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2014-05-20

Prosensa Holding N.V.: Prosensa Announces 1st Quarter 2014 Financial Results and Recent Corporate Developments

Regulatory Discussions Ongoing; Drisapersen Re-Dosing to Start in Q3

LEIDEN, The Netherlands, May 20, 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 first quarter ending March 31, 2014 and
provided an update on the next steps for its exon-skipping platform for the
treatment of Duchenne Muscular Dystrophy (DMD).

Hans Schikan, CEO of Prosensa said, "We have made excellent progress during
this period, and are very pleased that we are now in a position to commence
re-dosing with drisapersen of boys that have previously participated in
drisapersen trials beginning in the third quarter of 2014. In addition, our
250-patient Natural History study, designed to better understand the disease
progression of DMD, has almost completed enrollment."

"We are encouraged by our ongoing interactions with the regulatory
authorities, in both the United States and Europe, and remain on track to
communicating a potential regulatory path forward for drisapersen by the end
of June," he added. "Prosensa is dedicated to enabling long term patient
access to drisapersen and its follow-on candidates as novel treatments for
DMD. We are determined to accomplish this as soon as possible."

Recent Corporate Highlights

--Updates on the Drisapersen Development Program

* On January 13, we announced that we regained the rights to drisapersen from
GSK and retained the rights to all other programs for the treatment of DMD.
Prosensa now has full, unencumbered rights to continue the development of
drisapersen in addition to each of our other DMD programs. Since then, we
have made substantial progress in the transfer of the drisapersen program
from GSK.
* On March 17, encouraging 48-week data from Prosensa's U.S. Phase II
placebo-controlled study of drisapersen for the treatment of boys with DMD
(DEMAND V, DMD114876) were presented by the principal investigator, Craig
M. McDonald, M.D., Professor and Chair of Physical Medicine&Rehabilitation
and Professor of Pediatrics at the University of California, Davis School
of Medicine, at the Muscular Dystrophy Association 2014 Clinical Conference
in Chicago, Illinois (March 16-19). The results of this 51 patient-study
indicated that, compared to placebo, boys in the higher-dose drisapersen
group (6 mg/kg once weekly) experienced stabilization and even improvements
in their muscle function and physical activity as measured by the
six-minute walk test (6MWT) for the 24-week treatment phase and maintained
this improvement during the 24-week no-treatment follow-up period.
Additionally, when evaluating the percent-predicted six-minute walk
distance (6MWD), a clinically meaningful treatment difference of 5.2% was
observed at week 24 and 4.8% at week 48.
* On April 30, Dr. Nathalie Goemans, Head of the Neuromuscular Reference
Center for Children at the University Hospitals Leuven (UHL) in Belgium and
a key investigator in the drisapersen clinical program presented detailed
data up to week 48 (total of 96 weeks of treatment) from the second
open-label extension study of drisapersen in 113 boys with DMD (DEMAND IV,
DMD114349), who had previously completed a 48-week, double-blind,
placebo-controlled treatment phase in one of two feeder studies (DEMAND II,
DMD114117 and DEMAND III, DMD114044), during the 66th American Academy of
Neurology (AAN) Annual Meeting in Philadelphia, PA. The data are supportive
of the hypothesis that treating earlier in the disease and treating for a
longer duration confers a treatment benefit for boys with DMD.
* On May 1, we confirmed that following positive feedback from patients and
investigators regarding the willingness and desire of patients to go back
on drisapersen and encouraging analyses of further clinical trial data, we
will re-dose an initial group of boys, beginning with North America&Europe,
in the third quarter of 2014.

