~ Announced FDA clearance of Investigational New Drug (IND) application for AMT-260 in refractory mesial temporal lobe epilepsy ~
~ On track to provide clinical update from
~ uniQure and CSL win 2023
~ Announced strategic reorganization to focus on advancing multiple clinical-stage programs and expect to deliver
~ Strong cash position of approximately
“In the third quarter we made significant progress towards our goal of advancing multiple clinical-stage programs and focusing resources to generate value from these important investments,” stated
“As we prepare for 2024, we remain laser focused on execution across our portfolio of gene therapy product candidates with the goal of achieving clinical proof-of-concept as rapidly as possible. We also remain committed to carefully managing costs, prudently allocating capital, and thoughtfully evaluating strategies that can enhance value for shareholders in an increasingly challenging market environment.”
Recent Updates
- Advancing AMT-130 for the treatment of Huntington’s disease
- Later in the fourth quarter of 2023, the Company plans to provide a clinical update from the
U.S. and European Phase I/II studies of AMT-130, including 18- and 30-month follow-up data from the treated patients in the high- and low-doseU.S. cohorts, respectively. The data update will include safety and tolerability, biomarker, imaging and functional data across both high and low-dose cohorts. - A total of 33 patients have now been treated with AMT-130 across two dose cohorts in the
U.S. and EU clinical trials. The Company recently initiated patient screening for a third cohort in up to 12 patients to further investigate both doses in combination with perioperative immunosuppression, with a focus on evaluating near-term safety and tolerability. - In the first quarter of 2024, the Company plans to request regulatory interactions with the
Food and Drug Administration (FDA) to discuss theU.S. and EU data and potential late-stage development pathways for AMT-130.
- Later in the fourth quarter of 2023, the Company plans to provide a clinical update from the
- Advancing additional programs into the clinic
- AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (rMTLE) – In the third quarter of 2023, the Company announced the clearance of an investigational new drug (IND) for the Phase I/IIa clinical study of AMT-260. Screening and patient enrollment is expected to begin in the fourth quarter of 2023 with first patient dosing planned to occur in the first quarter 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 AMT-162 for the treatment of superoxide dismutase 1 (SOD1) ALS, a rare, genetic form of ALS. The Company expects to initiate patient screening in the fourth quarter of 2023 with first patient dosing planned to occur in the first quarter of 2024. - AMT-191 for the treatment of Fabry disease – The Company expects to submit an IND in the fourth quarter of 2023 and to begin patient dosing in 2024.
- AMT-260 for the treatment of refractory mesial temporal lobe epilepsy (rMTLE) – In the third quarter of 2023, the Company announced the clearance of an investigational new drug (IND) for the Phase I/IIa clinical study of AMT-260. Screening and patient enrollment is expected to begin in the fourth quarter of 2023 with first patient dosing planned to occur in the first quarter of 2024.
- Supporting the commercial launch of HEMGENIX® in the
United States andEuropean Union - In
October 2023 , uniQure andCSL Behring LLC (“CSL”) announced that the companies received the 2023Prix Galien USA Award in the category of Best Product for Rare/Orphan Diseases for HEMGENIX®. HEMGENIX® was one of several product nominations in this category. Created in 1970, the Prix Galien Awards recognizes outstanding innovation and scientific advancement and is regarded as the equivalent of the Nobel Prize for the life science industry. - In
July 2023 , uniQure received a$100 million milestone payment from CSL associated with the first commercial sale of HEMGENIX® inthe United States .
- In
- Implementing strategic reorganization focused on advancing multiple clinical-stage programs, achieving cost savings, and extending cash runway
- In
October 2023 , the Company announced a strategic reorganization plan which included a reduction of 28% of the workforce not related to manufacturing HEMGENIX®, the discontinuation of more than half of the research and technology projects, and a focus on executing across four clinical-stage programs. The Company expects a total cost savings of approximately$180 million over the next three years, which is expected to extend cash runway into the second quarter of 2027. - As part of the strategic reorganization and the significant reduction in research activities,
Ricardo Dolmetsch , Ph.D., the Company’s former chief scientific officer, departed the Company and will remain as a scientific consultant through the end of the year.Richard Porter , Ph.D., assumed responsibilities for research, as well as non-clinical and vector development in his new role as Chief Business and Scientific Officer.Dr. Porter has more than 25 years of neuroscience leadership in the biopharma industry and joined uniQure inJune 2021 through the acquisition of uniQure France SAS (formerly, Corlieve Therapeutics), where he was founder and chief executive officer.
