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ProQR Announces Financial Results for the Third Quarter of 2018

November 7, 2018

LEIDEN, Netherlands and CAMBRIDGE, Mass., Nov. 07, 2018 (GLOBE NEWSWIRE) -- ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe genetic rare diseases, today announced results for the third quarter of 2018.

“During the third quarter, we announced important clinical proof-of-concept data for QR-110, demonstrating a robust improvement in vision in patients with LCA10,” said Daniel A. de Boer, chief executive officer of ProQR. “These results build confidence in QR-110 and the broader ability of oligonucleotides to improve vision in patients with inherited retinal diseases. Based on these encouraging results we are rapidly advancing our pipeline for inherited blindness that includes programs for Usher syndrome, Stargardt’s diseases and FECD. With the recent addition of QR-1123 for autosomal dominant retinitis pigmentosa (adRP) from Ionis Pharmaceuticals we have strategically expanded our pipeline in this key therapeutic area. To fund these activities, we have raised capital that will allow us to fund operations into 2021.”

Third Quarter 2018 Corporate Highlights

-- Presented positive interim clinical data for lead program QR-110 at the Retinal Degeneration 2018 meeting (RD2018). In the interim analysis of the ongoing Phase 1/2 clinical trial, QR-110 demonstrated rapid and sustained improvement in vision in patients with LCA10, as well as being well-tolerated with no serious adverse events recorded related to treatment or procedure. The Company expects to start a pivotal Phase 2/3 clinical trial during the first half of 2019. -- Closed an underwritten public offering of 6,612,500 ordinary shares at a price of $15.75 per share including full exercise of underwriters’ option to purchase 862,500 additional ordinary shares. Gross proceeds totaled approximately $104.1 million. -- Delivered two presentations at the Oligonucleotide Therapeutics Society (OTS) conference on clinical data for QR-110 in patients with LCA10 and preclinical data for ProQR’s Axiomer® RNA-editing technology. Abstract titles: ○ Proof-of-concept for RNA-editing oligonucleotide QR-110 for treatment of inherited retinal dystrophy in adults and children with LCA10 ○Structure-based computational approach for optimizing oligonucleotides for A-to-I editing -- Presented at the International Symposium on Usher Syndrome (USH2018) and the Ophthalmology Innovation Summit (OIS) Retina conferences. Abstract titles: ○ Splice modulation to treat USH2A-associated retinal degeneration ○RNA treatments targeting rare diseases -- A paper with preclinical data for QR-110 targeting Leber’s congenital amaurosis 10 (LCA10) was published in Molecular Therapy – Nucleic Acids, an official journal of the American Society of Gene & Cell Therapy -- With current cash on hand of €113.7 million (at September 30, 2018), ProQR’s operations are funded into 2021.

Subsequent Events

-- In-licensed exclusive worldwide rights for IONIS-RHO-2.5Rx, now QR-1123, from Ionis Pharmaceuticals. QR-1123 is a first-in-class investigational oligonucleotide (gapmer) for the treatment of autosomal dominant retinitis pigmentosa (adRP), a rare inherited form of blindness with no approved therapy ○ A first in human Phase 1/2 clinical trial in adRP patients is expected to start in 2019 -- Received a conditional waiver of the €5 million Innovation credit that was awarded by the Dutch Ministry of Economic Affairs for the Company’s cystic fibrosis program. Consequently, the total repayment, including principal and interest, of €7.2 million (scheduled in three annual payments) has been waived and will be reviewed annually. -- Delivered presentations at European Oligonucleotide and Peptide Therapeutics (EuroTIDES) conference. Abstract titles: ○ Childhood Blindness due to a Photoreceptor Cilium Defect Treated with an Intravitreal Antisense Oligonucleotide (QR-110) ○Evaluating the impact of sterilization on oligonucleotide based drug products

Financial Highlights

At September 30, 2018, ProQR held cash and cash equivalents of €113.7 million, compared to €48.1 million at December 31, 2017. The increase in cash was due to the closing of an offering of ordinary shares for which net proceeds totaled €84.3 million. Net cash used in operating activities during the three month period ended September 30, 2018 was €4.3 million, compared to €7.6 million for the same period last year.

