OREANDA-NEWS. ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe orphan diseases such as cystic fibrosis (CF) and Leber's congenital amaurosis Type 10 (LCA10), today announced results for the first quarter of 2016.

“We continue to make great progress with our clinical development programs. Our first molecule, QR-010 for cystic fibrosis is in two active clinical trials and our second molecule, QR-110 for LCA10 is moving towards a first study in affected patients. We have also made a lot of progress in advancing our innovation pipeline", said Daniel de Boer, Chief Executive Officer of ProQR. “This quarter we organized an inaugural R&D Day where we, along with key opinion leaders presented our deep pipeline of RNA based molecules which hold the promise of restoring normal protein function for a range of genetic diseases with high unmet medical needs." 

Financial Highlights

At March 31, 2016, ProQR held cash and cash equivalents of €85.5 million, compared to €94.9 million at December 31, 2015. Net cash used in operating activities during the three month period ended March 31, 2016 was €7.8 million, compared to €5.3 million for the same period last year.

Research and development costs increased to €6.9 million for the quarter ended March 31, 2016 from €5.5 million for the same period last year and comprised of allocated employee costs including share-based payments, the costs of materials and laboratory consumables, outsourced activities, license and intellectual property costs and other allocated costs. The increase in expenses was primarily due to the clinical trial costs for QR-010 and clinic enabling studies for QR-110. The remainder represents increased investments in our other pipeline programs.

General and administrative costs increased to €2.6 million for the quarter ended March 31, 2016 from €1.6 million for the same period last year, primarily due to increased investments into our support organization. These costs include spend on the expansion of our lab and office facilities for the growing pipeline and headcount as well as increased spend on several compliance related activities, including internal controls for Sarbanes-Oxley (SOX).

Net result for the three month period ended March 31, 2016 was a €10.2 million loss or €0.44 per share, compared to a €0.2 million profit or €0.01 per share for the same period last year, which was driven primarily due to currency fluctuations. For further financial information for the period ending March 31, 2016, please refer to the financial statements appearing at the end of this release.

Corporate Highlights

  • On March 14th the company organized an R&D Day in New York where ProQR executives as well as external key opinion leaders presented more information on ProQRs pipeline products including programs for cystic fibrosis, Leber’s congenital amaurosis Type 10, Usher syndrome, Fuchs endothelial corneal dystrophy, dystrophic epidermolysis bullosa and Alzheimer’s disease.

Subsequent events

  • At the 2016 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) the company presented pre-clinical data for QR-110 for Leber’s congenital amaurosis Type 10 (LCA10). QR-110 is a single-stranded, chemically modified RNA oligonucleotide designed to skip the cryptic splice site caused by the p.Cys998X mutation resulting in mRNA that codes for a wild-type CEP290 protein. Following intravitreal injection in vivo, QR-110 was demonstrated to reach the outer nuclear layer of the retina, the target tissue. QR-110 was also demonstrated to increase wild-type mRNA in cells with the p.Cys998X mutation.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe orphan diseases such as cystic fibrosis and Leber’s congenital amaurosis. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Since 2012.

About QR-010

QR-010 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by repairing the mRNA defect encoded by the DF508 mutation in the CFTR gene of CF patients. The DF508 mutation is a deletion of three of the coding base pairs, or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally. QR-010 is designed to bind to the defective CFTR mRNA and guide the insertion of the three missing nucleotides, thus repairing the mRNA and subsequently producing wild-type, or normal CFTR protein. QR-010 is designed to be self-administered through a small, handheld aerosol delivery device, or nebulizer, in the form of a mist inhaled into the lungs. We believe this method could allow maximum exposure of QR-010 to the primary target organ, the lung, as well as significant exposure to other affected organs through systemic absorption into the blood. QR-010 has been granted orphan drug designation in the United States and the European Union. The QR-010 project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 633545.

About QR-110

QR-110 is a first-in-class oligonucleotide, designed to address the underlying cause of Leber’s congenital amaurosis Type 10 due to the p.Cys998X mutation in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional Cep290 protein. QR-110 is designed to restore wild-type CEP290 mRNA leading to the production of wild-type CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 is intended to be administered through intravitreal injections in the eye.

