Slide 1: ATTRibute-CM Part A topline results
ATTRibute-CM Part A topline results December 2021
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: ATTRibute-CM did not meet its Month 12 primary endpoint
ATTRibute-CM did not meet its Month 12 primary endpoint Primary endpoint p-value Change from baseline in 6MWD 0.76 Observed change from baseline in 6MWD at Month 12 Mean (SD) Median Acoramidis -9.28m (62.7) -4.83m Placebo -7.04m (59.7) -6.25m ATTRibute-CM placebo group outperformed tafamidis-treated
Slide 4: Observed ATTRibute-CM placebo substantially outperformed historical controls
Observed ATTRibute-CM placebo substantially outperformed historical controls Change from baseline in 6MWD Meters Healthy elderly adults1 NAC natural history (WT)3 0 ATTRibute-CM placebo2 ATTR-ACT placebo4 -10 -20 Approximate annual decline: Healthy elderly adult: -7m -30 ATTRibute-CM (placebo): -7m
Slide 5: Lack of placebo deterioration across NYHA classes is baffling
Lack of placebo deterioration across NYHA classes is baffling Change from baseline in 6MWT total distance at months 6, 9, 12 Baseline NYHA Class I Baseline NYHA Class II Baseline NYHA Class III 5
Slide 6: ATTRibute-CM enrolled a similar population as ATTR-ACT
ATTRibute-CM enrolled a similar population as ATTR-ACT Baseline Trait ATTRibute-CM (mITT) ATTR-ACT Age Mean 77.0 74.3 Median 78.0 74.6 NYHA Class Class I 11.2% 8.4% Class II 72.7% 59.6% Class III 16.1% 32.0% 6MWT (m) Mean 360 352 Genetic TTR status Variant 9.7% 24.0% Wildtype 90.3% 76.0% Geography U
Slide 7: ATTRibute-CM was relatively well-balanced
ATTRibute-CM was relatively well-balanced Baseline Trait Acoramidis (mITT) Placebo (mITT) Age Mean 77.2 76.9 Median 78.0 77.8 NYHA Class Class I 12.6% 8.5% Class II 70.2% 77.6% Class III 17.2% 13.9% 6MWT (m) Mean (SD) 364 (103) 351 (102) Genetic TTR status Variant 9.1% 9.5% Wildtype 90.6% 90.0% Geog
Slide 8: Trial population differences unlikely to contribute to lack of 6MWT benefit
Trial population differences unlikely to contribute to lack of 6MWT benefit Could the lack of 6MWD benefit be caused by… Preliminary answer Unlikely – our 6MWD result was modestly better in NYHA I/II …higher proportion of NYHA II vs III participants? than NYHA III participants Unlikely – our 6MWD re
Slide 9: Acoramidis improved NT-proBNP relative to placebo
Acoramidis improved NT-proBNP relative to placebo Percent change from baseline in NT-proBNP at Month 12 – mITT population Nominal p < 0.051 PRELIMINARY ANALYSIS – NOT VALIDATED 1Inference analysis (p-value) based on absolute change from baseline between groups 9
Slide 10: Acoramidis improved KCCQ-OS relative to placebo
Acoramidis improved KCCQ-OS relative to placebo KCCQ-OS by treatment and visit – MMRM without imputation Nominal p < 0.05 10
Slide 11: Acoramidis increased serum TTR concentrations
Acoramidis increased serum TTR concentrations Percent change from baseline in serum TTR by treatment and visit – mITT population Nominal Nominal p < 0.011 p < 0.011 1Inference analysis (p-value) based on absolute change from baseline between groups 11
Slide 12: No safety signals of potential clinical concern identified
No safety signals of potential clinical concern identified To protect the integrity of Part B, Sponsor’s access to adverse event data for Part A excludes AEs leading to a cardiovascular hospitalization excepting events with the outcome of death Preliminary safety results Treatment emergent adverse e
Slide 13: Preliminary summary and next steps
Preliminary summary and next steps After 12 months of ATTRibute-CM, acoramidis demonstrated relative to placebo: ▪ No improvement in functional status as measured by 6MWD ▪ Other measures positive, including improvements in: ▪ KCCQ-OS ▪ NT-proBNP ▪ Serum TTR ▪ No safety signals of clinical concern a
Slide 14: Major catalysts across the pipeline anticipated over the next 12 months
Major catalysts across the pipeline anticipated over the next 12 months Core value drivers Pipeline upside Low-dose infigratinib (FGFRi) for achondroplasia: Ph2 proof- COL7 replacement for RDEB: Data from Ph2 study (early ’22) of-concept data (1H22) AAV9 gene therapy for GALT: IND submission (2H22)
Slide 15: A sincere thank you to patients and families, investigators, referring physicians
(acoramidis for ATTR-CM) A sincere thank you to patients and families, investigators, referring physicians, clinical research staff, Eidos/BridgeBio employees, and collaborating research partners 15