BridgeBio Pharma, Inc. · NASDAQ:BBIO
BridgeBio Pharma — Technical Presentation (May 2024)
22 slides from BridgeBio Pharma's technical presentation, May 29, 2024
Slide 1: Featured research in the ATTR-CM landscape
Featured research in the ATTR-CM landscape May 29, 2024
Slide 2: Forward-Looking Statements and Disclaimer
Forward-Looking Statements and Disclaimer Statements in this Presentation that are not statements of historical fact are forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Such fo
Slide 3: A sincere thank you to patients, families, and research partners
A sincere THANK YOU to patients and families, advocates, physicians, clinical research staff, and collaborating research partners 3
Slide 4: Acoramidis was designed to achieve maximal stabilization and preserve native TTR
Acoramidis was designed to achieve maximal stabilization and preserve native TTR Design Objectives Rationale 1 Maximize TTR • Strong genotype/phenotype correlation between TTR instability and stabilization/minimize toxic disease severity1 monomer • Dose-dependent improvements in both TTR stabilizati
Slide 5: Connecting the dots: near-complete TTR stabilization leads to improved clinical outcomes
Connecting the dots: near-complete TTR stabilization leads to improved clinical outcomes Evidence of Disease Clinical and Genetics Stabilization (Activation and Functional Treatment Response) Outcomes ACM1,2 Serum TTR CVM3 Protective T119M CVH4 mutation NT-proBNP 6MWT KCCQ 1. Maurer et al., ISA 2024
Slide 6: Patients on acoramidis are surviving more and going to the hospital less
Patients on acoramidis are surviving more and going to the hospital less Near-complete TTR stabilization leads to improved clinical outcomes A B C Unprecedented Evidence of Early separation absolute improvement for in composite survival and large proportion outcomes hospitalization of patients rates
Slide 7: Patients on acoramidis are surviving more and going to the hospital less
Patients on acoramidis are surviving more and going to the hospital less Near-complete TTR stabilization leads to improved clinical outcomes A B C Unprecedented Evidence of Early separation absolute improvement for in composite survival and large proportion outcomes hospitalization of patients rates
Slide 8: Patients on acoramidis are surviving more and going to the hospital less
Patients on acoramidis are surviving more and going to the hospital less Near-complete TTR stabilization leads to improved clinical outcomes A B C Unprecedented Evidence of Early separation absolute improvement for in composite survival and large proportion outcomes hospitalization of patients rates
Slide 9: Acoramidis continues to expand upon its clinically differentiated safety and efficacy profile
Acoramidis continues to expand upon its clinically differentiated safety and efficacy profile 1 Category Title First Author Congress Early Increase in Serum Transthyretin Level is an Independent Predictor of Improved Survival in ATTR Cardiomyopathy: Insights Maurer, M. ISA ‘24 from Acoramidis Phase
Slide 10: New updates at ISA: Focus of today's call
New updates at ISA: Focus of today’s call ▪ Greater stabilization leads to better clinical outcomes – For every 5 mg/dL increase in serum TTR level, the risk of death was reduced by 30.9% by the logistic model and by 26.1% by the Cox proportional hazards model1 ▪ CVH is predictive of overall surviva
Slide 11: Early Increase in Serum Transthyretin Level is an Independent Predictor of Improved Survival
Early Increase in Serum Transthyretin Level is an Independent Predictor of Improved Survival in ATTR-CM: Insights From Acoramidis Phase 3 Study ATTRibute-CM Mathew S. Maurer, Nitasha Sarswat, Martha Grogan, Amrut Ambardekar, Anique Ducharme, Steen Hvitfeldt Poulsen, Satish Rao, Jean-François Tamby,
