Prosensa Announces Third Quarter 2013 Financial Results and Recent Corporate Developments – Working Closely With Collaboration Partner GSK to Analyze Benefit-to-Risk Profile of dris­apersen Across All Studies –

LEIDEN, The Netherlands, Nov. 18, 2013 (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 quar­ter end­ing September 30, 2013.
“Together with our col­lab­o­ra­tion part­ner, GSK, the ben­e­fit-to-risk pro­file of dris­apersen treat­ment across all stud­ies is being ful­ly eval­u­at­ed and is expect­ed to be com­plet­ed by the end of the year,” said Hans Schikan, Chief Executive Officer of Prosensa. “We have a very large dataset to work with, and we remain com­mit­ted to the over­all pro­gram and RNA-mod­u­la­tion tech­nol­o­gy plat­form.”
Mr. Schikan con­tin­ued, “With no long term dis­ease-mod­i­fy­ing ther­a­pies avail­able for patients with Duchenne mus­cu­lar dys­tro­phy (DMD), research and devel­op­ment of pos­si­ble treat­ment options is of vital impor­tance for the boys and their fam­i­lies affect­ed by this debil­i­tat­ing dis­ease. This has dri­ven us to devel­op a port­fo­lio of prod­ucts tar­get­ing DMD and con­tin­ues to dri­ve us to find a treat­ment. We remain stead­fast in our com­mit­ment to work close­ly with patient groups and to pro­vide as much infor­ma­tion about the dris­apersen pro­gram as soon as pos­si­ble.”
Prosensa’s cur­rent port­fo­lio 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.

Recent Corporate Highlights
— Reporting and Analysis of dris­apersen data
• On September 20, 2013, Prosensa and its col­lab­o­ra­tion part­ner, GSK, announced that the Phase III (DMD114044) clin­i­cal study of dris­apersen, an inves­ti­ga­tion­al anti­sense oligonu­cleotide for the treat­ment of DMD patients with an amenable muta­tion, did not meet the pri­ma­ry end­point of a sta­tis­ti­cal­ly sig­nif­i­cant improve­ment in the 6 Minute Walking Distance (6MWD) test com­pared to place­bo.

• On September 25, 2013, dur­ing the DIA/FDA Oligonucleotide based Therapeutics Conference in Washington, DC, Dr. Craig McDonald of UC Davis Children’s Hospital pre­sent­ed data from the DEMAND V (DMD114876) study, a Phase II explorato­ry study to assess two dos­es of dris­apersen in the treat­ment of ambu­lant boys with DMD. At the 6mg/kg/week dose, dris­apersen has a 27 meter ben­e­fit over place­bo on the 6MWD, which was clin­i­cal­ly mean­ing­ful but not sta­tis­ti­cal­ly sig­nif­i­cant.

• Full eval­u­a­tion of the ben­e­fit-to-risk pro­file of dris­apersen treat­ment across all stud­ies is antic­i­pat­ed to be com­plet­ed by year-end. This will include analy­ses of pooled results from var­i­ous dris­apersen stud­ies.

— PRO053 Phase I/II tri­al update
• On September 6, 2013, Prosensa announced dos­ing of the first patient in its Phase I/II clin­i­cal tri­al of PRO053, the company’s fourth com­pound in its clin­i­cal devel­op­ment port­fo­lio for DMD.

— FP7 Grant Awarded for Development of PRO045
• On August 1, 2013, Prosensa announced that it is part of a high qual­i­ty, pan-European con­sor­tium which has been award­ed a pres­ti­gious Framework Programme 7 (FP7) research grant of €6 mil­lion from the European Commission to sup­port the ongo­ing clin­i­cal study of Prosensa’s third nov­el DMD devel­op­ment can­di­date, PRO045. The project is expect­ed to run for three years.

Financial Highlights
• Cash Position and Cash Consumption Our cash and cash equiv­a­lents as of September 30, 2013 were €86.7 mil­lion, com­pared to €40.7 mil­lion as of December 31, 2012. Our cash con­sump­tion, exclud­ing cash flows from financ­ing in the first three quar­ters of 2013 was €17.8 mil­lion.

• Revenue & Deferred Revenue Revenue was €2.4 mil­lion for the third quar­ter 2013, com­pared to €2.3 mil­lion for the com­pa­ra­ble peri­od in 2012 due to decreased license income of €0.6 mil­lion and increased col­lab­o­ra­tion rev­enue of €0.7 mil­lion for the third quar­ter 2013. All of our license rev­enue and col­lab­o­ra­tion rev­enue were gen­er­at­ed under the GSK Agreement.

• R&D Expense Research and devel­op­ment expense was €4.9 mil­lion in the third quar­ter 2013, com­pared to €3.7 mil­lion for the com­pa­ra­ble peri­od in 2012. While we incurred expens­es for final­iza­tion of the pre­clin­i­cal safe­ty stud­ies for PRO045 and PRO053 in the three months end­ed September 30, 2012, our research and devel­op­ment expens­es in the three months end­ed September 30, 2013 main­ly relat­ed to the ongo­ing Phase I/II study of PRO045 and PRO053. In both 2012 and 2013 we incurred expens­es for the 3-months pre­clin­i­cal safe­ty stud­ies for PRO052 and in 2013 for prepar­ing for these stud­ies in PRO055.

• G&A Expense General and admin­is­tra­tive expense was €1.9 mil­lion in the third quar­ter 2013, com­pared to €0.9 mil­lion in the com­pa­ra­ble peri­od in 2012. The increase is pri­mar­i­ly due to share-based com­pen­sa­tion expense and costs asso­ci­at­ed with oper­at­ing as a pub­lic com­pa­ny.

• Net Loss Net loss for the third quar­ter 2013 was €4.3 mil­lion in the third quar­ter 2013 and €12.5 mil­lion for the nine months end­ed September 30, 2013, com­pared to €1.8 mil­lion and €6.9 mil­lion for the com­pa­ra­ble peri­ods in 2012.

Recent & Upcoming Conferences
Prosensa man­age­ment has recent­ly par­tic­i­pat­ed or will be par­tic­i­pat­ing in the fol­low­ing con­fer­ences through the end of 2013 and ear­ly 2014:
• Action Duchenne 12th International Conference, London, UK, Nov 8-9, 2013
• EURO-TIDES Oligonucleotides and Peptides, Prague, Czech Republic, Nov 13-14, 2013
• New York CEO Conference, New York, NY, Nov 12-13, 2013
• KBC Life Sciences Meeting, New York, NY, Nov 14, 2013
• World Orphan Drug Congress Europe 2013, Geneva, Switzerland, Nov 14-15, 2013
• Jefferies Global Healthcare Conference, London, UK, Nov 20-21, 2013
• J.P. Morgan Healthcare Conference, San Francisco, CA, January 13-16, 2014

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