LEIDEN, Netherlands and CAMBRIDGE, Mass., Nov. 07, 2018 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced results for the third quarter of 2018.

“During the third quarter, we announced important clinical proof-of-concept data for QR-110, demonstrating a robust improvement in vision in patients with LCA10,” said Daniel A. de Boer, chief executive officer of ProQR. “These results build confidence in QR-110 and the broader ability of oligonucleotides to improve vision in patients with inherited retinal diseases. Based on these encouraging results we are rapidly advancing our pipeline for inherited blindness that includes programs for Usher syndrome, Stargardt’s diseases and FECD. With the recent addition of QR-1123 for autosomal dominant retinitis pigmentosa (adRP) from Ionis Pharmaceuticals we have strategically expanded our pipeline in this key therapeutic area. To fund these activities, we have raised capital that will allow us to fund operations into 2021.”

Third Quarter 2018 Corporate Highlights

  • Presented positive interim clinical data for lead program QR-110 at the Retinal Degeneration 2018 meeting (RD2018). In the interim analysis of the ongoing Phase 1/2 clinical trial, QR-110 demonstrated rapid and sustained improvement in vision in patients with LCA10, as well as being well-tolerated with no serious adverse events recorded related to treatment or procedure. The Company expects to start a pivotal Phase 2/3 clinical trial during the first half of 2019.
  • Closed an underwritten public offering of 6,612,500 ordinary shares at a price of $15.75 per share including full exercise of underwriters’ option to purchase 862,500 additional ordinary shares. Gross proceeds totaled approximately $104.1 million.
  • Delivered two presentations at the Oligonucleotide Therapeutics Society (OTS) conference on clinical data for QR-110 in patients with LCA10 and preclinical data for ProQR’s Axiomer® RNA-editing technology. Abstract titles:
    ○  Proof-of-concept for RNA-editing oligonucleotide QR-110 for treatment of inherited retinal dystrophy in adults and children with LCA10
    ○  
    Structure-based computational approach for optimizing oligonucleotides for A-to-I editing
  • Presented at the International Symposium on Usher Syndrome (USH2018) and the Ophthalmology Innovation Summit (OIS) Retina conferences. Abstract titles:
    ○  Splice modulation to treat USH2A-associated retinal degeneration
    ○  
    RNA treatments targeting rare diseases
  • A paper with preclinical data for QR-110 targeting Leber’s congenital amaurosis 10 (LCA10) was published in Molecular Therapy – Nucleic Acids, an official journal of the American Society of Gene & Cell Therapy
  • With current cash on hand of €113.7 million (at September 30, 2018), ProQR’s operations are funded into 2021.

Subsequent Events

  • In-licensed exclusive worldwide rights for IONIS-RHO-2.5Rx, now QR-1123, from Ionis Pharmaceuticals. QR-1123 is a first-in-class investigational oligonucleotide (gapmer) for the treatment of autosomal dominant retinitis pigmentosa (adRP), a rare inherited form of blindness with no approved therapy
    ○  A first in human Phase 1/2 clinical trial in adRP patients is expected to start in 2019
  • Received a conditional waiver of the €5 million Innovation credit that was awarded by the Dutch Ministry of Economic Affairs for the Company’s cystic fibrosis program. Consequently, the total repayment, including principal and interest, of €7.2 million (scheduled in three annual payments) has been waived and will be reviewed annually.
  • Delivered presentations at European Oligonucleotide and Peptide Therapeutics (EuroTIDES) conference. Abstract titles:
    ○  Childhood Blindness due to a Photoreceptor Cilium Defect Treated with an Intravitreal Antisense Oligonucleotide (QR-110)
    ○  
    Evaluating the impact of sterilization on oligonucleotide based drug products

Financial Highlights

At September 30, 2018, ProQR held cash and cash equivalents of €113.7 million, compared to €48.1 million at December 31, 2017. The increase in cash was due to the closing of an offering of ordinary shares for which net proceeds totaled €84.3 million. Net cash used in operating activities during the three month period ended September 30, 2018 was €4.3 million, compared to €7.6 million for the same period last year.

Research and development costs totaled €6.3 million for the quarter ended September 30, 2018 compared to €7.2 million for the same period last year.

