EX-99.2 3 a19-22440_1ex99d2.htm EX-99.2

Exhibit 99.2

 

FINAL — FOR RELEASE

 

ProQR Announces Recent Progress and Financial Results for the Third Quarter of 2019

 

·                  Encouraging clinical data reported from Phase 1/2 trial of sepofarsen for LCA10

·                  Initial clinical data from Phase 1/2 trial of QR-421a for Usher syndrome type 2 on track for Q1 2020

·                  QR-1123 Investigational New Drug application active for autosomal dominant retinitis pigmentosa

·                  €49.3 million net proceeds from public offering extends cash runway into 2022

 

LEIDEN, Netherlands & CAMBRIDGE, Mass., Nov 06, 2019 — ProQR Therapeutics N.V. (Nasdaq: PRQR) (the “Company”), a company dedicated to changing lives through the creation of transformative RNA medicines for severe genetic rare diseases, today announced results for the third quarter of 2019.

 

“During the third quarter we made great progress towards our mission to bring novel RNA therapies to patients and we recently strengthened our capital position,” said Daniel A. de Boer, chief executive officer of ProQR. “In October we announced that LCA10 patients experienced a significant improvement in vision after treatment with sepofarsen in a Phase 1/2 trial. These results strengthen our confidence in the design of the ongoing pivotal Illuminate Phase 2/3 trial and our broader inherited retinal diseases pipeline. The Phase 1/2 trial of QR-421a in Usher syndrome is on track to deliver first clinical data in Q1 2020 and we are about to dose a first patient in a Phase 1/2 trial of QR-1123 for adRP. We strengthened our financial position with a public offering in October that will allow us to fund operations into 2022, well beyond the expected top-line readout of Illuminate.”

 

Corporate Highlights and Business Update

 

Sepofarsen (QR-110) for Leber’s congenital amaurosis 10 (LCA10)

 

·                  Presented positive top-line results from the Phase 1/2 clinical trial. In the trial, sepofarsen was observed to be well tolerated with rapid, significant and durable improvements in vision observed at month twelve. The target registration dose for the ongoing Phase 2/3 Illuminate trial showed a favorable benefit/risk profile in the Phase 1/2 trial.

·                  The top-line results from the Phase 1/2 trial support confidence in the design of the ongoing Phase 2/3 Illuminate trial that could be the sole registration trial for the program. Top-line data from Illuminate are expected during the first half of 2021.

·                  Received Rare Pediatric Disease designation from the Food and Drug Administration (FDA) for the treatment of LCA10.

 

QR-421a for Usher syndrome type 2

 

·                  The Phase 1/2 Stellar trial of QR-421a in patients with Usher syndrome type 2 is ongoing and on track to deliver interim data during the first quarter of 2020.

 

QR-1123 for autosomal dominant retinitis pigmentosa (adRP)

 

·                  The FDA cleared the Investigational New Drug (IND) application to start a first-in-human clinical trial in patients with adRP. ProQR plans to start enrolling patients in the Phase 1/2 Aurora trial in 2019.

·                  Received Fast Track designation from the FDA.

 


 

Business Updates

 

·                  Closed an underwritten public offering of 9,090,909 ordinary shares on October 18, 2019 at a price of $5.50 per share with full exercise of underwriters’ option to purchase 1,363,636 additional ordinary shares. Net proceeds totaled approximately €49.3 million. With the addition of this capital ProQR’s operations are funded into 2022.

 

Financial Highlights

 

At September 30, 2019, prior to the offering on October 18th, ProQR held cash and cash equivalents of €74.8 million, compared to €105.6 million at December 31, 2018. The cash balance at September 30, 2019 excludes the net proceeds of €49.3 million from the underwritten offering of ordinary shares in October 2019. Net cash used in operating activities during the three-month period ended September 30, 2019 was €8.8 million, compared to €4.3 million for the same period last year.

 

Research and development costs increased to €11.1 million for the quarter ended September 30, 2019 compared to €6.3 million for the same period last year due to increased clinical trial activity.

 

General and administrative costs increased to €2.9 million for the quarter ended September 30, 2019 compared to €2.6 million for the same period last year.

 

Net loss for the three-month period ended September 30, 2019 was €12.2 million or €0.31 per share, compared to a €6.0 million loss or €0.18 per share for the same period last year.

 

In October 2019 the Company sold an aggregate of 10.5 million ordinary shares, with net proceeds to the Company of €49.3 million.

 

For further financial information for the period ended September 30, 2019, please refer to the financial statements appearing at the end of this release.

 

About Sepofarsen

 

Sepofarsen (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 leads to aberrant splicing of the mRNA and non-functional CEP290 protein. Sepofarsen is designed to enable normal splicing, resulting in restoration of normal (wild type) CEP290 mRNA and subsequent production of functional CEP290 protein. Sepofarsen 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 and rare pediatric disease designation from the FDA as well as access to the PRIME scheme by the EMA.

