BridgeBio Pharma Inc shares have soared more than 55% after the company announced positive data from a Phase 2 clinical trial for its investigational therapy infigratinib in children with achondroplasia, a form of short-limbed dwarfism.
Infigratinib is an oral small molecule designed to inhibit FGFR3 and target achondroplasia at its source, BridgeBio noted in a statement.
The company said at six months, 80% of children responded to the therapy.
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At the highest dose level, the mean change from baseline in annualized height velocity (AHV), meaning how much a child is expected to grow in a year, at six months was plus 3.03 centimetres per year for the first 10 children with at least six months of follow-up, the company said.
The two remaining children who have not yet had six months of follow-up have a mean change from baseline in AHV of plus 8.8 centimetres per year based on three months of data, it added.
As a result of treatment, the median absolute AHV reached 7.6 centimetres per year, which is beyond the 99th percentile of growth for children living with achondroplasia, BridgeBio noted.
“We are thrilled to see these promising results and consider that AHV increases of this magnitude will translate to improvements in the medical and functional complications of achondroplasia,” commented the trial’s lead investigator Dr Ravi Savarirayan.
Based on the results of the Phase 2 trial, the company said it has begun enrollment for a Phase 3 trial.
BridgeBio shares were up 55.5% at US$16.90 in the early afternoon on Monday.
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