BridgeBio Pharma, Inc. · NASDAQ:BBIO
BridgeBio Pharma — Investor Presentation (August 2025)
55 slides from BridgeBio Pharma's investor presentation, Aug 5, 2025
Slide 1: Corporate Presentation
Corporate Presentation August 5th, 2025
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
Slide 3: Attruby is FDA-approved for ATTR-CM and approved in EU, Japan, UK
…is FDA-approved for ATTR-CM in the US …is approved in the EU, Japan, and UK 2
Slide 4: Three Phase 3 trials are now fully enrolled
Three Phase 3 trials are now fully enrolled Trial enrolled 112 patients to evaluate BBP-418 in Limb-Girdle Muscular Dystrophy 2I/R9 Trial enrolled 71 patients to evaluate encaleret in Autosomal Dominant Hypocalcemia Type 1 Trial enrolled 114 participants to evaluate infigratinib in Achondroplasia 3
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…
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
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&
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 Genetics ~3,000 without known molecular cause Macromolecules ~500 with approved therapies Clinical Diagnosis Ther
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
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 l in e To P patients in > 30 countries enrolled ip e trials > 500 sites pivotal trials a ti e P nt To > 70 > 35 19 < $10M and papers academic INDs created
Slide 11: A pipeline of products that sing 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
Slide 12: 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: LPLV De
Slide 13: Attruby Approved for ATTR-CM
Attruby® Approved for ATTR-CM
Slide 14: 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 3 months – the most rapid benefit seen in any Phase 3 study of ATTR-CM to date 42% reduction in composite of all-cause mortality and recurrent cardiovascular-rela
Slide 15: Committed to best access and affordability of any ATTR-CM medicine
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 The least expensive ATTR treatment: y Access Ensuring easy and fast access: rdability Medicine
Slide 16: 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 17: Performance to date indicates strong, continued commercial momentum
Performance to date indicates strong, continued commercial momentum 3,751 1,074 $71.5M Unique U.S. patient Unique U.S. Q2 Attruby net prescriptions for Attruby prescribing HCPs product sales As of August 1, 2025 Q2 2025 16
Slide 18: Positive feedback all around on market access
Positive feedback all around on market access Pricing: ForgingBridges: “ You priced responsibly and have a good label; we will have you at parity with tafamidis. Also, I have never seen a company offer free drug to their trial patients. Shows commitment.” - Payer the health system's mission of putti
Slide 19: Commercial infrastructure deployed across the US
Our commercial infrastructure is deployed across the US to ensure full provider coverage ATTR-CM Prescriber Market BridgeBio Coverage ✓ Dedicated commercial & US-field-based team with singular focus on serving patients with ATTR-CM ✓ Salesforce is built to adequately cover every HCP and institution
Slide 20: 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
Slide 21: 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 $14.0 Market growth drivers include: $12.0 • With more sponsors, $10.0 there is expanding $8.43 disease awareness $8.0 +52% $6.8 • Increased global $6.0 $4.2 adoption of non- $4.3 $4.0 $
Slide 22: 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 23: 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
Slide 24: OLE data support Attruby's benefit on ACM and recurrent CVH
Recently 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/R
Slide 25: Attruby Delivers Outstanding Results in Patients with Poor Prognosis
Looking Ahead: 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 & V122I participants vs. placebo
Slide 26: Acoramidis reduced risk of new-onset atrial fibrillation and CVH
Acoramidis reduced the risk of new-onset atrial fibrillation (AF), the risk of CVH irrespective of baseline AF, and the risk of CVH due to AF Incidence of CVH due to AF/AFL events* Incidence of new-onset AF/AFL events Occurrence of CVH Frequency of CVH RRR, 33% RRR, 43% RRR, 17% to AF-related events
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: First patient dosed in ACT-EARLY primary prevention trial
First patient dosed in 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 whet
Slide 29: Infigratinib
Infigratinib
Slide 30: 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)
Slide 31: 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 disease at the source Infigratinib For all the manifestations of ACH and HCH,
