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

February 28, 2018

 

 

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  ☒             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):  ☐

Attached 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 year ended December 31, 2017 and attached as Exhibit 99.2 to this Report on Form 6-K is a press release of ProQR Therapeutics N.V. dated February 28, 2018, announcing the Company’s results for the three months and year ended December 31, 2017.

The Company hereby incorporates by reference the information contained herein into the Company’s registration statement on Form F-3 (File No. 333-207245).

 

 

 


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: February 28, 2018     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 months and year ended December 31, 2017.
99.2    Press Release of ProQR Therapeutics N.V. dated February 28, 2018, announcing the Company’s results for the three months and year ended December 31, 2017.
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 at December 31, 2017 and December 31, 2016

     1  

Unaudited Condensed Consolidated Statement of Comprehensive Loss for the Years and Three Month Periods ended December 31, 2017 and 2016

     2  

Unaudited Condensed Consolidated Statement of Changes in Equity for the Years ended December 31, 2017 and 2016

     3  

Unaudited Condensed Consolidated Statement of Cash Flows for the Years and Three Month Periods ended December 31, 2017 and 2016

     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

 

     December 31,
2017
    December 31,
2016
 
   1,000     1,000  

Assets

    

Current assets

    

Cash and cash equivalents

     48,099       59,200  

Prepayments and other receivables

     2,064       2,420  

Social securities and other taxes

     396       395  
  

 

 

   

 

 

 

Total current assets

     50,559       62,015  
  

 

 

   

 

 

 

Property, plant and equipment

     2,505       3,438  

Intangible assets

     39       90  
  

 

 

   

 

 

 

Total assets

     53,103       65,543  
  

 

 

   

 

 

 

Equity and liabilities

    

Equity

    

Equity attributable to owners of the Company

     39,363       53,136  

Non-controlling interests

     (38     —    
  

 

 

   

 

 

 

Total equity

     39,325       53,136  
  

 

 

   

 

 

 

Current liabilities

    

Borrowings

     1,960       —    

Trade payables

     546       328  

Social securities and other taxes

     1,019       312  

Pension premiums

     —         13  

Deferred income

     347       —    

Other current liabilities

     4,622       6,057  

Total current liabilities

     8,494       6,710  

Borrowings

     5,284       5,697  
  

 

 

   

 

 

 

Total liabilities

     13,778       12,407  
  

 

 

   

 

 

 

Total equity and liabilities

     53,103       65,543  
  

 

 

   

 

 

 

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 December 31,
    Year ended December 31,  
     2017     2016     2017     2016  
   1,000     1,000     1,000     1,000  

Other income

     511       103       1,495       1,828  

Research and development costs

     (8,345     (8,100     (31,153     (31,923

General and administrative costs

     (2,891     (2,260     (10,840     (9,478
  

 

 

   

 

 

   

 

 

   

 

 

 

Total operating costs

     (11,236     (10,360     (41,993     (41,401
  

 

 

   

 

 

   

 

 

   

 

 

 

Operating result

     (10,725     (10,257     (40,498     (39,573

Finance income and expense

     (586     1,438       (3,175     470  

Result before corporate income taxes

     (11,311     (8,819     (43,673     (39,103

Income taxes

     —         —         (2     —    

Result for the period

     (11,311     (8,819     (43,675     (39,103

Other comprehensive income

     37       (16     151       (16
  

 

 

   

 

 

   

 

 

   

 

 

 

Total comprehensive income (attributable to owners of the Company)

     (11,274     (8,835     (43,524     (39,119
  

 

 

   

 

 

   

 

 

   

 

 

 

Result attributable to

        

Owners of the Company

     (11,283     (8,819     (43,637     (39,103

Non-controlling interests

     (28     —         (38     —    
  

 

 

   

 

 

   

 

 

   

 

 

 
     (11,311     (8,819     (43,675     (39,103
  

 

 

   

 

 

   

 

 

   

 

 

 

Share information

        

Weighted average number of shares outstanding1

     28,695,362       23,346,856       25,374,807       23,346,507  

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

        

Basic loss per share1

     (0.39     (0.38     (1.72     (1.67

Diluted loss per share1

     (0.39     (0.38     (1.72     (1.67

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


PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Changes in Equity

 

     Attributable to owners of the Company              
     Number of
shares
     Share
Capital
     Share
Premium
    Equity
Settled
Employee
Benefit
Reserve
     Translation
Reserve
    Accumulated
Deficit
    Total     Non-controlling
interests
    Total
Equity
 
                     
      1,000      1,000     1,000      1,000     1,000     1,000     1,000     1,000  

Balance at January 1, 2016

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

 

 

    

 

 

    

 

 

   

 

 

    

 

 

   

 

 

   

 

 

   

 

 

   

 

 

 

Result for the period

     —          —          —         —          —         (39,103     (39,103     —         (39,103

Other comprehensive income

     —          —          —         —          (16     —         (16     —         (16

Recognition of share-based payments

     —          —          —         2,454        —         —         2,454       —         2,454  

