FORM 6-K

 

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

 

FORM 6-K

 

 

Report of Foreign Private Issuer

Pursuant to Rule 13a-16 or 15d-16

of the Securities Exchange Act of 1934

August 17, 2016

 

 

PROQR THERAPEUTICS N.V.

 

 

Zernikedreef 9

2333 CK Leiden

The Netherlands

Tel: +31 88 166 7000

(Address, Including ZIP Code, and Telephone Number, Including Area Code, of Registrant’s Principal Executive Offices)

 

 

Indicate by check mark whether the registrant files or will file annual reports under cover of Form 20-F or Form 40-F.

Form 20-F  x             Form 40-F  ¨

Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(1):  ¨

Indicate by check mark if the registrant is submitting the Form 6-K in paper as permitted by Regulation S-T Rule 101(b)(7):  ¨

 

 

 


Furnished as Exhibit 99.1 to this Report on Form 6-K are the unaudited financial statements of ProQR Therapeutics N.V. (the “Company”) for the three and six month period ended June 30, 2016 and furnished as Exhibit 99.2 to this Report on Form 6-K is a press release of ProQR Therapeutics N.V. dated August 17, 2016, announcing the Company’s results for the three and six month period ended June 30, 2016.


SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned, thereunto duly authorized.

 

    PROQR THERAPEUTICS N.V.
Date: August 17, 2016     By:  

/s/ Smital Shah

      Smital Shah
      Chief Financial Officer


INDEX TO EXHIBITS

 

Number

  

Description

99.1    Unaudited financial statements of ProQR Therapeutics N.V. for the three and six month period ended June 30, 2016.
99.2    Press Release of ProQR Therapeutics N.V. dated August 17, 2016, announcing the Company’s results for the three and six month period ended June 30, 2016.
EX-99.1

Exhibit 99.1

PROQR THERAPEUTICS N.V.

Index to Unaudited Condensed Consolidated Financial Statements

 

     PAGE  

Unaudited Condensed Consolidated Statement of Financial Position as of June 30, 2016 and December 31, 2015

     1   

Unaudited Condensed Consolidated Statement of Comprehensive Loss for the Three Month and Six Month Periods ended June 30, 2016 and 2015

     2   

Unaudited Condensed Consolidated Statement of Changes in Equity for the Six Month Periods ended June 30, 2016 and 2015

     3   

Unaudited Condensed Consolidated Statement of Cash Flows for the Three Month and Six Month Periods ended June 30, 2016 and 2015

     4   
Notes to Unaudited Condensed Consolidated Financial Statements      5   


PAGE 1

Unaudited Condensed Consolidated Financial Statements

 

PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Financial Position

 

     June 30,
2016
     December 31,
2015
 
   1,000       1,000   

Assets

     

Current assets

     

Cash and cash equivalents

     76,311         94,865   

Prepayments and other receivables

     3,185         1,948   

Social securities and other taxes

     651         956   

Total current assets

     80,147         97,769   

Property, plant and equipment

     3,670         2,199   

Intangible assets

     116         141   

Total assets

     83,933         100,109   

Liabilities and shareholders’ equity

     

Current liabilities

     

Finance lease liabilities

     —           15   

Trade payables

     1,038         885   

Social securities and other taxes

     303         235   

Pension premiums

     43         16   

Deferred income

     —           144   

Other current liabilities

     6,349         4,191   

Total current liabilities

     7,733         5,486   

Borrowings

     5,267         4,824   

Total liabilities

     13,000         10,310   

Shareholders’ equity

     

Shareholders’ equity

     70,933         89,799   

Total liabilities and shareholders’ equity

     83,933         100,109   
  

 

 

    

 

 

 

The notes are an integral part of these condensed consolidated financial statements.

