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 17, 2016
PROQR THERAPEUTICS N.V.
Darwinweg 24
2333 CR Leiden
The Netherlands
Tel: +31 88 166 7000
(Address, Including ZIP Code, and Telephone Number,
Including Area Code, of Registrants 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 months and year ended December 31, 2015 and furnished as Exhibit 99.2 to this Report on Form 6-K is a press release of ProQR Therapeutics N.V. dated February 17, 2016, announcing the Companys results for the three months and year ended December 31, 2015.
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 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 months and year ended December 31, 2015. | |
99.2 | Press Release of ProQR Therapeutics N.V. dated February 17, 2016, announcing the Companys results for the three months and year ended December 31, 2015. |
Exhibit 99.1
PROQR THERAPEUTICS N.V.
Index to Unaudited Condensed Consolidated Financial Statements
PAGE | ||||
Unaudited Condensed Consolidated Statement of Financial Position as of December 31, 2015 and December 31, 2014 |
1 | |||
Unaudited Condensed Consolidated Statement of Comprehensive Loss for the Years and Three Month Periods ended December 31, 2015 and 2014 |
2 | |||
Unaudited Condensed Consolidated Statement of Changes in Equity for the Years ended December 31, 2015 and 2014 |
3 | |||
Unaudited Condensed Consolidated Statement of Cash Flows for the Years and Three Month Periods ended December 31, 2015 and 2014 |
4 | |||
Notes to Unaudited Condensed Consolidated Financial Statements |
5 |
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Financial Position
December 31, 2015 |
December 31, 2014 |
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| 1,000 | | 1,000 | |||||
Assets |
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Current assets |
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Cash and cash equivalents |
94,865 | 112,736 | ||||||
Prepayments and other receivables |
1,948 | 735 | ||||||
Social securities and other taxes |
956 | 426 | ||||||
Total current assets |
97,769 | 113,897 | ||||||
Property, plant and equipment |
2,199 | 1,187 | ||||||
Intangible assets |
141 | 163 | ||||||
Total assets |
100,109 | 115,247 | ||||||
Liabilities and shareholders equity |
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Current liabilities |
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Finance lease liabilities |
15 | 34 | ||||||
Trade payables |
885 | 1,247 | ||||||
Social securities and other taxes |
235 | 341 | ||||||
Pension premiums |
16 | 127 | ||||||
Deferred income |
144 | | ||||||
Other current liabilities |
4,191 | 1,265 | ||||||
Total current liabilities |
5,486 | 3,014 | ||||||
Finance lease liabilities |
| 15 | ||||||
Borrowings |
4,824 | 2,814 | ||||||
Total liabilities |
10,310 | 5,843 | ||||||
Shareholders equity |
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Shareholders equity |
89,799 | 109,404 | ||||||
Total liabilities and shareholders equity |
100,109 | 115,247 | ||||||
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The notes are an integral part of these condensed consolidated financial statements.
1
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, |
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2015 | 2014 | 2015 | 2014 | |||||||||||||
| 1,000 | | 1,000 | | 1,000 | | 1,000 | |||||||||
Other income |
958 | 309 | 3,235 | 313 | ||||||||||||
Research and development costs |
(6,494 | ) | (3,273 | ) | (23,401 | ) | (10,267 | ) | ||||||||
General and administrative costs |
(1,999 | ) | (1,997 | ) | (6,837 | ) | (6,507 | ) | ||||||||
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Total operating costs |
(8,493 | ) | (5,270 | ) | (30,238 | ) | (16,774 | ) | ||||||||
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Operating result |
(7,535 | ) | (4,961 | ) | (27,003 | ) | (16,461 | ) | ||||||||
Finance income and expense |
1,409 | 2,924 | 6,171 | 4,334 | ||||||||||||
Result before corporate income taxes |
(6,126 | ) | (2,037 | ) | (20,832 | ) | (12,127 | ) | ||||||||
Income taxes |
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Net loss attributable to equity holders of the Company |
(6,126 | ) | (2,037 | ) | (20,832 | ) | (12,127 | ) | ||||||||
Other comprehensive income |
1 | | 1 | | ||||||||||||
Total comprehensive loss (attributable to equity holders of the Company) |
(6,125 | ) | (2,037 | ) | (20,831 | ) | (12,127 | ) | ||||||||
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Share information |
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Weighted average number of shares outstanding1 |
23,345,860 | 23,338,154 | 23,343,262 | 11,082,801 | ||||||||||||
Earnings per share attributable to the equity holders of the Company (expressed in Euro per share) |
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Basic loss per share1 |
(0.26 | ) | (0.09 | ) | (0.89 | ) | (1.09 | ) | ||||||||
Diluted loss per share1 |
(0.26 | ) | (0.09 | ) | (0.89 | ) | (1.09 | ) |
The notes are an integral part of these condensed consolidated financial statements.
