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uniQure Announces Financial Results For Second Quarter 2016 and Update on Company Progress

2016-08-25 16:05 ET - News Release

~ Progress Across the Pipeline
~ Additional data on AMT-060 in Hemophilia B Expected Later this Year
~Data from Sanfilippo B and Parkinson’s Disease Studies Expected in Q1 2017
~Strong Balance Sheet to Support Advancement of Programs

LEXINGTON, Mass. and AMSTERDAM, the Netherlands, Aug. 25, 2016 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ:QURE), a leader in human gene therapy, today announced its financial results for the second quarter, ended June 30, 2016, and provided an update on corporate developments.

“We continue to make solid progress on our pipeline programs and are pleased to maintain a strong cash position that will enable us to advance our product candidates well into the future,” stated Daniel Soland, chief executive officer of uniQure. “We presented promising updated clinical data from the low-dose cohort of our ongoing Phase I/II study of AMT-060, which demonstrated durable, therapeutically relevant Factor IX activity in older patients with severe hemophilia B and advanced joint disease.  Importantly, we expect to present up to one year of follow-up data from our low dose cohort and up to 6 months of follow-up data from our second dose cohort of AMT-060 later this year.  Additionally, we look forward to clinical data presentations from studies in Sanfilippo B and Parkinson’s disease in the first quarter of 2017.”

Recent Corporate Highlights

Pipeline and Business Updates

Liver/Metabolic Diseases

  • Advancing AMT-060 in Hemophilia B – uniQure presented updated results from its ongoing Phase I/II clinical trial of AMT-060 in patients with severe hemophilia B and advanced joint disease at the World Federation of Hemophilia Congress. The clinical data from the low-dose cohort showed sustained Factor IX (FIX) activity and a substantial reduction in FIX usage. All patients had improvements in their disease phenotype and continued to maintain durable levels of FIX activity for up to 9 months post treatment.  Four out of five patients remained free of prophylactic FIX usage with mean steady-state FIX activity of 5.4% of normal and an 82% reduction in total FIX usage through up to 9 months.  Of note, AMT-060 continues to demonstrate a very low screening failure rate, with all patients screened in the study testing negative for pre-existing anti-AAV5 antibodies. All five patients in the second-dose cohort have now been treated, with 6-month data expected to be available for presentation later this year.    

Central Nervous System (CNS) Diseases

  • Ongoing Follow-up of AMT-110 in Sanfilippo B - uniQure anticipates that all four patients in this Phase I/II extension study will have completed their 30-month evaluation late this year and that clinical data will be presented in the first quarter of 2017.  uniQure assumed sponsorship of the Phase I/II study extension protocol earlier this year.

  • Advancing Preclinical Research of AMT-130 in Huntington’s Disease – uniQure is conducting ongoing IND-enabling studies of AMT-130 in non-human primates.  Earlier this year, uniQure published and presented data providing preclinical proof-of-concept and demonstrating the potential of a one-time administration of AAV5-delivered gene therapy to successfully silence the Huntingtin gene.

  • Continued Progress on Phase I/IIa Study of AMT-140 in Parkinson’s Disease – Two of the four, six-patient cohorts have now been fully enrolled since June 2016 in an ongoing Phase I/IIa clinical study of AMT-140, an AAV2-GDNF gene therapy for the treatment of Parkinson’s disease.  Top-line data, including safety, tolerability and preliminary efficacy results, are expected to be available for presentation in the first quarter of 2017.  The study is being conducted under the direction of Krystof Bankiewicz at the University of California, San Francisco (UCSF) and John Heiss at the National Institutes of Health.  uniQure has exclusive rights to GDNF in gene therapy and has the option to acquire the program from UCSF. 

Cardiovascular Diseases

  • Increasing Activity in Support of BMS Collaboration – uniQure and its collaboration partner, Bristol Myers Squibb (BMS), continue to conduct IND-enabling studies for S100A1, a proprietary gene therapy for chronic heart failure.  In addition to S100A1, uniQure and BMS have initiated work on 3 additional targets designated under their collaboration. 

Leadership Team and Board of Directors

  • Added Key Talent – In the second quarter, uniQure significantly strengthened its senior management team with the appointment of Paul Firuta as Chief Commercial Officer, Hans Janssen as Senior Vice President, Human Resources, Maria E. Cantor as Senior Vice President, Investor Relations & Communications, and Jonathan Garen as Chief Business Officer.

  • Completed Corporate Governance Changes – Following its Annual General Meeting in June, the Company transitioned from a two-tier Supervisory Board and Management Board to a single Board of Directors structure with executive and non-executive members. This new structure reflects corporate governance standards most familiar to the majority of the Company’s shareholder base.

