Tasquinimod

Projects

An immunomodulator with a unique mechanism of action for myelofibrosis

In development for the treatment of hematological malignancies. Clinical phase I/II studies in myelofibrosis are ongoing.

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Type of substance:

Small molecule

Effect:

Helps the immune system fight cancer

On this page:

Introduction

Tasquinimod in Brief

Tasquinimod is an orally active small molecule immunomodulator with a novel mode of action, blocking tumor supporting pathways in the bone marrow microenvironment. Tasquinimod is being developed for the treatment of blood cancers, with focus on myelofibrosis.

The tumor microenvironment in the bone marrow is essential for development of blood cancers and a key driver of disease recurrency as well as resistance to treatment.

Tasquinimod targets cells in the microenvironment of the bone marrow, immunosuppressive myeloid cells, endothelial cells, and mesenchymal cells, which play a central role in the development of blood cancers. Tasquinimod affects the function of these cells, leading to reduced tumor growth, reduced fibrosis, and restored hematopoiesis.

More about the project

How Tasquinimod Works

    Restores anti-tumour immunity

    Reduces tumour blood vessel formation (anti-angiogenic effect)

    Restores blood cell formation

    Reduces fibrosis

    Makes the tumour microenvironment less favourable for cancer and more receptive to the immune system

The Disease

Myelofibrosis

Myelofibrosis is a rare form of blood cancer. The sex- and age-adjusted incidence is estimated at approximately 1.5 cases per 100,000 people, with a prevalence of 12 patients per 100,000 people (Slowley et al., 2024). This would translate to a prevalence of more than 100,000 people with myelofibrosis in the EU, US, UK, and Japan.

More about the disease

The Project

Tasquinimod Targets the Tumor Microenvironment to Support Anti-Tumor Activity

Preclinical studies have shown that tasquinimod reduces myeloproliferation, splenomegaly (enlarged spleen), and fibrosis in models of myelofibrosis.

Preclinical experiments using malignant cells from patients have further shown that tasquinimod works synergistically with a JAK- or BET inhibitor to reduce spleen size and prolong survival. These promising results suggest that tasquinimod could be a valuable addition to the treatment options for myelofibrosis patients.

Tasquinimod was granted orphan designation in myelofibrosis by the US Food and Drug Administration (FDA) in May 2022.

Pipeline

    Discovery
    Preclinical
    Phase I
    Phase II
    Phase III

Clinical Development

Proof-of-concept studies with tasquinimod in myelofibrosis patients are ongoing in US and Europe. The study at MD Anderson Cancer Center in the US is evaluating tasquinimod as monotherapy and in combination with approved JAK2 inhibitors (JAKi). The European study, which is being conducted through a network of clinics in the Netherlands and Germany, is evaluating tasquinimod as monotherapy in JAKi-refractory patients.

As both studies are open-label, preliminary results may become available during the course of the studies. Pre-planned interim analyses will be conducted as part of the protocols and will be reported at scientific meetings, where applicable.

Tasquinimod har varit under utveckling för behandling av prostatacancer och har genomgått ett kliniskt utvecklingsprogram i fas I-III. Resultaten från fas III-prövningen i prostatacancer visade att tasquinimod förlängde den progressionsfria överlevnaden (progression free survival, PFS) men inte den totala överlevnadstiden (overall survival, OS) jämfört med placebo i denna patientpopulation och utvecklingen för prostatacancer avslutades. Tasquinimod studerades i både friska försökspersoner och cancerpatienter. Kliniska effekter och god säkerhet har visats i fler än 1 500 patienter, vilket motsvarar mer än 650 patientår av exponering för tasquinimod. Omfattande dataset inklusive regulatoriskt paket med preklinisk och klinisk säkerhet och CMC-dokumentation i full kommersiell skala har genererats.

  • Open Label Phase 1/2 Study of Tasquinimod in Patients With Primary Myelofibrosis (PMF), Post-Polycythemia Vera Myelofibrosis (Post-PV MF), or Post-Essential Thrombocytosis Myelofibrosis (Post-ET MF)

    Ongoing

    Phase I/II

    More about the study

    The study in the US is being conducted at MD Anderson Cancer Center, TX.

  • Tasquinimod in Patients with Myelofibrosis Refractory to or Intolerant for JAK2 Inhibition (HOVON 172 MF)

    Ongoing

    Phase I/II

    More about the study

    The European study is being conducted within the HOVON research network (Stichting Hemato-Oncologie voor Volwassenen Nederland), with participating clinics in the Netherlands and Germany.

  • Tasquinimod for the Treatment of Relapsed or Refractory Myeloma

    Completed

    Phase I/II

    More about the study

    Med prekliniska data och tidigare klinisk erfarenhet av tasquinimod som utgångspunkt påbörjades en klinisk studie och första patienten doserades i augusti 2020.

Publications

A collection of Publications

  • Heterogeneous bone-marrow stromal progenitors drive myelofibrosis via a druggable alarmin axis

    Leimkühler NB, Gleitz HFE, Ronghui L, Snoeren IAM, Fuchs SNR, Nagai JS, Banjanin B, Lam KH, Vogl T, Kuppe C, Stalmann USA, Büsche G, Kreipe H, Gütgemann I, Krebs P, Banz Y, Boor P, Tai EW, Brümmendorf TH, Koschmieder S, Crysandt M, Bindels E, Kramann R, Costa IG, Schneider RK

    Cell Stem Cell. 2021 Apr 1;28(4):637-652.e8. doi: 10.1016/j.stem.2020.11.004

  • Inhibiting the alarmin-driven hematopoiesis-stromal cell crosstalk in primary myelofibrosis ameliorates bone marrow fibrosis

    Gleitz HFE, Fuchs SNR, Snoeren IAM, Boys C, Nagai J, Tejeda-Mora H, Klöker V, Pritchard JE, Bakker IJ, Gargallo Garasa M, Bindels E, Saez-Rodriguez J, Vogl T, Kramann R, Dugourd A, Costa IG, Schneider RK

    Hemasphere. 2025 Aug 14;9(8):e70179. doi: 10.1002/hem3.70179

  • Preclinical efficacy of tasquinimod-based combinations in advanced myeloproliferative neoplasms in blastic phase

    Fiskus W, Masarova L, Mill CP, Birdwell CE, Das K, Hou H, Davis JA, Jain A, Malovannaya A, Manshouri T, Dunbar A, Sharma S, Kadia TM, DiNardo CD, Bose P, Pemmaraju N, Loghavi S, Su X, Rampal RK, Törngren M, Bhalla KN

    Blood Adv. 2025 Nov 11;9(21):5598-5609. doi: 10.1182/bloodadvances.2025016898.

  • S100A8/S100A9 Promote Progression of Multiple Myeloma via Expansion of Megakaryocytes

    Lin C, Garcia-Gerique L, Bonner EE, Mastio J, Rosenwasser M, Cruz Z, Lawler M, Bernabei L, Muthumani K, Liu Q, Poncz M, Vogl T, Törngren M, Eriksson H, Vogl DT, Gabrilovich DI, Nefedova Y.

    Cancer Res Commun. 2023 Mar 13;3(3):420-430