Pelabresib anhydrous
- Generic Name
- Pelabresib anhydrous
- Drug Type
- Small Molecule
- Chemical Formula
- C20H16ClN3O2
- CAS Number
- 1380087-89-7
- Unique Ingredient Identifier
- U4017GUQ06
New Phase 1 Trial Combines Tagraxofusp and Pacritinib for Treatment-Resistant Myelofibrosis
• A novel phase 1 trial is investigating the combination of tagraxofusp and pacritinib in patients with myelofibrosis who have failed or cannot receive JAK1/2 inhibitor therapy, targeting a significant unmet need in this population.
• The dual-agent approach leverages complementary mechanisms of action, with tagraxofusp targeting CD123 and pacritinib inhibiting specific kinases, potentially improving disease symptoms and reducing splenomegaly in treatment-resistant cases.
• The single-center study aims to enroll 20 patients with primary endpoints focused on spleen volume reduction and symptom improvement, with completion expected by December 2026.
JAK Inhibitors Pipeline Expands with 55+ Drug Candidates as Major Pharma Companies Drive Innovation
• The JAK inhibitors therapeutic landscape shows robust growth with over 50 pharmaceutical companies developing 55+ pipeline candidates across various clinical stages.
• Key drug candidates including Povorcitinib, Zasocitinib, and Ritlecitinib are advancing through late-stage clinical trials for conditions like vitiligo, psoriasis, and ulcerative colitis.
• Recent developments include promising results from Pelabresib-ruxolitinib combination therapy for myelofibrosis and expanded access initiatives by Eli Lilly for baricitinib in Africa.
Updates on Ruxolitinib Combinations for MPNs from ASH 2024
Recent updates from the ASH 2024 meeting highlight advancements in ruxolitinib combinations for treating myeloproliferative neoplasms (MPNs), including myelofibrosis (MF) and polycythemia vera (PV). Key studies discussed include MANIFEST-2, bomedemstat, and RESTORE trials, showing promising results in spleen volume reduction, symptom improvement, and addressing cytopenias.
Novartis to Close MorphoSys Sites, Cut 330 Jobs Amid Pelabresib Development Challenges
• Novartis announces closure of MorphoSys facilities in the US and Germany, affecting 330 jobs, following its $2.9 billion acquisition of the German biotech company.
• Development timeline for pelabresib, a potential myelofibrosis treatment, faces significant delays with regulatory submission pushed back to at least 2027 due to additional data requirements.
• Despite emerging safety concerns and mixed trial results for pelabresib, Novartis maintains commitment to the drug's development while restructuring operations.
Error parsing response
Error occurred while parsing the model response.
Novartis' Kisqali Receives EC Approval for Early Breast Cancer Treatment
• The European Commission (EC) has approved Novartis' Kisqali (ribociclib) in combination with an aromatase inhibitor for adjuvant treatment of HR+/HER2- early breast cancer.
• The approval is based on the Phase III NATALEE trial, demonstrating a 25.1% reduction in the risk of disease recurrence compared to endocrine therapy alone.
• Kisqali is now approved for a broader population of early breast cancer patients in Europe, including those with node-negative disease at high risk of recurrence.
• The decision follows positive recommendations and aligns with recent FDA approvals and NCCN guidelines, reinforcing Kisqali's role in early breast cancer treatment.
Novartis' Scemblix Shows Promising 96-Week Data in First-Line CML Treatment
• Scemblix (asciminib) demonstrates favorable safety and tolerability compared to investigator-selected TKIs in newly diagnosed CML-CP patients.
• The ASC4FIRST Phase III study's 96-week results support Scemblix as a potential first-line treatment option for chronic myeloid leukemia.
• Novartis is presenting over 65 abstracts at ASH and SABCS, highlighting their commitment to cancer and blood disorder research.
Novartis Highlights Extended Scemblix Data in CML and Kisqali Results in Early Breast Cancer at ASH and SABCS
• Scemblix (asciminib) demonstrates favorable safety and tolerability in newly diagnosed chronic myeloid leukemia in chronic phase (CML-CP) patients after 96 weeks, based on the Phase 3 ASC4FIRST study.
• A late-breaking 4-year analysis of Kisqali (ribociclib) from the Phase III NATALEE trial shows distant disease-free survival benefits in key subgroups with HR+/HER2- early breast cancer.
• Novartis is presenting data from over 65 abstracts at the American Society of Hematology (ASH) Annual Meeting and the San Antonio Breast Cancer Symposium (SABCS), showcasing advancements in hematologic diseases and cancers.
Novartis' Scemblix Shows Sustained Efficacy in Chronic Myeloid Leukemia at 96 Weeks
• Scemblix (asciminib) demonstrates favorable safety and tolerability compared to investigator-selected tyrosine kinase inhibitors in newly diagnosed chronic myeloid leukemia.
• Interim results from the Phase 2 ASC2ESCALATE trial show efficacy and safety of asciminib in CML patients after one prior tyrosine kinase inhibitor.
• The Phase 3 ASC4OPT study indicates high efficacy and favorable tolerability of asciminib at different dosages in CML patients previously treated with multiple TKIs.
Novartis Highlights Scemblix and Kisqali Data at ASH and SABCS
• Scemblix demonstrated favorable safety and tolerability in newly diagnosed chronic myeloid leukemia patients in the ASC4FIRST Phase III study.
• Kisqali showed promising distant disease-free survival across key subgroups in HR+/HER2- early breast cancer from the Phase III NATALEE trial.
• Novartis is presenting over 65 abstracts at ASH and SABCS, showcasing advancements in hematologic diseases and cancers.