 

PROQR THERAPEUTICS  |  ZERNIKEDREEF 9  |  2333 CK LEIDEN  |    THE NETHERLANDS  |  +31 88 166 7000  |   WWW.PROQR.COM


PAGE 2

Unaudited Condensed Consolidated Financial Statements

 

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 June 30,
    Six month period
ended June 30,
 
     2016     2015     2016     2015  
   1,000      1,000      1,000      1,000   

Other income

     589        748        1,278        1,086   

Research and development costs

     (8,606     (5,427     (15,504     (10,907

General and administrative costs

     (2,615     (1,777     (5,217     (3,380
  

 

 

   

 

 

   

 

 

   

 

 

 

Total operating costs

     (11,221     (7,204     (20,721     (14,287
  

 

 

   

 

 

   

 

 

   

 

 

 

Operating result

     (10,632     (6,456     (19,443     (13,201

Finance income and expense

     673        (2,168     (714     4,812   

Result before corporate income taxes

     (9,959     (8,624     (20,157     (8,389

Income taxes

     —          —          —          —     
  

 

 

   

 

 

   

 

 

   

 

 

 

Net loss attributable to equity holders of the Company

     (9,959     (8,624     (20,157     (8,389

Other comprehensive income

     (5     —          0        —     

Total comprehensive loss (attributable to equity holders of the Company)

     (9,964     (8,624     (20,157     (8,389
  

 

 

   

 

 

   

 

 

   

 

 

 

Share information

        

Weighted average number of shares outstanding1

     23,346,340        23,343,253        23,346,153        23,340,971   

Earnings per share attributable to the equity holders of the Company (expressed in Euro per share)

        

Basic loss per share1

     (0.43     (0.37     (0.86     (0.36

Diluted loss per share1

     (0.43     (0.37     (0.86     (0.36

The notes are an integral part of these condensed consolidated financial statements.

 

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  |  ZERNIKEDREEF 9  |  2333 CK LEIDEN  |    THE NETHERLANDS  |  +31 88 166 7000  |   WWW.PROQR.COM


PAGE 3

Unaudited Condensed Consolidated Financial Statements

 

PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Changes in Equity

 

     Number of
shares
     Total
Share
Capital
     Share
Premium
     Equity
Settled
Employee
Benefit
Reserve
     Translation
Reserve
     Accumulated
Deficit
    Total
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

     —           —           —           —           —           (8,389     (8,389

Recognition of share-based payments

     —           —           —           619         —           —          619   

Share options exercised

     5,217         0         6         —           —           —          6   
  

 

 

    

 

 

    

 

 

    

 

 

    

 

 

    

 

 

   

 

 

 

Balance at June 30, 2015

     23,343,371         934         123,587         1,306         —           (24,187     101,640   

Balance at January 1, 2016

     23,345,965         934         123,595         1,899         1         (36,630     89,799   

Net loss

     —           —           —           —           —           (20,157     (20,157

Other comprehensive income

     —           —           —           —           0         —          0   

Recognition of share-based payments

     —           —           —           1,289         —           —          1,289   

Share options exercised

     891         0         2         —           —           —          2   
  

 

 

    

 

 

    

 

 

    

 

 

    

 

 

    

 

 

   

 

 

 

Balance at June 30, 2016

     23,346,856         934         123,597         3,188         1         (56,787     70,933   
  

 

 

    

 

 

    

 

 

    

 

 

    

 

 

    

 

 

   

 

 

 

The notes are an integral part of these condensed consolidated financial statements.

 

PROQR THERAPEUTICS  |  ZERNIKEDREEF 9  |  2333 CK LEIDEN  |    THE NETHERLANDS  |  +31 88 166 7000  |   WWW.PROQR.COM


PAGE 4

Unaudited Condensed Consolidated Financial Statements

 

PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Cash Flows

 

     Three month period
ended June 30,
    Six month period
ended June 30,
 
     2016     2015     2016     2015  
   1,000      1,000      1,000      1,000   

Cash flows from operating activities

        

Net result

     (9,964     (8,624     (20,157     (8,389

Adjustments for:

        

— Depreciation

     360        117        694        212   

— Share-based compensation

     699        331        1,289        619   

— Financial income and expenses

     (673     2,168        714        (4,812

Changes in working capital

     1,242        (16     1,292        999   
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash used in operations

     (8,336     (6,024     (16,168     (11,371
  

 

 

   

 

 

   

 

 

   

 

 

 

Corporate income tax paid

     —          —          —          —     

Interest received/(paid)

     1        105        66        177   
  

 

 

   

 

 

   

 

 

   

 

 

 

