BridgeBio Reports Positive Pipeline Progress and Q3 2024 Financial Results
核心洞察
BridgeBio's acoramidis shows positive Phase 3 results for ATTR-CM (搜索), with an FDA PDUFA date set for November 29, 2024, and potential $500M milestone payment upon approval.
The company anticipates three Phase 3 readouts in 2025 and has completed screening for the CALIBRATE trial of encaleret and enrollment for the FORTIFY trial.
Infigratinib receives Breakthrough Therapy Designation for achondroplasia (搜索), highlighting advancements in BridgeBio's pipeline for genetic diseases.
BridgeBio Pharma, Inc. (Nasdaq: BBIO) reported its financial results for the third quarter ended September 30, 2024, alongside updates on its clinical pipeline. The company's lead drug candidate, acoramidis, is under review by the FDA for the treatment of transthyretin (搜索) amyloid cardiomyopathy (ATTR-CM (搜索)), with a PDUFA date set for November 29, 2024. If approved, BridgeBio anticipates receiving a $500 million milestone payment.
Acoramidis for ATTR-CM
Acoramidis, a near-complete transthyretin (搜索) (TTR) stabilizer, has demonstrated positive Phase 3 results in the ATTRibute-CM study. Data presented at the European Society of Cardiology (ESC) 2024 showed that increased serum TTR at Day 28 was correlated with reduced risk of all-cause mortality (ACM), cardiovascular mortality (CVM), and cardiovascular hospitalization (CVH) in ATTR-CM (搜索). A post-hoc analysis of ATTRibute-CM revealed a 42% reduction in composite ACM and recurrent CVH events at 30 months with acoramidis treatment compared to placebo (p=0.0005).
Encaleret for Autosomal Dominant Hypocalcemia Type 1 (ADH1)
BridgeBio is advancing encaleret, a calcium-sensing receptor (CaSR) antagonist, for the treatment of autosomal dominant hypocalcemia type 1 (ADH1 (搜索)). The Phase 3 CALIBRATE trial has completed screening, and the company anticipates completing enrollment in 2024. Proof-of-principle data presented at the American Society for Bone Mineral Research meeting demonstrated normalization of blood and urine calcium in 86% of participants within 5 days of encaleret treatment for post-surgical hypoparathyroidism.
BBP-418 (Ribitol) for Limb-Girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9)
The company has completed enrollment in the FORTIFY Phase 3 registrational study of BBP-418 (ribitol) in individuals with LGMD2I/R9 (搜索), with topline data readout from the interim analysis expected in 2025. The FDA has granted Rare Pediatric Disease Designation for BBP-418, potentially qualifying BridgeBio for a Priority Review Voucher upon approval.
Infigratinib for Achondroplasia and Hypochondroplasia
Infigratinib, an FGFR1-3 (搜索) inhibitor, has received Breakthrough Therapy Designation from the FDA for achondroplasia (搜索). The PROPEL 3 global Phase 3 registrational study in achondroplasia continues to enroll, with study completion anticipated by the end of the year. Additionally, ACCEL 2/3, a global Phase 2/3 study, will evaluate the efficacy and safety of infigratinib in children with hypochondroplasia (搜索).
BBP-812 for Canavan Disease
BridgeBio's BBP-812, an adeno-associated virus (AAV) 9 gene therapy for Canavan disease (搜索), has received Regenerative Medicine Advanced Therapy (RMAT) Designation. New data from the high-dose cohort of the CANaspire Phase 1/2 clinical trial shows progressive improvement in gross motor function. Statistically significant improvements in motor function and milestones were observed at 12 months after treatment with BBP-812 in the low-dose cohort, compared to the natural history of the disease.
Financial Highlights
BridgeBio reported revenue of $2.7 million for the three months ended September 30, 2024, compared to $4.1 million for the same period in the prior year. The net loss attributable to common stockholders was $162.0 million for the quarter, compared to $177.0 million in the prior year. As of September 30, 2024, the company's cash, cash equivalents, and short-term restricted cash totaled $405.7 million.