Share options exercised

     891        0        2       —          —         —         2       —         2  
  

 

 

    

 

 

    

 

 

   

 

 

    

 

 

   

 

 

   

 

 

   

 

 

   

 

 

 

Balance at December 31, 2016

     23,346,856        934        123,597       4,353        (15     (75,733     53,136       —         53,136  
  

 

 

    

 

 

    

 

 

   

 

 

    

 

 

   

 

 

   

 

 

   

 

 

   

 

 

 

Result for the period

     —          —          —         —          —         (43,637     (43,637     (38     (43,675

Other comprehensive income

     —          —          —         —          151       —         151       —         151  

Recognition of share-based payments

     —          —          —         4,024        —         —         4,024       —         4,024  

Issue of ordinary shares

     8,573,975        343        25,342       —          —         —         25,685       —         25,685  

Issue of treasury shares

     4,503,149        180        (180     —          —         —         0       —         0  

Share options exercised

     1,034        0        4       —          —         —         4       —         4  
  

 

 

    

 

 

    

 

 

   

 

 

    

 

 

   

 

 

   

 

 

   

 

 

   

 

 

 

Balance at December 31, 2017

     36,425,014        1,457        148,763       8,377        136       (119,370     39,363       (38     39,325  
  

 

 

    

 

 

    

 

 

   

 

 

    

 

 

   

 

 

   

 

 

   

 

 

   

 

 

 

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


PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Cash Flows

 

     Three month period
ended December 31,
    Year ended
December 31,
 
     2017     2016     2017     2016  
   1,000     1,000     1,000     1,000  

Cash flows from operating activities

        

Net result

     (11,274     (8,835     (43,524     (39,119

Adjustments for:

        

— Depreciation

     258       267       1,065       1,245  

— Share-based compensation

     934       537       4,024       2,454  

— Financial income and expenses

     586       (1,438     3,175       (470

Changes in working capital

     703       1,984       164       1,433  

Cash used in operations

     (8,793     (7,485     (35,096     (34,457

Corporate income tax paid

     —         —         (2     —    

Interest received/(paid)

     78       159       147       236  
  

 

 

   

 

 

   

 

 

   

 

 

 

Net cash used in operating activities

     (8,715     (7,326     (34,951     (34,221
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash flow from investing activities

        

Purchases of intangible assets

     —         —         —         —    

Purchases of property, plant and equipment

     (10     (44     (121     (2,539
  

 

 

   

 

 

   

 

 

   

 

 

 

Net cash used in investing activities

     (10     (44     (121     (2,539
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash flow from financing activities

        

Proceeds from issuance of shares, net of transaction costs

     16,923       —         25,685       —    

Proceeds from exercise of share options

     3       —         4       2  

Proceeds from borrowings

     100       177       301       370  

Proceeds from convertible loans

     500       —         650       —    

Redemption of financial lease

     —         —         —         (15
  

 

 

   

 

 

   

 

 

   

 

 

 

Net cash generated by financing activities

     17,526       177       26,640       357  
  

 

 

   

 

 

   

 

 

   

 

 

 

Net increase/(decrease) in cash and cash equivalents

     8,801       (7,193     (8,432     (36,403

Currency effect cash and cash equivalents

     (444     1,472       (2,669     738  

Cash and cash equivalents, at beginning of the period

     39,742       64,921       59,200       94,865  
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash and cash equivalents at the end of the period

     48,099       59,200       48,099       59,200  
  

 

 

   

 

 

   

 

 

   

 

 

 

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


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 VIII B.V. (100%);

 

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

 

    ProQR Therapeutics I Inc. (100%);

 

    Amylon Therapeutics B.V. (majority interest).

ProQR Therapeutics N.V. is also statutory director of Stichting Bewaarneming Aandelen ProQR (“ESOP Foundation”).

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 and the ESOP Foundation.

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, 2016. 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.

 

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


PAGE 6

Unaudited Condensed Consolidated Financial Statements

 

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, 2016. New Standards and Interpretations, which became effective as of January 1, 2017, did not have a material impact on our 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, 2016 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, 2016.

5. Cash and Cash Equivalents

At December 31, 2017, the Company’s cash and equivalents were € 48,099,000 as compared to € 59,200,000 at December 31, 2016. 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 December 31, 2017 and December 31, 2016, the other current liabilities consisted principally of accruals for services provided by vendors not yet billed and other miscellaneous liabilities.

7. Borrowings

 

     December 31,
2017
     December 31,
2016
 
   1,000      1,000  

Innovation credit

     4,899        4,598  

Accrued interest on innovation credit

     1,683        1,099  

Convertible notes

     662        —    

Total borrowings

     7,244        5,697  
  

 

 

    

 

 

 

Current portion

     (1,960      —    
  

 

 

    

 

 

 
     5,284        5,697  
  

 

 

    

 

 

 

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 2017. The credit covers 35% of the costs incurred in respect of the program up to an initial maximum of € 5.0 million through March 31, 2018.

