Naproxcinod for the treatment of Duchenne muscular dystrophy

Gloria Vigliani, MD
undisclosed company

25th October 2014

The company submitted a proposal to use Naproxcinod (HCT3012) for treatment of Duchenne muscular dystrophy (DMD). Naproxcinod is a naproxen-based nitric oxide (NO)-donating anti-inflammatory drug belonging to the cyclooxygenase (COX) –inhibiting NO donors (CINODs) class. The current standard of care for DMD is corticosteroids which have multiple functions, some of which are certainly anti-inflammatory, as well as a number of side-effects. A steroid sparing agent would be welcomed in this disease. NO is reduced in DMD and restoration of this signalling pathway is a major therapeutic goal. The company plans to combine the NO-donation with non-steroidal anti-inflammation activity to ameliorate DMD pathology and improve strength. The committee found the preclinical studies in the mdx mouse model encouraging. In addition, more than 4,000 subjects have been treated with Naproxcinod in trials in osteoarthritis (OA) suggesting a good safety and tolerability profile. The company outlined a placebo controlled clinical trial of 7-13 year old ambulatory boys with and without concomitant corticosteroids. This committee had several suggestions regarding the clinical plan. Naproxcinod was granted Orphan Drug Designation by the European Union in October 2013. The company plans to meet with the Health Authorities (FDA, European Agencies) to discuss the clinical plan and submit an Investigational New Drug (IND) to the Food and Drug Administration in 2015.

12 Apr 2017