RARE Daily

Cystic Fibrosis Foundation Makes $4.85 Million Equity Investment in Aridis Pharma

December 12, 2022

The Cystic Fibrosis Foundation has invested $4.85 million in Aridis Pharmaceuticals to support the ongoing development of AR-501, an inhalable broad-spectrum anti-infective currently under development for controlling debilitating chronic lung infections in cystic fibrosis patients.

Including this funding, the CF Foundation has provided a total of $12.5 million in support. Enrollment for a phase 2a study of AR-501 in CF patients was completed in November 2022 and Aridis anticipates reporting top-line results from the study in the first quarter of 2023.

“We want to thank the CF Foundation for their continued support for the clinical development of AR-501, an inhaled form of gallium, which we believe could be a more effective delivery route than intravenous administered gallium,” said Vu Truong, CEO of Aridis Pharmaceuticals. IV gallium has already demonstrated that it is well tolerated and improved lung function in a CF Foundation, National Institutes of Health, and FDA funded phase 2 clinical study.”

Cystic fibrosis (CF) is a genetic disorder caused by mutations in the CF transmembrane conductance regulator (CFTR) protein. This leads to numerous medical problems, including abnormal airway secretions, mucus accumulation, and opportunistic bacterial infections. Pseudomonas aeruginosa is the most significant pathogen, with greater than 80 percent of CF patients becoming chronically infected with P. aeruginosa by 18 years of age. Also of increasing prevalence in CF patients are infections caused by Staphylococcus aureus and non-tuberculosis mycobacterium. Extensive antibiotic treatment has led to selection of resistant strains, which are less responsive to drug treatment and exacerbate disease progression.

AR-501 is an inhalable broad-spectrum anti-infective currently under development for controlling debilitating chronic lung infections in CF patients. AR-501 functions as an iron analog, which is a mechanism of action that is distinct from antibiotics, and has broad antimicrobial activity against several common bacterial, fungal, and nontuberculous mycobacterial species which are sometimes found to colonize the lungs of CF patients.

The current study is a randomized, double-blind, placebo-controlled phase 2a trial investigating the safety and pharmacokinetics of multiple ascending doses of inhaled AR-501 in CF patients with chronic bacterial lung infections. Based on available blinded safety data of the on-going phase 2a study, the U.S. Food and Drug Administration has concurred with the company’s proposal to include an optional higher dose cohort after enrollment of the current dose cohorts.

AR-501 has been granted Orphan Drug, Fast Track, and Qualified Infectious Disease Product (QIDP) designations by the FDA. In addition, the European Medicines Agency granted Orphan Drug status to AR-501.

Photo: Vu Truong, CEO of Aridis Pharmaceuticals

Author: Rare Daily Staff

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