SEC Filing | ProQR Therapeutics

PROQR THERAPEUTICS N.V.

Date: August 16, 2017

/s/ Smital Shah

Smital Shah

Chief Financial Officer

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Form 6-K

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 6-K

Report of Foreign Private Issuer

Pursuant to Rule 13a-16 or 15d-16 of

the Securities Exchange Act of 1934

August 16, 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 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): ☐

Furnished as Exhibit 99.1 to this Report on Form 6-K are the unaudited financial statements of ProQR Therapeutics N.V. (the “Company”) for the three and six month period ended June 30, 2017 and furnished as Exhibit 99.2 to this Report on Form 6-K is a press release of ProQR Therapeutics N.V. dated August 16, 2017, announcing the Company’s results for the three and six month period ended June 30, 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.

INDEX TO EXHIBITS


Number		Description

99.1		Unaudited financial statements of ProQR Therapeutics N.V. for the three and six month period ended June 30, 2017.

99.2		Press Release of ProQR Therapeutics N.V. dated August 16, 2017, announcing the Company’s results for the three and six month period ended June 30, 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 June 30, 2017 and December 31, 2016		1

Unaudited Condensed Consolidated Statement of Comprehensive Loss for the Three Month and Six Month Periods ended June 30, 2017 and 2016		2

Unaudited Condensed Consolidated Statement of Changes in Equity for the Six Month Periods ended June 30, 2017 and 2016		3

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


	June 30, 2017		December 31, 2016
	€	1,000	€	1,000
Assets
Current assets
Cash and cash equivalents		42,321		59,200
Prepayments and other receivables		2,286		2,420
Social securities and other taxes		738		395

Total current assets		45,345		62,015

Property, plant and equipment		2,976		3,438
Intangible assets		65		90

Total assets		48,386		65,543

Liabilities and shareholders’ equity
Current liabilities
Trade payables		224		328
Social securities and other taxes		164		312
Pension premiums		39		13
Deferred income		729		—
Other current liabilities		4,363		6,057

Total current liabilities		5,519		6,710

Borrowings		6,085		5,697

Total liabilities		11,604		12,407

Shareholders’ equity
Shareholders’ equity		36,782		53,136

Total liabilities and shareholders’ equity		48,386		65,543

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

PAGE 2

Unaudited Condensed Consolidated Financial Statements

PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Profit or Loss and OCI

(€ in thousands, except share and per share data)


	Three month period ended June 30,						Six month period ended June 30,
	2017			2016			2017			2016
	€	1,000		€	1,000		€	1,000		€	1,000
Other income		265			589			658			1,278
Research and development costs		(7,552	)		(8,606	)		(15,582	)		(15,504	)
General and administrative costs		(2,892	)		(2,615	)		(5,196	)		(5,217	)

Total operating costs		(10,444	)		(11,221	)		(20,778	)		(20,721	)

Operating result		(10,179	)		(10,632	)		(20,120	)		(19,443	)
Finance income and expense		(1,184	)		673			(1,721	)		(714	)
Result before corporate income taxes		(11,363	)		(9,959	)		(21,841	)		(20,157	)
Income taxes		—			—			(2	)		—

Net result attributable to equity holders of the Company		(11,363	)		(9,959	)		(21,843	)		(20,157	)
Other comprehensive income		63			(5	)		65			0
Total comprehensive income (attributable to equity holders of the Company)		(11,300	)		(9,964	)		(21,778	)		(20,157	)

Share information
Weighted average number of shares outstanding¹		23,991,685			23,346,340			23,733,885			23,346,153
Earnings per share attributable to equity holders of the Company (expressed in Euro per share)
Basic loss per share¹		(0.47	)		(0.43	)		(0.92	)		(0.86	)
Diluted loss per share¹		(0.47	)		(0.43	)		(0.92	)		(0.86	)

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

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.

