Leiden, The Netherlands, Sept. 17, 2014 (GLOBE NEWSWIRE) — Prosensa Holding N.V. (NASDAQ: RNA), the biopharmaceutical company focusing on RNA-modulating therapeutics for rare diseases with high unmet need, today announced that a comprehensive program of re-dosing has commenced, with the first patients now re-dosed in the United States. All dosing in the drisapersen clinical program had been placed on hold by GSK on September 20, 2013, upon announcement of the DEMAND III study results.
The re-dosing program in North America will include up to 72 patients across 14 sites who had participated in the drisapersen DEMAND V (Phase II) & DEMAND III (Phase III) studies. Prosensa regained the rights to drisapersen from its previous partner GSK in January 2014, and soon thereafter committed to re-dose patients as quickly as possible, accepting that the transfer of data and clinical materials, and the re-engagement with clinical sites would be a complex process.
“We are very pleased that we have been able to keep our promise to commence re-dosing patients with drisapersen in the third quarter of this year, which is taking a staged approach,” said Dr. Giles Campion, Prosensa’s Chief Medical Officer. “While we know the wait has been incredibly difficult for the boys (and their families), we have been working diligently on this achievement since regaining the rights to drisapersen and are grateful to all that have helped us in attaining this goal. While today is a major milestone for the Company, it is an even greater one for these first patients and their families, who have been anxiously awaiting drisapersen treatment since dosing was stopped almost one year ago” he added.
Drisapersen is administered subcutaneously, with once weekly dosing. Prosensa is aware of the burden that clinical trials can place on the families, and in this protocol efforts have been made to alleviate burden and facilitate participation. Prosensa aims to enable home-dosing where feasible, and cost-free transport with straightforward logistics for clinical centers, through collaboration with Greenphire and the Medical Research Network (MRN).
Greenphire is the industry’s leading provider of clinical payment technology and has made its proprietary ClinCard System available for patients in the US and Canada. The ClinCard system with Travel Module will help Prosensa manage the complex travel arrangements required for the patients involved in this study, which will maintain a truly patient-centric environment. Patients, along with their caregivers and family members, can make use of Greenphire’s expert travel services to arrange travel for expected study visits with minimal out-of-pocket costs. Similarly, Greenphire’s logistical support allows clinician investigators to focus their time and attention where it’s needed: patient care and study execution.
MRN is the world’s leading provider of home healthcare for patients in clinical trials and will be making home-dosing options available to patients in the study.
“Given the weekly dosing regimen for drisapersen and the travel time and costs associated with these, we are pleased to collaborate with both Greenphire and MRN to ease the burden of this process to patients and their families, said Hans Schikan, Prosensa’s Chief Executive Officer. “While making treatment options available for DMD patients globally is our number one priority, we also want to do this in a manner that is as seamless as possible for patients and their families.”
Prosensa is also in in preparations for similar programs of re-dosing and support for all previously treated drisapersen patients.
In June, Prosensa announced that the United States Food and Drug Administration (FDA) outlined a regulatory path forward for drisapersen, under an accelerated approval pathway, based upon existing data, including the DEMAND II study, which was recently published in the Lancet Neurology. Prosensa remains on track to pursuing regulatory filings for drisapersen, initially in the US and Europe, with an FDA submission planned before the end of the year and an EMA submission shortly thereafter.
