ProQR Announces Results for the Third Quarter of 2016
November 14 2016 - 7:00AM
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 third quarter of 2016.
“This quarter we completed our QR-010 nasal potential difference
study and in October, we reported positive results from this study
in homozygous ∆F508 patients. The outcomes were both statistically
significant and clinically meaningful, marking an important step
for about half of the global CF population.” said Daniel de Boer,
Chief Executive Officer of ProQR “I’m proud of the team that has
designed and executed this study in the most rigorous way leading
to robust clinical proof of concept in the early phase of our
development program. I also want to thank the patients that
participated, and the clinical investigators that supported this
unique and important trial”.
Financial Highlights
At September 30, 2016, ProQR held cash and cash
equivalents of €64.9 million, compared to €76.3 million at June 30,
2016. Net cash used in operating activities during the three month
period ended September 30, 2016 was €10.8 million, compared to €6.3
million for the same period last year.
Research and development costs increased to €8.3
million for the quarter ended September 30, 2016 from €6.0 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 for our clinical
studies, 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 for CF, preparations for the start of the first clinical
trial of QR-110 for LCA10, and preclinical development activities
of QR-313 for epidermolysis bullosa.
General and administrative costs increased to €2.0
million for the quarter ended September 30, 2016 from €1.5 million
for the same period last year, primarily due to increased
investments in our facilities and our support organization.
Net result for the three month period ended
September 30, 2016 was a €10.1 million loss or €0.43 per share,
compared to a €6.3 million loss or €0.27 per share for the same
period last year. For further financial information for the period
ending September 30, 2016, please refer to the financial statements
appearing at the end of this release.
Corporate Highlights
- In July 2016, 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.
- During the 12th Annual Meeting of the Oligonucleotide
Therapeutics Society (OTS) September 25 – 28, 2016 the company
presented a poster titled: ‘QR-010 Restores CFTR Function in Models
of ∆F508 mediated Cystic Fibrosis’. The poster summarized some of
the exciting pre-clinical work published earlier and new data
showing that repeated nebulization of QR-010 did not change the
diffusion speed of QR-010 in in vitro models of CF-like mucus. The
poster also featured new data showing that QR-010 was stable in the
presence of clinically relevant levels of several CF
standard-of-care therapies.
- During OTS, the company also presented a poster titled ‘QR-110
Treatment for Leber’s Congenital Amaurosis Type 10 due to the
p.Cys998X Mutation in CEP290’. This data shows that QR-110 can
restore CEP290 mRNA and protein levels in primary LCA10 compound
heterozygous patient cells and homozygous optic cups in a dose
dependent manner. Based on this data, and other extensive
preclinical work, the company plans to start a first-in-human study
in adult and pediatric subjects in the first half of 2017.
- This quarter, the company advanced 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
COL7A1 RNA (exon skipping) that contains a disease causing mutation
with the aim to restore functional collagen type VII (C7) protein
and with that the anchoring fibrils that bind the layers of skin
together. The clinical program for QR-313 is expected to start in
2018.
Subsequent events
- During the North American Cystic Fibrosis conference (NACFC)
October 26 – 29, 2016 the company presented positive results from
PQ-010-002, a proof-of-concept study demonstrating that QR-010
restores CFTR function in patients homozygous for ∆F508. CFTR is
the protein channel that is defective in patients with CF, and
presence or absence of function of CFTR can be measured with the
nasal potential difference (NPD) assay. Following 4 weeks of
topical therapy, QR-010 improved the CFTR-mediated total chloride
response, a direct measure of CFTR function. QR-010 also restored
other indicators of CFTR function. In subjects that were compound
heterozygous for the ∆F508 mutation, no meaningful difference was
measured. QR-010 was observed to be safe and well-tolerated in all
subjects.
- During NACFC the company also announced that clinical study
PQ-010-001 completed all four single-dose cohorts and blinded
safety data from all cohorts was shared. PQ-010-001 is a
placebo-controlled Phase 1b study in subjects with CF homozygous
for ∆F508. QR-010 was observed to be safe and well-tolerated in all
cohorts. The multiple dose cohorts in this study are ongoing and
topline safety, tolerability and exploratory efficacy data from
this study are expected in mid-2017.
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 targeting the mRNA in CF patients that have the ∆F508
mutation. The ∆F508 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
to 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.
