Pipeline

Acoramidis is 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 for the prevention of ATTRv.

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 αDG, and to potentially stabilize or improve muscle function. FORTIFY, the Phase 3 clinical trial of BBP-418, successfully achieved all primary and secondary endpoints of its interim analysis and was well-tolerated with no new or unexpected safety findings observed. BBP-418 potentially represents the first approval of a therapy for any form of LGMD. Additional clinical studies of BBP-418 are planned.

Encaleret is an investigational oral negative allosteric modulator of the calcium-sensing receptor (CaSR) which has the potential to target low calcium levels in the blood (hypocalcemia) and high levels of calcium in the urine (hypercalciuria) by selectively modulating the CaSR. CALIBRATE, the Phase 3 clinical trial of encaleret in ADH1, a genetic form of hypoparathyroidism, successfully achieved all pre-specified primary and key secondary efficacy endpoints. We intend to submit a NDA to the FDA in the first half of 2026, and a Marketing Authorization Application to the EMA to follow. If approved, encaleret would be the first therapy specifically indicated for individuals living with ADH1. We have initiated a registrational clinical trial of encaleret in pediatric ADH1 in the first quarter of 2026.

Encaleret is an investigational oral negative allosteric modulator of the calcium-sensing receptor (CaSR) targeting low calcium levels in the blood (hypocalcemia) and high levels of calcium in the urine (hypercalciuria) by selectively modulating the CaSR. 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 a therapy for chronic hypoparathyroidism, acting on the CaSR to regulate calcium homeostasis. We plan to initiate a Phase 3 study of encaleret in chronic hypoparathyroidism in 2026.

*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.

Infigratinib is an investigational oral therapy designed to inhibit FGFR3 and target skeletal dysplasias, such as achondroplasia, at the source to potentially improve bone growth by decreasing the overactivity of FGFR3. Overactivating FGFR3 pathogenic variants drive downstream MAPK and STAT1 signaling that aberrates growth plate development, thereby causing disproportionate short stature and the potential for serious health complications stemming from cranial and spinal issues. We expect to share topline results of PROPEL 3, a Phase 3 clinical trial studying the efficacy and safety of infigratinib in children with achondroplasia, in early 2026. Both the 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 use in children with achondroplasia. Additionally, we have reached regulatory alignment with the FDA on the clinical development plan for infigratinib in infants and toddlers with achondroplasia from birth to less than 3 years old: this clinical trial is now enrolling.

Infigratinib is an investigational oral therapy designed to inhibit FGFR3 and target skeletal dysplasias, such as hypochondroplasia, at the source to potentially improve bone growth by decreasing the overactivity of FGFR3. Overactivating FGFR3 pathogenic variants drive downstream MAPK and STAT1 signaling that aberrates growth plate development, thereby causing a range of phenotypes that present as disproportionate short stature and other skeletal differences with the potential for medical complications. The first participant in the Phase 2 portion of ACCEL 2/3 in hypochondroplasia was dosed in April 2025 with data expected in the second half of 2026. Currently, no treatments for hypochondroplasia are approved in the U.S.

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.