QED Therapeutics

QED Therapeutics is a biotechnology company dedicated to developing innovative treatments for rare genetic diseases, including achondroplasia and hypochondroplasia. Their focus is on creating targeted therapies that address the underlying causes of these conditions, offering new hope and improved quality of life for patients and their families. Through rigorous research and a commitment to patient care, QED Therapeutics strives to make a meaningful impact in the lives of those affected by these conditions.

QED’s commitment to the skeletal dysplasia community1:
  • Listening to people with skeletal dysplasias, their families, and healthcare professionals to understand community needs
  • Pursuing research to address community needs
  • Sharing what they learn to help with care decisions and to support living well with skeletal dysplasias
learn more about Qed therapeutics

QED’S RESEARCH

QED is committed to pursuing research to address the needs of the skeletal dysplasia community. Their clinical programs involve multiple studies aimed at deepening the understanding of achondroplasia and hypochondroplasia and evaluating oral treatment options.

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.1

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

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

Image: https://qedtx.com/our-research/

QED’S PROPEL clinical study program

QED’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
  • 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 LAUNCHING a Phase 3 study in children with achondroplasia

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

LEARN MORE ABOUT ENROLLING INTO PROPEL, THE RUN-IN STUDY FOR PHASE 3

Outcomes being studied in the PROPEL clinical program:

PROPEL – Information about achondroplasia is collected without treatment

PROPEL2 – Children with achondroplasia are given different doses of the investigational treatment

PROPEL OPEN LABEL EXTENSION – Children who complete PROPEL 2 may be given the option to continue to receive the investigational treatment until they complete their growth

what is being studied?

PROPELPROPEL2PROPEL OLE
What is the most appropriate dose of the investigational treatment?No treatment is used✔️The dose will be selected in PROPEL 2
Safety and tolerability (side effects)No treatment is used✔️✔️
Growth measurements (height, limb length)✔️✔️✔️
Medical symptoms and complications (middle ear infections, sleep apnea, spinal stenosis, spinal surgery)✔️✔️✔️
Well-being measurements (health-related pain and functional independence questionnaires)✔️✔️✔️
How the body interacts with the treatment under investigation after oral dosing as it moves through the body (pharmacokinetics)No treatment is used✔️Not assessed
Source: https://qedtx.com/our-research/#clinical-studies

PARTICIPATING SITE LOCATIONS

for more information, visit clinicaltrials.gov

in the news

9 SEPTEMBER 2023 – BridgeBio Pharma Announces Positive Feedback from the U.S. FDA and EU EMA on the Regulatory Path for a Pivotal Phase 3 Trial of Infigratinib in Children with Achondroplasia

20 JUNE 2023 – BridgeBio Pharma Presents Updated Six Month Results from its Phase 2 Cohort 5 Trial of Infigratinib in Achondroplasia at the Endocrine Society (ENDO) 2023 Annual Conference

8 JUNE 2023 – BridgeBio Pharma to Present Updated Six Month Results from its Phase 2 Cohort 5 Study of Infigratinib in Achondroplasia in a Late Breaker Oral Presentation at the Endocrine Society (ENDO) 2023 Annual Conference

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. QED Therapeutics. Our Research. The PROPEL Clinical Study Program | QED Tx