Bridgebio Pharma, Inc. · NASDAQ:BBIO
Bridgebio Pharma — Q4 2025 Earnings Presentation
93 slides from Bridgebio Pharma's earnings presentation, Feb 24, 2026
Slide 1: Corporate Presentation March 2026
Corporate Presentation March 2026
Slide 2: Forward Looking Statements and Disclaimer
Forward Looking Statements and Disclaimer The presentation contains forward-looking statements. Statements made or presented may include statements that are not historica l facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Secti
Slide 3: BridgeBio's objective function
BridgeBio’s objective function Patient impact… …through sustainable value creation 𝑡 𝑁 ∆𝑄𝐴𝐿𝑌 (𝑖) Objective: max න ∗ 𝑝𝑎𝑡𝑖𝑒𝑛𝑡𝑠 (𝑖) Each program must be: 0 𝐷𝑟𝑢𝑔𝑠 𝑖=1 𝑝𝑎𝑡𝑖𝑒𝑛𝑡 • Based on beautiful science with a high probability of technical success (POTS) BridgeBio…
Slide 4: BridgeBio is a new type of biopharmaceutical company
BridgeBio is a new type of biopharmaceutical company From: To: Slow and bureaucratic decision making Rapid and decentralized decision making Expensive platforms with long lead times before Assets selected to target genetic diseases proof-of-concept data at their source High fixed costs Variablized a
Slide 5: The right approach: decentralized R&D, centralized infrastructure
The right approach: decentralized R&D, centralized infrastructure Build “minimum viable companies” Build central infrastructure for to de-risk programs as quickly and functions with economies of scale, efficiently as possible such as commercial Leverage hyper-experienced Leverage seasoned company R&
Slide 6: The right space: capitalizing on a scientific revolution to treat a massive unmet need
The right space: capitalizing on a scientific revolution to treat a massive unmet need for genetic diseases There are 10,000+ counted rare diseases affecting 450 million+ people globally ~3,000 without Genetics known molecular cause ~500 with Macromolecules approved therapies Clinical Diagnosis Ther
Slide 7: We have built a sustainable, high velocity engine to deliver hope and medicines
We have built a sustainable, high velocity engine to deliver hope and medicines to the communities that we serve > 9,500 individuals impacted by our therapies Obtained Approval for 3 Medicines 3 Positive + 1 Ongoing Phase 3 Results Pivotal Trials ✓ ✓ ✓ 15 > 70 > 35 19 < $40M active trials papers aca
Slide 8: We leverage lean operating teams that advance medicines quickly and efficiently
We leverage lean operating teams that advance medicines quickly and efficiently for people living with rare diseases Topline Avg. Team Size BBP-418 IND ~5.5 Years <30 FTEs for LGMD2I Topline IND ~6 Years Encaleret <30 FTEs for ADH1 Topline IND ~6 Years Infigratinib <60 FTEs for Achondroplasia Toplin
Slide 9: We target well-described diseases at their source, underpinning our probability of technical success
We target well-described diseases at their source, underpinning our industry-leading probability of technical success Our approach Probability of Technical Success (POTS) 1. Characterize the genetic source of the Clinical Dev. Industry BBIO disease Stage Benchmark1 Historical2 Phase 1 52% 86% 2. Def
Slide 10: A pipeline of products across the BridgeBio ecosystem
A pipeline of products that sing across the BrigeBio ecosystem Patients Market Program Indication Pre-Clinical Phase 1 Phase 2 Phase 3 Approved (US + EU) Opportunity ✓ Attruby Transthyretin Amyloidosis 500,000+ $20B+ (acoramidis) (ATTR-CM) ✓ Nulibry Molybdenum Cofactor Deficiency <100 Partnered (fos
Slide 11: We are well-financed to hit a drumbeat of potential milestones in 2026 and beyond
We are well-financed to hit a drumbeat of potential milestones in 2026 and beyond 1H 2026 ✓ Infigratinib: ACH Topline BridgeBio ended 2025 with $587.5M in cash, ✓ Encaleret: Initiate P2/3 pediatric ADH1 Encaleret: NDA submission cash equivalents, and marketable securities BBP-418: NDA submission 2H
Slide 12: Attruby Approved for ATTR-CM
