NovaBiotics has established a strong IP position, with granted and pending global patents providing exclusivity in the use of cysteamine, the active agent in Lynovex, for CF. Orphan drug designation has been granted by the FDA (in the United States) and COMP (in Europe) for Lynovex in the treatment of CF. The Lynovex development program is supported by the Cystic Fibrosis Trust.
Clinical and Commercial Development
Lynovex in oral (hard gel capsule) form successfully completed a phase IIa clinical study in 2014/15, demonstrating its tolerability, safety and utility in adult CF patients with stable disease. The first phase of the global, pre-registration CARE-CF clinical trials for oral Lynovex in acute infectious CF exacerbations is now underway. See https://clinicaltrials.gov/show/NCT03000348 and Eudra CT 2015-004986-99. To learn more, please visit www.lynovex.com.
The unique multi-action properties of Lynovex have been demonstrated in in vitro & in vivo studies, as well as an ex vivo sputum study of CF patients. Preclinical studies are currently underway for inhaled Lynovex and NovaBiotics intends to initiate a proof-of-concept clinical study for inhaled Lynovex Inhaled in 2017/18.
Mechanism of Action
Cysteamine based Treatments in CF and Other Conditions
Lynovex in hard gel capsule form should not be confused with cystagon, a medicine also containing cysteamine for use in the metabolic disease, cystinosis. The mechanism of action, dose, dose frequency and duration of treatment for both conditions with these medicines is very different. Cysteamine’s tolerability and safety has only been evidenced thus far in adult CF patients when used as an acute therapy (e.g. per the CARE-CF-1 study, for 14 days).
Cystic fibrosis (“CF”) is a life-limiting disease affecting around 70,000 individuals worldwide, including approximately 65,000 in the United States and Europe. The market for CF therapies is estimated at $6 billion. The disease is characterized by the build-up of thick mucus in a number of organs including the linings of the lungs; creating an ideal environment for bacterial infection and colonization. Resultant, progressive lung disease is the most critical manifestation in CF with mortality in >90% of all CF patients due to respiratory failure. CF patients generally undergo intensive, long-term antibiotic therapy, resulting in unavoidable resistance to many anti-infective drugs. The emergence of CF-specific multi-drug resistant pathogens is a source of major concern.
Exacerbations, intermittent episodes of acute worsening of CF symptoms often as a result of bacterial infection, are a key feature of cystic fibrosis. They are the principle cause of morbidity and mortality as a result of the decline in lung function. A typical adult patient would have multiple exacerbations annually.