Oct 27 (Reuters) - BridgeBio Pharma ( BBIO ) said on
Monday its experimental drug for a rare muscle disorder, which
currently has no approved treatments, showed improvements in
motor and lung function in a late-stage trial.
The oral drug, BBP-418, is being tested in patients with a
form of limb-girdle muscular dystrophy, or LGMD, a group of
inherited genetic disorders characterized by progressive muscle
weakness and wasting.
LGMD has an estimated global prevalence ranging from 1 to 6
per 100,000 people, with about 5,000 people in the U.S.
currently living with the condition.
BBP-418 met the main goal of significantly increasing
glycosylated αDG, a key marker of LGMD, by 1.8 times from
baseline when compared to placebo in an interim analysis of the
trial at three months.
Glycosylated αDG is a form of the protein αDG that has
undergone proper glycosylation, a biochemical modification
essential for its function in muscle cells.
The therapy at 12 months showed a statistically significant
difference in serum CK, a marker of muscle damage, an increase
in walking speed by 0.27 metre/second and an improvement of
about 5% in the volume of air participants could forcibly exhale
when compared to placebo.
BridgeBio intends to engage with the U.S. Food and Drug
Administration later this year to discuss these data and plans
to submit a marketing application for BBP-418 in the first half
of 2026.
The company plans to present detailed results from the trial
at a future medical meeting.