The credit is interest-bearing at a rate of 10% per annum. The credit, including accrued interest, is repayable in three instalments on November 30, 2018, November 30, 2019 and November 30, 2020, 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 € 3,000,000 consists of 37,500,000 ordinary shares and 37,500,000 preference shares with a par value of € 0.04 per share. At December 31, 2017, 36,425,014 ordinary shares were issued and fully paid in cash, of which 4,503,149 were held by the Company as treasury shares (2016: 1,173,958).

In October 2015, we entered into an agreement for an at-the-market offering facility, or ATM facility, pursuant to which we may issue shares of our common stock from time to time under our shelf registration statement up to a maximum of $ 60.0 million. In the fourth quarter of 2017, 60,865 shares were issued pursuant to our ATM facility, resulting in proceeds of € 240,000, net of € 7,000 of offering expenses. As at December 31, 2017, we have issued 976,477 shares pursuant to our ATM facility, resulting in proceeds of € 4,138,000, net of € 127,000 of offering expenses.

On June 28, 2017, the Company agreed to the issuance of 1,200,000 ordinary shares to institutional investors at an issue price of $ 5.00 per share in a registered direct offering with gross proceeds of € 5,278,000. The closing of the offering was effected on July 3, 2017. Transaction costs amounted to € 414,000, resulting in net proceeds of € 4,864,000.

In November 2017, the Company consummated an underwritten public offering and concurrent registered direct offering of 6,397,498 ordinary shares at an issue price of $ 3.25 per share. The gross proceeds from both offerings amounted to € 17,671,000 while the transaction costs amounted to € 988,000, resulting in net proceeds of € 16,683,000.

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 compensation expenses included in operating costs for this plan in 2017 were € 4,024,000 (2016: € 2,454,000), of which € 2,059,000 (2016: € 1,480,000) was recorded in general and administrative costs and € 1,965,000 (2016: € 974,000) was recorded in research and development costs.

 

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


PAGE 9

Unaudited Condensed Consolidated Financial Statements

 

9. Other income

 

     2017      2016  
   1,000      1,000  

Grant income

     870        1,632  

Rental income from property subleases

     625        196  
  

 

 

    

 

 

 
     1,495        1,828  
  

 

 

    

 

 

 

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: € 4.6 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 amount to € 31,153,000 for the year ended December 31, 2017 compared to € 31,923,000 for the year ended December 31, 2016 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 decrease in expenses was primarily due to the completion of the nasal potential difference (NPD) study for QR-010.

11. General and administrative costs

General and administrative costs amount to € 10,840,000 for the year ended December 31, 2017 compared to € 9,478,000 for the year ended December 31, 2016.

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

In January 2018, ProQR announced a research collaboration with Galapagos, where the Company’s Axiomer® technology will be applied to certain fibrosis targets identified by Galapagos. The Axiomer® platform may be applicable to more than 20,000 disease-causing mutations. The Company plans to develop its Axiomer® platform in select therapeutic areas and continue to validate and create value for this novel technology through licensing, partnering and other strategic relationships.

In February 2018, the Company entered into a partnership with Foundation Fighting Blindness in which ProQR will receive up to $7.5 million in funding from FFB for the pre-clinical and clinical development of QR-421a for Usher syndrome type 2A targeting mutations in exon 13. Pre-clinical development of QR-421a has begun and the Company plans to advance QR-421a towards the clinic in 2018 with data anticipated in 2019.

 

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 February 28, 2018

   LOGO

ProQR Announces Results for the Fourth Quarter and Full Year 2017 and Provides Business Update

Key updates

 

    Runway into the second half of 2019: cash of €48.0 million at year-end expected to fund operations through anticipated clinical data readouts in three different programs.

 

    First patient dosed in the Phase 1/2 safety & efficacy trial for QR-110 in children and adults with Leber’s congenital amaurosis 10 (LCA 10). Six-month treatment data is expected in 2018 and 12-month treatment data in 2019. QR-110 was granted FDA Fast Track designation and positive pre-clinical data were presented at the ARVO 2017 meeting. QR-110 has orphan drug designation (ODD) in the U.S. and Europe.

 

    Partnership with Foundation Fighting Blindness (FFB) where ProQR will receive up to $7.5 million in funding for the pre-clinical and clinical development of QR-421a for Usher syndrome type 2A targeting mutations in exon 13. QR-421a expected to advance towards the clinic in 2018 with clinical data anticipated in 2019. QR-421a was granted ODD in the U.S. and Europe.

 

    Completed IND-enabling studies for QR-313 for dystrophic epidermolysis bullosa (DEB), targeting mutations in exon 73, and presented positive pre-clinical data at two European scientific conferences. WINGS, a Phase 1/2 safety and efficacy clinical study of QR-313, is planned to commence in 2018, with interim data also expected in 2018 and full data in 2019. QR-313 was granted ODD in the U.S. and Europe.

 

    Axiomer® RNA-editing technology was introduced at the Company’s second annual Research & Development Investor Day and in vivo proof of concept data were presented at two scientific conferences.

