Aceragen Receives Grant from Cystic Fibrosis Foundation to Advance Novel Therapy

Cystic Fibrosis Foundation has given Aceragen a $3.5 million grant to develop a novel treatment for CF-related exacerbations.

FREMONT, CA: "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," comments John Taylor, Aceragen’s Chief Executive Officer. Aceragen, a clinical-stage biopharmaceutical company focused on developing novel therapeutics for orphan diseases with a high unmet medical need, has a wholly-owned subsidiary, Arrevus. The company has been awarded up to $3.5 million by the Cystic Fibrosis Foundation to assist the clinical development of ACG-701, a proprietary formulation of sodium fusidate, as a treatment for cystic fibrosis-related pulmonary exacerbations. There are no FDA-approved treatments for this indication at the moment.

As a biopharmaceutical firm, Aceragen is dedicated to discovering and developing treatments for the world's most unmet medical needs. Patients with Farber illness and other ceramide-metabolism-related diseases may benefit from ACG-801 (rhAC), an enzyme replacement medication currently being developed by the company. 

As part of its Arrevus subsidiary, the company plans to use sodium fusidate, ACG-701, as an oral formulation to treat severe, rare infectious disorders such as cystic fibrosis pulmonary exacerbations. Adding complementary assets to Aceragen's portfolio is a top priority.

Cystic fibrosis (CF) is characterized by persistent respiratory infections that culminate in pulmonary exacerbations. These exacerbations cause an immediate worsening of symptoms, progressive loss of lung function, and decreased survival. ACG-701 contains sodium fusidate, a chemical with a long history of usage outside the United States to treat methicillin-resistant Staphylococcus aureus (MRSA) and associated bacteria that cause exacerbations in the lungs of cystic fibrosis patients.

“The body of data supporting sodium fusidates safety and utility in treating CF pulmonary exacerbations is extensive,” comments Carl Kraus, MD, Aceragens 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.”