 

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 type 2 and non-syndromic retinitis pigmentosa (RP) due to mutations in exon 13 of the USH2A gene. QR-421a is designed to restore functional Usherin protein by using an exon skipping approach with the aim to stop or reverse vision loss in patients. QR-421a 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 from the FDA.

 


 

About QR-1123

 

QR-1123 is a first-in-class investigational RNA-based oligonucleotide that was discovered and 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 mutant 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 and received IND clearance in August 2019.

 

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, Usher syndrome and autosomal dominant retinitis pigmentosa. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind.

 

*Since 2012*

 

FORWARD-LOOKING STATEMENTS

 

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” and similar expressions. Such forward-looking statements include, but are not limited to, statements regarding sepofarsen (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 our financial position and cash runway. 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:

Sarah Kiely

ProQR Therapeutics N.V.

T: +1 617 599 6228

skiely@proqr.com

or

Hans Vitzthum

LifeSci Advisors

T: +1 617 535 7743

hans@lifesciadvisors.com

 

Media Contact:

Sara Zelkovic

LifeSci Public Relations

T: +1 646 876 4933

sara@lifescipublicrelations.com

 


 

PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Financial Position

 

 

 

September 30,

 

December 31,

 

 

 

2019

 

2018

 

 

 

€ 1,000

 

€ 1,000

 

Assets

 

 

 

 

 

Current assets

 

 

 

 

 

Cash and cash equivalents

 

74,792

 

105,580

 

Prepayments and other receivables

 

2,450

 

1,544

 

Social securities and other taxes

 

830

 

1,243

 

 

 

 

 

 

 

Total current assets

 

78,072

 

108,367

 

 

 

 

 

 

 

Property, plant and equipment

 

2,413

 

1,864

 

Investments in associates

 

579

 

 

 

 

 

 

 

 

Total assets

 

81, 064

 

110,231

 

 

 

 

 

 

 

Equity and liabilities

 

 

 

 

 

Equity

 

 

 

 

 

Equity attributable to owners of the Company

 

59,871

 

92,915

 

Non-controlling interests

 

(452

)

(230

)

Total equity

 

59,419

 

92,685

 

 

 

 

 

 

 

Current liabilities

 

 

 

 

 

Borrowings

 

260

 

 

Lease liabilities

 

890

 

 

Trade payables

 

668

 

135

 

Social securities and other taxes

 

15

 

 

Pension premiums

 

13

 

7

 

Deferred income

 

984

 

545

 

Other current liabilities

 

8,305

 

7,473

 

 

 

 

 

 

 

Total current liabilities

 

11,135

 

8,160

 

 

 

 

 

 

 

Borrowings

 

10,510

 

9,386

 

Lease liabilities

 

 

 

 

 

 

 

 

 

Total liabilities

 

21,645

 

17,546

 

 

 

 

 

 

 

Total equity and liabilities

 

81,064

 

110,231

 

 


 

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, 

 

 

 

2019

 

2018

 

2019

 

2018

 

 

 

€ 1,000

 

€ 1,000

 

€ 1,000

 

€ 1,000

 

Other income

 

530

 

2,958

 

1,509

 

4,428

 

 

 

 

 

 

 

 

 

 

 

Research and development costs

 

(11,074

)

(6,297

)

(32,560

)

(19,972

)

General and administrative costs

 

(2,903

)

(2,579

)

(8,970

)

(7,900

)

 

 

 

 

 

 

 

 

 

 

Total operating costs

 

(13,977

)

(8,876

)

(41,530

)

(27,872

)

 

 

 

 

 

 

 

 

 

 

Operating result

 

(13,447

)

(5,918

)

(40,021

)

(23,444

)

Finance income and expense

 

1,375

 

(74

)

1,339

 

(664

)

Results related to associates

 

(119

)

 

579

 

 

 

 

 

 

 

 

 

 

 

 

Result before corporate income taxes

 

(12,191

)

(5,992

)

(38,103

)

(24,108

)

Income taxes

 

 

 

(64

)

(1

)

 

 

 

 

 

 

 

 

 

 

Result for the period

 

(12,191

)

(5,992

)

(38,167

)

(24,109

)

Other comprehensive income

 

147

 

(4

)

121

 

(15

)

 

 

 

 

 

 

 

 

 

 

Total comprehensive income (attributable to owners of the Company)

 

(12,044

)

(5,996

)

(38,046

)

(24,124

)

 

 

 

 

 

 

 

 

 

 

Result attributable to

 

 

 

 

 

 

 

 

 

Owners of the Company

 

(12,139

)

(5,959

)

(37,945

)

(23,974

)

Non-controlling interests

 

(52

)

(33

)

(222

)

(135

)

 

 

(12,191

)

(5,992

)

(38,167

)

(24,109

)

 

 

 

 

 

 

 

 

 

 

Share information

 

 

 

 

 

 

 

 

 

Weighted average number of shares outstanding(1)

 

38,912,701

 

33,355,327

 

38,902,203

 

32,440,220

 

 

 

 

 

 

 

 

 

 

 

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

 

 

 

 

 

 

 

 

 

Basic loss per share(1)

 

(0.31

)

(0.18

)

(0.98

)