-- Other Research&Development Update

* Twelve abstracts from Prosensa and its collaborators have been accepted for
either poster or oral presentations for the 19thInternational World Muscle
Society Congress, taking place in Berlin, Germany, October 7-11, 2014.
* PRO044, the next most advanced product candidate, addresses a separate
sub-population of up to 6% of DMD patients. PRO044 has completed a Phase
I/II study in Europe, and results were presented in October 2013. An
extension study for PRO044 is planned for the second half of this year.
* PRO045 and PRO053 (each addressing a population for up to 8% of all DMD
patients) are currently in phase I/II clinical trials. We expect data for
PRO045 in the fourth quarter of 2014 and for PRO053 in the first quarter of
2015. We expect confirmatory studies for these compounds to start in the
first half of 2015.
* PRO052 and PRO055 are in advanced preclinical development.
* PROSPECT, which includes a new and innovative application our RNA
modulation technology platform, applies multiple exon skipping. Initial
efforts in the PROSPECT program are focused on the exon 10 to 30 region in
the dystrophin gene: a 10 to 30 multiple skip could be applicable to 13% of
all DMD patients. In vivo studies are currently ongoing.
* Natural History Study: 247 patients have been enrolled to date. The purpose
of the study is to characterize the natural history and progression of DMD
to help inform the design of future studies, to capture biomarkers of
safety and disease progression and to provide comparative data for the
development of rare exons for which formal controlled trials are not
feasible.

-- Supervisory Board Appointment

* On April 8, Prosensa announced that Michael S. Wyzga, former CEO of Radius
Health and CFO of Genzyme Corporation, was nominated for appointment to its
Supervisory Board. The appointment of Mr. Wyzga will be submitted to a
shareholder vote at the next shareholders' meeting on June 17, 2014.

Financial Highlights

* Cash Position and Cash Consumption: Prosensa's cash and cash equivalents as
of March 31, 2014 were €77.4 million, compared to €82.2 million as of
December 31, 2013. The decrease in cash and cash equivalents was mainly due
to operating activities. The company's cash consumption, excluding cash
flows from financing for the three months ended March 31, 2014 was €4.9
million.
* Revenue: Revenue for the three months ended March 31, 2014 was €14.8
million, compared with €2.4 million in 2013 due to an increase in license
revenue of €13.3 million and a decrease in collaboration revenue of €0.9
million. License revenue for the three months ended March 31, 2014 in an
amount of €14.7 million was exclusively related to the termination of the
research and collaboration agreement with GSK. This was due to a one-time
release of previously deferred revenue balances as well as €0.2 million
revenue related to other services delivered under the research and
collaboration agreement with GSK. In the three months ended March 31, 2014,
collaboration revenue was minimal due to the termination of the research
and collaboration agreement.
* R&D Expense: Research and development expense was €5.3 million for the
three months ended March 31, 2014, compared to €4.1 million for the
comparable period in 2013. PRO044 has completed a Phase I/II study in
Europe, and results were presented in October 2013. An extension study for
PRO044 is planned for the second half of this year which resulted in lower
expenses in the three month period ended March 31, 2014 compared to the
corresponding period in 2013. During the three month period ended March 31,
2014, we incurred expenses for the Phase I/II studies of both PRO045 and
PRO053. Our research and development expenses increased substantially in
connection with these clinical trials. In the three month period ended
March 31, 2014 we also incurred research and development expenses for
drisapersen as a result of the termination of the research and
collaboration agreement with GSK, in addition to expenses for our other
projects, such as the Natural History study, PROSPECT, PRO052 and PRO055.
* G&A Expense: General and administrative expense increased from €1.8 million
to €2.5 million in the three months ended March 31, 2013 and 2014,
respectively. The increase is primarily due to share-based compensation
expense and costs associated with operating as a public company.
* Net income/(loss): Net income for the three months ended March 31, 2014 was
€7.3 million or €0.20 per share (€0.19 diluted income per share), compared
to a loss of €3.5 million or €0.12 per share (€0.12 diluted loss per
share), for the comparative period in 2013.

Upcoming Conferences

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

* BioEquity Europe, May 21-22, Amsterdam, The Netherlands
* Jefferies Global Healthcare Conference, June 2-5, New York, NY
* ROTH Healthcare Corporate Access Day, June 24, London, UK
* Orphan Disease Forum at the BIO International Convention, June 24, San
Diego, CA
* 9thAnnual JMP Securities Healthcare Conference, June 24-25, New York, NY
* Parent Project Muscular Dystrophy (PPMD) Annual Conference, June 26-29,
Chicago, IL

Conference Call / Webcast Information

Prosensa will host a conference call on May 20, 2014 at 8:00 a.m. US Eastern
Time, 2:00 p.m. Central European Time to discuss the first 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 atwww.prosensa.com.

About Prosensa Holding N.V.

Prosensa (Nasdaq:RNA) is a Dutch 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 status 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 invari...

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