Aro Biotherapeutics Doses First Patient in Phase 1b Trial of ABX1100 in Late-Onset Pompe Disease (LOPD)

Trial aims to establish safety and provide proof of concept for GYS1 inhibition as promising new treatment strategy in patients with late-onset Pompe disease

Dosing milestone extends momentum of ABX1100 development program, with data from normal healthy volunteers to be presented at WORLDSymposium™ in February

Excerpt from the Press Release:

PHILADELPHIA–(BUSINESS WIRE)–Aro Biotherapeutics, a clinical-stage biotechnology company working to develop potent and tissue-targeted medicines, today announced the dosing of the first patient in its clinical trial of ABX1100, a novel treatment for late-onset Pompe disease (LOPD).

Excerpt from the Press Release:

“We are excited to initiate dosing in the Phase 1b trial, the results of which should provide the first proof of concept for GYS1 inhibition as a therapeutic strategy in Pompe disease,” said Susan Dillon, Ph.D., co-founder, president and chief executive officer of Aro. “The results build on promising data from healthy volunteers.”

ABX1100 is uniquely designed to deliver a glycogen synthase 1 (GYS1)-specific short-interfering RNA (siRNA) therapeutic payload directly to muscle tissue. Once inside the muscle tissue, the siRNA inhibits the production of GYS1, a key enzyme required for synthesizing glycogen. When combined with enzyme replacement therapy (ERT) of the genetically deficient enzyme, ABX1100 has shown significant reductions in glycogen in preclinical models and may reduce the need for frequent infusions of ERT.

“The Pompe disease community has long awaited the development of a treatment that overcomes the limitations of enzyme replacement therapy,” said Brad Crittenden, executive director of the Canadian Association of Pompe. “We are eager to see how well muscle-targeted GYS1 reduction with ABX1100 works to treat the disease.”

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