QR-010 has been granted orphan drug designation in the United
States and the European Union and fast-track status by the FDA. 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 and the timing
thereof, including those in our innovation pipeline, statements
regarding release of clinical data, 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, including that positive results
observed in our prior and ongoing studies may not be replicated in
later trials or guarantee approval of any product candidate by
regulatory authorities, 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.:Smital ShahChief
Financial OfficerT: +1 415 231 6431ir@proqr.com
PROQR THERAPEUTICS
N.V. |
Unaudited Condensed Consolidated Statement of Financial
Position |
|
|
September 30, 2016 |
|
December 31, 2015 |
|
|
€ 1,000 |
|
€ 1,000 |
Assets |
|
|
|
|
Current assets |
|
|
|
|
Cash and cash equivalents |
|
64,921 |
|
94,865 |
Prepayments and other receivables |
|
3,662 |
|
1,948 |
Social securities and other taxes |
|
490 |
|
956 |
|
|
|
|
|
|
|
|
|
|
Total current assets |
|
69,073 |
|
97,769 |
|
|
|
|
|
Property, plant and equipment |
|
3,644 |
|
2,199 |
Intangible assets |
|
103 |
|
141 |
|
|
|
|
|
Total assets |
|
72,820 |
|
100,109 |
|
|
|
|
|
Liabilities and shareholders' equity |
|
|
|
|
Current liabilities |
|
|
|
|
Borrowings |
|
1,768 |
|
-- |
Finance lease liabilities |
|
-- |
|
15 |
Trade payables |
|
237 |
|
885 |
Social securities and other taxes |
|
319 |
|
235 |
Pension premiums |
|
22 |
|
16 |
Deferred income |
|
-- |
|
144 |
Other current liabilities |
|
5,415 |
|
4,191 |
|
|
|
|
|
Total current liabilities |
|
7,761 |
|
5,486 |
|
|
|
|
|
Borrowings |
|
3,625 |
|
4,824 |
|
|
|
|
|
Total liabilities |
|
11,386 |
|
10,310 |
|
|
|
|
|
Shareholders' equity |
|
|
|
|
Shareholders' equity |
|
61,434 |
|
89,799 |
|
|
|
|
|
Total liabilities and shareholders' equity |
|
72,820 |
|
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 periodended September
30, |
|
Nine month periodended September
30, |
|
|
|
2016 |
|
|
|
2015 |
|
|
|
2016 |
|
|
|
2015 |
|
|
|
|
€ 1,000 |
|
|
|
€ 1,000 |
|
|
|
€ 1,000 |
|
|
|
€ 1,000 |
|
Other income |
|
|
447 |
|
|
|
1,191 |
|
|
|
1,725 |
|
|
|
2,277 |
|
|
|
|
|
|
|
|
|
|
Research
and development costs |
|
|
(8,319 |
) |
|
|
(6,000 |
) |
|
|
(23,823 |
) |
|
|
(16,907 |
) |
General
and administrative costs |
|
|
(2,001 |
) |
|
|
(1,458 |
) |
|
|
(7,218 |
) |
|
|
(4,838 |
) |
|
|
|
|
|
|
|
|
|
Total operating costs |
|
|
(10,320 |
) |
|
|
(7,458 |
) |
|
|
(31,041 |
) |
|
|
(21,745 |
) |
|
|
|
|
|
|
|
|
|
Operating result |
|
|
(9,873 |
) |
|
|
(6,267 |
) |
|
|
(29,316 |
) |
|
|
(19,468 |
) |
Finance income and expense |
|
|
(254 |
) |
|
|
(50 |
) |
|
|
(968 |
) |
|
|
4,762 |
|
|
|
|
|
|
|
|
|
|
Result before corporate income taxes |
|
|
(10,127 |
) |
|
|
(6,317 |
) |
|
|
(30,284 |
) |
|
|
(14,706 |
) |
Income
taxes |
|
|
-- |
|
|
|
-- |
|
|
|
-- |
|
|
|
-- |
|
|
|
|
|
|
|
|
|
|
Net loss attributable to equity holders of the
Company |
|
|
(10,127 |
) |
|
|
(6,317 |
) |
|
|
(30,284 |
) |
|
|
(14,706 |
) |
Other
comprehensive income |
|
|
-- |
|
|
|
-- |
|
|
|
-- |
|
|
|
-- |
|
|
|
|
|
|
|
|
|
|
Total comprehensive loss (attributable to
equity holders of the Company) |
|
|
(10,127 |
) |
|
|
(6,317 |
) |
|
|
(30,284 |
) |
|
|
(14,706 |
) |
|
|
|
|
|
|
|
|
|
Share information |
|
|
|
|
|
|
|
|
Weighted
average number of shares outstanding1 |
|
|
23,346,856 |
|
|
|
23,345,170 |
|
|
|
23,346,390 |
|
|
|
23,342,386 |
|
|
|
|
|
|
|
|
|
|
Earnings per share attributable to the equity holders of
the Company (expressed in Euro per share) |
|
|
|
|
|
|
|
|
Basic loss per share1 |
|
|
(0.43 |
) |
|
|
(0.27 |
) |
|
|
(1.30 |
) |
|
|
(0.63 |
) |
Diluted loss per share1 |
|
|
(0.43 |
) |
|
|
(0.27 |
) |
|
|
(1.30 |
) |
|
|
