BridgeBio surges as muscle disorder drug scores ‘best case’ trial win
BridgeBio Pharma (BBIO.O), opens new tab 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, sending its shares up 15%.
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.
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.
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.
Evercore ISI analyst Cory Kasimov called the results a “blowout best-case outcome” that materially increase confidence that BridgeBio is poised to become a multi-product biotech, with each asset representing a blockbuster opportunity.
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.
“We are going to be seeking alignment with the FDA if accelerated approval is still the right strategy, or if we should really be seeking a full approval here,” said Christine Siu, CEO of ML Bio Solutions, a subsidiary of BridgeBio.
BridgeBio said it has already built a lot of infrastructure for its heart disease drug Attruby, which it plans to leverage for the commercialization of BBP-418.