Attruby® Approved for ATTR-CM 11
Slide 13: Key Attruby highlights
Key Attruby highlights First and only approved product with a label specifying near-complete stabilization of TTR Effect seen as early as 1 month – reduction in cumulative cardiovascular outcomes within the first month of treatment in patients with ATTR-CM 42% reduction in composite of all-cause mor
Slide 14: $146M Q4 2025 Net Product Revenue
Continued Attruby commercial momentum $146M Q4 2025 Net Product Revenue 13
Slide 15: Continued commercial momentum and patient impact
NEW INFORMATION Continued commercial momentum and patient impact 7,804 1,856 >25% Unique U.S. patient Unique U.S. Estimated share of prescriptions for Attruby prescribing HCPs NBRx for Attruby As of Feb 20th, 2026 14
Slide 16: Our responsibility to patients spans beyond R&D: access and affordability
Our responsibility to patients spans beyond R&D – we are committed to ensuring the best access and affordability of any ATTR-CM medicine Dedicated patient support team Insurance navigation and support Ensuring easy and fast access: The least expensive ATTR treatment: Affo y Access Attruby can be del
Slide 17: We make it easy through simplified, differentiated, and generous access programs
We make it easy through simplified, differentiated, and generous access programs 28-day supply for patients new to Attruby Free Trial (newly diagnosed or switching from another therapy) Distribution Independent and rare/orphan disease–focused Specialty Pharmacies (SPs) Network where Institutional SP
Slide 18: Beyonttra has seen rapid EU uptake with leading guideline endorsements
Beyonttra® (acoramidis) has seen rapid EU uptake with leading guideline endorsements Beyonttra (acoramidis) Demonstrates Robust Commercial Global Guidelines Endorse Beyonttra (acoramidis) Uptake in Germany, Exceeding Projections for Clinical Confidence, Credibility, and Impact Achieved by Germany 8
Slide 19: ATTR is a multi-billion-dollar market primed for continued expansion
ATTR is a multi-billion-dollar market primed for continued expansion Global annual ATTR market sales1 $B $15-$202 $14 Market growth drivers include: $12 • With more sponsors, $10 $9.43 there is expanding $8 +55% disease awareness $6.8 $6 • Increased global $4.3 adoption of non- $4 $3.2 invasive diag
Slide 20: Demand study shows significant Attruby market potential
Demand study shows significant Attruby market potential vs. partial stabilizer and partial knockdown Surveyed HCPs expect 30 - 40%+ peak market share for Attruby HCP sentiment towards Attruby is positive Attruby Market Share as % of Future Competitive Market Acoramidis showed dramatic reduction in (
Slide 21: We continue to research the differentiated clinical role of Attruby
We continue to research the differentiated clinical role of Attruby 11 published manuscripts across 8 journals and 47 abstracts across 9 conferences in 2025 Clinical Outcomes Variant Biomarkers HEOR/Other 2026 Themes Prevention / Variant Arrhythmias Early Tx Effect Biomarkers And more… ACT-EARLY 20
Slide 22: Attruby's advantages stem from being the most potent stabilizer
Attruby’s advantages stem from the fact that it is the most potent stabilizer Acoramidis is the only stabilizer with “near The multiplicity of advantages to superior stabilization continue to be better understood complete” stabilization in the label and elucidated in novel research “Our thermodynami
Slide 23: Attruby significantly reduced the risk of CVM or recurrent CVH through Month 30
Attruby significantly reduced the risk of CVM or recurrent CVH through Month 30 vs. placebo by 49% with curve separation starting by Month 1 Estimated Mean Cumulative Events of CVM or Recurrent CVH Through Month 30 (mITT Population, Acoramidis, n = 409; Placebo, n = 202) Acoramidis Placebo (n = 409)
Slide 24: Published OLE data support Attruby's benefit on ACM and ACM/Recurrent CVH
