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
May 17, 2017
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 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 ☒ 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 month period ended March 31, 2017 and furnished as Exhibit 99.2 to this Report on Form 6-K is a press release of ProQR Therapeutics N.V. dated May 17, 2017, announcing the Companys results for the three month period ended March 31, 2017.
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: May 17, 2017 | 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 month period ended March 31, 2017. | |
99.2 | Press Release of ProQR Therapeutics N.V. dated May 17, 2017, announcing the Companys results for the three month period ended March 31, 2017. |
Exhibit 99.1
PROQR THERAPEUTICS N.V.
Index to Unaudited Condensed Consolidated Financial Statements
PAGE | ||||
Unaudited Condensed Consolidated Statement of Financial Position at March 31, 2017 and December 31, 2016 |
1 | |||
Unaudited Condensed Consolidated Statement of Comprehensive Loss for the Three Month Periods ended March 31, 2017 and 2016 |
2 | |||
Unaudited Condensed Consolidated Statement of Changes in Equity for the Three Month Periods ended March 31, 2017 and 2016 |
3 | |||
Unaudited Condensed Consolidated Statement of Cash Flows for the Three Month Periods ended March 31, 2017 and 2016 |
4 | |||
Notes to Unaudited Condensed Consolidated Financial Statements |
5 |
Unaudited Condensed Consolidated Financial Statements
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Financial Position
March 31, 2017 |
December 31, 2016 |
|||||||
1,000 | 1,000 | |||||||
Assets |
||||||||
Current assets |
||||||||
Cash and cash equivalents |
52,111 | 59,200 | ||||||
Prepayments and other receivables |
2,364 | 2,420 | ||||||
Social securities and other taxes |
387 | 395 | ||||||
|
|
|
|
|||||
Total current assets |
54,862 | 62,015 | ||||||
|
|
|
|
|||||
Property, plant and equipment |
3,187 | 3,438 | ||||||
Intangible assets |
78 | 90 | ||||||
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|
|
|
|||||
Total assets |
58,127 | 65,543 | ||||||
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|
|
|
|||||
Liabilities and shareholders equity |
||||||||
Current liabilities |
||||||||
Trade payables |
177 | 328 | ||||||
Social securities and other taxes |
193 | 312 | ||||||
Pension premiums |
27 | 13 | ||||||
Other current liabilities |
6,153 | 6,057 | ||||||
|
|
|
|
|||||
Total current liabilities |
6,550 | 6,710 | ||||||
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|
|
|
|||||
Borrowings |
5,840 | 5,697 | ||||||
|
|
|
|
|||||
Total liabilities |
12,390 | 12,407 | ||||||
|
|
|
|
|||||
Shareholders equity |
||||||||
Shareholders equity |
45,737 | 53,136 | ||||||
|
|
|
|
|||||
Total liabilities and shareholders equity |
58,127 | 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 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 March 31, |
||||||||
2017 | 2016 | |||||||
1,000 | 1,000 | |||||||
Other income |
393 | 689 | ||||||
Research and development costs |
(8,030 | ) | (6,898 | ) | ||||
General and administrative costs |
(2,304 | ) | (2,602 | ) | ||||
|
|
|
|
|||||
Total operating costs |
(10,334 | ) | (9,500 | ) | ||||
|
|
|
|
|||||
Operating result |
(9,941 | ) | (8,811 | ) | ||||
Finance income and expense |
(537 | ) | (1,387 | ) | ||||
Result before corporate income taxes |
(10,478 | ) | (10,198 | ) | ||||
Income taxes |
(2 | ) | | |||||
|
|
|
|
|||||
Net result attributable to equity holders of the Company |
(10,480 | ) | (10,198 | ) | ||||
Other comprehensive income |
2 | 5 | ||||||
Total comprehensive income (attributable to equity holders of the Company) |
(10,478 | ) | (10,193 | ) | ||||
|
|
|
|
|||||
Share information |
||||||||
Weighted average number of shares outstanding1 |
23,473,221 | 23,345,965 | ||||||
Earnings per share attributable to the equity holders of the Company (expressed in Euro per share) |
||||||||
Basic loss per share1 |
(0.45 | ) | (0.44 | ) | ||||
Diluted loss per share1 |
(0.45 | ) | (0.44 | ) |
The notes are an integral part of these condensed consolidated financial statements.
