BBIO Stock Soars After Experimental Drug Shows Improvement In Inherited Muscle Disorder In Late-stage Trial

• Limb-girdle muscular dystrophy type 2I/R9 patients treated with BBP-418 in a late-stage study demonstrated improvements in motor and pulmonary function at 12 months, the company said. 
• BridgeBio now intends to file a new drug application for the therapy's approval in the first half of 2026.
  

BridgeBio Pharma, Inc. (BBIO) on Monday announced that limb-girdle muscular dystrophy type 2I/R9 patients treated with BBP-418 in a late-stage study demonstrated improvements in motor and pulmonary function at 12 months.

Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) is a rare, inherited disorder leading to progressive weakness and wasting of the arm and leg muscles, commonly later accompanied by pulmonary muscle and cardiac muscle involvement. It is caused by mutations in the FKRP gene, impairing glycosylation of alpha-dystroglycan (αDG), a protein associated with stabilizing muscle cells.

Other Trial Observations

BridgeBio said that glycosylated αDG increased by 1.8x from baseline at 3 months, whereas the placebo group showed little change. These improvements were sustained at 12 months in BBP-418-treated individuals, it further added.

Furthermore, an average reduction of 82% from baseline in serum creatine kinase, a marker of muscle damage, was observed in BBP-418-treated individuals, it added.

Drug Safety

BBP-418 was well-tolerated in the study with no new or unexpected safety findings observed, the company said. Overall, interim analysis data are supportive of a favorable risk-benefit profile for BBP-418, the company said.

The company now intends to file a new drug application for approval of the therapy in the first half of 2026. It plans to engage with the U.S. Food and Drug Administration to discuss data from its studies later this year.

BBIO shares traded 9% higher in the pre-market session at the time of writing. On Stocktwits, retail sentiment around BBIO jumped from ‘neutral’ to ‘bullish’ territory over the past 24 hours, while message volume rose from ‘high’ to ‘extremely high’ levels.

BBIO stock has nearly doubled this year. 

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