FDA Clears Protocol Update for Tasquinimod in Myelofibrosis Trial

Active Biotech has received positive feedback from regulators on a protocol amendment to its ongoing proof-of-concept clinical trial evaluating tasquinimod in patients with myelofibrosis, a rare and serious blood cancer. Following approval of the amendment by the U.S. Food and Drug Administration (FDA) and The University of Texas MD Anderson Cancer Center Institutional Review Board, the study will resume patient recruitment.

According to Helén Tuvesson, CEO of Active Biotech, “We are very happy with the response to our protocol amendments, and we expect to report interim results from the study in 2026 and study results towards the end of 2027."

The trial is led by principal investigator Dr. Lucia Masarova, associate professor in the Department of Leukemia at MD Anderson Cancer Center.

Tasquinimod Study in Myelofibrosis Resumes With Expanded Options

The amended trial is designed to provide greater flexibility in tasquinimod administration and to broaden eligibility in the combination treatment group. The updated dosing schedule more closely mirrors the regimen used in prior phase 3 studies of tasquinimod in metastatic prostate cancer, where its safety profile was established.

Importantly, the amendment allows tasquinimod to be combined with either Jakafi (ruxolitinib), a commonly used Janus kinase 2 (JAK2) inhibitor, or Ojjaara (momelotinib), a recently approved dual JAK2/ACVR1 inhibitor. This change expands options for patients whose disease has not responded adequately to a JAK inhibitor alone.

The trial includes two distinct cohorts. One cohort is evaluating tasquinimod as a single agent in patients who are refractory to or intolerant of prior JAK2 inhibitor therapy. The second cohort is studying tasquinimod in combination with a JAK inhibitor in patients who have experienced a suboptimal response to JAK inhibitor treatment. The first patient was enrolled in 2025.

By expanding the dosing flexibility and combination strategies, investigators aim to better understand how tasquinimod may fit into the current treatment landscape for myelofibrosis, particularly for patients with limited therapeutic options.

Understanding Myelofibrosis and Current Treatment Limitations

Myelofibrosis is a rare myeloproliferative neoplasm characterized by abnormal production of blood-forming cells in the bone marrow. Over time, healthy bone marrow is replaced with scar tissue, a process known as fibrosis. The annual incidence of myelofibrosis is approximately 1.5 cases per 100,000 people across the United States, European Union, United Kingdom and Japan.

Patients frequently present with anemia and abnormal white blood cell counts. As the disease progresses, individuals may experience splenomegaly, fatigue, night sweats, fever and increased susceptibility to infections. Myelofibrosis is associated with reduced survival, and complications may include bone marrow failure or transformation to acute leukemia.

Current treatment options focus primarily on symptom control. Bone marrow transplantation offers a potential cure for select eligible patients, although many individuals are not candidates due to age or comorbidities. JAK inhibitors, including ruxolitinib and momelotinib, are commonly used to reduce spleen size and alleviate symptoms. Erythropoietin may help manage anemia. However, no approved therapies have been shown to reverse bone marrow fibrosis.

How Tasquinimod Works and What Researchers Hope to Learn

Tasquinimod is an oral small molecule immunomodulator with a novel mechanism of action. It targets tumor-supporting pathways within the bone marrow microenvironment, which play a role in sustaining abnormal blood cell production and fibrosis. In preclinical models of myelofibrosis, tasquinimod has demonstrated reductions in myeloproliferation, splenomegaly, and bone marrow fibrosis. Activity has been observed both as monotherapy and in combination with approved agents.

Tasquinimod has also been studied extensively in solid tumors, including a large phase 2 to 3 development program in metastatic prostate cancer. These prior trials have helped define its safety profile, providing a foundation for ongoing development in hematologic malignancies.

The current proof-of-concept study in Europe and the United States seeks to evaluate safety, tolerability, and early signals of clinical activity in patients with myelofibrosis. By including individuals who are refractory to JAK inhibitors and those with suboptimal responses, the trial addresses a population with significant unmet medical need.

Patients and caregivers seeking additional information about the study can visit ClinicalTrials.gov and search for identifier NCT06327100.

Reference

1. “Active Biotech receives positive feedback on its clinical study with tasquinimod in myelofibrosis,” by Active Biotech. News release; Feb. 10, 2026.

Editor's note: This article is for informational purposes only and is not a substitute for professional medical advice, as your own experience will be unique. Use this article to guide discussions with your oncologist. Content was generated with AI and reviewed by a human editor.

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