Acoramidis

FDA reviews 5 drugs with PDUFA dates in Nov 2024: DFD-29 for rosacea, eladocagene exuparvovec for AADC deficiency, inavolisib for PIK3CA-mutated HR-positive breast cancer, acoramidis for transthyretin amyloid cardiomyopathy, and zanidatamab for HER2-amplified biliary tract cancer.

The drug and biotech industry thrives on M&A, new drug launches, and pipeline successes, with obesity drugs from Novo Nordisk and Eli Lilly leading. Large drugmakers like Eli Lilly, AstraZeneca, Pfizer, Sanofi, and Bayer are profitable and innovative, focusing on areas like weight loss, inflammation, and Alzheimer’s. Despite headwinds such as pipeline setbacks and macroeconomic uncertainty, investment in drug/biotech stocks has surged. The Zacks Large Cap Pharmaceuticals industry ranks in the top 19% of Zacks industries, outperforming the Zacks Medical Sector but slightly underperforming the S&P 500.

Highly anticipated FDA approvals by year-end include acoramidis for ATTR-CM, olezarsen for FCS, sotagliflozin for type 1 diabetes, oral sulopenem for uUTI, and DFD-29 for rosacea.

BridgeBio Pharma to present 42-month outcomes data from ATTRibute-CM study at AHA Scientific Sessions in Chicago, Nov 16-18, 2024, including a featured science oral presentation on acoramidis' effects in ATTR-CM.

Transthyretin amyloidosis cardiomyopathy drug development, including Pfizer's Vyndamax and BridgeBio's acoramidis, is a major focus. FDA decisions on acoramidis, AstraZeneca's dato-dxd for lung cancer, Vertex's vanza triple for cystic fibrosis, PTC's Upstaza for AADC deficiency, and Bristol Myers' subcutaneous Opdivo for solid tumors are imminent, with significant market implications.

H.C. Wainwright's Ram Selvaraju reiterates Buy rating on BridgeBio Pharma (BBIO) with a $43.00 price target, citing promising clinical data and upcoming regulatory milestones for its drug candidates, including acoramidis for TTR cardiomyopathy. The analyst highlights BridgeBio's diverse pipeline and strategic positioning of assets, anticipating significant data readouts and market opportunities.

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.

The drug and biotech sector has outperformed the broader market, driven by new drug launches, pipeline successes, and increased M&A activity. Eli Lilly's obesity drugs have been particularly successful. Despite headwinds like pipeline setbacks and economic uncertainty, the sector has seen a surge in investments. Five large drugmakers—Pfizer, Eli Lilly, AstraZeneca, Bayer, and Sanofi—are recommended for portfolio growth, with strong financials and robust pipelines.

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.