Prosensa Hold (Nasdaq: RNA) reported Q1 EPS of EUR0.19, versus (EUR0.12) reported last year. Revenue for the quarter came in at EUR14.8 million, versus EUR2.3 million reported last year.

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.

For earnings history and earnings-related data on Prosensa Hold (RNA) click here.