Published data from the OLE further support Attruby’s statistically significant benefit on ACM and ACM/Recurrent CVH ACM1 ACM/Recurrent CVH2 M30 M42 M30 M42 0% -10% Relative Risk Reduction -20% -30% 25% -40% 34% -50% 42% 48% -60% Attruby resulted in a statistically significant ACM and ACM/Recurrent
Slide 25: Attruby delivers outstanding results in patients with poor prognosis
Attruby delivers outstanding results in patients with poor prognosis Natural History ATTRibute-CM mITT Population V122I ATTRv-CM has an aggressive Statistically significant benefit on composite ACM or first phenotype and poor prognosis1 CVH in ATTRv-CM participants vs. placebo2 Hazard Ratio 60 N (%)
Slide 26: Acoramidis reduced the risk of AF adverse events and AF-related hospitalizations
Acoramidis reduced the risk of AF adverse events and AF-related cardiovascular hospitalizations Incidence AF Adverse Event Annual Frequency of CVH Due to AF 30% RRR, 17% 0.035 RRR, 43% 0.03 Occurrence of atrial arrythmia event, % 25% to AF-related events per year, mean 25.1% 0.03 Annual frequency of
Slide 27: Elevated TTR levels are associated with improved survival
Additional data: Elevated TTR levels are associated with improved survival Higher TTR concentration associated with Each 5 mg/dL rise in serum TTR is associated greater life expectancy (N≈102K individuals)1 with ~32% lower odds of death by 30 months Acoramidis Hazard ratio (95% CI) TTR distribution
Slide 28: Recruitment continues for ACT-EARLY, the first ATTR primary prevention trial
Recruitment continues for ACT-EARLY, the first ATTR primary prevention clinical trial, opening a new potential therapeutic paradigm for patients Current therapeutic paradigm: slow the progression of disease in patients with diagnosed ATTR 130+ ATTR Variants ACT-EARLY is exploring whether acoramidis
Slide 29: Depleter for ATTR-CM
Depleter for ATTR-CM 28
Slide 30: BridgeBio has initiated a depleter program to explore ATTR-CM disease reversal
BridgeBio has initiated a depleter program to explore the potential of ATTR- CM disease reversal ATTR-CM Disease Pathophysiology Led by Renowned Antibody Expert Existing TTR therapies target upstream …but unmet need remains for patients with existing tetramer stabilization and synthesis… amyloid dep
Slide 31: BridgeBio's depleter is engineered across novel properties
BridgeBio’s depleter is engineered across 4 novel properties for potential differentiation on clinical efficacy and dosing convenience Depleter Mechanism of Action BridgeBio’s Differentiated Target Properties 1 Improved fibril:tetramer binding ratio • >10× preferential binding to misfolded TTR fibri
Slide 32: BBP-418
BBP-418 31
Slide 33: LGMD2I/R9 is a progressive neuromuscular disease with high unmet need
LGMD2I/R9 is a progressive neuromuscular disease with high unmet need, representing a >$1B market opportunity in the US and EU Addressable patients by indication Unmet need • LGMD2I/R9 is an inherited neuromuscular disorder 9K characterized by lower-limb weakness and loss of ambulation as well as re
Slide 34: BBP-418 is an oral, disease-modifying therapy targeting LGMD2I/R9
BBP-418 is an oral, disease-modifying therapy that targets LGMD2I/R9 at its source by restoring glycosylation of alpha-dystroglycan Mechanism Design Principles ✓ FKRP glycosylates alpha-dystroglycan (αDG), stabilizing muscle cells by binding extracellular ligands to act as a “shock absorber” for mus
Slide 35: FORTIFY is an ongoing randomized, placebo-controlled Phase 3 study
FORTIFY is an ongoing randomized, placebo-controlled Phase 3 study; we reported topline results from a planned interim analysis in Oct. 2025 BBP-418 (N=74) BBP-418 (N~74) Screening Interim Analysis • Genetically (N=73)2 Randomization Open Label confirmed, 2:1 (N=112) Extension symptomatic Topline LG
Slide 36: Interim analysis topline data reflect highly statistically significant results
