MedPath

AZD0780

Generic Name
AZD0780

AstraZeneca Exits Neuroscience Research to Focus on Core Therapeutic Areas

• AstraZeneca has officially closed its neuroscience research group, discontinuing programs for Alzheimer's disease, migraine, and pain conditions to redirect resources toward higher-value projects. • The strategic shift allows the pharmaceutical giant to concentrate on its core therapeutic areas including oncology, cardiovascular, respiratory diseases, weight management, and immunology. • Despite recording 10% revenue growth in Q1 2025 with nearly $13.6 billion in sales, AstraZeneca executives emphasized they "cannot be everywhere," stating CNS research is "better managed by other companies."

ACC.25 Highlights Novel Therapies and Strategies in Cardiovascular Care

• The SOUL trial demonstrated that oral semaglutide significantly reduces cardiovascular events in type 2 diabetes patients with atherosclerotic cardiovascular and/or chronic kidney disease. • The RIVAWAR trial investigated the efficacy of rivaroxaban versus warfarin in patients with acute left ventricular thrombus following myocardial infarction. • The FAIR-HF2 trial assessed the impact of ferric carboxymaltose on morbidity and mortality in patients with iron deficiency and chronic heart failure. • The ALPACA Phase 2 trial of Lepodisiran showed promising results for an extended duration small-interfering RNA targeting Lipoprotein(a).

AstraZeneca's Oral PCSK9 Inhibitor Shows Promise in Phase 1 Trial

• AstraZeneca's oral PCSK9 inhibitor, AZD0780, demonstrated a 52% reduction in LDL cholesterol when added to statin therapy in a Phase 1 trial. • The study included treatment-naive participants with hypercholesterolemia, showing a 78% total reduction from baseline in LDL-C levels. • AZD0780's efficacy appears comparable to injectable PCSK9 inhibitors, with the added convenience of oral administration, unaffected by food intake. • AstraZeneca plans to advance AZD0780 into a Phase 2 program, joining MSD in the race to provide oral alternatives to injectable cholesterol-lowering drugs.

2024 Advances in Lipid Management: A Year of Progress and Setbacks

2024 marked significant progress in lipid management, with notable advancements in the treatment of familial chylomicronemia syndrome (FCS) and lipoprotein(a) (Lp(a)) management. However, the year also saw challenges to the 'HDL hypothesis', highlighting the complexity of lipid-related cardiovascular disease prevention. Key developments included FDA approvals of novel therapies and promising results from clinical trials targeting triglycerides and Lp(a).

Verve Therapeutics Advances Pipeline with Focus on Gene Editing for Cardiovascular Disease

• Verve Therapeutics anticipates initial data from the Heart-2 Phase 1b trial of VERVE-102, targeting PCSK9, in Q2 2025, including safety and efficacy data. • VERVE-301 has been nominated as the development candidate targeting the LPA gene, triggering a milestone payment from Eli Lilly, who will fund Phase 1 development. • The Pulse-1 Phase 1b trial for VERVE-201, which targets ANGPTL3, is progressing, with an update expected in the second half of 2025. • Verve's strong financial position, boosted by the Lilly milestone payment, extends the company's cash runway into mid-2027.

Durvalumab Gains Priority Review for Muscle-Invasive Bladder Cancer Treatment

• The FDA has granted priority review to durvalumab (Imfinzi) for muscle-invasive bladder cancer (MIBC), potentially expediting its approval. • Phase 3 NIAGARA trial data supports the application, showing a 32% reduction in disease progression or recurrence with perioperative durvalumab. • The Imfinzi regimen also demonstrated a 25% reduction in the risk of death, offering a significant survival benefit. • Regulatory decisions are anticipated in the second quarter of 2025, with potential implications for MIBC treatment standards.
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