Phase 2 Trial of ACG-701 to Treat CF Exacerbations Starting in US

Add-on therapy an oral form of sodium fusidate, antibiotic in wide use

Patricia Inácio, PhD avatar

by Patricia Inácio, PhD |

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A Phase 2 trial of ACG-701, an investigational oral treatment for pulmonary exacerbations in people with cystic fibrosis (CF), is underway at clinical sites in the U.S.

The REPRIEVE trial (NCT05641298), launched in December, is evaluating the safety, pharmacokinetics, and effectiveness of ACG-701, being developed by Aceragen, when added to optimized background therapy in CF patients with pulmonary exacerbations. Pharmacokinetics refers to the movement of a medicine into, through, and out of the body.

Data from the placebo-controlled study is expected later this year, a company press release stated.

REPRIEVE is supported by a $3.5 million investment from the Cystic Fibrosis Foundation and working with the foundation’s Therapeutic Development Network.

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Trial to enroll up to 80 CF patients with an acute pulmonary exacerbation

Pulmonary exacerbations, the acute worsening of lung symptoms, are significant and typically linked with respiratory infections. No therapies specifically for pulmonary exacerbations in CF patients have been approved in the U.S.

ACG-701, originally developed as by Arrevus (later acquired by Aceragen), is an oral formulation of sodium fusidate (also known as fusidic acid), a bacteria-killing antibiotic. In particular, sodium fusidate is effective against Staphylococcus aureus, a type of bacteria that commonly infects the lungs of people with CF.

Sodium fusidate has been used for over 50 years outside the U.S., and is part of the treatment guidelines for pulmonary exacerbations in the U.K. and Australia, Aceragen reported. The compound is not approved in the U.S.

In the Phase 2 trial, up to 80 CF patients ages 12 and older will be randomly assigned to either ACG-701 or a placebo tablet, given twice a day for 14 days, in addition to optimized background therapy. Treatment will be preceded by a 24-hour screening period.

Participants, all with an acute pulmonary exacerbation diagnosis, can be hospitalized or treated as an outpatient and will be followed for about 28 days. A total of five clinic visits are required, as is the completion of a daily electronic symptom questionnaire. Contact information is available for the trial, but sites are not yet listed.

The U.S. Food and Drug Administration designated ACG-701, then known as ARV-1801, a qualified infectious disease product and an orphan drug, both intended to support and speed the development and review of needed therapies. ACG-701 also received fast track designation from the FDA.

Aceragen’s clinical program also includes ACG-801, a therapy candidate for Farber disease, a rare condition characterized by the progressive buildup of fat in the central nervous system (the brain and spinal cord) and organs.

“During the course of this year, we anticipate the achievement of significant clinical milestones that include two Phase 2 data read-outs for ACG-701, as well as lifting of the clinical hold and advancing toward the initiation of our Phase 2/3 trial in Farber disease for ACG-801,” said John Taylor, Aceragen’s CEO.

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