Triple-Negative Myelofibrosis: Disease Features, Response to Treatment and Outcomes

Luis E. Aguirre, Akriti Jain, Somedeb Ball, Najla Al Ali, Virginia O. Volpe, Sara Tinsley-Vance, David Sallman, Kendra Sweet, Jeffrey Lancet, Eric Padrom, Seongseok Yun, Andrew Kuykendall, Rami Komrokji Abstract Background Myelofibrosis is the most aggressive subtype among classical BCR::ABL1 negative myeloproliferative neoplasms. About 90% of cases are driven by constitutive activation of 1 of 3 genes […]

SHP2 inhibition displays efficacy as a monotherapy and in combination with JAK2 inhibition in preclinical models of myeloproliferative neoplasms

Garima Pandey 1, Lucia Mazzacurati 1, Tegan M Rowsell 1, Nathan P Horvat 2, Narmin E Amin 1, Guolin Zhang 3, Afua A Akuffo 2, Christelle M Colin-Leitzinger 2, Eric B Haura 3, Andrew T Kuykendall 4, Ling Zhang 5, Pearlie K Epling-Burnette 2, Gary W Reuther 1 4 Abstract Myeloproliferative neoplasms (MPNs), including polycythemia vera, essential thrombocytosis, and primary myelofibrosis, are clonal hematopoietic neoplasms driven by mutationally activated signaling by the JAK2 tyrosine kinase. Although JAK2 inhibitors can improve MPN […]

Combination Therapies and New Research Drive Progress in Myelofibrosis

March 7, 2024 Jordyn Sava 2023 brought a wave of positive developments for patients with myeloproliferative neoplasms (MPNs), particularly myelofibrosis. According to Raajit K. Rampal, MD, PhD, one study of particular interest was the phase 3 MANIFEST-2 trial (NCT04603495) of ruxolitinib (Jakafi) with pelabresib (CPI-0610).1  This study, in addition to the TRANSFORM-1 trial (NCT04472598), showed […]

Ryvu Therapeutics to Present Preclinical Data on RVU120 and Synthetic Lethality Programs at the 2024 AACR Annual Meeting

Published: Mar 06, 2024 Updated preclinical data will be presented from Ryvu’s synthetic lethality pipeline, including PRMT5 inhibitors in MTAP-Deficient cancers, WRN inhibitors for the treatment of microsatellite unstable (MSI-H) tumors, and Ryvu’s cutting-edge synthetic lethality platform based on primary cancer cells. Poster presentation to highlight the synergistic effects of RVU120 in combination with ruxolitinib […]

Study Reveals Real-World Characteristics of Patients With Secondary Myelofibrosis

February 6, 2024 Jonathan Goodman, MPhil A study published in the Annals of Hematology has highlighted the characteristics of patients with post-essential thrombocythemia or post-polycythemia vera myelofibrosis (PET-MF and PPV-MF, respectively) treated during the ruxolitinib era. Although the characteristics of patients with primary MF are well-established, less research has evaluated real-world data from patients with PET-MF or […]

Hobbs Highlights Key Research in Hematologic Malignancies at the 2023 ASH Annual Meeting

January 30, 2024 Courtney Flaherty Ruxolitinib (Jakafi)-based combinations continue to demonstrate promising ability to address splenomegaly and a signal toward improvement of tumor-related symptoms in myelofibrosis. As novel targets for development are unearthed and considered for evaluation in combination with standard JAK inhibition, the assessment of other meaningful end points is necessary to confirm the […]

European Commission Approves Momelotinib for Myelofibrosis/Anemia

January 29, 2024 Ariana Pelosci The European Commission granted marketing authorization to momelotinib (Omjjara) for patients with primary myelofibrosis who have disease-related splenomegaly or moderate to severe anemia, according to a press release from GSK.1 This indication also covers patients with post polycythemia vera myelofibrosis or post essential thrombocythemia myelofibrosis who are JAK inhibitor naïve […]

GB2064 Displays Preliminary Efficacy, Tolerability in Myelofibrosis

January 16, 2024 Kyle Doherty The potential first-in-class, oral, lysyl oxidase-like 2 (LOXL2) inhibitor GB2064 displayed efficacy with a generally acceptable tolerability profile in the treatment of patients with myelofibrosis, according to topline findings from the phase 2a MYLOX-1 trial (NCT04679870).1 Among evaluable patients with myelofibrosis who were treated with GB2064 monotherapy for a minimum […]

Karyopharm

 

Karyopharm Therapeutics is an innovation-driven pharmaceutical company focused on the discovery, development, and commercialization of medicines with the goal of improving the lives of patients with cancer. Our primary focus is on developing novel drugs that we hope will help treat patients with certain blood cancers or solid tumor malignancies.

Karyopharm Therapeutics is investigating the safety and efficacy of selinexor, a selective inhibitor of nuclear export, in combination with ruxolitinib in treatment-naïve patients with myelofibrosis.

INCA-033989 by Incyte for Myelofibrosis: Likelihood of Approval

January 12, 2024 INCA-033989 is under clinical development by Incyte and currently in Phase I for Myelofibrosis. According to GlobalData, Phase I drugs for Myelofibrosis have an 86% phase transition success rate (PTSR) indication benchmark for progressing into Phase II. GlobalData’s report assesses how INCA-033989’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication […]