Aro Biotherapeutics receives FDA Orphan Drug Designation for Pompe disease treatment

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for Aro Biotherapeutics’ ABX1100, an investigational Centyrin-siRNA conjugate that targets the Gys1 gene in the muscle, for the treatment of Pompe disease.

“We are pleased to have received this designation and are gratified by the FDA’s recognition of the potential of ABX1100 to improve the lives of patients living with Pompe disease,” said Mittie Doyle, MD, FACR, chief medical officer of Aro Biotherapeutics. “We believe our novel treatment approach has the opportunity to address the great unmet need that exists in Pompe disease, and we are excited to advance ABX1100 to clinical trials in the coming year.”

Pompe disease is a rare, genetic disease characterized by debilitating muscle weakness that progresses over time and arises from a mutation in the enzyme responsible for breakdown of glycogen in the muscle, acid alpha-glucosidase (GAA). As a result of this mutation, patients with Pompe disease have elevated levels of glycogen which drive disease progression.

Patients are currently treated with enzyme replacement therapy (ERT), with recombinant GAA administered intravenously. However, the inability to target and efficiently deliver ERT to skeletal muscle has limited the efficacy and safety profile of these therapies, and new medicines with novel mechanisms are needed to further improve patient outcomes.

ABX1100 targets the gene for glycogen synthase 1 (Gys1), an enzyme that is responsible for glycogen synthesis in muscle. Inhibition of Gys1 has been shown to reduce glycogen levels and thus represents a novel treatment approach for Pompe disease. At the TIDES USA 2022 meeting, Aro Biotherapeutics presented data demonstrating that ABX1100 profoundly reduces Gys1 mRNA and GYS1 protein, leading to meaningful reductions in glycogen levels in the skeletal muscle in the Pompe mouse disease model. Aro Biotherapeutics anticipates entering clinical trials with ABX1100 in mid-2023.

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