Novoteris announced both the US Food and Drug Administration and the Therapeutic Products Directorate of Health Canada have cleared its Phase 2 clinical trial application. The company will begin recruiting subjects into its Phase 2 trial that will be using inhaled nitric oxide for the treatment of Cystic Fibrosis (CF).
This multi-center trial will randomize sixty subjects recruited from five cystic fibrosis research centers to receive either Thiolanox nitric oxide product or a placebo treatment, delivered with its unique computerized trace-gas mixing system. The Novoteris investigators' Phase 1 pilot trial in Europe reported encouraging microbiological and significant lung function changes following two weeks of the same treatment in patients with CF. The broad-spectrum anti-microbial property of nitric oxide widens the enrollment eligibility of subjects in this Phase 2 trial with the only exclusion being for Non-Tuberculosis Mycobacterium (NTM), which will be studied in separately planned trials. Gaseous nitric oxide's potent antimicrobial properties, lack of bacterial resistance, and small molecule penetration capabilities could provide a promising alternative, non-antibiotic approach to treating infections in people living with the disease.
"The clearances by the FDA and Health Canada will enable us to advance our work with this novel therapy and represents an important step for Novoteris," said Alex Stenzler, President of Novoteris.
Cystic fibrosis is an autosomal recessive genetic disorder that affects the lungs most critically. Difficulty breathing is the most serious symptom, which results from frequent lung infections that are most often treated with lifelong inhaled antibiotics. Development of drug resistance to available antibiotics is a major concern for patients with CF.
Nitric oxide is a molecule naturally produced by the body and plays an important role in every organ system, including the innate defense system against microorganisms. Externally delivered nitric oxide has been demonstrated to be an effective antimicrobial agent against a broad spectrum of microbes, including drug resistant strains. A lower concentration of nitric oxide is already an approved drug on the market from other manufacturers for the treatment of pulmonary hypertension in newborns.