Myelofibrosis Treatment Landscape Evolves with Novel Therapies and Combination Strategies
• Emerging therapies like luspatercept, nuvisertib, and pelabresib show promise in addressing myelofibrosis symptoms and anemia, potentially improving patient outcomes.
• Combination strategies, particularly those involving JAK inhibitors with agents like pelabresib or navitoclax, aim to enhance spleen volume reduction and symptom control.
• Research focuses on identifying biomarkers and refining endpoints to better assess disease modification and personalize treatment approaches for myelofibrosis patients.
• Novel agents targeting calreticulin and hemojuvelin, along with advancements in JAK inhibitors, offer potential for more targeted and effective myelofibrosis therapies.
Novartis and Apellis Advance C3G Therapies Towards FDA Submission
• Novartis' Fabhalta (iptacopan) demonstrated significant proteinuria reduction in the Phase III APPEAR-C3G trial, meeting its primary endpoint and supporting regulatory submissions.
• Apellis' Empaveli (pegcetacoplan) showed a substantial reduction in proteinuria and C3c deposit clearance in the Phase III VALIANT trial, indicating early efficacy in C3G patients.
• Nephrologists express a strong interest in new C3G therapies that can slow eGFR decline and reduce proteinuria, addressing the urgent need for innovative treatments.
• Spherix data suggests treatment preferences may be influenced by administration route, with Fabhalta's oral administration potentially offering an advantage.
Caligan Partners Takes Stake in Verona Pharma, Citing Ohtuvayre's Potential
• Caligan Partners LP has acquired a stake in Verona Pharma, recognizing the potential value of its drug Ohtuvayre (ensifentrine) for COPD treatment.
• Ohtuvayre, a dual inhibitor of PDE3 and PDE4, has shown promising Phase III data with significant lung function improvement and reduced exacerbations.
• Caligan anticipates substantial revenue potential for Ohtuvayre, especially with possible expansion into non-cystic fibrosis bronchiectasis (NCFB) treatment.
• Caligan's investment is based on Verona's potential to capture a significant share of the COPD market and the possibility of a lucrative acquisition.
Pelabresib Plus Ruxolitinib Shows Significant Benefit in Myelofibrosis Patients
• Pelabresib combined with ruxolitinib significantly reduced splenomegaly in JAK inhibitor-naïve myelofibrosis patients, with 65.9% achieving a ≥35% spleen volume reduction at 24 weeks.
• The combination therapy also demonstrated improvements in total symptom score, with a mean absolute change of -15.99 compared to -14.05 in the placebo arm.
• Hemoglobin response was higher in the pelabresib plus ruxolitinib arm (10.7%) compared to the placebo arm (6.0%), with maintained differences at 48 weeks.
• While anemia and thrombocytopenia were observed as adverse effects, the study underscores the clinical benefit of the combination in treating myelofibrosis.
Ryvu Therapeutics Advances RVU305 PRMT5 Inhibitor to Preclinical Development; Reports Positive Financials and Clinical Trial Updates
• Ryvu Therapeutics is advancing RVU305, a PRMT5 inhibitor, into preclinical development with IND/CTA filing targeted for H2 2025.
• The company's RVU120 Phase II studies are progressing, with initial data expected at the ASH 2024 conference in December.
• Ryvu's cash position reached USD 65.3 million as of September 5, 2024, providing a financial runway through Q1 2026.
• Total operating revenues in H1 2024 increased to USD 12.1 million, a USD 4.2 million increase compared to H1 2023.
ASCO 2024 Highlights: Key Updates in Cancer Therapies
• Bristol Myers Squibb's Opdivo/Yervoy combo demonstrated superior overall survival in first-line unresectable hepatocellular carcinoma compared to Lenvima or Nexavar.
• Sanofi's Sarclisa combination reduced the risk of death or disease progression by 40% in transplant-ineligible multiple myeloma patients.
• AstraZeneca and Daiichi Sankyo's Enhertu showed significant progression-free survival improvement versus chemotherapy in HER2-low breast cancer.
Ryvu Therapeutics to Present Promising RVU120 and Synthetic Lethality Data at Major Oncology Conferences
• Ryvu Therapeutics will showcase clinical data for RVU120, their CDK8/19 inhibitor, demonstrating efficacy in 15 of 29 evaluable AML and HR-MDS patients, including complete remission in a patient with NPM1 and DNMT3A mutations.
• Preclinical studies reveal synergistic potential of RVU120 with venetoclax in AML and with ruxolitinib in myelofibrosis, supporting four planned Phase II trials targeting various hematologic malignancies.
• The company will present advances in its synthetic lethality pipeline, including novel MTA-cooperative PRMT5 inhibitors for MTAP-deleted cancers and WRN inhibitors for microsatellite unstable tumors, expanding their precision oncology portfolio.
Novartis Highlights New Data on Kisqali, Iptacopan, and Scemblix at SABCS and ASH
• Final analysis of the NATALEE trial showed Kisqali (ribociclib) significantly improved invasive disease-free survival in HR+/HER2- early breast cancer patients.
• APPLY-PNH trial data demonstrated iptacopan monotherapy's sustained efficacy and safety in anti-C5-treated PNH patients with persistent anemia over 48 weeks.
• Scemblix (asciminib) showed sustained efficacy and safety after nearly 4 years in CML-CP patients who failed ≥2 prior tyrosine kinase inhibitors.
Drug Development Updates
Stay informed with timely notifications on clinical trials, regulatory changes, and research advancements related to this medication.
© Copyright 2025. All Rights Reserved by MedPath