- In
Upcoming Investor Events
- Stifel 2023 Healthcare Conference,
November 14, 2023 –New York, NY
Financial Highlights
Cash position: As of
Revenues: Revenues 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 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. These forward-looking statements include, but are not limited to, the Company’s statements about its cash runway and its ability to fund operations into 2027, the Company’s cost savings related to the recently-announced strategic organization, whether there will be continued progress on the commercialization of HEMGENIX®, the Company’s plans to announce follow up data from 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 | ||||||||
2023 | 2022 | |||||||
(in thousands, except share and per share amounts) | ||||||||
Current assets | ||||||||
Cash and cash equivalents | $ | 229,484 | $ | 228,012 | ||||
Current investment securities | 429,428 | 124,831 | ||||||
Accounts receivable and contract asset | 1,644 | 102,376 | ||||||
Inventories | 15,654 | 6,924 | ||||||
Prepaid expenses | 14,884 | 11,817 | ||||||
Other current assets and receivables | 2,532 | 2,814 | ||||||
Total current assets | 693,626 | 476,774 | ||||||
Non-current assets | ||||||||
Property, plant and equipment, net | 45,946 | 50,532 | ||||||
Non-current investment securities | — | 39,984 | ||||||
Operating lease right-of-use assets | 30,360 | 32,726 | ||||||
Intangible assets, net | 57,976 | 58,778 | ||||||
25,273 | 25,581 | |||||||
Deferred tax assets, net | 12,351 | 14,528 | ||||||
Other non-current assets | 6,018 | 6,061 | ||||||
Total non-current assets | 177,924 | 228,190 | ||||||
Total assets | $ | 871,550 | $ | 704,964 | ||||
Current liabilities | ||||||||
Accounts payable | $ | 5,584 | $ | 10,984 | ||||
Accrued expenses and other current liabilities | 28,427 | 30,571 | ||||||
Current portion of contingent consideration | 26,708 | 25,982 | ||||||
Current portion of operating lease liabilities | 7,888 | 8,382 | ||||||
Total current liabilities | 68,607 | 75,919 | ||||||
Non-current liabilities | ||||||||
Long-term debt | 101,431 | 102,791 | ||||||
Liability from royalty financing agreement | 383,711 | — | ||||||
Operating lease liabilities, net of current portion | 28,977 | 31,719 | ||||||
Contingent consideration, net of current portion | 14,030 | 9,334 | ||||||
Deferred tax liability, net | 4,917 | 8,257 | ||||||
Other non-current liabilities | 1,093 | 935 | ||||||
Total non-current liabilities | 534,159 | 153,036 | ||||||
Total liabilities | 602,766 | 228,955 | ||||||
Shareholders' equity | ||||||||
Total shareholders' equity | 268,784 | 476,009 | ||||||
Total liabilities and shareholders' equity | $ | 871,550 | $ | 704,964 |
uniQure N.V. UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS | ||||||||
Three months ended | ||||||||
2023 | 2022 | |||||||
(in thousands, except share and per share amounts) | ||||||||
Total revenues | $ | 1,407 | $ | 1,449 | ||||
Operating expenses: | ||||||||
Cost of contract manufacturing revenues | (1,006 | ) | (861 | ) | ||||
Research and development expenses | (65,400 | ) | (48,068 | ) | ||||
Selling, general and administrative expenses | (18,074 | ) | (13,324 | ) | ||||
Total operating expenses | (84,480 | ) | (62,253 | ) | ||||
Other income | 1,424 | 1,485 | ||||||
Other expense | (228 | ) | (199 | ) | ||||
Loss from operations | (81,877 | ) | (59,518 | ) | ||||
Non-operating items, net | (7,763 | ) | 11,332 | |||||
Loss before income tax (expense) / benefit | $ | (89,640 | ) | $ | (48,186 | ) | ||
Income tax (expense) / benefit | 69 | 329 | ||||||
Net loss | $ | (89,571 | ) | $ | (47,857 | ) | ||
Basic and diluted net loss per ordinary share | $ | (1.88 | ) | $ | (1.02 | ) | ||
Weighted average shares used in computing basic and diluted net loss per ordinary share | 47,770,101 | 46,772,430 |
Source: uniQure Inc.
2023 GlobeNewswire, Inc., source