BridgeBio's growth disorder drug tops expectations in Phase II update

2024-06-04

临床结果临床3期临床2期上市批准

BridgeBio Pharma's investigational therapy infigratinib surpassed expectations in the latest readout of the Phase II PROPEL 2 study, demonstrating sustained and statistically significant increases in annualised height velocity (AHV) in children with achondroplasia.

In Cohort 5 of the study, which evaluated the highest dose of 0.25 mg/kg per day, the oral FGFR3 inhibitor resulted in a mean change from baseline of +2.51 cm/year in AHV at 12 months, and a change of +2.50 cm/year at the 18-month mark.

'Best-in-class data'

Analysts were anticipating only a 2-cm improvement, according to Salim Syed of Mizuho Securities. While growth did slow between the sixth and twelfth months, he said that "the question was regarding its potential magnitude… Today's reported best-in-class data should settle any debate on this."

Syed pointed out that body proportions also improved. At month 18, there was a statistically significant improvement in the ratio between the upper and lower body segments, with a mean ratio of 1.88 compared to 2.02 at baseline.

By contrast, there were no significant improvements in upper/lower body segment ratio in children receiving BioMarin Pharmaceutical's once-daily injection Voxzogo (vosoritide) during its pivotal 52-week study, "so we'd argue that the body proportionality data may represent a point of differentiation from Voxzogo in investor debates," the analyst said.

BioMarin's modified C-type natriuretic peptide was first approved in 2021 based on data showing it improved annualised growth velocity by 1.57 cm/year versus placebo. The drug's label was expanded last October to include children under the age of 5.

Clean safety profile

Meanwhile, infigratinib's safety profile in PROPEL 2 is encouraging as well, with no treatment-related adverse events tied to the drug in any Cohort 5 participant. There were no cases of hyperphosphatemia or hypergrowth either.

BridgeBio has also launched a Phase III registrational study, PROPEL 3 that is currently recruiting and is expected to be fully enrolled by year-end. Mizhuo has said it estimates peak sales of $1.2 billion for infigratinib in achondroplasia.

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