ilginatinib (NS-018) / Nippon Shinyaku - LARVOL DELTA

XPO1 inhibition by selinexor blocks nucleo-cytoplasmic trafficking of multiple members of the NF-kb family in MPN cell lines and primary myelofibrosis cells (ASH 2025) - P3 | "The patient was previously treated with ruxolitinib, fedratinib, and ilginatinib/NS018, with adverse events and lack of response. Selinexor induced G0/G1 cell cycle arrest at 16 hours in HEL cells. Preliminary data showed that exposure of HEL cells to selinexor led to pronounced nuclear sequestration of several NF-κB family members. Cells with nuclear localization of RELA increased from 39.4% in DMSO-treated controls to 93.2% following selinexor treatment."

IO biomarker • Preclinical • Acute Myelogenous Leukemia • Fibrosis • Hematological Disorders • Hematological Malignancies • Immunology • Leukemia • Myelofibrosis • Polycythemia Vera • CD33 • CD34 • JAK2 • NFKBIA • NRAS • PTPRC • RELA • RUNX1 • SRSF2 • TET2

This Month in JAAD International: March 2025: Weight gain and Janus kinase inhibitors. (PubMed, J Am Acad Dermatol) - No abstract available

Journal • Atopic Dermatitis • Dermatitis • Dermatology • Immunology • Psoriasis

Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis (clinicaltrials.gov) - P2 | N=7 | Terminated | Sponsor: NS Pharma, Inc. | N=120 ➔ 7 | Recruiting ➔ Terminated; The study was stopped due to a business decision.

Enrollment change • Trial termination • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Thrombocytopenia • Thrombocytosis

Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis (clinicaltrials.gov) - P2 | N=120 | Recruiting | Sponsor: NS Pharma, Inc. | Phase classification: P2b ➔ P2

Phase classification • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Thrombocytopenia • Thrombocytosis

Novel janus-kinase (JAK) inhibitors in myelofibrosis. (PubMed, Expert Opin Investig Drugs) - "This review includes the current status of JAKi treatment for myelofibrosis, mainly focusing on investigational JAKis; jaktinib, lestaurtinib, itacitinib, gandotinib, BMS-911543, ilginatinib, TQ05105, and flonoltinib maleate...In patients with myelofibrosis, momelotinib was effective in treating anemia, whereas jaktinib was effective in both anemia and Total Symptom Score (TSS)...The increasing variety of JAKis will allow for more personalized treatment options for myelofibrosis in the future. The potential impact on disease progression, molecular responses, and the duration of this response will become important parameters for future evaluations of these drugs."

Journal • Review • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Oncology

NS-018, an Investigational Treatment for Myelofibrosis, Receives Orphan Drug Designation from the European Commission (PRNewswire) - "NS Pharma, Inc. announced today that the European Commission (EC) has granted Orphan Drug Designation to NS-018 (ilginatinib) an oral, selective JAK2 inhibitor which is being investigated for the treatment of myelofibrosis (MF)."

European regulatory • Hematological Malignancies • Myelofibrosis • Myeloproliferative Neoplasm • Oncology

A RANDOMIZED, CONTROLLED CLINICAL TRIAL (PHASE 2B) OF NS-018 VERSUS BEST AVAILABLE THERAPY IN SUBJECTS WITH PRIMARY OR SECONDARY MYELOFIBROSIS WITH SEVERE THROMBOCYTOPENIA (EHA 2023) - P1/2, P2b | "A total of 120 subjects are expected to be enrolled and randomized 1:1 to either NS-018 or BAT (may include hydroxyurea, danazol, fedratinib, ruxolitinib, or no therapy). NS-018-201 study is currently enrolling at sites worldwide. Co-primary endpoints of reduction of spleen size and symptomatic scores, as well as safety will be elucidated. Other exploratory endpoints which will benefit to understand and manage the disease are also investigated."

Clinical • P2b data • Anemia • Fatigue • Hematological Disorders • Immunology • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Thrombocytopenia

NS Pharma Announces First Patient Enrolled in Phase 2 Study to Assess Efficacy and Safety of NS-018 Compared to Best Available Therapy (BAT) in Patients With Myelofibrosis (PRNewswire) - "NS Pharma, Inc. announced today the first patient enrollment in a Phase 2 clinical study of NS-018 (ilginatinib), an investigational treatment for myelofibrosis (MF), a rare and incurable blood cancer."

