The US Food and Drug Administration (FDA) has granted Orphan Drug Designation for Aro Biotherapeutics’ ABX1100 to treat Pompe disease.
An investigational Centyrin-small interfering ribonucleic acid (siRNA) conjugate, ABX1100 acts on the glycogen synthase 1 (Gys1) gene in the muscle.
Gys1 is an enzyme that synthesises glycogen in muscle. Hindering this enzyme was found to lower glycogen levels and is a new approach to treat Pompe disease.
In a Pompe mouse disease model, ABX1100 was demonstrated to greatly reduce the Gys1 mRNA and GYS1 protein, causing a meaningful decline in glycogen levels in the skeletal muscle.
The company plans to advance ABX1100 into clinical trials in the middle of next year.
Aro Biotherapeutics chief medical officer Mittie Doyle said: “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.
“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.”
A biotechnology firm, Aro Biotherapeutics focuses on developing tissue-targeted genetic treatments.
Pompe disease is a rare genetic ailment that causes debilitating weakness of muscles which advances over time.
It is caused by a mutation in the acid alpha-glucosidase (GAA) enzyme, which disintegrates glycogen in the muscle.
Due to the mutation, Pompe disease patients have increased glycogen levels which leads to the progression of the ailment.
At present, patients receive enzyme replacement therapy (ERT) with recombinant GAA given intravenously.
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