Net cash used in operating activities

     (8,335     (5,919     (16,102     (11,194
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash flow from investing activities

        

Purchases of intangible assets

     —          (28     —          (28

Purchases of property, plant and equipment

     (1,571     (549     (2,073     (1,037
  

 

 

   

 

 

   

 

 

   

 

 

 

Net cash used in investing activities

     (1,571     (577     (2,073     (1,065
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash flow from financing activities

        

Proceeds from exercise of share options

     2        1        2        6   

Proceeds from borrowings

     —          1,254        193        1,254   

Redemption of financial lease

     (7     (8     (15     (20
  

 

 

   

 

 

   

 

 

   

 

 

 

Net cash generated by financing activities

     (5     1,247        180        1,240   
  

 

 

   

 

 

   

 

 

   

 

 

 

Net increase/(decrease) in cash and cash equivalents

     (9,911     (5,249     (17,995     (11,019

Currency effect cash and cash equivalents

     755        (2,184     (559     4,665   

Cash and cash equivalents, at beginning of the period

     85,467        113,815        94,865        112,736   
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash and cash equivalents at the end of the period

     76,311        106,382        76,311        106,382   
  

 

 

   

 

 

   

 

 

   

 

 

 

The notes are an integral part of these condensed consolidated financial statements.

 

PROQR THERAPEUTICS  |  ZERNIKEDREEF 9  |  2333 CK LEIDEN  |    THE NETHERLANDS  |  +31 88 166 7000  |   WWW.PROQR.COM


PAGE 5

Unaudited Condensed Consolidated Financial Statements

 

PROQR THERAPEUTICS N.V.

Notes to Unaudited Condensed Consolidated Financial Statements

1. General information

ProQR Therapeutics N.V., or “ProQR” or the “Company”, is a development stage company that primarily focuses on the development and commercialization of novel therapeutic medicines.

Since September 18, 2014, the Company’s ordinary shares are listed on the NASDAQ Global Market under ticker symbol PRQR.

The Company was incorporated in the Netherlands, on February 21, 2012 and has been reorganized from a private company with limited liability to a public company with limited liability on September 23, 2014. The Company has its statutory seat in Leiden, the Netherlands. The address of its headquarters and registered office is Zernikedreef 9, 2333 CK Leiden, the Netherlands.

ProQR Therapeutics N.V. is the ultimate parent company of the following entities:

 

    ProQR Therapeutics Holding B.V. (100%);

 

    ProQR Therapeutics I B.V. (100%);

 

    ProQR Therapeutics II B.V. (100%);

 

    ProQR Therapeutics III B.V. (100%);

 

    ProQR Therapeutics IV B.V. (100%);

 

    ProQR Therapeutics VI B.V. (100%);

 

    ProQR Therapeutics VII B.V. (100%);

 

    ProQR Therapeutics I Inc. (100%).

As used in these condensed consolidated financial statements, unless the context indicates otherwise, all references to “ProQR” or the “Company” refer to ProQR Therapeutics N.V. including its subsidiaries.

2. Significant Accounting Policies

These condensed consolidated financial statements have been prepared in accordance with International Financial Reporting Standards (“IFRS”), as issued by the International Accounting Standards Board (“IASB”), in particular IAS 34 - Interim Financial Reporting. Certain information and disclosures normally included in financial statements prepared in accordance with IFRS have been condensed or omitted. Accordingly, these condensed consolidated financial statements should be read in conjunction with the Company’s annual financial statements for the year ended December 31, 2015. In the opinion of management, all adjustments, consisting of normal recurring nature, considered necessary for a fair presentation have been included in the condensed consolidated financial statements.

The Company’s financial results have varied substantially, and are expected to continue to vary, from period to period. The Company believes that its ordinary activities are not linked to any particular seasonal factors.

The Company operates in one reportable segment, which comprises the discovery and development of innovative, RNA based therapeutics.

3. Adoption of new and revised International Financial Reporting Standards

The accounting policies adopted in the preparation of the condensed consolidated financial statements are consistent with those applied in the preparation of the Company’s annual financial statements for the year ended December 31, 2015. New Standards and Interpretations, which became effective as of January 1, 2016, did not have a material impact on our condensed consolidated financial statements.

