Bridgebio Pharma, Inc. · NASDAQ:BBIO

Bridgebio Pharma — Investor Presentation (October 2025)

71 slides from Bridgebio Pharma's investor presentation, Oct 29, 2025

  1. Slide 1: Corporate Presentation

    Corporate Presentation October 29th, 2025

  2. 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 historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Sectio

  3. Slide 3: Acoramidis approved for ATTR-CM in US, EU, Japan, UK

    …is FDA-approved for ATTR-CM in the US …is approved in the EU, Japan, and UK 2

  4. Slide 4: Two positive Phase 3 trial readouts announced this week

    Two positive Phase 3 trial readouts announced this week Phase 3 trial met all primary and key Positive Phase 3 results announced secondary interim analysis endpoints in Limb-Girdle Muscular Dystrophy 2I/R9 Phase 3 trial met all primary and key Positive Phase 3 results announced secondary endpoints i

  5. Slide 5: 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 𝑝𝑎𝑡𝑖𝑒𝑛𝑡 𝐷𝑟𝑢𝑔𝑠 𝑖=1 • Based on beautiful science with a high probability of technical success (POTS) BridgeBio…

  6. Slide 6: 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 Assets selected to target genetic diseases before proof-of-concept data at their source High fixed costs Variablized a

  7. Slide 7: The right approach: decentralized R&D, centralized infrastructure

    The right approach: decentralized R&D, centralized infrastructure Build “minimum viable companies” to Build central infrastructure for de-risk programs as quickly and functions with economies of scale, efficiently as possible such as commercial Leverage hyper-experienced Leverage seasoned company R&

  8. Slide 8: The right space: capitalizing on a scientific revolution to treat 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 approved Macromolecules therapies Clinical Diagnosis Ther

  9. Slide 9: Our leadership team has world-renowned drug hunters and operators

    Our leadership team has world-renowned drug hunters and operators Neil Kumar, PhD Thomas Trimarchi, PhD Christine Siu Ananth Sridhar Justin To Eric David, MD, JD Founder and President and Chief Executive Officer, Chief Operating Officer, Chief Executive Officer, Chief Executive Officer, Chief Execut

  10. Slide 10: We have built a sustainable, high velocity engine to deliver medicines

    We have built a sustainable, high velocity engine to deliver medicines From Pipette Obtained Approval for 3 Medicines > 2,000 > 30 trials 3 fully + 2 Positive l in e To P patients in > 30 countries enrolled ip e Phase 3 Results trials > 500 sites pivotal trials a ti e P nt To > 70 > 35 19 < $10M and

  11. Slide 11: We leverage lean operating teams that advance medicines quickly

    We leverage lean operating teams that advance medicines quickly and efficiently for patients Topline BBP-418 IND ~5.5 Years for LGMD2I Topline IND ~6 Years Encaleret for ADH1 Topline IND ~6 Years Acoramidis for ATTR-CM Data on file. 10

  12. Slide 12: We target well-described diseases at their source

    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 Clinical Dev. Industry BBIO source of the disease Stage Benchmark1 Historical2 Phase 1 52% 86% 2. Def

  13. Slide 13: A pipeline of products across the BridgeBio ecosystem

    A pipeline of products that sing across the BridgeBio ecosystem Pre- Patients Market Program Indication Phase 1 Phase 2 Phase 3 Approved Clinical (US + EU) Opportunity ✓ Attruby Transthyretin Amyloidosis 500,000+ $20B+ (acoramidis) (ATTR-CM) ✓ Nulibry Molybdenum Cofactor Deficiency <100 Partnered (f

  14. Slide 14: We are well-financed and expect to hit numerous milestones in 2025

    We are well-financed and expect to hit numerous milestones in 2025 1H 2025 2H 2025 ✓ Beyonttra: EU, Japan, UK approvals ✓ Q3 Earnings Call ✓ Q1 Earnings Call ✓ Encaleret: LPLV, Topline ✓ Q2 Earnings Call ✓ BBP-418: LPLV, Topline ✓ financing via HCRx and Blue Owl Secured $300M royalty Infigratinib: L

