CF is an autosomal, recessive inheritable genetic disease affecting 70,000-80,000 patients world-wide. It has the highest incidence and prevalence in Caucasian populations and is less common in other population groups.
The disease is caused by a defective gene that results in the accumulation of mucus in the respiratory tract, pancreas, intestine and other organs. The excess of thick mucus in the lungs makes patients susceptible to serious and recurring lung infections that are difficult to eradicate, eventually leading to permanent lung damage. Patients also suffer from digestive abnormalities resulting in pain, bloating, constipation and intestinal obstruction also known as DIOS (distal intestinal obstructive syndrome).
Some CF patients may take as many as 30+ different medications and supplements every day, and in the western world the predicted median age of survival for a person with CF is in the mid 40’s.
AlgiPharma’s two product candidates for CF include a dry powder for inhalation (DPI), and a solution for nebulisation containing Oligo-G. The DPI drug is inhaled using a monodose dry powder inhaler.
Results from pre-clinical models and clinical trials have shown that the drug candidate reduces the viscosity of CF sputum and improves the efficacy of antibiotics.
Reduction in sputum viscosity will enable patients to ease clearing of mucus from the lungs and airways and thus reduce microbial burden and reduce the need for physical therapy.
The potentiating effect on antibiotic treatment may also increase efficacy in eradicating the bacteria causing respiratory infections, a common complication for CF patients.
Phase 1, 2a, 2b, and drug deposition studies have shown that the product candidate is safe and well tolerated.