There is an urgent need for a new form of therapy, one that is designed to mediate anti-tumor, disease modifying activity that may prolong life, reduce both treatment and disease‐related morbidity, and improve quality of life without a significant demonstration of cytotoxicity.

New clinical endpoints are needed to evaluate anti‐cancer agents in studies with respect to their potential to prolong and improve the life of patients with advanced cancer. XBiotech has embarked on groundbreaking studies in oncology that are expected to antagonize the effects of tumor pathophysiology without principally acting as a cytotoxic agent. In doing so we have opened the door to novel thinking about how cancer agents may be conceived and developed to improve the health and overall survival of patients living with cancer.

Colorectal cancer

XBiotech has recently concluded a pivotal double-blind, placebo-controlled Phase III trial for Xilonix™ in symptomatic colorectal cancer patients refractory to standard therapy that has met its end point. This study resulted from a collaborative process between the sponsor and the Scientific Advisory Group of the European Medicines’ Agency (EMA).

The objective response criteria devised was consistent with an EMA Mandate to find new measures of efficacy for anti-tumor agents in advanced cancer based on improvement in disease-related symptoms. Relevant clinical endpoints were thus developed around unique findings from an earlier study where patients receiving the antibody were found to exhibit remarkable symptomatic recovery that in turn was associated with overall survival benefit.

Findings in the present study provide the first evidence that:

  1. new endpoints based on symptom recovery may be used to evaluate an anti‐tumor agent in advanced cancer
  2. in patients with advanced cancer, clinical trajectory can be positively altered in the presence of recalcitrant tumor
  3. Xilonix met its primary endpoint—and thus represents a novel therapy for a serious unmet medical need in advanced colorectal cancer

In addition, a Global Phase III registration study for advanced colorectal cancer patients is currently underway after receiving Fast Track designation by the U.S. FDA.

NSCLC (Non-Small Cell Lung Cancer)

XBiotech is planning to launch a Phase II study to assess Xilonix™ in combination with Tarceva® for the treatment of NSCLC. The proposed study will enroll approximately 75 patients. This study is following up on results from a previous study which suggested the potential for Xilonix™ therapy in combination with EGFR (Epidermal Growth Factor Receptor) inhibition in patients with advanced NSCLC.