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our product platform

Our drug engineering platform leverages and efficiently translates innovation into therapies that matter.

discover novel genetic disease targets

Well described diseases that can be targeted at their source

  • Our genetic disease target engine allows us to identify genetic diseases to pursue based on the impact we might deliver for patients
  • We use computational genomics and statistical genetics, along with systematic disease mapping and partnerships with top academic institutions to identify potential disease-modifying targets
  • Our team is made up of industry leaders with proven track records who bring decades of scientific insight to the table when selecting genetic disease targets for our pipeline

create medicines with our industry research capabilities

Tailored therapeutic technologies that create potential first or best-in-class medicines

  • We select a therapeutic approach to target each genetic disease at its source
  • Our capabilities allow us to work across multiple modalities, including molecular dynamics assisted chemistry, gene therapy, therapeutic proteins and antisense oligonucleotides as well as multiple other technologies, including de novo QGS, NMR trapping and molecular dynamics
  • Our small, expert teams led by industry veterans focus on addressing the greatest unmet patient needs. We pair each disease with the modality that is suited to maximize patient impact

test our drugs through global development footprint

Broad clinical development capabilities across therapeutic areas and geographies

  • We have a global clinical development footprint
  • We have ongoing trials across five different therapeutic areas and over 25 countries
  • Our patient-centric clinical and regulatory strategies are focused on efficiency and speed and deploys our vast experience across enrollment, protocol quality, site activation, CRO quality and regional performance
  • We have expert, dedicated R&D teams in each therapeutic area and focused scientific and clinical advisors to guide our programs

deliver our products to patients through commercial infrastructures

Capabilities built to deliver genetic medicines to patients globally

  • We are building the capabilities and collaborating with partners to deliver our approved products to patients across the globe
  • We look for new diagnostic partnerships to identify more patients in need of our medicines
  • We are committed to deepening the early partnerships we have built with patient advocacy groups
  • We are implementing commercial partnerships in strategic geographics and establishing country-specific Early Access Programs (EAP) and patient assistance programs

We are poised to potentially help 4.5m+ patients across our pipeline

Our platform is delivering:

2approved products

30+programs in our pipeline

30+clinical trials across the globe

The Pipeline

Precision Cardiorenal

Precision Cardiorenal

Mendelian

Precision Oncology

Gene Therapy

Precision cardiorenal targets genetically-validated mechanisms underlying heart and kidney disease.

Pre-clinical
Phase 1
Phase 2
Phase 3
Commercial
Acoramidis

TTR stabilizer for transthyretin amyloidosis (ATTR)

disease

ATTR-CM, ATTR-PN

genetic source

TTR (transthyretin)

estimated prevalence

>400,000

modality

small molecule

Program summary

Acoramidis (AG10) is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin (TTR). Acoramidis was designed to mimic a naturally-occurring variant of the TTR gene (T119M) that is considered a “rescue mutation” because it has been shown to prevent or minimize ATTR in individuals carrying pathogenic, or disease-causing, mutations in the TTR gene. Results from the ongoing Phase 3 study investigating acoramidis for symptomatic transthyretin amyloid cardiomyopathy (ATTR-CM), which includes mortality and cardiovascular-related hospitalizations, are expected in 2023.

Encaleret

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

disease

Autosomal dominant hypocalcemia type 1 and hypoparathyroidism

genetic source

CaSR

estimated prevalence

12,000 / 200,000

modality

small molecule

Program summary

Encaleret is an investigational small molecule antagonist of the calcium sensing receptor (CaSR) being studied in disorders of calcium homeostasis, including autosomal dominant hypocalcemia type 1 (ADH1). Individuals with ADH1 have gain-of-function mutations in the CaSR, causing low serum calcium and a range of debilitating symptoms. ADH1 may also lead to relatively high levels of calcium in urine, a condition called hypercalciuria, which can result in impaired kidney function and can cause kidney stone formation. Encaleret has been administered to approximately 1,300 healthy volunteers and osteoporosis patients, demonstrating tolerability and showing clear modification of ADH1 disease drivers, encouraging our investigation of the compound in ADH1 patients. Encaleret is a potential first-in-class CaSR antagonist for ADH1 and initiated its Phase 3 clinical trial at the end of 2022.