General and administrative costs decreased to €2.6 million for the quarter ended September 30, 2018 compared to €2.8 million for the same period last year.

Net loss for the three month period ended September 30, 2018 was €6.0 million or €0.18 per share, compared to a €10.5 million loss or €0.42 per share for the same period last year. For further financial information for the period ended September 30, 2018, please refer to the financial statements appearing at the end of this release.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as Leber’s congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis. 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-110

QR-110 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G 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 normal (wild-type) CEP290 mRNA leading to the production of normal 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 and has been granted orphan drug designation in the United States and the European Union and received fast-track designation by the FDA.

About QR-421a

QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of vision loss in Usher syndrome 2A due to mutations in exon 13 of the USH2A gene. Mutations in this exon can cause loss of functional usherin protein that causes the disease. QR-421a is designed to exclude the genetic defect from the RNA in the eye, such that it leads to the expression of a shortened but functional protein, thereby modifying the underlying disease. QR-421a has received orphan drug designation in the United States and the European Union.

About QR-1123

QR-1123 is a first-in-class investigational oligonucleotide (gapmer) that was developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology for the treatment of adRP due to the P23H mutation in the RHO gene. The therapy aims to inhibit the formation of the mutated toxic version of the rhodopsin protein by specifically binding the mutated RHO mRNA. Binding of QR-1123 causes allele specific knockdown of the mutated mRNA by a mechanism called RNase H mediated cleavage without affecting the normal RHO mRNA. QR-1123 is intended to be administered through intravitreal injections in the eye.

About Axiomer® Technology Platform

ProQR is pioneering a next-generation RNA technology called Axiomer®, which could potentially yield a new class of medicines for genetic diseases. Axiomer® “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells. The Axiomer® EONs are designed to recruit an endogenously expressed RNA editing system called ADAR, which it can direct to the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G).

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. Such forward-looking statements include, but are not limited to, statements regarding QR-110 and the clinical development and the therapeutic potential thereof, statements regarding our pipeline of programs targeting inherited retinal dystrophies, statements regarding QR-421a, and the clinical development and the therapeutic potential thereof,  statements regarding QR-1123 and the clinical development and therapeutic potential thereof, and statements regarding our Axiomer® Technology Platform and the preclinical development and therapeutic potential thereof. 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. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. 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, except as required by law.

ProQR Therapeutics N.V.
Investor Contact:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431
ir@proqr.com

Media Contact:
Sara Zelkovic
LifeSci Public Relations
T: +1 646 876 4933
Sara@lifescipublicrelations.com

     
PROQR THERAPEUTICS N.V.    
Unaudited Condensed Consolidated Statement of Financial Position    
     
  September 30,  December 31, 
  2018  2017 
  € 1,000  € 1,000 
Assets    
Current assets    
Cash and cash equivalents 113,716  48,099 
Prepayments and other receivables 1,976  2,064 
Social securities and other taxes 1,195  396 
     
Total current assets  116,887   50,559 
     
Property, plant and equipment 2,065  2,505 
Intangible assets 39  39 
     
Total assets  118,991   53,103 
     
Equity and liabilities    
Equity    
Equity attributable to owners of the Company 102,575  39,363 
Non-controlling interests (173) (38)
Total equity  102,402   39,325 
     
Current liabilities    
Borrowings 2,000  1,960 
Trade payables 771  546 
Social securities and other taxes 7  1,019 
Pension premiums    
Deferred income 1,122  347 
Other current liabilities 6,364  4,622 
     
Total current liabilities  10,264   8,494 
     
Borrowings 6,325  5,284 
     
Total liabilities  16,589   13,778 
     
Total equity and liabilities  118,991   53,103 
       


 
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
  Nine month period
 
  ended September 30,
  ended September 30,
 
             
  2018  2017  2018  2017 
  € 1,000  € 1,000  € 1,000  € 1,000 
Other income  2,958   326   4,428   984 
         
Research and development costs  (6,297)  (7,226)  (19,972)  (22,808)
General and administrative costs  (2,579)  (2,753)  (7,900)  (7,949)
         
Total operating costs  (8,876)  (9,979)  (27,872)  (30,757)
         
Operating result  (5,918)  (9,653)  (23,444)  (29,773)
Finance income and expense  (74)  (868)  (664)  (2,589)
         
