Acoramidis

Bayer submitted an MAA to EMA for elinzanetant, seeking approval for treating moderate to severe VMS associated with menopause or adjuvant endocrine therapy. The submission is based on OASIS phase III studies showing elinzanetant's efficacy and safety. The FDA also accepted Bayer's NDA for elinzanetant, and Bayer continues global submissions. Bayer aims to strengthen its pharmaceutical portfolio through new drug approvals.

FDA reviews 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, with PDUFA dates in November 2024.

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.