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Japan-based Nippon Shinyaku has entered into an option agreement with US-based Elixirgen Therapeutics, Inc. under which Nippon Shinyaku may obtain the exclusive worldwide rights to develop and commercialise EXG-7001 for the treatment of Duchenne muscular dystrophy (DMD).
The agreement was facilitated by the NS Pharma Innovation Research Partnering (IRP) team, located in Cambridge, MA.
Duchenne muscular dystrophy (DMD) is a severe, progressive genetic disorder causing muscle weakness due to the absence or dysfunction of dystrophin.
"Collaboration is essential in furthering scientific innovation, and we are proud to partner with Elixirgen to expand treatment options for the Duchenne community," said NS Pharma President Yukiteru Sugiyama.
EXG-7001 is an investigational, locally administered, mRNA based therapeutic drug that expresses full-length human dystrophin protein. Regardless of genetic mutation, it is intended to suppress local muscle dysfunction by expressing the full-length dystrophin protein, which is deficient within the muscle cells of patients with DMD. EXG-7001 is in preclinical development for the treatment of DMD. Elixirgen is currently preparing for clinical trials in the United States.
Following the exercise of the option rights by Nippon Shinyaku and regulatory approval in the United States, NS Pharma intends to commercialize EXG-7001.
