ProQR Therapeutics Announces Results for the First Quarter of 2015

LEIDEN, the Netherlands, May 21, 2015 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe diseases such as cystic fibrosis and Leber's congenital amaurosis, today announced results for the first quarter of 2015.

"The first quarter was one in a year that is going to be a very exciting one for ProQR," said Daniel de Boer, Chief Executive Officer of ProQR. "We advanced our first pipeline program, QR-110, into preclinical development for Leber's congenital amaurosis, the most common genetic blindness, which leads to vision loss in very young children. In addition, we have made a lot of progress towards initiating our clinical studies for QR-010 in patients with cystic fibrosis due to the ΔF508 mutation and are on track to dose a first patient in this second quarter of 2015."

Financial Highlights

As of March 31, 2015, we held cash and cash equivalents of €113.8 million, compared to €112.7 million at December 31, 2014. The net increase in cash and cash equivalents is mainly the result of a foreign exchange gain of €6.8 million. Net cash used in operating activities during the three month period ended March 31, 2015 was €5.3 million, compared to €1.9 million for the same period last year.

Research and development costs were €5.5 million for the three month period ended March 31, 2015, compared to €1.3 million for the same period in 2014. The increase reflects the expansion of research and development activities to support the further development of our lead product candidate, QR-010, for the treatment of cystic fibrosis, as well as the development of our other pipeline product candidates, including QR-110 for the treatment of Leber's congenital amaurosis, the leading genetic cause of childhood blindness.

General and administrative costs were €1.6 million for the three month period March 31, 2015, compared to €0.6 million for the same period in 2014. The increase is related to the expansion of the Company through 2014.

Net result for the three month period ended March 31, 2015 was a €0.2 million profit or €0.01 per share, compared to a €1.9 million loss or €0.30 per share for the same period in 2014. For further financial information for the period ending March 31, 2015, please refer to the financial statements appearing at the end of this release.

Corporate Highlights

  • To further strengthen the capabilities of our Supervisory Board, the Supervisory Board nominated Paul Baart as a member and the Chair of the Audit Committee. Paul brings significant experience to the Supervisory Board and Audit Committee through his extensive career in public accounting in both the Netherlands and the USA. At PwC Netherlands he served on the Management and Supervisory Boards and was also a member of the Global Board of PwC International. While at PwC, Paul's clients included several large private and public multinational companies across a variety of industries. His appointment is subject to approval of the Annual General Meeting of Shareholders in June 2015.

Subsequent Events

  • The European Committee (EC) granted ProQR and our academic partners a grant of €6 million under the Horizon 2020 program to finance the Phase Ib and PoC clinical studies of QR-010. Horizon 2020 is one of the largest research and innovation programs in the European Union with nearly €80 billion in available funding for qualified projects from 2014 to 2020. Under the Horizon 2020, the EC works closely with the European Research Council, the European Institute of Innovation and Technology and EURATOM and evaluates projects on three key pillars of Excellent Science, Industrial Leadership and Societal Challenges.
  • We have selected the product candidate in our second program QR-110 for the treatment of Leber's congenital amaurosis (LCA), the most common genetic blindness in childhood. QR-110, a first-in-class oligonucleotide, is designed to treat the disease by repairing the underlying cause in the mRNA, which results in the production of wild-type CEP290 protein. In the first half of 2016 we intend to start our first clinical trial for QR-110 in patients suffering from the most common mutation in LCA, the p.Cys998X mutation, also known as c.2991+1655A>G mutation in the CEP290 gene.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe 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.


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 initiation of clinical trials of QR-010 and QR-110 and statements regarding our ongoing and planned discovery and development of 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 clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and 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. 

