OREANDA-NEWS. 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 third quarter of 2017 and highlighted recent progress across its business.

“In recent months, we have made tremendous progress advancing our gene therapy candidates in hemophilia B and Huntington’s disease, and continue to build momentum as we near the end of the year,” stated Matthew Kapusta, chief executive officer of uniQure. “Based on recent meetings with the FDA and EMA, we now have what we believe is a clear pathway to initiate a pivotal study in 2018 with AMT-061, an AAV5-Padua gene therapy.  We believe AMT-061 has the potential to provide consistent, long-term benefits to nearly all patients with hemophilia B, without the complications often associated with immune responses to the capsid.  Preparations for the pivotal study are underway and the manufacturing of AMT-061 for clinical use has been initiated.”

“Regarding AMT-130 in Huntington’s disease, we received Orphan Drug Designation from the FDA and presented new preclinical data demonstrating functional and survival benefit,” added Mr. Kapusta.  “We are also pleased to announce that we recently completed dosing of a toxicology study that will support an IND filing in 2018.  For the remainder of this year, we remain focused on the execution across our programs and look forward to sharing updates on our progress in the near future.”

Third Quarter 2017 and Recent Company Progress:

•  Achieved significant progress with hemophilia B gene therapy program by introducing the Padua transgene

  • Based on recent meetings with the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA), the Company plans to initiate a pivotal study in 2018 with AMT-061, an AAV5-Padua FIX gene therapy.
  • The FDA agreed that AMT-061 will fall under the existing Breakthrough Therapy Designation and Investigational New Drug (IND) for AMT-060, and the EMA also agreed that AMT-061 will fall under the current PRIME designation. The Company also achieved general agreement with the FDA and EMA on the AMT-061 comparability plan and proposed pivotal trial design.
  • A Good Laboratory Practices (GLP), nonclinical study of AMT-061 performed in non-human primates demonstrated a strong correlation between dose and human FIX (hFIX) expression levels, as well as the biological activity of the expressed hFIX protein. AMT-061 demonstrated substantial increases in hFIX clotting activity compared to AMT-060, and was well-tolerated with no evidence of any significant toxicological findings.
  • uniQure has initiated production of multiple clinical-grade batches of AMT-061 in its state-of-the-art Lexington, MA manufacturing facility. Production is at commercial scale and utilizing current Good Manufacturing Practices.  Data reviewed to date support comparability between AMT-061 and AMT-060.

•  Acquired exclusive patent family covering the use of Padua in gene therapy for hemophilia B

  • The intellectual property broadly covers the Padua FIX variant (R338L) and its use in gene therapy and includes a patent issued in the U.S., as well as pending patent applications in Europe.
  • The patent family was acquired from Professor Paolo Simioni, a renowned hemophilia expert at the University of Padua, Italy, who is widely recognized as the first to identify the mutation. Professor Simioni is serving as an advisor and consultant exclusively to uniQure for the development of gene therapy products using his invention.

•  Continued progress on AMT-130 gene therapy in Huntington’s disease

  • Preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) 25th Anniversary Congress in Berlin, Germany demonstrated substantial improvement in motor symptoms and median survival following a single administration of AMT-130 (AAV5-miHTT) gene therapy in a mouse model with highly aggressive disease.
  • The Company has completed the dosing of non-human primates in a GLP toxicology study of AMT-130.  This study will support the filing of an IND in 2018, with the goal of being the first one-time administered gene therapy to enter a clinical study for Huntington’s disease.
  • Orphan Drug Designation (ODD) from the FDA was granted to AMT-130 in October 2017.

•  Added talent to leadership team and Board of Directors

  • On August 7, the Company announced the appointments of Dr. Scott McMillan as Chief Operating Officer and Dr. Sander van Deventer as Chief Scientific Officer & General Manager, Amsterdam.
  • On September 14, the Company’s shareholders approved the appointments of Dr. Jeremy Springhorn and Madhavan Balachandran to the Board of Directors.

•  Completed follow-on offering

  • On October 27, the Company completed an underwritten public offering of 5,000,000 of its ordinary shares at a public offering price of $18.25 per share. The gross proceeds from the offering, before deducting the underwriting discounts and commissions and estimated offering expenses, were approximately $91,250,000. uniQure has granted to the underwriters a 30-day option to purchase up to 750,000 additional ordinary shares at the public offering price, less underwriting discounts and commissions.

