Biogen

The HSE's Model of Care for Dementia in Ireland aims to ensure equitable access to services, addressing variability in timely diagnosis and care. Published in May 2023, it includes 37 targets and practice recommendations for assessment, treatment, and support. Despite funding consistency, the HSE recruitment moratorium has delayed planned developments. A dementia registry is essential for service planning, with €200,000 allocated in Budget 2025. The model outlines three levels of assessment, with minimum staffing requirements and maximum waiting times. The roll-out coincides with significant scientific activity on modifiable risk factors and disease-modifying treatments for early Alzheimer’s disease, influencing future updates to the model.

Alzheimer's disease research saw a decade of stagnation until FDA approved aducanumab in 2021. Since then, therapies like donanemab and lecanemab have shown promise. NKGen's SNK01, an NK cell therapy, and Longeveron's Lomecel-B, an MSC therapy, are in phase 2 trials, demonstrating efficacy and safety. Other novel therapies, including Biogen's BIIB080 and AAV2-BDNF gene therapy, are also in development.

Scholar Rock's apitegromab, a muscle-preserving therapy for spinal muscular atrophy, succeeded in a Phase 3 trial, showing significant motor function improvement when combined with standard SMA therapies. The company plans to seek approvals in the U.S. and Europe early next year. The drug's success has implications for ongoing research into muscle preservation and potential treatments for obesity.

FDA placed a clinical hold on Kezar Life Sciences' zetomipzomib development for lupus nephritis after 4 patients died in Phase IIb PALIZADE trial. Kezar observed common symptoms and proximity to dosing in the deaths and other serious adverse events, leading to voluntary suspension of enrollment and dosing. The company is investigating potential risk mitigation strategies and expects a formal FDA letter. Kezar continues zetomipzomib development for autoimmune hepatitis unaffected by the hold.

The immunoglobulin market, valued at US$ 13.36 billion in 2023, is projected to reach US$ 28.70 billion by 2034, driven by its use in treating immunodeficiency and other applications. Key growth factors include immunodeficiency disorders, infections, and thrombocytopenia. North America leads with a 46% share, while Asia-Pacific is expected to grow fastest due to increased health spending and prevalence of autoimmune diseases. Recent advancements include subcutaneous injection therapies and acquisitions by major pharmaceutical companies.

Sanofi sells global rights to Enjaymo to Recordati for $825M upfront, with potential $250M more if sales goals are met. Enjaymo, a treatment for cold agglutinin disease, adds to Recordati's rare disease portfolio, expected to generate €150M+ in 2025.

CMS’s former CMO, Lee Fleisher, says Alzheimer’s mAb policies won’t set precedent for accelerated approval, noting access restrictions were based on unusual circumstances. He emphasized these policies shouldn’t signal routine Medicare access limits for accelerated approvals, highlighting FDA’s broader approval labels and trial diversity issues as factors influencing CMS’s decision.

The global immunomodulators market is projected to grow from $317.06 billion in 2024 to $1,237.66 billion by 2034, driven by their use in treating autoimmune diseases, cancer, preventing infections, and managing organ transplant rejection. Key factors include advancements in precision medicine, regional growth in Asia-Pacific, and recent acquisitions and partnerships in the sector.

FDA approves 420 mg strength of Hercessi (trastuzumab-strf) biosimilar; UnitedHealth to remove Humira from preferred drug lists in favor of biosimilars; Evernorth offers interchangeable ustekinumab biosimilar at no cost; Samsung Bioepis receives positive opinion for aflibercept biosimilar.

Remedy Pharmaceuticals' CIRARA, previously scrapped by Biogen, showed 78% of patients treated with CIRARA and endovascular thrombectomy (EVT) walked independently at 12 months post-large hemispheric infarction (LHI), compared to 51% in the placebo group. Remedy plans a Phase III confirmatory trial.