1. | For the periods presented in these financial statements, the potential exercise of share options and the conversion of preferred shares into ordinary shares in 2014 are 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. |
2
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 | |||||||||||||||||||
Balance at January 1, 2014 |
6,108,152 | 59 | 3,482 | 41 | | (3,671 | ) | (89 | ) | |||||||||||||||||||
Net loss |
| | | | | (12,127 | ) | (12,127 | ) | |||||||||||||||||||
Recognition of share-based payments |
| | | 646 | | | 646 | |||||||||||||||||||||
Shares issued in the period |
17,755,515 | 880 | 122,291 | | | | 123,171 | |||||||||||||||||||||
Treasury shares issued |
(525,513 | ) | (5 | ) | (2,192 | ) | | | | (2,197 | ) | |||||||||||||||||
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Balance at December 31, 2014 |
23,338,154 | 934 | 123,581 | 687 | | (15,798 | ) | 109,404 | ||||||||||||||||||||
Net loss |
| | | | | (20,832 | ) | (20,832 | ) | |||||||||||||||||||
Other comprehensive income |
| | | | 1 | | 1 | |||||||||||||||||||||
Recognition of share-based payments |
| | | 1,212 | | | 1,212 | |||||||||||||||||||||
Share options exercised |
7,811 | 0 | 14 | | | | 14 | |||||||||||||||||||||
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Balance at December 31, 2015 |
23,345,965 | 934 | 123,595 | 1,899 | 1 | (36,630 | ) | 89,799 | ||||||||||||||||||||
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The notes are an integral part of these condensed consolidated financial statements.
3
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows
Three month period ended December 31, |
Year ended December 31, |
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2015 | 2014 | 2015 | 2014 | |||||||||||||
| 1,000 | | 1,000 | | 1,000 | | 1,000 | |||||||||
Cash flows from operating activities |
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Net loss |
(6,125 | ) | (2,037 | ) | (20,831 | ) | (12,127 | ) | ||||||||
Adjustments for: |
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Depreciation |
142 | 61 | 480 | 126 | ||||||||||||
Share-based compensation |
293 | 240 | 1,212 | 646 | ||||||||||||
Financial income and expenses |
(1,409 | ) | (2,924 | ) | (6,171 | ) | (4,334 | ) | ||||||||
Changes in working capital |
165 | (315 | ) | 637 | 1,090 | |||||||||||
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Cash used in operations |
(6,934 | ) | (4,975 | ) | (24,673 | ) | (14,599 | ) | ||||||||
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Corporate income tax paid |
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Interest received/(paid) |
160 | (6 | ) | 441 | 142 | |||||||||||
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Net cash used in operating activities |
(6,774 | ) | (4,981 | ) | (24,232 | ) | (14,457 | ) | ||||||||
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Cash flow from investing activities |
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Purchases of intangible assets |
| (124 | ) | (28 | ) | (124 | ) | |||||||||
Purchases of property, plant and equipment |
(203 | ) | (515 | ) | (1,296 | ) | (1,109 | ) | ||||||||
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Net cash used in investing activities |
(203 | ) | (639 | ) | (1,324 | ) | (1,233 | ) | ||||||||
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Cash flow from financing activities |
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Proceeds from issuance of shares, net of transaction costs |
| 150 | | 118,250 | 1 | |||||||||||
Proceeds from exercise of share options |
0 | | 14 | | ||||||||||||
Proceeds from borrowings |
386 | | 1,640 | 1,667 | ||||||||||||
Redemption of financial lease |
(7 | ) | (7 | ) | (34 | ) | (34 | ) | ||||||||
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Net cash generated by financing activities |
379 | 143 | 1,620 | 119,883 | ||||||||||||
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Net increase/(decrease) in cash and cash equivalents |
(6,598 | ) | (5,477 | ) | (23,936 | ) | 104,193 | |||||||||
Currency effect cash and cash equivalents |
1,451 | 2,956 | 6,065 | 4,414 | ||||||||||||
Cash and cash equivalents, at beginning of the period |
100,012 | 115,257 | 112,736 | 4,129 | ||||||||||||
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Cash and cash equivalents at the end of the period |
94,865 | 112,736 | 94,865 | 112,736 | ||||||||||||
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The notes are an integral part of these condensed consolidated financial statements.