Research and development costs totaled €6.3 million for the quarter ended September 30, 2018 compared to €7.2 million for the same period last year.

General and administrative costs decreased to €2.6 million for the quarter ended September 30, 2018 compared to €2.8 million for the same period last year.

Net loss for the three month period ended September 30, 2018 was €6.0 million or €0.18 per share, compared to a €10.5 million loss or €0.42 per share for the same period last year. For further financial information for the period ended September 30, 2018, 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 Leber’s congenital amaurosis 10, dystrophic epidermolysis bullosa and cystic fibrosis. 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-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 (also known as the c.2991+1655A>G mutation) in the CEP290 gene. The p.Cys998X mutation is a substitution of one nucleotide in the pre-mRNA that leads to aberrant splicing of the mRNA and non-functional CEP290 protein. QR-110 is designed to restore normal (wild-type) CEP290 mRNA leading to the production of normal CEP290 protein by binding to the mutated location in the pre-mRNA causing normal splicing of the pre-mRNA. QR-110 is intended to be administered through intravitreal injections in the eye and has been granted orphan drug designation in the United States and the European Union and received fast-track designation by the FDA.

About QR-421a

QR-421a is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of vision loss in Usher syndrome 2A due to mutations in exon 13 of the USH2A gene. Mutations in this exon can cause loss of functional usherin protein that causes the disease. QR-421a is designed to exclude the genetic defect from the RNA in the eye, such that it leads to the expression of a shortened but functional protein, thereby modifying the underlying disease. QR-421a has received orphan drug designation in the United States and the European Union.

About QR-1123

QR-1123 is a first-in-class investigational oligonucleotide (gapmer) that was developed by Ionis Pharmaceuticals using Ionis’ proprietary antisense technology for the treatment of adRP due to the P23H mutation in the RHO gene. The therapy aims to inhibit the formation of the mutated toxic version of the rhodopsin protein by specifically binding the mutated RHO mRNA. Binding of QR-1123 causes allele specific knockdown of the mutated mRNA by a mechanism called RNase H mediated cleavage without affecting the normal RHO mRNA. QR-1123 is intended to be administered through intravitreal injections in the eye.

About Axiomer® Technology Platform

ProQR is pioneering a next-generation RNA technology called Axiomer®, which could potentially yield a new class of medicines for genetic diseases. Axiomer® “Editing Oligonucleotides”, or EONs, mediate single nucleotide changes to RNA in a highly specific and targeted way using molecular machinery that is present in human cells. The Axiomer® EONs are designed to recruit an endogenously expressed RNA editing system called ADAR, which it can direct to the change of an Adenosine (A) to an Inosine (I) in the RNA – an Inosine is translated as a Guanosine (G).

FORWARD-LOOKING STATEMENTSThis 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. Such forward-looking statements include, but are not limited to, statements regarding QR-110 and the clinical development and the therapeutic potential thereof, statements regarding our pipeline of programs targeting inherited retinal dystrophies, statements regarding QR-421a, and the clinical development and the therapeutic potential thereof, statements regarding QR-1123 and the clinical development and therapeutic potential thereof, and statements regarding our Axiomer® Technology Platform and the preclinical development and therapeutic potential thereof. 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. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, 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:Smital ShahChief Financial Officer T: +1 415 231 6431 ir@proqr.com

Media Contact:Sara ZelkovicLifeSci Public RelationsT: +1 646 876 4933 Sara@lifescipublicrelations.com

