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 Type 10 (LCA10), today
announced results for the second quarter of 2016.
“We continue to make good progress with our three different
development programs, all RNA oligonucleotides for serious genetic
diseases. For QR-010 for patients with CF due to the ∆F508
mutation, we will present top-level results from clinical
proof-of-concept study, PQ-010-002 later this year during the North
American Cystic Fibrosis Conference (NACFC)", said Daniel de Boer,
Chief Executive Officer of ProQR. “For QR-110 for patients with
LCA10, we presented strong pre-clinical proof-of-concept data at
the Association for Research in Vision and Ophthalmology (ARVO)
conference. We plan to move this program towards the clinic this
year. For QR-313 for dystrophic epidermolysis bullosa (DEB)
patients due to mutations in Exon 73, we continue to strengthen the
pre-clinical proof-of-concept in important clinically relevant
models. In addition to our three molecules in development, we are
strengthening our pipeline of RNA oligonucleotides for severe
genetic diseases through our innovation unit.”
Financial Highlights
At June 30, 2016, ProQR held cash and cash equivalents of €76.3
million, compared to €85.5 million at March 31, 2016. Net cash used
in operating activities during the three month period ended June
30, 2016 was €8.3 million, compared to €5.9 million for the same
period last year.
Research and development costs increased to €8.6 million for the
quarter ended June 30, 2016 from €5.4 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 increase in expenses
was primarily due to the advancement of our pipeline, which
included clinical development of QR-010, preclinical development of
QR-110 and progress of our innovation programs such as QR-313 for
epidermolysis bullosa, which moved into pre-clinical development
this quarter and other pipeline programs.
General and administrative costs increased to €2.6 million for
the quarter ended June 30, 2016 from €1.8 million for the same
period last year, in line with the growth of our organization.
Net result for the three month period ended June 30, 2016 was a
€10.0 million loss or €0.43 per share, compared to a €8.6 million
loss or €0.37 per share for the same period last year. For further
financial information for the period ending June 30, 2016, please
refer to the financial statements appearing at the end of this
release.
Corporate Highlights
- QR-010 for patients with CF due to the ∆F508 mutation is being
studied in two global clinical trials. PQ-010-002 is a
proof-of-concept study evaluating the effect of QR-010 on an
important measurement of CFTR function, the nasal potential
difference (NPD). PQ-010-002 is an open label 28-day study of
topical exposure of QR-010 in 16 CF patients, 8 homozygous
(carrying two copies) for the ΔF508 mutation and 8 compound
heterozygous (one copy of the ΔF508 plus one other CF disease
causing mutation) with the option to enroll an additional 16.
Top-line data from the first 16 patients is expected to be
presented during the NACFC, Orlando, Florida, October 27 - 29,
2016. The other study, PQ-010-001 is a Phase 1b randomized,
double-blind, placebo-controlled, dose-escalation 28-day study. The
safety, tolerability and pharmacokinetics of single and multiple
ascending doses of inhaled QR-010 will be evaluated in 64 CF
patients carrying two copies (homozygotes) of the ∆F508 mutation.
In addition, exploratory efficacy endpoints in this study include
sweat chloride, weight gain, CFQ-R Respiratory Symptom Score and
lung function, measured by FEV1. This study is not powered for
statistical significance on any of these exploratory endpoints. The
company expects to present preliminary safety data from the single
ascending dose cohorts at the same time as results for study
PQ-010-002.
- At the 2016 European Cystic Fibrosis Conference (ECFS, June 10
2016) the company presented additional pre-clinical data for QR-010
in a presentation titled “QR-010 penetrates the CF-like mucus
barrier in vitro and in vivo.” QR-010 was shown to diffuse rapidly
through CF-like mucus layers in in vitro and in vivo models. QR-010
was also shown to be stable in CF sputum, in the presence of CF
lung bacteria and in the presence of clinically relevant levels of
CF standard-of-care.
- QR-110, for patients with LCA10 due to the p.Cys998X mutation
received orphan drug designation (ODD) from both the U.S. Food and
Drug Administration (FDA) and the European Medicines Agency (EMA).
ODD in the U.S. and the European Union confers a special status for
investigational drugs that are being developed for rare
diseases.
