Jakafi (ruxolitinib)

These novel time-dependent transfusion burden analyses demonstrate that momelotinib is associated with anemia-related benefits in most patients and greater transfusion burden reduction versus comparators.

P=N/A, N=28, Not yet recruiting, The First Affiliated Hospital, School of Medicine, Zhejiang University; The First Affiliated Hospital, College of Medicine, Zhejiang University

P2, N=82, Recruiting, Memorial Sloan Kettering Cancer Center | Trial completion date: Nov 2024 --> Nov 2025 | Trial primary completion date: Nov 2024 --> Nov 2025

P2, N=102, Recruiting, Institute of Hematology & Blood Diseases Hospital, China | Trial primary completion date: Jun 2024 --> Oct 2024

P2, N=40, Recruiting, Institute of Hematology & Blood Diseases Hospital, China | Trial primary completion date: Jun 2024 --> Oct 2024

P2, N=41, Recruiting, H. Lee Moffitt Cancer Center and Research Institute | Trial completion date: Jan 2025 --> Dec 2026 | Trial primary completion date: Jan 2025 --> Dec 2025

P2, N=112, Active, not recruiting, Incyte Corporation | Recruiting --> Active, not recruiting | N=200 --> 112

P2, N=16, Terminated, University of California, San Francisco | Completed --> Terminated; Low accrual

P2, N=28, Recruiting, Jonathan Brammer | Trial primary completion date: Dec 2024 --> Dec 2025

P2, N=120, Not yet recruiting, H. Lee Moffitt Cancer Center and Research Institute

Our results revealed the relationships among the ERα, PI3K/AKT, and MAPK signaling pathways in ER+ breast cancer cells. Understanding the interactions among ERα, PI3K-AKT-mTOR, and MAPK could lead to novel combination therapies.

P1/2, N=147, Active, not recruiting, NRG Oncology | Trial completion date: Sep 2022 --> Aug 2025

P2, N=200, Recruiting, Incyte Corporation | Trial completion date: Sep 2024 --> Sep 2027 | Trial primary completion date: Sep 2024 --> Sep 2027

Current therapeutic strategies include JAK inhibitors like Ruxolitinib, which target the JAK-STAT pathway, alongside supportive treatments such as blood transfusions, erythropoiesis-stimulating agents and developing combinatorial approaches...Recently approved JAK inhibitors, including Fedratinib, Pacritinib, and Momelotinib, have expanded the therapeutic landscape...These technologies elucidate the role of the spleen in MF, highlighting its transformation into a site of abnormal hematopoietic activity, fibrotic changes, and immune cell infiltration, functioning as a "tumor surrogate." By profiling diverse cell populations and molecular alterations within the BM and spleen, SRT facilitates a deeper understanding of MF pathophysiology, helping identify novel therapeutic targets and biomarkers. Ultimately, integrating spatial transcriptomics into MF research promises to enhance diagnostic precision and therapeutic innovation, addressing the multifaceted challenges of this disease.

Various therapies have been discovered for psoriasis, including topical treatments, phototherapy, conventional systemic agents such as methotrexate, retinoids and ciclosporine, as well as biologics...Ruxolitinib cream has been investigated in various dermatologic diseases, including atopic dermatitis, vitiligo, psoriasis, and alopecia areata. However, there is limited data on the efficacy of oral ruxolitinib in patients with psoriasis vulgaris. Here, we report a patient diagnosed with myelofibrosis coexisting with psoriasis vulgaris, successfully treated with oral ruxolitinib.

P1/2, N=54, Recruiting, Veronika Bachanova | Trial completion date: Dec 2026 --> Jul 2027 | Trial primary completion date: Sep 2024 --> Jul 2025

P1/2, N=55, Recruiting, Masonic Cancer Center, University of Minnesota | Trial primary completion date: Sep 2024 --> Sep 2025

P2, N=23, Active, not recruiting, Dana-Farber Cancer Institute | Trial completion date: Aug 2024 --> Dec 2024

Notably, pleural effusion and mediastinal mass significantly improved the post-chest cavity infusion of dexamethasone combined with etoposide at the same stage. The patient also underwent allogeneic hematopoietic stem cell transplantation upon achieving bone marrow remission and was followed up until January 2024. Ruxolitinib combined with venetoclax and azacytidine has shown promising efficacy and safety in treating refractory T-ALL harboring the JAK1, JAK3, and STAT5B mutations, providing a novel therapeutic approach for such patients.

P3, N=66, Active, not recruiting, MaaT Pharma | Recruiting --> Active, not recruiting | Trial completion date: Sep 2024 --> Nov 2025 | Trial primary completion date: Sep 2023 --> Nov 2024

P1, N=18, Recruiting, University of Chicago | Not yet recruiting --> Recruiting

P2, N=36, Recruiting, Aaron Logan, MD | Not yet recruiting --> Recruiting | Trial completion date: May 2027 --> Nov 2027 | Initiation date: Feb 2024 --> Oct 2024 | Trial primary completion date: May 2027 --> Nov 2027

P2, N=29, Active, not recruiting, H. Lee Moffitt Cancer Center and Research Institute | Trial completion date: Sep 2024 --> Sep 2025

In patients with myelofibrosis and platelets ≤ 100 × 109/L, achieving SVR on pacritinib, but not BAT (including ruxolitinib), was associated with significant OS benefit, suggesting that pacritinib may offer a unique survival advantage in patients with myelofibrosis and thrombocytopenia who achieve any SVR. Trial Registration: ClinicalTrials.gov number: NCT02055781.

