Aceragen announces dosing of first patient with ACG-701 in clinical trial for treatment of melioidosis

$45 million clinical program funded by the Defense Threat Reduction Agency-

Trial initiates clinical development effort in support of ACG-701 as a treatment for multiple life-threatening orphan pulmonary diseases, including cystic fibrosis

RALEIGH-DURHAM, NC – June 15, 2022 – Aceragen, Inc., a clinical-stage biopharmaceutical company developing therapies for rare pulmonary and rheumatic diseases with high unmet medical need, announced today that the first patient has been dosed in a clinical trial evaluating ACG-701 (formerly ARV-1801) for the treatment of patients with melioidosis (NCT05105035). Melioidosis is an infection caused by the pathogen Burkholderia pseudomallei, which can result in a serious and life-threatening disease. Treatment failure rates for these infections can exceed 40% with current standard of care, resulting in persistently high morbidity and mortality.

Aceragen’s wholly owned subsidiary, Arrevus, Inc., received funding from the Department of Defense’s Defense Threat Reduction Agency (DTRA) to execute a study in Southeast Asia where melioidosis is endemic, resulting in over 150,000 cases per year.

“Melioidosis is a neglected infection with limited development programs focusing on reducing serious outcomes,” said Carl Kraus, MD, Chief Medical Officer of Aceragen and the principal investigator of the program. “It is rarely seen in the United States, although there were four highly publicized cases last year. For this reason, it’s a disease that remains of particular concern as a biodefense pathogen, with a highly antibiotic-resistant profile. I am pleased that we will be able to address both concerns with the launch of this clinical trial.”

“Melioidosis is a devastating illness with high disease burden and poor outcomes in resource-limited geographies,” added Dr. Erin Reichert, chief for DTRA’s vaccine and therapeutic development efforts. “Confirming ACG-701’s effectiveness against this pathogen will address the U.S. Joint Force’s requirement as well as advance humanitarian interests in establishing a safe and effective treatment.”

Aceragen previously announced the $45 million agreement between Arrevus and DTRA to fund manufacturing, clinical execution, and associated clinical and non-clinical development activities for ACG-701 as a medical countermeasure for the treatment and post-exposure prophylaxis (PEP) of melioidosis. ACG-701 is a patented oral formulation of sodium fusidate that rapidly achieves steady state concentration in circulation. The compound has a distinct anti-infective activity profile with potency that dramatically increases in the low-pH intracellular environment where Burkholderia pseudomallei tends to evade the host immune system. Aceragen has orphan and QIDP designations for ACG-701.

“I would like to congratulate the Aceragen team and DTRA on the initiation of this study,” said Dan Salain, Aceragen’s Chief Operating Officer. “This is an important milestone as the first multi-center, placebo-controlled clinical study to investigate a treatment for melioidosis, and the first of many studies Aceragen has planned for ACG-701 in infectious and inflammatory orphan diseases that particularly impact the lungs. We see an enormous opportunity to increase the standard of care in the orphan pulmonary space with ACG-701, a potent, oral anti-infective with anti-inflammatory properties and an extensive history of safety.”

About Aceragen

Aceragen is a biopharmaceutical company developing innovative therapeutics for rare and orphan pulmonary and rheumatic diseases with high unmet medical need. The company’s development programs include:

• ACG-801 (recombinant acid ceramidase), an investigational enzyme replacement therapy for the treatment of acid ceramidase deficiency presenting as Farber disease, and potentially other diseases associated with the dysregulation of ceramide metabolism, including cystic fibrosis.
• ACG-701 (sodium fusidate), a patented oral formulation of sodium fusidate as a medical countermeasure for melioidosis, and as a treatment for cystic fibrosis-associated pulmonary exacerbations, as well as other severe and progressive orphan pulmonary diseases.

About DTRA

DTRA enables Department of Defense (DoD), the U.S. Government, and International Partners to counter and deter weapons of mass destruction (WMD) and emerging threats. Under the auspice of the Chemical and Biological Defense Program, DTRA has the responsibility to manage and integrate the DoD chemical and biological defense science and technology programs. DTRA’s continued effort to enhance the combat support mission also advances public health services by developing innovative technologies that protect against biological threats. For more information, visit http://www.dtra.mil/.

Contact:
Aceragen, Inc.
investorrelations@aceragen.com