Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD)

Phase 2 Completed 16 enrolled NS Pharma, Inc. Cooperative International Neuromuscular Research Group · collab Nippon Shinyaku Co., Ltd. · collab Therapeutic Research in Neuromuscular Disorders Solutions · collab

RandomizedParallel-groupQuadruple-blindTreatment

Summary

The main objective of this study is to evaluate the safety of a high (80mg/kg) and low (40mg/kg) dose of NS-065/NCNP-01 delivered as an intravenous infusion in patients with Duchenne Muscular Dystrophy (DMD) amendable to exon 53 skipping. Additional objectives include tolerability, muscle function and strength, pharmacokinetics and pharmacodynamics.

Timeline

Start: 2016-12
Primary completion: 2018-03
Completion: 2018-04

Outcome

Met primary endpoint

paper After 20 to 24 weeks of treatment, significant drug-induced dystrophin production was seen in both viltolarsen dose cohorts PMID 32453377 ↗

paper Viltolarsen was well tolerated; no treatment-emergent adverse events required dose reduction, interruption, or discontinuation of the study drug. PMID 32453377 ↗

Publications

Clemens PR, Rao VK, Connolly AM, Harper AD, Mah JK, Smith EC, McDonald CM, Zaidman CM, Morgenroth LP, Osaki H, Satou Y, Yamashita T, Hoffman EP; CINRG DNHS Investigators. Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping: A Phase 2 Randomized Clinical Trial. JAMA Neurol. 2020 Aug 1;77(8):982-991. doi: 10.1001/jamaneurol.2020.1264.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Viltolarsen	Unknown	40 mg/kg	Intravenous
Subject	Viltolarsen	Unknown	80 mg/kg	Intravenous