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BridgeBio Announces Positive Phase 2 Cohort 5 Results of Infigratinib in Achondroplasia Demonstrating Mean Increase in Annualized Height Velocity of 3.03 cm/year with No Treatment-related Adverse Events
- In the highest dose level (Cohort 5, 0.25 mg/kg once daily), the mean change from baseline in annualized height velocity (AHV) at six months was +3.03 cm/yr
About this update from Bridgebio Pharma, Inc.
[{"type":"text","content":"- In the highest dose level (Cohort 5, 0.25 mg/kg once daily), the mean change from baseline in annualized height velocity (AHV) at six months was +3.03 cm/yr (p = 0.0022) for the first 10 children with at least six months of follow-up in Cohort 5. The two remaining children who have not yet had six months of follow-up have a mean change from baseline in AHV of +8.8 cm/yr based on three months data - 80% of children at six months were responders, as defined by an increase from baseline AHV of at least 25%. The mean change from baseline in AHV of responders was 3.81 cm/yr - As a result of treatment, the median absolute AHV reached 7.6 cm/yr, which is beyond the 99th percentile of growth for children living with achondroplasia - Infigratinib demonstrated clear dose-responsiveness as a single daily oral therapy and was well-tolerated with no adverse events (AEs) assessed as treatment-related in Cohort 5 - Based on the positive Phase 2 results, BridgeBio has started to enroll children for a pivotal Phase 3 trial - BridgeBio expects to initiate clinical development of infigratinib for hypochondroplasia, a skeletal dysplasia closely related to achondroplasia and driven by fibroblast growth factor receptor 3 (FGFR3) gain-of-function variants, and will continue to explore the impact on the medical and functional complications of achondroplasia in future studies of infigratinib PALO ALTO, Calif., March 06, 2023 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) (BridgeBio), a commercial-stage biopharmaceutical company focused on genetic diseases and cancers, today announced positive results from PROPEL2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, demonstrating potential best-in-class efficacy and a clean safety profile. Infigratinib is an oral small molecule designed to inhibit FGFR3 and target achondroplasia at its source. BridgeBio will also host an investor call on March 6, 2023, at 7:30 am ET to discuss the results from the Phase 2 study. To date, key results from the clinical trial include: At the highest dose level evaluated to date (Cohort 5, 0.25 mg/kg once daily), the mean increase from baseline in annualized height velocity (AHV) for the 10 children that have had six-month visits was +3.03 cm/yr (p = 0.0022). Individual data can be found in Figure 1 belowThe baseline AHV ...