Author: Willem van Lawick

Regulatory Discussions Ongoing; Drisapersen Re-Dosing to Start in Q3
LEIDEN, The Netherlands, May 20, 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 financial results for the first quarter ending March 31, 2014 and provided an update on the next steps for its exon-skipping platform for the treatment of Duchenne Muscular Dystrophy (DMD).
Hans Schikan, CEO of Prosensa said, “We have made excellent progress during this period, and are very pleased that we are now in a position to commence re-dosing with drisapersen of boys that have previously participated in drisapersen trials beginning in the third quarter of 2014. In addition, our 250-patient Natural History study, designed to better understand the disease progression of DMD, has almost completed enrollment.”
“We are encouraged by our ongoing interactions with the regulatory authorities, in both the United States and Europe, and remain on track to communicating a potential regulatory path forward for drisapersen by the end of June,” he added. “Prosensa is dedicated to enabling long term patient access to drisapersen and its follow-on candidates as novel treatments for DMD. We are determined to accomplish this as soon as possible.” (more…)

Re-dosing plans are underway and 96-week data from an open-label extension study of drisapersen for the treatment of DMD presented at the American Academy of Neurology (AAN)

Leiden, The Netherlands, May 1, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA) the Dutch biopharmaceutical company focusing on rare diseases with a high unmet medical need, yesterday provided the data analysis of its second open-label extension study (DEMAND IV/DMD114349) of drisapersen in boys with Duchenne Muscular Dystrophy (DMD) during the 66th American Academy of Neurology (AAN) Annual Meeting in Philadelphia, PA. The Company also provided an update on the drisapersen clinical program earlier this week in a letter sent to patient group representatives and clinical investigators and will present a corporate update during an investor briefing on May 1. (more…)

Groningen, the Netherlands, April 15, 2014 — Biotechnology company Mucosis B.V. today announced that it entered into a long-term collaboration and license agreement with China-based Changchun BCHT Biotechnology Co (BCHT) and raised EUR 5 million, in a new financing round.
Under the agreement with BCHT, Mucosis will receive an equity investment along with customary payments including royalties in return for a license to its SynGEM® prefusion F vaccine candidate for prevention of RSV and the Mimopath® platform for other disease targets within the People’s Republic of China on an exclusive basis and non-exclusively in certain other Asian countries. Further details of the agreement were not disclosed. (more…)

Leiden, The Netherlands, April 8, 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 Michael S. Wyzga has been nominated for appointment to its Supervisory Board at the next shareholders’ meeting, scheduled for June 17, 2014.
“We welcome Michael as an instrumental addition to our Supervisory Board,” said Hans Schikan, Prosensa’s CEO. “Michael’s extensive financial and transactional expertise will support the company significantly in our efforts to bring much-needed, life-saving treatments for rare genetic diseases to patients. His long-term tenure at Genzyme, one of the most successful companies in the rare disease space, will be invaluable as we continue developing therapies for boys with Duchenne muscular dystrophy and building our company.” (more…)

Amsterdam, The Netherlands, March 24, 2014 – Kiadis Pharma B.V., a clinical stage biopharmaceutical company developing T-cell immunotherapy treatments for blood cancers, today announces that it will organize a satellite symposium on March 30, 2014 at the 40th Annual Meeting of the European Society for Blood and Marrow Transplantation (EBMT) in Milan, Italy. (more…)