Summary of Clinical Use

Arbekacin is a potent aminoglycoside antibacterial already approved in some countries to treat multidrug-resistant bacteria. An inhalation solution of arbekacin (ME1100) is being developed for the treatment of hospital-acquired and ventilator-associated bacterial pneumonia. Several aminoglycoside antibacterials, including arbekacin, have been reported to have the ability to promote mRNA translation even in the presence of premature termination codons (PTCs) in mutated genes. In the absence of pharmaceutical intervention, the ribosomal translation process produces truncated, often non-functional protein products. However, in the presence of such compounds the ribosomes 'readthrough' the PTCs, producing increased amounts of full-length, functional protein. This developing pharmaceutical intervention has been termed 'readthrough strategy' and has led to a repurposing effort, with the compound now being evaluated in clinical trial as a treatment for Duchenne muscular dystrophy (DMD), in patients where the inherited genetic mutation causes production of truncated, non-functional dystrophin. Dystrophin is a protein critical for muscle function. For more information regarding current clinical trials testing arbekacin, visit www.clinicaltrials.gov.

Mechanism Of Action and Pharmacodynamic Effects

Arbekacin promotes ribosomal readthrough of PTCs, thereby increasing transcription of full-length, functional proteins.

Clinical Trials
Clinical Trial ID	Title	Type	Source	Comment	References
NCT02459158	A Study to Assess the Pharmacokinetic Profile, the Safety, and the Tolerability of ME1100 Inhalation Solution in Patients With Mechanically Ventilated Bacterial Pneumonia	Phase 1 Interventional	Meiji Seika Pharma Co., Ltd.		2
NCT01918384	Phase II Study of NPC-14 (Arbekacin Sulfate) to Explore Safety, Tolerability, and Efficacy in Duchenne Muscular Dystrophy	Phase 2 Interventional	Kobe University