Bridgebio Pharma, Inc. · NASDAQ:BBIO
Bridgebio Pharma — Investor Day (January 2026)
36 slides from Bridgebio Pharma's investor day presentation, Jan 9, 2026
Slide 1: Infigratinib Clinical Program in Achondroplasia
Infigratinib Clinical Program in Achondroplasia Investor Webinar Jan 9th, 2026
Slide 2: Forward-Looking Statements and Disclaimer
Forward-Looking Statements and Disclaimer Statements made or presented in this Presentation may include statements that are not historical fact and are considered forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities
Slide 3: Agenda
Agenda Janet Legare, MD Overview of 1 Achondroplasia University of Wisconsin School of Medicine and Public Health Daniela Rogoff, MD, PhD Infigratinib Clinical 2 Development Program Chief Medical Officer Skeletal Dysplasias, BridgeBio Justin To The Opportunity for a 3 Daily Oral in ACH CEO Skeletal
Slide 4: Overview of Achondroplasia
Overview of Achondroplasia Janet Legare, MD Director of Midwest Regional Bone Dysplasia Clinic Professor of Pediatrics University of Wisconsin School of Medicine and Public Health 4
Slide 5: Achondroplasia has health consequences beyond stature
Achondroplasia has health consequences beyond stature Most common form of disproportionate short stature Achondroplasia presents in all ethnicities equally – more than 90% of disproportionate short stature Incidence 1: 25,000-30,000 About 300,000 affected persons worldwide; 15,000 in North Ame
Slide 6: When does diagnosis usually occur?
When does diagnosis usually occur? Detectable during the third trimester – usually after 26 weeks – only diagnosed prenatally if mother is having growth ultrasounds during this time —Most cases are diagnosed by the 1st month of life About 33% diagnosed prenatally, 33-40% in first 48 hours, anoth
Slide 7: There are two types of bone growth or ossification
There are two types of bone growth or ossification: endochondral- and intramembranous- ossification Intramembranous bones There are two types of ossification: endochondral and intramembranous1,2 Endochondral bones Growth plates 1. National Cancer Institute. SEER Training Modules. Bone Development
Slide 8: Achondroplasia affects bones that grow through cartilage growth plates
Achondroplasia affects bones that grow through cartilage growth plates, specifically endochondral bone Endochondral bones Endochondral ossification occurs in all bones except for top of skull, lower jaw, and collar bones, and contributes to length of bones1,2 1. National Cancer Institute. SEER Tra
Slide 9: Typical vs. Irregular Growth at Growth Plate
Typical vs. Irregular Growth at Growth Plate Typical Growth Plate Achondroplasia Growth Plate Epiphyseal Epiphyseal growth plate growth plate Resting zone Resting zone Proliferation Proliferation zone zone Chondrocyte hypertrophy accounts for ~60% of longitudinal bone growth Hypertrophic Hypertrop
Slide 10: FGFR3 gain-of-function variants disrupt endochondral ossification
In achondroplasia, FGFR3 gain-of-function variants disrupt endochondral ossification and limit bone growth 1 Figure from Su, N., Jin, M. & Chen, L. Role of FGF/FGFR signaling in skeletal development and homeostasis: learning from mouse models. Bone Res 2, 14003 (2014). https://doi.org/10.1038/bonere
Slide 11: FGFR3 gain-of-function variants disrupt endochondral ossification and limit bone growth
In achondroplasia, FGFR3 gain-of-function variants disrupt endochondral ossification and limit bone growth FGFR3 tyrosine kinase inhibitors directly target overactive FGFR3 signaling, potentially addressing the mechanistic driver of skeletal dysplasia2-4 1Figure from Su, N., Jin, M. & Chen, L. Role
Slide 12: Clinical Picture
Clinical Picture Examples of Features Clinical Presentation Short stature: average height of 4’4” in males and 4’1” in females Rhizomesomelia Short fingers/trident configuration Macrocephaly with frontal and biparietal bossing Midface hypoplasia Small chest Tibial varus Frontal bossing