1. | Net of non-cash conversion of convertible loan. |
4
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 Companys 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 Darwinweg 24, 2333 CR 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 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 Companys annual financial statements for the year ended December 31, 2014. 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 Companys 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 Companys annual financial statements for the year ended December 31, 2014. New Standards and Interpretations, which became effective as of January 1, 2015, did not have a material impact on our condensed consolidated financial statements.
5
4. Critical Accounting Estimates and Judgments
In the application of the Companys 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 Companys 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 Companys annual financial statements for the year ended December 31, 2014 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 on September 17, 2014, the Company used the opening price of the Companys stock on September 18, 2014, the first day of trading of the Companys stock on the Nasdag Global Market, which amounted to US$13.00 (10.03) as the value of its ordinary shares.
For all options granted subsequent to the initial public offering, the Company uses the closing price of the ordinary shares on the previous business day as the 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 Companys 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. Managements 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) 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 vary in any particular period.
6
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 Companys annual financial statements for the year ended December 31, 2014.
5. Cash and Cash Equivalents
At December 31, 2015, the Companys cash and equivalents were 94,865,000 as compared to 112,736,000 at December 31, 2014. 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, 2015 and December 31, 2014, 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 December 31, 2015 increased significantly compared to December 31, 2014 as both our activities and our number of employees increased significantly compared to last year.
7. Borrowings
December 31, 2015 |
December 31, 2014 |
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| 1,000 | | 1,000 | |||||
Innovation credit |
4,228 | 2,588 | ||||||
Accrued interest on innovation credit |
596 | 226 | ||||||
Total borrowings |
4,824 | 2,814 | ||||||
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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 Companys cystic fibrosis program. The credit was increased in the course of 2013, 2014 and 2015. 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.
8. Shareholders equity
The authorized share capital of the Company amounting to 934,000 consists of 23,345,965 ordinary shares with a nominal value of 0.04 per share. All issued shares have been fully paid in cash.
On September 15, 2014, the general meeting of shareholders of the Company resolved to approve and effect a capital reorganization, including a share split and bonus share issuance. The combined effect of the share split and bonus share issuance was a 101.804232-for-1 share split of the outstanding ordinary and preferred shares held by the Companys shareholders. This share split became effective on September 15, 2014.
All share, per-share and related information presented in the comparative figures of these condensed consolidated financial statements and accompanying footnotes have been retroactively adjusted, where applicable, to reflect the impact of the share split.
7
On September 18, 2014, the Company was listed at the NASDAQ Global Market under ticker symbol PRQR. In connection with this listing, the Company issued a total of 8,625,000 ordinary shares against the initial public offering price of $ 13.00, resulting in gross proceeds of $ 112,125,000 ( 87,202,000). The number of shares issued includes the exercise of the overallotment option granted to the underwriters. The net proceeds raised in the offering amounted to 80,376,000, net of 8,589,000 of underwriting discounts and offering expenses, of which 6,826,000 was processed through share premium and 1,763,000 was included in the statement of comprehensive loss as general and administrative costs.
All of the issued preferred shares were converted into the Companys ordinary shares. The conversion rate for the preferred shares was one-to-one, adjusted for the stock splits.
Treasury shares
All treasury shares presented in the statement of changes in equity relate to ordinary shares that have legally been issued, but that are within control of the Company. Therefore, these shares are presented as treasury shares.
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. Total compensation expenses included in operating costs for this plan in 2015 were 1,212,000 (2014: 646,000), of which 801,000 (2014: 404,000) was recorded in general and administrative costs and 411,000 (2014: 242,000) was recorded in research and development costs.
9. Other income
Other income increased to 3,235,000 for the year ended December 31, 2015 from 313,000 for the year ended December 31, 2014 and comprised income related to grants. Other income particularly increased in 2015 resulting from the 6 million grant from the European Commission (EC) under the Horizon 2020 program to finance the clinical development of QR-010.
