uniQure Announces Second Quarter 2018 Financial Results and Highlights Company Progress

~ Patient Enrollment Underway in Global Phase III HOPE-B Pivotal Study of AMT-061

~ First Patient Successfully Screened into Phase IIb Dose-Confirmation Trial of AMT-061

~ Robert Gut, M.D., Ph.D., Named Chief Medical Officer and Other Appointments to Clinical Development Leadership

~ Ended Second Quarter with $259 Million in Cash and Cash Equivalents; Expected to Fund Operations into 2021

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Aug. 08, 2018 (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 second quarter of 2018 and highlighted recent progress across its business.   

“We have made tremendous progress over the past several months advancing our gene therapy programs in hemophilia B and Huntington’s disease, as well as in expanding our intellectual property portfolio and executing on our manufacturing plan,” said Matt Kapusta, chief executive officer of uniQure. “Our Phase III pivotal trial of AMT-061 is actively enrolling patients, and we have consented and successfully screened the first of three patients in our Phase IIb dose-confirmation study.” 

“The initiation of our registrational study provides the opportunity for uniQure to be first to market with a one-time treatment that has the potential to transform the standard of care for patients with hemophilia B,” Mr. Kapusta added. “We continue to work toward what we expect will be an equally productive second half of 2018 with the achievement of multiple value-creating catalysts across the company, including top-line data from our dose-confirmation study, the submission of an Investigational New Drug application for AMT-130 in Huntington’s disease, and hosting a Research and Development Day that will feature expansion of our early-stage pipeline.”

Recent Company Progress

Near-Term Company Milestones

The company expects to achieve the following milestones in the second half of 2018:

Financial Highlights

The company strengthened its financial position through completion of an underwritten public offering in May 2018.  The gross proceeds from the offering, before deducting underwriting discounts and commissions and estimated offering expenses payable by uniQure, were $147.5 million.

Cash Position: As of June 30, 2018, the Company held cash and cash equivalents of $259.2 million, compared to $159.4 million as of December 31, 2017. The Company currently expects cash and cash equivalents will be sufficient to fund operations into 2021.

Revenues: Revenue for the three months ended June 30, 2018 was $3.1 million, compared to $4.9 million for the comparable period in 2017. Collaboration revenue for the three months ended June 30, 2018 was $0.9 million, compared to $4.2 million for the comparable period in 2017. The decrease in collaboration revenue was primarily due to the termination of the Chiesi co-development agreement in July 2017.

R&D Expenses: Research and development expenses were $18.5 million for the three months ended June 30, 2018, compared to $16.9 million for the comparable period in 2017. The increase was primarily related to costs incurred preparing for the initiation of the AMT-061 pivotal study and continued IND-enabling nonclinical studies of AMT-130.

SG&A Expenses: Selling, general and administrative expenses were $5.9 million for the three months ended June 30, 2018, compared to $5.4 million for the comparable period in 2017.

Other income, net: Other income, net was an income of $0.1 million for the three months ended June 30, 2018, compared to an expense of $2.4 million for the comparable period in 2017.

Net Loss: The net loss was $20.6 million, or $0.57 per share, for the three months ended June 30, 2018, compared to $21.3 million, or $0.83 per share, for the comparable period in 2017.

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 and partnered gene therapies to treat patients with liver/metabolic, central nervous system and cardiovascular diseases.

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," "estimate," "expect," "goal," "intend," "look forward to", "may," "plan," "potential," "predict," "project," "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 completion of our Phase IIb study, the release of top-line clinical data, the opportunity to be first to market with a Hemophilia B product, the filing of an IND or other regulatory filings or their acceptance by regulatory authorities, initiating a Huntington’s Disease program or other program, trial or study that is the first in the clinic, completion of our GLP safety and toxicology study, our heart function study or other studies, the achievement of any of our planned near term or other milestones, the development of our gene therapy product candidates, the transition to our AMT-061 product candidate, the success of our collaborations and the risk of cessation, delay or lack of success of any of our ongoing or planned clinical studies and/or development of our product candidates. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our and our collaborators’ clinical development activities, collaboration arrangements, corporate reorganizations and strategic shifts, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading "Risk Factors" in uniQure’s Annual Report on Form 10-K filed on April 30th, 2018. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

uniQure Contacts:
Maria E. Cantor Eva M. Mulder Tom Malone
Direct: 339-970-7536 Direct: +31 20 240 6103 Direct: 339-970-7558
Mobile:  617-680-9452 Mobile: +31 6 52 33 15 79 Mobile: 339-223-8541 

uniQure N.V.


 June 30, December 31,
 2018  2017
 in thousands, except share and per share amounts
Current assets   
Cash and cash equivalents$  259,180 $  159,371
Accounts receivables and accrued income   1,037    1,586
Prepaid assets and other current assets   2,489    1,826
Total current assets   262,706     162,783
Non-current assets   
Property, plant and equipment, net   32,126    34,281
Intangible assets and goodwill   11,249    10,100
Restricted cash   2,458    2,480
Total non-current assets    45,833     46,861
Total assets$   308,539  $   209,644
Current liabilities   
Accounts payable$  3,866 $  2,908
Accrued expenses and other current liabilities   8,920    8,838
Current portion of long-term debt   8,028    1,050
Current portion of deferred rent   1,082    737
Current portion of deferred revenue   8,463    4,613
Current portion of contingent consideration   1,081    1,084
Total current liabilities   31,440     19,230
Non-current liabilities   
Long-term debt, net of current portion   12,840    19,741
Deferred rent, net of current portion   8,464    9,114
Deferred revenue, net of current portion   32,853    67,408
Contingent consideration, net of current portion   2,704    2,880
Derivative financial instruments related party   1,309    1,298
Other non-current liabilities   513    614
Total non-current liabilities   58,683     101,055
Total liabilities   90,123     120,285
Total shareholders' equity   218,416     89,359
Total liabilities and shareholders' equity$   308,539  $   209,644

 uniQure N.V.


 Three months ended June 30,  
  2018   2017  
 in thousands, except share and per share amounts 
Total revenues$   3,050   $   4,942   
Operating expenses:    
Research and development expenses   (18,493)    (16,866) 
Selling, general and administrative expenses   (5,896)    (5,410) 
Total operating expenses   (24,389)    (22,276) 
Other income   565     266  
Other expense   (429)    (2,640) 
Loss from operations   (21,203)    (19,708) 
Non operating items, net   972     (1,561) 
Loss before income tax expense   (20,231)    (21,269) 
Income tax expense   (361)    -  
Net loss$   (20,592) $   (21,269) 
Basic and diluted net loss per common share$  (0.57) $  (0.83) 
Weighted average shares used in computing basic and diluted net loss per common share   36,205,061     25,560,348