Key updates
ProQR Therapeutics N.V. (Nasdaq:PRQR), today announced results for
the first quarter of 2017.
“As we announced earlier this week, we are all devastated by the
unexpected passing of our co-founder and vice-chairman of the
supervisory board, Henri A. Termeer,” said Daniel de Boer, Chief
Executive Officer of ProQR. “Henri was a great mentor, a passionate
patient advocate and a key factor in establishing ProQR. His
passion to do the right thing in the interest of patients is
unparalleled. We are honored to have had the opportunity to work
with him so closely and learn from his wealth of experience. He was
a true inspiration for all of us, and we will continue to build on
the path he helped us to set out. Although he will be deeply
missed, our supervisory board, with co-founder and chairman Dinko
Valerio, James Shannon, Alison Lawton and Antoine Papiernik,
continues to be very strong with broad experience in all aspects of
running a biotechnology business and we are extremely motivated to
continue to build on the path he helped us set.”
“With that in mind, this is an important phase for the Company
in our goal to translate our rich pipeline into a diversified
portfolio of development programs. We are very pleased with the
clearance of the IND for QR-110, enabling us to now advance our
second molecule into clinical development aiming to make a
meaningful difference for LCA 10 patients. We are also excited to
add Dave Rodman to our team who will further strengthen our
development team, with the goal of efficiently and rapidly driving
our programs through to patients. Looking forward to the upcoming
summer, we are very excited for the expected completion of the
Phase 1b trial in our lead program QR-010. We have been treating
>64 CF patients with our lead compound QR-010 and are excited to
get to the data and progress the compound into next trials.”
Corporate Highlights
- In March, the Company announced that it appointed David M.
Rodman, MD as Chief Development Strategy Officer. Dr. Rodman has
had a long career in drug development including leadership roles in
translational medicine, rare disease drug development, and RNA
therapeutics. Dr. Rodman’s experience includes a leadership role in
developing two approved medicines for CF at Vertex Pharmaceuticals,
as Vice President and head of respiratory drug development. He was
also the head of translational medicine at Novartis Institute for
Biomedical Research. More recently, he was the Chief Medical
Officer at MiRagen and Nivalis. Expansion of the ProQR management
team will allow the Company to further realize the potential of RNA
therapeutics as well as expand business capabilities needed to
advance the development of our product candidates.
Subsequent events
- In April, the Company announced that with the clearance of the
investigational new drug (IND) application by the U.S. Food and
Drug Administration (FDA), ProQR can now start clinical development
of QR-110 in Leber’s congenital amaurosis Type 10 (LCA 10)
patients. The trial, named PQ-110-001, is an open-label trial that
will include approximately 6 children (age 6-17 years) and 6 adults
(≥ 18 years) that have LCA 10 due to one or two copies of the
p.Cys998X mutation. During the trial, patients will receive four
intravitreal injections of QR-110 into one eye; one every three
months for one year and the second eye will serve as a control. The
QR-110 trial is expected to be conducted in three centers with
significant expertise in genetic retinal disease in the US and
Europe. The primary objective will be to assess safety and
tolerability of QR-110. Secondary objectives are to evaluate
pharmacokinetics and efficacy, which is measured by specialized
ophthalmic tests including visual acuity, full field stimulus
testing (FST), optical coherence tomography (OCT), pupillary light
reflex (PLR) and a mobility course. Fixation stability and changes
in quality of life in LCA subjects will also be evaluated. Top-line
results from the trial are expected to be available in 2018.
- In April, the Company announced the grant of two key patents
protecting QR-010 in the US and EU. These patents provide the
Company exclusive rights for QR-010 for the treatment of CF until
at least July 2033. US patent no. 9,605,255 is directed to methods
of targeting RNA for the most common mutation in CF, called
F508del, using oligonucleotides to restore the function of the CFTR
protein. Last year, ProQR also received the grant of the equivalent
European patent (EP 2 852 668 B1). Apart from these ProQR owned
patents, ProQR has an exclusive license to US patent no. 9,617,535
from Massachusetts General Hospital covering QR-010.
- During the 2017 Annual Meeting of the Association for Research
in Vision and Ophthalmology (ARVO) held on May 7 – 11, 2017 in
Baltimore, MD, USA, the Company presented three abstracts,
including additional positive pre-clinical proof-of-concept data
for QR-110 in LCA 10 and pre-clinical data for two programs,
QRX-411 and QRX-421, each targeting specific mutations that result
in Usher syndrome.
- The Company announced that it will host an R&D Day on June
15th in New York where Company executives and external experts will
present ProQR’s pipeline of development and early stage programs in
detail.
Financial Highlights
At March 31, 2017, ProQR held cash and cash equivalents of €52.1
million, compared to €59.2 million at December 31, 2016. Net cash
used in operating activities during the three month period ended
March 31, 2017 was €8.8 million, compared to €7.8 million for the
same period last year.