 

    ProQR and Galapagos N.V. entered into a research collaboration to apply Axiomer® to fibrosis targets selected by Galapagos.

 

    Positive results from the Phase 1b safety and tolerability clinical trial of eluforsen, formerly known as QR-010, in cystic fibrosis (CF) patients with F508del mutation were presented at the North American CF conference. A Phase 2 study is currently being designed and planned to start in 2018 subject to a potential partnership. Eluforsen has FDA Fast Track designation and ODD in the U.S. and Europe.

 

    Amylon Therapeutics was spun out as a privately-held company focused on central nervous system therapies. ProQR retains majority ownership and is eligible to receive milestones and royalties.

 

    David M. Rodman, MD joined the Company as Chief Development Strategy Officer and recently was promoted to Executive Vice President of Research & Development,

 

    Appointed several thought leaders to Scientific Advisory Board (Drs. Phil Zamore, Cy Stein, Scott Armstrong and Thaddeus (Ted) Dryja).

LEIDEN, the Netherlands, February 28, 2018 — ProQR Therapeutics N.V. (Nasdaq: PRQR), today announced results for the fourth quarter and full year 2017 and provided a business update.

“In the past year we made solid progress on our mission to create meaningful medicines for patients in need. I am proud of the milestones our team has achieved on all fronts—advancing our pipeline of ophthalmology programs, with QR-110 now in the clinic, positive proof-of-concept for eluforsen for CF, getting QR-313 ready for clinical trials and validating our novel RNA-editing technology, Axiomer®,” said Daniel A. de Boer, CEO of

 

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


ProQR. “We are also excited to be working with several new strategic partners: Galapagos in applying our Axiomer® technology to novel fibrosis targets, Foundation Fighting Blindness to advance our QR-421a candidate for Usher syndrome 2A patients, and the team at Amylon, which we spun out as a privately-owned company that is focused on therapies for CNS. The readouts expected in three separate clinical trials for therapies that have the potential to make a meaningful difference for patients are important and exciting next milestones, as well as advancing the Axiomer® technology further towards development and forging new partnerships.”

Corporate highlights of 2017 and Business Update

QR-110 for LCA 10

 

    In April, ProQR’s QR-110 for Leber’s congenital amaurosis 10 (LCA 10) caused by the p.Cys998X mutation in CEP290 received IND clearance by the FDA to start a Phase 1/2 trial in both adult and pediatric LCA 10 patients. In May, the Company received Fast Track designation from the FDA and presented positive pre-clinical proof of concept data at the ARVO 2017 meeting.

 

    In November, the first patient was dosed in the Phase 1/2 open-label trial (PQ-110-001) to assess the safety, tolerability, pharmacokinetics and efficacy of QR-110 in patients with LCA 10, the most common cause of blindness due to genetic disease in children. The trial will enroll six children (³ 6 years) and six adults (³ 18 years) who have LCA 10 due to one or two copies of the p.Cys998X mutation in the CEP290 gene. During the trial, patients will receive four intravitreal injections of QR-110 into one eye; once every three months. The trial is being conducted in three centers with significant expertise in genetic retinal disease in the U.S. and Europe. The objectives of the trial include safety, tolerability, pharmacokinetics and efficacy as measured by restoration or improvement of visual function and retinal structure through ophthalmic endpoints such as visual acuity, full field stimulus testing (FST), optical coherence tomography (OCT), pupillary light reflex (PLR), mobility course and fixation stability. Changes in quality of life in the trial subjects will also be evaluated. The Company expects to obtain six-month treatment data in 2018 and 12-month data in 2019.

QR-421a and QR-411 for Usher syndrome 2A

 

    In May, the Company presented positive pre-clinical data for QR-421a and QR-411 at the ARVO conference. QR-421a is being developed for the ophthalmic manifestations of Usher syndrome 2A due to exon 13 mutations in USH2A and QR-411 is targeting the ophthalmic manifestations of Usher syndrome 2A due to the PE40 mutation in USH2A. Both candidates are in pre-clinical development; QR-421a advancing to the clinic at the end of 2018. Both QR-421a and QR-411 have received ODD from the FDA and EMA. QR-411 and QR-421a are part of the Company’s growing ophthalmology pipeline, which also includes lead candidate, QR-110 for LCA 10 currently in clinical trials, and two additional discovery programs: QRX-1011 for Stargardt’s disease, and QRX-504 for Fuchs endothelial corneal dystrophy.

QR-313 for DEB

 

    QR-313 for dystrophic epidermolysis bullosa received ODD from the FDA in September and ODD from the EMA in November. QR-313 is a first-in-class RNA-based oligonucleotide designed to target the underlying cause of DEB due to mutations in exon 73 of the COL7A1 gene. DEB is a rare genetic disease that can lead to severe blistering of the skin resulting in high treatment burden and poor quality of life for patients with DEB.

 

    In September, positive QR-313 pre-clinical data were presented at the EB2017 Research Conference and ESDR meeting.