BBP-711

GO inhibitor for primary hyperoxaluria type 1 (PH1) and recurrent kidney stone formers

disease

PH1 and FSF

genetic source

AGXT

estimated prevalence

5,000 / 1.5M

modality

small molecule

Program summary

BBP-711 is an orally-administered small molecule inhibitor of glycolate oxidase (GO) that is being developed to treat conditions of excess oxalate, including primary hyperoxaluria type 1 (PH1) and frequent kidney stone formation. In PH1, loss of function mutation in the AGXT gene results in accumulation of glyoxylate, which is converted into oxalate and leads to kidney stones and organ damage. Targeting GO is a clinically validated approach to reduce urinary oxalate by lowering the concentration of glyoxylate. The first-in-human Phase 1 trial of BBP-711 for hyperoxaluria was initiated in May 2021.

Mendelian diseases arise from defects in a single gene.

Pre-clinical
Phase 1
Phase 2
Phase 3
Commercial
NULIBRY (fosdenopterin)*

Synthetic cPMP for molybdenum cofactor deficiency (MocD) type a

disease

MocD Type A

genetic source

MOCS1

estimated prevalence

100

modality

small molecule

Program summary

Patients with MoCD Type A have mutations in the MOCS1 gene leading to deficient MOCS1A/B dependent synthesis of the intermediate substrate, cPMP. Substrate replacement therapy with fosdenopterin provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase (SOX), an enzyme that reduces levels of neurotoxic sulfites. NULIBRY™ (fosdenopterin) for Injection is a substrate replacement therapy that provides an exogenous source of cPMP, which is converted to molybdopterin. Molybdopterin is then converted to molybdenum cofactor, which is needed for the activation of molybdenum-dependent enzymes, including sulfite oxidase, an enzyme that reduces levels of neurotoxic sulfites. It is the first and only FDA-approved therapy indicated to reduce the risk of mortality in patients with MoCD Type A, and clinical trials have demonstrated that patients treated with NULIBRY or rcPMP had an improvement in overall survival compared to the untreated, genotype-matched, historical control group. In 2022, BridgeBio announced an asset acquisition agreement for NULIBRY with Sentynl Therapeutics, Inc. Sentynl gained full, exclusive licensing rights to ongoing development and commercialization of NULIBRY in the U.S. and is responsible for developing, manufacturing and commercializing fosdenopterin globally.

Low-dose infigratinib

FGFR1-3 inhibitor for achondroplasia

disease

Achondroplasia

genetic source

FGFR

estimated prevalence

55,000

modality

small molecule

Program summary

Infigratinib (BGJ398), is an orally administered, ATP-competitive, FGFR1-3 tyrosine kinase inhibitor in development for the treatment of FGFR-driven conditions, including achondroplasia, a bone growth disorder in children. Overactivating FGFR3 mutations drive downstream MAPK and STAT1 signaling that aberrates growth plate development, thereby causing disproportionate short stature and serious health complications stemming from cranial and spinal defects. Low dose infigratinib has the potential to help these patients due to its direct inhibition of the mutant FGFR3 receptor. In mouse models of achondroplasia, infigratinib demonstrated robust bone growth and clear improvement in cranial and spinal defects. BridgeBio is currently conducting a Phase 2 clinical trial in achondroplasia patients to evaluate the safety and tolerability of low-dose infigratinib, as well as clinical proof-of-concept.

BBP-418

Glycosylation substrate pro-drug for limb-girdle muscular dystrophy type 2i (LGMD2i)

disease

Limb-girdle muscular dystrophy type 2i (LGMD2i)