Our candidate approach identifies FOXN3-AS1 as a prognostic indicator of survival in AML with a potential immune-related role. The preliminary observations we made on FOXN3-AS1/DNMT1 crosstalk warrant more in-depth invested immunotherapeutic approaches in AML.

Ruxolitinib reduces the expression of p-JAK2, PD-1, and PD-L1 in JAK2 V617F-positive cells by specifically inhibiting the JAK2 signaling pathway, thereby suppressing the progression of MPNs.

P1, N=20, Recruiting, Baylor Research Institute | Not yet recruiting --> Recruiting | Trial completion date: Dec 2025 --> Dec 2026 | Trial primary completion date: Jun 2025 --> Jun 2026

P2, N=30, Recruiting, M.D. Anderson Cancer Center | Not yet recruiting --> Recruiting

Interestingly, pacritinib, but not other JAK inhibitors such as ruxolitinib, upadacitinib, or filgotinib, acted synergistically with lenvatinib in HCC cells. Immunohistochemical analysis of tumor sections revealed that pacritinib reduced Ki67 staining and phosphorylated IRAK1. Our findings suggest that pacritinib may be a promising therapeutic option for the treatment of advanced HCC, particularly in patients who have developed resistance to lenvatinib.

The JAK1/2 inhibitor, ruxolitinib, which increased the uSTAT5:pSTAT5 ratio, had similar effects on murine HSC function: it constrained HSC differentiation and proliferation, promoted HSC maintenance and upregulated transcriptional programs associated with stemness...Our results therefore reveal a previously unrecognized interplay between pSTAT5 and uSTAT5 in the control of HSC function and highlight JAK inhibition as a potential strategy for enhancing HSC function during ex vivo culture. Increased levels of uSTAT5 may also contribute to the failure of JAK inhibitors to eradicate myeloproliferative neoplasms.

Currently, ruxolitinib is the only FDA-approved medication for vitiligo; however, it carries a black box warning for serious adverse effects, including infections, malignancy, and major cardiovascular events, limiting its use...Although limited by the number of clinical studies, this review underscores the potential of using AhR agonists, such as tapinarof, as a transformative approach to vitiligo management. Future clinical trials are necessary to evaluate the safety, efficacy, and long-term outcomes of AhR agonists.

This unique system allowed the authors to identify a link between IDH2 and TET2 CH mutations, histone demethylases, and altered cytokine signaling, which enabled targeting by ruxolitinib leading to the elimination of CH clones, offering a possible path for preventing the development of malignancy. See related article by Waarts et al., p. 1860.

P3, N=252, Active, not recruiting, Incyte Corporation | Trial completion date: Jul 2024 --> Nov 2024

Ruxolitinib and fedratinib can cause myelosuppression and are recommended for patients with myeloproliferative MF. Approval of 2 less myelosuppressive JAKi, pacritinib and momelotinib, provided essential treatment options for patients with severe thrombocytopenia and anemia, respectively...Judicious treatment decisions of JAKi can be made with in-depth understanding of the pivotal clinical trials on JAKi and their therapeutic attributes and should be guided by the dominant clinical manifestations and the type/degree of cytopenia(s). This article reviews our clinical approach to treatment with JAKi and their sequencing in MF patients by presenting 3 clinical vignettes.

Western blotting analysis showed that the JAK1/2-specific inhibitor ruxolitinib could rescue alterations induced by IGFBP7 overexpression in GC cells...This study emphasizes the clinical relevance of IGFBP7 in GC and its influence on cell proliferation and migration via the JAK/STAT signalling pathway. This study also highlights the regulation of IGFBP7 in GC by DNA and m6A RNA methylation.

P2, N=1, Terminated, Shanghai General Hospital, Shanghai Jiao Tong University School of Medicine | N=30 --> 1 | Trial completion date: Oct 2021 --> Sep 2024 | Unknown status --> Terminated | Trial primary completion date: Oct 2021 --> Sep 2024; Participants recruitment failure

P2, N=56, Recruiting, Medical College of Wisconsin | Trial completion date: Dec 2025 --> Sep 2025 | Trial primary completion date: Dec 2025 --> Sep 2025

P2, N=230, Recruiting, Ryvu Therapeutics SA | Not yet recruiting --> Recruiting

P2, N=30, Not yet recruiting, M.D. Anderson Cancer Center

In order to potentially increase clinical benefit for patients with MF, several novel agents are being partnered with ruxolitinib (RUX) with the ongoing hypothesis to augment greater measures of MF disease modification. The novel agents are either 'added-on' to RUX or as a combo in JAKi naïve patients. Also, the mutant-targeting era of therapies is now beginning with novel CALR-mutated, novel JAK2 V617F mutation-specific and type II JAK2i in the initial stages of drug development, representing a new approach to treatment.

P1/2, N=39, Not yet recruiting, University of Michigan Rogel Cancer Center

P3, N=572, Not yet recruiting, Incyte Corporation