- ProQR strengthened its Supervisory Board with the appointment
of James Shannon, MD in June 2016. James was the former Chief
Medical Officer at GlaxoSmithKline and Global Head of Pharma
Development at Novartis. We believe that James’ broad knowledge and
expertise in drug development and pharma will be of significant
value to the Supervisory Board.
- The company moved headquarters to Zernikedreef 9, 2333 CK
Leiden, the Netherlands.
Subsequent events
- The company has moved its product candidate for the third
program, QR-313 (previously named QRX-313) into pre-clinical
development for the treatment of dystrophic epidermolysis bullosa
(DEB). QR-313 is an RNA oligonucleotide designed to induce the
exclusion of a part of the RNA (exon skipping) that contains a
disease causing mutation with the aim to restore functional COL7a1
protein and with that the anchoring fibrils that bind the layers of
skin together. QR-313 is the second program to be added to the
pipeline behind the CF and LCA10 programs from ProQR’s internal
innovation (discovery) unit.
- QR-010 received a Fast Track designation by the US Food and
Drug Administration (FDA). Drugs that are under development for
serious conditions and have the potential to fulfill an unmet
medical need can receive this designation. It was established with
the intention to bring promising drugs to patients sooner by
facilitating the development with more frequent FDA interactions
and expediting the review process.
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.
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
in CF patients that have the DF508 mutation. 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 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. We believe this route of administration 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 QR-110
QR-110 is a first-in-class RNA-based oligonucleotide designed to
address the underlying cause of Leber’s congenital amaurosis Type
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.
About QR-313
QR-313 is a first-in-class RNA-based oligonucleotide designed to
address the underlying cause of dystrophic epidermolysis bullosa
(DEB) due to mutations in exon 73 of the COL7A1 gene. Mutations in
this exon can cause loss of functional collagen type VII (C7)
protein. Absence of C7 results in the loss of anchoring fibrils
that normally link the dermal and epidermal layers of the skin
together. QR-313 is designed to exclude exon 73 from the mRNA (exon
skipping) and produce truncated but functional C7 protein and
thereby restores functionality of the anchoring fibrils.
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 QR-313, and the clinical development and the therapeutic
potential thereof, statements regarding our ongoing and planned
discovery and development of product candidates, including those in
our innovation pipeline, statements regarding release of clinical
data, statements regarding the appointment of Dr. Shannon, 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, except as required by
law.
ProQR Therapeutics N.V.:Sariette WitteHead of
CommunicationsT: +1 213 261 8891ir@proqr.com
|
PROQR THERAPEUTICS N.V. |
Unaudited Condensed Consolidated Statement of
Financial Position |
|
|
June 30, |
|