Slide 32: 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/
Slide 33: Fully enrolled Phase 3 PROPEL 3 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
Slide 34: 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
Slide 35: Hypochondroplasia expansion opportunity; first participant dosed in Phase 2
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 overactivity1,2 Observational run-in study for
Slide 36: Encaleret
~ oO t= a © (S] c Lu
Slide 37: ADH1 is a serious and rare genetic condition with no indicated therapies
ADH1 is a serious and rare genetic condition for which there are no indicated therapies and diagnosis rates are low An analogous ADH1 market is XLH 12K ADH1 Carriers XLH ADH1 ~12K carriers of ADH1 Prevalence (US) 12K5 12K causing variants in the US Acute hypocalcemia risk, based on 4 population Dise
Slide 38: Encaleret is a potentially first-in-class, disease-modifying therapy
Encaleret is a potentially first-in-class, disease-modifying investigational therapy that targets the underlying disease mechanism of ADH1 Mechanism Design Principles Normal CaSR senses and Encaleret is a CaSR antagonist regulates serum Ca levels to designed to decrease the sensitivity maintain home
Slide 39: Phase 2B results showed rapid and sustained normalization of Ca
Phase 2B results demonstrated rapid and sustained normalization of serum Ca, and urine Ca without need for dose escalation Mean responses through 42 months of treatment (N=13) Participants with blood & urine Ca in the target range 11 10 100% ** ** Blood cCa 9 (mg/dL) 80% 69% % of Participants 8 7 6
Slide 40: Fully enrolled Phase 3 CALIBRATE study of encaleret in ADH1
We have fully enrolled the Phase 3 study (CALIBRATE) of encaleret in ADH1 and expect topline results in Fall 2025 Screening and SoC Encaleret Optimization Period Encaleret Titration Period (N ~ 47) Maintenance Period • Age > 16 years of age SoC Long-Term Extension • Documented pathogenic Maintenance
Slide 41: Encaleret potential in broader chronic hypoparathyroidism population
Encaleret has the potential to address unmet need and provide an oral medicine for a broader chronic hypoparathyroidism population 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 (<1
Slide 42: Preliminary data support encaleret as oral option for hypoparathyroidism
Preliminary data support the potential for encaleret as an effective oral option for chronic hypoparathyroidism (HP) Ongoing proof-of-principle study of encaleret in post- Individual Exposure surgical hypoparathyroidism (PSH) N=7, Preliminary Results1 n=3 Individual Exposure n=3 n=4 n=4 0.05 • 25 Hi
Slide 43: BBP-418
ie) ei + BBP-
Slide 44: 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 45: BBP-418 is a potentially first-in-class, disease-modifying therapy
BBP-418 is a potentially first-in-class, disease-modifying therapy with potential to be the first approved therapy for LGMD2I/R9 Mechanism Design Principles ✓ FKRP glycosylates alpha-dystroglycan (αDG), stabilizing muscle cells by binding extracellular ligands to act as a “shock absorber” for muscle
Slide 46: Phase 2 study of BBP-418 shows increased glycosylated αDG
Significant increases in glycosylated αDG in muscle and stabilization of ambulatory measures were observed in a Phase 2 study of BBP-418 Increase in glycosylated αDG in muscle observed post dosing Change from baseline in 10-meter walk test with BBP-418 (median ± 95% CI) (m/s) L276I/L276I homozygous
Slide 47: Fully enrolled Phase 3 FORTIFY study of BBP-418
We have fully enrolled a Phase 3 study (FORTIFY) of BBP-418 in LGMD2I/R9 and expect topline interim analysis data readout in 2H 2025 BBP-418 (N ~ 75) BBP-418 (N ~ 75) Screening Interim Analysis • Genetically Randomization (N ~ 70) Open Label confirmed, 2:1 (N ~ 112) Extension symptomatic Topline Dat
Slide 48: BBP-812
BBP-812
Slide 49: 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
Slide 50: BBP-812 is a potentially first-in-class gene therapy for Canavan disease
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 51: Reductions in NAA levels 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
Slide 52: BridgeBio Ecosystem Highlights
BridgeBio Ecosystem Highlights
Slide 53: BBOT pipeline of RAS-focused molecules in 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 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
Slide 54: The GondolaBio pipeline across therapeutic areas and modalities
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) 20k Galactosemia 10k Alpha-1 Antitrypsin Deficiency (AATD) 200k Neuro
Slide 55: 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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