current liabilities | 73,675 | 75,919 | ||||
Non-current liabilities | ||||||
Long-term debt | 101,749 | 102,791 | ||||
Liability from royalty financing agreement | 394,241 | — | ||||
Operating lease liabilities, net of current portion | 28,316 | 31,719 | ||||
Contingent consideration, net of current portion | 14,795 | 9,334 | ||||
Deferred tax liability, net | 7,543 | 8,257 | ||||
Other non-current liabilities | 3,700 | 935 | ||||
Total non-current liabilities | 550,344 | 153,036 | ||||
Total liabilities | 624,019 | 228,955 | ||||
Shareholders' equity | ||||||
Total shareholders' equity | 207,670 | 476,009 | ||||
Total liabilities and shareholders' equity | $ | 831,689 | $ | 704,964 | ||
Balancing check | - | - |
uniQure N.V. UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS |
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Years ended |
|||||||||||
2023 | 2022 | 2021 | |||||||||
(in thousands, except share and per share amounts) | |||||||||||
License revenues | $ | 2,758 | $ | 100,000 | $ | 517,400 | |||||
Contract manufacturing revenues | 10,835 | 1,717 | - | ||||||||
Collaboration revenues | 2,250 | 4,766 | 6,602 | ||||||||
Total revenues | 15,843 | 106,483 | 524,002 | ||||||||
Operating expenses: | |||||||||||
Cost of license revenues | (65 | ) | (1,254 | ) | (24,976 | ) | |||||
Cost of contract manufacturing revenues | (13,563 | ) | (2,089 | ) | - | ||||||
Research and development expenses | (214,864 | ) | (197,591 | ) | (143,548 | ) | |||||
Selling, general and administrative expenses | (74,591 | ) | (55,059 | ) | (56,290 | ) | |||||
Total operating expenses | (303,083 | ) | (255,993 | ) | (224,814 | ) | |||||
Other income | 6,059 | 7,171 | 12,306 | ||||||||
Other expense | (1,690 | ) | (820 | ) | (876 | ) | |||||
(Loss) / income from operations | (282,871 | ) | (143,159 | ) | 310,618 | ||||||
Non-operating items, net | (23,686 | ) | 14,900 | 22,188 | |||||||
(Loss) / income before income tax (expense) / benefit | $ | (306,557 | ) | $ | (128,259 | ) | $ | 332,806 | |||
Income tax (expense) / benefit | (1,921 | ) | 1,470 | (3,217 | ) | ||||||
Net (loss) / income | $ | (308,478 | ) | $ | (126,789 | ) | $ | 329,589 | |||
Earnings per ordinary share - basic | |||||||||||
Basic net (loss) / income per ordinary share | $ | (6.47 | ) | $ | (2.71 | ) | $ | 7.17 | |||
Earnings per ordinary share - diluted | |||||||||||
Diluted net (loss) / income per ordinary share | $ | (6.47 | ) | $ | (2.71 | ) | $ | 7.04 | |||
Weighted average shares - basic | 47,670,986 | 46,735,045 | 45,986,467 | ||||||||
Weighted average shares - diluted | 47,670,986 | 46,735,045 | 46,840,972 | ||||||||
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