1. | For this period presented in these financial statements, the potential exercise of share options is not included in the diluted earnings per share calculation as the Company was loss-making in all periods. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal in this period. |
PROQR THERAPEUTICS | ZERNIKEDREEF 9 | 2333 CK LEIDEN | THE NETHERLANDS | +31 88 166 7000 | WWW.PROQR.COM
PAGE 2
Unaudited Condensed Consolidated Financial Statements
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Changes in Equity
Number of shares |
Total Share Capital |
Share Premium |
Equity Settled Employee Benefit Reserve |
Translation Reserve |
Accumulated Deficit |
Total Equity |
||||||||||||||||||||||
1,000 | 1,000 | 1,000 | 1,000 | 1,000 | 1,000 | |||||||||||||||||||||||
Balance at January 1, 2016 |
23,345,965 | 934 | 123,595 | 1,899 | 1 | (36,630 | ) | 89,799 | ||||||||||||||||||||
Net loss |
| | | | | (10,198 | ) | (10,198 | ) | |||||||||||||||||||
Other comprehensive income |
| | | | 5 | | 5 | |||||||||||||||||||||
Recognition of share-based payments |
| | | 590 | | | 590 | |||||||||||||||||||||
Share options exercised |
| | | | | | | |||||||||||||||||||||
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|
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Balance at March 31, 2016 |
23,345,965 | 934 | 123,595 | 2,489 | 6 | (46,828 | ) | 80,196 | ||||||||||||||||||||
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|
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Balance at January 1, 2017 |
23,346,856 | 934 | 123,597 | 4,353 | (15 | ) | (75,733 | ) | 53,136 | |||||||||||||||||||
Net loss |
| | | | | (10,480 | ) | (10,480 | ) | |||||||||||||||||||
Other comprehensive income |
| | | | 2 | | 2 | |||||||||||||||||||||
Recognition of share-based payments |
| | | 927 | | | 927 | |||||||||||||||||||||
Shares issued in the period |
518,162 | 21 | 2,130 | | | | 2,151 | |||||||||||||||||||||
Share options exercised |
127 | 0 | 1 | | | | 0 | |||||||||||||||||||||
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Balance at March 31, 2017 |
23,865,145 | 955 | 125,728 | 5,280 | (13 | ) | (86,213 | ) | 45,737 | |||||||||||||||||||
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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 3
Unaudited Condensed Consolidated Financial Statements
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows
Three month period ended March 31, |
||||||||
2017 | 2016 | |||||||
1,000 | 1,000 | |||||||
Cash flows from operating activities |
||||||||
Net result |
(10,478 | ) | (10,193 | ) | ||||
Adjustments for: |
||||||||
Depreciation |
268 | 334 | ||||||
Share-based compensation |
927 | 590 | ||||||
Financial income and expenses |
537 | 1,387 | ||||||
Changes in working capital |
(93 | ) | 50 | |||||
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|
|
|
|||||
Cash used in operations |
(8,839 | ) | (7,832 | ) | ||||
|
|
|
|
|||||
Corporate income tax paid |
(2 | ) | | |||||
Interest received/(paid) |
58 | 65 | ||||||
|
|
|
|
|||||
Net cash used in operating activities |
(8,783 | ) | (7,767 | ) | ||||
|
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Cash flow from investing activities |
||||||||
Purchases of intangible assets |
| | ||||||
Purchases of property, plant and equipment |
(45 | ) | (502 | ) | ||||
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|
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Net cash used in investing activities |
(45 | ) | (502 | ) | ||||
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Cash flow from financing activities |
||||||||
Proceeds from issuance of shares, net of transaction costs |
2,151 | | ||||||
Proceeds from exercise of share options |
1 | | ||||||
Proceeds from borrowings |
| 193 | ||||||
Redemption of financial lease |
| (8 | ) | |||||
|
|
|
|
|||||
Net cash generated by financing activities |
2,152 | 185 | ||||||
|
|
|
|
|||||
Net increase/(decrease) in cash and cash equivalents |
(6,676 | ) | (8,084 | ) | ||||
Currency effect cash and cash equivalents |
(413 | ) | (1,314 | ) | ||||
Cash and cash equivalents, at beginning of the period |
59,200 | 94,865 | ||||||
|
|
|
|
|||||
Cash and cash equivalents at the end of the period |
52,111 | 85,467 | ||||||
|
|
|
|
The notes are an integral part of these condensed consolidated financial statements.