Interim analysis topline data reflect highly statistically significant results on all pre- specified endpoints, including biomarker and clinical measures at 12 months BBP-418 in LGMD2I/R9: FORTIFY Phase 3 interim analysis topline results Primary endpoint (3 months) p-value Change from baseline in gl
Slide 37: Consistent improvement across primary and key secondary endpoints
Unprecedented and consistent improvement across primary and all key secondary efficacy endpoints combined with well-tolerated safety profile Ph. 3 FORTIFY interim analysis Type Endpoint Upside case target Outcome observed • Statistically significant increase vs. placebo ✓ Highly statistically signif
Slide 38: Statistically significant increase in glycosylated αDG sustained at 12 months
Interim analysis showed statistically significant increase (+17% of control) from baseline in glycosylated αDG for BBP-418 treated, which was sustained at 12 months Mean glycosylated αDG (% of control)* (± standard error of the mean, SEM) 50 BBP-418 Placebo Mean Glycosylated αDG 40 (% of control)* 3
Slide 39: Rapid reduction in serum CK sustained at 12 months
In addition, patients treated with BBP-418 experienced a rapid reduction in serum CK from baseline at 3 months that was sustained at 12 months Mean CK (U/L) (± SEM)* 3,500 BBP-418 Placebo Mean Serum CK (U/L) 3,000 2,500 2,000 1,500 Among patients treated with BBP-418 for 12 months: 59.6% had CK with
Slide 40: Difference of 0.27 m/s in 100MTT at 12 months
Results translated to clinical endpoints with a difference of 0.27 m/s in 100MTT at 12 months and separation from placebo as early as 3 months LS Mean Change from Baseline in 100MTT (±95% CI) 0.20 LS Mean Change With 95% CI 0.10 100MTT (m/s) 0.00 -0.10 BBP-418 Placebo -0.20 Timepoint 3 months 12 mon
Slide 41: Post hoc analysis of time to complete 100MTT
A post hoc analysis of time to complete 100MTT was significantly different between the treatment and placebo arms, favoring BBP-418 by 31 seconds LS mean time to complete LS mean (99% CI) change from baseline to Endpoint 100MTT at 12 months (s) 12 months (s) BBP-418 85.1 -12.4 (-26.9, 2.0) Placebo 1
Slide 42: BBP-418 patients registered increase in predicted FVC volume
BBP-418 treated patients also registered ~3% increase in predicted volume from baseline FVC, resulting in a difference of ~5% predicted volume versus placebo LS Mean Change from Baseline in FVC (% predicted) (±95% CI) 6.00 LS Mean Change With 95% CI BBP-418 Placebo 4.00 FVC (% Predicted, Sitting Pos
Slide 43: Clinically meaningful 2.6 point benefit on NSAD relative to placebo
In an exploratory analysis, BBP-418 treated patients experienced highly clinically meaningful 2.6 point benefit on NSAD relative to placebo even at early 12-month timepoint Improved gross motor function Even a 1-point difference in NSAD can mean… Change from baseline in NSAD (points) Requiring Able
Slide 44: More than stabilization: meaningful improvements across clinical endpoints
More than stabilization: BBP-418 treated individuals experienced meaningful improvements across clinical endpoints at 12 months Improved ambulatory function Improved pulmonary function Improved gross motor function CFB in 100MTT (m/s) CFB in FVC (% predicted) CFB in NSAD (points) 0.15 3 1.5 0.10 2 1
Slide 45: Planned subgroup analyses show consistent benefit of BBP-418
Planned subgroup analyses show consistent benefit of BBP-418 vs. placebo in all subgroups across α-controlled endpoints at 12 months Biomarker Glycosylated αDG (% of control) Serum creatine kinase (U/L) LS Mean Difference (95% CI) LS Mean Difference (95% CI) L276I homozygous L276I homozygous Other F
Slide 46: Safety data support a tolerable safety profile for BBP-418
Safety data support a tolerable and well-characterized safety profile for BBP- 418 in LGMD2I/R9, reinforcing favorable risk-benefit profile Safety Population Treatment-Emergent Adverse Events* • 112 randomized participants Any TEAE (%) 100% 93.2% ‒ BBP-418: n=74 5.3% ‒ Placebo: n=38 Grade ≥3 TEAEs (
Slide 47: Most common TEAE was diarrhea; no new safety findings