  15. Slide 15: Attruby Approved for ATTR-CM

    Attruby® Approved for ATTR-CM

  16. Slide 16: 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

  17. Slide 17: Our responsibility to patients spans beyond R&D

    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 Affo y Access Ensuring easy and fast access: The least expensive ATTR treatment: rdability Attruby

  18. Slide 18: 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

  19. Slide 19: Performance to date indicates strong, continued commercial momentum

    Performance to date indicates strong, continued commercial momentum 5,259 1,355 $108.1M Unique U.S. patient Unique U.S. Q3 Attruby net prescriptions for Attruby prescribing HCPs product sales As of October 25, 2025 Q3 2025 18

  20. Slide 20: 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 6

  21. Slide 21: Our Attruby team has experienced industry leaders

    Our Attruby team has experienced industry leaders who have built and launched blockbuster drugs Matt Outten Julie Everett John Whang Chief Commercial Officer Chief Business Officer Chief Medical Affairs Officer • Broad commercial leadership expertise with success • Successfully led cross-functional

  22. Slide 22: 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 there is expanding $8.53 disease awareness $8 +52% $6.8 $4.1 • Increased global $6 adoption of non- $4.3 $4 $3.2 invasive

  23. Slide 23: 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 (

  24. Slide 24: Committed to advancing Attruby's scientific narrative

    BridgeBio is committed to advancing Attruby’s scientific narrative to ensure as many appropriate patients as possible can benefit 2024 2025+ ATTR-CM ATTR-CM WT and hereditary WT and hereditary Extended Ph. 3 data disclosures at Attruby lifecycle management and further ACC, ESC-HF, ESC, HFSA, and AHA

  25. Slide 25: Acoramidis significantly reduced cumulative cardiovascular outcomes within first month

    Acoramidis significantly reduced cumulative cardiovascular outcomes within the first month of treatment Acoramidis Placebo (n = 409)​ (n = 202)​ Participants with CVM or 136 (33.3)​ 98 (48.5)​ recurrent CVH, n (%)​ Hazard ratio 0.51 (95% CI)​a (0.43, 0.62)​ p value < 0.0001 Source: Masri et al. (202

  26. Slide 26: 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 M36 M30 M36 0% -10% Relative Risk Reduction -20% -30% 25% -40% 36% -50% 42% 46% -60% Attruby resulted in a statistically significant ACM and ACM/Recurrent

  27. Slide 27: 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 phenotype and poor prognosis1 first CVH in ATTRv-CM participants vs. placebo2 Hazard Ratio 60 N (%)

  28. Slide 28: Acoramidis reduced the risk of AF adverse events

    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% Occurrence of atrial arrythmia event, % 0.03 to AF-related events per year, mean 25% 25.1% 0.03 Annual frequency of

  29. Slide 29: 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

  30. Slide 30: Recruitment continues for ACT-EARLY 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 ~600 Participants ACT-EARLY is exploring w

  31. Slide 31: Encaleret

    ~ oO t= a © (S] c Lu

  32. Slide 32: Thank you to patients, families and investigators

    encaleret for Autosomal Dominant Hypocalcemia Type 1 (ADH1) A sincere THANK YOU to the patients, families, advocates, investigators, clinical research staff, referring providers, and collaborating research partners 31

  33. Slide 33: 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) 12K5 12K population in the US based on Acute - hypocalcemia Disease burden Hypophosphatemia 4 populati

  34. Slide 34: Encaleret targets the underlying disease mechanism of 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

  35. Slide 35: Phase 3 study (CALIBRATE) of encaleret in ADH1

    Phase 3 study (CALIBRATE) of encaleret in ADH1 Encaleret Screening and SOC Encaleret Titration Period (N=45) Maintenance Optimization Period Period • Age > 16 years of age Participants randomized to encaleret stopped SoC prior to • Documented pathogenic first dose Long-Term Extension SoC Randomizati

  36. Slide 36: 76% of participants on encaleret met the primary endpoint

    76% of participants randomized to encaleret met the primary endpoint, achieving serum calcium and 24-hour urine calcium in the target ranges Week 4 Week 24 Primary Analysis – Within Group SoC Encaleret p-value4 (N=45) (N=45) Number of Participants Meeting The Primary Endpoint 2 34 (Responder status)