PROQR THERAPEUTICS | ZERNIKEDREEF 9 | 2333 CK LEIDEN | THE NETHERLANDS | +31 88 166 7000 | WWW.PROQR.COM
PAGE 4
Unaudited Condensed Consolidated Financial Statements
PROQR THERAPEUTICS N.V.
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 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%). |
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 34Interim 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, 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 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.
PROQR THERAPEUTICS | ZERNIKEDREEF 9 | 2333 CK LEIDEN | THE NETHERLANDS | +31 88 166 7000 | WWW.PROQR.COM
PAGE 5
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 Companys 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 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, 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 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) 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 6
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 Companys annual financial statements for the year ended December 31, 2016.
5. Cash and Cash Equivalents
At March 31, 2017, the Companys cash and equivalents were 52,111,000 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 March 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. The accrued liabilities as at March 31, 2017 increased compared to December 31, 2016 as a result of the increased level of research and development activities.
7. Borrowings
March 31, 2017 |
December 31, 2016 |
|||||||
1,000 | 1,000 | |||||||
Innovation credit |
4,598 | 4,598 | ||||||
Accrued interest on innovation credit |
1,242 | 1,099 | ||||||
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|
|
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Total borrowings |
5,840 | 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 Companys cystic fibrosis program. The credit was increased in the course of 2013 through 2016. The credit covers 35% of the costs incurred in respect of the program up to an initial maximum of 5.0 million through 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 7
Unaudited Condensed Consolidated Financial Statements
8. Shareholders equity
The authorized share capital of the Company amounting to 934,000 consists of 23,865,145 ordinary shares with a nominal value of 0.04 per share. All issued shares have been fully paid in cash.
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. As at March 31, 2017, we have issued 518,162 shares pursuant to our ATM facility, resulting in proceeds of 2,151,000, net of 66,000 of underwriting discounts and offering expenses.
Translation reserve
The translation reserve comprises all foreign currency differences arising from the translation of the financial statements of foreign operations.
Share options
The Company operates an equity-settled share-based compensation plan which was introduced in 2013. The supervisory board may grant options to employees, members of the supervisory board, members of the management board and consultants. The quarterly compensation expenses included in operating costs for this plan were 927,000 (2016: 590,000), of which 530,000 (2016: 363,000) was recorded in general and administrative costs and 397,000 (2016: 227,000) was recorded in research and development costs.
9. Other income
Three month period ended March 31, | ||||||||
2017 | 2016 | |||||||
1,000 | 1,000 | |||||||
Grant income |
273 | 643 | ||||||
Rental income from property subleases |
120 | 46 | ||||||
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|
|
|
|||||
393 | 689 | |||||||
|
|
|
|
In August 2014, the Company entered into an agreement with Cystic Fibrosis Foundation Therapeutics, Inc., or CFFT, a subsidiary of the Cystic Fibrosis Foundation, pursuant to which CFFT agreed to provide the Company with up to $ 3 million to support the clinical development of QR-010.
In 2015, the European Commission (EC) through its Horizon 2020 program awarded ProQR and its academic partners a grant of 6 million (ProQR: 4.4 million) to support the clinical development of QR-010 in the period up till December 31, 2017. Horizon 2020 is one of the largest research and innovation programs in the European Union with nearly 80 billion in available funding for qualified projects from 2014 to 2020.
Both grants are recognized in other income in the same period in which the related R&D costs are recognized.
