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Prosensa Announces Full Year 2013 Financial Results and Recent Corporate Developments
Prosensa Committed to Defining a Path Forward for Its Lead Product, Drisapersen
LEIDEN, The Netherlands, March 18, 2014 (GLOBE NEWSWIRE) -- Prosensa Holding N.V. (Nasdaq:RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today reported financial results for the full year ending December 31, 2013, and provided an update on the next steps for its exon-skipping platform for the treatment of Duchenne Muscular Dystrophy (DMD).
"As we have previously stated, we are encouraged by initial findings from further analyses of the aggregate drisapersen data that suggest that treating earlier in DMD and treating longer shows a delay in the progression of the disease. These data give us the confidence to engage patient groups, clinical experts and regulators to explore a path forward for drisapersen," said Hans Schikan, CEO of Prosensa.
Mr. Schikan continued, "While the past few months have been a very important transition period for Prosensa, our primary focus has not changed, which is to improve the lives and outcomes of boys with DMD. At the start of the year we regained the rights to drisapersen and retained rights to our other development-stage compounds for DMD from GlaxoSmithKline (GSK). We are currently working closely with GSK to transfer data and other elements covered in the collaboration as soon as practicable and within 120 days of the January 12 effective date of the agreement. Concurrently, we are focused on the continued analysis of the complete drisapersen data set, preparing for possible re-dosing of boys and having a meaningful dialogue with regulatory authorities. We expect to communicate the status of these events during the second quarter of this year."
With data from more than 300 patients, Prosensa has the largest clinical data set in DMD. The Company is actively working to define the most optimal path forward for its DMD pipeline, which includes formulating a re-dosing plan for boys who have previously participated in clinical trials. In addition, it has enrolled 190 patients in a planned 250 patient study to better understand the natural history of DMD and potential biomarkers. Prosensa's current portfolio of drug candidates includes six compounds for the treatment of DMD with four compounds currently in clinical development, all of which have received orphan drug status in the United States and the European Union. The compounds utilize an innovative technique called exon-skipping to provide a personalized medicine approach to treat different populations of DMD patients. Furthermore, the Company has progressed PROSPECT, a unique research program which could enable expedited development of DMD therapeutic candidates that target multiple exons.
At of the end of 2013, Prosensa had a cash position of EUR82.2 million.
Recent Corporate Highlights
-- Updates on the Drisapersen Development Program
-- Rare Disease Expert Joined Company Supervisory Board
-- New FP7 Grant Awarded for Development of DMD Imaging Biomarkers
Financial Highlights
Upcoming Conferences
Prosensa management will be participating in the following conferences and events:
Conference Call / Webcast Information
Prosensa will host a conference call on March 18 at 8:00am ET, 1:00pm CET to discuss the full year 2013 financial results and 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 www.prosensa.com .
About Prosensa Holding N.V.
Prosensa (Nasdaq:RNA) is an innovative biotechnology company engaged in the discovery and development of ribonucleic acid-modulating, or 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, myotonic dystrophy and Huntington's disease. Its clinical portfolio of RNA-based product candidates is focused on the treatment of Duchenne muscular dystrophy, or DMD. Each of its DMD compounds has been granted orphan drug status in the United States and the European Union. Its first product candidate, drisapersen, can address a variety of mutations in the dystrophin gene, such as a deletion of exon 50 or exons 48 to 50.
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 and 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 containing this press release and the Company's Annual Report on Form 20-F. In addition, any forward-looking statements represent its 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 disclaim any obligation to do so, even if its views change.
CONTACT: Prosensa Holding N.V.
Celia Economides, Senior Director IR &
Corporate Communications
Phone: +1 917 941 9059
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