 

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


    IND-enabling studies have been completed and a first in human study of QR-313 in DEB exon 73 patients is expected to commence in 2018. The Phase 1/2 safety and efficacy study is referred to as WINGS (A First in Human, Double-Blind, Randomized, Intra-Subject Placebo-Controlled, Multiple Dose Study of QR-313 Evaluating Safety, Proof of Mechanism, Preliminary Efficacy and Systemic Exposure in Subjects With Recessive Dystrophic Epidermolysis Bullosa (RDEB) due to Mutation(s) in Exon 73 of the COL7A1 Gene). The WINGS study will be conducted in two cohorts: the first cohort will recruit eight RDEB patients with an exon 73 mutation, and a second cohort of DEB patients will be open for enrollment after an interim analysis of the first cohort. The study will evaluate safety, tolerability and pharmacokinetics of QR-313. Interim data from the first trial is expected in 2018, and full results in 2019.

Eluforsen (formerly QR-010) for CF

 

    In April, ProQR was granted two key patents protecting eluforsen in the U.S. and EU which provides the Company with exclusive rights for eluforsen for the treatment of CF until at least 2033. U.S patent no. 9,605,255 is directed to methods of targeting RNA for the most common mutation in CF, called F508del, using oligonucleotides to restore the function of the CFTR protein. In 2016, ProQR was also granted the equivalent European patent (EP 2 852 668 B1). Apart from these ProQR-owned patents, the Company has an exclusive license to U.S. patent no. 9,617,535 from Massachusetts General Hospital covering eluforsen.

 

    The Company presented two abstracts at the European Cystic Fibrosis Conference held in June. Steve Rowe, MD, professor of Pulmonary, Allergy and Critical Care Medicine at University of Alabama and Director of the Gregory Fleming James Cystic Fibrosis Research Center, and Director of the CFF Therapeutics Development Network gave an oral presentation on the final results of study PQ-010-002, a nasal potential difference proof-of-concept study (title “QR-010, an investigational RNA therapeutic, improves CFTR activity in cystic fibrosis subjects homozygous for the F508del mutation”). A poster was also presented on preliminary data from the single ascending dose cohorts of study PQ-010-001, the Company’s Phase 1b safety and tolerability trial (title “QR-010 via inhalation is safe, well-tolerated, and achieves systemic concentrations in a single ascending dose study in subjects with cystic fibrosis homozygous for the F508del CFTR mutation”).

 

    In September, the Company announced positive preliminary top-line results from the multiple-dose cohorts of the Phase 1b randomized, double-blind, placebo-controlled, dose escalation study (Study PQ-010-001) to evaluate the safety, tolerability, pharmacokinetics and exploratory efficacy of eluforsen in adults with CF homozygous for the F508del mutation. A total of 4 dose levels were studied: 6.25, 12.5, 25 and 50 mg of eluforsen administered via inhalation using the PARI eFlow® nebulizer. Patients eligible to participate were males and females of 18 years and over with a ppFEV1 of ³70% at time of inclusion, homozygous for the F508del mutation, and not taking CFTR modulator drugs. The study was designed to enroll 8 cohorts of 8 subjects each (6 subjects receiving eluforsen, 2 subjects receiving placebo). In cohorts 1-4, a single dose of eluforsen was administered, and in cohorts 5-8, twelve doses of eluforsen were administered over a 4-week period. In this study, eluforsen was safe and well-tolerated across all dose levels and we believe that eluforsen may have therapeutic benefit for CF patients. Most patients in the trial reported a significant reduction of their CF symptoms after receiving eluforsen (as measured by the validated questionnaire Cystic Fibrosis Questionnaire-Revised Respiratory Symptom Score, or CFQ-R RSS) compared to placebo. A supportive trend was observed in the improvement of lung function (as measured by percent predicted forced expiratory volume in 1 second, or ppFEV1) compared to placebo. Subjects that received placebo did not report this reduction in CF symptoms or improvement in lung function. As expected, no changes were observed in sweat chloride and weight gain.

 

   

In November, J. Stuart Elborn, Clinical Chair in Respiratory Medicine at Imperial College, Consultant at Royal Brompton Hospital, and immediate past-president of the European Cystic Fibrosis Society, presented data from the Phase 1b trial of eluforsen at the North American Cystic Fibrosis Conference

 

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


 

(NACFC). During the conference, the Company also held an investor and analyst event to discuss the Phase 1b data, new opportunities to target other CFTR mutations in the CFTR gene that can potentially be treated using its RNA technologies and potential design of future studies of eluforsen.

 

    ProQR is currently designing a 12-week Phase 2 safety and efficacy study of eluforsen in CF subjects with the F508del mutation. The trial is expected to be conducted at clinical centers in North America, EU and other countries. The Company plans to initiate the trial in 2018 subject to a partnership.

Spin-out of Amylon Therapeutics

 

    In September, ProQR spun out Amylon Therapeutics as a privately-held company to a group of private and institutional investors. Amylon is developing therapeutics for central nervous system disorders, with an initial focus on a RNA-based treatment for a rare genetic disease that leads to strokes at mid-adulthood, called Hereditary Cerebral Hemorrhage with Amyloidosis of the Dutch type (HCHWA-D). ProQR maintains a majority ownership in Amylon and is entitled to future milestones and royalties.