Upcoming Anticipated Milestones

  • Presentation of nonclinical data of AMT-061 at the 59th American Society of Hematology (ASH) Meeting & Exposition
  • Presentation of up to two year follow-up data of AMT-060 from an ongoing Phase I/II clinical trial in hemophilia B at the 59th ASH Meeting & Exposition
  • Completion of the comparability analysis for AMT-061 and submission of data to regulatory agencies for review
  • Submission of the IND amendment for AMT-061
  • Commencement of patient enrollment in the AMT-061 dose confirmation and pivotal Phase III studies
  • Submission of the IND for AMT-130 in Huntington’s disease
  • Initiation of a preclinical therapeutic heart study for AMT-126, an S100A1 gene therapy targeting congestive heart failure

Financial Highlights

Cash Position:  As of September 30, 2017, the Company held cash and cash equivalents of $88.9 million, compared to $132.5 million as of December 31, 2016. The decrease in cash was primarily related to the advancement of its clinical and preclinical gene therapy targets, general corporate activities and capital expenditures related to its facilities. 

Revenues: Revenues for the three months ended September 30, 2017 were $2.3 million compared to $7.2 million for the same period in 2016. Collaboration revenues for the third quarter of 2017 were $1.1 million, compared to $6.0 million for the comparable period in 2016.  The decrease in collaboration revenue was primarily due to the termination of the Chiesi co-development agreement in July 2017, as well as nonrecurring revenue recognized in the prior year period associated with the production of AMT-126 product supplies.

R&D Expenses: Research and development expenses for the three months ended September 30, 2017 were $20.1 million compared to $16.6 million for the same period in 2016. The increase primarily results from non-cash changes in fair value of the contingent consideration related to the Company’s 2014 acquisition of InoCard.

SG&A Expenses: Selling, general and administrative expenses for the three months ended September 30, 2017 were $5.6 million compared to $5.1 million for the same period in 2016. The increase was primarily related to larger share-based compensation expenses in the current year period.

Other income: Other income for the three months ended September 30, 2017 were $14.4 million compared to $0.3 million for the same period in 2016. The current year period includes the full amortization of the outstanding deferred revenue of $13.8 million following the termination of the Company’s collaboration with Chiesi in July 2017.

Net Loss: The net loss for the third quarter of 2017 was $10.2 million, or $0.40 per share, compared to $15.3 million, or $0.61 per share, for the third quarter of 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.

uniQure N.V.
  September 30,   December 31,
 2017    2016
  in thousands, except share and per share amounts
Current assets      
Cash and cash equivalents $   88,934   $   132,496
Accounts receivables and accrued income     1,945       9,180
Prepaid assets and other current assets     1,436       2,270
Total current assets     92,315       143,946
Non-current assets      
Property, plant and equipment, net     34,653       35,702
Intangible assets and goodwill     9,549       8,789
Other non-current assets     2,469       1,828
Total non-current assets      46,671       46,319
Total assets $    138,986   $    190,265
Current liabilities      
Accounts payable $   2,987   $   5,524
Accrued expenses and other current liabilities     10,165       9,766
Current portion of long-term debt     6,232       605
Current portion of deferred rent     724       684
Current portion of deferred revenue     4,249       6,142
Current portion of contingent consideration     1,017       -
Total current liabilities     25,374       22,721
Non-current liabilities      
Long-term debt, net of current portion     14,353       19,631
Deferred rent, net of current portion     8,829       6,781
Deferred revenue, net of current portion     67,863       75,612
Contingent consideration, net of current portion     2,593       1,838
Other non-current liabilities     367       51
Total non-current liabilities     94,005       103,913
Total liabilities     119,379       126,634
Total shareholders' equity     19,607       63,631
Total liabilities and shareholders' equity $    138,986   $    190,265
uniQure N.V.  
  Three months ended September 30,  
   2017   2016  
  in thousands, except share and per share amounts  
Total revenues $    2,260     $    7,221    
Operating expenses:        
Research and development expenses     (20,103 )       (16,604 )  
Selling, general and administrative expenses     (5,584 )       (5,113 )  
Total operating expenses     (25,687 )       (21,717 )  
Other income     14,413         336    
Other expense     (261 )       -    
Loss from operations     (9,275 )       (14,160 )  
Non operating items, net     (1,248 )       (935 )  
Loss before income tax expense     (10,523 )       (15,095 )  
Income tax benefit / (expense)     278         (177 )  
Net loss $    (10,245 )   $    (15,272 )  
Basic and diluted net loss per common share $   (0.40 )   $   (0.61 )  
Weighted average shares used in computing basic and diluted net loss per common share     25,632,642         25,142,660