Slide 13: Children with achondroplasia have a different growth trajectory
Children living with achondroplasia have a very different growth trajectory from those of the general population Height-for-age growth chart Height z-scores (or height SDS) is the difference of an individual’s height from the average height expected for their age and sex, expressed in standard dev
Slide 14: Proportionality links changes in linear growth to potential benefits beyond height
Proportionality links changes in linear growth to potential benefits beyond height Upper to Lower (U:L) body segment ratio is a measurable marker of disproportion in ACH2 U:L body segment ratio never reaches 1 in achondroplasia because trunk growth continues while lower extremity growth is limited
Slide 15: Achondroplasia is associated with multisystem medical challenges across the lifespan
Achondroplasia is associated with multisystem medical challenges across the lifespan Musculoskeletal/Orthopedic1–5 Neurologic1–3 Hypotonia Foramen magnum stenosis (FMS) Ligament laxity Hydrocephalus Thoracolumbar kyphosis (TLK) Lumbar lordosis Respiratory and Sleep1–3 Obstructive sleep apnea (OSA) L
Slide 16: Goals of Therapeutic Intervention
Goals of Therapeutic Intervention Improve health Decrease medical sequelae FDA requires a measurement – height/annual growth velocity Emerging data have associated increased height with greater mobility, function, and QoL1,2 Height is the metric, but improved health is the goal QoL, quality
Slide 17: Infigratinib Clinical Development Program
Infigratinib Clinical Development Program Daniela Rogoff, MD, PhD Chief Medical Officer, Skeletal Dysplasias 17
Slide 18: Infigratinib is an oral, potentially first-in-class FGFR1-3 inhibitor
Infigratinib is an oral, potentially first-in-class FGFR1-3 inhibitor in development as a therapeutic option for achondroplasia Mechanism of the condition Infigratinib Mechanism of Action FGFR3 Gain-of-function variant FGFR3 Infigratinib (FGFR3 TKI) STAT1 Pathway MAPK Pathway STAT1 Pathway MAPK Path
Slide 19: Infigratinib sprinkle capsules are being developed for oral administration
Infigratinib sprinkle capsules are being developed for oral administration1,2 • Infigratinib is being studied in children over 3 Capsules Granules years of age with achondroplasia (0.25 (17 mm long*) (2 mm long) kg/mg/day) as a sprinkle capsule • Capsules can be swallowed whole or content (granules)
Slide 20: The infigratinib clinical program encompasses 5 clinical studies
The infigratinib clinical program for achondroplasia encompasses 5 clinical studies, evaluating safety and efficacy in ~300 children ages 0 to <18 years* Observational Run-in Phase 2 Open-Label Dose-Escalation Open-label Extension Participants: Children (3–11 years) who Duration: Dose Escalation: 18
Slide 21: PROPEL 2: Infigratinib was well-tolerated, with no safety signals identified
PROPEL 2: Infigratinib was well-tolerated, with no safety signals identified In Cohort 5 (highest dose escalation level of 0.25 mg/kg/day): • No serious adverse events (SAEs) • 0 subjects with grade 3 TEAEs • No adverse events (AEs) that required • 0 retinal adverse events treatment discontinuation
Slide 22: PROPEL 2 Cohort 5: Change from baseline in AHV over time
PROPEL 2 – Cohort 5: Change from baseline in AHV over time demonstrated durability of treatment effect Mean change from baseline in annualized height velocity (AHV) 3.38cm/yr 2.51cm/yr 2.50cm/yr 3.51 cm/yr Baseline Month 6 Month 12 Month 18 (n=12) (n=12) (n=11) (n=11) p=0.0015 At each timepoint, cha
Slide 23: PROPEL 2 Cohort 5: Cumulative increase in AHV and height z score
PROPEL 2 – Cohort 5: Cumulative increase in AHV corresponds to an increase in the height z score referenced to ACH-specific growth charts Mean height Z-score 0.76 1.06 1.15 1.32 Mean change from baseline in ACH height Z- +0.29SDs +0.36SDs +0.54SDs score Baseline Month 6 Month 12 Month 18 (n=12) (n=1
