uniQure Announces Third Quarter 2022 Financial Results and Highlights Recent Company Progress
~ Patient enrollment at the higher dose of AMT-130 to resume in European Phase Ib/II study following Data Safety Monitoring Board recommendation ~
~
~ Virtual investor event focused on next pipeline program, AMT-260 investigational gene therapy for refractory temporal lobe epilepsy, to be held on
“We are pleased that following a comprehensive review of all available safety, biomarker and imaging data, the Data Safety Monitoring Board (DSMB) has recommended that we resume patient enrollment at the higher dose in the ongoing Phase Ib/II study evaluating AMT-130 in Huntington’s disease,” stated
“In the third quarter, the
Recent Updates
- Advancing etranacogene dezaparvovec for the treatment of hemophilia B
- In
May 2022 , the Company’s global commercialization partner,CSL Behring , announced that the biologic license application (BLA) for etranacogene dezaparvovec (AMT-061) was accepted by the FDA for priority review. This followed the EMA validation of the marketing application authorization (MAA) inMarch 2022 . In accordance with the Company’s commercialization and license agreement,CSL Behring is solely responsible for all regulatory activities, including any filings and agency interactions associated with etranacogene dezaparvovec and the companion diagnostic test for neutralizing antibodies to AAV5. - In
the United States , the BLA remains under Priority Review with etranacogene dezaparvovec having Breakthrough Therapy Designation. InJuly 2022 ,CSL Behring was notified by the Committee for Advanced Therapies (CAT) inEurope that they will be unable to complete their review in accordance with the accelerated assessment timetable and will switch to a standard review procedure. - In July and
August 2022 , respectively, the EMA and the FDA conducted comprehensive, multi-day, pre-approval inspections of the Company’sLexington manufacturing site. Following the inspection, EMA issued a GMP certificate related to the production of commercial supply of etranacogene dezaparvovec.
- In
- Advancing AMT-130 for the treatment of Huntington’s disease
- In
October 2022 , the DSMB reviewed all available safety, biomarker and imaging data from the ongoing Phase I/II clinical trials of AMT-130 and recommended resuming treatment at the higher dose. As previously announced inAugust 2022 , patient enrollment at the higher dose had been voluntarily paused following the reporting of suspected unexpected significant adverse reactions (SUSARs) in three patients shortly after they received the higher dose of AMT-130. The SUSAR events in all three patients have since fully resolved.- The DSMB recommended implementing additional risk mitigation procedures during the first two weeks after the surgical administration of AMT-130, including a seven-day, in-person post-surgical visit. The DSMB also made no change to the treatment protocol regarding the use of immunosuppression that will continue to be at the discretion of the treating physician.
- The Company plans to resume patient dosing in the open-label European clinical study as soon as possible and complete enrollment in the first half of 2023. To date, 10 patients have been treated in the European study, including all six patients in the lower-dose cohort and four of nine patients in the higher-dose cohort.
- The DSMB recommended implementing additional risk mitigation procedures during the first two weeks after the surgical administration of AMT-130, including a seven-day, in-person post-surgical visit. The DSMB also made no change to the treatment protocol regarding the use of immunosuppression that will continue to be at the discretion of the treating physician.
- The Company still plans to announce one to two-years of follow up data from the
U.S. Phase I/II clinical study in the second quarter of 2023.- All 26 patients have been enrolled in the first two cohorts of the randomized, controlled and double-blinded
U.S. Phase I/II study of AMT-130, including 10 patients in the lower-dose cohort (6 treated patients and 4 control patients) and 16 patients in the higher-dose cohort (10 treated patients and 6 control patients). One control patient from the higher-dose cohort was successfully crossed over to treatment in the third quarter of 2022 and received the lower dose of AMT-130.
- All 26 patients have been enrolled in the first two cohorts of the randomized, controlled and double-blinded
- In
June 2022 , the Company announced 12-month follow-up data from the lower-dose cohort of theU.S. Phase I/II study of AMT-130.- The lower dose was generally well-tolerated with no serious adverse events related to treatment.
- In the four treated patients with evaluable data from this cohort, mean levels of mutant Huntingtin protein (mHTT) in the cerebral spinal fluid (CSF) declined at all timepoints compared to baseline and decreased by 53.8% at 12 months of follow-up.
- In the six treated patients in the lower-dose cohort, measurements of neurofilament light chain (NfL) in the CSF, a biomarker of neuronal damage, initially increased as expected following the AMT-130 surgical procedure and declined thereafter, nearing baseline at 12 months of follow-up.
- The lower dose was generally well-tolerated with no serious adverse events related to treatment.
