BridgeBio's growth disorder drug tops expectations in Phase II update
04 Jun 2024
Clinical ResultPhase 3Phase 2Drug Approval
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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