10. Research and development costs
Research and development costs increased to 23,401,000 for the year ended December 31, 2015 from 10,267,000 for the year ended December 31, 2014 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 QR-010 into clinical development and QR-110 from research into development as well as increased investments in our other research programs.
8
11. General and administrative costs
General and administrative costs amount to 6,837,000 for the year ended December 31, 2015 compared to 6,507,000 for the year ended December 31, 2014.
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.
9
Exhibit 99.2
ProQR Announces Results for the Fourth Quarter and Full Year 2015
LEIDEN, the Netherlands, February 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 genetic diseases including cystic fibrosis (CF) and Lebers congenital amaurosis (LCA), today announced results for the fourth quarter and full year 2015.
2015 was a year of execution for ProQR. From the inception of ProQR in 2012, we had been working towards taking QR-010 into the clinic and in 2015 we started two global clinical studies, said Daniel de Boer, Chief Executive Officer of ProQR. During 2015, we have further executed on our strategy to build ProQR into a global company with multiple programs that include QR-110 for LCA patients moving into development and the advancement of our discovery programs in several severe genetic disorders. With the opening of our U.S. office in Palo Alto, the growing talent in our organization, now at 125 ProQRians, and the pipeline of potential new product candidates we look forward to an exciting 2016.
Financial Highlights
At December 31, 2015, ProQR held cash and cash equivalents of 94.9 million, compared to 112.7 million at December 31, 2014. The decrease in cash was driven by operating expenses, partially offset by receipt of grants and foreign currency gains. Net cash used in operating activities during the three month period and full year ended December 31, 2015 was 6.8 million and 24.2 million respectively, compared to 5.0 million and 14.5 million for the same period last year.
Other income increased to 3.2 million for the year ended December 31, 2015 from 0.3 million in 2014 and comprised income related to grants. For the full year ended 2015, other income increased due to the 6.0 million grant from the European Commission (EC) under the Horizon 2020 program to finance the clinical development of QR-010.
Research and development costs increased to 6.5 million for the quarter ended December 31, 2015 from 3.3 million for the same period in 2014. Research and development costs for the year ended December 31, 2015 were 23.4 million, compared to 10.3 million for the same period in 2014. The increase was primarily driven by the start of our clinical studies, expansion of our toxicology studies, increased investments in our other research programs and the overall growth in the Company.
General and administrative costs remained at 2.0 million for the quarter ended December 31, 2015 as it was for the same period in 2014. General and administrative costs for the year ended December 31, 2015 were 6.8 million, compared to 6.5 million for the same period in 2014.
Net result for the three month period ended December 31, 2015 was a 6.1 million loss or 0.26 per share, compared to a 2.0 million loss or 0.09 per share for the same period in 2014. Net loss for the year ended December 31, 2015 was 20.8 million or 0.89 per share, compared to 12.1 million, or 1.09 per share for the same period ended December 31, 2014. For further financial information for the period ending December 31, 2015, please refer to the financial statements appearing at the end of this release.
Corporate Highlights in 2015
| In 2015, just three and-a-half years after its inception, ProQR started two global clinical studies of QR-010, a novel investigational RNA therapeutic designed to repair the genetic mutation in the mRNA of CF patients due to the DF508 mutation. The first study called PQ-010-001 is a global Phase 1b clinical study that is a randomized, double-blind, placebo-controlled, 28-day study being conducted in approximately 20 centers worldwide. This first-in-human study is evaluating the safety, tolerability and pharmacokinetics of single and multiple ascending doses of inhaled QR-010 in 64 CF patients homozygous (carrying two copies) for the DF508 mutation. As exploratory efficacy endpoints this study is also assessing sweat chloride, weight gain, CFQ-R Respiratory Symptom Score and lung function, measured by FEV1. The study is not powered for statistical significance on these exploratory endpoints. The second study called 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). This proof-of-concept study is a case-controlled, open label 28-day study being conducted in 5 specialized centers in the US and Europe. The study plans to enroll 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. The NPD assay selectively measures the activity of the impaired CFTR protein in the nasal epithelium in CF patients. Restoration of NPD to normal levels will demonstrate an important proof of the effect of QR-010 on CFTR function, a benefit that has already been demonstrated in a mouse model with the DF508 mutation. Both study PQ-010-001 and PQ-010-002 are actively enrolling patients and top-line results for both studies are expected mid to late 2016. |
