uniQure Announces 2023 Financial Results and Highlights Recent Company Progress
- Promising clinical update for AMT-130 in Huntington’s disease with favorable trends in key clinical rating scales and biomarkers
- FDA clearance for IND applications for Phase I/II trials in mesial temporal lobe epilepsy, Fabry disease, and SOD1-ALS
- Strategic reorganization to deliver $180 million of cost savings over the next three years
- Cash position of approximately $618 million as of December 31, 2023, expected to fund operations into the second quarter of 2027
- Revenue of $15.8 million for the year ended December 31, 2023, with an increase in contract manufacturing revenues for HEMGENIX®
- Increase in R&D and SG&A expenses, with a net loss of $308.5 million for the year ended December 31, 2023
- Significant net loss of $308.5 million for the year ended December 31, 2023
- Revenue decline from $106.5 million in 2022 to $15.8 million in 2023
- Increase in R&D and SG&A expenses impacting the company's financials
Insights
The financial health of uniQure N.V. is indicated by its strong cash position, with approximately $618 million expected to sustain operations into Q2 2027. This liquidity is crucial for the company's strategic reorganization aimed at delivering $180 million in cost savings over three years, which is significant for a company of its size. The strategic reorganization suggests a focus on efficiency and could be a response to the increased R&D and SG&A expenses reported for 2023. The net loss increase year-over-year from $126.8 million to $308.5 million is concerning, yet it's partially mitigated by the robust cash reserves and the royalty agreement inflow. However, the decrease in revenues from $106.5 million to $15.8 million year-over-year is a red flag, as it shows a heavy reliance on milestone payments rather than steady revenue streams.
From a medical research perspective, the IND clearances and the initiation of Phase I/II clinical trials for multiple treatments including mTLE, SOD1-ALS and Fabry disease represent significant progress in uniQure's pipeline. The advancement of AMT-130 for Huntington’s disease is particularly noteworthy, with the treatment showing promising trends against key clinical rating scales. The neurofilament light chain (NfL) biomarker data suggest potential efficacy in slowing disease progression, which is a critical factor in evaluating the treatment's long-term potential. However, the therapeutic area of gene therapy is highly complex and competitive, with regulatory challenges and high costs of development. The expected regulatory interactions in 2024 will be pivotal in determining the future of AMT-130 and could have major implications for the company's valuation and strategic direction.
Analyzing the market implications, uniQure's strategic reorganization to streamline operations and the initiation of multiple clinical trials could position the company favorably in the gene therapy market. The company's commercial supply of HEMGENIX® to CSL Behring indicates a move towards generating recurring revenue, which is a positive signal to investors looking for sustainable business models. However, the gene therapy sector is highly volatile, with success heavily dependent on clinical outcomes and regulatory approvals. The market will closely watch the upcoming regulatory interactions concerning AMT-130, as clarity on the approval pathway could significantly impact investor sentiment and stock performance.
~ Presented promising clinical update from U.S. and European Phase I/II trials of AMT-130 in Huntington’s disease; Up to three years of follow-up data to be presented in mid-2024; Regulatory interactions and clarity on potential strategies for clinical development expected in 2024 ~
~ 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
LEXINGTON, Mass. and AMSTERDAM, Feb. 28, 2024 (GLOBE NEWSWIRE) -- 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 fourth quarter and full year 2023 and highlighted recent progress across its business.
“We are pleased with the progress made across the company in 2023 and are now laser-focused on execution across multiple clinical programs,” stated Matt Kapusta, chief executive officer of uniQure. “Our top priorities are to engage with regulatory authorities to clarify the approval pathway for AMT-130 in Huntington’s disease and to initiate patient enrollment across three additional clinical trials as expeditiously as possible.”
“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 ongoing U.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 December 2023, the Company announced updated clinical data from the ongoing U.S. and European Phase I/II studies of AMT-130 for the treatment of early-stage Huntington’s disease.
- 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 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.
- 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 in Lexington, 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 CSL Behring and manufactures the product at its cGMP facility in Lexington, MA. The Company is one of a select number of gene therapy companies with a qualified facility producing routine commercial manufacturing for the market.
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 December 31, 2023, the Company held cash and cash equivalents and investment securities of
Revenues: Revenue for the year ended December 31, 2023 was
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 December 31, 2023, was
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 Huntington's disease, refractory mesial temporal lobe epilepsy, amyotrophic lateral sclerosis (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. 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 U.S. and European Phase I/II clinical studies of AMT-130; the Company’s plans to initiate interactions with regulatory authorities regarding the further development of AMT-130 and the timing of regulatory clarity from such interactions; and the Company’s plans to initiate patient enrollment for AMT-260, AMT-162 and AMT-191 in the first half of 2024. The Company’s actual results could differ materially from those anticipated in these forward-looking statements for many reasons. These risks and uncertainties include, among others: risks associated with the clinical results and the development and timing of the Company’s programs; the Company’s interactions with regulatory authorities, which may affect the initiation, timing and progress of clinical trials and pathways to approval; the Company’s ability to continue to build and maintain the company infrastructure and personnel needed to achieve its goals; the Company’s effectiveness in managing current and future clinical trials and regulatory processes; the continued development and acceptance of gene therapies; the Company’s ability to demonstrate the therapeutic benefits of its gene therapy candidates in clinical trials; the Company’s ability to obtain, maintain and protect intellectual property; the Company’s ability to fund its operations and to raise additional capital as needed; and the impact of global economic uncertainty, rising inflation, rising interest rates or market disruptions on its business. These risks and uncertainties are more fully described under the heading "Risk Factors" in the Company’s periodic filings with the U.S. Securities & Exchange Commission (“SEC”), including its Annual Report on Form 10-K filed February 28, 2024 and in other filings that the Company makes with the SEC from time to time. 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 |
| uniQure N.V. UNAUDITED CONSOLIDATED BALANCE SHEETS | ||||||
| December 31, | December 31, | |||||
| 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 | ||||
| Goodwill | 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 | |||||||||||
| Years ended December 31, | |||||||||||
| 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 | ||||||||
FAQ
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