10. Research and development costs
Research and development costs increased to 8,030,000 for the quarter ended March 31, 2017 from 6,898,000 for the same period in 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 increase in expenses was primarily due to the advancement of our pipeline, which included clinical development of QR-010 and QR-110, preclinical development of QR-313. The remainder represents increased investments in our other pipeline programs.
PROQR THERAPEUTICS | ZERNIKEDREEF 9 | 2333 CK LEIDEN | THE NETHERLANDS | +31 88 166 7000 | WWW.PROQR.COM
PAGE 8
Unaudited Condensed Consolidated Financial Statements
11. General and administrative costs
General and administrative costs amount to 2,304,000 for the quarter ended March 31, 2017 compared to 2,602,000 for the quarter ended March 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
On April 27 the Company announced that it has received clearance of the investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA). ProQR can therefore start clinical development of QR-110 in Lebers congenital amaurosis Type 10 (LCA 10) patients.
PROQR THERAPEUTICS | ZERNIKEDREEF 9 | 2333 CK LEIDEN | THE NETHERLANDS | +31 88 166 7000 | WWW.PROQR.COM
PAGE 9
Exhibit 99.2
ProQR Therapeutics N.V. Press Release May 17, 2017 |
ProQR Announces Results for the First Quarter of 2017
Key updates
| QR-010 Phase 1b clinical trial on track to present top-line data in cystic fibrosis (CF) patients in mid-2017. |
| Investigational new drug (IND) application for QR-110, ProQRs lead program in ophthalmology, was cleared by the FDA to start a clinical trial in both adult and pediatric LCA 10 patients. |
| David M. Rodman, MD was appointed as Chief Development Strategy Officer of ProQR, and will lead the translational development effort to rapidly advance the pipeline programs into the clinic. |
| ProQR was granted two key patents, protecting QR-010 for cystic fibrosis in the US and EU. |
| Pre-clinical data was presented for three programs in the ophthalmology pipeline targeting LCA 10 and Usher syndrome at the ARVO annual meeting in May 2017. |
LEIDEN, the Netherlands, May 17, 2017 ProQR Therapeutics N.V. (Nasdaq: PRQR), today announced results for the first quarter of 2017.
As we announced earlier this week, we are all devastated by the unexpected passing of our co-founder and vice-chairman of the supervisory board, Henri A. Termeer said Daniel de Boer, Chief Executive Officer of ProQR. Henri was a great mentor, a passionate patient advocate and a key factor in establishing ProQR. His passion to do the right thing in the interest of patients is unparalleled. We are honored to have had the opportunity to work with him so closely and learn from his wealth of experience. He was a true inspiration for all of us, and we will continue to build on the path he helped us to set out. Although he will be deeply missed, our supervisory board, with co-founder and chairman Dinko Valerio, James Shannon, Alison Lawton and Antoine Papiernik, continues to be very strong with broad experience in all aspects of running a biotechnology business and we are extremely motivated to continue to build on the path he helped us set.
With that in mind, this is an important phase for the Company in our goal to translate our rich pipeline into a diversified portfolio of development programs. We are very pleased with the clearance of the IND for QR-110, enabling us to now advance our second molecule into clinical development aiming to make a meaningful difference for LCA 10 patients. We are also excited to add Dave Rodman to our team who will further strengthen our development team, with the goal of efficiently and rapidly driving our programs through to patients. Looking forward to the upcoming summer, we are very excited for the expected completion of the Phase 1b trial in our lead program QR-010. We have been treating >64 CF patients with our lead compound QR-010 and are excited to get to the data and progress the compound into next trials.
