XBiotech Receives Fast Track Designation from FDA for Anti-Cachexia Drug Xilonix™

Initiation of Phase III Pivotal Trial Using Proprietary True Human™ Monoclonal Antibody Targeting Inflammatory Cytokine IL-1α

AUSTIN, Texas, Oct. 3, 2012 /PRNewswire/ -- XBiotech, a privately held biotechnology company, announced today that the company has been granted Fast Track designation for its anti-cachexia drug Xilonix.  The Company's groundbreaking therapeutic antibody was shown in a previous clinical study at MD Anderson Cancer Center in Texas to stop or reverse cachexia in about a third of all advanced cancer patients treated. Anti-tumor and anti-cachexia effects were seen in many different types of cancer. In patients with advanced colorectal cancer, the reversal of cachexia resulted in a dramatic improvement in life expectancy, with patients that responded to treatment having a median survival of 474 days, versus a median of 72 days in others.

Xilonix is a first-in-class True Human antibody that blocks an inflammatory substance (IL-1α) produced by the body in response to tumor growth. This so called sterile inflammatory response plays a role in cancer progression and the development of lethal symptoms such as cachexia.

XBiotech will now launch a pivotal Phase III study under the FDA's Fast Track program to treat advanced colorectal cancer patients that are suffering from cachexia.  The study will involve sites in the United States, Canada and Europe with the results intended to provide the basis for international marketing approval. The international study will be a randomized, controlled trial to evaluate overall survival in patients treated with Xilonix.

"The Fast Track study design reflects our belief that Xilonix will substantially prolong life in this advanced cancer population," said John Simard, president and CEO. "That is why our clinical study examines overall survival compared to other anti-cachexia agents in development, which are only attempting to provide symptomatic relief to these patients. We believe that this makes Xilonix a very important and unique therapy for a large patient population that currently has few other treatment options."

"We are very grateful to the FDA for the careful analysis of our clinical results and for granting Fast Track designation to Xilonix," said Michael Stecher, M.D., Medical Director. "This designation highlights the significance of the data generated to date, and the potential for this treatment to address the needs of a patient population that has no effective therapies available."

Cachexia is a fatal wasting condition that develops in patients with chronic inflammatory conditions such as cancer, chronic obstructive pulmonary disease, heart disease, and even infectious diseases such as AIDS. There are over 400,000 cancer patients in the US with cachexia and over 5,000,000 patients suffering with clinical symptoms of the disease. It has been estimated that 2% of the general population is afflicted with early-stage cachexia (defined as weight loss in association with a chronic disease). In advanced cancer it has been estimated that 40% of all deaths are directly caused by the cachexic process rather than by tumor progression. Colorectal cancer affects about 5% of the US population, with up to 150,000 new cases per year in the US alone.

XBiotech is pioneering the development of novel antibodies, production processes and therapies to address vast unmet medical need. The Company has created a novel antibody discovery platform that is generating a dynamic pipeline of first-in-class True Human™ therapeutic antibodies. A diverse clinical program blocking chronic inflammation has created anticipation for a new therapeutic for treating multiple diseases. The use of new simpler manufacturing technology is also enabling a compelling, cost-efficient commercialization path. The Company's lead True Human antibody (Xilonix™) is a novel agent for neutralizing a master regulator of chronic inflammation—interleukin-1 alpha (IL-1α). The Company has completed or is conducting clinical studies in acne, psoriasis, cachexia, cancer, type 2 diabetes and cardiovascular disease.  For more information please visit, www.xbiotech.com.

John Simard
[email protected]

Aaron DeLucia
Porter Novelli
[email protected]