Most common TEAE associated with BBP-418 was diarrhea; no new or unexpected safety findings were observed Most Common TEAEs (>10%)* Laboratory and Cardiac Assessments Diarrhea 52.6% Procedural pain 10.5% 39.2% • No laboratory pattern suggestive of 20.3% Falls 26.3% 18.9% hepatic, renal, cardiac, hem
Slide 48: Successful FDA meeting post interim analysis
We completed a successful meeting with FDA post interim analysis, and they recommend orienting our NDA toward traditional approval Oct. 2025 ✓ Positive topline File New Engage 1H 2026 2026 results from Drug regulatory Phase 3 FORTIFY Application agencies in study interim (NDA) with Europe analysis F
Slide 49: Encaleret
Encaleret 48
Slide 50: No therapies currently indicated to treat ADH1
There are no therapies currently indicated to treat ADH1, a serious and rare genetic condition An analogous ADH1 market is XLH 12K Individuals with ADH1 XLH ADH1 ~12K ADH1 prevalent Prevalence (US) 12K4 12K population in the US based on 4 Acute - hypocalcemia Disease burden Hypophosphatemia populati
Slide 51: Encaleret is an investigational potential first-in-class therapy for ADH1
Encaleret is an investigational, potential first-in-class therapy that targets the underlying disease mechanism of ADH1 Design Principles Only investigational treatment directly targeting ADH1 at its source Potential to restore physiologic mineral homeostasis that is disrupted by CaSR oversensitivit
Slide 52: HCPs report low satisfaction with conventional therapy
Endocrinologists & nephrologists report low satisfaction with conventional therapy, leading to strong interest in novel investigational agents like encaleret High likelihood HCPs would HCPs are not satisfied with conventional therapy prescribe encaleret Dissatisfaction with Currently Available Treat
Slide 53: Encaleret phase 3 registrational study design
Encaleret phase 3 registrational study design Screening and SOC Encaleret Optimization Period Encaleret Titration Period (N=45) Maintenance Period • Age > 16 years of age Participants randomized to encaleret stopped SoC Long-Term • Documented pathogenic, prior to first dose Extension likely pathogen
Slide 54: CALIBRATE participants demonstrated a pharmacologic response
>90% of CALIBRATE participants administered encaleret demonstrated a pharmacologic response Week 4 Week 24 Primary Analysis – Within Group SoC Encaleret p-value3 (N=45) (N=45) Number of Participants Meeting The Primary Endpoint 2 34 (Responder status)1,2 Proportion, % 4% 76% Difference in Proportion
Slide 55: Encaleret was well-tolerated with no TEAE discontinuations
Encaleret was well-tolerated with no TEAEs resulting in encaleret or study discontinuation Period 1 Periods 2 and 3 SoC SoC Encaleret N=67 N=22 N=45 Participants experiencing any Serious TEAE 2 (3%) 3 (14%) 4 (9%) Serious Related TEAE 1 (2%) 0 (0%) 1 (2%) Participants experiencing any TEAE 30 (45%)
Slide 56: Encaleret restored physiologic mineral homeostasis through CaSR
Encaleret was found to restore physiologic mineral homeostasis through its action on the CaSR • 76% of participants randomized to encaleret achieved serum and urine calcium in the target range compared to the same individuals on conventional therapy (difference 71%, p<0.0001) 1,2 • Among encaleret r
Slide 57: CALIBRATE achieved and exceeded all upside target criteria
CALIBRATE achieved & exceeded all criteria set forth as an upside target, with a 76% responder rate following 24 weeks of encaleret treatment Upside Target Clinical Profile Outcome Observed Statistically significant primary analysis result Primary endpoint met (p <0.0001) demonstrating ✓ compared to
Slide 58: Pioneering efforts to enable the successful launch of encaleret
BridgeBio is pioneering efforts to enable the successful launch of encaleret, potentially the first calcilytic molecule to be approved for any condition Expanded availability of Enabled creation of new Enabled update to treatment testing through sponsored ICD-10 code dedicated to guidelines specifyi
