Prosensa Announces 3rd Quarter 2014 Financial Results and Recent Corporate Developments
(Thomson Reuters ONE) -
* Rolling NDA submission drisapersen underway
* EMA filing for conditional approval anticipated to follow NDA submission
* Re-dosing has commenced in United States, Belgium & Sweden
* Cash & cash equivalents at quarter-end ?62M
LEIDEN, The Netherlands, Nov. 17, 2014 (GLOBE NEWSWIRE) -- Prosensa Holding N.V.
(Nasdaq:RNA), the biopharmaceutical company focusing on RNA-modulating
therapeutics for rare diseases with high unmet need, today reported financial
results for the third quarter ending September 30, 2014 and provided an update
on the regulatory plans and re-dosing for drisapersen, our lead exon-skipping
drug candidate, and next steps for our follow-on exon skipping compounds for the
treatment of Duchenne muscular dystrophy (DMD).
Hans Schikan, CEO of Prosensa said, "We are incredibly pleased with the
substantial progress we have accomplished during this period. We have delivered
on our promise of initiating the re-dosing process for patients who were
previously in drisapersen clinical trials with a total of 15 boys now back on
treatment, and our rolling New Drug Application (NDA) submission with the US
Food & Drug Administration (FDA) is currently well underway. We continue to work
as diligently and quickly as possible to fulfill our promise of bringing much
needed therapies to boys with DMD."
Recent Corporate Highlights
Drisapersen Development Program
* On September 17, we announced that a staged program of re-dosing had
commenced, with the first patients re-dosed in the United States. The re-
dosing program in North America will include up to 72 patients across 14
sites who had participated in the drisapersen DEMAND V (Phase II) & DEMAND
III (Phase III) studies.
* On September 26, we announced that we had extended our re-dosing program of
drisapersen in patients with DMD into Europe, starting with Belgium.
* On October 10, we announced that we submitted the first module for an NDA
regulatory filing for drisapersen to the FDA for treating DMD. Drisapersen
was granted "Fast Track status" and "Breakthrough Therapy designation" from
the FDA, making it eligible for a rolling review of the NDA.
Publications & Scientific Presentations
* On September 8, we announced that results from an exploratory, double-blind,
placebo-controlled Phase II study (DEMAND II/DMD114117) of drisapersen in
patients with DMD were published in The Lancet Neurology. The publication of
the results of the study, which investigated the efficacy and safety of
drisapersen over 48 weeks, describes the positive outcome of this study,
meeting its primary endpoint, without any reported major safety concerns.
Top-line study results were first presented in April 2013.
* On September 24, the results of our research into developing an accurate and
reproducible method for the measurement of dystrophin in patients with DMD
and Becker's muscular dystrophy (BMD) were published in the online peer-
reviewed journal PLOS ONE. The publication, by Chantal Beekman et al from
Prosensa, describes the Company's semi-automated image analysis method,
which was shown to be objective (operator independent), reproducible (in
multiple samples and experiments) and sensitive for assessing dystrophin
levels by immunofluorescence in muscle biopsies from BMD and DMD patients in
natural history studies or clinical studies with compounds aiming to restore
dystrophin expression.
* On October 7-11, during the 19(th) International Congress of the World
Muscle Society (WMS) in Berlin, Germany, we and our collaborators presented
12 abstracts, including abstracts discussing biomarkers to measure disease
progression, clinical results and our pre-clinical work on multi-exon
skipping, which could address the rarer mutations (region 10-40) in the
dystrophin gene.
Financial Highlights
* Net loss: Net loss for the third quarter 2014 was ?10.6 million, or ?0.29
basic and diluted loss per share, compared to a net loss of ?4.3 million, or
?0.12 basic and diluted loss per share, for the third quarter 2013.
* Revenue: Revenue for the three months ended September 30, 2014 was nil,
compared with ?2.4 million in the same period in 2013 due to a decrease in
license revenue of ?1.3 million and a decrease in collaboration revenue of
?1.1 million due to the termination of the research and collaboration
agreement with GSK.
* R&D Expense: Research and development expense increased from ?4.9 million in
the three months ended September 30, 2013 to ?8.4 million in the three
months ended September 30, 2014. Research and development expenses mainly
increased due to the expansion of our development and regulatory
capabilities, directly impacted by the termination of the research and
collaboration agreement with GSK, as well as the costs of preparing a
regulatory file for drisapersen and the progressing clinical studies of
PRO044, PRO045 and PRO053.
* G&A Expense: General and administrative expense increased from ?1.9 million
to ?2.4 million in the three months ended September 30, 2013 and 2014,
respectively. The increase is primarily due to share-based compensation and
costs associated with the regulatory filing in the three months period ended
September 30, 2014 compared to the same period in 2013. On July 3, 2014, we
filed a shelf registration statement (Form F-3) that provides the
flexibility to raise up to $150 million in a primary offering if we choose
to do so.
* Cash Position and Cash Consumption: Cash and cash equivalents as of
September 30, 2014, were ?62.0 million.
2014 Guidance
* We expect that cash and cash equivalents as of December 31, 2014 will range
from ?52 million to ?54 million. This guidance does not account for the
impact of any future acquisitions, dispositions, partnerships, license
transactions or changes to the Company's capital structure, including future
securities offerings.
