Author: Willem van Lawick

Leiden, The Netherlands, Nov. 7, 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 announced the successful enrollment of the 100th patient into the Natural History Study of Duchenne muscular dystrophy (DMD).
The goal of this observational study is to characterize DMD at various stages of progression using the same measures used in ongoing clinical studies, such as the “six minute walk test.” No medication is being tested in this study. (more…)

Amsterdam, The Netherlands, October 7, 2013 – Kiadis Pharma B.V., a clinical stage biopharmaceutical company developing treatments for blood cancers, announced today that a Services Agreement has been signed with the German Red Cross Blood Donor Service Baden-Wuerttemberg-Hessen (GRCBDS) whereby GRCBDS will provide pharmaceutical development and GMP manufacturing services for Kiadis Pharma’s ongoing Phase II clinical study with ATIR™. The transfer of the manufacturing process for ATIR™ has already successfully been completed by GRCBDS at their facility in Frankfurt am Main, Germany and a first batch of Phase II clinical trial material for the European study has now been successfully manufactured. (more…)

LONDON and LEIDEN, Netherlands, Sept. 20, 2013 (GLOBE NEWSWIRE) — GlaxoSmithKline (GSK) and Prosensa today announced that GSK’s Phase III clinical study of drisapersen, an investigational antisense oligonucleotide, for the treatment of Duchenne Muscular Dystrophy (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. (more…)

Amsterdam, The Netherlands, September 11, 2013 – Kiadis Pharma B.V., a clinical stage biopharmaceutical company developing treatments for blood cancers, announced today that the five-year follow-up of patients with high-risk malignancies from its Phase I/II clinical study confirms long-term safety and efficacy of ATIR™ over a broad dose range. ATIR™ is a cell-based product designed to enable stem cell transplantations from partially mismatched (haploidentical) family donors for patients who do not have a standard of care stem cell donor available. The results demonstrate proof of concept and show that ATIR™ infusion after a T-cell depleted haploidentical hematopoietic stem cell transplantation (HSCT) provides immune protection shortly after the transplantation and improves long-term outcome in high-risk patients with very poor prognosis. (more…)

Leiden, The Netherlands, Sept. 6, 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 announced the dosing of the first patient in its Phase I/II clinical trial of PRO053, an exon-skipping compound for the treatment of Duchenne muscular dystrophy (DMD).
“We are pleased that our fourth exon-skipping compound, PRO053, has advanced into clinical trials.” Giles Campion, Prosensa’s Chief Medical Officer, said. “Bringing new drug candidates, such as PRO053, into the clinic exemplifies how we can leverage our scientific and regulatory expertise gleaned from the experience in advancing the investigational development compound, drisapersen, through the clinic. Most importantly, a broader clinical portfolio may offer additional patients with subtypes of DMD a much-needed potential treatment option.” (more…)