Slide 59: Over 1,700 unique patients diagnosed with ADH in the US
Over a 24-month period, >1,700 unique patients were diagnosed with ADH in the US 1,800 Cumulative ADH Patients 1,600 1,400 Diagnosed2 17% 1,200 CMGR 1,000 800 600 400 200 0 10/23 01/24 04/24 07/24 10/24 01/25 04/25 07/25 10/25 ~50% of HCPs diagnosing ADH patients manage ≥5 non-surgical hypoparathyro
Slide 60: Encaleret potential in other etiologies of chronic hypoparathyroidism
Encaleret also has potential as an oral medication for other etiologies of chronic hypoparathyroidism (HP) Encaleret expansion Chronic Hypoparathyroidism opportunity (~200K in US+EU)1,2,3 (RECLAIM-HP trial) Post-surgical Genetic Autoimmune Idiopathic (65%-75%)4,5 (~12.5%)5 (<10%)5 (<10%)5 Encaleret
Slide 61: Normalization of calcium homeostasis in post-surgical hypoparathyroidism
Normalization of calcium homeostasis in post-surgical hypoparathyroidism (PSH) patients is indicative of encaleret’s broader potential Effect on PTH in PSH patients1,2 Effect on calcium homeostasis in PSH patients1,2 (N = 10) (N = 10) Fractional Excretion of Calcium Parathyroid Hormone (pg/mL) Corre
Slide 62: Encaleret as an oral option for chronic hypoparathyroidism
Encaleret has the potential to be an orally administered option for patients with chronic hypoparathyroidism (CHP) Announcing the RECLAIM-HP Encaleret has the potential to normalize Phase 3 Study of Encaleret in CHP blood and urine calcium in CHP patients Initiating in summer 2026 • CHP patients pre
Slide 63: RECLAIM-HP Phase 3 study to initiate in summer 2026
RECLAIM-HP Phase 3 study of encaleret in chronic hypoparathyroidism to initiate in summer 2026 RECLAIM-HP: Global, multi-center, randomized, double-blind, placebo-controlled study Double-blinded treatment period Encaleret + Conventional Therapy1 Encaleret + Conventional Therapy1 Titration Period Mai
Slide 64: CALIBRATE-PEDs Phase 2/3 registrational trial in pediatric ADH1
CALIBRATE-PEDs Phase 2/3 registrational trial has initiated to NEW INFORMATION study encaleret in pediatric ADH1 patients CALIBRATE-PEDS: Global, multi-center, open-label, single-arm study Screening Inpatient N = 7 per cohort PK Sampling & Outpatient Outpatient Long- • Cohort 1: ≥ 12 and < 18 YOA Ou
Slide 65: NDA submission for ADH1 planned in first half of 2026
NDA submission for ADH1 planned in the first half of 2026 with two additional registrational studies to initiate this year ✓ Positive topline ✓ Pre-NDA interaction Submit New Drug results from Oct. 2025 with FDA Application (NDA) 1H 2026 Q1 2026 Phase 3 to FDA CALIBRATE ✓ Initiate Phase 2/3 registra
Slide 66: Infigratinib
Infigratinib 65
Slide 67: Infigratinib for Achondroplasia
“Taken together, these best-in-class results highlight Infigratinib for Achondroplasia the transformative potential for infigratinib to address aspects of achondroplasia beyond linear height, and with a product administered orally.” THANK YOU to the study Ravi Savarirayan, M.D., Ph.D. of Murdoch Chi
Slide 68: Significant unmet need for children with skeletal dysplasias
There remains a significant unmet need for many children with skeletal dysplasias; this represents a large and rapidly growing market Addressable people by indication in US/EU1 (current population eligible for treatment) 55,000 Represents 15-25K individuals with diagnosed and addressable ACH 1.5-2.5
Slide 69: Infigratinib is a potentially best-in-class FGFR3 inhibitor
Infigratinib is a potentially best-in-class FGFR3 inhibitor that targets achondroplasia and hypochondroplasia at their source Mechanism Design Principles FGFR3 FGFR3 Gain-of-function Maximize efficacy by targeting condition variant at the source Infigratinib For all the manifestations of ACH and (FG
Slide 70: Infigratinib is a sprinkle capsule