    In addition, the Company appointed Jack Kaye, a seasoned financial executive with more than 40 years of diversified experience, to its Board of Directors.  Company shareholders also approved the appointments of Daniel Soland, chief executive officer, and Matthew Kapusta, chief financial officer, as executive directors of the Board.  Finally, as part of the governance transition, Mr. Ferdinand Verdonck and Mr. Joseph Fezcko retired from the board.

Business Development

  • In the second quarter, uniQure revised its existing license contract with Protein Sciences Corporation for the use of its expresSF+ (SF+) insect cell line to provide the Company with a royalty free, perpetual right and license to the licensed technology in the field of AAV-based gene therapy.

Financial Highlights for Q2 2016

As of June 30, 2016, the Company held cash and cash equivalents of €165.6 million, compared with of €203.5 million as of December 31, 2015, a decrease of €37.9 million. The decrease includes €36.4 million of cash used in operating, investing and financing activities during the six months ended June 30, 2016, in addition to a €1.5 million loss related to foreign currency effects on U.S. dollar-denominated deposits. The Company maintains its expectation that existing cash resources will be sufficient to fund operations into the second half of 2018.   

Licensing and collaboration revenues for the three months ended June 30, 2016 were €3.9 million, compared with €1.6 million for the comparable period in 2015. The increase relates to increased research activity associated with S100A1 for heart failure, which are fully reimbursed by BMS in accordance with the Company’s collaboration agreement.

Research and development expenses were €17.3 million for the three months ended June 30, 2016, compared with €10.6 million for the comparable period in 2015. The increase is mainly due to increased activity in support of our collaboration agreement with BMS, validation and scale-up activities related to AMT-060 in the Company’s Lexington facility, increased facility costs in Amsterdam and the continued progression of our gene therapy product candidates targeting Sanfilippo B, Huntington’s disease and hemophilia A.    

Selling, general and administrative expenses were €5.5 million for the three months ended June 30, 2016, compared with €4.5 million for the comparable period in 2015. The change is primarily related to an increase in personnel expense, as well as an increase in expenses associated with the Glybera global registry and Phase IV study, which are currently classified as selling, general and administrative expenses, but were previously capitalized in the same period in 2015 prior to the commencement of Glybera commercialization.

The net loss for the second quarter 2016 was €19.3 million, or €0.77 per share, compared with €18.9 million, or €0.87 per share, for the second quarter of 2015. The Company had 25,137,009 ordinary shares outstanding as of June 30, 2016.

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. 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," "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, statements regarding the development of our gene therapy product candidates, 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 collaboration arrangements, our and our collaborators’ clinical development activities, 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 2015 Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 4, 2016. 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
Direct: 339-970-7536
Mobile:  617-680-9452
m.cantor@uniQure.com

Eva Mulder
Direct: +31 20 240 6103
Mobile: +31 6 52 33 15 79
e.mulder@uniQure.com

 
UNIQURE N.V.
Unaudited Condensed Consolidated Statement of Financial Position
(€ in thousands)
 
  December 31, June 30,
  2015   2016 
Assets    
Non-current assets    
Goodwill  442   442 
Intangible assets other than Goodwill  7,209   8,956 
Property, plant and equipment  23,820   24,041 
Other non-current assets  1,142   1,675 
Total non-current assets  32,613   35,114 
Current assets    
Receivables from related parties  3,792   1,502 
Trade and other receivables  1,730   5,195 
Inventories  435   363 
Cash and cash equivalents  203,532   165,646 
Total current assets  209,489   172,706 
Total assets    242,102      207,820  
Equity    
Share capital  1,216   1,257 
Share premium  344,803   346,732 
Other reserves  26,026   29,785 
Accumulated deficit  (252,561)  (292,468)
Total equity  119,484   85,306 
Liabilities    
Non-current liabilities    
Borrowings  13,434   18,356 
Derivative financial instruments - related party  530   136 
Deferred rent  5,737   5,789 
Deferred revenue  75,852   73,507 
Contingent consideration  2,687   2,915 
Total non-current liabilities  98,240   100,703 
Current liabilities    
Trade and other payables  11,220   12,568 
Derivative financial instruments - related parties  992   222 
Borrowings  5,124   - 
Borrowings - derivative  238   51 
Deferred rent  579   592 
Deferred revenue  6,225   5,978 
Provisions  -   2,400 
Total current liabilities  24,378   21,811 
Total liabilities    122,618      122,514  
Total equity and liabilities    242,102      207,820  
         


UNIQURE N.V.
Unaudited Condensed Consolidated Statements of Comprehensive Income
(€ in thousands except share and per share data)
 
