Pelabresib anhydrous

JAK inhibitors, including ruxolitinib, momelotinib, pacritinib, and fedratinib, are key in myelofibrosis treatment, each with unique benefits and side effects. Momelotinib, approved in 2023, is notable for addressing anemia, spleen size, and symptoms. Combination therapies and new agents like CALR-directed antibodies are under investigation, offering potential for improved disease management. Prognostic models consider genetic and clinical features to guide treatment decisions.

Ruxolitinib, approved in 2011 for myelofibrosis (MF), is key in treating MF and polycythemia vera (PV). Despite its benefits, some patients don't respond or stop due to mutations or progression. Recent studies at ASH 2024 explored combinations like pelabresib, bomedemstat, and elritercept with ruxolitinib, showing promise in improving symptoms, spleen volume reduction, and addressing cytopenias in MF patients.

The Essential Thrombocythemia (ET) market is set to grow significantly by 2034, driven by increasing disease prevalence and awareness. Key companies like Merck and PharmaEssentia are advancing treatments such as Bomedemstat and Ropeginterferon alfa-2b. ET, a rare blood disorder, is diagnosed through blood tests and genetic mutations, with treatments aiming to reduce thrombosis or bleeding risks.

Orna Therapeutics signs a $4.35B deal with Vertex for LNP tech. Avenzo and Duality partner on a cancer ADC, potentially worth $1.15B. Hoth's phase IIa data boosts stock. Novartis closes Morphosys, laying off 330. China approves first MSC therapy. Dimerix licenses kidney drug to Fuso for ¥10.5B. FDA clarifies confirmatory trial timing. Athira settles research misconduct for $4M. BioWorld recaps 2024 trends.

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.

JAK inhibitor monotherapy successes in myelofibrosis led to development of combination regimens aiming to increase spleen volume reduction (SVR) and decrease symptom burden. Momelotinib, approved by the FDA in 2023 for myelofibrosis with anemia, showed noninferiority to ruxolitinib in SVR but not in total symptom score (TSS) reduction. Clinical data from SIMPLIFY-1 and MOMENTUM trials supported its use, particularly in patients with anemia. Momelotinib offers advantages over older JAK inhibitors by improving anemia without exacerbating thrombocytopenia. Future therapeutic targets and combination strategies with JAK inhibitors are promising, focusing on comprehensive disease control and patient-specific benefits.

The MANIFEST-2 trial (NCT04603495) showed pelabresib (CPI-0610; Novartis) plus ruxolitinib (Jakafi; Incyte Corp) improved spleen volume, total symptom score (TSS), anemia, and bone marrow fibrosis (BMF) in JAK inhibitor-naïve myelofibrosis (MF) patients compared to ruxolitinib and placebo. At week 24, 65.9% achieved SVR35, and at week 48, 56.5% achieved SVR35 with pelabresib and ruxolitinib, versus 37.5% with placebo and ruxolitinib. The data will be presented at the 66th ASH Annual Meeting and Exposition on December 8, 2024.

Novartis presents over 65 abstracts at ASH and SABCS, highlighting new data on Scemblix, Kisqali, and other treatments, emphasizing commitment to cancer and blood disorder patients. The company also showcases personal breast cancer stories at SABCS, aiming to uplift and unite the community.

Novartis to present over 65 abstracts at ASH and SABCS, including 96-week Scemblix results and Kisqali 4-year analysis. Data highlights commitment to cancer and blood disorder treatments.