Active Biotech (NASDAQ STOCKHOLM: ACTI) has announced the enrollment of its first patient in a groundbreaking clinical study evaluating tasquinimod for myelofibrosis patients who have limited treatment options. The Phase Ib/II trial targets patients who are either refractory to JAK2 inhibitor treatment or ineligible for such therapy, representing a significant advancement in addressing an urgent medical need.
Study Design and Objectives
The single-arm, multi-center open-label study is structured in two parts. The initial Phase Ib safety component will evaluate dose-limiting toxicity during a 28-day treatment period. Following this assessment, the trial will progress to its Phase II efficacy stage. Researchers will primarily focus on measuring the proportion of patients achieving at least a 35% reduction in spleen volume after six 4-week cycles of tasquinimod treatment.
Secondary endpoints include evaluating tasquinimod's effects on bone marrow fibrosis, myelofibrosis-related symptoms, changes in variant allele frequency, and overall survival. The study will be conducted across the Stichting Haemato-Oncologie Volwassenen Netherland (HOVON) network of study centers in the Netherlands and Germany.
Novel Therapeutic Approach
Tasquinimod represents a innovative approach to treating myelofibrosis, functioning as an orally active small molecule immunomodulator. The drug's mechanism of action uniquely targets tumor-supporting pathways in the bone marrow microenvironment, differentiating it from existing treatments.
"We are pleased to have enrolled the first patient into the study where tasquinimod will be evaluated in a patient population with high unmet medical need for new treatment options with a different mechanism of action," stated Dr. Erik Vahtola, CMO of Active Biotech. "I am excited to follow the study progress."
Disease Impact and Current Treatment Landscape
Myelofibrosis affects approximately 1.5 people per 100,000 annually across the EU, US, UK, and Japan. This rare blood cancer leads to the replacement of healthy bone marrow with scar tissue, resulting in abnormal blood cell production and various complications including enlarged spleen, increased infection risk, and potential transformation into acute leukemia.
Current treatment options are limited, with bone marrow transplantation available only for eligible patients, while JAK inhibitors primarily address spleen size reduction. Notably, no approved therapies currently exist that can reverse bone marrow fibrosis, highlighting the potential significance of tasquinimod's development in this space.
Research Collaboration and Support
The study emerges from a global patent license agreement between Active Biotech and Oncode Institute, established in February 2022. Oncode Institute, representing Erasmus Universiteit Medisch Centrum (Erasmus MC), will serve as the main financial supporter of the trial, while HOVON acts as the legal sponsor.
