Bridgebio Pharma, Inc. · NASDAQ:BBIO

Bridgebio Pharma — Technical Presentation (September 2023)

13 slides from Bridgebio Pharma's technical presentation, Sep 20, 2023

  1. Slide 1: Preliminary Results from MLB-01-003: Open Label Phase 2 Study of BBP-418 in LGMD2I/R9

    Preliminary Results from MLB-01-003: An Open Label Phase 2 Study of BBP-418 in Patients with Limb-girdle Muscular Dystrophy Type 2I/R9 Amy Harper, MD, Virginia Commonwealth University

  2. Slide 2: Disclosures

    Disclosures • I have the following conflict/s of interest to declare: • I receive VCU contracted funds from several sponsors for clinical research studies (PI or Co-I), including NSPharma, Italafarmaco, Santhera (ReveraGen), Dyne, Novartis (Avexis), Astellas, Fulcrum and ML Bio. • Additionally, I re

  3. Slide 3: LGMD2I/R9 is caused by mutations in FKRP with an established genotype/phenotype association

    LGMD2I/R9 is caused by mutations in FKRP and characterized by an established genotype/phenotype association Birth Early Childhood Late Childhood Adolescence to adulthood L276I  Age of symptom onset Loss of ambulation: 25% by age 40 Homozygotes 18 ± 3 years old Asymptomatic Asymptomatic Respiratory

  4. Slide 4: Alpha Dystroglycan, disrupted in LGMD2I, is part of the dystrophin-glycoprotein complex

    Alpha Dystroglycan (⍺DG), disrupted in LGMD2I (LGMD R9 FKRP-related), is an integral part of the dystrophin-glycoprotein complex​ Figure from Wicklund et al., The limb-girdle muscular dystrophies. Neurologic Clinics, 2014. 4 BridgeBio Pharma, Inc. © 2023

  5. Slide 5: Oral BBP-418 is under investigation as an upstream substrate supplement

    Oral BBP-418 is under investigation as an upstream substrate supplement to drive residual activity of mutant FKRP in LGMD2I/R9, targeting the disease at its source LGMD2I/R9 Disease Mechanism Proposed BBP-418 Therapeutic Approach Functional FKRP fully glycosylates alpha-dystroglycan (αDG) which Supp

  6. Slide 6: Natural history data: αDG glycosylation mirrors disease severity and remains stable

    Natural history data supports the premise that glycosylation of αDG in muscle mirrors the severity of LGMD2I/R9 disease and remains stable over time Reduced ⍺DG glycosylation in other FKRP genotypes vs. Glycosylated αDG levels remain stable over 6─12 months in L276I/L276I homozygous LGMD2I/R9 patien

  7. Slide 7: BBP-418 is under investigation in a small, open label Phase 2 study

    BBP-418 is under investigation in a small, open label Phase 2 study in individuals with LGMD2I/R9 Key Endpoints Dose Maximum Long-term escalation dose extension  NSAD  10-meter walk test/100-meter timed test  FVC 90 days (N=14) 90 days (N=14) 24 months  PUL2.0  Glycosylated αDG levels After Par

  8. Slide 8: BBP-418 has been well tolerated, with only minor GI related adverse events

    BBP-418 has been well tolerated, with only minor GI related adverse events recorded in the Phase 2 study TEAE # of incidents Severity  189 adverse events (AEs) were recorded in Diarrhea 9 66% mild, 33 % moderate the study with 19 possibly or probably related to BBP-418 treatment and 4 Dehydration 1

  9. Slide 9: Sustained increases in glycosylated αDG and decreases in serum creatine kinase

    Sustained increases in levels of glycosylated αDG in muscle and decreases in serum creatine kinase observed in Phase 2 study of BBP-418 Increase in glycosylated αDG in muscle observed post dosing Reduction in mean serum creatine kinase (CK) observed post with BBP-418 (median ± 95% CI) dosing with BB

  10. Slide 10: Stabilization in ambulatory and clinical measures after 21 months of treatment

    Stabilization in ambulatory and clinical measures observed after 21 months of treatment with BBP-418 in Phase 2 study Change from baseline in 10MWT (m/s) Change from baseline in 100MTT (s) Change from baseline in NSAD Blue lines denote natural history data and red lines denote on-treatment data coll

  11. Slide 11: Early changes in glycosylated αDG at 3 months may be associated with clinical improvements

    Phase 2 data support that early changes in glycosylated αDG levels at 3 months may be associated with subsequent clinical improvements glycosylated αDG Levels at 3 months vs. 100MTT at 9 months glycosylated αDG Levels at 3 months vs. 10MWT at 9 months 11 BridgeBio Pharma, Inc. © 2023

  12. Slide 12: Summary

    Summary Phase 2 Study (MLB-01-003) • Increased glycosylation of ⍺DG observed following BBP-418 dosing which is sustained over time • Large, sustained reduction in creatine kinase observed over an extended (up to 21-months) treatment period • Stabilization in NSAD and ambulatory measures observed ove

  13. Slide 13: Thank You!

    Thank You! • ML Bio study participants • LGMD2I/R9 patients, families, and patient advocates

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