Research and development costs increased to €8.0 million for the
quarter ended March 31, 2017 from €6.9 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 and QR-110, pre-clinical
development of QR-313. The remainder represents increased
investments in our other pipeline programs.
General and administrative costs decreased to €2.3 million for
the quarter ended March 31, 2017 from €2.6 million for the same
period last year.
Net result for the three month period ended March 31, 2017 was a
€10.5 million loss or €0.45 per share, compared to a €10.2 million
loss or €0.44 per share for the same period last year. For further
financial information for the period ending March 31, 2017, 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 cystic fibrosis, Leber’s
congenital amaurosis Type 10 and dystrophic epidermolysis
bullosa. 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 F508del mutation. The F508del 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 investigational 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 our
patent estate. 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 |
|
|
|
|
|
|
|
|
|
March 31,2017 |
|
December 31,2016 |
|
|
|
€ 1,000 |
|
€ 1,000 |
|
Assets |
|
|
|
|
|
Current assets |
|
|
|
|
|
Cash and cash equivalents |
|
52,111 |
|
59,200 |
|
Prepayments and other receivables |
|
2,364 |
|
2,420 |
|
Social securities and other taxes |
|
387 |
|
395 |
|
|
|
|
|
|
|
|
|
|
|
|
|
Total current assets |
|
54,862 |
|
62,015 |
|
|
|
|
|
|
|
Property, plant and equipment |
|
3,187 |
|
3,438 |
|
Intangible assets |
|
78 |
|
90 |
|
|
|
|
|
|
|
Total assets |
|
58,127 |
|
65,543 |
|
|
|
|
|
|
|
Liabilities and shareholders' equity |
|
|
|
|
|
Current liabilities |
|
|
|
|
|
Trade payables |
|
177 |
|
328 |
|
Social securities and other taxes |
|
193 |
|
312 |
|
Pension premiums |
|
27 |
|
13 |
|
Other current liabilities |
|
6,153 |
|
6,057 |
|
|
|
|
|
|
|
Total current liabilities |
|
6,550 |
|
6,710 |
|
|
|
|
|
|
|
Borrowings |
|
5,840 |
|
5,697 |
|
|
|
|
|
|
|
Total liabilities |
|
12,390 |
|
12,407 |
|
|
|
|
|
|
|
Shareholders' equity |
|
|
|
|
|
Shareholders' equity |
|
45,737 |
|
53,136 |
|
|
|
|
|
|
|
Total liabilities and shareholders' equity |
|
58,127 |
|
65,543 |
|
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 March
31, |
|
|
|
2017 |
|
|
2016 |
|
|
|
|
€ 1,000 |
|
|
€ 1,000 |
|
|
Other income |
|
393 |
|
|
689 |
|
|
|
|
|
|
|
|
|
| Research
and development costs |
|
(8,030 |
) |
|
(6,898 |
) |
| General
and administrative costs |
|
(2,304 |
) |
|
(2,602 |
) |
|
|
|
|
|
|
|
|
|
Total operating costs |
|
(10,334 |
) |
|
(9,500 |
) |
|
|
|
|
|
|
|
|
|
Operating result |
|
(9,941 |
) |
|
(8,811 |
) |
| Finance
income and expense |
|
(537 |
) |
|
(1,387 |
) |
|
|
|
|
|
|
|
|
|
Result before corporate income taxes |
|
(10,478 |
) |
|
(10,198 |
) |
| Income
taxes |
|
(2 |
) |
|
-- |
|
|
|
|
|
|
|
|
|
|
Net result attributable to equity holders of the
Company |
|
(10,480 |
) |
|
(10,198 |
) |
| Other
comprehensive income |
|
2 |
|
|
5 |
|
|
|
|
|
|
|
|
|
|
Total comprehensive income (attributable to equity holders
of the Company) |
|
(10,478 |
) |
|
(10,193 |
) |
|
|
|
|
|
|
|
|
|
Share information |
|
|
|
|
|
|
| Weighted
average number of shares outstanding1 |
|
23,473,221 |
|
|
23,345,965 |
|
|
|
|
|
|
|
|
|
|
Earnings per share attributable to the equity holders of
the Company (expressed in Euro per share) |
|
|
|
|
|
|
| Basic
loss per share1 |
|
(0.45 |
) |
|
(0.44 |
) |
| Diluted
loss per share1 |
|
(0.45 |
) |
|
(0.44 |
) |
| |
|
|
|
|
|
|