Infigratinib is a sprinkle capsule that can be taken intact whole, or sprinkled Infigratinib is being studied in children over 3 years of age with achondroplasia (0.25 kg/mg/day) as a sprinkle capsule Capsules Granules (17 mm long*) (2 mm long) Capsules can be swallowed whole or content (granules) s
Slide 71: Infigratinib exceeded the bar for a potential best-in-class option
Infigratinib exceeded the bar for a potential best-in-class therapeutic option, in the broadest age range studied across any achondroplasia trial Target Clinical Profile for Commercial Outcomes Success CFBL in AHV: More than +1.5 cm/yr against Met the primary endpoint with mean difference against pl
Slide 72: PROPEL 3 registrational study design
PROPEL 3 - Phase 3 registrational study design PROPEL: PROPEL 3: Ph3 randomized, double-blinded PROPEL OLE: Observational run-in pivotal trial (n=114 consented) 1 Open-label extension 0.25 mg/kg/day infigratinib (n=74) Observational 0.25 mg/kg/day Children are followed for a 2:1 infigratinib minimum
Slide 73: Demographic and baseline characteristics
Demographic and baseline characteristics Infigratinib Placebo All Participants Baseline demographics (n=74) (n=39) (n=113) Age (yrs), mean (SD) 7.92 (2.7) 7.74 (2.5) 7.86 (2.7) (min, max) (3.7 – 14.4) (3.9 – 14.9) (3.7 – 14.9) Age group, n (%) 3 – <5 10 (13.5) 4 (10.3) 14 (12.4) 5 – <11 50 (67.6) 31
Slide 74: Primary endpoint: Change from baseline to Week 52 in AHV
Primary endpoint: Change from baseline to Week 52 in AHV Infigratinib Placebo (n=74) (n=39) Baseline AHV (cm/yr), Mean (SE) 4.28 (0.16) 4.57 (0.23) Week 52 AHV (cm/yr), Mean (SE) 5.75 (0.15) 3.95 (0.14) CFBL AHV at Week 52, Mean (SE) 1.48 (0.22) -0.62 (0.29) Infigratinib vs. placebo difference, Mean
Slide 75: Subgroup analyses: Change in AHV by age group
Subgroup analyses: Change from baseline to Week 52 in AHV by age group Age subgroups 3 to < 5 years 5 to < 11 years 11 to < 18 years Infigratinib Placebo Infigratinib Placebo Infigratinib Placebo (n=10) (n=4) (n=50) (n=31) (n=14) (n=4) Infigratinib vs. placebo difference, 2.30 1.93 2.78 Mean Infigra
Slide 76: Secondary endpoint: Absolute AHV at Week 52
Secondary endpoint: Absolute AHV at Week 52 Absolute Annualized Height Velocity (AHV) Average reference AHV 6 Annualized height velocity (average stature) (6 cm/year)2 Infigratinib Placebo Absolute AHV at Week 52 (cm/yr, mean) (n=74) (n=39) 5 LS Mean 5.96 4.22 Infigratinib vs. placebo Average refere
Slide 77: Key secondary endpoint: Change in height Z-score
Key secondary endpoint: Change from baseline in height Z-score in relation to an achondroplasia population over 52 weeks Change from baseline (CFBL) in ACH height Z-score 0.4 Infigratinib CFBL in ACH height Z-score Placebo ACH Height Z-score at Infigratinib Placebo 0.3 Week 52 (n=74) (n=39) LS Mean
Slide 78: Key secondary endpoint: Upper-to-lower body segment ratio
Key secondary endpoint: Change from baseline to Week 52 in upper-to-lower body segment ratio Change from baseline in upper to lower body segment ratio (UL:LL) at Week 52 All ages 3 to < 8 years Infigratinib (n=74) Placebo (n=39) Infigratinib (n=43) Placebo (n=23) 0.00 UL:LL (Change from Baseline) LS
Slide 79: Infigratinib at 0.25 mg/kg was well-tolerated
Infigratinib at 0.25 mg/kg was well-tolerated, with no safety signal indicating inhibition of FGFR1 or 2 ▪ The majority of treatment-emergent adverse events (TEAEs) were grade 1 or 2 in severity and typical for children of these ages ▪ TEAEs were balanced between the treatment and placebo arms ▪ No
Slide 80: Mean serum phosphate levels comparable across infigratinib and placebo
Mean serum phosphate levels were comparable across infigratinib and placebo at all measured time points Mean Serum Phosphate Levels Mean serum phosphate levels 7 Infigratinib Placebo 6 Mean serum phosphate levels For all participants, the (mg/dL) maximal change in (mg/dL) 5 phosphate levels was with
Slide 81: NDA and MAA submissions expected in 2H 2026
