Prosensa Holding N.V.: Prosensa Announces Full Year 2013 Financial Results and Recent Corporate Developments

Prosensa Holding N.V.: 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 €82.2 million.

Recent Corporate Highlights

-- Updates on the Drisapersen Development Program

  oOn January 13, 2014, Prosensa and GlaxoSmithKline (GSK) announced that
    Prosensa regained all rights from GSK to drisapersen and retained rights
    to all other programs for the treatment of DMD. This transfer of rights
    represents the termination of the collaboration agreement between GSK and
    Prosensa executed in 2009 and gives Prosensa full, unencumbered rights to
    continue the development of drisapersen as well as each of its DMD
    programs.
    
  oOn January 16, 2014, Prosensa announced initial findings from further
    analyses of the aggregate data obtained from the clinical development
    program of drisapersen for the treatment of DMD. The analyses, which are
    based on the results from three placebo controlled studies and two long
    term open label studies, suggest that treating earlier in DMD and treating
    longer shows a delay in the progression of the disease, as measured by the
    six-minute walk test (6MWT). Key safety findings are consistent with
    previous observations.
    
  oOn February 18, 2014, the Investigational New Drug (IND) for drisapersen
    was transferred from GSK. As a first step in developing the path forward
    for drisapersen, Prosensa communicated to patient advocacy groups and
    investigators that it is exploring options for study participants to be
    re-dosed with drisapersen. Furthermore, it has initiated a survey to seek
    feedback from patients and investigators regarding the willingness and
    desire of patients to resume treatment. Based on the respondents to date,
    the majority of patients wish to be re-dosed with drisapersen.

-- Rare Disease Expert Joined Company Supervisory Board

  oOn December 10, 2013, Prosensa announced that Dr. Georges Gemayel, an
    expert in the rare disease space and strategic corporate leadership, was
    nominated for the Prosensa Supervisory Board. The appointment of Dr.
    Gemayel was approved in the extraordinary general meeting of shareholders
    on January 23, 2014.

-- New FP7 Grant Awarded for Development of DMD Imaging Biomarkers

  oOn November 25, 2013, Prosensa and Newcastle University, UK, announced the
    award of a second Framework Programme 7 (FP7) research grant from the
    European Commission. This new FP7 research grant totals approximately €6
    million to support the development of imaging biomarkers for DMD. The
    project "Developing imaging technologies for therapeutic interventions in
    rare diseases" will be known as "BIOIMAGE-NMD" and is expected to run for
    three and a half years. Earlier in the year, Prosensa was part of a
    pan-European consortium that received a €6 million FP7 research grant to
    support the ongoing clinical study of its third novel DMD development
    candidate, PRO045.

Financial Highlights

  oCash Position and Cash Consumption: Prosensa's cash and cash equivalents
    as of December 31, 2013 were €82.2 million, compared to €40.7 million as
    of December 31, 2012 and €86.7 million as of the quarter ended September
    30, 2013. The increase in cash and cash equivalents was due to €64.0
    million in proceeds (before issuance cost) from the issuance of 6.9
    million shares of common stock upon the IPO on July 3, 2013. The company's
    cash consumption, excluding cash flows from financing for the 12-months
    ended December 31, 2013 was €22.4 million.
    
  oRevenue: Revenue for the 12-months ended December 31, 2013 was €8.9
    million, compared with €7.9 million in 2012 due to increased collaboration
    revenue of €1.2 million and lower license income of €0.1 million.
    
  oR&D Expense: Research and development expense was €18.5 million for the
    12-months ended December 31, 2013, compared to €14.4 million for the
    comparable period in 2012. While we incurred expenses for preclinical
    safety studies for PRO045 and PRO053 in the 12-months ended December 31,
    2012, our research and development expenses in 2013 mainly related to the
    ongoing Phase I/II study of PRO045 and PRO053. In the twelve months ended
    December 31, 2013 we also incurred expenses for the preclinical safety
    studies for PRO052, the Natural History and PROSPECT programs.
    
  oG&A Expense: General and administrative expense was €7.7 million for the
    12-months ended December 31, 2013, compared to €4.0 million in 2012. The
    increase is primarily due to higher share-based compensation expense in
    2013 of €1.1 million compared to €180 thousand in 2012 and costs
    associated with operating as a public company.
    
  oNet Loss: Net loss for the full year 2013 was €16.6 million or €0.51 per
    share, compared to €9.9 millionor €0.37 per share for the full year 2012.

Upcoming Conferences

Prosensa management will be participating in the following conferences and
events:

  oMuscular Dystrophy Association Clinical Conference, March 16-19, Chicago,
    IL
  oCommercialisation of Orphan & Rare Diseases Drugs Forum, March 20-21,
    London, UK
  oBioCapital Europe, March 27, Amsterdam, The Netherlands
  oWorld Orphan Drug Congress, April 24-25, 2014, Washington, DC
  oAmerican Academy of Neurology (AAN), April 26-May 3, Philadelphia, PA
  oUS TIDES 2014, May 12-15, Providence, RI
  oBioEquity Europe, May 21-22, Amsterdam, The Netherlands
  oParent Project Muscular Dystrophy (PPMD) Annual Conference, June 26-29,
    Chicago, IL
  oOrphan Disease Forum at the BIO International Convention, June 24, San
    Diego, CA

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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Source: Prosensa Holding N.V. via Globenewswire
HUG#1769472
 
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