QED and Parent Company BridgeBio Announce Preclinical Data Supporting Tolerability and Activity of Low-dose Infigratinib in Treating Achondroplasia
Achondroplasia is the most common cause of dwarfism and is caused by mutation of the gene for fibroblast growth factor receptor 3 (FGFR3). FGFR3 has been shown to be crucial for the process of bone elongation.1,2 Infigratinib, an investigational agent, binds to FGFR3 and has been previously shown to increase bone growth in preclinical mouse models of achondroplasia.3 The data presented today showed a positive dose-response relationship between infigratinib and bone growth.
"Observing a dose-response relationship is an important step in developing infigratinib as a therapy for achondroplasia," said
The preclinical study used a dwarf mouse model (Fgfr3Y367C/+), which mimics achondroplasia. In each of the three dose groups, treatment with infigratinib led to an increase in all nine measures of bone size studied compared to the control group (See figure). At the highest dose examined (0.5 mg/kg/day), treatment with infigratinib demonstrated a statistically significant (P < 0.01) improvement in bone length of 7-14% in the upper limbs, 10% to 17% in the lower limbs and 12% in the foramen magnum (an opening at the base of the skull). No apparent toxicity of infigratinib was noted in this study.
"We are encouraged by this preclinical data supporting the ability of a low dose, or even intermittently dosed, infigratinib to increase the length of the limbs as well as other bones in the skeleton. In particular, we believe the increase in foramen magnum size has significant implications, given that narrowing of this area of the skull can, in some cases, cause cervical spinal cord compression in achondroplastic children," said
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QED Therapeutics, a subsidiary of
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BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic diseases at their source. BridgeBio was founded in 2015 to identify and advance transformative medicines to treat patients who suffer from Mendelian diseases, which are diseases that arise from defects in a single gene, and cancers with clear genetic drivers. BridgeBio's pipeline of over 15 development programs includes product candidates ranging from early discovery to late-stage development.
As the first European center of research, care and education on genetic diseases, the
Since its creation,
Some 8,000 identified genetic diseases affect 30 million patients in
BridgeBio Pharma Forward Looking Statements
This press release contains forward-looking statements. Statements we make in this press release may include statements which are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended (the Securities Act), and Section 21E of the Securities Exchange Act of 1934, as amended (the Exchange Act), which are usually identified by the use of words such as "anticipates," "believes," "estimates," "expects," "intends," "may," "plans," "projects," "seeks," "should," "will," and variations of such words or similar expressions. We intend these forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act and Section 21E of the Securities Exchange Act and are making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements, including statements relating to expectations, plans, and prospects regarding QED Therapeutics' clinical development plans, clinical trial results, timing and completion of clinical trials, competitive environment, and the clinical and therapeutic potential of infigratinib for the treatment of achondroplasia, reflect our current views about our plans, intentions, expectations, strategies and prospects, which are based on the information currently available to us and on assumptions we have made. Although we believe that our plans, intentions, expectations, strategies and prospects as reflected in or suggested by those forward-looking statements are reasonable, we can give no assurance that the plans, intentions, expectations or strategies will be attained or achieved. Furthermore, actual results may differ materially from those described in the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control including, without limitation, QED Therapeutics' ability to initiate and continue its planned clinical trials of infigratinib, its ability to advance infigratinib in clinical development, including QED Therapeutics' plans to file an IND in 2020, and the timing and success of any such continued clinical development, as well as those set forth in the Risk Factors section of
1Rousseau, F., Bonaventure, J., Legeai-Mallet, L., Pelet, A., Rozet, J.M., Maroteaux, P.,
2Shiang R, Thompson LM, Zhu YZ, Church DM, Fielder TJ, Bocian M, Winokur ST, Wasmuth JJ. "Mutations in the transmembrane domain of FGFR3 cause the most common genetic form of dwarfism, achondroplasia." Cell 78 (1994): 335–342.
3Komla-Ebri, D., Dambroise, E., Kramer, I., Benoist-Lasselin,C., Kaci, N.,
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SOURCE QED Therapeutics
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