Conference Call / Webcast Information
Prosensa will host a conference call on November 17, 2014 at 8:00 a.m. US
Eastern Time, 2:00 p.m. Central European Time, to discuss the third quarter
financial results and provide a corporate update. In order to participate in the
conference call, please dial +1-877-407-9170 (US domestic toll-free).
International dial-in numbers and an audio webcast can be accessed under "Events
& Presentations" through the Investors & Media section of the Prosensa corporate
website at www.prosensa.com.
About Prosensa Holding N.V.
Prosensa (Nasdaq:RNA) is a biotechnology company engaged in the discovery and
development of RNA-modulating therapeutics for the treatment of genetic
disorders. Its primary focus is on rare neuromuscular and neurodegenerative
disorders with a large unmet medical need, including Duchenne muscular dystrophy
(DMD), myotonic dystrophy and Huntington's disease.
Prosensa's current portfolio includes six compounds for the treatment of DMD,
all of which have received orphan drug designation in the United States and the
European Union. The compounds use an innovative technique called exon-skipping
to provide a personalized medicine approach to treat different populations of
DMD patients. www.prosensa.com
About DMD
DMD is one of the most prevalent rare genetic diseases globally affecting up to
1 in 3,500 boys and is invariably fatal. There is currently no approved disease-
modifying therapy for DMD. The progressive muscle-wasting that characterizes
this disease is caused by inadequate production of dystrophin, a protein
necessary for muscle function, as a result of mutations in the dystrophin gene.
The different mutations, which are mostly deletions of one or more exons, found
in the dystrophin gene result in distinct sub-populations of DMD patients.
Forward Looking Statement
This press release contains certain forward-looking statements. All statements,
other than statements of historical facts, contained in this press release,
including statements regarding the Company's strategy, future operations, future
financial position, future revenues, projected costs, prospects, plans and
objectives of management, are forward-looking statements. The words
"anticipate," "believe," "estimate," "expect," "intend," "may," "plan,"
"predict," "project," "target," "potential," "will," "would," "could," "should,"
"continue," and similar expressions are intended to identify forward-looking
statements, although not all forward-looking statements contain these
identifying words. Forward-looking statements in this press release include
statements around the Company's exon-skipping drug pipeline, regulatory review
of its product candidates, its intellectual property position and its financial
position. Actual results may differ materially from those projected or implied
in such forward-looking statements. Such forward-looking information involves
risks and uncertainties that could significantly affect expected results. These
risks and uncertainties are discussed in the Company's SEC filings, including,
but not limited to, the Company's Form 6-K's and the Company's Annual Report on
Form 20-F. In addition, any forward-looking statements represent the Company's
views only as of today and should not be relied upon as representing its views
as of any subsequent date. While the Company may elect to update these forward-
looking statements at some point in the future, the Company specifically
disclaims any obligation to do so, even if its views change.
CONDENSED CONSOLIDATED STATEMENT OF COMPREHENSIVE INCOME (UNAUDITED)
Three months ended Nine months ended
September 30, September 30,
2014 2013 2014 2013
-------------------------------------------------------------------------------
? ('000 except per share data)
License revenue -- 1,319 14,695 4,012
Collaboration revenue -- 1,060 60 2,751
------------------------------------------------------
Total revenue -- 2,379 14,755 6,763
Other income 250 186 702 220
Research and development
expense (8,395) (4,919) (19,191) (13,528)
General and
administrative expense (2,403) (1,939) (7,554) (5,808)
Other gains - net 26 11 122 19
------------------------------------------------------
Operating loss (10,522) (4,282) (11,166) (12,334)
Finance income 177 166 633 458
Finance costs (304) (177) (787) (576)
------------------------------------------------------
Finance cost - net (127) (11) (154) (118)
------------------------------------------------------
Net loss (10,649) (4,293) (11,320) (12,452)
------------------------------------------------------
Other comprehensive
income -- -- -- --
------------------------------------------------------
Total comprehensive
loss* (10,649) (4,293) (11,320) (12,452)
------------------------------------------------------
Loss per share from
operations attributable
to the equity holders of
the Company during the
period (in ? per share)
Basic and diluted loss
per share (0.29) (0.12) (0.31) (0.40)
* Total comprehensive loss is fully attributable to equity holders of the
company
BALANCE SHEET HIGHLIGHTS (UNAUDITED)
? ('000) As of September 30, As of December 31,
2014 2013
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Cash and cash equivalents 61,984 82,232
Total assets 70,361 90,790
Total equity 53,495 63,210
Total liabilities 16,866 27,580
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CONTACT: Prosensa Holding N.V.
Celia Economides, Senior Director IR &
Corporate Communications
Phone: +1 917 941 9059
Email: c.economides(at)prosensa.nl
This announcement is distributed by GlobeNewswire on behalf of
GlobeNewswire clients. The owner of this announcement warrants that:
(i) the releases contained herein are protected by copyright and
other applicable laws; and
(ii) they are solely responsible for the content, accuracy and
originality of the information contained therein.
Source: Prosensa Holding N.V. via GlobeNewswire
[HUG#1872008]
Unternehmensinformation / Kurzprofil:
Datum: 17.11.2014 - 13:00 Uhr
Sprache: Deutsch
News-ID 353144
Anzahl Zeichen: 14984
contact information:
Town:
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Kategorie:
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