Corporate Highlights
| In March, the Company announced that it appointed David M. Rodman, MD as Chief Development Strategy Officer. Dr. Rodman has had a long career in drug development including leadership roles in translational medicine, rare disease drug development, and RNA therapeutics. Dr. Rodmans experience includes a leadership role in developing two approved medicines for CF at Vertex Pharmaceuticals, as Vice President and head of respiratory drug development. He was also the head of translational medicine at Novartis Institute for Biomedical Research. More recently, he was the Chief Medical Officer at MiRagen and Nivalis. Expansion of the ProQR management team will allow the Company to further realize the potential of RNA therapeutics as well as expand business capabilities needed to advance the development of our product candidates. |
Subsequent events
| In April, the Company announced that with the clearance of the investigational new drug (IND) application by the U.S. Food and Drug Administration (FDA), ProQR can now start clinical development of QR-110 in Lebers congenital amaurosis Type 10 (LCA 10) patients. The trial, named PQ-110-001, is an open-label trial that will include approximately 6 children (age 6- 17 years) and 6 adults (³ 18 years) that have LCA 10 due to one or two copies of the p.Cys998X mutation. During the trial, patients will receive four intravitreal injections of QR-110 into one eye; one every three months for one year and the second eye will serve as a control. The QR-110 trial is expected to be conducted in three centers with significant expertise in genetic retinal disease in the US and Europe. The primary |
objective will be to assess safety and tolerability of QR-110. Secondary objectives are to evaluate pharmacokinetics and efficacy, which is measured by specialized ophthalmic tests including visual acuity, full field stimulus testing (FST), optical coherence tomography (OCT), pupillary light reflex (PLR) and a mobility course. Fixation stability and changes in quality of life in LCA subjects will also be evaluated. Top-line results from the trial are expected to be available in 2018. |
| In April, the Company announced the grant of two key patents protecting QR-010 in the US and EU. These patents provide the Company exclusive rights for QR-010 for the treatment of CF until at least July 2033. US 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. Last year, ProQR also received the grant of the equivalent European patent (EP 2 852 668 B1). Apart from these ProQR owned patents, ProQR has an exclusive license to US patent no. 9,617,535 from Massachusetts General Hospital covering QR-010. |
| During the 2017 Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) held on May 7 11, 2017 in Baltimore, MD, USA, the Company presented three abstracts, including additional positive pre-clinical proof-of-concept data for QR-110 in LCA 10 and pre-clinical data for two programs, QRX-411 and QRX-421, each targeting specific mutations that result in Usher syndrome. |
| The Company announced that it will host an R&D Day on June 15th in New York where Company executives and external experts will present ProQRs pipeline of development and early stage programs in detail. |
Financial Highlights
At March 31, 2017, ProQR held cash and cash equivalents of 52.1 million, compared to 59.2 million at December 31, 2016. Net cash used in operating activities during the three month period ended March 31, 2017 was 8.8 million, compared to 7.8 million for the same period last year.
Research and development costs increased to 8.0 million for the quarter ended March 31, 2017 from 6.9 million for the same period last year and comprised of allocated employee costs including share-based payments, the costs of materials and laboratory consumables, outsourced activities, license and intellectual property costs and other allocated costs. The increase in expenses was primarily due to the advancement of our pipeline, which included clinical development of QR-010 and QR-110, pre-clinical development of QR-313. The remainder represents increased investments in our other pipeline programs.
General and administrative costs decreased to 2.3 million for the quarter ended March 31, 2017 from 2.6 million for the same period last year.
Net result for the three month period ended March 31, 2017 was a 10.5 million loss or 0.45 per share, compared to a 10.2 million loss or 0.44 per share for the same period last year. For further financial information for the period ending March 31, 2017, please refer to the financial statements appearing at the end of this release.
About ProQR
ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases such as cystic fibrosis, Lebers congenital amaurosis Type 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*
About QR-010
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 F508del 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 to restore CFTR function. QR-010 is designed to be self-administered via an optimized eFlow® Nebulizer (PARI Pharma GmbH). eFlow® is a small, handheld aerosol delivery device which nebulizes QR-010 into a mist inhaled directly into the lungs. QR-010 has been granted orphan drug designation in the United States and the European Union and fast-track status by the FDA. 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 investigational RNA-based oligonucleotide designed to address the underlying cause of Lebers congenital amaurosis Type 10 due to the p.Cys998X mutation in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. QR-110 is designed to restore wild-type CEP290 mRNA leading to the production of wild-type CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union.
