May 20, 2014

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 19th International 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
  • 9th Annual 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 at www.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 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 disclaims 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

         Email: c.economides@prosensa.nl


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