ProQR Announces Financial Results for the Third Quarter of 2018
November 07 2018 - 7:00AM
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
4933Sara@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 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
- 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, 2017 |
|
23,346,856 |
|
934 |
|
123,597 |
|
4,353 |
|
(15 |
) |
|
(75,733 |
) |
|
53,136 |
|
|
— |
|
|
53,136 |
|
Result
for the period |
|
— |
|
— |
|
— |
|
— |
|
— |
|
|
(32,354 |
) |
|
(32,354 |
) |
|
(10 |
) |
|
(32,364 |
) |
Other
comprehensive income |
|
— |
|
— |
|
— |
|
— |
|
114 |
|
|
— |
|
|
114 |
|
|
— |
|
|
114 |
|
Recognition of share-based payments |
|
— |
|
— |
|
— |
|
3,090 |
|
— |
|
|
— |
|
|
3,090 |
|
|
— |
|
|
3,090 |
|
Issue of
ordinary shares |
|
2,115,612 |
|
85 |
|
8,677 |
|
— |
|
— |
|
|
— |
|
|
8,762 |
|
|
— |
|
|
8,762 |
|
Share
options exercised |
|
381 |
|
|
|
1 |
|
— |
|
— |
|
|
— |
|
|
1 |
|
|
— |
|
|
1 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at September 30, 2017 |
|
25,462,849 |
|
1,019 |
|
132,275 |
|
7,443 |
|
99 |
|
|
(108,087 |
) |
|
32,749 |
|
|
(10 |
) |
|
32,739 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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 |
|
Issue of
ordinary shares |
|
6,612,500 |
|
265 |
|
84,691 |
|
|
|
|
|
|
|
84,956 |
|
|
|
|
84,956 |
|
Share
options exercised |
|
— |
|
|
|
|
|
— |
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
— |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at September 30, 2018 |
|
43,037,514 |
|
1,722 |
|
233,454 |
|
10,622 |
|
121 |
|
|
(143,344 |
) |
|
102,575 |
|
|
(173 |
) |
|
102,402 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
PROQR THERAPEUTICS N.V. |
Unaudited Condensed Consolidated Statement of
Cash Flows |
|
|
|
|
|
|
|
|
|
|
|
Three month period |
|
|
Nine month period |
|
|
|
ended September 30, |
|
|
ended September 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 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
ProQR Therapeutics NV (NASDAQ:PRQR)
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From Aug 2024 to Sep 2024
ProQR Therapeutics NV (NASDAQ:PRQR)
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From Sep 2023 to Sep 2024