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 has been granted Fast Track designation by the U.S. FDA. Enrollment in this study has been completed and the first interim analysis is upcoming.

Combination Therapies

XBiotech continues to consider other possible oncology indications for Xilonix, particularly as a combination therapy. The Company believes Xilonix may work, for example, to improve the efficacy of chemotherapy where immunomodulatory effects of treatment may be partly related to disease progression. XBiotech is confident in Xilonix’s potential as both a monotherapy and as a combination therapy for the treatment of various types of cancer and will continue to explore these treatment opportunities.

There remains interest to assess Xilonix in combination with Tarceva® for the treatment of NSCLC. The interest in combining Xilonix with an EGFR inhibitor was based on results, which we published in Investigational New Drugs recently, of treatment for non-small cell lung cancer patients who have previously been treated with an EGFR inhibitor. Patients receiving anti-EGFR therapy prior to Xilonix had considerably better outcomes than those that had not received EGFR inhibition. We will continue to look for opportunities in combination therapy for non-small cell lung cancer and other indications involving anti EGFR treatment.

Relevant Publications