About QR-313
QR-313 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of dystrophic epidermolysis bullosa (DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in this exon can cause loss of functional collagen type VII (C7) protein. Absence of C7 results in the loss of anchoring fibrils that normally link the dermal and epidermal layers of the skin together. QR-313 is designed to exclude exon 73 from the mRNA (exon skipping) and produce truncated but functional C7 protein and thereby restores functionality of the anchoring fibrils.
FORWARD-LOOKING STATEMENTS
This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, look forward to, may, plan, potential, predict, project, should, will, would and similar expressions. Forward-looking statements are based on 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, QR-110 and QR-313, and the clinical development and the therapeutic potential thereof, statements regarding our ongoing and planned discovery and development of product candidates and the timing thereof, including those in our innovation pipeline, statements regarding release of clinical data, and statements regarding our patent estate. 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.:
Smital Shah
Chief Financial Officer
T: +1 415 231 6431
ir@proqr.com
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Financial Position
March 31, 2017 |
December 31, 2016 |
|||||||
1,000 | 1,000 | |||||||
Assets |
||||||||
Current assets |
||||||||
Cash and cash equivalents |
52,111 | 59,200 | ||||||
Prepayments and other receivables |
2,364 | 2,420 | ||||||
Social securities and other taxes |
387 | 395 | ||||||
|
|
|
|
|||||
Total current assets |
54,862 | 62,015 | ||||||
|
|
|
|
|||||
Property, plant and equipment |
3,187 | 3,438 | ||||||
Intangible assets |
78 | 90 | ||||||
|
|
|
|
|||||
Total assets |
58,127 | 65,543 | ||||||
|
|
|
|
|||||
Liabilities and shareholders equity |
||||||||
Current liabilities |
||||||||
Trade payables |
177 | 328 | ||||||
Social securities and other taxes |
193 | 312 | ||||||
Pension premiums |
27 | 13 | ||||||
Other current liabilities |
6,153 | 6,057 | ||||||
|
|
|
|
|||||
Total current liabilities |
6,550 | 6,710 | ||||||
|
|
|
|
|||||
Borrowings |
5,840 | 5,697 | ||||||
|
|
|
|
|||||
Total liabilities |
12,390 | 12,407 | ||||||
|
|
|
|
|||||
Shareholders equity |
||||||||
Shareholders equity |
45,737 | 53,136 | ||||||
|
|
|
|
|||||
Total liabilities and shareholders equity |
58,127 | 65,543 | ||||||
|
|
|
|
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 March 31, |
||||||||
2017 | 2016 | |||||||
1,000 | 1,000 | |||||||
Other income |
393 | 689 | ||||||
Research and development costs |
(8,030 | ) | (6,898 | ) | ||||
General and administrative costs |
(2,304 | ) | (2,602 | ) | ||||
|
|
|
|
|||||
Total operating costs |
(10,334 | ) | (9,500 | ) | ||||
|
|
|
|
|||||
Operating result |
(9,941 | ) | (8,811 | ) | ||||
Finance income and expense |
(537 | ) | (1,387 | ) | ||||
Result before corporate income taxes |
(10,478 | ) | (10,198 | ) | ||||
Income taxes |
(2 | ) | | |||||
|
|
|
|
|||||
Net result attributable to equity holders of the Company |
(10,480 | ) | (10,198 | ) | ||||
Other comprehensive income |
2 | 5 | ||||||
Total comprehensive income (attributable to equity holders of the Company) |
(10,478 | ) | (10,193 | ) | ||||
|
|
|
|
|||||
Share information |
||||||||
Weighted average number of shares outstanding1 |
23,473,221 | 23,345,965 | ||||||
Earnings per share attributable to the equity holders of the Company (expressed in Euro per share) |
||||||||
Basic loss per share1 |
