April 14, 2023 / Clinical Trials

NS Pharma Announces FDA Clearance of IND for Clinical Trial of NS-089/NCNP-02 Exon 44 Skipping Candidate

PPMD is excited to share that NS Pharma has announced that they have received clearance from the FDA on their Investigational New Drug (IND) application for NS-089/NCNP-02, a potential exon skipping therapy targeting people with Duchenne who have genetic mutations amenable to skipping exon 44 of the dystrophin gene.

With this clearance, NS Pharma can move towards initiating its Phase II trial investigating NS-089/NCNP-02. Additional information will be provided once the trial is ready to begin enrolling.

We look forward to hearing more from NS Pharma at PPMD’s 2023 Annual Conference in Dallas, TX, June 29- July 1.

Read NS Pharma’s Press Release

NS Pharma Announces FDA Clearance to Initiate Phase II Study for NS-089/NCNP-02, an Exon 44 Skipping Candidate for the Treatment of Duchenne Muscular Dystrophy NS Pharma, Inc.

PARAMUS, NJ: April 14, 2023, – NS Pharma, Inc. announced today the U.S. Food & Drug Administration (FDA) has agreed to the planned Phase II study of NS-089/NCNP02 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.

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