Axiomer® RNA editing technology platform

 

    In June, ProQR introduced its next-generation Axiomer® RNA technology platform during the Company’s second annual R&D Investor Day held in NYC. Axiomer®’s editing oligonucleotides, or EONs, are designed to recruit endogenous Adenosine Deaminases Acting on RNA, or ADAR, enzymes to make single nucelotide changes in the RNA in a highly specific and targeted manner at the desired location.

 

    In September, in vivo proof of concept data for the Company’s proprietary Axiomer® RNA technology were presented at the Oligonucleotide Therapeutics Society meeting in Bordeaux, France and at a Drug Information Association industry event. The data demonstrated that in an in vivo research model of Hurler syndrome, treatment with the Axiomer® EONs resulted in editing of RNA and partial restoration of the enzymatic activity that is missing in this syndrome. Additionally, the increase in enzymatic activity correlated well with reduced levels of the enzyme’s substrate, the accumulation of which results in the characteristics of the syndrome.

Other corporate updates

 

    In March, David M. Rodman, MD joined ProQR to serve as Chief Development Strategy Officer and was recently promoted to Executive Vice President of Research & Development. Dr. Rodman has extensive experience in rare disease drug development, translational medicine and RNA therapeutics, having previously served in leadership roles with Novartis Institute for Biomedical Research (NIBR), Vertex Pharmaceuticals, miRagen Therapeutics and Nivalis Therapeutics.

 

    In June, ProQR strengthened its cash balance by completing a $6.0 million registered direct offering with high quality institutional investors. In November, the Company received gross process of approximately $20 million in public and registered direct offerings. Investors included several new and existing shareholders, along with significant participation from the Management team and Supervisory Board in the underwritten public offering. Current cash is expected to fund operations into second half of 2019 and through the anticipated data readouts in three different clinical programs.

 

    In August, Drs. Phil Zamore, Cy Stein and Scott Armstrong were appointed to the Scientific Advisory Board (SAB), and in October and Dr. Thaddeus (Ted) Dryja was appointed to the SAB. The new members joined SAB members Drs. Art Levin and Annemieke Aartsma-Rus. The SAB, chaired by Gerard Platenburg, Chief Innovation Officer of ProQR, will play a key strategic role as the Company develops its pipeline of RNA therapeutics and novel proprietary technology platforms for severe genetic rare diseases.

 

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


Subsequent events

 

    In January 2018, ProQR announced a research collaboration with Galapagos, where the Company’s Axiomer® technology will be applied to certain fibrosis targets identified by Galapagos. The Axiomer® platform may be applicable to more than 20,000 disease-causing mutations. The Company plans to develop its Axiomer® platform in select therapeutic areas and continue to validate and create value for this novel technology through licensing, partnering and other strategic relationships.

 

    In February 2018, the Company entered into a partnership with Foundation Fighting Blindness in which ProQR will receive up to $7.5 million in funding from FFB for the pre-clinical and clinical development of QR-421a for Usher syndrome type 2A targeting mutations in exon 13. Pre-clinical development of QR-421a has begun and the Company plans to advance QR-421a towards the clinic in 2018 with data anticipated in 2019.

Financial highlights

At December 31, 2017, ProQR held cash and cash equivalents of €48.1 million, compared to €59.2 million at December 31, 2016. The decrease in cash was driven by operating expenses, partially offset by the receipt of cash generated from financing activities. Net cash used in operating activities during the three month period and full year ended December 31, 2017 was €8.7 million and €35.0 million respectively, compared to €7.3 million and €34.2 million for the same period last year.

Research and development costs increased to €8.3 million for the quarter ended December 31, 2017 from €8.1 million for the same period in 2016. Research and development costs for the year ended December 31, 2017 were €31.2 million, compared to €31.9 million for the same period in 2016.

General and administrative costs increased to €2.9 million for the quarter ended December 31, 2017 from €2.3 million for the same period in 2016. General and administrative costs for the year ended December 31, 2017 were €10.8 million, compared to €9.5 million for the same period in 2016.

Net result for the three month period ended December 31, 2017 was a €11.3 million loss or €0.39 per share, compared to a €8.8 million loss or €0.38 per share for the same period in 2016. Net loss for the year ended December 31, 207 was €43.7 million or €1.72 per share, compared to €39.1 million, or €1.67 per share for the same period ended December 31, 2016. For further financial information for the period ending December 31, 2017, please refer to the financial statements appearing at the end of this release.

2017 Annual Reports

The consolidated statement of financial position of ProQR Therapeutics N.V. as of December 31, 2017 and December 31, 2016, the consolidated statements of comprehensive loss for the years and the three month periods ended December 31, 2017 and 2016, the related consolidated statement of changes in equity for the years ended December 31, 2017 and 2016 and the consolidated statements of cash flows for years and three months periods ended December 31, 2017 and 2016 as presented in this press release are unaudited. ProQR Therapeutics N.V. will publish its 2017 Annual Report on Form 20-F, Statutory Annual Report, and Compensation Report later in Q1 2018. The reports will be published on our website at www.proqr.com.

