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- BridgeBio Pharma Inc (NASDAQ:BBIO) has announced interim results from PROPEL 2 Phase 2 trial of infigratinib in children with achondroplasia, the most common form of short-limbed dwarfism.
- At the highest dose level (0.128 mg/kg once daily), the mean increase in annualized height velocity (AHV) was 1.52 cm/yr over baseline in children five years and older.
- Earlier cohorts in PROPEL 2 did not achieve the target efficacious exposure as suggested by preclinical data, and no dose-response was observed.
- An increase in AHV over a baseline of 0.22 cm/yr was observed across these earlier combined cohorts.
- Related: BridgeBio Pharma Posts Promising Data From Gene Therapy Trial In Ultra-Rare Disease.
- A single case of mild hyperphosphatemia (abnormally high serum phosphate levels) led to a dose reduction in a participant in Cohort 3 (dose: 0.064 mg/kg once daily).
- No children have discontinued treatment as the result of an adverse event. No bone-related AEs were observed to date.
- BridgeBio has begun enrolling Cohort 5 participants who will receive approximately twice the dose of Cohort 4 (0.25 mg/kg once vs. 0.128 mg/kg once daily).
- Price Action: BBIO shares are up 6.53% at $11.75 during the premarket session on the last check Tuesday.
Crypto Whales Are Loading Up — Are You?
New research shows the biggest crypto buyers are back. And this time? They could hold for the possibility that Bitcoin will surpass $100,000 in 2024. You don’t want to miss the next massive crypto bull run like we saw in 2020 and 2021. To know exactly what’s going on and what to buy… Get Access To Benzinga’s Best Crypto Research and Investments For Only $1.
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