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

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