BridgeBio Pharma, Inc. · NASDAQ:BBIO
BridgeBio Pharma — Technical Presentation (September 2024)
20 slides from BridgeBio Pharma's technical presentation, September 2024
Slide 1: Infigratinib Clinical Development Program Update
Infigratinib Clinical Development Program Update Ravi Savarirayan Murdoch Children’s Research Institute, Royal Children’s Hospital Victoria University of Melbourne Parkville, Victoria, Australia September 2024 ©2024 BridgeBio Inc. Do not duplicate or distribute without written permission. MAT-EU—006
Slide 2: Disclosures
Disclosures • Honoraria from QED, BioMarin, Ascendis and Tyra. ©2024 BridgeBio Inc. Do not duplicate or distribute without written permission. 2
Slide 3: Forward-looking statements
Forward-looking statements This presentation contains forward-looking statements. Statements made or presented may include statements that 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), an
Slide 4: Presentation overview
Presentation overview QED Clinical Development Program Update Infigratinib & the PROPEL Program for ACH PROPEL 2 Cohort 5: Data Update PROPEL 3: Update Hypochondroplasia: ACCEL Program Closing and Q&A ACH: Achondroplasia ©2024 BridgeBio Inc. Do not duplicate or distribute without written permission.
Slide 5: Achondroplasia: The PROPEL Program
Achondroplasia The PROPEL Program 5 ©2024 BridgeBio Inc. Do not duplicate or distribute without written permission. 5
Slide 6: Infigratinib is an oral FGFR3 inhibitor in development for achondroplasia & hypochondroplasia
Infigratinib* is an oral FGFR3 inhibitor in development as a treatment option for achondroplasia & hypochondroplasia Mechanism of action Infigratinib FGFR3 NPR2 • Oral FGFR1-3 tyrosine G380R variant (ACH) kinase inhibitor N540K variant (HCH) • Inhibits all pathways responsible for the clinical TK RA
Slide 7: The PROPEL Program in Achondroplasia
The PROPEL Program in Achondroplasia Observational Run-in (N ≈ 250) Participants: Children and adolescents (2.5 to <17 years) with achondroplasia Open-label Extension 1° endpoint: AHV (N ≈ 280) Duration: ≤2 years (≥6 months required for PROPEL interventional studies) Participants: Children and adole
Slide 8: PROPEL 2: Trial Overview
PROPEL 2: Trial Overview ©2024 BridgeBio Inc. Do not duplicate or distribute without written permission. 8 Infigratinib is an investigational agent that is not approved for use by any regulatory authority.
Slide 9: PROPEL 2: Safety Summary
PROPEL 2: Safety Summary Cohort 5 (highest dose escalation level of 0.25 mg/kg/day): • No serious adverse events (SAEs) • No adverse events (AEs) that required treatment discontinuation • Most treatment-emergent adverse events (TEAEs) were grade 1 in severity and none of the TEAEs were assessed as r
Slide 10: PROPEL 2: Safety Profile
PROPEL 2: Safety Profile Common AEs across all cohorts AEs occurring in ≥10% of study participants Total (%) N = 72 Nasopharyngitis 29 (40.3%) COVID-19 24 (33.3%) Headache 24 (33.3%) Vomiting 22 (30.6%) Pain in extremity 20 (27.8%) Ear infection 19 (26.4%) Pyrexia 18 (25.0%) Abdominal pain 11 (15.3%
Slide 11: PROPEL 2: Cohort 5 Baseline Characteristics
PROPEL 2: Cohort 5 Baseline Characteristics Female : Male ratio 7:5 Mean age at screening (years) 7.24 <5 8% 5 – <8 58% 8 – <11 25% ≥11 8% Baseline AHV (cm/year) 3.51 (1.3) Mean (SD) Data on file ©2024 BridgeBio Inc. Do not duplicate or distribute without written permission. 11 Infigratinib is an in
Slide 12: PROPEL 2: Cohort 5 Efficacy Results
PROPEL 2: Cohort 5 Efficacy Results Mean change from baseline in annualized height velocity (AHV) +3.38 cm/yr +2.51 cm/yr +2.50 cm/yr 3.51 cm/yr Baseline Month 6 Month 12 Month 18 (n=12) (n=12) (n=11) (n=11) p=0.0015 Change from baseline in AHV over time demonstrates durability of treatment effect D
Slide 13: PROPEL 2: Cohort 5 Efficacy Results
PROPEL 2: Cohort 5 Efficacy Results Absolute AHV individual values (cm/yr) 91% of participants had an increase in AHV from Baseline to Month 18 73% of participants had an increase of greater than 25% in AHV from Baseline to Month 18 Data on file ©2024 BridgeBio Inc. Do not duplicate or distribute wi
Slide 14: PROPEL 2: Cohort 5 Efficacy
PROPEL 2: Cohort 5 Efficacy Upper to lower body segment ratio Infigratinib* showed continued improvement in upper to lower body segment ratio Data on file. NOTE: 1 child dropped out before month 12 assessments as study site closed due to PI departure. This child was a ©2024 BridgeBio Inc. Do not dup
Slide 15: PROPEL 3: Last patient in expected by end of 2024
PROPEL 3: Last patient in expected by end of 2024 PROPEL3: Ph3 randomized, double-blinded pivotal PROPEL OLE: PROPEL: Observational run-in trial (n=~110) Open-label extension 0.25 mg/kg/day Infigratinib* Observational Children are followed for a 2:1 0.25 mg/kg/day minimum of 6 months Infigratinib* P
Slide 16: Hypochondroplasia: The ACCEL Program
Hypochondroplasia The ACCEL Program 16 ©2024 BridgeBio Inc. Do not duplicate or distribute without written permission. 16
Slide 17: Hypochondroplasia is an FGFR3-related skeletal dysplasia with a need for treatment options
Hypochondroplasia is an FGFR3-related skeletal dysplasia with a need for treatment options FGFR3 NPR2 • Disproportionate short stature genetic condition due to N540K variant heterogeneous FGFR3 pathogenic variants (primarily N540K)1 TK RAS RAF • Similar incidence to achondroplasia1 MEK cGKII STAT1 •
Slide 18: The ACCEL Program in Hypochondroplasia
The ACCEL Program in Hypochondroplasia Observational Phase 2/3 Open-Label Phase followed by a Double-Blinded, Open-Label Run-in Randomized, Placebo-Controlled Study Extension (planned) Phase 2: Open-Label Phase Children and adolescents Children (5 – 11 yr with growth potential) completed ≥6 Eligible
Slide 19: Summary
Summary In the PROPEL 2 study, the selected dose of 0.25mg/kg/day of oral infigratinib* was considered safe and well-tolerated Infigratinib* demonstrated a durable increase from baseline in AHV for up to 18 months with a statistically significant improvement in upper to lower body segment ratio PROP
Slide 20: Thank you
To the children, families, advocates, and physicians who have been a part of this program: Thank you Developing new treatment options relies entirely on your guidance, dedication, and effort. ©2024 BridgeBio Inc. Do not duplicate or distribute without written permission. 20
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