 

PROQR THERAPEUTICS  |  ZERNIKEDREEF 9  |  2333 CK LEIDEN  |    THE NETHERLANDS  |  +31 88 166 7000  |   WWW.PROQR.COM


PAGE 6

Unaudited Condensed Consolidated Financial Statements

 

4. Critical Accounting Estimates and Judgments

In the application of the Company’s accounting policies, management is required to make judgments, estimates and assumptions about the carrying amounts of assets and liabilities that are not readily apparent from other sources. The estimates and associated assumptions are based on historical experience and other factors that are considered to be relevant. Actual results may differ from these estimates.

The estimates and underlying assumptions are reviewed on an ongoing basis. Revisions to accounting estimates are recognized in the period in which the estimate is revised if the revision affects only that period or in the period of the revision and future periods if the revision affects both current and future periods.

(a) Share-based payments

Share options granted to employees and consultants are measured at the fair value of the equity instruments granted. Fair value is determined through the use of the Black-Scholes option-pricing model, which is considered the most appropriate model for this purpose by management.

Initially, the Company’s ordinary shares were not publicly traded and consequently the Company needed to estimate the fair value of its share and the expected volatility of that value. Please refer to the Company’s annual financial statements for the year ended December 31, 2015 for the assumptions used in those estimates. The value of the underlying shares was determined on the basis of the prior sale of company stock method. As such, the Company has benchmarked the value per share to external transactions of Company shares and external financing rounds.

For options granted from the moment of listing, the Company uses the closing price of the ordinary shares on the previous business day as exercise price of the options granted.

The result of the share option valuations and the related compensation expense is dependent on the model and input parameters used. Even though Management considers the fair values reasonable and defensible based on the methodologies applied and the information available, others might derive a different fair value for the Company’s share options.

(b) Corporate income taxes

The Company recognizes deferred tax assets arising from unused tax losses or tax credits only to the extent that the Company has sufficient taxable temporary differences or there is convincing evidence that sufficient taxable profit will be available against which the unused tax losses or unused tax credits can be utilized. Management’s judgment is that such convincing evidence is currently not sufficiently available and a deferred tax asset is therefore only recognized to the extent that the Company has sufficient taxable temporary differences.

(c) Grant income

Grants (to be) received are reflected in the balance sheet as other receivables or deferred income. At each balance sheet date, for grants approved, the Company estimates the associated costs incurred, the level of service performed and the progress of the associated projects. Based on this analysis grant income is recognized.

 

PROQR THERAPEUTICS  |  ZERNIKEDREEF 9  |  2333 CK LEIDEN  |    THE NETHERLANDS  |  +31 88 166 7000  |   WWW.PROQR.COM


PAGE 7

Unaudited Condensed Consolidated Financial Statements

 

(d) Research and development expenditures

Research expenditures are currently not capitalized but are reflected in the income statement because the criteria for capitalization are not met. At each balance sheet date, the Company estimates the level of service performed by the vendors and the associated costs incurred for the services performed.

Although we do not expect the estimates to be materially different from amounts actually incurred, the understanding of the status and timing of services performed relative to the actual status and timing of services performed may vary and could result in reporting amounts that are too high or too low in any particular period.

The condensed consolidated financial statements do not include all disclosures for critical accounting estimates and judgments that are required in the annual consolidated financial statements and should be read in conjunction with the Company’s annual financial statements for the year ended December 31, 2015.

5. Cash and Cash Equivalents

At June 30, 2016, the Company’s cash and equivalents were € 76,311,000 as compared to € 94,865,000 at December 31, 2015. A significant portion of the cash balance is denominated in US dollars. The cash balances are held at banks with investment grade credit ratings. The cash at banks is at full disposal of the Company.

6. Current liabilities

At June 30, 2016 and December 31, 2015, the other current liabilities consisted principally of accruals for services provided by vendors not yet billed and other miscellaneous liabilities. The accrued liabilities as at June 30, 2016 increased compared to December 31, 2015 as a result of the increased level of research and development activities.