genetic source

FKRP

estimated prevalence

7,000

modality

small molecule

Program summary

BridgeBio is investigating what could be the first-ever oral disease-modifying treatment for limb girdle muscular dystrophy type 2i (LGMD2i) – BBP-418 (ribitol), which preclinical studies have shown is converted into the phosphorylated substrate that is inefficiently added to the alpha-dystroglycan “shock absorber” protein by the mutated FKRP gene product. Dysfunctional alpha-dystroglycan leads to progressive leg and arm muscle weakness, and in some cases severe respiratory and cardiovascular compromise. Currently, people with LGMD2i have symptomatic and palliative treatment options only, such as walking assists and ventilatory support, but no disease-modifying therapies exist. The Phase 2 trial is currently enrolling patients with a genetically-confirmed diagnosis of LGMD2i. In addition to safety, key efficacy measurements include changes in muscle αDG glycosylation levels, muscle biomarkers including creatine kinase, and changes in functional measures including 10 meter walk and North Star for Dysferlinopathy (NSAD). Initial Phase 2 data indicates the potential for BBP-418 to increase glycosylation of αDG and drive functional improvements for patients, as well as reduce CK, a key marker of muscle breakdown. Furthermore, the 90- and 180-day data show improvements on walk tests from baseline, which the Company believes suggests a potential impact on clinical function and on the rate of disease progression. BBP-418 has received Fast Track Designation and Orphan Drug Designation for the treatment of LGMD2i from the FDA and for LGMD from the European Medicines Agency.

BBP-671

PanK activator for pantothenate kinase-associated neurodegeneration (PKAN) & organic acidemias

disease

PKAN, organic acidemias and CASTOR

genetic source

PANK2 (pantothenate kinase)

estimated prevalence

7,000

modality

small molecule

Program summary

Coenzyme A (CoA) plays a crucial role in energy metabolism and is implicated in a large number of disorders, including ultra-rare diseases like pantothenate-kinase associated neurodegeneration, or PKAN, and organic acidemias (OAs), such as propionic acidemia (PA) and methylmalonic acidemia (MMA). BBP-671 is a novel small-molecule approach designed to modulate Coenzyme A (CoA) levels by leveraging recent research about the CoA synthetic pathway. While PanK2 is inactivated in PKAN patients, other isoforms known as PanK1 and PanK3 are still active. This leads to decreased PanK activity and impaired CoA synthesis. Conversely, in organic acidemias, build-up of metabolic intermediates can inhibit the activity of all three isoforms of PanK and impair CoA synthesis. Early experiments have shown that our small molecules can bind to all three PanK isoforms, prevent feedback inhibition, and thereby increase PanK activity and increase CoA synthesis. BridgeBio believes initial findings from its Phase 1 study of healthy volunteers demonstrate target engagement and proof of mechanism of BBP-671 provided by evidence that BBP-671 can cross the blood brain barrier. Based on these data, BridgeBio intends to move forward with the second part of the Phase 1 clinical study in patients with propionic acidemia and methylmalonic acidemia in the second half of 2022, and plans to initiate a pivotal Phase 2/3 study in PKAN in 2023.

Precision oncology targets clear genetic aberrations that drive cancer.

Pre-clinical
Phase 1
Phase 2
Phase 3
Commercial
TRUSELTIQ (high-dose infigratinib)*

FGFR1-3 inhibitor for FGFR+ cancers

disease

Fibroblast Growth Factor Receptor

genetic source

FGFR

estimated prevalence

37,000

modality

small molecule

Program summary

Infigratinib is an orally administered, ATP-competitive, FGFR1-3 tyrosine kinase inhibitor in development for the treatment of FGFR-driven cancers. Multiple Phase 3 clinical trials are currently enrolling; please explore www.clinicaltrials.gov for more information. TRUSELTIQ™ (infigratinib) obtained accelerated approval by FDA, was conditionally approved by Health Canada and received approval in Australia. TRUSELTIQ™ is not approved for use by any other health authority. For the current approval status and more information please visit www.truseltiq.com. In 2022, Helsinn Group gained full, exclusive licensing rights to commercialize high-dose infigratinib worldwide in oncology indications except in mainland China, Hong Kong and Macau. BridgeBio has established a strategic collaboration with LianBio for the development and commercialization of infigratinib in oncology indications in mainland China, Hong Kong and Macau.

