This multiple-‐dose study will assess thesafety, tolerability, efficacy, and pharmacokinetics of SRP-‐4053 in DMD patients with genotypes amenable to exon-‐53 skipping.
Professor Francesco Muntoni, the Chief Investigator and SKIP-‐NMD Project Coordinator from UCL Institute of Child Health & Great Ormond Street Hospital in London said, “It is certainly pleasing to see that the long-‐term collaboration with our European partners from London, Paris, Newcastle, and Rome – and with Sarepta in the U.S-‐ – has come to fruition with the dosing of the first patient recruited into the dose escalation part of the study.
This project, which started two years ago, enters now into the in-‐patient phase in which we aim to demonstrate safety of this new antisense oligonucleotide, before proceeding to the subsequent maintenance phase in which exploratory efficacy will also be determined using a variety of techniques, including novel and innovative outcome measures.”
The study will be conducted at four sites in Europe under a consortium agreement between Sarepta and
various European hospitals, institutions, and scientists established to conduct the study and which is funded in part by the European Union’s Seventh Programme for research, technological development and demonstration under grant agreement No. 305370.
“We are excited to add a second exon-‐skipping drug to our clinical development pipeline,” said Edward Kaye, M.D., Sarepta’s Chief Medical Officer. “With the support of the SKIP-‐NMD partners,this marks a significant milestone in the expansion of our DMD program and a major step toward our goal of providing treatments to those children with Duchenne who may benefit from our exon-‐skipping technology.”
“Recruitment of patients into this and other clinical trials is made possible through collaboration with the French network of clinicians who have identified and followed up in a natural history with all patients theoretically treatable by skipping exon 53,” Laurent Servais, M.D., of the Institut de Myologie added. “We are happy that the first patient has received this new drug, to induce skipping of exon 53, here in Paris.”