Lynovex (NM001)

Lynovex® (NM001) is a first-in-class orphan drug candidate for cystic fibrosis, an inherited life-limiting disease affecting around 70,000 individuals worldwide. NovaBiotics is developing two formulations, oral Lynovex for CF exacerbations, and inhaled Lynovex for long term treatment/maintenance.

Lynovex is a novel, highly differentiated therapy for the treatment of CF-associated lung disease, with a unique multi-action:

  • Breaking down of the mucus (in a more potent and distinct mechanism than other mucolytic agents; one more likely to facilitate sputum expectoration) that not only obstructs the airways of CF patients but which also creates an environment in which bacteria can thrive
  • Killing the bacteria responsible for the recurrent respiratory infections and disrupting the biofilms in which they colonise.
  • Potentiatating the activity of standard of care CF antibiotics (boosting their function and reversing antibiotic resistance).

Lynovex is not a mutation-specific CFTR-targeting intervention and can be used to treat all genotypes. The unique multi-activity of Lynovex has been demonstrated in clinical trials and a range of laboratory experiments. See our publications Pubmed page for NovaBiotics

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

Lynovex is being developed as an oral medicine as an intervention for pulmonary exacerbations of CF and is the only treatment to specifically target these lung function critical events. In parallel, a dry powder inhaler form of Lynovex has been developed as a continuous therapy for the maintenance of respiratory function in stable CF patients.

Game-changing therapies

There are no other products on the market or in development that compare to Lynovex. The application of the oral and inhaled forms of Lynovex in exacerbating and stable patients respectively provides the opportunity for a paradigm shift in CF standard of care therapy. Modulator and other therapies do not address CF lung disease symptomology nor do they prevent exacerbations from occurring. Lynovex’s impact in the CF therapy regimen for patients who will live longer with complex disease could be potentially game-changing.

Clinical and Commercial Development

Lynovex in oral (tablet) form recently completed an exploratory phase 2b global study (CARE CF 2) 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 Lynovex in exacerbating (adult) CF patients who took Lynovex 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 Lynovex is/are optimal for efficacy. The endpoint and dose(s) were successfully identified from CARE CF 1 and, following interactions and feedback from the FDA and EMA, Lynovex will be taken forward in a confirmatory phase 2b study (CARE CF 2) and thereafter phase 3 clinical trial (CARE CF 3). Paediatric studies are planned alongside the phase 3 study in adult patients.

The inhaled (dry powder) formulation of Lynovex has recently been optimised and from a platform of very encouraging preclinical efficacy and safety data, NovaBiotics is working towards initiating clinical studies for inhaled Lynovex in 2020.

To learn more about Lynovex, please visit www.lynovex.com.