ASTELLAS PHARMA

Ferhina S. Ali, MD, MPH, discusses managing geographic atrophy (GA) by considering treatment timing, imaging, phase 3 trial data, and patient adherence. Ali uses an individual treatment approach, focusing on the eye with better vision in bilateral GA cases. She administers GA treatments pegcetacoplan and avacincaptad pegol every 4-6 weeks, guided by trial extension data and patient motivation. Ali emphasizes the importance of monitoring for choroidal neovascularization and progressive vision loss, and ongoing patient education.

Young biotechs should reduce risk, differentiate products, and focus on drugs over platforms to succeed, according to a BioPharma Dive panel. Key strategies include accelerating drug development, leveraging genetic insights, and ensuring clear communication with investors.

FDA approved zolbetuximab-clzb (Vylov) plus chemotherapy for first-line treatment of HER2-negative, CLDN18.2-positive gastric or gastroesophageal junction adenocarcinoma, based on SPOTLIGHT and GLOW trials. Dr. Jaffer A. Ajani discussed clinical use considerations, including adverse effects and CLDN18.2 as a biomarker.

Kronos Bio discontinues istisociclib development due to safety concerns, plans strategic alternatives to maximize shareholder value, and explores partnerships for preclinical assets KB-9558 and KB-7898.

Pharma companies are using unconventional marketing strategies, such as Astellas' Veozah Super Bowl ad, Reckitt Benckiser's Mr. Mucus Tinder profile, and Genentech's SMA musical, to promote their drugs and raise disease awareness.

Japan is leveraging sovereign AI to enhance healthcare, addressing a projected shortage of 500,000 healthcare workers by 2024. AI tools, trained on country-specific data and local infrastructure, are deployed in drug discovery, genomic medicine, healthcare imaging, and robotics. Key technologies include NVIDIA BioNeMo for drug discovery, NVIDIA MONAI for medical imaging, NVIDIA Parabricks for genomics, and NVIDIA Holoscan for healthcare robotics. These innovations are showcased at the NVIDIA AI Summit Japan, highlighting real-time AI applications in surgery and radiology, and digital health systems for patient care.

Zolbetuximab, a CLDN18.2-targeted IgG1 monoclonal antibody, combined with frontline chemotherapy, extended PFS and OS in HER2-negative, locally advanced unresectable or metastatic gastric or GEJ adenocarcinoma patients positive for CLDN18.2, according to phase 3 SPOTLIGHT and GLOW trials. The FDA approved zolbetuximab in October 2024 for first-line treatment of these patients. Median PFS was 9.2 months with zolbetuximab vs 8.2 months with placebo, and median OS was 16.4 months vs 13.7 months, respectively. Zolbetuximab was also approved in Japan and Europe in 2024.

The neuroscience market, valued at USD 35.3 billion in 2023, is projected to reach USD 50.2 billion by 2032, growing at a CAGR of 4.0%. This expansion is driven by increasing awareness of neurological disorders, advancements in medical technology, and rising healthcare spending. Key technologies like Brain-Computer Interfaces (BCIs), AI, and machine learning are transforming diagnostics and treatments. Notable initiatives such as the BRAIN Initiative and China’s Brain Project are also contributing to market growth.

FDA considers removing oral phenylephrine from OTC guidelines due to lack of efficacy, not safety concerns. Public comments invited until May 7, 2025, before final decision. Phenylephrine is in many nasal decongestants like Sudafed and Mucinex. FDA's proposal does not affect nasal spray decongestants.

The Patient Recruitment for Rare Disease Trials Summit tackles challenges like geographical dispersion and low awareness by exploring innovative strategies to efficiently identify and engage eligible participants, featuring actionable insights, knowledge on patient feedback integration, best practices for data sharing, and fair patient reimbursement discussions.