PROQR THERAPEUTICS N.V. Unaudited Condensed Consolidated Statement of Financial Position ---------------------------------------------------------------- --------------- -------------- September 30, December 31, 2018 2017 ---------------------------------------------------------------- ------------- - ------------ - € 1,000 € 1,000 ---------------------------------------------------------------- ------------- - ------------ - Assets ---------------------------------------------------------------- --------------- -------------- Current assets ---------------------------------------------------------------- --------------- -------------- Cash and cash equivalents 113,716 48,099 ---------------------------------------------------------------- ------------- - ------------ - Prepayments and other receivables 1,976 2,064 ---------------------------------------------------------------- ------------- - ------------ - Social securities and other taxes 1,195 396 ---------------------------------------------------------------- --------------- -------------- Total current assets 116,887 50,559 ---------------------------------------------------------------- --------------- -------------- Property, plant and equipment 2,065 2,505 ---------------------------------------------------------------- ------------- - ------------ - Intangible assets 39 39 ---------------------------------------------------------------- --------------- -------------- Total assets 118,991 53,103 ---------------------------------------------------------------- --------------- -------------- Equity and liabilities ---------------------------------------------------------------- --------------- -------------- Equity ---------------------------------------------------------------- --------------- -------------- Equity attributable to owners of the Company 102,575 39,363 ---------------------------------------------------------------- ------------- - ------------ - Non-controlling interests (173 ) (38 ) ---------------------------------------------------------------- ------------- - ------------ - Total equity 102,402 39,325 ---------------------------------------------------------------- --------------- -------------- Current liabilities ---------------------------------------------------------------- --------------- -------------- Borrowings 2,000 1,960 ---------------------------------------------------------------- ------------- - ------------ - Trade payables 771 546 ---------------------------------------------------------------- ------------- - ------------ - Social securities and other taxes 7 1,019 ---------------------------------------------------------------- ------------- - ------------ - Pension premiums — — ---------------------------------------------------------------- ------------- - ------------ - Deferred income 1,122 347 ---------------------------------------------------------------- ------------- - ------------ - Other current liabilities 6,364 4,622 ---------------------------------------------------------------- --------------- -------------- Total current liabilities 10,264 8,494 ---------------------------------------------------------------- --------------- -------------- Borrowings 6,325 5,284 ---------------------------------------------------------------- --------------- -------------- Total liabilities 16,589 13,778 ---------------------------------------------------------------- --------------- -------------- Total equity and liabilities 118,991 53,103 ---------------------------------------------------------------- ------------- - ------------ -

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 Nine month period ended September 30, ended September 30, 2018 2017 2018 2017 € 1,000 € 1,000 € 1,000 € 1,000 ----------------------------------------------------- ---------- - ---------- - ---------- - ---------- - Other income 2,958 326 4,428 984 ----------------------------------------------------- ------------ ------------ ------------ ------------ Research and development costs (6,297 ) (7,226 ) (19,972 ) (22,808 ) ----------------------------------------------------- ---------- - ---------- - ---------- - ---------- - General and administrative costs (2,579 ) (2,753 ) (7,900 ) (7,949 ) ----------------------------------------------------- ------------ ------------ ------------ ------------ Total operating costs (8,876 ) (9,979 ) (27,872 ) (30,757 ) ----------------------------------------------------- ------------ ------------ ------------ ------------ Operating result (5,918 ) (9,653 ) (23,444 ) (29,773 ) ----------------------------------------------------- ---------- - ---------- - ---------- - ---------- - Finance income and expense (74 ) (868 ) (664 ) (2,589 ) ----------------------------------------------------- ------------ ------------ ------------ ------------ Result before corporate income taxes (5,992 ) (10,521 ) (24,108 ) (32,362 ) ----------------------------------------------------- ---------- - ---------- - ---------- - ---------- - Income taxes — — (1 ) (2 ) ----------------------------------------------------- ------------ ------------ ------------ ------------ Result for the period (5,992 ) (10,521 ) (24,109 ) (32,364 ) ----------------------------------------------------- ---------- - ---------- - ---------- - ---------- - Other comprehensive income (4 ) 49 (15 ) 114 ----------------------------------------------------- ------------ ------------ ------------ ------------ Total comprehensive income (5,996 ) (10,472 ) (24,124 ) (32,250 ) ----------------------------------------------------- ------------ ------------ ------------ ------------ Result attributable to ----------------------------------------------------- ------------ ------------ ------------ ------------ Owners of the Company (5,959 ) (10,511 ) (23,974 ) (32,354 ) ----------------------------------------------------- ---------- - ---------- - ---------- - ---------- - Non-controlling interests (33 ) (10 ) (135 ) (10 ) ----------------------------------------------------- ---------- - ---------- - ---------- - ---------- - (5,992 ) (10,521 ) (24,109 ) (32,364 ) ----------------------------------------------------- ------------ ------------ ------------ ------------ Share information ----------------------------------------------------- ------------ ------------ ------------ ------------ Weighted average number of shares outstanding1 33,355,327 25,282,588 32,440,220 24,255,792 ----------------------------------------------------- ------------ ------------ ------------ ------------ Earnings per share attributable to the equity holders of the Company (expressed in Euro per share) ----------------------------------------------------- ------------ ------------ ------------ ------------ Basic loss per share1 (0.18 ) (0.42 ) (0.74 ) (1.33 ) ----------------------------------------------------- ---------- - ---------- - ---------- - ---------- - Diluted loss per share1 (0.18 ) (0.42 ) (0.74 ) (1.33 ) ----------------------------------------------------- ---------- - ---------- - ---------- - ---------- -

