BridgeBio Pharma Shares One Year Data From Dwarfism Candidate

Author: Vandana Singh | June 04, 2024 03:29pm

Tuesday, BridgeBio Pharma Inc (NASDAQ:BBIO) announced sustained results from the PROPEL 2 Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, a disorder of bone growth that causes the most common type of dwarfism.

The data demonstrated continued efficacy and an encouraging safety profile.

Infigratinib is an oral small molecule designed to inhibit FGFR3 signaling and target achondroplasia and hypochondroplasia at their source.

To date, key results from the Cohort 5 dose-escalation cohort in PROPEL 2 trial include:

Sustained and statistically significant mean increase in annualized height velocity (AHV) of +2.51cm/year from baseline at 12 months and +2.50 cm/yr at 18 months.
Statistically significant improvement in body proportionality, from 2.02 at baseline to 1.88 at Month 18.
A continued well-tolerated safety profile, with no treatment-related adverse events assessed as related to infigratinib

PROPEL 3 Phase 3 registrational study of infigratinib in achondroplasia continues to enroll on schedule, with completion of enrollment expected by the end of the year.

BridgeBio announced that the first child consented in ACCEL 2/3 observational run-in study for 0.25mg/kg/day of infigratinib in children with hypochondroplasia.

Unlike achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short stature, people with hypochondroplasia typically present with less severe short stature and milder disproportionality.

Price Action: BBIO shares are down 6.94% at $26.97 at last check Tuesday.

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Posted In: BBIO