Pelabresib anhydrous

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.

Novartis to present over 65 abstracts at ASH and SABCS, including 96-week Scemblix Phase III ASC4FIRST results and Kisqali 4-year NATALEE trial analysis. The presentations highlight advancements in hematologic diseases and cancers, emphasizing earlier disease stages and new treatment options.

In a recent program, hematologists Swoboda and Kuykendall discussed current myelofibrosis treatments and potential advancements, focusing on JAK inhibitors and the MANIFEST-2 trial with pelabresib. They emphasized the need for combination therapies and better biomarkers to improve patient outcomes, particularly in spleen volume reduction.

Verona Pharma, a biopharmaceutical company, focuses on respiratory disease treatments, with ensifentrine in Phase 3 trials for COPD, asthma, and cystic fibrosis. Caligan Partners LP, an activist investor, sees potential in Verona's ensifentrine, predicting significant revenue growth and possible indication expansion for non-cystic fibrosis bronchiectasis.