  37. Slide 37: Encaleret was well-tolerated with no TEAEs resulting in discontinuation

    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%)

  38. Slide 38: 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 re

  39. Slide 39: NDA submission planned in first half of 2026

    NDA submission planned in the first half of 2026 with two additional registrational studies planned for initiation next year Generate clinical evidence for encaleret in Advance encaleret towards registration in ADH1 additional patient populations Submit New Drug Application to FDA Initiate Phase 2/3

  40. Slide 40: Encaleret potential in chronic hypoparathyroidism

    Encaleret also has potential as an oral medication for other etiologies of chronic hypoparathyroidism (HP) Encaleret potential Chronic Hypoparathyroidism expansion (~200K in US+EU)1,2,3 opportunity Post-surgical Genetic Autoimmune Idiopathic (65%-75%)4,5 (~12.5%)5 (<10%)5 (<10%)5 Encaleret current A

  41. Slide 41: Preliminary data supports potential expansion to chronic HP

    Preliminary data supports potential expansion to chronic HP, showing rapid, PTH-independent, effect on blood and urine calcium Proof-of-principle phase 2 study of encaleret in post-surgical hypoparathyroidism (PSH), N=10 0.04 0.03 100% FECa 0.02 ULN % of Participants with blood & 24- * * hr urine Ca

  42. Slide 42: Potential path for improved calcium control in a convenient pill

    Proof-of-concept study findings are suggestive of a potential path for improved calcium control in a convenient pill form Potential Differentiating Features For Encaleret Orally administered tablet Benefit in 24-hour urinary calcium excretion Avoidance of long-term PTH-mediated impact on bone health

  43. Slide 43: BBP-418

    ie) ei + BBP-

  44. Slide 44: Thank you to patients, families and investigators

    BBP-418 for Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9) A sincere THANK YOU to patients, families, advocates, investigators, clinical research staff, and collaborating research partners 43

  45. Slide 45: LGMD2I/R9 is a progressive disease representing a >$1B market

    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

  46. Slide 46: BBP-418 targets LGMD2I/R9 by restoring glycosylation of alpha-dystroglycan

    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

  47. Slide 47: FORTIFY Phase 3 study design and interim analysis

    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=72) Randomization Open Label confirmed, 2:1 (N=112) Extension symptomatic Topline LGM

  48. Slide 48: Interim topline results highly statistically significant on all endpoints

    Interim analysis topline data reflect highly statistically significant results on all pre-specified endpoints, including biomarker and clinical measures at 12 mo. BBP-418 in LGMD2I/R9: FORTIFY Phase 3 interim analysis topline results Primary endpoint (3 months) p-value Change from baseline in glycos

  49. Slide 49: 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

  50. Slide 50: 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 mo. Change from baseline in glycosylated αDG (+/- 99% CI) Glycosylated αDG (% of control) 40 BBP-418 Placebo p<0.0001 LS Mean Estimate CFB in

  51. Slide 51: Reduction in serum CK of 82% at 12 months

    In addition, BBP-418 treated patients experienced a large, statistically significant reduction in serum CK of 82% from baseline at 12 months Reduction in muscle damage Change from baseline in serum creatine kinase (U/L) 400 186 0 -400 -800 -1,869 -1,200 -1,600 -2,000 Placebo BBP-418 (n=22) p<0.0001

  52. Slide 52: Improved ambulatory function on 100MTT

    Results translated to clinical endpoints with a difference of 0.27 m/s between BBP-418 and placebo in 100MTT, translating to a difference of ~14 seconds faster Improved ambulatory function Change from baseline in 100-meter timed test (m/s) 0.15 0.10 0.14 0.05 0.00 -0.05 -0.12 -0.10 -0.15 Placebo BBP

  53. Slide 53: Improved pulmonary function on FVC

    BBP-418 treated individuals also registered ~3% increase in predicted volume from baseline FVC, resulting in a difference of ~5% predicted volume vs. placebo Improved pulmonary function Change from baseline in forced vital capacity, sitting position (% predicted) 3 2 3.0% 1 ~2% decline is consistent