BBP-398

SHP2 inhibitor monotherapy for MAPK/RAS driven cancers

disease

MAPK/RAS driven cancers

genetic source

PTPN11 (SHP2) and MAPK pathway

estimated prevalence

>500,000

modality

small molecule

Program summary

BBP-398

SHP2 inhibitor combo therapy for MAPK/RAS driven cancers

disease

MAPK/RAS driven cancers

genetic source

PTPN11 (SHP2) and MAPK pathway

estimated prevalence

>500,000

modality

small molecule

Program summary

BBO-8520

KRASG12C dual inhibitor for KRAS cancers

disease

KRAS+ cancers

genetic source

KRAS

estimated prevalence

40,000

modality

small molecule

Program summary

PI3Kα:RAS breaker

PI3Kα:RAS breaker for KRAS cancers

disease

KRAS+ cancers

genetic source

KRAS

estimated prevalence

>500,000

modality

small molecule

Program summary

Pan-KRAS inhibitor

Pan-KRAS inhibitor for KRAS cancers

disease

KRAS+ cancers

genetic source

KRAS

estimated prevalence

>500,000

modality

small molecule

Program summary

BBP-954

GPX4 inhibitor for multiple tumors

disease

Multiple tumors

estimated prevalence

>500,000

modality

small molecule

Program summary

GPX4 neutralizes toxic free radicals at the lipid membrane, protecting cells from death by ferroptosis. BridgeBio is developing covalent inhibitors of GPX4 designed to induce ferroptosis in cancer cells. BridgeBio has entered into a strategic collaboration with Helsinn to co-develop and co-commercialize a potentially first-in-class inhibitor designed to target glutathione peroxidase 4 (GPX4) with the hope of providing an effective new therapy for patients with difficult-to-treat tumors. Our approach has demonstrated monotherapy activity, reducing tumor volume in a mouse xenograft model of renal cell carcinoma. Recent high profile publications provide preclinical in vivo rationale for monotherapy and combinations with immuno-oncology agents, kinase inhibitors, and chemotherapy. Optimization of oral lead compounds is ongoing.

Gene therapy aims to replace functional copies of single genes harboring loss-of-function mutations. Genetic material is delivered into cells via non-replicating virus particles, offering the potential for one-time, curative therapy.

Pre-clinical
Phase 1
Phase 2
Phase 3
Commercial
BBP-631

AAV5 gene therapy for congenital adrenal hyperplasia (CAH)

disease

CAH

genetic source

CYP21A2

estimated prevalence

>75,000

modality

gene therapy

Program summary

BBP-631 is an investigational adeno-associated virus (AAV) gene therapy to treat CAH due to 21-hydroxylase deficiency at its source. BBP-631 is designed to deliver a functional copy of the 21-hydroxylase gene and has been shown through multiple animal studies to result in efficient and persistent delivery to the adrenal gland, where hormones are naturally made. If successful, we hope to restore the body’s hormone and steroid balance by enabling people with CAH to make their own cortisol and aldosterone — something that is not possible with any treatment on the market or currently in clinical trials for CAH. BridgeBio believes a gene therapy has the potential to restore the delicate balance of hormone production that is dysregulated in this disease. The initial Phase 1/2 data readout is expected in the second half of 2023.

BBP-812

AAV9 gene therapy for Canavan disease

disease

Canavan Disease

genetic source

ASPA

estimated prevalence

1,000

modality

gene therapy

Program summary

BBP-812 is an investigational adeno-associated virus (AAV) gene therapy for Canavan disease, which begins in infancy and progresses rapidly to severe neuromuscular symptoms and early mortality. Using AAV gene therapy, BridgeBio seeks to deliver functional copies of the ASPA gene throughout the body and into the brain, correcting the disease. To date, results have shown promising pharmacodynamic, tolerability and preliminary functional efficacy. A broader Phase 1/2 data readout, including safety and efficacy findings and updates on the pharmacodynamic data, is expected in the first half of 2023.

AAV gene therapy

Tuberous sclerosis (TSC) 1/2

disease

Tuberous sclerosis (TSC) 1/2

genetic source

TSC

estimated prevalence

100,000

modality

gene therapy

Program summary

AAV gene therapy

Cystinuria

disease

Cystinuria

genetic source

SLC3A1, SLC7A9

estimated prevalence

30,000

modality

gene therapy

Program summary

* BridgeBio has licensed these therapies to partners with the hope of reaching as many people with unmet need as possible.

Visit publications for selected literature and presentations for recent data presented at medical meetings about each program.

our commitment to patients

BridgeBio ultimately exists to help patients. We are committed to discover, create, test and deliver breakthrough medicines for genetic diseases to patients as quickly and safely as possible.

Elliot, living with MoCD Type A Read More
Porter, living with cholangiocarcinoma Read More
Lesley, living with CAH Read More