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 ------------------------------------------------------------------------------------------------------------------------ Attributable to owners of the Company Equity Settled Employee Non- Number of Share Share Benefit Translation Accumulated controlling Total shares Capital Premium Reserve Reserve Deficit Total interests Equity ------------- € 1,000 € 1,000 € 1,000 € 1,000 € 1,000 € 1,000 € 1,000 € 1,000 ------------- ---------- ------- ------- -------- ----------- - ----------- - ------- - ----------- - ------- - Balance at January 1, 23,346,856 934 123,597 4,353 (15 ) (75,733 ) 53,136 — 53,136 2017 ------------- ---------- ------- ------- -------- ----------- - ----------- - ------- - ----------- - ------- - Result for — — — — — (32,354 ) (32,354 ) (10 ) (32,364 ) the period ------------- ---------- ------- ------- -------- ----------- - ----------- - ------- - ----------- - ------- - Other comprehensive — — — — 114 — 114 — 114 income ------------- ---------- ------- ------- -------- ----------- - ----------- - ------- - ----------- - ------- - Recognition of — — — 3,090 — — 3,090 — 3,090 share-based payments ------------- ---------- ------- ------- -------- ----------- - ----------- - ------- - ----------- - ------- - Issue of ordinary 2,115,612 85 8,677 — — — 8,762 — 8,762 shares ------------- ---------- ------- ------- -------- ----------- - ----------- - ------- - ----------- - ------- - Share options 381 1 — — — 1 — 1 exercised ------------- ---------- ------- ------- -------- ------------- ------------- --------- ------------- --------- Balance at September 30, 25,462,849 1,019 132,275 7,443 99 (108,087 ) 32,749 (10 ) 32,739 2017 ------------- ---------- ------- ------- -------- ------------- ------------- --------- ------------- --------- Balance at January 1, 36,425,014 1,457 148,763 8,377 136 (119,370 ) 39,363 (38 ) 39,325 2018 ------------- ---------- ------- ------- -------- ----------- - ----------- - ------- - ----------- - ------- - Result for — — — — — (23,974 ) (23,974 ) (135 ) (24,109 ) the period ------------- ---------- ------- ------- -------- ----------- - ----------- - ------- - ----------- - ------- - Other comprehensive — — — — (15 ) — (15 ) — (15 ) income ------------- ---------- ------- ------- -------- ----------- - ----------- - ------- - ----------- - ------- - Recognition of — — — 2,245 — — 2,245 — 2,245 share-based payments ------------- ---------- ------- ------- -------- ----------- - ----------- - ------- - ----------- - ------- - Issue of ordinary 6,612,500 265 84,691 84,956 84,956 shares ------------- ---------- ------- ------- -------- ------------- ------------- ------- - ------------- ------- - Share options — — — — — — — exercised ------------- ---------- ------- ------- -------- ------------- ------------- --------- ------------- --------- Balance at September 30, 43,037,514 1,722 233,454 10,622 121 (143,344 ) 102,575 (173 ) 102,402 2018 ------------- ---------- ------- ------- -------- ----------- - ----------- - ------- - ----------- - ------- -