| During the North American Cystic Fibrosis Conference (NACFC) in October, ProQR presented encouraging pre-clinical data that shows QR-010 is stable in CF mucus, transits through the mucus in a clinically meaningful time, is absorbed systemically after inhalation, and is stable in the presence of commonly used inhaled medications (dornase alfa (Pulmozyme®), fluticasone and salbutamol). In vitro, QR-010 was demonstrated to be stable in CF sputum and diffuse easily through in vitro CF mucus models. Using a mouse model with CF-like lung phenotype, it was shown that after inhalation, QR-010 is absorbed systemically and is delivered to extra pulmonary organs that are also compromised in CF. |
| ProQR progressed QR-110, its second molecule, into preclinical development for the indication of Lebers congenital amaurosis (LCA) due to the p.Cys998X mutation in the CEP290 gene. LCA is a rare disease without any known treatment and is the most common cause of genetic blindness in childhood. QR-110 is an RNA oligonucleotide designed to repair the p.Cys998X mutation in the CEP290 mRNA in LCA patients. Preclinical data will be presented at the Association for Research in Vision and Ophthalmology (ARVO) conference in May 2016. The company is moving this program through pre-clinical development towards clinical studies in 2016. |
| Over 2015, ProQR grew its in house discovery group, strengthening its RNA technologies and patent portfolio. ProQR announced that future programs amongst many others may include RNA based oligonucleotides for the treatment of dystrophic epidermolysis bullosa, Usher syndrome, Fuchs endothelial corneal dystrophy (FECD), Huntingtons disease, Friedreichs ataxia, and Alzheimers disease. ProQR will host an R&D Day for investors on March 14, 2016 in New York City and will highlight the efforts across the therapeutic areas of pulmonology, ophthalmology, dermatology, and neurology. |
| In 2015 the company and its academic partners received a grant from the European Union under the Horizon 2020 program. The maximum amount of 6.0 million was granted to support the clinical development of QR-010. ProQR also received additional tranches totaling 1.6 million under the Innovation credit program or Innovatiekrediet by the Dutch government, through its agency RVO (previously: AgentschapNL) of the Ministry of Economic Affairs, for the cystic fibrosis development program. |
| ProQR strengthened its Supervisory Board with the appointment of Paul Baart in June 2015. He is also the new Chair of the Audit Committee. Paul was a former member of the Global Board at Price Waterhouse Cooper International and is a renowned expert in accounting and finance. |
| ProQR opened an office in Palo Alto, California. This provides ProQR the opportunity to build the company on a global scale. |
Subsequent events
| James Shannon, MD has been nominated to serve as a full member of the Supervisory Board, to be appointed at the 2016 Annual General Shareholders Meeting. James brings significant experience to the Supervisory Board through his extensive career in drug development and pharma. From 2012 until his retirement in 2015, James was Chief Medical Officer at GlaxoSmithKline. Prior to that he was Global Head of Pharma Development at Novartis and Senior Vice-President, Clinical Development at Sterling Winthrop Pharmaceuticals. He held board positions at companies including Biotie, Circassia, Crucell, Endocyte, MannKind and Cerimon Pharmaceuticals. He received his undergraduate and postgraduate degrees at Queens University of Belfast and is a Member of the Royal College of Physicians (UK). The company believes James broad knowledge and expertise will be of significant value to the Supervisory Board. |
About QR-010
QR-010 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by repairing the mRNA defect encoded by the DF508 mutation in the CFTR gene of CF patients. The DF508 mutation is a deletion of three of the coding base pairs, or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally. QR-010 is designed to bind to the defective CFTR mRNA and guide the insertion of the three missing nucleotides, thus repairing the mRNA and subsequently producing wild-type, or normal CFTR protein. QR-010 is designed to be self-administered through a small, handheld aerosol delivery device, or nebulizer, in the form of a mist inhaled into the lungs. We believe this method could allow maximum exposure of QR-010 to the primary target organ, the lung, as well as significant exposure to other affected organs through systemic absorption into the blood. QR-010 has been granted orphan drug designation in the United States and the European Union. The QR-010 project has received funding from the European Unions Horizon 2020 research and innovation programme under grant agreement No 633545.