PAGE 3

Unaudited Condensed Consolidated Financial Statements

PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Changes in Equity


	Number of shares		Total Share Capital		Share Premium		Equity Settled Employee Benefit Reserve		Translation Reserve			Accumulated Deficit			Total Equity
			€	1,000	€	1,000	€	1,000	€	1,000		€	1,000		€	1,000
Balance at January 1, 2016		23,345,965		934		123,595		1,899		1			(36,630	)		89,799
Net loss		—		—		—		—		—			(20,157	)		(20,157	)
Other comprehensive income		—		—		—		—		0			—			0
Recognition of share-based payments		—		—		—		1,289		—			—			1,289
Share options exercised		891		0		2		—		—			—			2

Balance at June 30, 2016		23,346,856		934		123,597		3,188		1			(56,787	)		70,933

Balance at January 1, 2017		23,346,856		934		123,597		4,353		(15	)		(75,733	)		53,136
Net loss		—		—		—		—		—			(21,843	)		(21,843	)
Other comprehensive income		—		—		—		—		65			—			65
Recognition of share-based payments		—		—		—		2,200		—			—			2,200
Shares issued in the period		758,012		30		3,193		—		—			—			3,223
Share options exercised		381		0		1		—		—			—			1

Balance at June 30, 2017		24,105,249		964		126,791		6,553		50			(97,576	)		36,782

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

PAGE 4

Unaudited Condensed Consolidated Financial Statements

PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Cash Flows


	Three month period ended June 30,						Six month period ended June 30,
	2017			2016			2017			2016
	€	1,000		€	1,000		€	1,000		€	1,000
Cash flows from operating activities
Net result		(11,300	)		(9,964	)		(21,778	)		(20,157	)
Adjustments for:
— Depreciation		272			360			540			694
— Share-based compensation		1,273			699			2,200			1,289
— Financial income and expenses		1,184			(673	)		1,721			714
Changes in working capital		(1,275	)		1,242			(1,368	)		1,292

Cash used in operations		(9,846	)		(8,336	)		(18,685	)		(16,168	)

Corporate income tax paid		—			—			(2	)		—
Interest received/(paid)		1			1			59			66

*Net cash used in operating activities*		*(9,845*	)		*(8,335*	)		*(18,628*	)		*(16,102*	)

Cash flow from investing activities
Purchases of intangible assets		—			—			—			—
Purchases of property, plant and equipment		(48	)		(1,571	)		(93	)		(2,073	)

*Net cash used in investing activities*		*(48*	)		*(1,571*	)		*(93*	)		*(2,073*	)

Cash flow from financing activities
Proceeds from issuance of shares, net of transaction costs		1,072			—			3,223			—
Proceeds from exercise of share options		0			2			1			2
Proceeds from borrowings		101			—			101			193
Redemption of financial lease		—			(7	)		—			(15	)

*Net cash generated by financing activities*		*1,173*			(5	)		*3,325*			*180*

Net increase/(decrease) in cash and cash equivalents		(8,720	)		(9,911	)		(15,396	)		(17,995	)
Currency effect cash and cash equivalents		(1,070	)		755			(1,483	)		(559	)
Cash and cash equivalents, at beginning of the period		52,111			85,467			59,200			94,865

Cash and cash equivalents at the end of the period		42,321			76,311			42,321			76,311

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%).

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

2. Significant Accounting Policies

These condensed consolidated financial statements have been prepared in accordance with International Financial Reporting Standards (“IFRS”), as issued by the International Accounting Standards Board (“IASB”), in particular IAS 34—Interim Financial Reporting. Certain information and disclosures normally included in financial statements prepared in accordance with IFRS have been condensed or omitted. Accordingly, these condensed consolidated financial statements should be read in conjunction with the Company’s annual financial statements for the year ended December 31, 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.

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.

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 June 30, 2017, the Company’s cash and equivalents were € 42,321,000 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 June 30, 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


	June 30, 2017		December 31, 2016
	€	1,000	€	1,000
Innovation credit		4,699		4,598
Accrued interest on innovation credit		1,386		1,099
Total borrowings		6,085		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.

PAGE 8

Unaudited Condensed Consolidated Financial Statements

8. Shareholders’ equity

The authorized share capital of the Company amounting to € 964,000 consists of 24,105,249 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 June 30, 2017, we have issued 758,012 shares pursuant to our ATM facility, resulting in proceeds of € 3,223,000, net of € 99,000 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 € 1,273,000 (2016: € 699,000), of which € 627,000 (2016: € 459,000) was recorded in general and administrative costs and € 646,000 (2016: € 240,000) was recorded in research and development costs.

9. Other income


	Three month period ended June 30,
	2017		2016
	€	1,000	€	1,000
Grant income		154		544
Rental income from property subleases		111		45
		265		589

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.