 
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Financial Position
    March 31,    December 31, 
    2016   2015
    € 1,000   € 1,000
Assets        
Current assets        
Cash and cash equivalents   85,467   94,865
Prepayments and other receivables   2,478   1,948
Social securities and other taxes   533   956
         
         
Total current assets   88,478   97,769
         
Property, plant and equipment   2,790   2,199
Intangible assets   128   141
         
Total assets   91,396   100,109
         
Liabilities and shareholders' equity        
Current liabilities        
Finance lease liabilities   7   15
Trade payables   1,026   885
Social securities and other taxes   153   235
Pension premiums   31   16
Deferred income   --   144
Other current liabilities   4,841   4,191
         
Total current liabilities   6,058   5,486
         
Finance lease liabilities   --   --
Borrowings   5,142   4,824
         
Total liabilities   11,200   10,310
         
Shareholders' equity        
Shareholders' equity   80,196   89,799
         
Total liabilities and shareholders' equity   91,396   100,109
 
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Profit or Loss and OCI
(€ in thousands, except share and per share data)
    Three month period
ended March 31,
      2016       2015  
      € 1,000       € 1,000  
Other income     689       338  
         
Research and development costs       (6,898 )       (5,480 )
General and administrative costs       (2,602 )       (1,603 )
         
Total operating costs       (9,500 )       (7,083 )
         
Operating result       (8,811 )       (6,745 )
Finance income and expense       (1,387 )       6,980  
         
Result before corporate income taxes       (10,198 )     235  
Income taxes     --       --  
         
Net result attributable to equity holders of the Company       (10,198 )     235  
Other comprehensive income     5       --  
         
Total comprehensive income (attributable to equity       (10,193 )     235  
holders of the Company)        
         
Share information        
Weighted average number of shares outstanding1 – basic     23,345,965       23,338,663  
Weighted average number of shares outstanding1 – diluted     23,345,965       24,378,072  
         
Earnings per share attributable to the equity holders        
of the Company (expressed in Euro per share)        
Basic loss per share     (0.44)1       0.01  
Diluted loss per share     (0.44)1       0.01  
         
1.  For this period presented in these financial statements, the potential exercise of share options is not included in the diluted earnings per share calculation as the Company was loss-making in the period. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal in this period.
 
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Changes in Equity
                Equity            
                Settled            
        Total       Employee            
    Number of   Share   Share   Benefit   Translation    Accumulated   Total
     shares   Capital   Premium   Reserve   Reserve   Deficit   Equity
        € 1,000   € 1,000   € 1,000   € 1,000     € 1,000       € 1,000  
Balance at January 1, 2015   23,338,154   934   123,581   687   --       (15,798 )     109,404  
Net loss   --   --   --   --   --     235       235  
Recognition of share-based payments   --   --   --   288   --     --       288  
Share options exercised   5,090   0   5   --   --     --       5  
                             
Balance at March 31, 2015   23,343,244   934   123,586   975   --       (15,563 )     109,932  
                             
Balance at January 1, 2016   23,345,965   934   123,595   1,899   1       (36,630 )     89,799  
Net loss   --   --   --   --   --     (10,198 )     (10,198 )
Other comprehensive income   --   --   --   --   5     --       5  
Recognition of share-based payments   --   --   --   590   --     --       590  
Share options exercised   --   --   --   --   --     --       --  
                             
Balance at March 31, 2016   23,345,965   934   123,595   2,489   6     (46,828 )     80,196  
 
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows
    Three month period
ended March 31,
      2016       2015  
      € 1,000       € 1,000  
Cash flows from operating activities        
Net result       (10,193 )       235  
Adjustments for:        
— Depreciation       334         95  
— Share-based compensation       590         288  
— Financial income and expenses       1,387         (6,980 )
         
Changes in working capital       50         1,015  
Cash used in operations       (7,832 )       (5,347 )
         
Corporate income tax paid      --       --  
Interest received/(paid)       65         72  
         
Net cash used in operating activities       (7,767 )       (5,275 )
         
Cash flow from investing activities        
Purchases of intangible assets     --        --  
Purchases of property, plant and equipment       (502 )       (488 )
         
Net cash used in investing activities       (502 )       (488 )
         
Cash flow from financing activities        
Proceeds from exercise of share options     --         5  
Proceeds from borrowings       193       --   
Redemption of financial lease       (8 )       (12 )
         
Net cash generated by financing activities       185         (7 )
         
Net increase/(decrease) in cash and cash equivalents       (8,084 )       (5,770 )
         
Currency effect cash and cash equivalents       (1,314 )       6,849  
Cash and cash equivalents, at beginning of the period       94,865         112,736  
         
Cash and cash equivalents at the end of the period       85,467         113,815