About QR-110
QR-110 is a first-in-class oligonucleotide, designed to address the underlying cause of Lebers congenital amaurosis 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 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.
2015 Annual Reports
The consolidated statement of financial position of ProQR Therapeutics N.V. as of December 31, 2015 and December 31, 2014, the consolidated statements of comprehensive loss for the years and the three month periods ended December 31, 2015 and 2014, the related consolidated statement of changes in equity for the years ended December 31 ,2015 and 2014 and the consolidated statements of cash flows for years and three months periods ended December 31, 2015 and 2014 as presented in this press release are unaudited. ProQR Therapeutics N.V. will publish its 2015 Annual Report on Form 20-F, Statutory Annual Report, and Compensation Report later in Q1 2015. 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 orphan diseases such as cystic fibrosis and Lebers 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.
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 managements 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 and QR-110, statements regarding our ongoing and planned discovery and development of existing and future product candidates, statements regarding the expected timing of results from our clinical studies, and statements regarding the appointment Dr. Shannon to our Supervisory Board. 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.
Contact:
Sariette Witte
Investor Relations
T: +1 213 261 8891
ir@proqr.com
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Financial Position
December 31, 2015 |
December 31, 2014 |
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| 1,000 | | 1,000 | |||||
Assets |
||||||||
Current assets |
||||||||
Cash and cash equivalents |
94,865 | 112,736 | ||||||
Prepayments and other receivables |
1,948 | 735 | ||||||
Social securities and other taxes |
956 | 426 | ||||||
Total current assets |
97,769 | 113,897 | ||||||
Property, plant and equipment |
2,199 | 1,187 | ||||||
Intangible assets |
141 | 163 | ||||||
Total assets |
100,109 | 115,247 | ||||||
Liabilities and shareholders equity |
||||||||
Current liabilities |
||||||||
Finance lease liabilities |
15 | 34 | ||||||
Trade payables |
885 | 1,247 | ||||||
Social securities and other taxes |
235 | 341 | ||||||
Pension premiums |
16 | 127 | ||||||
Deferred income |
144 | | ||||||
Other current liabilities |
4,191 | 1,265 | ||||||
Total current liabilities |
5,486 | 3,014 | ||||||
Finance lease liabilities |
| 15 | ||||||
Borrowings |
4,824 | 2,814 | ||||||
Total liabilities |
10,310 | 5,843 | ||||||
Shareholders equity |
||||||||
Shareholders equity |
89,799 | 109,404 | ||||||
Total liabilities and shareholders equity |
100,109 | 115,247 | ||||||
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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, |
|||||||||||||||
2015 | 2014 | 2015 | 2014 | |||||||||||||
| 1,000 | | 1,000 | | 1,000 | | 1,000 | |||||||||
Other income |
958 | 309 | 3,235 | 313 | ||||||||||||
Research and development costs |
(6,494 | ) | (3,273 | ) | (23,401 | ) | (10,267 | ) | ||||||||
General and administrative costs |
(1,999 | ) | (1,997 | ) | (6,837 | ) | (6,507 | ) | ||||||||
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Total operating costs |
(8,493 | ) | (5,270 | ) | (30,238 | ) | (16,774 | ) | ||||||||
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Operating result |
(7,535 | ) | (4,961 | ) | (27,003 | ) | (16,461 | ) | ||||||||
Finance income and expense |
1,409 | 2,924 | 6,171 | 4,334 | ||||||||||||
Result before corporate income taxes |
(6,126 | ) | (2,037 | ) | (20,832 | ) | (12,127 | ) | ||||||||
Income taxes |
| | | | ||||||||||||
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Net loss attributable to equity holders of the Company |
(6,126 | ) | (2,037 | ) | (20,832 | ) | (12,127 | ) | ||||||||
Other comprehensive income |
1 | | 1 | | ||||||||||||
Total comprehensive loss (attributable to equity holders of the Company) |
(6,125 | ) | (2,037 | ) | (20,831 | ) | (12,127 | ) | ||||||||
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Share information |
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Weighted average number of shares outstanding1 |
23,345,860 | 23,338,154 | 23,343,262 | 11,082,801 | ||||||||||||
Earnings per share attributable to the equity holders of the Company (expressed in Euro per share) |
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Basic loss per share1 |