Pursuant to the terms of the agreement, the Company was obligated to make a one-time milestone payment of CFFT of up to approximately $80 million, payable in three equal annual installments following the first commercial sale of QR-010, as well as certain other milestones and royalties. In August 2017, the one-time milestone payment was amended to approximately $16 million, payable in the same schedule. Further, an amendment was made to the effect that the approximately $6 million payable in case of a change of control transaction may be set-off against the milestone payment mentioned in the preceding sentence. There were no other changes to the milestones or royalties.

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 amount to € 7,552,000 for the quarter ended June 30, 2017 compared to € 8,606,000 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 decrease in expenses was primarily due to the completion of the nasal potential difference (NPD) study for QR-010.

PAGE 9

Unaudited Condensed Consolidated Financial Statements

11. General and administrative costs

General and administrative costs amount to € 2,892,000 for the quarter ended June 30, 2017 compared to € 2,615,000 for the quarter ended June 30, 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 June 28, 2017, the Company agreed to the issuance of 1.2 million ordinary shares to institutional investors at an issue price of $ 5.00 per share in a registered direct offering with gross proceeds of $ 6.0 million. The closing of the offering was effected on July 3, 2017.

EX-99.2

Exhibit 99.2

ProQR Therapeutics N.V.

Press Release August 16, 2017

ProQR Announces Results for the Second Quarter of 2017

Key updates

•

Data from two clinical trials of QR-010 presented at the European Cystic Fibrosis Conference. Enrollment completed in the Phase 1b clinical trial in cystic fibrosis (CF) and top-line data expected to be announced in September.

•

The Investigational New Drug Application (IND) and the Clinical Trial Authorization (CTA) cleared and Fast Track designation received from US Food and Drug Administration (FDA) for QR-110 in patients with Leber’s congenital amaurosis 10 (LCA 10).

•

Two key patents granted protecting QR-010 for cystic fibrosis in the US and EU.

•

Pre-clinical data for three programs in the ophthalmology pipeline targeting LCA 10 and Usher syndrome presented at the ARVO annual meeting.

•

Second annual Research & Development Day featured presentations by ProQR senior management and key opinion leaders and introduced next-generation Axiomer^® RNA technology platform.

LEIDEN, the Netherlands, August 16, 2017 — ProQR Therapeutics N.V. (Nasdaq: PRQR), today announced results for the second quarter of 2017.

“I am pleased with the numerous advancements our team has made this quarter in expanding the depth and breadth of our pipeline with the goal of developing medicines for patients in need,” said Daniel de Boer, Chief Executive Officer of ProQR. “We have made significant progress in all of our programs as well as in our innovation efforts. Our QR-010 program for CF has completed enrollment of the Phase 1b trial and we look forward to announcing the results from this study in September. Furthermore, for the first program in our ophthalmology pipeline, QR-110 for LCA 10, the FDA and EMA have cleared the IND and CTA, enabling us to start a clinical trial in children and adults to study safety and efficacy. QR-110 was also granted Fast Track designation by the FDA underlining the need for therapeutics for these patients. Additionally, two other programs in our ophthalmology pipeline continue to generate exciting pre-clinical data in Usher syndrome including our QRX-411, which recently was granted orphan drug designation. Our QR-313 program for dystrophic epidermolysis bullosa (DEB) is also progressing in IND-enabling development, on track to start and complete a clinical trial in 2018. Beyond that, we are excited about the Axiomer^® platform technology we announced at our R&D day. I’m proud of our scientific team to have invented such a groundbreaking scientific technology that holds the potential to bring a large number of new therapeutics to patients in need.”

Corporate Highlights

•

In April, ProQR’s QR-110 received clearance of its IND application by FDA to commence a clinical trial in both adult and pediatric LCA 10 patients. In May, the Company received Fast Track designation for QR-110 from the FDA. The planned Phase 1/2 open-label trial (PQ-110-001) 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 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 are 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 data from the trial are expected in 2018.

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

•

In April, ProQR received grants of two key patents protecting QR-010 in the US and EU which provides the Company with 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. In 2016, ProQR also received the grant of the equivalent European patent (EP 2 852 668 B1). Apart from these ProQR owned patents, the Company has an exclusive license to US patent no. 9,617,535 from Massachusetts General Hospital covering QR-010.

