[A second clinical trial site has been added!]
Aro Biotherapeutics has launched a Phase 1b clinical trial for ABX1100, a novel therapy designed for adults with late-onset Pompe disease (LOPD). ABX1100 is based on substrate reduction therapy (SRT), a therapeutic approach that aims to slow disease progression by reducing the production of harmful substrates—in this case, glycogen. In Pompe disease, an enzyme deficiency leads to excess glycogen buildup in muscle cells, causing significant muscle weakness and respiratory issues.
ABX1100 utilizes Aro’s Centyrin platform to deliver small interfering RNA (siRNA) directly to muscle tissue by binding to the CD71 receptor on muscle cells. This siRNA targets and reduces the activity of glycogen synthase 1 (GYS1), an enzyme responsible for glycogen production. By limiting glycogen production, ABX1100 addresses the root cause of muscle damage by controlling the amount of excess glycogen stored in the cell.
In a recently completed Phase 1a trial involving healthy volunteers, ABX1100 was well tolerated and demonstrated durable GYS1 mRNA knockdown in muscle biopsies, with effects lasting at least 10 weeks following a single dose. These findings support the progression to the Phase 1b study in LOPD patients. This is for LOPD patients ≥ 18 years of age.
ABX1100 has received Orphan Drug Designation and Rare Pediatric Disease status from the U.S. Food and Drug Administration (FDA), highlighting its potential as a novel therapeutic approach for Pompe disease.
Aro Biotherapeutics is using its Centyrin platform to develop therapies that change the course of treatment for devastating diseases. Their pipeline focuses on novel medicines that enable targeted delivery of oligonucleotides to specific cell types.
The first clinical trial site to open is at the M.A.G.I.C. Clinic in Calgary, Alberta, Canada. Dr Aneal Khan, a pediatrician and medical geneticist, is the principal investigator.
A second trial site has been added at the Neuromuscular & Neurometabolic Clinic, in Hamilton, Ontario,
For more information
Olu Olunaike
Clinical Research Coordinator
Genetic Treatments and Clinical Trials Unit
M.A.G.I.C Clinic
971 64 Ave NE, Suite #215
Calgary, AB. T2E 7Z4
Email: olugbenga.olunaike@magiccalgary.ca
Phone: 587-885-3076
– or –
Erin Hatcher
Clinical Research Coordinator
Neuromuscular & Neurometabolic Clinic
1200 Main Street West – 2H
Hamilton, Ontario
Email: hatchere@hhsc.ca
Phone: 905-521-2100 ext 76929
Source
Clinical trial: clinicaltrials.gov/study/NCT06109948