7. Borrowings

 

     June 30,
2016
     December 31,
2015
 
   1,000       1,000   

Innovation credit

     4,421         4,228   

Accrued interest on innovation credit

     846         596   

Total borrowings

     5,267         4,824   
  

 

 

    

 

 

 

Innovation credit (“Innovatiekrediet”)

On June 1, 2012, ProQR was awarded an Innovation credit by the Dutch government, through its agency RVO (previously: “AgentschapNL”) of the Ministry of Economic Affairs, for the Company’s cystic fibrosis program. The credit was increased in the course of 2013 through 2016. The credit covers 35% of the costs incurred in respect of the program up to an initial maximum of € 5.0 million through December 31, 2016.

The credit is interest-bearing at a rate of 10% per annum. The credit, including accrued interest, is repayable in three instalments on August 31, 2017, August 31, 2018 and August 31, 2019, depending on the technical success of the program.

The assets which are co-financed with the granted innovation credit are subject to a right of pledge for the benefit of RVO.

 

PROQR THERAPEUTICS  |  ZERNIKEDREEF 9  |  2333 CK LEIDEN  |    THE NETHERLANDS  |  +31 88 166 7000  |   WWW.PROQR.COM


PAGE 8

Unaudited Condensed Consolidated Financial Statements

 

8. Shareholders’ equity

The authorized share capital of the Company amounting to € 934,000 consists of 23,346,856 ordinary shares with a nominal value of € 0.04 per share. All issued shares have been fully paid in cash.

Translation reserve

The translation reserve comprises all foreign currency differences arising from the translation of the financial statements of foreign operations.

Share options

The Company operates an equity-settled share-based compensation plan which was introduced in 2013. The supervisory board may grant options to employees, members of the supervisory board, members of the management board and consultants. The quarterly compensation expenses included in operating costs for this plan in Q2 2016 were € 699,000 (Q2 2015: € 331,000), of which € 459,000 (Q2 2015: € 227,000) was recorded in general and administrative costs and € 240,000 (Q2 2015: € 104,000) was recorded in research and development costs.

9. Other income

 

     Three month period
ended June 30, 2016
     Three month period
ended June 30, 2015
 
   1,000       1,000   

Grant income

     544         748   

Rental income from property subleases

     45         —     
     589         748   
  

 

 

    

 

 

 

In August 2014, the Company entered into an agreement with Cystic Fibrosis Foundation Therapeutics, Inc., or CFFT, a subsidiary of the Cystic Fibrosis Foundation, pursuant to which CFFT agreed to provide the Company with up to $ 3 million to support the clinical development of QR-010.

In 2015, the European Commission (EC) through its Horizon 2020 program awarded ProQR and its academic partners a grant of € 6 million (ProQR: € 4.4 million) to support the clinical development of QR-010 in the period up till December 31, 2017. 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.

Both grants are recognized in other income in the same period in which the related R&D costs are recognized.

10. Research and development costs

Research and development costs increased to € 8,606,000 for the quarter ended June 30, 2016 from € 5,427,000 for the same period in 2015 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 advancement of our pipeline, which included clinical development of QR-010, preclinical development of QR-110 and progress of our innovation programs such as QRX-313 for Epidermolysis bullosa and QRX-411 for Usher syndrome, as well as other programs in ophthalmology, neuromuscular and central nervous system (CNS) diseases.

 

PROQR THERAPEUTICS  |  ZERNIKEDREEF 9  |  2333 CK LEIDEN  |    THE NETHERLANDS  |  +31 88 166 7000  |   WWW.PROQR.COM


PAGE 9

Unaudited Condensed Consolidated Financial Statements

 

11. General and administrative costs

General and administrative costs amount to € 2,615,000 for the quarter ended June 30, 2016 compared to € 1,777,000 for the same period in 2015 primarily due to increased investments in our facilities and our support organization.

12. Income taxes

Due to the operating losses incurred since inception the Company has no tax provisions as of the balance sheet date. Furthermore, no significant temporary differences exist between accounting and tax results. Realization of deferred tax assets is dependent on future earnings, if any, the timing and amount of which are uncertain. Accordingly, the Company has not yet recognized any deferred tax asset related to operating losses.

13. Events after balance sheet date

No significant events have occurred after balance sheet date.

