First Myelofibrosis Patient Dosed in US Tasquinimod Phase II Study

Researchers are investigating the effectiveness of the drug tasquinimod in treating patients with specific types of myelofibrosis. This study aims to evaluate tasquinimod as a monotherapy for patients who have either relapsed after using JAK inhibitors or are ineligible for such treatments. Additionally, the study examines the combination of tasquinimod with the JAK inhibitor ruxolitinib for patients who have not achieved a sufficient response from ruxolitinib alone.

The primary goal of this clinical trial is to assess the anti-tumor activity of tasquinimod through the objective response rate (ORR) after six cycles of treatment. The ORR is a critical measure, representing the proportion of patients with a significant reduction in tumor size or disease markers. By focusing on ORR, researchers can determine the effectiveness of tasquinimod in reducing myelofibrosis symptoms and tumor presence.

Alongside the primary objective, the study is also set to evaluate several secondary outcomes. These include the safety and tolerability of tasquinimod, which are essential for understanding potential side effects and risks associated with the treatment. Researchers will also monitor the symptom burden experienced by patients, aiming to identify any improvements or exacerbations resulting from the treatment. Additionally, the study will assess the duration of response, indicating how long patients benefit from the treatment, and examine changes in the grade of bone marrow fibrosis, a condition often associated with myelofibrosis that can affect overall patient health.

This significant clinical trial is being carried out at The University of Texas MD Anderson Cancer Center in Houston, Texas. Dr. Lucia Masarova, an assistant professor of Leukemia at MD Anderson, is the principal investigator overseeing the study. Active Biotech, a company involved in the development of tasquinimod, is providing support for the trial, supplying the Investigational Medicinal Product and covering related expenses. For those interested in more detailed information about the study, resources are available on clinicaltrials.gov under the identifier NCT06327100.

Dr. Erik Vahtola, Chief Medical Officer at Active Biotech, expressed enthusiasm about the study, highlighting the potential of tasquinimod to positively influence the bone marrow microenvironment, which is often suppressive in myelofibrosis patients. This approach aligns with ongoing research into how modulating this environment can improve treatment outcomes for patients with this challenging condition.

Overall, this study represents a crucial step in exploring new therapeutic options for myelofibrosis patients, particularly those who have limited responses to current treatments. By focusing on both monotherapy and combination therapy, the trial aims to provide comprehensive insights into how tasquinimod can be integrated into myelofibrosis treatment regimens. The outcomes of this research may lead to improved management strategies and enhanced quality of life for patients affected by this disease.