Besremi (ropeginterferon alfa-2b-njft)

Its favorable efficacy-toxicity balance and convenient dosing support long-term use, particularly in younger or treatment-naïve patients. Future research should refine patient selection, explore predictive biomarkers, and define its role among disease-modifying agents capable of transforming PV into a chronic, potentially controllable disorder.

The most common adverse events were elevated liver enzymes (AST: 0.28; ALT: 0.32), influenza-like illness (0.11), and anemia (0.09), with unresolved heterogeneity in all outcomes.Ropeginterferon alfa-2b shows promising efficacy in achieving hematological and molecular responses in patients with PV. However, notable heterogeneity and safety concerns, particularly liver-related adverse effects, warrant further investigation in large-scale trials.

Our findings suggest that ropeginterferon alfa-2b could be considered as a second-line treatment option for patients with essential thrombocythaemia and leukocytosis.

The Janus kinase (JAK)/signal transducers and activators of transcription (STAT) pathway has recently been shown to play an important role in the pathogenesis of inflammation in TAFRO syndrome, and inhibitors of the JAK/STAT pathway may be effective as therapeutic agents for TAFRO syndrome. We herein report the successful treatment using combination therapy with ruxolitinib and ropeginterferon alfa-2b of a case of TAFRO-like syndrome with a long history of polycythemia vera with JAK2 V617F refractory to several treatments.

P2, N=11, Terminated, Mayo Clinic | Completed --> Terminated; Study drug became commercially available

Key trials include the phase 2 LSD1 inhibitor bomedemstat trial showing significant platelet-count reduction and mutation-burden improvement the phase 3 SURPASS-ET trial comparing ropeginterferon alfa-2b versus anagrelide, ongoing investigations of JAK - STAT pathway modulators, and emerging data on the anti-calreticulin (CALR) monoclonal antibody INCA033989, which selectively targets mutCALR progenitors to suppress malignant hematopoiesis while sparing normal hematopoiesis. Drawing on recent clinical trials, expert consensus, and emerging data presented at hematology meetings (2018-2025), we highlight established cytoreductive strategies - hydroxyurea, interferon-α (including pegylated formulations), and anagrelide - and evaluate emerging targeted agents. Future advances in ET management hinge on integrating molecular risk stratification into treatment algorithms, optimizing combination regimens to deepen molecular remissions, and prioritizing agents with favorable safety profiles. Personalized approaches leveraging allele-burden dynamics and symptom-control metrics are likely to define the next era of ET pharmacotherapy.

P1, N=38, Recruiting, H. Lee Moffitt Cancer Center and Research Institute | Initiation date: Dec 2025 --> Aug 2025

Most effects of IFN-α on Jak2V617F cells were preserved in Jak2V617F;Stat1 -/- mice but to a moderate degree compared with Jak2V617F mice. Our study reveals essential roles of TYK2 for the preferential suppressive effect of IFN-α on Jak2V617F progenitors and HSCs.

P1, N=38, Recruiting, H. Lee Moffitt Cancer Center and Research Institute | Initiation date: Jul 2025 --> Dec 2025

P2, N=91, Active, not recruiting, PharmaEssentia | Trial primary completion date: Mar 2027 --> May 2025

P3, N=111, Active, not recruiting, PharmaEssentia | Trial completion date: Jul 2025 --> Jul 2027 | Trial primary completion date: Dec 2024 --> Jun 2025

P3, N=174, Active, not recruiting, PharmaEssentia | Trial completion date: Dec 2025 --> Aug 2029