IONIS PHARMACEUTICALS, INC.

WAINZUA (eplontersen) recommended by CHMP for hATTR-PN treatment in EU, based on NEURO-TTRansform Phase 3 results showing consistent benefits in neuropathy impairment and QoL. If approved, WAINZUA will be the only self-administered monthly treatment for ATTRv-PN in the EU.

Ionis Pharmaceuticals and AstraZeneca's WAINZUA (eplontersen) recommended for EU approval for treating hereditary transthyretin-mediated amyloidosis in adults with stage 1 or 2 polyneuropathy, based on NEURO-TTRansform Phase 3 results showing consistent and sustained benefits in neuropathy impairment and quality of life.

AstraZeneca and Ionis' Wainzua (eplontersen) recommended for approval by EU's CHMP for treating hereditary transthyretin-mediated amyloidosis in adults with stage 1 or 2 polyneuropathy. If approved, Wainzua will be the only self-administered monthly treatment in the EU for ATTRv-PN. The CHMP's opinion is based on the NEURO-TTRansform Phase III trial showing consistent and sustained benefits on serum TTR concentration, neuropathy impairment, and quality of life. Wainzua is a once-monthly silencer designed to reduce TTR production at its source in the liver.

Bio-pharma advances in endocrinology, dermatology, cardiovascular, oncology, rare diseases, pulmonology, gastroenterology, and neurology highlight promising therapies like Eli Lilly’s orforglipron, Amgen’s MariTide, Novo Nordisk’s oral semaglutide, Johnson & Johnson’s JNJ-2113, Sanofi’s amlitelimab, Ionis’s olezarsen, BridgeBio’s acoramidis, NewAmsterdam Pharma’s obicetrapib, Jazz Pharmaceuticals’ zanidatamab, Syndax’s revumenib, Merus’ zenocutuzumab, Johnson & Johnson’s nipocalimab, Novo Nordisk’s Mim8, Solid Biosciences’ SGT-003, Merck’s sotatercept, Johnson & Johnson’s Tremfya, and Crossject’s Zepizure, with analyses including clinical data, revenue forecasts, and expected launch dates.

Tau Inhibitors Clinical Trial Pipeline Analysis shows 25+ key companies expected to transform treatment, with increased funding accelerating advancements in neurodegenerative disease therapies.

David Baker, Demis Hassabis, and John Jumper won the 2023 Nobel Prize in chemistry for computational protein design and AlphaFold development. Alnylam Pharmaceuticals seeks FDA approval for vutrisiran in another form of transthyretin amyloidosis. Johnson & Johnson terminated TAR-200 trial in bladder cancer. Gemma Biotherapeutics partners with Brazil's Fiocruz for rare disease gene therapies. AstraZeneca acquires a preclinical cholesterol-lowering drug from CPSC Pharmaceutical Group. Astellas invests in AviadoBio's experimental gene therapy for frontotemporal dementia.

AstraZeneca entered an exclusive licensing deal with CSPC Pharmaceutical Group to develop a preclinical lipid-lowering drug candidate, YS2302018, aiming to treat dyslipidemia and related cardiometabolic diseases. The deal includes $100 million upfront and up to $1.92 billion in milestones, with CSPC eligible for royalties. YS2302018 is an oral small molecule disruptor of Lp(a), a type of LDL cholesterol linked to higher heart attack and stroke risks. AstraZeneca joins Eli Lilly and others in the Lp(a) drug development arena.

Ionis Pharmaceuticals announced the FDA granted zilganersen Fast Track designation for treating Alexander disease, an ultra-rare neurological disorder. Zilganersen aims to address the underlying cause by reducing excess glial fibrillary acidic protein production. Topline data from the pivotal study is expected in 2025.