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 cystic fibrosis, Leber’s congenital amaurosis 10 and dystrophic epidermolysis bullosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

*Since 2012*

 

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


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 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-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 a functional C7 protein, thereby restoring functionality of the anchoring fibrils. QR-313 has been granted orphan drug designation in the United States and the European Union.

About QR-421a

QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of 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 repair the genetic defect in 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-411

QR-411 is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Usher syndrome due to the c.7595-2144A>G mutation in the USH2A gene. The mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional or absence of USH2A protein. QR-411 is designed to restore wild-type USH2A mRNA leading to the production of wild-type USH2A protein by binding the mutated pre-mRNA causing normal splicing of the pre-mRNA. QR-411 has been granted orphan drug designation in the United States and the European Union.

About Eluforsen

Eluforsen, formerly known as QR-010, is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by targeting the mRNA in CF patients that have the F508del mutation. The technology was exclusively licensed from Massachusetts General Hospital. The F508del mutation results in the production of a misfolded CFTR protein that does not function normally. Eluforsen is a single agent designed to bind to the defective CFTR mRNA and to restore CFTR function. Eluforsen is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a small, handheld aerosol delivery device which nebulizes eluforsen into a mist inhaled directly into the lungs. Eluforsen has been granted orphan drug designation in the United States and the European Union and fast-track status by the FDA. The eluforsen project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 633545.

 

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


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® 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® “Editing Oligo Nucleotides”, or EONs, recruit an endogenously expressed RNA editing system called ADAR, which it can direct to change 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. 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 eluforsen (formerly known as QR-010), QR-110, QR-411, QR-421a, QR-313 and the clinical development and the therapeutic potential thereof, including our ongoing clinical trials of these product candidates, statements regarding our ongoing and planned discovery and development of product candidates and the timing thereof, including those in our innovation pipeline and the potential of our Axiomer® technology, statements about Amylon and our collaborations with FFB and Galapagos, statements regarding release of clinical data, and statements regarding our financial resources and cash runway. 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, including that positive results observed in our prior and ongoing studies may not be replicated in later trials or guarantee approval of any product candidate by regulatory authorities, 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.:

Contact:

Smital Shah

Chief Financial Officer

T: +1 415 231 6431

ir@proqr.com

 

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


PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Financial Position

 

     December 31,
2017
     December 31,
2016
 
   1,000      1,000  

Assets

     

Current assets

     

Cash and cash equivalents

     48,099        59,200  

Prepayments and other receivables

     2,064        2,420  

Social securities and other taxes

     396        395  
  

 

 

    

 

 

 

Total current assets

     50,559        62,015  
  

 

 

    

 

 

 

Property, plant and equipment

     2,505        3,438  

Intangible assets

     39        90  
  

 

 

    

 

 

 

Total assets

     53,103        65,543  
  

 

 

    

 

 

 

Equity and liabilities

     

Equity

     

Equity attributable to owners of the Company

     39,363        53,136  

Non-controlling interests

     (38      —    
  

 

 

    

 

 

 

Total equity

     39,325        53,136  
  

 

 

    

 

 

 

Current liabilities

     

Borrowings

     1,960        —    

Trade payables

     546        328  

Social securities and other taxes

     1,019        312  

Pension premiums

     —          13  

Deferred income

     347        —    

Other current liabilities

     4,622        6,057  
  

 

 

    

 

 

 

Total current liabilities

     8,494        6,710  
  

 

 

    

 

 

 

Borrowings

     5,284        5,697  

Total liabilities

     13,778        12,407  
  

 

 

    

 

 

 

Total equity and liabilities

     53,103        65,543  
  

 

 

    

 

 

 

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

 

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


PAGE 1

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 December 31,
     Year ended December 31,  
     2017      2016      2017      2016  
   1,000      1,000      1,000      1,000  

Other income

     511        103        1,495        1,828  

Research and development costs

     (8,345      (8,100      (31,153      (31,923

General and administrative costs

     (2,891      (2,260      (10,840      (9,478
  

 

 

    

 

 

    

 

 

    

 

 

 

Total operating costs

     (11,236      (10,360      (41,993      (41,401
  

 

 

    

 

 

    

 

 

    

 

 

 

Operating result

     (10,725      (10,257      (40,498      (39,573

Finance income and expense

     (586      1,438        (3,175      470  

Result before corporate income taxes

     (11,311      (8,819      (43,673      (39,103

Income taxes

     —          —          (2      —    

Result for the period

     (11,311      (8,819      (43,675      (39,103

Other comprehensive income

     37        (16      151        (16

Total comprehensive income (attributable to owners of the Company)

     (11,274      (8,835      (43,524      (39,119
  

 

 

    

 

 

    

 

 

    

