- Prosensa Therapeutics
- Wassenaarseweg 72
- 2333 AL Leiden
- Luc Dochez
- +31 (71) 3322-100
Prosensa Initiates Clinical Development of PRO044 in an Open Label Phase I/II Study
Prosensa's second clinical candidate for treatment of Duchenne Muscular Dystrophy
The objective of this open label, doseescalating study is to evaluate the safety and tolerability of the systemic delivery of PRO044 in twelve Duchenne Muscular Dystrophy (DMD) patients receiving weekly subcutaneous injections for a period of five weeks. The effect of PRO044 will be assessed at the RNA level, to demonstrate specific exon 44 skipping, and at the protein level, to demonstrate novel dystrophin expression in muscle biopsies.
The first patients have been enrolled at the University Hospital Leuven in Belgium by principal investigator Dr. Nathalie Goemans, department of Pediatric Neurology. Prosensa also received approval from the regulatory authorities in Sweden, where patient screening has been initiated, and Italy. Participating centers are the Queen Silvia Children's Hospital in Gothenburg, Sweden and the University of Ferrara in Italy.
PRO044 is Prosensa's second RNA based therapeutic candidate for the treatment of DMD. Prosensa's lead DMD compound, PRO051/GSK2402968, is close to entering phase III clinical development and was recently partnered with GlaxoSmithKline (GSK). GSK also has an exclusive option on PRO044, which will be triggered upon successful completion of this phase I/II study.
"We are delighted to advance a second promising candidate for the treatment of DMD into clinical development. With the addition of PRO044 to our drug candidate portfolio, we will be able to address a broader patient population upon successful completion of these clinical development programs." commented Dr. Giles Campion, Chief Medical Officer of Prosensa. "This important clinical development step confirms Prosensa's commitment to the treatment of neuromuscular disorders."
About DMD and exon skipping
Duchenne muscular dystrophy is a severely debilitating childhood neuromuscular disease that affects 1 in 3,500 newborn boys. The young patients suffer from progressive loss of muscle strength due to the absence of the protein dystrophin, often making them wheelchair bound before the age of 12. Most patients die in early adulthood due to respiratory and cardiac failure. Today, there is no treatment to prevent the eventual fatal outcome. The disease is caused by mutations in the DMD gene, resulting in the absence of the dystrophin protein, which is crucial for the integrity of muscle fiber membranes.
RNAbased therapeutics, specifically antisense oligonucleotides inducing exon skipping, are currently amongst the most promising therapies for DMD. More specifically, antisense oligonucleotides have the capacity to skip an exon and thereby correct the reading frame of DMD transcripts aiming at the synthesis of a largely functional dystrophin protein. Different mutations in the gene require different oligonucleotide drugs. Prosensa's lead product candidates, PRO051/GSK2402968 and PRO044 may be suitable for approximately 20% of all DMD patients.
Prosensa is a privately held biopharmaceutical company, backed by a consortium of highly regarded investors such as Abingworth, AGF Private Equity, GIMV, LSP and MedSciences Capital.
For more information about Prosensa, please visit www.prosensa.com.
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