NM001

NM001 is a novel, highly differentiated therapy candidate in development for CF and NCFB-associated lung disease.  NM001 has a unique multi-action including anti-inflammatory effects, potent mucolytic action, antimicrobial, anti-virulence and antibiotic-potentiating effects.

NovaBiotics is developing two forms of NM001.  Oral NM001 for pulmonary exacerbations of CF and NCFBE and inhaled NM001 for the maintenance of ventilatory function in CF and NCFB patients. The application of the oral and inhaled forms of NM001 in exacerbating and stable patients respectively could provide the opportunity for a paradigm shift in standard of care therapy.  Importantly, as a CF candidate therapy, NM001 is not a mutation-specific CFTR-targeting intervention.  The unique multi-activity of NM001 has been demonstrated in our clinical trials and a range of laboratory experiments. See our publications page here [opens in new window].

NM001 in oral (tablet) form recently completed an exploratory phase 2b global study (CARE CF 1) in CF patients following an earlier, positive phase 2a trial.  CARE CF 1 was designed to determine which clinical endpoints are the most appropriate to demonstrate the benefits of NM001 in exacerbating (adult) CF patients who took NM001 alongside their other ‘standard’ medicines for 14 days (or placebo).  CARE CF 1 was also designed to determine which dose(s) and treatment regimen(s) of NM001 is/are optimal for efficacy.  The endpoint and dose(s) were successfully identified from CARE CF 1. We now plan to progress NM001’s development in registrational studies comprising both CF and NCFB patient cohorts. 

The inhaled (dry powder) formulation of NM001 has recently been optimised and from a platform of very encouraging preclinical efficacy and safety data, NovaBiotics is working towards initiating phase 1 clinical studies for inhaled NM001.

Orphan drug designation has been granted in the US and Europe for NM001 in the treatment of CF.  NM001 gained Fast Track Designation in the US in 2018.  Oral NM001 was designated as a priority review medicine by the Central European Health Technology Assessment body in July 2019.