December 31, |
|
|
2016 |
|
2015 |
|
|
€ 1,000 |
|
€ 1,000 |
Assets |
|
|
|
|
Current
assets |
|
|
|
|
Cash and cash
equivalents |
|
76,311 |
|
94,865 |
Prepayments and other
receivables |
|
3,185 |
|
1,948 |
Social securities and other
taxes |
|
651 |
|
956 |
|
|
|
|
|
|
|
|
|
|
Total current assets |
|
80,147 |
|
97,769 |
|
|
|
|
|
Property, plant and
equipment |
|
3,670 |
|
2,199 |
Intangible assets |
|
116 |
|
141 |
|
|
|
|
|
Total assets |
|
83,933 |
|
100,109 |
|
|
|
|
|
Liabilities and shareholders' equity |
|
|
|
|
Current liabilities |
|
|
|
|
Finance lease
liabilities |
|
-- |
|
15 |
Trade payables |
|
1,038 |
|
885 |
Social securities and other
taxes |
|
303 |
|
235 |
Pension premiums |
|
43 |
|
16 |
Deferred income |
|
-- |
|
144 |
Other current
liabilities |
|
6,349 |
|
4,191 |
|
|
|
|
|
Total current liabilities |
|
7,733 |
|
5,486 |
|
|
|
|
|
Borrowings |
|
5,267 |
|
4,824 |
|
|
|
|
|
Total liabilities |
|
13,000 |
|
10,310 |
|
|
|
|
|
Shareholders' equity |
|
|
|
|
Shareholders' equity |
|
70,933 |
|
89,799 |
|
|
|
|
|
Total liabilities and shareholders' equity |
|
83,933 |
|
100,109 |
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 |
|
|
|
Six month period |
|
|
|
|
ended June 30, |
|
|
|
ended June 30, |
|
|
|
|
2016 |
|
|
|
2015 |
|
|
|
2016 |
|
|
|
2015 |
|
|
|
|
€ 1,000 |
|
|
|
€ 1,000 |
|
|
|
€ 1,000 |
|
|
|
€ 1,000 |
|
Other income |
|
|
589 |
|
|
|
748 |
|
|
|
1,278 |
|
|
|
1,086 |
|
|
|
|
|
|
|
|
|
|
Research
and development costs |
|
|
(8,606 |
) |
|
|
(5,427 |
) |
|
|
(15,504 |
) |
|
|
(10,907 |
) |
General
and administrative costs |
|
|
(2,615 |
) |
|
|
(1,777 |
) |
|
|
(5,217 |
) |
|
|
(3,380 |
) |
|
|
|
|
|
|
|
|
|
Total operating costs |
|
|
(11,221 |
) |
|
|
(7,204 |
) |
|
|
(20,721 |
) |
|
|
(14,287 |
) |
|
|
|
|
|
|
|
|
|
Operating result |
|
|
(10,632 |
) |
|
|
(6,456 |
) |
|
|
(19,443 |
) |
|
|
(13,201 |
) |
Finance income and expense |
|
|
673 |
|
|
|
(2,168 |
) |
|
|
(714 |
) |
|
|
4,812 |
|
|
|
|
|
|
|
|
|
|
Result before corporate income taxes |
|
|
(9,959 |
) |
|
|
(8,624 |
) |
|
|
(20,157 |
) |
|
|
(8,389 |
) |
Income
taxes |
|
|
-- |
|
|
|
-- |
|
|
|
-- |
|
|
|
-- |
|
|
|
|
|
|
|
|
|
|
Net loss attributable to equity holders of the
Company |
|
|
(9,959 |
) |
|
|
(8,624 |
) |
|
|
(20,157 |
) |
|
|
(8,389 |
) |
Other
comprehensive income |
|
|
(5 |
) |
|
|
-- |
|
|
|
0 |
|
|
|
-- |
|
|
|
|
|
|
|
|
|
|
Total comprehensive loss (attributable to
equity holders of the Company) |
|
|
(9,964 |
) |
|
|
(8,624 |
) |
|
|
(20,157 |
) |
|
|
(8,389 |
) |
|
|
|
|
|
|
|
|
|
Share information |
|
|
|
|
|
|
|
|
Weighted
average number of shares outstanding1 |
|
|
23,346,340 |
|
|
|
23,343,253 |
|
|
|
23,346,153 |
|
|
|
23,340,971 |
|
|
|
|
|
|
|
|
|
|
Earnings per share attributable to the equity holders of
the Company (expressed in Euro per share) |
|
|
|
|
|
|
|
|
Basic loss per share1 |
|
|
(0.43 |
) |
|
|
(0.37 |
) |
|
|
(0.86 |
) |
|
|
(0.36 |
) |
Diluted loss per share1 |
|
|
(0.43 |
) |
|
|
(0.37 |
) |
|
|
(0.86 |
) |
|
|
(0.36 |
) |
|
|
|
|
|
|
|
|
|
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 |
|
|
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, 2015 |
|
23,338,154 |
|
934 |
|
123,581 |
|
687 |
|
-- |
|
|
(15,798 |
) |
|
|
109,404 |
|
Net
loss |
|
-- |
|
-- |
