CF Foundation Awards $3.5M to Develop Antibiotic ACG-701

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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The Cystic Fibrosis Foundation has awarded up to $3.5 million to Aceragen to support the clinical development of ACG-701, an investigational antibiotic to treat pulmonary exacerbations in people with cystic fibrosis (CF).

“We appreciate the generous support of the Cystic Fibrosis Foundation and are looking forward to 2022 as we work closely with the Foundation to initiate clinical development for ACG-701 in CF and multiple other indications,” John Taylor, CEO of Aceragen, said in a press release.

Aceragen gained rights to ACG-701 last year when it acquired the therapy’s original developer Arrevus. The company has announced plans to start a Phase 2/3 clinical trial that will test the safety and effectiveness of ACG-701 (formerly ARV-1801 and ACG-721) in CF patients with pulmonary exacerbations. The trial is expected to launch later this year.

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Pulmonary exacerbations, also called flares or flare-ups, are times when lung-related symptoms worsen suddenly. These exacerbations typically are associated with lung infections — the thick mucus that is characteristic of CF provides a fertile breeding ground for disease-causing bacteria.

In the U.S., there are no therapies specifically approved to treat pulmonary exacerbations of CF. The U.S. Food and Drug Administration (FDA) has granted ACG-701 a form of priority review and an orphan drug designation, both of which aim to speed the development and review of needed therapies.

ACG-701 is an oral dosing formulation of fusidic acid, which is a bacteria-killing antibiotic medicine. In particular, fusidic acid is effective for killing a type of bacteria called Staphylococcus aureus, thought to infect most people with CF.

In the U.K. and Australia, fusidic acid is a first-line treatment for methicillin-resistant S. aureus, or MRSA, a strain of this bacteria that can cause serious lung infections and is resistant to other commonly used antibiotics.

“The body of data supporting sodium fusidate’s safety and utility in treating CF pulmonary exacerbations is extensive,” said Carl Kraus, MD, chief medical officer of Aceragen.

In addition to its bacteria-killing effects, fusidic acid also is believed to have anti-inflammatory properties.

“Because it has the unusual advantage of its potency increasing many fold under acidic conditions, as well as meaningful anti-inflammatory activity, sodium fusidate is well-suited to treat the frequent, tenacious infections in the inflamed, low-pH environment of the CF lung,” Kraus said.

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