Trial status • Hematological Malignancies • Myelofibrosis • Oncology

NS Pharma Announces Receipt of Orphan Drug Designation from the U.S. FDA for NS-018, an Investigational Treatment for Myelofibrosis (PRNewswire) - "NS Pharma...announced that the U.S. FDA has granted Orphan Drug Designation to NS-018 (ilginatinib), an investigational treatment for myelofibrosis (MF), a rare and incurable blood cancer.....NS-018 is a highly selective and potent inhibitor of JAK2 developed by scientists from Nippon Shinyaku."

Orphan drug • Hematological Malignancies • Myelofibrosis • Myeloproliferative Neoplasm • Oncology

Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis (clinicaltrials.gov) - P2b | N=120 | Recruiting | Sponsor: NS Pharma, Inc. | Not yet recruiting ➔ Recruiting

Enrollment open • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Polycythemia Vera • Thrombocytopenia • Thrombocytosis

JAK Be Nimble: Reviewing the Development of JAK Inhibitors and JAK Inhibitor Combinations for Special Populations of Patients with Myelofibrosis. (PubMed, J Immunother Precis Oncol) - "In this review, we highlight several specific areas of unmet need within MF. Subsequently, we review agents that target those areas of unmet need, focusing specifically on the JAK inhibitors, momelotinib, pacritinib, itacitinib, and NS-018 as well as JAK inhibitor combination approaches using CPI-0610, navitoclax, parsaclisib, and luspatercept."

Journal • Review • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Rare Diseases

Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis (clinicaltrials.gov) - P2b; N=120; Not yet recruiting; Sponsor: NS Pharma, Inc.; Initiation date: Dec 2021 ➔ Mar 2022

Clinical • Trial initiation date • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Polycythemia Vera • Thrombocytopenia • Thrombocytosis • MRI

Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis (clinicaltrials.gov) - P2b; N=100; Not yet recruiting; Sponsor: NS Pharma, Inc.; Initiation date: Jul 2021 ➔ Dec 2021

Clinical • Trial initiation date • Hematological Disorders • Myelofibrosis • Myeloproliferative Neoplasm • Polycythemia Vera • Thrombocytopenia • Thrombocytosis • MRI

Safety and Dose Finding Study of NS-065/NCNP-01 in Boys With Duchenne Muscular Dystrophy (DMD) (clinicaltrials.gov) - P2b; N=16; Completed; Sponsor: NS Pharma, Inc.; Phase classification: P2 ➔ P2b

Clinical • Phase classification • Duchenne Muscular Dystrophy • Genetic Disorders • Hematological Disorders • Muscular Dystrophy • Myelofibrosis • Polycythemia Vera • Thrombocytopenia • Thrombocytosis

Study to Assess Efficacy and Safety of NS-018 Compared to BAT in Patients With Myelofibrosis (clinicaltrials.gov) - P2b; N=100; Not yet recruiting; Sponsor: NS Pharma, Inc.

New P2b trial • Hematological Disorders • Myelofibrosis • Polycythemia Vera • Thrombocytopenia • Thrombocytosis • MRI

Safety and Tolerability Study of Oral NS-018 in Patients With Primary Myelofibrosis (MF), Post-polycythemia Vera MF or Post-essential Thrombocythemia MF (clinicaltrials.gov) - P1/2; N=77; Completed; Sponsor: NS Pharma, Inc.; Active, not recruiting ➔ Completed; Trial completion date: Nov 2020 ➔ May 2020; Trial primary completion date: Nov 2020 ➔ May 2020

Clinical • Trial completion • Trial completion date • Trial primary completion date • Myelofibrosis • Polycythemia Vera • Thrombocytosis

Q2 '12 Results (Nippon Shinyaku) - Anticipated NME submission for myelofibrosis in 2013

Anticipated FDA event

Safety and Tolerability Study of Oral NS-018 in Patients With Primary Myelofibrosis (MF), Post-polycythemia Vera MF or Post-essential Thrombocythemia MF (clinicaltrials.gov) - P1/2; N=77; Active, not recruiting; Sponsor: NS Pharma, Inc.; Trial completion date: Nov 2019 ➔ Nov 2020; Trial primary completion date: Nov 2019 ➔ Nov 2020

Clinical • Trial completion date • Trial primary completion date