•

In May, three abstracts were presented on three of ProQR’s RNA editing approaches for inherited blindness at the Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) in Baltimore, MD. Additional positive pre-clinical proof-of-concept data was presented for QR-110 in LCA 10 together with pre-clinical data for two programs, QRX-411 and QRX-421, each targeting specific mutations that result in Usher syndrome.

•

In June, the Company presented two abstracts at the European Cystic Fibrosis Conference (ECFS) in Seville, Spain. Steve Rowe, M.D., 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, an ongoing 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 June, ProQR held its second annual Research & Development Day in New York featuring presentations by ProQR senior management and perspectives from several key opinion leaders on the Company’s development pipeline. During the event, the Company also introduced its next-generation Axiomer^® RNA technology platform, which has the potential to yield a new class of medicines for genetic diseases. The archived webcast of the presentation is accessible from the ‘Investor Relations’ section of ProQR’s website (www.proqr.com) under ‘Events and Presentations’.

Subsequent Events

•

In July, the Company’s QRX-411 for Usher syndrome received orphan drug designation from the FDA and European Medicines Agency (EMA). QRX-411 is part of ProQR’s ophthalmology pipeline that currently also includes one clinical compound, QR-110 for Leber’s congenital amaurosis 10, and three pipeline programs, QRX-421 for Usher syndrome, QRX-1011 for Stargardt’s disease and QRX-504 for Fuchs endothelial corneal dystrophy.

•

In June, the Company agreed to issue 1.2 million ordinary shares to high quality institutional investors based on an inbound request at an issue price of $5.00 per share in a registered direct offering with gross proceeds of $6.0 million. The closing of the offering occurred on July 3, 2017.

Financial Highlights

At June 30, 2017, ProQR held cash and cash equivalents of €42.3 million, compared to €59.2 million at December 31, 2016. Net cash used in operating activities during the three month period ended June 30, 2017 was €9.8 million, compared to €8.3 million for the same period last year.

Research and development costs totaled €7.6 million for the quarter ended June 30, 2017 compared to €8.6 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 decrease in expenses was primarily due to the completion of the nasal potential difference (NPD) study for QR-010.

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

General and administrative costs increased to €2.9 million for the quarter ended June 30, 2017 compared to €2.6 million for the quarter ended June 30, 2016.

Net result for the three month period ended March 31, 2017 was a €11.3 million loss or €0.47 per share, compared to a €10.0 million loss or €0.43 per share for the same period last year. For further financial information for the period ending June 30, 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, 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*

About QR-010

QR-010 is a first-in-class investigational 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 Union’s 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 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 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 and fast track status by the FDA.

About QRX-411

QRX-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. QRX-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. QRX-411 has been granted orphan drug designation in the United States and the European Union.

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

FORWARD-LOOKING STATEMENTS

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Forward-looking statements are based on management’s beliefs and assumptions and on information available to management only as of the date of this press release. These forward-looking statements include, but are not limited to, statements regarding QR-010, QR-110 and QRX-411, and the clinical development and the therapeutic potential thereof, including regarding our PQ-110-001 clinical trial of QR-110, 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 regarding release of clinical data, including that from our Phase 1b trial of QR-010 for CF, 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.:

Investor Contact:

Bonnie Ortega

T: +1 858 245 3983

ir@proqr.com

Media Contact:

Sariette Witte

T: +31 6 2970 4513 (NL)

T: + 1 213 261 8891 (US)

pr@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


	June 30, 2017		December 31, 2016
	€	1,000	€	1,000
Assets
Current assets
Cash and cash equivalents		42,321		59,200
Prepayments and other receivables		2,286		2,420
Social securities and other taxes		738		395

Total current assets		45,345		62,015

Property, plant and equipment		2,976		3,438
Intangible assets		65		90

Total assets		48,386		65,543

Liabilities and shareholders’ equity
Current liabilities
Trade payables		224		328
Social securities and other taxes		164		312
Pension premiums		39		13
Deferred income		729		—
Other current liabilities		4,363		6,057

Total current liabilities		5,519		6,710

Borrowings		6,085		5,697

Total liabilities		11,604		12,407

Shareholders’ equity
Shareholders’ equity		36,782		53,136

Total liabilities and shareholders’ equity		48,386		65,543

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 Profit or Loss and OCI

(€ in thousands, except share and per share data)