Slide 12: Early Increase in Serum TTR Predicts Improved Survival: ATTRibute-CM Insights
Early Increase in Serum Transthyretin Level is an Independent Predictor of Improved Survival in ATTR-CM: Insights From Acoramidis Phase 3 Study ATTRibute-CM Mathew S. Maurer, Nitasha Sarswat, Martha Grogan, Amrut Ambardekar, Anique Ducharme, Steen Hvitfeldt Poulsen, Satish Rao, Jean-François Tamby,
Slide 13: Rapid and sustained increase in serum TTR over 30 months
Patients on acoramidis observed a rapid and sustained increase in serum TTR over 30 months FIGURE 1: Change From Baseline in Serum TTR Levels—mITT Population Acoramidis treatment increased serum TTR levels at Day 28, which remained stable through Month 30 Source: Maurer et al., ISA 2024 “Early Incre
Slide 14: Correlation of increasing serum TTR levels and decreasing risk of death
Stat-sig correlation of increasing serum TTR levels and decreasing risk of death was observed in ATTR-CM patients on acoramidis FIGURE 2: Probability of All-Cause Mortality as a Function of Change from Baseline Serum TTR Level at Day 28 in Acoramidis Treated Cohort Probability of All-Cause Mortality
Slide 15: Real-World Outcomes of Tafamidis in Transthyretin Cardiomyopathy
Real-World Outcomes of Tafamidis in Transthyretin Cardiomyopathy Ahmad Masri, Priyanka Bhattacharya, Brent Medoff, Ain U. Ejaz, Pranav Chandrashekar, Lauren Ives, Alfonsina Mirabal Santos, Sergio L. Teruya, Yuanzi Zhao, Shuaiqi Huang, Xiaofeng Wang, Brett W. Sperry, Mathew S. Maurer, Prem Soman, Maz
Slide 16: Methods
Methods • Patients with ATTR-CM who received tafamidis between 1/1/2018 and 10/15/2021 at 5 amyloidosis centers in the US • Cleveland Clinic Foundation, University of Pittsburgh Medical Center, Oregon Health & Science University, Columbia University, and University of Missouri - Kansas City • Primar
Slide 17: Baseline Characteristics
Baseline Characteristics Total wtATTR vATTR Variable (N=624) (N=515) (N=109) p value Age <0.0001 Median (Q1, Q3) 78.0 (72.0, 83.0) 79.0 (73.1, 84.0) 71.0 (65.0, 76.0) Male sex (%) 546 (87.5%) 469 (91.1%) 77 (70.6%) Race (%) <0.0001 White 507 (81.3%) 472 (91.7%) 35 (32.1%) <0.0001 Black 109 (17.5%) 3
Slide 18: Primary Outcome: All-Cause Mortality
Primary Outcome: All-Cause Mortality Freedom from death at 30 months 70% (95% CI 65%, 74%) Median Follow-up (years) : 1.8 (1.1, 2.5) 142 (23%) Died 18
Slide 19: Tafamidis' survival rates comparable to ATTR-ACT
Tafamidis’ survival rates in the contemporary ATTR-CM population were comparable to ATTR-ACT Acoramidis & Placebo Tafamidis (ATTR-ACT & RWE) ATTR-ACT Placebo -33 19
Slide 20: Survival based on previous CV hospitalizations (Tafamidis)
Survival based on previous CV hospitalizations (Tafamidis) Non-CVH: 82% (76% - 87%) CVH: 56% (49% - 63%) 20
Slide 21: Survival based on previous CV hospitalizations (Acoramidis)
Survival based on previous CV hospitalizations (Acoramidis) Source: ATTRibute-CM, Data on file. Note: “+” symbol used to represent when a patient is censored at a given time. Cardiovascular-related hospitalization includes both CEC adjudicated CV-related hospitalization and Events of clinical intere
Slide 22: Tying it all together: Near-complete TTR stabilization leads to improved clinical outcomes
Tying it all together: Near-complete TTR stabilization leads to improved clinical outcomes ▪ Patients on acoramidis observed unprecedented 81-100% absolute survival at month 301,2 ▪ Patients with at least one CVH have a significantly higher risk of mortality, highlighting the need for ATTR-CM treatm