BridgeBio Pharma, Inc. - BBIO STOCK NEWS

BridgeBio Pharma announced FDA approval of Attruby™ (acoramidis) for treating adults with ATTR-CM to reduce cardiovascular death and hospitalization. The approval is based on the ATTRibute-CM Phase 3 study results, showing a 42% reduction in composite all-cause mortality and recurrent cardiovascular-related hospitalizations at Month 30. Attruby demonstrated rapid benefits within 3 months and achieved a 50% reduction in cardiovascular-related hospitalization events. As the first approved product with near-complete TTR stabilization (≥90%), Attruby preserves native TTR function. BridgeBio will receive a $500 million payment under their royalty funding agreement and has submitted for European approval, expected in 2025.

BridgeBio Pharma (BBIO) presented positive initial outcomes from the ATTRibute-CM open-label extension study of acoramidis in ATTR-CM. Key results show that acoramidis achieved a 36% reduction in All-Cause Mortality at Month 36 and demonstrated the earliest known time to separation in cardiovascular outcomes (3 months). The study showed a 46% reduction in the composite endpoint of mortality and cardiovascular-related hospitalizations at Month 36, increasing to 48% at Month 42. The drug continues to be well-tolerated with no new safety concerns. A New Drug Application is under FDA review with a PDUFA date of November 29, 2024.

BridgeBio Pharma (BBIO) has published positive 18-month results from its Phase 2 PROPEL 2 trial of infigratinib for children with achondroplasia in the New England Journal of Medicine. The study showed statistically significant results with a +2.50cm/year increase in annualized height velocity at Month 18 using a 0.25mg/kg daily oral dose. The treatment demonstrated improved body proportionality and was well-tolerated with no serious adverse events. The drug has received multiple FDA designations, including Breakthrough Therapy Designation. The company's Phase 3 PROPEL 3 study continues enrollment, expected to complete by end of 2024.

BridgeBio Pharma (BBIO) reported Q3 2024 financial results with revenue of $2.7M and a net loss of $162.0M. Cash position ended at $405.7M. The company's lead drug acoramidis showed positive Phase 3 results in ATTR-CM with FDA PDUFA date set for November 29, 2024. Upon approval, BBIO anticipates receiving a $500M milestone payment and $105M in additional regulatory milestones for European and Japanese territories.

Three Phase 3 readouts are expected in 2025. Notable pipeline progress includes completed screening for CALIBRATE Phase 3 trial of encaleret, completed enrollment for FORTIFY Phase 3 trial, and Breakthrough Therapy Designation for infigratinib in achondroplasia.

BridgeBio Pharma (BBIO) announced the publication of a case study in The Journal of Portfolio Management, exploring how portfolio theory impacts biomedical innovation. The study, co-authored by BridgeBio's senior management and MIT professor Andrew Lo, examines the company's unique business model that applies portfolio theory to drug development. Founded in 2015, BridgeBio's approach involves diversifying risk by investing in multiple uncorrelated drug development programs, rather than focusing on a single lead candidate. This strategy aims to increase success probability, create stable returns, and attract broader investment, particularly for early-stage genetic disease research.

BridgeBio Pharma presented positive preliminary data from eleven participants in the CANaspire Phase 1/2 clinical trial of BBP-812, an investigational gene therapy for Canavan disease. The study showed significant motor function improvements and milestone achievements at 12-months post-treatment, contrasting with the natural disease progression observed in their CANinform study.

Key findings include sustained reductions in N-acetylaspartate levels across urine, cerebrospinal fluid, and brain, with urine NAA reduced by 64% in low dose and 73% in high dose cohorts. The therapy demonstrated improved myelination and continued motor function progress, with some children achieving independent sitting and walking. BBP-812 was generally well-tolerated and has received multiple FDA designations including RMAT, Orphan Drug, and Fast Track.

BridgeBio Pharma (Nasdaq: BBIO) announced that outcomes data through 42 months from the open-label extension of ATTRibute-CM, its Phase 3 study of acoramidis in ATTR-CM, will be presented at the 2024 AHA Scientific Sessions. The featured science oral presentation, titled 'Acoramidis Reduces All-Cause Mortality (ACM) and Cardiovascular-Related Hospitalization (CVH): Initial Outcomes from the ATTRibute-CM Open-Label Extension (OLE) Study', will be delivered by Dr. Daniel Judge on November 18.

Additionally, BridgeBio will present three moderated digital posters on ATTR-CM, covering topics such as healthcare costs, baseline risk evolution, and acoramidis' impact on survival. These presentations will take place on November 17. The presentation and posters will be available on BridgeBio's website after the sessions.

BridgeBio Pharma announced the completion of enrollment for its Phase 3 FORTIFY study evaluating BBP-418 in Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). The study exceeded its target enrollment, with topline data expected in 2025. BBP-418 could potentially receive Accelerated Approval in the U.S. based on the biomarker glycosylated alpha-dystroglycan (αDG). If successful, BBP-418 would be the first approved therapy for LGMD2I/R9 in the U.S. The study's primary endpoint is the North Star Assessment for limb-girdle type muscular dystrophies, evaluated at 36 months. BBP-418 has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations from the FDA and Orphan Drug Designation from the EMA. The completion announcement coincided with the 10th annual LGMD Awareness Day on September 30, 2024.

BridgeBio Pharma (Nasdaq: BBIO) has announced that the FDA granted Breakthrough Therapy Designation to oral infigratinib for children with achondroplasia. This designation, based on preliminary clinical evidence from the PROPEL 2 trial, is designed to expedite development and regulatory review. In Cohort 5 of PROPEL 2, infigratinib showed a statistically significant increase in annualized height velocity (AHV) and improved body proportionality. The global Phase 3 study, PROPEL 3, is enrolling on schedule. If approved, infigratinib could become the first-in-class oral therapeutic option for children with achondroplasia, potentially offering families greater control and choice in treatment options.