Slide 24: PROPEL 2 Cohort 5: Persistent decrease in upper/lower body segment ratio
PROPEL 2 – Cohort 5: Infigratinib showed a persistent decrease in upper/lower body segment ratio Upper to lower body segment ratio 2.05 2.02 2.00 p=0.001 1.95 1.95 Mean change from 1.95 Baseline 1.90 1.88 1.85 1.80 Baseline Month 6 Month 12 Month 18 (n=12) (n=12) (n=11) (n=11) Infigratinib showed st
Slide 25: PROPEL 3 study design and key endpoints
PROPEL 3 study design and key endpoints1 PROPEL 3: Ph3 randomized, double-blinded pivotal PROPEL OLE: PROPEL: Observational run-in trial (n=~110) Open-label extension 0.25 mg/kg/day Infigratinib* Observational 0.25 mg/kg/day Children are followed for a 2:1 Infigratinib* minimum of 6 months Placebo F
Slide 26: The Opportunity for a Daily Oral in Achondroplasia
The Opportunity for a Daily Oral in Achondroplasia Justin To CEO, Skeletal Dysplasias
Slide 27: Defining characteristics of a potentially best-in-class program in the ACH landscape
Infigratinib: Defining characteristics of a potentially best-in-class program in the ACH landscape Designed to target achondroplasia at its genetic source: FGFR3 overactivation Addresses not just overactivation of the MAPK pathway (chondrocyte hypertrophy), but also STAT1 (chondrocyte proliferation)
Slide 28: BBIO has a validated evidence-based perspective to forecasting market share
BBIO has developed a validated evidence-based perspective to forecasting market share performance Comprehensive market surveys Analog research shows 2nd entrants ML-based empirical analysis enables indicated 30-40%+ Attruby share achieve average ~37% share high fidelity drug launch modeling Attruby
Slide 29: Infigratinib could capture >50% of treated market share
Using these techniques, our market research indicates that infigratinib could capture >50% of treated market share, primarily driven by the favorable oral administration and MOA Attribute TPP for testing market share Potential share for ACH children % of treated children • Children (3 – 18 years) wi
Slide 30: Insights on market share: leading prescription drivers
A) Insights on market share: The leading prescription drivers for clinicians are having a child-friendly oral option and a mechanism targeting FGFR3 Relevance of Reasons to Switch TO Infigratinib1 (% of HCPs; n=100 HCPs) Relevance of Reasons Reasons (% of HCPs rating on a 9-point scale; n=100 HCPs)
Slide 31: Insights on market size: $5B market opportunity globally
B) Insights on market size: There is a $5B market opportunity globally, with the vast majority of individuals not on a treatment option Addressable achondroplasia population1-4 Estimated number of ACH children currently treated1-4 ~55K addressable globally Represents 55,000 diagnosed and addressable
Slide 32: Insights on market growth: injection-related burden is the leading barrier
C) Insights on market growth: Injection-related burden is frequently cited as the leading barrier against adoption of approved options today Barriers to treatment uptake despite approved option Top reasons for families not being treated today2 among U.S. clinicians experienced with therapy1 For thos
Slide 33: Connecting the dots between genetics, nonclinical data, and clinical data
Infigratinib: Connecting the dots between genetics, nonclinical data, and clinical data to do more and to do better for families living with achondroplasia 1994 2016 2024 Topline Q1 2026 (Last participant last visit completed) • Safety: No injection site reactions or symptomatic hypotension. Less th
Slide 34: PROPEL 3 topline in Q1 2026 and expansion opportunities
Infigratinib: PROPEL 3 topline in Q1 2026 and expansion opportunities PROPEL 3 topline results expected in Q1 2026 • Last participant last visit achieved • Ph. 3 topline results to be shared in future webinar, with full results presented at a medical congress Advancing toward regulatory milestones a
Slide 35: Q&A
Q&A
Slide 36: Thank you
bridael . briagebio Thank you