BBP-418 shows promise in LGMDR9 phase 3 trial

3 November 2025

BBP-418 is a potential treatment for limb-girdle muscular dystrophy R9 (LGMDR9). People with LGMDR9 have changes in a gene called FKRP, which affects how a protein called alpha-dystroglycan is made. Normally, this protein needs to go through a process called glycosylation to work properly. In LGMDR9, this process doesn’t happen as it should, and that leads to muscle damage over time.

BBP-418 is designed to help improve glycosylation by giving the body more of the building blocks it needs.

Scientists are testing BBP-418 in a phase 3 trial, which is a large study to check if the medicine works and is safe. Some people in the trial got BBP-418, and others got a placebo (a dummy treatment).

After three months of treatment, early results showed that glycosylation nearly doubled, which means the protein was processed better. But even with this improvement, levels were still much lower than normal, reaching only about 17% of what’s seen in people without LGMDR9. The treatment also lowered a marker in the blood called creatine kinase, which shows muscle damage.

After one year, results suggest that BBP-418 may help improve walking speed and lung function. People who took the treatment walked faster and had better breathing scores compared to the start of the trial, while those who got the placebo walked more slowly and their lung function got worse over time.

More results from the trial will be shared soon. The company behind BBP-418 hopes to start the approval process in the United States next year. There’s no news yet about plans for the UK.