(0.26 | ) | (0.09 | ) | (0.89 | ) | (1.09 | ) | ||||||||
Diluted loss per share1 |
(0.26 | ) | (0.09 | ) | (0.89 | ) | (1.09 | ) |
1. | For the periods presented in these financial statements, the potential exercise of share options and the conversion of preferred shares into ordinary shares in 2014 are 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. |
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 | |||||||||||||||||||
Balance at January 1, 2014 |
6,108,152 | 59 | 3,482 | 41 | | (3,671 | ) | (89 | ) | |||||||||||||||||||
Net loss |
| | | | | (12,127 | ) | (12,127 | ) | |||||||||||||||||||
Recognition of share-based payments |
| | | 646 | | | 646 | |||||||||||||||||||||
Shares issued in the period |
17,755,515 | 880 | 122,291 | | | | 123,171 | |||||||||||||||||||||
Treasury shares issued |
(525,513 | ) | (5 | ) | (2,192 | ) | | | | (2,197 | ) | |||||||||||||||||
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Balance at December 31, 2014 |
23,338,154 | 934 | 123,581 | 687 | | (15,798 | ) | 109,404 | ||||||||||||||||||||
Net loss |
| | | | | (20,832 | ) | (20,832 | ) | |||||||||||||||||||
Other comprehensive income |
| | | | 1 | | 1 | |||||||||||||||||||||
Recognition of share-based payments |
| | | 1,212 | | | 1,212 | |||||||||||||||||||||
Share options exercised |
7,811 | 0 | 14 | | | | 14 | |||||||||||||||||||||
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Balance at December 31, 2015 |
23,345,965 | 934 | 123,595 | 1,899 | 1 | (36,630 | ) | 89,799 | ||||||||||||||||||||
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PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows
Three month period ended December 31, |
Year ended December 31, |
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2015 | 2014 | 2015 | 2014 | |||||||||||||
| 1,000 | | 1,000 | | 1,000 | | 1,000 | |||||||||
Cash flows from operating activities |
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Net loss |
(6,125 | ) | (2,037 | ) | (20,831 | ) | (12,127 | ) | ||||||||
Adjustments for: |
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Depreciation |
142 | 61 | 480 | 126 | ||||||||||||
Share-based compensation |
293 | 240 | 1,212 | 646 | ||||||||||||
Financial income and expenses |
(1,409 | ) | (2,924 | ) | (6,171 | ) | (4,334 | ) | ||||||||
Changes in working capital |
165 | (315 | ) | 637 | 1,090 | |||||||||||
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Cash used in operations |
(6,934 | ) | (4,975 | ) | (24,673 | ) | (14,599 | ) | ||||||||
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Corporate income tax paid |
| | | |||||||||||||
Interest received/(paid) |
160 | (6 | ) | 441 | 142 | |||||||||||
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Net cash used in operating activities |
(6,774 | ) | (4,981 | ) | (24,232 | ) | (14,457 | ) | ||||||||
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Cash flow from investing activities |
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Purchases of intangible assets |
| (124 | ) | (28 | ) | (124 | ) | |||||||||
Purchases of property, plant and equipment |
(203 | ) | (515 | ) | (1,296 | ) | (1,109 | ) | ||||||||
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Net cash used in investing activities |
(203 | ) | (639 | ) | (1,324 | ) | (1,233 | ) | ||||||||
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Cash flow from financing activities |
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Proceeds from issuance of shares, net of transaction costs |
| 150 | | 118,250 | 1 | |||||||||||
Proceeds from exercise of share options |
0 | | 14 | | ||||||||||||
Proceeds from borrowings |
386 | | 1,640 | 1,667 | ||||||||||||
Redemption of financial lease |
(7 | ) | (7 | ) | (34 | ) | (34 | ) | ||||||||
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Net cash generated by financing activities |
379 | 143 | 1,620 | 119,883 | ||||||||||||
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Net increase/(decrease) in cash and cash equivalents |
(6,598 | ) | (5,477 | ) | (23,936 | ) | 104,193 | |||||||||
Currency effect cash and cash equivalents |
1,451 | 2,956 | 6,065 | 4,414 | ||||||||||||
Cash and cash equivalents, at beginning of the period |
100,012 | 115,257 | 112,736 | 4,129 | ||||||||||||
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Cash and cash equivalents at the end of the period |
94,865 | 112,736 | 94,865 | 112,736 | ||||||||||||
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1. | Net of non-cash conversion of convertible loan. |