BBP-812

BridgeBio Pharma, with a $5.3B market cap, focuses on genetic diseases. Its FDA-approved Acoramidis could significantly boost revenue. Analysts rate it a 'Strong Buy', predicting a 150% stock increase to $70, despite a 31.5% YTD drop. The company faces competition and R&D challenges but has a promising pipeline.

TD Cowen maintains Buy rating on BridgeBio Pharma (NASDAQ:) shares due to early approval of Attruby, a drug effective in reducing cardiovascular hospitalizations and deaths, priced 10% lower than competitor taf. A Key Opinion Leader survey indicates over 30% of first-line patients likely to be treated with Attruby, suggesting commercial success. BridgeBio's market capitalization is $5.63 billion, with analysts projecting sales growth, though the company is not yet profitable.

H.C. Wainwright raises BridgeBio Pharma's price target to $49.00 from $43.00, maintaining a Buy rating, following FDA approval of Attruby for ATTR-CM. Attruby, priced at $18,759.12 for a 28-day supply, is expected to generate sales early next year. BridgeBio's market cap is $4.43 billion, with negative operating income of -$516.2 million, highlighting the significance of Attruby's commercialization.

BridgeBio Pharma (BBIO) reported Q3 2024 revenue of $2.7M and a net loss of $162.0M, with a cash position of $405.7M. Acoramidis, their lead drug for ATTR-CM, showed positive Phase 3 results, with an FDA PDUFA date set for November 29, 2024. Upon approval, BBIO expects a $500M milestone payment and $105M in additional regulatory milestones for European and Japanese territories. Three Phase 3 readouts are expected in 2025, with notable pipeline progress including completed screening for CALIBRATE and enrollment for FORTIFY trials, and Breakthrough Therapy Designation for infigratinib in achondroplasia.

BridgeBio presented positive data on gene therapy BBP-812 for Canavan Disease at ESCGT meeting, showing potential to correct the aspartoacylase gene and reduce toxic N-acetyl-aspartate levels. Participants in the CANaspire trial are showing improvements, including walking unassisted. The therapy has received RMAT designation, enabling early FDA interactions for Accelerated Approval. BridgeBio's financial model focuses on rare diseases, balancing smaller and larger rare disease programs. The company emphasizes community engagement and patient input for successful rare disease treatments.

MYR-101, an rAAV gene therapy for Canavan disease, showed >80% reduction in NAA levels and myelin volume increases in 7 patients. Functional improvements were noted, contrary to expected deterioration. The FDA included rAAV-Olig001-ASPA in its START Pilot Program and granted multiple designations. BridgeBio's BBP-812 also received RMAT designation for CD treatment.

BridgeBio Pharma completes enrollment for Phase 3 FORTIFY study of BBP-418, targeting Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9), with topline data expected in 2025. BBP-418 has received Orphan Drug, Fast Track, and Rare Pediatric Disease Designations, and could become the first disease-modifying therapy for LGMD2I/R9 if approved. BridgeBio also reports updates in its drug development pipeline, including Breakthrough Therapy Designation for infigratinib and discontinuation of BBP-631 gene therapy program.

BridgeBio Pharma's infigratinib receives FDA Breakthrough Therapy Designation for treating achondroplasia, with PROPEL 2 trial showing significant height velocity increase. Ongoing PROPEL 3 study aims for completion by year-end. BridgeBio's diverse pipeline includes other genetic diseases, and recent developments include expedited review for BBP-812 gene therapy and discontinuation of BBP-631 program. Analysts maintain positive ratings, highlighting anticipated sales growth despite stock volatility.

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