Aceragen Announces $3.5 Million Development Award from Cystic Fibrosis Foundation to Progress Novel Therapy for CF-Related Exacerbations

Agreement supports late-stage clinical study for ACG-701 beginning in 2022

Advances Aceragen’s development effort to treat life-threatening rare and orphan diseases

RALEIGH-DURHAM, NC – January 06, 2022 – Aceragen, Inc., a clinical-stage biopharmaceutical company developing novel therapeutics for orphan diseases with high unmet medical need, today announced that its wholly-owned subsidiary, Arrevus, Inc., has received an award from the Cystic Fibrosis Foundation for up to $3.5 million to support the clinical development of ACG-701, a proprietary formulation of sodium fusidate, as a treatment for cystic fibrosis-related pulmonary exacerbations. There are currently no FDA-approved therapies for this indication.

Cystic fibrosis (“CF”) is characterized by chronic respiratory infections that can lead to pulmonary exacerbations, which result in acute worsening of symptoms, progressive loss of lung function and decreased survival. The active ingredient in ACG-701 is sodium fusidate, a compound with significant history outside the United States in the treatment of methicillin- resistant Staphylococcus aureus (“MRSA”) and related pathogens that cause exacerbations in the lungs of patients with cystic fibrosis.

“The body of data supporting sodium fusidate’s safety and utility in treating CF pulmonary exacerbations is extensive,” commented Carl Kraus, MD, Aceragen’s Chief Medical Officer. “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.”

“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,” added John Taylor, Chief Executive Officer of Aceragen.

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About Aceragen

Aceragen is a biopharmaceutical company developing innovative therapeutics for rare and orphan diseases. The Company is advancing ACG-801 (rhAC) as an investigational enzyme replacement therapy for the treatment of patients with Farber disease and potentially other diseases associated with the dysregulation of ceramide metabolism, including cystic fibrosis. Via its Arrevus subsidiary the Company is developing a patented oral formulation of sodium fusidate, ACG-701, as a treatment for cystic fibrosis pulmonary exacerbations and other severe, orphan infectious diseases. Aceragen is actively seeking complimentary assets to expand its portfolio.


Aceragen, Inc.