 

PROQR THERAPEUTICS  |  ZERNIKEDREEF 9  |  2333 CK LEIDEN  |    THE NETHERLANDS  |  +31 88 166 7000  |   WWW.PROQR.COM

EX-99.2

Exhibit 99.2

 

ProQR Therapeutics N.V.

Press Release August 17, 2016

   LOGO

FINAL – FOR RELEASE

ProQR Announces Results for the Second Quarter of 2016

LEIDEN, the Netherlands, August 17, 2016 — 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 second quarter of 2016.

“We continue to make good progress with our three different development programs, all RNA oligonucleotides for serious genetic diseases. For QR-010 for patients with CF due to the DF508 mutation, we will present top-level results from clinical proof-of-concept study, PQ-010-002 later this year during the North American Cystic Fibrosis Conference (NACFC).”, said Daniel de Boer, Chief Executive Officer of ProQR. “For QR-110 for patients with LCA10, we presented strong pre-clinical proof-of-concept data at the Association for Research in Vision and Ophthalmology (ARVO) conference. We plan to move this program towards the clinic this year. For QR-313 for dystrophic epidermolysis bullosa (DEB) patients due to mutations in Exon 73, we continue to strengthen the pre-clinical proof-of-concept in important clinically relevant models. In addition to our three molecules in development, we are strengthening our pipeline of RNA oligonucleotides for severe genetic diseases through our innovation unit.”

Financial Highlights

At June 30, 2016, ProQR held cash and cash equivalents of €76.3 million, compared to €85.5 million at March 31, 2016. Net cash used in operating activities during the three month period ended June 30, 2016 was €8.3 million, compared to €5.9 million for the same period last year.

Research and development costs increased to €8.6 million for the quarter ended June 30, 2016 from €5.4 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 advancement of our pipeline, which included clinical development of QR-010, preclinical development of QR-110 and progress of our innovation programs such as QR-313 for epidermolysis bullosa, which moved into preclinical development this quarter and other pipeline programs.

General and administrative costs increased to €2.6 million for the quarter ended June 30, 2016 from €1.8 million for the same period last year, in line with the growth of our organization.

Net result for the three month period ended June 30, 2016 was a €10.0 million loss or €0.43 per share, compared to a €8.6 million loss or €0.37 per share for the same period last year. For further financial information for the period ending June 30, 2016, please refer to the financial statements appearing at the end of this release.

Corporate Highlights

 

    QR-010 for patients with CF due to the DF508 mutation is being studied in two global clinical trials. PQ-010-002 is a proof-of-concept study evaluating the effect of QR-010 on an important measurement of CFTR function, the nasal potential difference (NPD). PQ-010-002 is an open label 28-day study of topical exposure of QR-010 in 16 CF patients, 8 homozygous (carrying two copies) for the DF508 mutation and 8 compound heterozygous (one copy of the DF508 plus one other CF disease causing mutation) with the option to enroll an additional 16. Top-line data from the first 16 patients is expected to be presented during the NACFC, Orlando, Florida, October 27 - 29, 2016. The other study, PQ-010-001 is a Phase 1b randomized, double-blind, placebo-controlled, dose-escalation 28-day study. The safety, tolerability and pharmacokinetics of single and multiple ascending doses of inhaled QR-010 will be evaluated in 64 CF patients carrying two copies (homozygotes) of the DF508 mutation. In addition, exploratory efficacy endpoints in this study include sweat chloride, weight gain, CFQ-R Respiratory Symptom Score and lung function, measured by FEV1. This study is not powered for statistical significance on any of these exploratory endpoints. The company expects to present preliminary safety data from the single ascending dose cohorts at the same time as results for study PQ-010-002.

 

ProQR Therapeutics N.V. | Zernikedreef 9, 2333 CK Leiden, The Netherlands | +31 88 166 7000 | info@proqr.com | www.proqr.com


    At the 2016 European Cystic Fibrosis Conference (ECFS, June 10 2016) the company presented additional pre-clinical data for QR-010 in a presentation titled “QR-010 penetrates the CF-like mucus barrier in vitro and in vivo.” QR-010 was shown to diffuse rapidly through CF-like mucus layers in in vitro and in vivo models. QR-010 was also shown to be stable in CF sputum, in the presence of CF lung bacteria and in the presence of clinically relevant levels of CF standard-of-care.