  54. Slide 54: Significant improvements across clinical endpoints at 12 months

    More than stabilization: BBP-418 treated individuals experienced significant, clinically meaningful improvements across clinical endpoints at 12 months Key: Placebo BBP-418 Improved ambulatory function Improved pulmonary function CFB in 100MTT (m/s) CFB in FVC (% predicted) 0.15 3 0.10 2 0.14 3.0% 0

  55. Slide 55: Favorable safety profile of BBP-418

    Interim analysis results continue to reflect highly favorable safety profile of BBP-418 • No new or unexpected safety findings have been observed; results consistent with Ph. 2 • Discontinuation rate was low overall and higher in the placebo group • No treatment-related serious TEAEs were observed I

  56. Slide 56: Next steps

    Next steps ✓ Topline results from Phase 3 FORTIFY study interim analysis October 2025 Engage FDA prior to NDA submission Late 2025 / Early 2026 Present Phase 3 FORTIFY study interim analysis results MDA Clinical & Scientific Conference March 2026 File New Drug Application (NDA) with FDA 1H 2026 U.S.

  57. Slide 57: Infigratinib

    Infigratinib

  58. Slide 58: Significant unmet need in skeletal dysplasias; $4B+ market

    There remains a significant unmet need for many children with skeletal dysplasias; this represents a large ($4B+) and rapidly growing market Addressable people by indication in US/EU1 Annualized achondroplasia product sales2 (current population eligible for treatment) ($M WW, annualized by quarter)

  59. Slide 59: 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 variant condition at the source Infigratinib For all the manifestations of ACH and HCH

  60. Slide 60: At 18 months, infigratinib shows persistent improvement in AHV

    At 18 months, infigratinib has shown persistent improvement in AHV and body segmentation, along with a favorable safety profile Mean change from baseline in annualized height velocity Upper body to lower body segment ratio (AHV) (Lower value means improved proportionality) +3.38 cm/yr 2.05 +2.51 cm/

  61. Slide 61: Phase 3 PROPEL 3 study of infigratinib in Achondroplasia

    We have fully enrolled a Phase 3 study (PROPEL 3) of infigratinib in Achondroplasia and expect topline results in early 2026 0.25 mg/kg/day Infigratinib (N ~ 76) Open Label Screening and Extension Observational Period Randomization Children are Children are followed 2:1 followed until for a minimum

  62. Slide 62: 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 d

  63. Slide 63: 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 Observational run-in study for

  64. Slide 64: BBP-812

    BBP-812

  65. Slide 65: 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 is an ultra-rare neurodegenerative disease with ~1,000 patients across the US and EU • Canavan is usually fatal within the first two decades of life, and >25% of pat

  66. Slide 66: BBP-812 is a potentially first-in-class AAV9 gene 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

  67. Slide 67: Sustained reductions in NAA and improved motor function with BBP-812

    Significant, sustained reductions in NAA levels and improvement in motor function after 12-months of BBP-812 dosing in Phase 1/2 study Urine N-acetylaspartic acid (NAA) levels Hammersmith Infant Neurological Examination (HINE-2) Trajectory Change in HINE-2 Total Score Months since Baseline Natural B

  68. Slide 68: BridgeBio Ecosystem Highlights

    BridgeBio Ecosystem Highlights

  69. Slide 69: BBOT pipeline of molecules in the RAS-focused space

    BBOT has progressed a pipeline of molecules into the clinic that are poised to close key activity gaps in the RAS-focused therapeutic space Program Mechanism of Action Status • First direct inhibitor of KRASG12C ON BBO-8520 Phase 1 • Inhibits both KRASG12C GTP (active) and GDP (inactive) states KRAS

  70. Slide 70: The GondolaBio pipeline

    The GondolaBio pipeline features a diverse set of programs across therapeutic areas and modalities Lead IND Est. Patient Pop. Indication Discovery Phase 1 Phase 2 Optimization Enabling (US + EU) Erythropoietic Protoporphyria (EPP) 25k Alpha-1 Antitrypsin Deficiency (AATD) 200k Hereditary Pancreatiti

  71. Slide 71: 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 well-tolerated. The most commo

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