- 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 |
|
Total Share
Capital |
|
Share Premium |
|
Equity Settled
Employee Benefit
Reserve |
|
TranslationReserve |
|
Accumulated
Deficit |
|
Total Equity |
|
|
|
|
|
€ 1,000 |
|
€ 1,000 |
|
€ 1,000 |
|
€ 1,000 |
|
|
€ 1,000 |
|
|
€ 1,000 |
|
|
Balance at January 1, 2016 |
|
23,345,965 |
|
934 |
|
123,595 |
|
1,899 |
|
1 |
|
|
(36,630 |
) |
|
89,799 |
|
| Net
loss |
|
-- |
|
-- |
|
-- |
|
-- |
|
-- |
|
|
(10,198 |
) |
|
(10,198 |
) |
| Other
comprehensive income |
|
-- |
|
-- |
|
-- |
|
-- |
|
5 |
|
|
-- |
|
|
5 |
|
|
Recognition of share-based payments |
|
-- |
|
-- |
|
-- |
|
590 |
|
-- |
|
|
-- |
|
|
590 |
|
| Share
options exercised |
|
-- |
|
-- |
|
-- |
|
-- |
|
-- |
|
|
-- |
|
|
-- |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at March 31, 2016 |
|
23,345,965 |
|
934 |
|
123,595 |
|
2,489 |
|
6 |
|
|
(46,828 |
) |
|
80,196 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at January 1, 2017 |
|
23,346,856 |
|
934 |
|
123,597 |
|
4,353 |
|
(15 |
) |
|
(75,733 |
) |
|
53,136 |
|
| Net
loss |
|
-- |
|
-- |
|
-- |
|
-- |
|
-- |
|
|
(10,480 |
) |
|
(10,480 |
) |
| Other
comprehensive income |
|
-- |
|
-- |
|
-- |
|
-- |
|
2 |
|
|
-- |
|
|
2 |
|
|
Recognition of share-based payments |
|
-- |
|
-- |
|
-- |
|
927 |
|
-- |
|
|
-- |
|
|
927 |
|
| Shares
issued in the period |
|
518,162 |
|
21 |
|
2,130 |
|
-- |
|
-- |
|
|
-- |
|
|
2,151 |
|
| Share
options exercised |
|
127 |
|
0 |
|
1 |
|
-- |
|
-- |
|
|
-- |
|
|
0 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Balance at March 31, 2017 |
|
23,865,145 |
|
955 |
|
125,728 |
|
5,280 |
|
(13 |
) |
|
(86,213 |
) |
|
45,737 |
|
|
PROQR THERAPEUTICS N.V. |
|
Unaudited Condensed Consolidated Statement of Cash
Flows |
|
|
|
|
| |
|
Three month period ended March
31, |
|
|
|
2017 |
|
|
2016 |
|
|
|
|
€ 1,000 |
|
|
€ 1,000 |
|
|
Cash flows from operating activities |
|
|
|
|
|
|
| Net
result |
|
(10,478 |
) |
|
(10,193 |
) |
|
Adjustments for: |
|
|
|
|
|
|
| —
Depreciation |
|
268 |
|
|
334 |
|
| —
Share-based compensation |
|
927 |
|
|
590 |
|
| —
Financial income and expenses |
|
537 |
|
|
1,387 |
|
|
|
|
|
|
|
|
|
| Changes
in working capital |
|
(93 |
) |
|
50 |
|
| Cash used
in operations |
|
(8,839 |
) |
|
(7,832 |
) |
|
|
|
|
|
|
|
|
| Corporate
income tax paid |
|
(2 |
) |
|
-- |
|
| Interest
received/(paid) |
|
58 |
|
|
65 |
|
|
|
|
|
|
|
|
|
|
Net cash used in operating activities |
|
(8,783 |
) |
|
(7,767 |
) |
|
|
|
|
|
|
|
|
|
Cash flow from investing activities |
|
|
|
|
|
|
| Purchases
of intangible assets |
|
-- |
|
|
-- |
|
| Purchases
of property, plant and equipment |
|
(45 |
) |
|
(502 |
) |
|
|
|
|
|
|
|
|
|
Net cash used in investing activities |
|
(45 |
) |
|
(502 |
) |
|
|
|
|
|
|
|
|
|
Cash flow from financing activities |
|
|
|
|
|
|
| Proceeds
from issuance of shares, net of transaction costs |
|
2,151 |
|
|
-- |
|
| Proceeds
from exercise of share options |
|
1 |
|
|
-- |
|
| Proceeds
from borrowings |
|
-- |
|
|
193 |
|
|
Redemption of financial lease |
|
-- |
|
|
(8 |
) |
|
|
|
|
|
|
|
|
|
Net cash generated by financing activities |
|
2,152 |
|
|
185 |
|
|
|
|
|
|
|
|
|
|
Net increase/(decrease) in cash and cash
equivalents |
|
(6,676 |
) |
|
(8,084 |
) |
|
|
|
|
|
|
|
|
| Currency
effect cash and cash equivalents |
|
(413 |
) |
|
(1,314 |
) |
| Cash and
cash equivalents, at beginning of the period |
|
59,200 |
|
|
94,865 |
|
|
|
|
|
|
|
|
|
|
Cash and cash equivalents at the end of the
period |
|
52,111 |
|
|
85,467 |
|
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