	Three month period ended June 30,						Six month period ended June 30,
	2017			2016			2017			2016
	€	1,000		€	1,000		€	1,000		€	1,000
Other income		265			589			658			1,278
Research and development costs		(7,552	)		(8,606	)		(15,582	)		(15,504	)
General and administrative costs		(2,892	)		(2,615	)		(5,196	)		(5,217	)

Total operating costs		(10,444	)		(11,221	)		(20,778	)		(20,721	)

Operating result		(10,179	)		(10,632	)		(20,120	)		(19,443	)
Finance income and expense		(1,184	)		673			(1,721	)		(714	)
Result before corporate income taxes		(11,363	)		(9,959	)		(21,841	)		(20,157	)
Income taxes		—			—			(2	)		—

Net result attributable to equity holders of the Company		(11,363	)		(9,959	)		(21,843	)		(20,157	)
Other comprehensive income		63			(5	)		65			0
Total comprehensive income (attributable to equity holders of the Company)		(11,300	)		(9,964	)		(21,778	)		(20,157	)

Share information
Weighted average number of shares outstanding¹		23,991,685			23,346,340			23,733,885			23,346,153
Earnings per share attributable to equity holders of the Company (expressed in Euro per share)
Basic loss per share¹		(0.47	)		(0.43	)		(0.92	)		(0.86	)
Diluted loss per share¹		(0.47	)		(0.43	)		(0.92	)		(0.86	)

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 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		—		—		—		—		—			(20,157	)		(20,157	)
Other comprehensive income		—		—		—		—		0			—			0
Recognition of share-based payments		—		—		—		1,289		—			—			1,289
Share options exercised		891		0		2		—		—			—			2

Balance at June 30, 2016		23,346,856		934		123,597		3,188		1			(56,787	)		70,933

Balance at January 1, 2017		23,346,856		934		123,597		4,353		(15	)		(75,733	)		53,136

Net loss		—		—		—		—		—			(21,843	)		(21,843	)
Other comprehensive income		—		—		—		—		65			—			65
Recognition of share-based payments		—		—		—		2,200		—			—			2,200
Shares issued in the period		758,012		30		3,193		—		—			—			3,223
Share options exercised		381		0		1		—		—			—			1

Balance at June 30, 2017		24,105,249		964		126,791		6,553		50			(97,576	)		36,782

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 June 30,						Six month period ended June 30,
	2017			2016			2017			2016
	€	1,000		€	1,000		€	1,000		€	1,000
Cash flows from operating activities
Net result		(11,300	)		(9,964	)		(21,778	)		(20,157	)
Adjustments for:
— Depreciation		272			360			540			694
— Share-based compensation		1,273			699			2,200			1,289
— Financial income and expenses		1,184			(673	)		1,721			714
Changes in working capital		(1,275	)		1,242			(1,368	)		1,292

Cash used in operations		(9,846	)		(8,336	)		(18,685	)		(16,168	)

Corporate income tax paid		—			—			(2	)		—
Interest received/(paid)		1			1			59			66

*Net cash used in operating activities*		*(9,845*	)		*(8,335*	)		*(18,628*	)		*(16,102*	)

Cash flow from investing activities
Purchases of intangible assets		—			—			—			—
Purchases of property, plant and equipment		(48	)		(1,571	)		(93	)		(2,073	)

*Net cash used in investing activities*		*(48*	)		*(1,571*	)		*(93*	)		*(2,073*	)

Cash flow from financing activities
Proceeds from issuance of shares, net of transaction costs		1,072			—			3,223			—
Proceeds from exercise of share options		0			2			1			2
Proceeds from borrowings		101			—			101			193
Redemption of financial lease		—			(7	)		—			(15	)

*Net cash generated by financing activities*		*1,173*			(5	)		*3,325*			*180*

Net increase/(decrease) in cash and cash equivalents		(8,720	)		(9,911	)		(15,396	)		(17,995	)
Currency effect cash and cash equivalents		(1,070	)		755			(1,483	)		(559	)
Cash and cash equivalents, at beginning of the period		52,111			85,467			59,200			94,865

Cash and cash equivalents at the end of the period		42,321			76,311			42,321			76,311

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