 

    QR-110, for patients with LCA10, due to the p.Cys998X mutation received orphan drug designation (ODD) from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). ODD in the U.S. and the European Union confers a special status for investigational drugs that are being developed for rare diseases.

 

    ProQR strengthened its Supervisory Board with the appointment of James Shannon, MD in June 2016. James was the former Chief Medical Officer at GlaxoSmithKline and Global Head of Pharma Development at Novartis. We believe that James’ broad knowledge and expertise in drug development and pharma will be of significant value to the Supervisory Board.

 

    The company moved headquarters to Zernikedreef 9, 2333 CK Leiden, the Netherlands.

Subsequent events

 

    The company has moved its product candidate for the third program, QR-313 (previously named QRX-313) into pre-clinical development for the treatment of dystrophic epidermolysis bullosa (DEB). QR-313 is an RNA oligonucleotide designed to induce the exclusion of a part of the RNA (exon skipping) that contains a disease causing mutation with the aim to restore functional COL7a1 protein and with that the anchoring fibrils that bind the layers of skin together. QR-313 is the second program to be added to the pipeline behind the CF and LCA10 programs from ProQR’s internal innovation (discovery) unit.

 

    QR-010 received a Fast Track designation by the US Food and Drug Administration (FDA). Drugs that are under development for serious conditions and have the potential to fulfill an unmet medical need can receive this designation. It was established with the intention to bring promising drugs to patients sooner by facilitating the development with more frequent FDA interactions and expediting the review process.

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 in CF patients that have the DF508 mutation. 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 restore CFTR function. QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a small, handheld aerosol delivery device which nebulizes QR-010 into a mist inhaled directly into the lungs. We believe this route of administration 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 RNA-based 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 and has been granted orphan drug designation in the United States and the European Union.

About QR-313

QR-313 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in this exon can cause loss of functional collagen type VII (C7) protein. Absence of C7 results in the loss of anchoring fibrils that normally link the dermal and epidermal layers of the skin together. QR-313 is designed to exclude exon 73 from the mRNA (exon skipping) and produce truncated but functional C7 protein and thereby restores functionality of the anchoring fibrils.

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 QR-010, QR-110 and QR-313, and the clinical development and the therapeutic potential thereof, statements regarding our ongoing and planned discovery and development of product candidates, including those in our innovation pipeline, statements regarding release of clinical data, statements regarding the appointment of Dr. Shannon, and statements regarding the Horizon 2020 program. 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, except as required by law.

ProQR Therapeutics N.V.:

Sariette Witte

Head of Communications

T: +1 213 261 8891

ir@proqr.com


PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Financial Position

 

     June 30,
2016
     December 31,
2015
 
   1,000       1,000   

Assets

     

Current assets

     

Cash and cash equivalents

     76,311         94,865   

Prepayments and other receivables

     3,185         1,948   

Social securities and other taxes

     651         956   

Total current assets

     80,147         97,769   

Property, plant and equipment

     3,670         2,199   

Intangible assets

     116         141   

Total assets

     83,933         100,109   

Liabilities and shareholders’ equity

     

Current liabilities

     

Finance lease liabilities

     —           15   

Trade payables

     1,038         885   

Social securities and other taxes

     303         235   

Pension premiums

     43         16   

Deferred income

     —           144   

Other current liabilities

     6,349         4,191   

Total current liabilities

     7,733         5,486   

Borrowings

     5,267         4,824   

Total liabilities

     13,000         10,310   

Shareholders’ equity

     

Shareholders’ equity

     70,933         89,799   

Total liabilities and shareholders’ equity

     83,933         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 June 30,
    Six month period
ended June 30,
 
     2016     2015     2016     2015  
   1,000      1,000      1,000      1,000   

Other income

     589        748        1,278        1,086   

Research and development costs

     (8,606     (5,427     (15,504     (10,907

General and administrative costs

     (2,615     (1,777     (5,217     (3,380
  

 

 

   

 

 

   

 

 

   

 

 

 

Total operating costs

     (11,221     (7,204     (20,721     (14,287
  

 

 

   

 

 

   

 

 

   

 

 

 