|
-- |
|
-- |
|
-- |
|
|
(8,389 |
) |
|
|
(8,389 |
) |
Recognition of share-based payments |
|
-- |
|
-- |
|
-- |
|
619 |
|
-- |
|
-- |
|
|
619 |
|
Share
options exercised |
|
5,217 |
|
0 |
|
6 |
|
-- |
|
-- |
|
-- |
|
|
6 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at June 30, 2015 |
|
23,343,371 |
|
934 |
|
123,587 |
|
1,306 |
|
-- |
|
|
(24,187 |
) |
|
|
101,640 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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 |
|
PROQR THERAPEUTICS N.V. |
Unaudited Condensed Consolidated Statement of
Cash Flows |
|
|
|
Three month period |
|
|
|
Six month period |
|
|
|
|
ended June 30, |
|
|
|
ended June 30, |
|
|
|
|
2016 |
|
|
|
2015 |
|
|
|
2016 |
|
|
|
2015 |
|
|
|
|
€
1,000 |
|
|
|
€
1,000 |
|
|
|
€
1,000 |
|
|
|
€
1,000 |
|
Cash flows from operating activities |
|
|
|
|
|
|
|
|
Net
result |
|
|
(9,964 |
) |
|
|
(8,624 |
) |
|
|
(20,157 |
) |
|
|
(8,389 |
) |
Adjustments for: |
|
|
|
|
|
|
|
|
—
Depreciation |
|
|
360 |
|
|
|
117 |
|
|
|
694 |
|
|
|
212 |
|
—
Share-based compensation |
|
|
699 |
|
|
|
331 |
|
|
|
1,289 |
|
|
|
619 |
|
—
Financial income and expenses |
|
|
(673 |
) |
|
|
2,168 |
|
|
|
714 |
|
|
|
(4,812 |
) |
|
|
|
|
|
|
|
|
|
Changes
in working capital |
|
|
1,242 |
|
|
|
(16 |
) |
|
|
1,292 |
|
|
|
999 |
|
Cash used
in operations |
|
|
(8,336 |
) |
|
|
(6,024 |
) |
|
|
(16,168 |
) |
|
|
(11,371 |
) |
|
|
|
|
|
|
|
|
|
Corporate
income tax paid |
|
|
-- |
|
|
|
-- |
|
|
|
-- |
|
|
|
-- |
|
Interest
received/(paid) |
|
|
1 |
|
|
|
105 |
|
|
|
66 |
|
|
|
177 |
|
|
|
|
|
|
|
|
|
|
Net cash used in operating activities |
|
|
(8,335 |
) |
|
|
(5,919 |
) |
|
|
(16,102 |
) |
|
|
(11,194 |
) |
|
|
|
|
|
|
|
|
|
Cash flow from investing activities |
|
|
|
|
|
|
|
|
Purchases
of intangible assets |
|
|
-- |
|
|
|
(28 |
) |
|
|
-- |
|
|
|
(28 |
) |
Purchases
of property, plant and equipment |
|
|
(1,571 |
) |
|
|
(549 |
) |
|
|
(2,073 |
) |
|
|
(1,037 |
) |
|
|
|
|
|
|
|
|
|
Net cash used in investing activities |
|
|
(1,571 |
) |
|
|
(577 |
) |
|
|
(2,073 |
) |
|
|
(1,065 |
) |
|
|
|
|
|
|
|
|
|
Cash flow from financing activities |
|
|
|
|
|
|
|
|
Proceeds
from exercise of share options |
|
|
2 |
|
|
|
1 |
|
|
|
2 |
|
|
|
6 |
|
Proceeds
from borrowings |
|
|
-- |
|
|
|
1,254 |
|
|
|
193 |
|
|
|
1,254 |
|
Redemption of financial lease |
|
|
(7 |
) |
|
|
(8 |
) |
|
|
(15 |
) |
|
|
(20 |
) |
|
|
|
|
|
|
|
|
|
Net cash generated by financing activities |
|
|
(5 |
) |
|
|
1,247 |
|
|
|
180 |
|
|
|
1,240 |
|
|
|
|
|
|
|
|
|
|
Net increase/(decrease) in cash and cash
equivalents |
|
|
(9,911 |
) |
|
|
(5,249 |
) |
|
|
(17,995 |
) |
|
|
(11,019 |
) |
|
|
|
|
|
|
|
|
|
Currency
effect cash and cash equivalents |
|
|
755 |
|
|
|
(2,184 |
) |
|
|
(559 |
) |
|
|
4,665 |
|
Cash and
cash equivalents, at beginning of the period |
|
|
85,467 |
|
|
|
113,815 |
|
|
|
94,865 |
|
|
|
112,736 |
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents at the end of the
period |
|
|
76,311 |
|
|
|
106,382 |
|
|
|
76,311 |
|
|
|
106,382 |
|
ProQR Therapeutics NV (NASDAQ:PRQR)
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From Apr 2024 to May 2024
ProQR Therapeutics NV (NASDAQ:PRQR)
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From May 2023 to May 2024