BridgeBio Nears 52-Week High On Strong Dwarfism Trial Data, Plans FDA Filing In 2026
BridgeBio Pharma Inc. (NASDAQ:BBIO) stock is surging on Thursday and nearing the upper end of its 52-week range of $28.33-$84.94, according to Benzinga Pro data.
The biopharmaceutical company focused on genetic conditions, shared positive topline results from PROPEL 3 Phase 3 pivotal study of oral infigratinib in children with achondroplasia, the most common form of skeletal dysplasia leading to disproportionate short-limb dwarfism.
PROPEL 3 evaluated infigratinib in children with achondroplasia aged 3 to 18 years with open growth plates.
Key Results
Change from baseline in annualized height velocity (AHV) superior to placebo, with an LS mean treatment difference of +1.74 cm/year and a mean treatment difference of +2.10 cm/year
The secondary endpoint of absolute AHV at week 52 showed a significant improvement with infigratinib compared to placebo, with the infigratinib arm achieving the highest LS mean absolute AHV reported to date in a randomized trial in achondroplasia (5.96 cm/year versus 4.22 cm/year on placebo).
In a pre-specified exploratory analysis (children younger than 8; more than 50% of the participants) of the key secondary endpoint of change from baseline in upper-to-lower body proportionality at week 52, oral infigratinib is the first therapeutic option to show statistical significance against placebo in a randomized trial for achondroplasia, demonstrating an LS mean decrease of -0.05 against placebo.
In the overall population, infigratinib achieved an LS mean decrease of -0.05, the largest reduction observed in a treatment arm in a randomized achondroplasia trial, with a favorable LS treatment difference of -0.02 versus placebo at week 52 (p=0.1849)
Infigratinib was well-tolerated, with no discontinuations related to the study drug, and no serious adverse events related to the study drug were observed.
Regulatory Plans
BridgeBio intends to meet with regulatory authorities to discuss plans for submission of a New Drug Application (NDA) and Marketing Authorization Application (MAA) for infigratinib in the second half of 2026 to support approval.
Other Rare Indications
The company also intends to accelerate the development of infigratinib for hypochondroplasia, and is enrolling participants in the observational run-in for the phase 3 trial.
The company also has an ongoing clinical trial of infigratinib for newborns to three-year-old age groups in achondroplasia in the PROPEL Infant and Toddler trial.
Competitive Landscape
The data comes just ahead of the upcoming PDUFA target action date is Feb. 28, for the rival Ascendis Pharma A/S‘ (NASDAQ:ASND) New Drug Application (NDA) for TransCon CNP (navepegritide) for children with achondroplasia.
BioMarin Pharmaceutical Inc.'s (NASDAQ: BMRN) Voxzogo (vosoritide) was the first FDA-approved drug (November 2021) for achondroplasia.
BBIO Price Action: BridgeBio shares are up 6.92% at $78.41 at the time of publication on Thursday. The stock hit a high of $84.94 and dipped to a low of $77.45 as of publication on Thursday. The stock is sitting approximately 5.5% below its 52-week high, according to Benzinga Pro data.
Photo: Shutterstock
Disclaimer: The content of this article solely reflects the author's opinion and does not represent the platform in any capacity. This article is not intended to serve as a reference for making investment decisions.
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