 

 

 

Result attributable to

           

Owners of the Company

     (11,283      (8,819      (43,637      (39,103

Non-controlling interests

     (28      —          (38      —    
     (11,311      (8,819      (43,675      (39,103

Share information

           

Weighted average number of shares outstanding1

     28,695,362        23,346,856        25,374,807        23,346,507  

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

           

Basic loss per share1

     (0.39      (0.38      (1.72      (1.67

Diluted loss per share1

     (0.39      (0.38      (1.72      (1.67

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


PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Changes in Equity

 

     Attributable to owners of the Company              
     Number of
shares
     Share
Capital
     Share
Premium
    Equity
Settled
Employee
Benefit
Reserve
     Translation
Reserve
    Accumulated
Deficit
    Total     Non-controlling
interests
    Total
Equity
 
                     
      1,000      1,000     1,000      1,000     1,000     1,000     1,000     1,000  

Balance at January 1, 2016

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

Result for the period

     —          —          —         —          —         (39,103     (39,103     —         (39,103

Other comprehensive income

     —          —          —         —          (16     —         (16     —         (16

Recognition of share-based payments

     —          —          —         2,454        —         —         2,454       —         2,454  

Share options exercised

     891        0        2       —          —         —         2       —         2  
  

 

 

    

 

 

    

 

 

   

 

 

    

 

 

   

 

 

   

 

 

   

 

 

   

 

 

 

Balance at December 31, 2016

     23,346,856        934        123,597       4,353        (15     (75,733     53,136       —         53,136  
  

 

 

    

 

 

    

 

 

   

 

 

    

 

 

   

 

 

   

 

 

   

 

 

   

 

 

 

Result for the period

     —          —          —         —          —         (43,637     (43,637     (38     (43,675

Other comprehensive income

     —          —          —         —          151       —         151       —         151  

Recognition of share-based payments

     —          —          —         4,024        —         —         4,024       —         4,024  

Issue of ordinary shares

     8,573,975        343        25,342       —          —         —         25,685       —         25,685  

Issue of treasury shares

     4,503,149        180        (180     —          —         —         0       —         0  

Share options exercised

     1,034        0        4       —          —         —         4       —         4  
  

 

 

    

 

 

    

 

 

   

 

 

    

 

 

   

 

 

   

 

 

   

 

 

   

 

 

 

Balance at December 31, 2017

     36,425,014        1,457        148,763       8,377        136       (119,370     39,363       (38     39,325  
  

 

 

    

 

 

    

 

 

   

 

 

    

 

 

   

 

 

   

 

 

   

 

 

   

 

 

 

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

 

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


PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Cash Flows

 

     Three month period
ended December 31,
     Year ended
December 31,
 
     2017      2016      2017      2016  
   1,000      1,000      1,000      1,000  

Cash flows from operating activities

           

Net result

     (11,274      (8,835      (43,524      (39,119

Adjustments for:

           

— Depreciation

     258        267        1,065        1,245  

— Share-based compensation

     934        537        4,024        2,454  

— Financial income and expenses

     586        (1,438      3,175        (470

Changes in working capital

     703        1,984        164        1,433  
  

 

 

    

 

 

    

 

 

    

 

 

 

Cash used in operations

     (8,793      (7,485      (35,096      (34,457
  

 

 

    

 

 

    

 

 

    

 

 

 

Corporate income tax paid

     —          —          (2      —    

Interest received/(paid)

     78        159        147        236  
  

 

 

    

 

 

    

 

 

    

 

 

 

Net cash used in operating activities

     (8,715 )       (7,326 )       (34,951 )       (34,221 ) 
  

 

 

    

 

 

    

 

 

    

 

 

 

Cash flow from investing activities

           

Purchases of intangible assets

     —          —          —          —    

Purchases of property, plant and equipment

     (10      (44      (121      (2,539
  

 

 

    

 

 

    

 

 

    

 

 

 

Net cash used in investing activities

     (10 )       (44 )       (121 )       (2,539 ) 
  

 

 

    

 

 

    

 

 

    

 

 

 

Cash flow from financing activities

           

Proceeds from issuance of shares, net of transaction costs

     16,923        —          25,685        —    

Proceeds from exercise of share options

     3        —          4        2  

Proceeds from borrowings

     100        177        301        370  

Proceeds from convertible loans

     500        —          650        —    

Redemption of financial lease

     —          —          —          (15
  

 

 

    

 

 

    

 

 

    

 

 

 

Net cash generated by financing activities

     17,526        177        26,640        357  
  

 

 

    

 

 

    

 

 

    

 

 

 

Net increase/(decrease) in cash and cash equivalents

     8,801        (7,193      (8,432      (36,403

Currency effect cash and cash equivalents

     (444      1,472        (2,669      738  

Cash and cash equivalents, at beginning of the period

     39,742        64,921        59,200        94,865  
  

 

 

    

 

 

    

 

 

    

 

 

 

Cash and cash equivalents at the end of the period

     48,099        59,200        48,099        59,200  

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

 

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