PROQR THERAPEUTICS N.V. Unaudited Condensed Consolidated Statement of Cash Flows ------------------------------------------------------------------------------------------------------ Three month period Nine month period ended September ended September 30, 30, 2018 2017 2018 2017 € 1,000 € 1,000 € 1,000 € 1,000 ---------------------------------------------------------- ------- - ------- - ------- - ------- - Cash flows from operating activities ---------------------------------------------------------- --------- --------- --------- --------- Net result (5,992 ) (10,521 ) (24,109 ) (32,364 ) ---------------------------------------------------------- ------- - ------- - ------- - ------- - Adjustments for: ---------------------------------------------------------- --------- --------- --------- --------- — Depreciation 242 267 725 807 ---------------------------------------------------------- ------- - ------- - ------- - ------- - — Share-based compensation 734 890 2,245 3,090 ---------------------------------------------------------- ------- - ------- - ------- - ------- - — Financial income and expenses 74 868 664 2,589 ---------------------------------------------------------- ------- - ------- - ------- - ------- - — Net foreign exchange gain / (loss) (4 ) 49 (15 ) 114 ---------------------------------------------------------- --------- --------- --------- --------- Changes in working capital 656 829 1,074 (539 ) ---------------------------------------------------------- ------- - ------- - ------- - ------- - Cash used in operations (4,290 ) (7,618 ) (19,416 ) (26,303 ) ---------------------------------------------------------- --------- --------- --------- --------- Corporate income tax paid 1 — — (2 ) ---------------------------------------------------------- ------- - ------- - ------- - ------- - Interest received/(paid) 32 10 25 69 ---------------------------------------------------------- --------- --------- --------- --------- Net cash used in operating activities (4,257 ) (7,608 ) (19,391 ) (26,236 ) ---------------------------------------------------------- --------- --------- --------- --------- Cash flow from investing activities ---------------------------------------------------------- --------- --------- --------- --------- Purchases of intangible assets — — — — ---------------------------------------------------------- ------- - ------- - ------- - ------- - Purchases of property, plant and equipment (99 ) (18 ) (285 ) (111 ) ---------------------------------------------------------- --------- --------- --------- --------- Net cash used in investing activities (99 ) (18 ) (285 ) (111 ) ---------------------------------------------------------- --------- --------- --------- --------- Cash flow from financing activities ---------------------------------------------------------- --------- --------- --------- --------- Proceeds from issuance of shares, net of transaction costs 84,295 5,539 84,295 8,762 ---------------------------------------------------------- ------- - ------- - ------- - ------- - Proceeds from exercise of share options 637 — 660 1 ---------------------------------------------------------- ------- - ------- - ------- - ------- - Proceeds from borrowings — 100 101 201 ---------------------------------------------------------- ------- - ------- - ------- - ------- - Proceeds from convertible loans 115 150 430 150 ---------------------------------------------------------- --------- --------- --------- --------- Net cash generated by financing activities 85,047 5,789 85,486 9,114 ---------------------------------------------------------- --------- --------- --------- --------- Net increase/(decrease) in cash and cash equivalents 80,691 (1,837 ) 65,810 (17,233 ) ---------------------------------------------------------- --------- --------- --------- --------- Currency effect cash and cash equivalents 57 (742 ) (193 ) (2,225 ) ---------------------------------------------------------- ------- - ------- - ------- - ------- - Cash and cash equivalents, at beginning of the period 32,968 42,321 48,099 59,200 ---------------------------------------------------------- --------- --------- --------- --------- Cash and cash equivalents at the end of the period 113,716 39,742 113,716 39,742 ---------------------------------------------------------- ------- - ------- - ------- - ------- -

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