Author: Willem van Lawick

Clinically meaningful improvement from 24-week treatment period was maintained for 24 weeks after drisapersen administration ceased

Chicago, IL, March 17, 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 encouraging 48-week data from its U.S.-based, Phase II placebo-controlled study (DMD114876 or DEMAND V) of its lead compound, drisapersen, for the treatment of Duchenne Muscular Dystrophy (DMD).
The results of this study indicate 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 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. (more…)

Prof. Dr. Angela Krackhardt from the Klinikum rechts der Isar of the Technische Universität München to lead the project.

Amsterdam, The Netherlands, January 29, 2014 – Kiadis Pharma B.V., a clinical stage biopharmaceutical company developing treatments for blood cancers, today announces that it has initiated a collaboration to identify and characterize leukemia-specific T-cells in Kiadis Pharma’s lead product ATIRTM, that would be responsible for the Graft-versus-Leukemia effect of the T-cell immunotherapy product. The collaboration will be run with Prof. Dr. Angela Krackhardt who leads the group of Translational Immunotherapy at the Department of Hematology (Prof. Christian Peschel) of the Klinikum rechts der Isar of the Technische Universität München. (more…)

Corporate update presentation given at the 32nd Annual J.P. Morgan Healthcare Conference in San Francisco on Thursday, January 16, 2014 at 10:00 AM Pacific Time

Leiden, The Netherlands, Jan. 16, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the Dutch biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet medical need, today announced initial findings from further analyses from the aggregate data from the clinical development program of drisapersen for the treatment of Duchenne Muscular Dystrophy (DMD).
“We are encouraged by these results that suggest that treating earlier in the disease and treating longer shows a delay in the progression of the disease,” said Hans Schikan, Prosensa’s Chief Executive Officer. “These data encourage us to engage patient groups, clinical experts and regulators to explore a path forward for drisapersen, which includes the possibility of re-dosing.” (more…)

LEIDEN, Netherlands and LONDON, Jan. 13, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (Nasdaq:RNA) and GlaxoSmithKline (GSK) today announced that Prosensa has regained all rights from GSK to drisapersen and will retain rights to all other programs for the treatment of Duchenne Muscular Dystrophy (DMD). This transfer of rights represents the termination of the collaboration agreement between GSK and Prosensa executed in 2009. Prosensa will now have the full, unencumbered rights to continue the development of drisapersen as well as each of its DMD programs. (more…)

Leiden, The Netherlands, Dec. 19, 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, commented on a recent update by GlaxoSmithKline (GSK) to patient groups and investigators regarding the ongoing analyses for drisapersen, an investigational antisense oligonucleotide, for the treatment of Duchenne Muscular Dystrophy (DMD) patients with an amenable mutation, which is exclusively licensed to GSK. (more…)