QED Therapeutics (QEDtx), a BridgeBio company, is dedicated to developing meaningful treatment options for those with achondroplasia. In August 2018, BridgeBio launched QEDtx to focus on precision medicine for FGFR-driven diseases.1 During Dwarfism Awareness Month (October) in 2019, QED and BridgeBio announced their preclinical data supporting tolerability and activity of low-dose Infigratinib in treating achondroplasia.

what is qed researching?

Genetic variants that cause FGFR3 overactivity can lead to skeletal conditions such as achondroplasia. We are studying whether an investigational agent (infigratinib) has the potential to improve the irregular bone growth in achondroplasia by decreasing the overactivity of the FGFR3, thereby targeting the condition at its source.2

Infigratinib is not currently approved for the treatment of achondroplasia by the U.S. Food and Drug Administration (FDA) or any other health authority.

It is the only oral agent under investigation for achondroplasia and is currently being studied in the PROPEL clinical program (see below for study details).

Learn more about the process required to develop a new treatment option

The PROPEL clinical study program

QEDtx’s clinical trials are currently underway and may change over time. PROPEL is an observational study to collect information about achondroplasia. Participants who complete PROPEL may enroll in investigational studies of oral infigratinib sponsored by QED Therapeutics.2

Eligibility and study duration

  • Children with achondroplasia 2.5 years to <17 years (at screening)
  • Eligible children may participate in PROPEL for a minimum of 6 months to a maximum of 2 years

Goals of the observational study are:

  • To learn more about overall health, growth, and possible medical complications in children with achondroplasia

Eligibility and study duration

  • Children with achondroplasia ages 3 to 11 years who completed at least 6 months in the PROPEL study
  • Eligible children may receive the investigational treatment for up to 18 months in PROPEL 2
  • After completion of the PROPEL 2 study, eligible children may rollover to the PROPEL Open Label Extension

Goals of the study with infigratinib are:

  • To find the most appropriate dose of the investigational treatment to use in children with achondroplasia
  • To learn more about the safety and what side effects may occur with the treatment under investigation
  • To learn how it may affect growth, symptoms, medical complications, and well-being in children with achondroplasia

Eligibility and study duration:

  • Children who complete PROPEL 2 may be given the option to continue to receive the investigational treatment until they complete their growth

Goals of the study with infigratinib are:

  • To further explore the effect of this investigational treatment on overall health, growth, and possible medical complications

qed is planning for a Phase 3 study in children with achondroplasia

This is the next step in the process of developing a new treatment option

Outcomes being studied in the PROPEL clinical program

qed in the news

6 March 2023 – BridgeBio Announces Positive Phase 2 Cohort 5 Results of Infigratinib in Achondroplasia Demonstrating Mean Increase in Annualized Height Velocity of 3.03 cm/year with No Treatment-related Adverse Events

3 March 2023 – BridgeBio to Host Investor Call to Discuss Phase 2 Cohort 5 Data for Achondroplasia on March 6, 2023

13 February 2023 – BridgeBio Pharma to Host Educational Roundtable with Esteemed Achondroplasia Expert on February 16, 2023

26 July 2022 – BridgeBio Pharma Announces Positive Interim Results from a Phase 2 Trial of Infigratinib in Achondroplasia Demonstrating an Increase in Annualized Height Velocity of 1.52 cm/year in Children 5 Years of Age and Older, and Adds 5th Cohort to Trial

15 July 2020 – BridgeBio Pharma’s QED Therapeutics Doses First Child in Phase 2 Clinical Trial of the Investigational Medicine Infigratinib in Achondroplasia

11 May 2020 – BridgeBio Pharma’s QED Therapeutics Announces Preclinical Data Demonstrating Potential of Low-Dose Infigratinib in Achondroplasia

17 October 2019 – QED and Parent Company BridgeBio Announce Preclinical Data Supporting Tolerability and Activity of Low-dose Infigratinib in Treating Achondroplasia

15 August 2018 – QEDtx – A New Company Working On A Treatment For Achondroplasia

  1. Beyond Achondroplasia. QED Tx – a new company working on a treatment for achondroplasia
  2. QED Therapeutics. Our Research. The PROPEL Clinical Study Program | QED Tx