CLINICAL TRIAL ACTIVITIES

XBiotech has conducted a number of Phase I/II clinical trials for its lead product candidate Xilonix™. This remarkable therapeutic antibody targets and neutralizes a small protein substance produced by the body known as IL-1α. To date, multiple clinical trials testing Xilonix™ monotherapy have been conducted, including the treatment of late stage cancer, diabetes, vascular disease, acne and psoriasis. Results from these studies have formed the basis for USFDA Fast Track designations for the treatment of colorectal cancer, as well as for treatment of patients undergoing treatment for vascular disease. A Phase III pivotal study was launched in 2013 for the use of Xilonix™ in improving survival in metastatic colorectal cancer patients. XBiotech has also worked with the European Medicines Agency (EMA) to establish a regulatory path in Europe and launched a Phase III registration study there in March 2014 evaluating Xilonix™ in symptomatic colorectal cancer patients refractory to standard therapy.


milestones



A short list of clinical milestones is shown on a timeline from 2010 through expected milestones over the near future. Notably, the timeline depicts XBiotech's launch of its True Human™ antibody therapy into human clinical trials for advanced cancer treatment in 2010 at MD Anderson Cancer Center in Texas. Based on results from this study, the most advanced programs depicted are an ongoing Phase III Pivotal studies in the US and Europe for treatment of colorectal cancer. The US study is headed by the Mayo Clinic in Rochester, Minnesota. This study involves over 100 clinical sites around the USA. The second Phase III pivotal study in Europe is also underway with roughly 40 sites enrolled in 8 countries across Europe. Proof of concept clinical studies conduced in Type II diabetes, Psoriasis, and Acne have each yielded promising results to justify advancement of respective clinical programs. Based on clinical efficacy seen with the anti-IL-1α therapeutic antibody in dermatology, a Phase II study was recently launched for a rare but debilitating disorder, Pyoderma Gangrenosum (PG). Based on the small patient population and severity of the disease, this indication is expected to be classified as an orphan indication. In 2013, XBiotech discovered a True Human antibody for treating methicillin resistant staphylococcus aureus (S. aureus) infections. The Company’s antibody therapy is designed to neutralize a key immune evasion mechanism of the bacteria, enabling the body’s immune system to naturally, safely and effectively eradicate S. aureus infection. This possible breakthrough antibody therapy for drug resistant bacterial infections is expected to reach clinical development before the end of 2014.

List of Enrolling or Completed Clinical Studies*

Title
Enrolling
Completed
Study of MABp1 in patients with Advanced Cancers X
Study of CA-18C3 in Subjects with Advanced Hematologic Malignancies X
Open Label, Randomized Study of the Safety, Pharmacokinetics, and Preliminary Efficacy of an Anti-Inflammatory Therapeutic Antibody (CV-18C3) in Reducing Restenosis in Patients Undergoing Percutaneous Femoro-Popliteal Revascularization X
Open Label Study of the Safety, Pharmacokinetics, and Efficacy of a True Human™ Anti-Inflammatory Therapeutic Antibody (RA-18C3) in Subjects with Moderate to Severe Plaque Psoriasis X
Study of the Safety and Pharmacokinetics of an Anti-Inflammatory Therapeutic Antibody (MABp1) in Patients with Type 2 Diabetes Mellitus X
Open Label Study of the Safety, Pharmacokinetics, and Efficacy of a True Human™ Anti-Inflammatory Therapeutic Antibody (RA-18C3) in Subjects with Moderate to Severe Acne Vulgaris X
A Phase III Double-blinded, Placebo Controlled Study of Xilonix™ for Improving Survival in Metastatic Colorectal Cancer X
A Phase II Open Label Study of RA-18C3 in Subjects with Pyoderma Gangrenosum X
A Double Blind, Placebo Controlled Pivotal Phase III Study Evaluating Xilonix™ in Symptomatic Colorectal Cancer Patients Refractory to Standard Therapy X

*The MABp1 anti-IL-1α therapeutic antibody has been assigned different names depending on the clinical indication, such as MABp1, Xilonix™, CA-18C3, CV-18C3, RA-18C3, T2-18C3, etc. Each of these, however, contain the same True Human™ antibody MABp1.