QED Achondroplasia Clinical Update
QED Therapeutics, Inc. (“QED”), a subsidiary of BridgeBio Pharma, Inc. (“BridgeBio”) focuses on bringing medicine to people living with conditions that are caused by changes in fibroblast growth factor receptor (FGFR) genes.
QED is developing a medicine which may improve the overall health and quality of life of people with achondroplasia. This investigational medicine is called infigratinib (formerly called BGJ398) and may be an option for children to take while they are still growing. Infigratinib is a small tablet taken by mouth that works by reducing the activity of proteins called fibroblast growth factor receptors, or FGFRs. Because increased activity in one of the FGFRs – FGFR3 – leads to the changes in bone development associated with achondroplasia, limiting its activity may have benefits for children with achondroplasia.
Other conditions can occur as a result of different changes to the FGFRs, which is why infigratinib is also being studied (at higher doses) as a potential treatment for rare forms of cancer in adults. While the exact way infigratinib works to treat cancer is different from how it works in achondroplasia, these studies may give us an understanding of infigratinib’s safety and side effects in adults with cancer.
Studies using low doses of infigratinib in animals have shown an ability to improve irregular bone growth. Now, QED and its scientific partners are doing more research in animals to better understand how infigratinib may work in children with achondroplasia.
In the meantime, QED has started a research study called PROPEL that seeks to better understand the growth patterns and possible medical complications among children with achondroplasia by observing changes over time with routine medical care. Much will be learned through PROPEL even though no medicine will be administered in the study. For more information and a list of open sites, visit http://www.clinicaltrials.gov using identifier NCT04035811.
In 2020 QED plans to start the first clinical trial that studies the investigational medicine infigratinib in achondroplasia, which will explore the safety and potential effectiveness of infigratinib in children with achondroplasia. Children who have participated in the PROPEL study for at least six months may be eligible to participate in this or other future trials with infigratinib.
QED is committed to the achondroplasia community. Our goal is to one day give families an option that may minimize the impact of irregular bone growth and promote better health and well-being for children with achondroplasia.
Note: The safety and efficacy of infigratinib has not been established. There is no guarantee that infigratinib will receive health authority approval or become commercially available in any country for the uses being investigated.
About QED Therapeutics
QED Therapeutics, a subsidiary of BridgeBio Pharma, is a biotechnology company focused on bringing medicine to people living with FGFR-driven conditions. Infigratinib (BGJ398) is an investigational, oral, FGFR1-3 inhibitor that has shown meaningful clinical activity in patients with cholangiocarcinoma with FGFR2 fusions and advanced urothelial carcinoma with FGFR3 genomic alterations. QED is also evaluating infigratinib in preclinical studies for the treatment of achondroplasia and other skeletal dysplasias.
For more information on QED Therapeutics, please visit the company’s website at
About BridgeBio Pharma
BridgeBio is a team of experienced drug discoverers, developers and innovators working to create life-altering medicines that target well-characterized genetic conditions at their source. Founded in 2015, BridgeBio’s mission is to identify and advance transformative medicines to treat people who suffer from Mendelian conditions, which are conditions 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.
For more information on BridgeBio, please visit the company’s website at
Forward-Looking Statements
This press release contains forward-looking statements. All statements contained herein other than statements of historical fact constitute 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 and clinical and therapeutic potential of infigratinib. These forward-looking statements are subject to a number of risks, uncertainties and assumptions, including, but not limited to, QED Therapeutics’ ability to initiate and continue its planned clinical trials of infigratinib and to advance infigratinib in clinical development. Moreover, QED Therapeutics operates in a very competitive and rapidly changing environment in which new risks emerge from time to time. These forward-looking statements are based upon the current expectations and beliefs of QED Therapeutics’ management as of the date of this release and are subject to certain risks and uncertainties that could cause actual results to differ materially from those described in the forward-looking statements. All forward-looking statements in this press release are based on information available to QED Therapeutics as of the date hereof, and QED Therapeutics disclaims any obligation to update these forward-looking statements except as required by applicable law.