EX-99.2 3 d214953dex992.htm EX-99.2 EX-99.2

Exhibit 99.2

 

 

ProQR Therapeutics N.V.

 

Press Release November 23, 2015

 

   LOGO

FINAL – FOR RELEASE

ProQR Announces Results for the Third Quarter of 2015 and Provides Update on Clinical Trials

LEIDEN, the Netherlands, November 23, 2015 — ProQR Therapeutics N.V. (Nasdaq:PRQR), a company dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe orphan diseases such as cystic fibrosis (CF) and Leber’s congenital amaurosis (LCA), today announced results for the third quarter of 2015 and a delay in top-line data readout for the ongoing clinical studies in CF. Data is now expected to be released in mid to late 2016.

“During the third quarter we announced we started our second clinical study of QR-010 (PQ-010-002) in patients with CF and the DF508 mutation, that is running in parallel with the Phase 1b (PQ-010-001) study. While patient enrollment is going well in both studies, the ramp up of enrollment is slower than expected. We attribute this to very robust but stringent protocols and the large number of competing studies”, said Daniel de Boer, Chief Executive Officer of ProQR. “Our teams have taken a number of steps to accelerate enrollment without compromising on quality. The results from both studies together will give us a strong indication of the potential of QR-010 to make a difference in the lives of patients with CF.”

Financial Highlights

At September 30, 2015, ProQR held cash and cash equivalents of €100.0 million, compared to €106.4 million at June 30, 2015. Net cash used in operating activities during the three month period ended September 30, 2015 was €6.3 million, compared to €4.3 million for the same period last year.

Research and development costs increased to €6.0 million for the quarter ended September 30, 2015 from €2.5 million for the same period in 2014 and comprised of costs of clinical trials, allocated employee costs including share-based payments, the costs of materials and laboratory consumables, license and intellectual property costs and other allocated costs. The increase in expenses was due to clinical development of QR-010 for patients with CF due to the DF508 mutation; development activities related to QR-110 for LCA due to the p.Cys998X mutation; and increased investment into our RNA based therapeutics pipeline.

General and administrative costs decreased to €1.5 million for the quarter ended September 30, 2015 from €2.4 million for the same period in 2014, driven by the costs the Company’s IPO on the Nasdaq Global market in September 2014.

Net result for the three month period ended September 30, 2015 was a €6.3 million loss or €0.27 per share, compared to a €3.4 million loss or €0.43 per share for the same period in 2014. For further financial information for the period ending September 30, 2015, please refer to the financial statements appearing at the end of this release.

Corporate Highlights

 

    Enrollment has started in study PQ-010-002, a global proof-of-concept nasal potential difference (NPD) study of QR-010. PQ-010-002 is a case-controlled, open label 28-day study to be conducted in approximately 5 specialized centers with planned enrollment of 8 CF patients that are homozygous

 

ProQR Therapeutics N.V.  |  Darwinweg 24, 2333 CR Leiden, The Netherlands  |  +31 88 166 7000  |  info@proqr.com  |  www.proqr.com


 

(carrying two copies) for the DF508 mutation and 8 CF patients that are compound heterozygous (one copy of the DF508 plus one other CF disease causing mutation). This second human study in affected patients will evaluate the change in NPD after topical administration of QR-010. QR-010 is a novel investigational RNA therapeutic designed to repair the genetic mutation in the mRNA of cystic fibrosis (CF) patients due to the DF508 mutation.

Subsequent events

 

    The company presented an oral presentation and poster with pre-clinical data on the delivery of QR-010 to the CF diseased lung at the North American Cystic Fibrosis Conference (NACFC) [October 8-11 in Phoenix, Arizona]. QR-010 was demonstrated to be stable in sputum collected from CF patients. QR-010 easily diffuses through CF-like mucus in vitro and in vivo in a clinically relevant timeframe. Solute percentage in the mucus will slow diffusion slightly but it is not a clinically meaningful difference. QR-010 is absorbed systemically after orotracheal administration in normal and betaENaC mice, a mouse model where the lungs have similar mucus and related findings to humans with CF. Quantitative assays showed no difference between normal and betaENaC mice. QR-010 remained stable in mixing studies with three commonly used CF medications: dornase alfa (Pulmozyme®), fluticasone and salbutamol.

 

    Update on the Phase 1b (PQ-010-001) and NPD (PQ-010-002) trials: Patient enrollment continues in Europe and North America for QR-010 for patients with CF associated with the DF508 mutation. ProQR is now expecting to release data in mid to late 2016. Patient screening and enrollment is going well, however due to stringent inclusion/exclusion criteria and the recent increase of clinical studies competing for the same patient population, the predicted acceleration of enrollment has not occurred. ProQR is actively implementing recruitment strategies.

