Bridgebio Pharma, Inc. · NASDAQ:BBIO
Bridgebio Pharma — Investor Day (September 2020)
24 slides from Bridgebio Pharma's investor day presentation, Sep 29, 2020
Slide 1: R&D Day September 29, 2020
R&D Day September 29, 2020 Exhibit 99.1
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: BridgeBio Pharma: Hope through rigorous science
BridgeBio Pharma: Hope through rigorous science Our mission: To discover, create, test and deliver transformative medicines to treat patients who suffer from genetic diseases and cancers with clear genetic drivers
Slide 4: BridgeBio corporate overview: Strategy
BridgeBio corporate overview Strategy Platform Products
Slide 5: We are at Day 1 in the era of genetic medicine
Source: Claussnitzer et al., Nature 2020 Draft human genome High-throughput sequencing UKBiobank recruitment begins GWAS results catalogue initiated ClinVar launches >1 million GWAS samples Hundreds of monogenic disease-causing variants are discovered every year… …and common disease associations are
Slide 6: A vast opportunity to help patients
~ > US prevalence, mn Americans are living with a genetic diseases Of people affected are children Of these diseases have an approved therapy 57% of cancers have genetic drivers A vast opportunity to help patients ~27 million 50% Only 5% Source: Global Genes, American Cancer Society, American Heart
Slide 7: Our strategy is simple
Our strategy is simple Right playing field Genetic disease History teaches us about strategy: BBIO applications: Stay adaptive Beautiful science, NPV positive Right tenets No initial focus on TA, disease, or modality. Repeated application
Slide 8: Our organizational principles enable scale
Our organizational principles enable scale Simple rules repeated at many levels Simple rules – put patients first, think independently and let science speak, be efficient History teaches us about growth: BBIO applications: De-centralized approach – small teams that focus and are incented at the leve
Slide 9: Result: Pipeline momentum
Result: Pipeline momentum 20+ programs Multiple $1B+ opportunities in Ph3 Two NDA submissions >10 INDs in 5 years BridgeBio founded 1 pipeline program 10 pipeline programs 3 clinical compounds 20 10 1
Slide 10: Increasing returns to scale – BridgeBio since IPO
Increasing returns to scale – BridgeBio since IPO 7 INDs filed Six new clinical trials initiated (16 total), >350 trial sites across 25 countries NDA for MoCD Type A accepted, ODD & Fast Track received for 2L CCA program 8 new programs, including LGMD2i and ADH1, both in the clinic Achievement: TTR
Slide 11: BridgeBio corporate overview: Platform
BridgeBio corporate overview Strategy Platform Products
Slide 12: BridgeBio drug engineering basics: our platform
Well described diseases than can be targeted at their source Tailored therapeutic technologies to create first or best-in-class medicines Broad clinical development capabilities across therapeutic areas and geographies Building the capabilities to deliver genetic medicines to patients globally Disco
Slide 13: Discover: capabilities to identify new genetic disease targets at scale
Mining of large genotype-phenotype databases De novo target discovery Target validation Indication expansion Manual annotation and prioritization of the 7K known genetic diseases 15 current partnerships Our target identification engine is driven by three core areas of strength: Computational genomic
Slide 14: Scientific insight and judgment from industry leaders
Scientific insight and judgment from industry leaders with a proven track record Richard Scheller, PhD Chairman of R&D Charles Homcy, MD Founder and Chairman of Pharmaceuticals Frank McCormick, PhD Founder and Chairman of Oncology Len Post, PhD Advisor Phil Reilly, MD, JD Advisor Discover
Slide 15: Create: capabilities across modalities
Industry-leading capabilities across 4 modalities: Medicinal chemistry Gene therapy Molecular dynamics Reversible and irreversible chemistry Topical formulations Vector optimization Novel capsid engineering Analytical assay development Optimal use: Inhibition of GOF or allosteric activation of LOF m
Slide 16: Research leaders with a productive history developing novel therapeutics
Mendelian Oncology Gene therapy Research leaders with a productive history developing novel therapeutics Create Uma Sinha, PhD Chief Scientific Officer Robert Zamboni, PhD Chemistry Eli Wallace, PhD Chief Scientific Officer, Oncology Pedro Beltran, PhD SVP, Biology Clayton Beard, PhD SVP, Research a
Slide 17: Test: our global clinical development footprint
16 ongoing trials across 5 different therapeutic areas, >350 trial sites, and 25 countries Creative clinical and regulatory strategy, e.g., unique, nested Phase 3 trial design for acoramadis in ATTR Central operations toolkit for enrollment, protocol quality, site activation, CRO quality, regional p
Slide 18: Deliver: building capabilities to deliver products to patients globally
Global commercial infrastructure to leverage our drug and disease expertise Diagnostic partnerships to identify patients in need of our medicines Disease awareness strategies including close partnerships with patient advocacy groups Country-specific Early Access Programs (EAP) and patient assistance
Slide 19: The platform is delivering
Discover Novel genetic disease targets Create Medicines with industry-leading research capabilities Deliver Our products to patients through commercial infrastructure Test Our drugs through global development footprint 20+ Disclosed programs in the pipeline >10 INDs since 2015 16 Clinical trials acr
Slide 20: BridgeBio corporate overview: Products
BridgeBio corporate overview Strategy Platform Products
Slide 21: Pipeline by portfolio segment and phase
Portfolio segment Program Drug mechanism Diseases Patient pop. (US+EU) Modality Preclinical Clinical Discovery IND-enabling Phase1 Phase 2 Phase 3 Mendelian Acoramidis TTR stabilizer ATTR-CM >400K Fosdenopterin cPMP replacement MoCD type A 100 Infigratinib Low-dose FGFR1-3i Achondroplasia 55K Encale
Slide 22: Four core value drivers over the next 12-24 months
Low-dose infigratinib (FGFRi) for achondroplasia Enrolling Ph2 study Dose first child Phase 2 data 2021 ATTR-CM Ph3 ongoing GLP tox ongoing File IND Phase 1/2 data 2021 Ph2 ongoing FPI in Ph2 study Phase 2 data 2021 Acoramidis: TTR stabilizer for ATTR Gene therapy for congenital adrenal hyperplasia
Slide 23: A pipeline with multi-blockbuster potential
A pipeline with multi-blockbuster potential $1B+ opportunities in the pipeline Acoramidis for ATTR CM and PN Low-dose infigratinib for achondroplasia AAV5 gene therapy for congenital adrenal hyperplasia High-dose infigratinib for adjuvant urothelial carcinoma Pan-mutant KRAS inhibitor for KRAS+ canc
Slide 24: Thank you to our speakers
Speaker Low-dose infigratinib (FGFRi) for achondroplasia Acoramidis: TTR stabilizer for ATTR cardiomyopathy Gene therapy for congenital adrenal hyperplasia (BBP-631) Encaleret: CaSR inhibitor for autosomal dominant hypocalcemia type 1 (ADH1) Oncology research, KRAS Related program Thank you to our s