Patients with a rare and aggressive form of liposarcoma need treatment advances

Dedifferentiated liposarcoma, or DDLPS, is a rare and aggressive form of sarcoma with a poor prognosis. Patients impacted by this devastating cancer have had no new first-line treatment options in almost 50 years. The first patient was enrolled at UZ Leuven (Belgium) by Patrick Schöffski’s team in our clinical trial called Brightline-1 (ClinicalTrials.gov Identifier: NCT05218499), studying an investigational treatment option for patients with advanced DDLPS. This compound is an investigational agent and has not been approved for use. The efficacy and safety of this investigational compound have not been established.

Brightline-1, a Phase II/III, randomized, open-label, multi-center study, is investigating the Boehringer Ingelheim investigational compound compared to doxorubicin as a first-line treatment for patients with advanced DDLPS. Globally, 400 patients were enrolled in the study with sites in Europe, North America, and Asia.

In 2023, the Brightline-1 clinical trial program progressed to Phase III and the European Medicines Agency granted our MDM2-p53 antagonist with Orphan Drug Designation for the potential treatment of soft tissue sarcoma, including DDLPS. The U.S. Food and Drug Administration (FDA) provided Fast Track Designation for the investigation of our MDM2-p53 antagonist in DDLPS.

Through this trial and other upcoming ones, and in partnership with experts in this field, we intend to expand our knowledge, and that of the medical community, about the role of the MDM2-p53 pathway in cancer development. This pathway is an area of high interest for the medical community as approximately 3.5-7% of tumors display MDM2 amplifications, including lung cancer and other malignancies, and nearly all DDLPS patients have MDM2 amplification.

Our goal is to transform the lives of people with cancer by delivering medical innovations in areas of high unmet need. Boehringer Ingelheim is committed to pioneering treatment advancements in some of the most challenging and most impactful areas of cancer.

Our rich pipeline of next-generation oncology treatments is comprised of cancer cell-directed and immuno-oncology therapies, the smart combinations of which may hold the greatest benefit for most patients​. We proactively collaborate with patients, advocacy organizations, and the world’s leading academic centers and industry, because we believe together, we can make a bigger impact in transforming the lives of people with cancer.

We hope the results of this clinical trial will provide sarcoma specialists with more therapeutic options that ultimately improve the lives of patients with DDLPS.