- In
- Progress towards 2023 Investigational New Drug (IND) Applications
- 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. - AMT-191 for the treatment of Fabry disease – In
August 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. - The Company plans to host a virtual investor event on
Tuesday, November 29, 2022 to highlight the unmet medical need of patients with refractory temporal lobe epilepsy and its gene therapy candidate AMT-260, as well as advancements in platform technology and AAV manufacturing capabilities.
- AMT-260 for the treatment of refractory temporal lobe epilepsy (rTLE) – In
- Strong cash position to advance the Company’s programs
- As of
September 30, 2022 , the Company had cash and cash equivalents of$440.3 million . The Company expects that its cash and cash equivalents will fund operations into the first half of 2025 assuming the achievement of the first commercial sales milestones under the CSL Behring Agreement.
- As of
Upcoming Investor Events
• Stifel 2022 Healthcare Conference,
• uniQure virtual investor event,
Financial Highlights
Cash position: As of
Revenues: Collaboration revenue for the three months ended
Cost of contract manufacturing: Cost of contract manufacturing 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 income, net was income of
Net loss:
The net loss for the three months ended
About uniQure
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,
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 we will announce 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 |
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2022 | 2021 | |||||||
(in thousands, except share and per share amounts) | ||||||||
Current assets | ||||||||
Cash and cash equivalents | $ | 440,313 | $ | 556,256 | ||||
Accounts receivable and contract asset | 3,603 | 58,768 | ||||||
Inventories | 4,075 | - | ||||||
Prepaid expenses | 13,692 | 10,540 | ||||||
Other current assets and receivables | 2,894 | 2,675 | ||||||
Total current assets | 464,577 | 628,239 | ||||||
Non-current assets | ||||||||
Property, plant and equipment, net | 47,886 | 43,505 | ||||||
Operating lease right-of-use assets | 27,804 | 25,573 | ||||||
Intangible assets, net | 53,837 | 62,686 | ||||||
23,418 | 27,633 | |||||||
Deferred tax assets, net | 14,627 | 15,647 | ||||||
Other non-current assets | 6,085 | 5,897 | ||||||
Total non-current assets | 173,657 | 180,941 | ||||||
Total assets | $ | 638,234 | $ | 809,180 | ||||
Current liabilities | ||||||||
Accounts payable | $ | 7,566 | $ | 2,502 | ||||
Accrued expenses and other current liabilities | 26,437 | 28,487 | ||||||
Current portion of contingent consideration | 23,537 | - | ||||||
Current portion of operating lease liabilities | 6,434 | 5,774 | ||||||
Total current liabilities | 63,974 | 36,763 | ||||||
Non-current liabilities | ||||||||
Long-term debt | 102,394 | 100,963 | ||||||
Operating lease liabilities, net of current portion | 29,893 | 28,987 | ||||||
Contingent consideration, net of current portion | 9,158 | 29,542 | ||||||
Deferred tax liability, net | 8,592 | 12,913 | ||||||
Other non-current liabilities | 3,053 | 4,236 | ||||||
Total non-current liabilities | 153,090 | 176,641 | ||||||
Total liabilities | 217,064 | 213,404 | ||||||
Shareholders' equity | ||||||||
Total shareholders' equity | 421,170 | 595,776 | ||||||
Total liabilities and shareholders' equity | $ | 638,234 | $ | 809,180 |
uniQure N.V. UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS |
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Three months ended |
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2022 | 2021 | |||||||
(in thousands, except share and per share amounts) | ||||||||
Total revenues | $ | 1,449 | $ | 1,989 | ||||
Operating expenses: | ||||||||
Cost of contract revenues | - | - | ||||||
Cost of contract manufacturing | (861 | ) | ||||||
Research and development expenses | (48,068 | ) | (36,432 | ) | ||||
Selling, general and administrative expenses | (13,324 | ) | (12,023 | ) | ||||
Total operating expenses | (62,253 | ) | (48,455 | ) | ||||
Other income | 1,485 | 1,680 | ||||||
Other expense | (199 | ) | (214 | ) | ||||
Loss from operations | (59,518 | ) | (45,000 | ) | ||||
Non-operating items, net | 11,332 | 8,558 | ||||||
Loss before income tax benefit / (expense) | $ | (48,186 | ) | $ | (36,442 | ) | ||
Income tax benefit / (expense) | 329 | (89 | ) | |||||
Net loss | $ | (47,857 | ) | $ | (36,531 | ) | ||
Earnings per ordinary share - basic | ||||||||
Basic net (loss) per ordinary share | $ | (1.02 | ) | $ | (0.79 | ) | ||
Earnings per ordinary share - diluted | ||||||||
Diluted net (loss) per ordinary share | $ | (1.02 | ) | $ | (0.79 | ) | ||
Weighted average shares - basic | 46,772,430 | 46,152,404 | ||||||
Weighted average shares - diluted | 46,772,430 | 46,152,404 |