NDA and MAA submissions expected in 2H 2026 NDA and MAA submissions expected in 2H 2026 Advance infigratinib towards registration in achondroplasia Submit New Drug Application to FDA 2H 2026 Submit Marketing Authorization Application to EMA 2H 2026 Detailed PROPEL 3 results planned for presentation
Slide 82: Hypochondroplasia is a FGFR3-related skeletal dysplasia
Hypochondroplasia is a FGFR3-related skeletal dysplasia • Autosomal dominant condition Infigratinib directly targets FGFR3 signaling • Similar incidence to achondroplasia • Greater genetic heterogeneity in FGFR3 pathogenic variants (e.g. N540K in addition to others) • Clinical features: Moderate dis
Slide 83: Hypochondroplasia is a large, mechanistically de-risked expansion opportunity
Hypochondroplasia is a large, mechanistically de-risked expansion opportunity; First participant dosed in Phase 2 study Rationale for infigratinib Recent progress and upcoming milestones Directly targets the underlying cause of hypochondroplasia, FGFR3 overactivity 1,2 The observational run-in study
Slide 84: We are on Day 1 of the opportunity for infigratinib
We are on Day 1 of the opportunity for infigratinib Achondroplasia (0-3) sNDA-enabling trial, FPI achieved Now enrolling Hypochondroplasia Genotype-driven Accelerating development, (FGFR3 gain-of-function) Now enrolling run-in for Ph. 3 Achondroplasia (Adults) Exploring unique opportunity to potenti
Slide 85: BBP-812
BBP-812 84
Slide 86: Canavan disease is a severe, fatal, ultra-rare pediatric disease
Canavan disease is a severe, fatal, and ultra-rare neurodegenerative pediatric disease with no approved therapies Unmet need • Canavan disease (CD) is an ultra-rare neurodegenerative disease with ~1,000 patients across the US and EU • CD is usually fatal within the first two decades of life, and >25
Slide 87: BBP-812 is a potentially first-in-class, disease-modifying therapy
BBP-812 is a potentially first-in-class, disease-modifying therapy that targets Canavan disease directly at its source Mechanism Design Principles Provide first disease-modifying therapy Target the condition directly at the source, utilize single registrational study & biomarker for accelerated appr
Slide 88: Current path to a potential BLA filing in 2027
Current path to a potential BLA filing in 2027 based on reductions in urine NAA (surrogate endpoint) supported by motor function improvements Urine N-acetylaspartic acid (NAA) levels Hammersmith Infant Neurological Examination (HINE-2) Trajectory Change in HINE-2 Total Score Months since Baseline Na
Slide 89: BBP-812 is changing the lives of children with Canavan
BBP-812 is changing the lives of children with Canavan and the lives of their families Study Investigator Quotes Post-Treatment Patient Progression Patient is showing “lots of improvement at 3-month visit, + gene therapy (22 months old) emotional presence, visual tracking, reaching for things” 12 mo
Slide 90: BridgeBio Ecosystem Highlights
BridgeBio Ecosystem Highlights 89
Slide 91: BBOT has progressed a pipeline of molecules into the clinic
BBOT has progressed a pipeline of molecules into the clinic that are poised to close key activity gaps in the RAS-focused therapeutic space BBO-8520 BBO-11818 BBO-10203 First direct inhibitor Potent, direct inhibitor Novel RAS:PI3Kα Breaker of KRASG12C ON/OFF with of panKRAS ON/OFF specifically bloc
Slide 92: The GondolaBio pipeline features a diverse set of programs
The GondolaBio pipeline features a diverse set of programs across therapeutic areas and modalities Patient Population IND Indication Discovery Lead Op Phase 1 Phase 2 (US+EU) Enabling Erythropoietic Protoporphyria (EPP) 25k Autosomal Dominant Polycystic Kidney Disease 300k (ADPKD) Alpha-1 Antitrypsi
Slide 93: About Attruby and BridgeBio
About Attruby® and BridgeBio About Attruby® (acoramidis) Attruby is the only near-complete (≥90%) stabilizer of Transthyretin (TTR) approved in the U.S. for the treatment of adult patients with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization. Attruby was generally w