BridgeBio Pharma has taken another step toward the potential approval of its muscle weakness drug candidate BBP-418, reporting statistically significant phase 3 efficacy data that analysts have deemed impressive.
The drugmaker revealed that the Fortify study met its primary and secondary interim analysis endpoints in October. BridgeBio then released detailed findings yesterday at the MDA Clinical and Scientific Conference in Florida. The biotech plans to include the data in a filing for full FDA approval, which could lead to a U.S. launch for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9) around the end of the year. The rare genetic disorder causes gradual muscle weakness and wasting, with no existing treatment currently available.
William Blair analysts said in a note to investors that creatine kinase (CK) level response rates were particularly impressive, given the significant elevations observed on placebo. Almost 60% of people treated with BBP-418 had CK levels within two times the upper limit of normal. More than 38% of patients on the study drug had normal CK levels.
Viewed alongside improvements in functional outcomes over baseline and relative to placebo, the data led the analysts to conclude BBP-418 is disease-modifying in LGMD2I/R9. The analysts flagged the safety data, which favored BBP-418 over placebo, as another strength of the readout.
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As BridgeBio revealed previously, the FDA recommended the company seek a traditional full approval, not an accelerated nod. The analysts believe the recommendation shows the strength and consistency of the data favoring BBP-418 over placebo.
The analysts said BridgeBio will need to find people with the rare disease—which the biotech said (PDF) affects 7,000 people in the U.S. and EU—but they cited Fortify enrollment as evidence there is “a bolus of available patients for treatment.” BridgeBio enrolled the trial eight months faster than expected while exceeding the original participant target by 20%, the analysts said.
BridgeBio estimates that LGMD2I/R9 represents an opportunity of more than $1 billion in the U.S. and EU, while analysts forecast worldwide sales of $1.1 billion in 2035. Launching BBP-418 could give BridgeBio a growth driver to supplement Attruby, which powered the company to net product sales of $362 million last year.