First patient dosed in Phase Ib Pompe disease trial

Aro Biotherapeutics has dosed the first patient in a Phase Ib trial of ABX1100, a novel treatment for late-onset Pompe disease (LOPD), a rare inherited disorder that causes progressive muscle weakness. The condition is brought about by a deficiency in alpha-glucosidase, an enzyme necessary for the breakdown of complex carbohydrates into glucose in the small intestine.

The first-in-human trial is an open-label study evaluating the drug’s safety and tolerability in LOPD patients currently receiving enzyme replacement therapy (ERT).

Philadelphia-based Aro Biotherapeutics’sABX-1100 is designed to deliver a glycogen synthase 1 (GYS1) specific short-interfering RNA (siRNA) therapeutic payload directly to muscle tissue. According to the company, the siRNA inhibits the production of GYS1 andwhen paired withERT, ABX1100 has shown significant reductions in glycogen in preclinical models.

Susan Dillon, president and chief executive officer of Aro, said: “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. The results build on promising data from healthy volunteers.”

The Phase Ib trial (NCT06109948) is seeing patients receive one dose of ABX1100 on day one of the study and a booster dose on day 29. Subjects will then be followed for 16 weeks to assess response. Aro Biotherapeuticsrecently completed a Phase Ia trial, conducted in healthy volunteers, which demonstrated a durable GYS1 mRNA knockdown in muscle biopsies. A gene knockdown is a technique by which the expression of one or more genes is reduced.

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

Elsewhere in the world of advanced therapies, the UK has seen a 70% rise in Phase I advanced therapy medicinal product (ATMP) studies, with research into oncology taking top place.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

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