CF Foundation Invests Another $4.85M to Help Develop AR-501
Aridis therapy for chronic lung infections in CF now in clinical trial
The Cystic Fibrosis Foundation has invested another $4.85 million — termed an equity investment — in Aridis Pharmaceuticals to advance the clinical development of AR-501, an inhaled treatment for chronic lung infections in people with cystic fibrosis (CF).
The total funding from the CF Foundation for the clinical program of AR-501 now stands at $12.5 million, according to an Aridis press release. The new investment was in Aridis common stock on market terms.
A Phase 2a clinical trial, which is fully enrolled, is testing the safety and pharmacokinetics of increasing doses of AR-501 compared with a placebo in CF patients with Pseudomonas aeruginosa infection. P. aeruginosa is a key bacterial agent in lung disease in cystic fibrosis. Pharmacokinetics refers to the movement of a medicine into, through, and out of the body
Top-line results are expected in early 2023, the company said.
“We want to thank the CF Foundation for their continued support for the clinical development of AR-501,” said Vu Truong, PhD, CEO of Aridis.
AR-501 shows promise in CF
AR-501 is an inhaled formulation of gallium citrate. Gallium is an iron-like molecule that can suppress iron-dependent metabolic functions of bacteria, including those involved in infection and antibiotic resistance.
When delivered delivered intravenously (into-the-vein) in a Phase 2 study (NCT02354859), gallium was deemed safe and effective for CF patients. Notably, however, IV treatment requires five days of continuous infusion.
In contrast, AR-501 can be self-administered weekly using a hand-held nebulizer that delivers the medication directly to the lungs.
“IV gallium has already demonstrated that it is well tolerated and improved lung function in a CF Foundation, National Institutes of Health, and [U.S. Food and Drug Administration, FDA] funded Phase 2 clinical study,” Truong said.
The inhaled form of the therapy “could be a more effective delivery route than intravenous (IV) administered gallium,” Truong said.
According to the company, AR-501 has broad antimicrobial activity against bacterial, fungal, and nontuberculous mycobacterial species that can be found in the lungs of CF patients. The therapy has a distinct mode of action from antibiotics.
In the first part of the ongoing Phase 1/2a study (NCT03669614), AR-501 was found to be safe and well-tolerated in 48 healthy adults. This Phase 1 part of the trial is delivering the therapy in a single ascending dose (SAD) or a multiple ascending dose (MAD) regimen.
In Phase 2a, three groups of patients are randomly assigned to receive three weekly MAD of AR-501 or a placebo. In a fourth group, patients are randomly assigned to three weekly doses of AR-501 — at those same doses — or a placebo. All are followed for 28 days after the last dose.
After reviewing the latest and still blinded safety data from the trial, the FDA agreed to include a higher dose group after enrollment of the current dose groups is complete.
In the U.S., the investigational therapy has won orphan drug designation, and was granted fast track and qualified infectious disease product designations for lung infections in CF patients. In Europe, it also received orphan drug designation for the same indication.