Aridis Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to AR-501, the Company's inhaled formulation of gallium citrate for treatment of lung infection in patients with cystic fibrosis.

Cystic fibrosis patients often suffer from severe, persistent secondary bacterial lung infections due to their underlying lung disease which results in an immune-compromised state. AR-501 is a non-antibiotic, small molecule anti-infective in which gallium functions as an iron analog that antagonizes multiple iron-dependent pathways in microbes and thus, subverts multiple key functions in bacteria. Preclinical efficacy and safety data have demonstrated that AR-501 works synergistically with multiple antibiotics, is effective against antibiotic resistant strains, and has a low intrinsic resistance profile. AR-501 is being developed as a self-administered, weekly treatment which is being evaluated in an-going Phase 1/2a clinical trial funded by the Cystic Fibrosis Foundation (ClinicalTrials.gov Identifier: NCT03669614).  The Company expects to report data from the Phase 1 portion of the trial which consists of healthy subjects in Q1 2020 and the Phase 2a segment with cystic fibrosis subjects in Q2 2021.

"We are pleased to receive the Orphan Drug Designation for AR-501. This candidate diversifies our portfolio of novel non-antibiotic anti-infective therapies and potentially provides a novel, convenient treatment option for cystic fibrosis patients to manage their chronic, life-long lung infections," commented Vu Truong, PhD, Chief Executive Officer of Aridis Pharmaceuticals, Inc.

The FDA's Office of Orphan Products Development (OOPD) advances the evaluation and development of products that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the U.S. The OOPD evaluates scientific and clinical data submissions from sponsors to identify and designate products as promising for rare diseases and to further advance scientific development of such promising medical products. Orphan drug designation provides incentives for sponsors to develop products for rare diseases. These incentives may include a partial tax credit for certain clinical trial expenditures, the waiver of certain FDA user fees, and if approved, potential eligibility for seven years of orphan drug marketing exclusivity.

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