NULIBRY™ (fosdenopterin) – Synthetic cPMP for MoCD Type A

Fosdenopterin – SSIEM 2019

TRUSELTIQ™ (high-dose infigratinib) – FGFR1-3 inhibitor for cholangiocarcinoma

High-dose Infigratinib – ASCO 2020 – Phase 2 Javle 4591

Acoramidis (AG10) – TTR stabilizer for transthyretin amyloidosis (ATTR)

ATTRibute-CM Detailed Results From ESC Congress 2023

Acoramidis (AG10) – ESC 2023 – ATTRibute-CM-Efficacy and Safety of Acoramidis in Transthyretin Amyloid Cardiomyopathy

Acoramidis (AG10) – ESC 2023 – Acoramidis Produces Near-Complete TTR Stabilization in Variant ATTR Patients that is Greater than that Achieved with Tafamidis

ATTRibute-CM Phase 3 Topline Results

Acoramidis (AG10) – ACC 2022 – Updated Analysis Ongoing Phase 2 OLE

Acoramidis (AG10) TTR – ISA 2022 – Updated Analysis Ongoing Phase 2 OLE

Acoramidis (AG10) – ACC 2022 – Unmet Needs Among Patients with ATTR

Patient and family experience with transthyretin amyloid cardiomyopathy (ATTR-CM) and polyneuropathy (ATTR-PN) amyloidosis: results of two focus groups

Rintell, D. et al. Orphanet J Rare Dis. 2021. 16:70. https://doi.org/10.1186/s13023-021-01706-7.

Acoramidis (AG10) – ACC 2019 – Phase 2 ATTR-CM Subgroup Analysis

Acoramidis (AG10) – AHA 2019 – Phase 3 Design

Acoramidis (AG10) – AHA 2019 – Phase 2 OLE

Acoramidis (AG10) – AHA 2019 – Differential Transthyretin Binding by AG10 Compared to Tafamidis

Acoramidis (AG10) – AHA 2019 – Differential Ex Vivo Stabilization of TTR by AG10 and Tafamidis in TTR Variants

High-dose infigratinib – FGFR1-3 inhibitor for urothelial carcinoma (urinary tract and bladder cancer)

High-dose Infigratinib – ASCO 2020 – UC Ph1_2 Lyou 5038

High-dose Infigratinib – ASCO 2020 – Adjuvant Ph3 TiP

Low-dose infigratinib – FGFR1-3 inhibitor for achondroplasia

Low-dose Infigratinib – Achondroplasia – ENDO 2023 – Evaluation of Bone Mineral Density

Low-dose Infigratinib – Achondroplasia – ENDO 2023 – PROPEL, PROPEL 2 and PROPEL OLE

Low-dose Infigratinib – Achondroplasia – PROPEL2 Topline Results

Low-dose Infigratinib – Achondroplasia – ISDS 2022- Qualitative Research in Children and Parents of Children with Achondroplasia

Low-dose Infigratinib – Achondroplasia – ENDO 2022 – PROPEL Medical History Clinical Assessment with Baseline Parameters

Low-dose Infigratinib – Hypochondroplasia – ENDO 2022 – Preclinical Data

Low-dose Infigratinib – Achondroplasia – ENDO 2022 – QoL Measurements and Medical Challenges

Low-dose Infigratinib – Achondroplasia – ENDO 2022 – PROPEL, PROPEL2 and PROPEL OLE TiP

Low-dose Infigratinib – Achondroplasia – ENDO 2022 – Evaluation of BMI and Metabolic Parameters

Low-dose Infigratinib – Achondroplasia – ENDO 2022 – QoL Measurements and Patient-reported Outcome Measures

Low-dose Infigratinib – Achondroplasia – ASBMR 2021 – PROPEL2 Trial Design

Low-dose Infigratinib – Achondroplasia – ASBMR 2021 – PROPEL Trial Design & Baseline Characteristics

Low-dose Infigratinib – Achondroplasia – ENDO 2021 – PROPEL2 Phase 2 Study Design

Low-dose Infigratinib – Achondroplasia – ENDO 2020 – PROPEL TiP

Low-dose Infigratinib – Achondroplasia – ENDO 2020 – Demuynck

Low-dose Infigratinib – Achondroplasia – ENDO 2020 – Dobscha Lit Review

Low-dose Infigratinib – Achondroplasia – ENDO 2020 – Reyes, Preclinical Phosphate Levels

Low-dose Infigratinib – Achondroplasia – ASHG 2019 – ACH Mouse Model v. Human Phosphorus

Encaleret – Ca sensing receptor antagonist for autosomal dominant hypocalcemia type 1 (ADH1)

