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The Pipeline

Product
Disease
Preclinical
Phase 1
Phase 2
Phase 3
Commercial
Attruby™ (acoramidis)
Disease

Transthyretin-mediated amyloidosis cardiomyopathy (ATTR-CM)

Commercial
genetic source

TTR (transthyretin)

estimated prevalence

>400,000 (Globally)

modality

small molecule

molecule icon
Program summary

Attruby is a prescription medicine used to treat adults with a disease that affects the heart muscle called cardiomyopathy of wild-type or variant transthyretin-mediated amyloidosis (ATTR-CM), to reduce death and hospitalization related to heart problems. It is not known if Attruby is safe and effective in children.
The Prescribing Information for Attruby includes information about gastrointestinal adverse reactions.

Please Visit Attruby.com/PI for Full Prescribing Information.
Acoramidis
Disease

TTR stabilizer for transthyretin amyloidosis (ATTR)

Phase 3
genetic source

TTR (transthyretin)

estimated prevalence

~50,000 (Globally)

modality

small molecule

molecule icon
Program summary

Acoramidis is now also under Phase 3 investigation among asymptomatic individuals who carry a pathogenic TTR variant for the prevention of transthyretin amyloidosis (ATTR) cardiomyopathy or polyneuropathy (ATTR-CM or ATTR-PN, whichever occurs first). The Phase 3 ACT-EARLY trial was initiated at the end of 2024 to evaluate the potential of acoramidis as a first-in-class TTR stabilizer for prevention of ATTRv.

Encaleret
Disease

Autosomal dominant hypocalcemia type 1

Phase 3
genetic source

CASR

estimated prevalence

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

modality

small molecule

molecule icon
Program summary

Encaleret is an investigational small molecule negative allosteric modulator 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 variants in the CASR, which can cause low serum calcium and inappropriately low parathyroid hormone secretion, leading to a range of debilitating symptoms. ADH1 may also lead to excess calcium in urine, a condition called hypercalciuria, which can result in impaired kidney function and kidney stones. Encaleret is being evaluated in a global Phase 3 study for its potential to restore physiologic mineral homeostasis in patients with ADH1. If succesful, we expect encaleret would be the first approved therapy specifically indicated for individuals living 
with ADH1. Encaleret has received Fast Track Designation from the FDA and was granted Orphan Drug Designation in the US, EU, and Japan.

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Disease

Chronic hypoparathyroidism

Phase 2
genetic source

N/A

estimated prevalence

200,000 (US & EU)

modality

small molecule

molecule icon
Program summary

Encaleret is an investigational small molecule negative allosteric modulator of the calcium-sensing receptor (CaSR) being studied in disorders of calcium homeostasis, including chronic hypoparathyroidism. Chronic hypoparathyroidism is characterized by inadequate or impaired parathyroid hormone secretion causing low serum calcium and can lead to a range of debilitating symptoms. Hypoparathyroidism is also associated with excess calcium in urine, a condition called hypercalciuria, and increased phosphate in the blood. Most cases of hypoparathyroidism occur as a complication of surgery. Encaleret is being evaluated for its potential to be an orally-administered therapy for chronic hypoparathyroidism, acting uniquely on the CaSR to regulate calcium homeostasis. *This is an investigator-initiated study conducted at the NIH and Calcilytix. Calcilytix has a Cooperative Research and Development Agreement (CRADA) in place with NIH.

BBP-418
Disease

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

Phase 3
genetic source

FKRP

estimated prevalence

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

modality

small molecule

molecule icon
Program summary

BBP-418 is an investigational small molecule with the potential to be the first disease-modifying oral therapy intended to saturate the partially functional FKRP enzyme with substrate, to drive increased glycosylation of aDG, and to potentially stabilize or improve muscle function. Based on recent interactions with the US 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 US FDA and Orphan Drug Designation for LGMD from the European Medicines Agency.

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Low-dose infigratinib
Disease

Achondroplasia

Phase 3
genetic source

FGFR

estimated prevalence

55,000 (U.S. & EU)

modality

small molecule

molecule icon
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 is being studied for its 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 US 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.

Disease

Hypochondroplasia

Phase 2
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
Disease

Canavan Disease

Phase 2
genetic source

ASPA

estimated prevalence

1,000 (U.S. & EU)

modality

gene therapy

gene therapy icon
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.

Preclinical programs
Disease

Multiple assets in large genetic 
disease populations

Pre-clinical
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.

Alexis and Jackson, living with ADH1 Read More
Elliot, living with MoCD Type A Read More
Miguel, living with achondroplasia Read More
Tobin, a girl with Canavan disease Read More