Leiden University Medical Center and Prosensa B.V. Announce First Successful Clinical Study with RNA-based Therapeutic PRO051
Novel RNA-based therapeutic PRO051 to enter Phase I/II clinical trials in Duchenne Muscular Dystrophy
Leiden University Medical Center (LUMC) and Prosensa announced in the New England Journal of medicine (van Deutekom et al.) positive results from the first ever clinical study with RNA-based therapeutic PRO051 in four patients with Duchenne muscular dystrophy (DMD), in which PRO051 has restored dystrophin Expression in the treated muscle fibers of all four patients tested.
In this study, DMD patients between 10 and 13 years of age received a single injection of PRO051 (a 2-'O-methyl antisense oligonucleotide) in a small area of a muscle in the lower leg. In a biopsy taken four weeks later, novel dystrophin expression was observed in the vast majority of muscle fibers with protein levels that are expected to be clinically relevant. This pilot study is the first ever with an RNA-based therapeutic agent for DMD and is an important step towards treatment of this devastating disease, for which as yet no therapy is available. Following this first clinical proof-of-mechanism, Prosensa has started the preparations for a phase I/II clinical study to explore the effects and safety of PRO051 after repeated systemic injections.
Gerard Platenburg, Prosensa's CEO, commented: "This first clinical study demonstrates that PRO051, an RNA-Therapeutic based on our proprietary exon skipping technology, was able to correct this genetic error in locally injected muscle tissue of Duchenne Muscular Dystrophy patients. We are excited to have demonstrated clinical proof-of-mechanism and will be progressing PRO051 into systemic Phase I/II trials in order to assess the broader benefits available to patients. RNA-based therapeutics hold great promise as an approach to create a whole new class of innovative medicines and we are well positioned to exploit this opportunity."
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