Encaleret – ENDO 2023 18 Month Phase 2b Data

Encaleret – ENDO 2023 Preliminary Results from Genetic Testing Program

Encaleret – ESE 2023 12 Month Phase 2b Data

Encaleret – ESE 2023 12 Month Phase 2b Data

Encaleret – ASN 2022 Mineral Homeostasis Phase 2b Data

Encaleret – ASBMR 2022 6 month Phase 2 data

Encaleret – ENDO 2022 Complete Phase 2B Data

Encaleret – ECTS 2022 – Phase 2B Period 2 Data

Encaleret – ECE 2022 – Phase 2B Data on Mineral Physiology

Encaleret – AHA 2021 – Phase 2B Data on Corrected QT Interval

Encaleret – ASBMR 2021 – ADH1 Review

Encaleret – ASBMR 2021 – Phase 2B Period 2 Data

Encaleret – ENDO 2021 – Phase 2B Data

Encaleret – ENDO 2021 – Phase 2B Study Design

BBP-418 – Glycosylation substrate pro-drug for limb-girdle muscular dystrophy 21 (LGMD2i)

BBP-418 – MDA 2022 – Oral Presentation – Phase 2 Data at 15 Months

BBP-418 – MDA 2022 – Poster – Phase 2 Data at 15 Months

BBP-418 – MDA 2022 – Preliminary Data on Bioassay Measuring ⍺DG

BBP-418 – WMS 2022 – aDG assay poster

BBP-418 – WMS 2022 – Ph2 data

BBP-418 – ICNMD 2022 – Phase 2 Data

BBP-418 – MDA 2022 – Preliminary Phase 2 Data

BBP-418 – MDA 2022 – Phase 1 Data

BBP-418 – MDA 2021 – Analysis of Patient Biopsies with Ratio-metric Western Blot Method

BBP-589 – Recombinant collagen for recessive dystrophic epidermolysis bullosa (RDEB)

BBP-589 – SID 2022 – Phase 2 Data

BBP-589 – SID 2021 – Interim Update on Phase 1/2 Data

BBP-681 – Topical PI3Ka inhibitor for venous malformations (VMs), lymphatic malformations (LMs) and venolymphatic malformations (VLMs) associated with PIK3CA or TEK mutations

VT30 (BBP-681) Overview

VT30 (BBP-681) – ISSVA 2022 – Phase 1b Data

BBP-812 – AAV9 gene therapy for Canavan disease

BBP-812 – CNS 2022 – CANaspire Phase 1/2 Study

BBP-812 – CNS 2022 – CANinform Natural History Study

BBP-812 – ASGCT 2021 – NHP

BBP-398 – SHP2 inhibitor for tumors driven by RAS and receptor tyrosine kinase mutations

BBP-398 – 2021 AACR-NCI-EORTC – Preclinical Data in NSCLC Models

BBP-398 – 2019 AACR-NCI-EORTC – Preclinical osimertinib combo in NSCLC models

BBP-711 – Glycolate oxidase (GO) inhibitor for primary hyperoxaluria type 1 (PH1) and frequent stone formers

BBP-711 – ESPN 2022 – Phase 1 Data in Healthy Volunteers & Phase 2/3 Trial Design

BBP-671 – PanK activator for pantothenate kinase-associated neurodegeneration (PKAN) and organic acidemias (OA)

BBP-671 – PAS 2022 – Positive Phase 1 Data in Healthy Volunteers

Pantothenate kinase activation relieves coenzyme A sequestration and improves mitochondrial function in mice with propionic acidemia

Subramanian C, et al. Sci Transl Med. 2021 Sep 15;13(611):eabf5965. doi: 10.1126/scitranslmed.abf5965.

A therapeutic approach to pantothenate kinase associated neurodegeneration

Sharma LK, et al. Nat Commun. 2018 Oct 23;9(1):4399. doi: 10.1038/s41467-018-06703-2. PMID: 30352999; PMCID: PMC6199309.

BBP-631 – AAV5 gene therapy for congenital adrenal hyperplasia (CAH)

BBP-631 – ENDO 2022 – Pre-screening Protocol

BBP-631 – ENDO 2021 – Phase 1/2 Study Design

BBP-631 – ASGCT 2021 – NHP

BBP-631 – ESGCT 2019 – NHP

BBP-454 – KRAS inhibitor for KRAS cancers

BBIO – KRAS Cancers – RAS Initiative Symposium 2022 – KRASG12C GTP/GDP dual inhibitor and PI3Kα:RAS breaker

BridgeBio

J.P. Morgan presentation, 2023

Oncology overview, Q4 2022

Corporate presentation