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Aro Biotherapeutics Receives FDA Orphan Drug Designation for ABX1100 for the Treatment of Pompe Disease

Thursday, August 11, 2022

Aro Biotherapeutics, a leading biotechnology company dedicated to advancing tissue-targeted genetic medicines, has announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ABX1100, an investigational Centyrin-siRNA conjugate designed to target the Gys1 gene in muscle tissue for the treatment of Pompe disease.

Dr. Mittie Doyle, Chief Medical Officer of Aro Biotherapeutics, expressed their satisfaction with the designation, stating, "We are delighted to have received this recognition and appreciate the FDA's acknowledgement of the potential of ABX1100 to enhance the lives of patients suffering from Pompe disease. We believe our innovative treatment approach holds promise in addressing the significant unmet need in Pompe disease, and we are excited to advance ABX1100 into clinical trials in the upcoming year."

Pompe disease is a rare genetic disorder characterized by progressive muscle weakness caused by a mutation in the acid alpha-glucosidase (GAA) enzyme responsible for glycogen breakdown in the muscles. This mutation results in elevated glycogen levels that contribute to disease progression. Currently, patients with Pompe disease undergo treatment with intravenous enzyme replacement therapy (ERT) involving recombinant GAA. However, the effectiveness and safety of these therapies have been limited by the inability to efficiently target and deliver ERT to skeletal muscle. There is a pressing need for new medications with novel mechanisms to further improve patient outcomes.

ABX1100 specifically targets the Gys1 gene, which encodes for the glycogen synthase 1 enzyme responsible for glycogen synthesis in muscle. Inhibiting Gys1 has shown promise in reducing glycogen levels and presents a new approach to treating Pompe disease. Aro Biotherapeutics presented data at the TIDES USA 2022 meeting demonstrating that ABX1100 significantly reduces Gys1 mRNA and GYS1 protein levels, leading to meaningful reductions in glycogen levels in the skeletal muscle of a Pompe disease mouse model. Aro Biotherapeutics plans to initiate clinical trials with ABX1100 in mid-2023, marking a significant step forward in their research and development efforts.

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