science & pipeline

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The Pipeline

Acoramidis

TTR stabilizer for transthyretin amyloidosis (ATTR)

disease

ATTR-CM

genetic source

TTR (transthyretin)

estimated prevalence

>500,000 (U.S. & EU)

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” due to its ability to prevent or minimize ATTR in individuals carrying pathogenic, or disease-causing, mutations in the TTR gene. BridgeBio submitted its NDA to the U.S. FDA in 2023 with regulatory filings in additional markets to follow in 2024.

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Encaleret

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

disease

Autosomal dominant hypocalcemia type 1

genetic source

CaSR

estimated prevalence

20,000 - 25,000 (U.S. & EU)

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.

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BBP-418

Glycosylation substrate pro-drug for limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)

disease

Limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9)

genetic source

FKRP

estimated prevalence

7,000 - 9,000 (Includes all potentially addressable indications in U.S., EU & Japan)

modality

small molecule

Program summary

BBP-418 has the potential to be the first oral therapy indicated for LGMD2I/R9, which is developed to supply supraphysiological levels of a substrate upstream of the mutant FKRP enzyme, helping to drive residual activity of the enzyme to glycosylate alpha-dystroglycan (αDG). Based on recent interactions with the U.S. FDA, BridgeBio believes there is potential to file for Accelerated Approval. BBP-418 has received Rare Pediatric Disease Designation, Fast Track Designation, and Orphan Drug Designation for the treatment of LGMD2I/R9 from the U.S. FDA and Orphan Drug Designation for LGMD from the European Medicines Agency.

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Low-dose infigratinib

FGFR inhibitor for achondroplasia

disease

Achondroplasia

genetic source

FGFR

estimated prevalence

55,000 (U.S. & EU)

modality

small molecule

Program summary

Infigratinib is an oral small molecule 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 issues. Low dose infigratinib has the potential to help children due to its direct inhibition of the mutant FGFR3 receptor. BridgeBio initiated PROPEL 3, a Phase 3 clinical trial studying the efficacy and safety of infigratinib in children with achondroplasia. Both the U.S. FDA and the EU EMA indicated the trial design for PROPEL 3 would be acceptable as a registrational study to support a marketing application for the treatment of children with achondroplasia.

Low-dose infigratinib

FGFR inhibitor for hypochondroplasia

disease

Hypochondroplasia

genetic source

FGFR

estimated prevalence

55,000 (U.S. & EU)

modality

small molecule

Program summary

Infigratinib is an oral small molecule designed to inhibit FGFR3 and target skeletal dysplasias, such as hypochondroplasia, at the source to improve uncommon bone growth by decreasing the overactivity of FGFR3. Hypochondroplasia is an FGFR3-associated skeletal dysplasia and is a rare condition affecting approximately 55,000 people in the United States and EU, including up to 10,000 children and adolescents with open growth plates. Hypochondroplasia presents with a wide spectrum of phenotypes including disproportionate short stature, mild joint laxity and macrocephaly. Currently, no treatments for hypochondroplasia are approved in the United States. ACCEL, the observational lead-in program for hypochondroplasia, was initiated with the first participant consented in May 2024.

BBP-812

AAV9 gene therapy for Canavan disease

disease

Canavan Disease

genetic source

ASPA

estimated prevalence

1,000 (U.S. & EU)

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.

Preclinical programs

Multiple assets in large genetic disease populations

Program summary

Our preclinical programs include gene therapies under development for tuberous sclerosis (TSC) 1/2, cystinuria and LMNA cardiomyopathy, as well as discovery research targeting large, complex genetic diseases with high unmet need.

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

Miguel, living with achondroplasia Read More

Tobin, a girl with Canavan disease Read More