Operating result

     (10,632     (6,456     (19,443     (13,201

Finance income and expense

     673        (2,168     (714     4,812   

Result before corporate income taxes

     (9,959     (8,624     (20,157     (8,389

Income taxes

     —          —          —          —     
  

 

 

   

 

 

   

 

 

   

 

 

 

Net loss attributable to equity holders of the Company

     (9,959     (8,624     (20,157     (8,389

Other comprehensive income

     (5     —          0        —     

Total comprehensive loss (attributable to equity holders of the Company)

     (9,964     (8,624     (20,157     (8,389
  

 

 

   

 

 

   

 

 

   

 

 

 

Share information

        

Weighted average number of shares outstanding1

     23,346,340        23,343,253        23,346,153        23,340,971   

Earnings per share attributable to the equity holders of the Company (expressed in Euro per share)

        

Basic loss per share1

     (0.43     (0.37     (0.86     (0.36

Diluted loss per share1

     (0.43     (0.37     (0.86     (0.36

 

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

 

     Number of
shares
     Total
Share
Capital
     Share
Premium
     Equity
Settled
Employee
Benefit
Reserve
     Translation
Reserve
     Accumulated
Deficit
    Total
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

     —           —           —           —           —           (8,389     (8,389

Recognition of share-based payments

     —           —           —           619         —           —          619   

Share options exercised

     5,217         0         6         —           —           —          6   
  

 

 

    

 

 

    

 

 

    

 

 

    

 

 

    

 

 

   

 

 

 

Balance at June 30, 2015

     23,343,371         934         123,587         1,306         —           (24,187     101,640   

Balance at January 1, 2016

     23,345,965         934         123,595         1,899         1         (36,630     89,799   

Net loss

     —           —           —           —           —           (20,157     (20,157

Other comprehensive income

     —           —           —           —           0         —          0   

Recognition of share-based payments

     —           —           —           1,289         —           —          1,289   

Share options exercised

     891         0         2         —           —           —          2   
  

 

 

    

 

 

    

 

 

    

 

 

    

 

 

    

 

 

   

 

 

 

Balance at June 30, 2016

     23,346,856         934         123,597         3,188         1         (56,787     70,933   
  

 

 

    

 

 

    

 

 

    

 

 

    

 

 

    

 

 

   

 

 

 


PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Cash Flows

 

     Three month period
ended June 30,
    Six month period
ended June 30,
 
     2016     2015     2016     2015  
   1,000      1,000      1,000      1,000   

Cash flows from operating activities

        

Net result

     (9,964     (8,624     (20,157     (8,389

Adjustments for:

        

— Depreciation

     360        117        694        212   

— Share-based compensation

     699        331        1,289        619   

— Financial income and expenses

     (673     2,168        714        (4,812

Changes in working capital

     1,242        (16     1,292        999   
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash used in operations

     (8,336     (6,024     (16,168     (11,371
  

 

 

   

 

 

   

 

 

   

 

 

 

Corporate income tax paid

     —          —          —          —     

Interest received/(paid)

     1        105        66        177   
  

 

 

   

 

 

   

 

 

   

 

 

 

Net cash used in operating activities

     (8,335     (5,919     (16,102     (11,194
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash flow from investing activities

        

Purchases of intangible assets

     —          (28     —          (28

Purchases of property, plant and equipment

     (1,571     (549     (2,073     (1,037
  

 

 

   

 

 

   

 

 

   

 

 

 

Net cash used in investing activities

     (1,571     (577     (2,073     (1,065
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash flow from financing activities

        

Proceeds from exercise of share options

     2        1        2        6   

Proceeds from borrowings

     —          1,254        193        1,254   

Redemption of financial lease

     (7     (8     (15     (20
  

 

 

   

 

 

   

 

 

   

 

 

 

Net cash generated by financing activities

     (5     1,247        180        1,240   
  

 

 

   

 

 

   

 

 

   

 

 

 

Net increase/(decrease) in cash and cash equivalents

     (9,911     (5,249     (17,995     (11,019

Currency effect cash and cash equivalents

     755        (2,184     (559     4,665   

Cash and cash equivalents, at beginning of the period

     85,467        113,815        94,865        112,736   
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash and cash equivalents at the end of the period

     76,311        106,382        76,311        106,382