Treatment for antibiotic resistant lung and urinary tract infections
The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for AB569 in the treatment of Pseudomonas aeruginosa (P. aeruginosa) pulmonary infections in patients with cystic fibrosis.
Treatment for Respiratory Pseudomonas aeruginosa Infections
Two deadly diseases, cystic fibrosis (CF) and chronic obstructive pulmonary disease (COPD), are characterized by airway bacterial infections that significantly impact the overall quality of patient’s lives. There are approximately 40,000 CF patients and some 14.2 million individuals diagnosed with COPD in the United States. In both diseases, the Gram-negative bacterium, Pseudomonas aeruginosa (P. aeruginosa), constitutes a significant and problematic cause of the pulmonary exacerbations that result in frequent hospitalizations of these patients.
In particular, the mucoid form of P. aeruginosa is a very challenging infection to treat due to its high resistance to both antibiotics and phagocyte–mediated killing. Once patients present with the mucoid form of P. aeruginosa, their overall lung function precipitously declines resulting in a poor prognosis.
CF patients are predisposed to lung infections due to abnormal mucus production in the lungs and airways. P. aeruginosa infects 40% of CF patients between the ages of 6 and 10 years of age. By the age of 17, the frequency of infection increases to 60% and reaches approximately 75% of all CF patients between the ages of 25 and 34. Thus, there is an urgent clinical need for the development of novel effective treatments in this area.
AB569 constitutes an innovative, bactericidal method to treat mucoid and nonmucoid P. aeruginosa pulmonary infections that are resistant to traditional antibiotics.
Orphan Drug Designation for AB569
In November, 2015, Arch Biopartners received Orphan Drug Designation on AB569 from the U.S. Food and Drug Administration for the treatment of P. aeruginosa lung infections in cystic fibrosis (CF) patients.
The Orphan Drug Designation has been granted for the combination of two active ingredients of AB569: sodium nitrite and ethylenediaminetetraacetic acid. AB569 is to be administered to patients as a nebulized (inhaled) solution. AB569 was invented at the University of Cincinnati in the lab of Dr. Daniel Hassett.
The FDA Office of Orphan Products Development grants Orphan Drug Designation to drugs and biologics to encourage the development of new medicines for the safe and effective treatment of underserved, rare diseases or disorders that affect less than 200,000 patients in the U.S.
The Orphan Drug Designation qualifies Arch for a seven-year term of market exclusivity to sell AB569 in the U.S. following FDA approval of the drug. Additionally, as Arch takes AB569 through the regulatory and human trial process, the Orphan Drug Designation provides an accelerated review and approval process, potential grant funding, tax benefits and an exemption from certain user fees.
Human Trial Plans
Arch Biopartners, d3 Medicine and Dr. Hassett’s team at the University of Cincinnati are preparing to conduct a human trial to test the efficacy of AB569 for the treatment of P. aeruginosa lung infections in CF patients.
During 2015, AB569 successfully completed pre-clinical in vivo and in vitro validation studies. In all studies, AB569 has demonstrated significant efficacy against P. aeruginosa. This includes a recent study involving a chronic pulmonary infection model in mice.
The Arch team believes these results provide the scientific rationale for pursuing a human trial to test the safety and efficacy of AB569 for cystic fibrosis (CF) patients whose airways are chronically infected with P. aeruginosa. In this regard, Arch management is currently arranging the necessary toxicology program to support an Investigational New Drug Application for the first human trial for AB569.