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BridgeBio Pharma’s QED Therapeutics Announces Preclinical Data Demonstrating Potential of Low-Dose Infigratinib in Achondroplasia
Data Accepted to ENDO 2020 SAN FRANCISCO, May 11, 2020 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) affiliate QED Therapeutics announced today
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[{"type":"text","content":"Data Accepted to ENDO 2020\nSAN FRANCISCO, May 11, 2020 (GLOBE NEWSWIRE) -- BridgeBio Pharma, Inc. (Nasdaq: BBIO) affiliate QED Therapeutics announced today that in vitro and in vivo data from two studies support QED’s plans to evaluate a low dose of infigratinib as a treatment option for children with achondroplasia, the most common cause of disproportionate short stature. Data were accepted for presentation at the Endocrine Society's Annual Meeting (“ENDO 2020”), which was cancelled due to COVID-19, and the studies were published in the special supplemental section of the Journal of the Endocrine Society.\n The first study, entitled “Support for a new therapeutic approach of using a low-dose FGFR tyrosine kinase inhibitor (infigratinib) for achondroplasia,” showed that in a mouse model of achondroplasia, treatment with infigratinib led to dose-dependent improvement in achondroplasia-associated phenotypes. At a low dose of 0.5 mg/kg, infigratinib was associated with a statistically significant improvement in bone length of 7% to 14% in the upper limbs, 10% to 17% in the lower limbs and 12% in the foramen magnum (the opening at the base of the skull). Also presented in this study was in vitro data demonstrating that, at similar concentrations, infigratinib had greater activity over a CNP analog (vosoritide) in an achondroplasia cell line. The data suggest that inhibition of multiple key pathways downstream of FGFR3 controlling either proliferation or differentiation of the chondrocytes may lead to better efficacy compared with MAPK inhibition alone. The second study, entitled “Low-dose infigratinib treatment does not lead to changes in phosphorus in preclinical animal studies,” showed that rats and mice treated with infigratinib at or below 5 mg/kg showed no relationship between dose and blood levels of phosphorus, a potential safety concern for infigratinib treatment. In addition, two other posters accepted to ENDO 2020, including the design of QED’s PROPEL natural history study in achondroplasia, are available for review on the QED corporate website. Titles for the presentations are: Prospective clinical assessment study in children with achondroplasia: the PROPEL trialFGFR-selective tyrosine kinase inhibitors, such as infigratinib, show potency and selectivity for FGFR3 at pharmacologically relevant doses for the potential tr...