(0.45 | ) | (0.44 | ) | ||||
Diluted loss per share1 |
(0.45 | ) | (0.44 | ) |
1. | For this period presented in these financial statements, the potential exercise of share options is not included in the diluted earnings per share calculation as the Company was loss-making in all periods. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal in this period. |
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Changes in Equity
Number of shares |
Total Share Capital |
Share Premium |
Equity Settled Employee Benefit Reserve |
Translation Reserve |
Accumulated Deficit |
Total Equity |
||||||||||||||||||||||
1,000 | 1,000 | 1,000 | 1,000 | 1,000 | 1,000 | |||||||||||||||||||||||
Balance at January 1, 2016 |
23,345,965 | 934 | 123,595 | 1,899 | 1 | (36,630 | ) | 89,799 | ||||||||||||||||||||
Net loss |
| | | | | (10,198 | ) | (10,198 | ) | |||||||||||||||||||
Other comprehensive income |
| | | | 5 | | 5 | |||||||||||||||||||||
Recognition of share-based payments |
| | | 590 | | | 590 | |||||||||||||||||||||
Share options exercised |
| | | | | | | |||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||
Balance at March 31, 2016 |
23,345,965 | 934 | 123,595 | 2,489 | 6 | (46,828 | ) | 80,196 | ||||||||||||||||||||
Balance at January 1, 2017 |
23,346,856 | 934 | 123,597 | 4,353 | (15 | ) | (75,733 | ) | 53,136 | |||||||||||||||||||
Net loss |
| | | | | (10,480 | ) | (10,480 | ) | |||||||||||||||||||
Other comprehensive income |
| | | | 2 | | 2 | |||||||||||||||||||||
Recognition of share-based payments |
| | | 927 | | | 927 | |||||||||||||||||||||
Shares issued in the period |
518,162 | 21 | 2,130 | | | | 2,151 | |||||||||||||||||||||
Share options exercised |
127 | 0 | 1 | | | | 0 | |||||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|||||||||||||||
Balance at March 31, 2017 |
23,865,145 | 955 | 125,728 | 5,280 | (13 | ) | (86,213 | ) | 45,737 | |||||||||||||||||||
|
|
|
|
|
|
|
|
|
|
|
|
|
|
PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows
Three month period ended March 31, |
||||||||
2017 | 2016 | |||||||
1,000 | 1,000 | |||||||
Cash flows from operating activities |
||||||||
Net result |
(10,478 | ) | (10,193 | ) | ||||
Adjustments for: |
||||||||
Depreciation |
268 | 334 | ||||||
Share-based compensation |
927 | 590 | ||||||
Financial income and expenses |
537 | 1,387 | ||||||
Changes in working capital |
(93 | ) | 50 | |||||
|
|
|
|
|||||
Cash used in operations |
(8,839 | ) | (7,832 | ) | ||||
|
|
|
|
|||||
Corporate income tax paid |
(2 | ) | | |||||
Interest received/(paid) |
58 | 65 | ||||||
|
|
|
|
|||||
Net cash used in operating activities |
(8,783 | ) | (7,767 | ) | ||||
|
|
|
|
|||||
Cash flow from investing activities |
||||||||
Purchases of intangible assets |
| | ||||||
Purchases of property, plant and equipment |
(45 | ) | (502 | ) | ||||
|
|
|
|
|||||
Net cash used in investing activities |
(45 | ) | (502 | ) | ||||
|
|
|
|
|||||
Cash flow from financing activities |
||||||||
Proceeds from issuance of shares, net of transaction costs |
2,151 | | ||||||
Proceeds from exercise of share options |
1 | | ||||||
Proceeds from borrowings |
| 193 | ||||||
Redemption of financial lease |
| (8 | ) | |||||
|
|
|
|
|||||
Net cash generated by financing activities |
2,152 | 185 | ||||||
|
|
|
|
|||||
Net increase/(decrease) in cash and cash equivalents |
(6,676 | ) | (8,084 | ) | ||||
Currency effect cash and cash equivalents |
(413 | ) | (1,314 | ) | ||||
Cash and cash equivalents, at beginning of the period |
59,200 | 94,865 | ||||||
|
|
|
|
|||||
Cash and cash equivalents at the end of the period |
52,111 | 85,467 | ||||||
|
|
|
|