(0.74

)

Diluted loss per share(1)

 

(0.31

)

(0.18

)

(0.98

)

(0.74

)

 


(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, 2018

 

36,425,014

 

1,457

 

148,763

 

8,377

 

136

 

(119,370

)

39,363

 

(38

)

39,325

 

Result for the period

 

 

 

 

 

 

(23,974

)

(23,974

)

(135

)

(24,109

)

Other comprehensive income

 

 

 

 

 

(15

)

 

(15

)

 

(15

)

Recognition of share-based payments

 

 

 

 

2,245

 

 

 

2,245

 

 

2,245

 

Issuance of ordinary shares

 

6,612,500

 

265

 

84,032

 

 

 

 

84,297

 

 

84,297

 

Share options exercised

 

 

 

659

 

 

 

 

659

 

 

659

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Balance at September 30, 2018

 

43,037,514

 

1,722

 

233,454

 

10,622

 

121

 

(143,344

)

102,575

 

(173

)

102,402

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Balance at January 1, 2019

 

43,149,987

 

1,726

 

235,744

 

10,780

 

108

 

(155,443

)

92,915

 

(230

)

92,685

 

Result for the period

 

 

 

 

 

 

(37,945

)

(37,945

)

(222

)

(38,167

)

Other comprehensive income

 

 

 

 

 

121

 

 

121

 

 

121

 

Recognition of share-based payments

 

 

 

 

4,614

 

 

 

4,614

 

 

4,614

 

Share options lapsed

 

 

 

 

(33

)

 

 

33

 

 

 

 

Share options exercised

 

 

 

166

 

(115

)

 

115

 

166

 

 

166

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Balance at September 30, 2019

 

43,149,987

 

1,726

 

235,910

 

15,246

 

229

 

(193,240

)

59,871

 

(452

)

59,419

 

 


 

PROQR THERAPEUTICS N.V.
Unaudited Condensed Consolidated Statement of Cash Flows

 

 

 

Three month period 

 

Nine month period

 

 

 

ended September 30, 

 

ended September 30, 

 

 

 

2019

 

2018

 

2019

 

2018

 

 

 

€ 1,000

 

€ 1,000

 

€ 1,000

 

€ 1,000

 

Cash flows from operating activities

 

 

 

 

 

 

 

 

 

Net result

 

(12,191

)

(5,992

)

(38,167

)

(24,109

)

Adjustments for:

 

 

 

 

 

 

 

 

 

— Depreciation

 

506

 

242

 

1,543

 

725

 

— Share-based compensation

 

1,226

 

734

 

4,614

 

2,245

 

— Financial income and expenses

 

(1,375

)

74

 

(1,339

)

664

 

— Results related to associates

 

119

 

 

(579

)

 

— Net foreign exchange gain / (loss)

 

148

 

(4

)

122

 

(15

)

 

 

 

 

 

 

 

 

 

 

Changes in working capital

 

2,718

 

656

 

1,744

 

1,074

 

Cash used in operations

 

(8,849

)

(4,290

)

(32,062

)

(19,416

)

 

 

 

 

 

 

 

 

 

 

Corporate income tax paid

 

 

1

 

(64

)

 

Interest received

 

90

 

32

 

176

 

25

 

Interest paid

 

(13

)

 

(64

)

 

 

 

 

 

 

 

 

 

 

 

Net cash used in operating activities

 

(8,772

)

(4,257

)

(32,014

)

(19,391

)

 

 

 

 

 

 

 

 

 

 

Cash flow from investing activities

 

 

 

 

 

 

 

 

 

Purchases of intangible assets

 

 

 

 

 

Purchases of property, plant and equipment

 

(32

)

(99

)

(341

)

(285

)

 

 

 

 

 

 

 

 

 

 

Net cash used in investing activities

 

(32

)

(99

)

(341

)

(285

)

 

 

 

 

 

 

 

 

 

 

Cash flow from financing activities

 

 

 

 

 

 

 

 

 

Proceeds from issuance of shares, net of transaction costs

 

 

84,295

 

 

84,295

 

Proceeds from exercise of share options

 

2

 

637

 

166

 

660

 

Proceeds from borrowings

 

 

 

 

101

 

Proceeds from convertible loans

 

 

115

 

690

 

430

 

Repayment of lease liability

 

(290

)

 

(861

)

 

 

 

 

 

 

 

 

 

 

 

Net cash (used in)/generated by financing activities

 

(288

)

85,047

 

(5

)

85,486

 

 

 

 

 

 

 

 

 

 

 

Net increase/(decrease) in cash and cash equivalents

 

(9,092

)

80,691

 

(32,360

)

65,810

 

 

 

 

 

 

 

 

 

 

 

Currency effect cash and cash equivalents

 

1,420

 

57

 

1,572

 

(193

)

Cash and cash equivalents, at beginning of the period

 

82,464

 

32,968

 

105,580

 

48,099

 

 

 

 

 

 

 

 

 

 

 

Cash and cash equivalents at the end of the period

 

74,792

 

113,716

 

74,792

 

113,716

 

 

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