NS Pharma announced the U.S. Food & Drug Administration (FDA) has agreed to the planned Phase II study of NS-089/NCNP-02 for Duchenne muscular dystrophy.  NS-089/NCNP-02 is an investigational candidate for patients with Duchenne muscular dystrophy amenable to exon 44 skipping therapy.

"Progress has been made in the treatment of Duchenne, but patients and families need new and more treatment options," said Vamshi Rao, MD, Ann & Robert H. Lurie Children's Hospital of Chicago. "There are currently no available antisense treatments that target Duchenne patients amenable to exon 44 skipping therapy, which is why I am excited about this program and the potential advance of effective treatments for Duchenne muscular dystrophy."

Study efficacy measures will include the expression of dystrophin protein and motor function. Trial details will be made available through ClinicalTrials.gov. Additional information will be provided once the trial is ready to begin enrolling.

"We are pleased to announce FDA's clearance to proceed with our Phase II clinical trial in our endeavor to help patients with Duchenne amenable to exon 44 skipping therapy," said Takeshi Seita, Vice President, R&D at NS Pharma, Inc. "We are confident in our exon skipping drug discovery platform and excited about the future potential of our development program."

In addition to NS-089/NCNP-02, NS Pharma's parent company, Nippon Shinyaku, has four investigational exon skipping candidates in various stages of preclinical development.