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Expert Interview

Slingshot members are talking to an expert! The topic is:

Examining BridgeBio's Infigratinib PROPEL 2 Trial Results in Achondroplasia and Beyond

Ticker(s): BBIO

Who's the expert?

Institution: University Hospital Cologne

  • Head of the Center for Rare Skeletal Disorders in Children and Adolescents at the University of Cologne & member of the Osteogenesis Imperfecta Federation Europe (OIFE) Medical Advisory Board
  • Treats 52 patients with Achondroplasia older than 5 years of age and is currently prescribing Voxzogo to 26 patients.
  • Specializes in the research of brittle bone disease and directs the special ambulance for rare pediatric skeletal diseases and pediatric rheumatology.

Interview Questions
Q1.

The PROPEL 2 trial showed a sustained and statistically significant increase in annualized height velocity (AHV) of +2.51 cm/year at 12 months and +2.50 cm/year at 18 months in children with achondroplasia. Can you elaborate on the clinical significance of these results?

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Q2.

At month 18, infigratinib treatment led to a statistically significant improvement in body proportionality. How might these changes impact the overall health and daily life of children with achondroplasia?

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Q3.

Infigratinib was reported to be well-tolerated with no treatment-related adverse events in Cohort 5 of the PROPEL 2 trial. How important is this safety profile in the context of long-term treatment for children with achondroplasia?

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Q4.

How does infigratinib compare to other treatments currently available or in development for achondroplasia, particularly in terms of efficacy and safety?

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Q5.

BridgeBio has initiated the ACCEL study for hypochondroplasia following positive interactions with the FDA and EMA. What are the key challenges and opportunities in extending infigratinib treatment to this related condition?

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Q6.

With PROPEL 3, the global Phase 3 registrational study of infigratinib in achondroplasia, underway, what outcomes are you most eager to see, and how might they influence the potential approval of infigratinib?

Added By: catalin_admin
Q7.

Besides height velocity and body proportionality, what other quality-of-life measures are being considered in these trials, and why are they important for patients and families dealing with achondroplasia?

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Q8.

Given the promising results in achondroplasia, what other FGFR-related skeletal dysplasias or genetic conditions might benefit from infigratinib, and what are BridgeBio’s plans for exploring these possibilities?

Added By: catalin_admin

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