Result before corporate income taxes  (5,992)  (10,521)  (24,108)  (32,362)
Income taxes  —   —   (1)  (2)
         
Result for the period  (5,992)  (10,521)  (24,109)  (32,364)
Other comprehensive income  (4)  49   (15)  114 
         
Total comprehensive income  (5,996)  (10,472)  (24,124)  (32,250)
         
Result attributable to        
Owners of the Company  (5,959)  (10,511)  (23,974)  (32,354)
Non-controlling interests  (33)  (10)  (135)  (10)
   (5,992)  (10,521)  (24,109)  (32,364)
         
Share information        
Weighted average number of shares outstanding1  33,355,327   25,282,588   32,440,220   24,255,792 
         
Earnings per share attributable to the equity holders of the Company (expressed in Euro per share)        
Basic loss per share1  (0.18)  (0.42)  (0.74)  (1.33)
Diluted loss per share1  (0.18)  (0.42)  (0.74)  (1.33)
             
  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 all periods. 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
                   
  Attributable to owners of the Company    
                   
        Equity          
        Settled          
        Employee       Non-   
  Number of Share Share Benefit Translation  Accumulated    controlling  Total 
  shares Capital Premium Reserve Reserve  Deficit  Total  interests  Equity 
    € 1,000 € 1,000 € 1,000 € 1,000  € 1,000  € 1,000  € 1,000  € 1,000 
Balance at January 1, 2017  23,346,856  934  123,597  4,353  (15)  (75,733)  53,136   —   53,136 
Result for the period       (32,354) (32,354) (10) (32,364)
Other comprehensive income     114    114    114 
Recognition of share-based payments    3,090     3,090    3,090 
Issue of ordinary shares 2,115,612 85 8,677      8,762    8,762 
Share options exercised 381   1      1    1 
                   
Balance at September 30, 2017  25,462,849  1,019  132,275  7,443  99   (108,087)  32,749   (10)  32,739 
                   
Balance at January 1, 2018  36,425,014  1,457  148,763  8,377  136   (119,370)  39,363   (38)  39,325 
Result for the period       (23,974) (23,974) (135) (24,109)
Other comprehensive income     (15)   (15)   (15)
Recognition of share-based payments    2,245     2,245    2,245 
Issue of ordinary shares 6,612,500 265 84,691       84,956    84,956 
Share options exercised                
                   
Balance at September 30, 2018  43,037,514  1,722  233,454  10,622  121   (143,344)  102,575   (173)  102,402 
                        


 
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows
         
  Three month period
  Nine month period
 
  ended September 30,
  ended September 30,
 
         
  2018 2017 2018 2017
  € 1,000  € 1,000  € 1,000  € 1,000 
Cash flows from operating activities        
Net result (5,992) (10,521) (24,109) (32,364)
Adjustments for:        
— Depreciation 242  267  725  807 
— Share-based compensation 734  890  2,245  3,090 
— Financial income and expenses 74  868  664  2,589 
— Net foreign exchange gain / (loss) (4) 49  (15) 114 
         
Changes in working capital 656  829  1,074  (539)
Cash used in operations  (4,290)  (7,618)  (19,416)  (26,303)
         
Corporate income tax paid 1      (2)
Interest received/(paid) 32  10  25  69 
         
Net cash used in operating activities  (4,257)  (7,608)  (19,391)  (26,236)
         
Cash flow from investing activities        
Purchases of intangible assets        
Purchases of property, plant and equipment (99) (18) (285) (111)
         
Net cash used in investing activities  (99)  (18)  (285)  (111)
         
Cash flow from financing activities        
Proceeds from issuance of shares, net of transaction costs 84,295  5,539  84,295  8,762 
Proceeds from exercise of share options 637    660  1 
Proceeds from borrowings   100  101  201 
Proceeds from convertible loans 115  150  430  150 
         
Net cash generated by financing activities  85,047   5,789   85,486   9,114 
         
Net increase/(decrease) in cash and cash equivalents  80,691   (1,837)  65,810   (17,233)
         
Currency effect cash and cash equivalents 57  (742) (193) (2,225)
Cash and cash equivalents, at beginning of the period 32,968  42,321  48,099  59,200 
         
Cash and cash equivalents at the end of the period  113,716   39,742   113,716   39,742