NOVARTIS

Ionis Pharmaceuticals' shares rose 5% after FDA approved its RNA-targeted therapy, Tryngolza, for familial chylomicronemia syndrome (FCS), marking the first treatment for FCS in the U.S. The approval is supported by phase III BALANCE study results showing significant triglyceride-lowering and reduced acute pancreatitis attacks. Tryngolza is Ionis' first wholly-owned drug and independent commercial launch, expected by year-end. The company is also evaluating Tryngolza for severe hypertriglyceridemia and has collaborations with major drugmakers for various treatments.

The Pediatric Clinical Trials Market is projected to grow from US$ 20.1 Billion in 2023 to US$ 33.0 Billion by 2033, with a CAGR of 5.0%. North America led in 2023 with a 39.10% share. Challenges include ethical concerns, limited recruitment, and high costs. Innovations like adaptive trials and digital technologies are enhancing efficiency.

Roche's experimental Parkinson’s drug, prasinezumab, failed another key trial but shows potential in levodopa users, prompting further exploration. Despite mixed results, Roche plans continued studies and data evaluation.

DelveInsight's 'Pompe Disease Pipeline Insights, 2024' covers 15+ companies and 20+ pipeline drugs, including clinical and nonclinical stage products, with insights on product type, stage, route of administration, and molecule type. Key companies include Spark Therapeutics, Amicus Therapeutics, and Novartis AG. Report highlights recent clinical trial results and emerging therapies.

Novartis plans to close MorphoSys' U.S. and German sites, affecting 330 jobs, due to R&D priorities and pelabresib approval delays. Despite safety concerns, Novartis acquired MorphoSys for $2.9B to access pelabresib, now delayed until at least 2027 for regulatory submission. Novartis has been restructuring, including a 2022 major restructuring and shutting a San Diego gene therapy facility.

Roche and Prothena's prasinezumab, targeting alpha-synuclein for Parkinson's, missed primary endpoint in phase 2b PADOVA trial but showed numerical delay in motor progression and positive trends on secondary endpoints. Roche plans further exploration and collaboration with Parkinson's community.

Rising ACR20 response rates in placebo arms of rheumatoid arthritis trials may be linked to global recruitment shifts, with recent trials including more patients from less affluent countries.

The 'Macular Edema - Global Clinical Trials Review, 2024' report by ResearchAndMarkets.com offers an overview of global clinical trials on Macular Edema, including trial numbers, enrollment, regions, countries, phases, statuses, and sponsor types. It highlights prominent drugs and companies involved, and provides enrollment trends and recent news.

David Epstein, after selling Seagen to Pfizer, joined Francesco de Rubertis' startup Ottimo Pharma, developing a cancer drug targeting PD-1 and VEGF pathways. Ottimo raised $140 million, aiming to outperform Keytruda and other immunotherapies, with a unique bifunctional antibody design.

Nano-biotechnology revolutionizes healthcare with precise diagnostics, advanced drug delivery, and innovative therapies. The market, valued at USD 157.5 billion in 2023, is projected to grow at a CAGR of 9.3% to surpass USD 421.5 billion by 2034, driven by chronic disease management and innovations in personalized medicine and AI integration.