 Three months ended Six months ended
 June 30, June 30,
        
 2015   2016   2015   2016 
License revenues 492   1,101   713   2,202 
Collaboration revenues 1,123   2,840   2,009   5,641 
Total revenues   1,615      3,941      2,722      7,843  
Cost of goods sold -   (76)  -   (152)
Other income 140   423   346   824 
Research and development expenses (10,613)  (17,335)  (20,719)  (32,463)
Selling, general and administrative expenses (4,509)  (5,504)  (8,668)  (12,108)
Other gains/(losses), net (5,241)  (321)  (996)  (4,379)
Total operating costs   (20,223)    (22,813)    (30,037)    (48,278)
Operating loss   (18,608)    (18,872)    (27,315)    (40,435)
Finance income 46   64   65   1,584 
Finance expense (325)  (523)  (4,275)  (1,056)
Finance income/(expense), net   (279)    (459)    (4,210)    528  
Result before corporate income tax   (18,887)    (19,331)    (31,525)    (39,907)
Provision for income taxes -   -   -   - 
Net loss   (18,887)    (19,331)    (31,525)    (39,907)
Items that may be subsequently reclassified to profit or loss       
Currency translation differences on foreign operations (285)  356   1,086   (301)
Other comprehensive income/(loss) (285)  356   1,086   (301)
Total comprehensive loss   (19,172)    (18,975)    (30,439)    (40,208)
Loss per share attributable to the equity holders of the       
Company during the period:       
Basic and diluted loss per share   (0.87)    (0.77)    (1.58)    (1.60)
                


UNIQURE N.V.
Unaudited Condensed Consolidated Statements of Changes in Equity/Deficit
(€ in thousands)
           
  Total        
  share Share Other Accumulated Total
  capital premium reserves deficit equity
Balance at January 1, 2015   905     206,111      17,149      (181,081)    43,084  
Loss for the period -  -   -   (31,525)  (31,525)
Other comprehensive income/(loss) -  -   1,086   -   1,086 
Total comprehensive income/(loss)   -     -      1,086      (31,525)    (30,439)
Capital contributions 231  101,747   -   -   101,978 
Share issuance costs -  (612)  -   -   (612)
Share based payment/expense -  -   3,416   -   3,416 
Balance at June 30, 2015   1,136     307,246      21,651      (212,606)    117,427  
Loss for the period -  -   -   (39,955)  (39,955)
Other comprehensive income/(loss) -  -   164   -   164 
Total comprehensive income/(loss)   -     -      164      (39,955)    (39,791)
Capital contributions 80  37,557   -   -   37,637 
Share based payment/expense -  -   4,211   -   4,211 
Balance at December 31, 2015   1,216     344,803      26,026      (252,561)    119,484  
Loss for the period -  -   -   (39,907)  (39,907)
Other comprehensive income/(loss) -  -   (301)  -   (301)
Total comprehensive income/(loss)   -     -      (301)    (39,907)    (40,208)
Capital contributions 41  1,929   -   -   1,970 
Share based payment/expense -  -   4,060   -   4,060 
Balance at June 30, 2016   1,257     346,732      29,785      (292,468)    85,306  


UNIQURE N.V.
Unaudited Condensed Consolidated Statements of Cash Flows
(€ in thousands)
 
 Six months ended
 June 30,
    
  2015   2016 
Cash flow from operating activities   
Net loss (31,525)  (39,907)
Adjustments for:   
Amortization on intangible assets -   378 
Depreciation 1,877   2,462 
Lease incentive 333   (398)
Loss/(gain) on derivatives 1,666   (1,351)
Loss/(gain) on foreign exchanges 1,873   1,280 
Share-based expenses 3,416   4,060 
Changes in provisions -   2,400 
Changes in trade and other receivables (1,363)  (1,174)
Changes in inventories (214)  72 
Changes in other current liabilities (232)  810 
Changes in deferred revenue 52,769   (2,592)
Cash generated from/(used in) operations   28,600      (33,960)
Interest paid (929)  (1,006)
Net cash generated from/(used in) operating activities   27,671      (34,966)
Cash used in investing activities   
Purchases of intangible assets (1,729)  (10)
Purchases of property, plant and equipment (1,855)  (2,776)
Changes in restricted cash -   (555)
Interest received 59   33 
Net cash used in investing activities   (3,525)    (3,308)
Cash flow from financing activities   
Proceeds from shares issued 101,366   1,970 
Payments of finance lease (82)  (88)
Net cash generated from financing activities   101,284      1,882  
Net increase/(decrease) in cash and cash equivalents   125,430      (36,392)
Currency effect cash and cash equivalents 3,206   (1,494)
Cash and cash equivalents at January 1 53,219   203,532 
Cash and cash equivalents at June 30   181,855      165,646  
        

 

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