Active Biotech's Tasquinimod Myelofibrosis Trial Resumes After FDA Approval of Protocol Amendment
核心洞察
Active Biotech (搜索) received FDA approval for protocol amendments to its tasquinimod myelofibrosis (搜索) trial, allowing the study to resume recruitment with increased dosing flexibility.
The clinical study evaluates tasquinimod both as monotherapy in JAK2 (搜索) inhibitor refractory patients and in combination with JAK2 inhibitors for suboptimal responders.
The company expects to report interim results in 2026 and final study results by the end of 2027 for this rare blood cancer affecting approximately 1.5 cases per 100,000 people.
Active Biotech (搜索) announced that the US Food and Drug Administration (FDA) and MD Anderson Institutional Review Board have approved protocol amendments for its clinical proof-of-concept trial evaluating tasquinimod in myelofibrosis (搜索) patients. The approval enables the Swedish biotechnology company to resume recruitment for the study, which first enrolled patients in 2025.
"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," said Active Biotech (搜索) CEO Helén Tuvesson.
Study Design and Patient Population
The clinical trial consists of two distinct cohorts designed to address different patient populations with myelofibrosis (搜索). The first cohort evaluates tasquinimod as monotherapy in patients who are refractory to or intolerant of JAK2 (搜索) inhibitors. The second cohort examines tasquinimod in combination with JAK2 inhibitors for patients showing suboptimal response to JAK inhibitor monotherapy.
Dr. Lucia Masarova, associate professor of Leukemia at The University of Texas MD Anderson Cancer Center, serves as the study's principal investigator. The trial is registered under ClinicalTrials.gov identifier NCT06327100.
Protocol Amendments Enhance Treatment Flexibility
The approved protocol amendment introduces several key modifications to improve study conduct and patient access. The dosing schedule of tasquinimod now closely reflects the regimen used in previous phase III prostate cancer (搜索) studies, leveraging the drug's well-characterized safety profile from earlier clinical programs.
Significantly, the amendment expands combination therapy options by allowing tasquinimod to be paired with either ruxolitinib, an established JAK2 (搜索) inhibitor, or momelotinib, the newly approved JAK2/ACVR1 (搜索) inhibitor. This flexibility addresses the evolving treatment landscape for myelofibrosis (搜索) patients.
Tasquinimod's Novel Mechanism of Action
Tasquinimod represents an orally active small molecule immunomodulator with a distinctive mode of action that blocks tumor-supporting pathways in the bone marrow microenvironment. Preclinical studies demonstrate that tasquinimod reduces myeloproliferation, splenomegaly, and fibrosis in myelofibrosis (搜索) models, showing efficacy both as monotherapy and in combination with approved therapies.
The drug's development history includes extensive clinical evaluation in solid tumors, particularly a phase II-III program in metastatic prostate cancer (搜索) patients, which established its safety profile and informed current dosing strategies.
Addressing Unmet Medical Need in Myelofibrosis
Myelofibrosis (搜索) affects approximately 1.5 cases per 100,000 people annually in the EU, US, UK, and Japan. This rare blood cancer belongs to the myeloproliferative neoplasm family and causes abnormal blood cell production, leading to healthy bone marrow replacement with scar tissue.
Patients typically present with anemia (搜索), altered white blood cell counts, spleen enlargement, increased infection risk, night sweats, and fever. The disease shortens survival, with death often resulting from bone marrow failure or transformation to acute leukemia (搜索).
Current treatment options include bone marrow transplantation for eligible patients, erythropoietin for anemia (搜索) management, and JAK inhibitors for spleen size reduction. However, no approved therapies currently reverse bone marrow fibrosis in myelofibrosis (搜索) patients, highlighting the significant unmet medical need that tasquinimod aims to address.