BridgeBio Pharma, Inc. products

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 best-in-class potential due to its design to inhibit 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.

BridgeBio is working to create a potential first-ever systemic treatment for dystrophic epidermolysis bullosa (DEB), a disease caused by loss of function of Collagen 7 (C7) protein. PTR-01 is a C7 protein replacement therapy designed to restore the missing collagen protein (C7) at the root of the disease. Mutations in the gene that encodes C7 enable the epidermis to separate from the dermis. Even minor friction or trauma can cause debilitating blistering, tearing and scarring of the skin, along with severe pain and itching. The disease can also affect the lining of the mouth, esophagus, eye, anus and vagina. BridgeBio completed a Phase 2 clinical trial evaluating safety and tolerability of PTR-01, as well as clinical proof of concept, and has initiated a Phase 2 extension study.

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.

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 initiation of Phase 3 is planned in 2022.

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.