Author: Willem van Lawick

Leiden, The Netherlands, June 27, 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 announced that Dr. Judith van Deutekom, Prosensa’s Vice President of Drug Discovery, has been awarded a $200,000 research grant from Parent Project Muscular Dystrophy (PPMD), the largest non-profit organization in the United States focused on finding a cure for Duchenne Muscular Dystrophy (DMD), to fund the development of a multi exon-skipping strategy for exons 10-30 of the dystrophin gene.
The project titled “Single AON-induced multiple exon skipping in the mdx mouse model” will focus on obtaining proof-of-concept in the mdx mouse model. The studies are expected to be completed in the second half of 2015. (more…)

Leiden, The Netherlands, June 25, 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 announced the successful completion of enrollment in its prospective Natural History Study designed to increase the understanding of Duchenne muscular dystrophy (DMD).
The study enrolled in total 269 boys with confirmed DMD between the ages three and 18 from 10 countries across 16 centers in North & South America and Europe, 80% of whom are ambulatory and 20% non-ambulatory. (more…)

San Diego, CA, June 23, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA) the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, today announced that Hans GCP Schikan, Chief Executive Officer of Prosensa, has been appointed to the Biotechnology Industry Organization’s (BIO) Emerging Companies Section Governing Board. (more…)

Plans to Submit a New Drug Application to the FDA this Year; Dialogue with EMA continues with intent to seek approval

Leiden, The Netherlands, June 3, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA) the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, today announced that the United States Food and Drug Administration (FDA) has outlined a regulatory path forward, under an accelerated approval pathway, for drisapersen, the Company’s lead program for the potential treatment of Duchenne Muscular Dystrophy (DMD). In addition, the company has been interacting with the European Medicines Agency (EMA) and based on these interactions intends to file in Europe as well.
Following the positive feedback from the FDA, Prosensa has confirmed that it will pursue a New Drug Application (NDA) filing for drisapersen with the FDA, under an accelerated approval pathway based on existing data. The company plans to submit a file later this year and will commit to the initiation of two confirmatory post-approval studies. “Given the urgent need to find effective therapies for boys afflicted with this devastating disease, we could not be more pleased with this favorable outcome and with the regulatory authorities’ willingness to advance investigational products for the treatment of DMD” said Hans Schikan, Chief Executive Officer of Prosensa. (more…)

Groningen, the Netherlands, May 22, 2014 – Biotechnology company Mucosis B.V. today announced that it has received an innovation credit line of up to €5 million from the Netherlands Enterprise Agency, an agency of the Dutch Ministry of Economic Affairs.
Mucosis will use these funds to further advance the clinical development of its proprietary SynGEM® prefusion F RSV vaccine candidate through human proof-of-concept studies. These studies aim to demonstrate both safety and protection against a challenge with RSV in healthy volunteers. (more…)