NOVARTIS

Two new drugs, an siRNA and an oral agent, reduced Lp(a) by 80% in phase II studies. Zerlasiran, the siRNA, lowered Lp(a) by 85% over 36 weeks, while muvalaplin, the oral agent, reduced Lp(a) by 70% with a traditional assay and over 85% with an intact assay. Both therapies offer potential for treating cardiovascular disease, with muvalaplin providing an oral alternative to injectables.

Muvalaplin, an oral drug, significantly reduced lipoprotein(a) (Lp[a]) by 70% in a traditional blood test and 85.5% in a new test, according to a phase 2 study presented at the 2024 AHA Scientific Sessions. The drug, developed by Eli Lilly, targets Lp(a), a genetically driven variant of LDL cholesterol linked to cardiovascular disease, with no current treatments. Muvalaplin disrupts the bonding of apo(a) to apoB, preventing Lp(a) formation, and also reduced oxidized phospholipids. The study involved doses of 10 mg, 60 mg, and 240 mg over 12 weeks, with 97% of participants achieving Lp(a) levels below 125 nmol/L. Phase 3 trials and cardiovascular outcomes trials are next steps before market potential.

Novartis' NVP-DFV890, an NLRP3 inhibitor with a unique sulfonimidamide motif, shows reduced hydrolysis and promising PK in humans. It is advancing in Ph. II trials for coronary heart disease and knee osteoarthritis, with discovery and preclinical data presented by Angela Mackay at EFMC-ISMC 2024.

Syndax Pharmaceuticals wins FDA approval for Revuforj, a leukemia treatment; Cybin's psilocin-based drug shows efficacy in treating depression; Neurogene halts high-dose gene therapy for Rett syndrome due to safety concerns; Eli Lilly's Zepbound reduces heart failure-related hospitalizations in trial.

Vera Therapeutics appoints Jason S. Carter as Chief Legal Officer, enhancing its team for the pivotal ORIGIN 3 trial of atacicept in IgAN, with topline results expected in Q2 2025 and a planned BLA submission to the FDA.

Medicus Pharma Ltd appoints Faisal Mehmud, MD as Chief Medical Officer and promotes Edward Brennan, MD, FACS to Chief Scientific Officer & Head of R&D, focusing on expanding drug development pipeline and commercializing novel non-invasive treatments for basal cell carcinoma.

The Global Cell and Gene Therapy Market is projected to reach USD 78 Billion by 2032, driven by regulatory approvals, technological innovations, and personalized medicine. North America leads with a 49.6% market share. Challenges include high costs and complex manufacturing. Recent developments include Novartis AG's FDA approval for KYMRIAH and Biogen Inc.'s collaboration with Scribe Therapeutics. The market is set for extensive growth, supported by strategic partnerships and ongoing R&D.

Alzheimer’s disease therapeutics market includes symptomatic treatments (cholinesterase inhibitors, NMDA receptor antagonists) and disease-modifying therapies (anti-amyloid monoclonal antibodies). Market growth driven by aging population, diagnostic advancements, and R&D investments. Market expected to grow from USD 5.5 billion in 2023 to USD 30.8 billion by 2033 at 18.8% CAGR. Major companies include Biogen, Merck, GSK, Novartis, Johnson & Johnson, Bristol Myers Squibb, Sanofi, Pfizer, Teva Pharmaceutical, and AbbVie, each with strategic initiatives and collaborations in Alzheimer’s research.