(0.63 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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 ofshares |
|
TotalShareCapital |
|
SharePremium |
|
EquitySettledEmployeeBenefitReserve |
|
TranslationReserve |
|
AccumulatedDeficit |
|
TotalEquity |
|
|
|
|
€ 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 |
|
-- |
|
-- |
|
-- |
|
-- |
|
-- |
|
|
(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 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at January 1, 2016 |
|
23,345,965 |
|
934 |
|
123,595 |
|
1,899 |
|
1 |
|
|
(36,630 |
) |
|
|
89,799 |
|
Net
loss |
|
-- |
|
-- |
|
-- |
|
-- |
|
-- |
|
|
(30,284 |
) |
|
|
(30,284 |
) |
Other
comprehensive income |
|
-- |
|
-- |
|
-- |
|
-- |
|
0 |
|
|
-- |
|
|
|
0 |
|
Recognition of share-based payments |
|
-- |
|
-- |
|
-- |
|
1,917 |
|
-- |
|
|
-- |
|
|
|
1,917 |
|
Share
options exercised |
|
891 |
|
0 |
|
2 |
|
-- |
|
-- |
|
|
-- |
|
|
|
2 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at September 30, 2016 |
|
23,346,856 |
|
934 |
|
123,597 |
|
3,816 |
|
1 |
|
|
(66,914 |
) |
|
|
61,434 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
PROQR THERAPEUTICS N.V. |
Unaudited Condensed Consolidated Statement of
Cash Flows |
|
|
Three month periodended September
30, |
|
Nine month periodended September
30, |
|
|
|
2016 |
|
|
|
2015 |
|
|
|
2016 |
|
|
|
2015 |
|
|
|
|
€ 1,000 |
|
|
|
€ 1,000 |
|
|
|
€ 1,000 |
|
|
|
€ 1,000 |
|
Cash flows from operating activities |
|
|
|
|
|
|
|
|
Net
result |
|
|
(10,127 |
) |
|
|
(6,317 |
) |
|
|
(30,284 |
) |
|
|
(14,706 |
) |
Adjustments for: |
|
|
|
|
|
|
|
|
—
Depreciation |
|
|
284 |
|
|
|
126 |
|
|
|
978 |
|
|
|
338 |
|
—
Share-based compensation |
|
|
628 |
|
|
|
300 |
|
|
|
1,917 |
|
|
|
919 |
|
—
Financial income and expenses |
|
|
254 |
|
|
|
50 |
|
|
|
968 |
|
|
|
(4,762 |
) |
|
|
|
|
|
|
|
|
|
Changes
in working capital |
|
|
(1,843 |
) |
|
|
(527 |
) |
|
|
(551 |
) |
|
|
472 |
|
Cash used
in operations |
|
|
(10,804 |
) |
|
|
(6,368 |
) |
|
|
(26,972 |
) |
|
|
(17,739 |
) |
|
|
|
|
|
|
|
|
|
Corporate
income tax paid |
|
|
-- |
|
|
|
-- |
|
|
|
-- |
|
|
|
-- |
|
Interest
received/(paid) |
|
|
11 |
|
|
|
104 |
|
|
|
77 |
|
|
|
281 |
|
|
|
|
|
|
|
|
|
|
Net cash used in operating activities |
|
|
(10,793 |
) |
|
|
(6,264 |
) |
|
|
(26,895 |
) |
|
|
(17,458 |
) |
|
|
|
|
|
|
|
|
|
Cash flow from investing activities |
|
|
|
|
|
|
|
|
Purchases
of intangible assets |
|
|
-- |
|
|
|
-- |
|
|
|
-- |
|
|
|
(28 |
) |
Purchases
of property, plant and equipment |
|
|
(422 |
) |
|
|
(56 |
) |
|
|
(2,495 |
) |
|
|
(1,093 |
) |
|
|
|
|
|
|
|
|
|
Net cash used in investing activities |
|
|
(422 |
) |
|
|
(56 |
) |
|
|
(2,495 |
) |
|
|
(1,121 |
) |
|
|
|
|
|
|
|
|
|
Cash flow from financing activities |
|
|
|
|
|
|
|
|
Proceeds
from exercise of share options |
|
|
-- |
|
|
|
8 |
|
|
|
2 |
|
|
|
14 |
|
Proceeds
from borrowings |
|
|
-- |
|
|
|
-- |
|
|
|
193 |
|
|
|
1,254 |
|
Redemption of financial lease |
|
|
-- |
|
|
|
(7 |
) |
|
|
(15 |
) |
|
|
(27 |
) |
|
|
|
|
|
|
|
|
|
Net cash generated by financing activities |
|
|
-- |
|
|
|
1 |
|
|
|
180 |
|
|
|
1,241 |
|
|
|
|
|
|
|
|
|
|
Net increase/(decrease) in cash and cash
equivalents |
|
|
(11,215 |
) |
|
|
(6,319 |
) |
|
|
(29,210 |
) |
|
|
(17,338 |
) |
|
|
|
|
|
|
|
|
|
Currency
effect cash and cash equivalents |
|
|
(175 |
) |
|
|
(51 |
) |
|
|
(734 |
) |
|
|
4,614 |
|
Cash and
cash equivalents, at beginning of the period |
|
|
76,311 |
|
|
|
106,382 |
|
|
|
94,865 |
|
|
|
112,736 |
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents at the end of the
period |
|
|
64,921 |
|
|
|
100,012 |
|
|
|
64,921 |
|
|
|
100,012 |
|
ProQR Therapeutics NV (NASDAQ:PRQR)
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From Mar 2024 to Apr 2024
ProQR Therapeutics NV (NASDAQ:PRQR)
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From Apr 2023 to Apr 2024