Pelabresib anhydrous

Novartis to present over 65 abstracts at ASH and SABCS, including 96-week Scemblix results and 4-year Kisqali analysis, highlighting advancements in hematologic diseases and cancers.

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.

In 2024, smaller deals have dominated pharma M&A, with no single deal surpassing $5 billion. Despite this, M&A remains crucial for refilling pipelines and supporting smaller biotechs. Notable deals include Vertex's $4.9 billion acquisition of Alpine Immune Sciences, Eli Lilly's $3.2 billion acquisition of Morphic, and Gilead's $4.3 billion purchase of CymaBay. Oncology and neuroscience have seen significant activity, with Novartis and AstraZeneca leading in oncology, and Bristol Myers Squibb and Lundbeck in neuroscience.

Pelabresib combined with ruxolitinib significantly reduced splenomegaly and improved anemia in JAKi-naïve myelofibrosis patients, according to the phase 3 MANIFEST-2 trial. The combination therapy showed substantial spleen volume reduction, symptom relief, and anemia management, with manageable adverse effects.

Ryvu Therapeutics reported H1 2024 operating revenues of USD 12.1 million, up USD 4.2 million from H1 2023. As of September 5, 2024, Ryvu's cash position was USD 65.3 million, providing a runway through Q1 2026. The company received the final EUR 6 million tranche from the European Investment Bank and advanced its PRMT5 inhibitor RVU305 for preclinical development, targeting IND/CTA filing in H2 2025. Ryvu is progressing with RVU120 Phase II studies and expects initial data updates at ASH 2024.

The article highlights advancements in myelofibrosis (MF) treatment, focusing on novel therapeutic strategies beyond JAK inhibitors. It discusses the development of drugs like momelotinib, pacritinib, and imetelstat, which address unmet needs such as anemia and thrombocytopenia. These therapies aim to improve spleen and symptom responses, modify disease course, and extend overall survival. The article also emphasizes the importance of combination therapies and the potential for personalized treatment approaches based on patient-specific factors.