About QR-010

QR-010 is a first-in-class RNA-based oligonucleotide designed to address the underlying cause of the disease by repairing the mRNA defect encoded by the DF508 mutation in the CFTR gene of CF patients. The DF508 mutation is a deletion of three of the coding base pairs, or nucleotides, in the CFTR gene, which results in the production of a misfolded CFTR protein that does not function normally. QR-010 is designed to bind to the defective CFTR mRNA and guide the insertion of the three missing nucleotides, thus repairing the mRNA and subsequently producing wild-type, or normal CFTR protein. QR-010 is designed to be self-administered through a small, handheld aerosol delivery device, or nebulizer, in the form of a mist inhaled into the lungs. We believe this method could allow maximum exposure of QR-010 to the primary target organ, the lung, as well as significant exposure to other affected organs through systemic absorption into the blood. QR-010 has been granted orphan drug designation in the United States and the European Union. The QR-010 project has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No 633545.

About ProQR

ProQR Therapeutics is dedicated to changing lives through the creation of transformative RNA medicines for the treatment of severe orphan diseases such as cystic fibrosis and Leber’s congenital amaurosis. Based on our unique proprietary RNA repair platform technologies we are growing our pipeline with patients and loved ones in mind. Since 2012.

FORWARD-LOOKING STATEMENTS

This press release contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward to”, “may,” “plan,” “potential,” “predict,” “project,”


“should,” “will,” “would” and similar expressions. Forward-looking statements are based on 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 and QR-110, statements regarding our ongoing and planned discovery and development of product candidates, statements regarding the expected timing of results from our clinical studies and statements regarding the Horizon 2020 program. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with our clinical development activities, manufacturing processes and facilities, regulatory oversight, product commercialization, intellectual property claims, and the risks, uncertainties and other factors in our filings made with the Securities and Exchange Commission, including certain sections of our annual report filed on Form 20-F. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements, even if new information becomes available in the future.

ProQR Therapeutics N.V.:

Sariette Witte

Investor Relations

T: +1 213 261 8891

ir@proqr.com


PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Financial Position

 

 

     September 30,
2015
     December 31,
2014
 
     €1,000      €1,000  

Assets

     

Current assets

     

Cash and cash equivalents

     100,012         112,736   

Prepayments and other receivables

     1,788         735   

Social securities and other taxes

     845         426   
  

 

 

    

 

 

 

Total current assets

     102,645         113,897   
  

 

 

    

 

 

 

Property, plant and equipment

     1,980         1,187   

Intangible assets

     153         163   
  

 

 

    

 

 

 

Total assets

     104,778         115,247   
  

 

 

    

 

 

 

Liabilities and shareholders’ equity

     

Current liabilities

     

Finance lease liabilities

     22         34   

Trade payables

     98         1,247   

Social securities and other taxes

     11         341   

Pension premiums

     198         127   

Deferred income

     700         —     

Other current liabilities

     3,784         1,265   
  

 

 

    

 

 

 

Total current liabilities

     4,813         3,014   
  

 

 

    

 

 

 

Finance lease liabilities

     —           15   

Borrowings

     4,334         2,814   
  

 

 

    

 

 

 

Total liabilities

     9,147         5,843   
  

 

 

    

 

 

 

Shareholders’ equity

     

Shareholders’ equity

     95,631         109,404   
  

 

 

    

 

 

 

Total liabilities and shareholders’ equity

     104,778         115,247   
  

 

 

    

 

 

 


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 September 30,
    Nine month period
ended September 30,
 
     2015     2014     2015     2014  
     €1,000     €1,000     €1,000     €1,000  

Other income

     1,191        —          2,277        4   

Research and development costs

     (6,000     (2,457     (16,907     (6,994

General and administrative costs

     (1,458     (2,380     (4,838     (4,510
  

 

 

   

 

 

   

 

 

   

 

 

 

Total operating costs

     (7,458     (4,837     (21,745     (11,504
  

 

 

   

 

 

   

 

 

   

 

 

 

Operating result

     (6,267     (4,837     (19,468     (11,500

Finance income and expense

     (50     1,462        4,762        1,410   

Result before corporate income taxes

     (6,317     (3,375     (14,706     (10,090

Income taxes

     —          —          —          —     
  

 

 

   

 

 

   

 

 

   

 

 

 

Net loss attributable to equity holders of the Company

     (6,317     (3,375     (14,706     (10,090

Other comprehensive income

     —          —          —          —     
  

 

 

   

 

 

   

 

 

   

 

 

 

Total comprehensive loss (attributable to equity holders of the Company)

     (6,317     (3,375     (14,706     (10,090
  

 

 

   

 

 

   

 

 

   

 

 

 

Share information

        

Weighted average number of shares outstanding1

     23,345,170        7,916,686        23,342,386        6,952,792   

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

        

Basic loss per share1

     (0.27     (0.43     (0.63     (1.45

Diluted loss per share1

     (0.27     (0.43     (0.63     (1.45

 

1. For the periods presented in these financial statements, the potential exercise of share options and the conversion of preferred shares into ordinary shares in 2014 are not included in the diluted earnings per share calculation as the Company was loss-making in all periods. Due to the anti-dilutive nature of the outstanding options, basic and diluted earnings per share are equal.


PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Changes in Equity

 

 

     Number of shares     Total
Share
    Share     Equity
Settled
Employee
Benefit
     Accumulated     Total  
     Ordinary     Preferred     Capital     Premium     Reserve      Deficit     Equity  
                 €1,000     €1,000     €1,000      €1,000     €1,000  

Balance at January 1, 2014

     6,108,152        —          59        3,482        41         (3,671     (89

Net loss

     —          —          —          —          —           (10,090     (10,090

Recognition of share-based payments

     —          —          —          —          406         —          406   

Shares issued in the period

     9,490,336        8,265,179        880        122,291        —           —          123,171   

Conversion of preferred shares

     8,265,179        (8,265,179     —          —          —           —          —     

Treasury shares issued

     (525,513     —          (5     (2,191     —           —          (2,196
  

 

 

   

 

 

   

 

 

   

 

 

   

 

 

    

 

 

   

 

 

 

Balance at September 30, 2014

     23,338,154        —          934        123,582        447         (13,761     111,202   
  

 

 

   

 

 

   

 

 

   

 

 

   

 

 

    

 

 

   

 

 

 

Balance at January 1, 2015

     23,338,154        —          934        123,581        687         (15,798     109,404   

Net loss

     —          —          —          —          —           (14,706     (14,706

Recognition of share-based payments

     —          —          —          —          919         —          919   

Share options exercised

     7,684        —          0        14        —           —          14   
  

 

 

   

 

 

   

 

 

   

 

 

   

 

 

    

 

 

   

 

 

 

Balance at September 30, 2015

     23,345,838        —          934        123,595        1,606         (30,504     95,631   
  

 

 

   

 

 

   

 

 

   

 

 

   

 

 

    

 

 

   

 

 

 


PROQR THERAPEUTICS N.V.

Unaudited Condensed Consolidated Statement of Cash Flows

 

 

     Three month period
ended September 30,
    Nine month period
ended September 30,
 
     2015     2014     2015     2014  
     €1,000     €1,000     €1,000     €1,000  

Cash flows from operating activities

        

Net loss

     (6,317     (3,375     (14,706     (10,090

Adjustments for:

        

— Depreciation

     126        26        338        65   

— Share-based compensation

     300        178        919        406   

— Financial income and expenses

     50        (1,462     (4,762     (1,410

Changes in working capital

     (527     205        472        1,405   
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash used in operations

     (6,368     (4,428     (17,739     (9,624
  

 

 

   

 

 

   

 

 

   

 

 

 

Corporate income tax paid

     —          —          —          —     

Interest received/(paid)

     104        129        281        148   
  

 

 

   

 

 

   

 

 

   

 

 

 

Net cash used in operating activities

     (6,264     (4,299     (17,458     (9,476
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash flow from investing activities

        

Purchases of property, plant and equipment

     (56     (344     (1,093     (594

Purchases of intangible assets

     —          —          (28     —     
  

 

 

   

 

 

   

 

 

   

 

 

 

Net cash used in investing activities

     (56     (344     (1,121     (594
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash flow from financing activities

        

Proceeds from issuance of shares, net of transaction costs

     —          80,258        —          118,100 1 

Proceeds from exercise of share options

     8        —          14        —     

Proceeds from borrowings

     —          1        1,254        1,667   

Redemption of financial lease

     (7     (10     (27     (27
  

 

 

   

 

 

   

 

 

   

 

 

 

Net cash generated by financing activities

     1        80,249        1,241        119,740   
  

 

 

   

 

 

   

 

 

   

 

 

 

Net increase/(decrease) in cash and cash equivalents

     (6,319     75,606        (17,338     109,670   

Currency effect cash and cash equivalents

     (51     1,458        4,614        1,458   

Cash and cash equivalents, at beginning of the period

     106,382        38,193        112,736        4,129   
  

 

 

   

 

 

   

 

 

   

 

 

 

Cash and cash equivalents at the end of the period

     100,012        115,257        100,012        115,257   
  

 

 

   

 

 

   

 

 

   

 

 

 

 

1. Net of non-cash conversion of convertible loan.