ABX1100

ABX1100, an experimental substrate reduction therapy for late-onset Pompe disease (LOPD), was well tolerated and seemed to work as designed in an early clinical trial involving nine adults with LOPD, new data show. Developer Aro Biotherapeutics noted that, in addition to meeting the study’s primary endpoints, or goals,…

ABX1100, an experimental substrate reduction therapy being developed by Aro Biotherapeutics, showed strong and sustained biological activity and early signs of clinical benefit in people with late-onset Pompe disease (LOPD), according to preliminary trial results. The therapy was also well tolerated. The preliminary results are based on the…

A Phase 1b clinical trial testing substrate reduction therapy ABX1100 in late-onset Pompe disease (LOPD) has completed enrollment, developer Aro Biotherapeutics said. This part of the trial (NCT06109948) investigates the medication’s safety, tolerability, and pharmacological profile in adults with LOPD. ABX1100 is given by intravenous (into-the-vein) infusion.

The U.S. Food and Drug Administration (FDA) has cleared Aro Biotherapeutics to conduct a Phase 1 clinical trial to test ABX1100, its experimental substrate reduction therapy, in people with late-onset Pompe disease (LOPD). The clearance of Aro’s investigational new drug (IND) application comes as the company is…

The first healthy volunteer has been enrolled in a Phase 1 study testing ABX1100 — a novel substrate reduction therapy from Aro Biotherapeutics — as a treatment for Pompe disease. This will be the first-in-human trial for the experimental therapy, meaning it’s the first time ABX1100 has been…

The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease designation to Aro Biotherapeutics’ ABX1100, an investigational substrate reduction therapy for Pompe disease. This designation is awarded to experimental therapies for rare pediatric disorders with serious or life-threatening manifestations that primarily affect young people from birth…

ABX1100, an experimental substrate reduction therapy that Aro Biotherapeutics is developing for Pompe disease, has been granted an orphan drug designation by the U.S. Food and Drug Administration (FDA). The FDA gives this designation to investigational treatments designed to improve care for rare diseases affecting fewer than…