ProQR Therapeutics N.V.:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431

Media inquiries:
Gretchen Schweitzer
MacDougall Biomedical Communications
Direct: +49 172 861 8540
Main: +49 89 2424 3494

Unaudited Condensed Statement of Financial Position
(€ in thousands)
  March 31, 
December 31, 
Current assets    
Cash and cash equivalents 113,815 € 112,736
Prepayments and other receivables 1,551 735
Social securities and other taxes 323 426
Total current assets 115,689 113,897
Property, plant and equipment 1,747 1,187
Intangible assets 152 163
Total assets 117,588 115,247
Liabilities and shareholders' equity    
Current liabilities    
Finance lease liabilities 30 34
Trade payables 2,412 1,247
Social securities and other taxes 274 341
Pension premiums 130 127
Other current liabilities 1,918 1,265
Total current liabilities 4,764 3,014
Finance lease liabilities 7 15
Borrowings 2,885 2,814
Total liabilities 7,656 5,843
Shareholders' equity    
Shareholders' equity 109,932 109,404
Total liabilities and shareholders' equity 117,588 € 115,247
Unaudited Condensed Statement of Profit or Loss and OCI 
(€ in thousands, except share and per share data) 
  2015 2014
Other income 338 4
Research and development costs (5,480) (1,265)
General and administrative costs (1,603) (595)
Total operating costs (7,083) (1,860)
Operating result (6,745) (1,856)
Finance income and expense 6,980 (63)
Result before corporate income taxes 235 (1,919)
Income taxes -- --
Net profit (loss) attributable to equity holders of the Company 235 (1,919)
Other comprehensive income -- --
Total comprehensive profit (loss) (attributable to equity holders of the Company) 235 (1,919)
Share information    
Weighted average number of shares outstanding 1 - basic 23,338,663 6,467,419
Weighted average number of shares outstanding 1 - diluted 24,378,072 6,467,419
Earnings per share attributable to the equity holders of the Company (expressed in Euro per share) 0.01 (0.30)
Basic profit (loss) per share 0.01 (0.30) 1
Diluted profit (loss) per share 0.01 (0.30) 1
1 For the comparative figures included in these financial statements, the potential exercise of share options and the conversion of preferred shares into ordinary shares are not included in the diluted earnings per share calculation as the Company was loss-making in that period. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal in that period.
Unaudited Condensed Statement of Changes in Equity
(€ in thousands, except share data)
  Number of shares Total
Total Equity
  Ordinary Preferred          
Balance at January 1, 2014 6,108,152 59 3,482 41 (3,671) (89)
Net loss -- -- -- -- -- (1,919) (1,919)
Recognition of share-based payments -- -- -- -- 104 -- 104
Shares issued in the period -- -- -- -- -- -- --
Treasury shares issued -- -- -- -- -- -- --
Balance at March 31, 2014 6,108,152 -- 59 3,482 145 (5,590) (1,904)
Balance at January 1, 2015 23,338,154 € 934 € 123,581 € 687 € (15,798) € 109,404
Net profit (loss)   -- -- -- -- 235 235
Recognition of share-based payments -- -- -- -- 288 -- 288
Shares options exercised 5,090  -- 0 5 -- -- 5
Balance at March 31, 2015 23,343,244 -- 934 123,586 975 (15,563) 109,932
Unaudited Condensed Statement of Cash Flows
(€ in thousands)
  2015 2014
Cash flows from operating activities    
Net profit (loss) 235 (1,919)
Adjustments for:    
— Depreciation 95 17
— Share-based compensation 288 104
— Financial income and expenses (6,980) 63
Changes in working capital 1,015 (199)
Cash used in operations (5,347) (1,934)
Corporate income tax paid -- --
Interest received/(paid) 72 (2)
Net cash used in operating activities (5,275) (1,936)
Cash flow from investing activities    
Purchases of property, plant and equipment (488) (156)
Purchases of intangible assets -- --
Net cash used in investing activities (488) (156)
Cash flow from financing activities    
Proceeds from exercise of share options 5 --
Proceeds from borrowings -- --
Redemption of financial lease (12) (15)
Net cash used in financing activities (7) (15)
Net increase in cash and cash equivalents (5,770) (2,107)
Currency effect cash and cash equivalents 6,849 --
Cash and cash equivalents, at beginning of the period 112,736 4,129
Cash and cash equivalents at the end of the period 113,815 2,022

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