Biotech Short Profile

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2 The Company X is leading the development of next generation biological therapies and production systems. The Company is in two pivotal Phase III clinical studies in colorectal cancer for a unique anticancer agent that makes patients feel better, while undermining the tumor s ability to do harm. Proof of concept studies have been completed in Phase II clinical studies in diabetes, peripheral vascular disease, acne, and psoriasis. The Company has a rich discovery pipeline of first-in-class True Human antibodies a better, safer platform from which to produce therapeutic antibodies. And X is dramatically reducing the capital footprint to bring breakthrough biological therapies to market with new technology that collapses the complex, capital intensive infrastructure of biological manufacturing. In the end, all our efforts are aimed at getting safe, well tolerated breakthrough therapies to patients with better value for stakeholders and shareholders alike. Uncompromising Values Relentless Innovation Breakthrough Therapies X campus located in Austin, Tx incorporates 46,000 ft 2 of research and development laboratories, manufacturing and G&A operations Contact 8201 E Riverside Dr. Bldg 4 Ste 100 Austin, TX (Ph) Ms. Ashley Otero aotero@xbiotech.com Please visit our website at 2

3 X scientists working on Company s manufacturing process. UNCOMMON DEPTH AND VALUE X Inc. was founded in 2005 in Vancouver, Canada and now is also incorporated in Zurich, Frankfurt and Tokyo, with operations headquartered in Austin, Texas. The Company was founded to develop therapeutic antibodies from those that occur naturally in human beings. This contrasts all other marketed antibodies to date, which are engineered in vitro. Based on this fundamental biology, True Human antibodies were expected to be safer, better drug products. A concept that today is supported by compelling clinical evidence. To identify True Human antibodies, X pioneered technology to recognize these precious proteins and clone the unique gene sequences from healthy human donors. To better control costs and timelines, and facilitate a dynamic clinical program, in 2008 the Company began to explore manufacturing technology. By 2010, the first clinical trial was launched using drug product manufactured from disposable bioreactors. In 2014, X now has engineered its own bioreactors and downstream equipment to operate what it believes is the most cost efficient and most readily compliant biologics manufacturing platform in the industry. BREAKTHROUGH THERAPY EXCEPTIONAL CLINICAL DEVELOPMENT PROGRAM X has conducted a number of Phase I/II clinical trials for its lead product candidate. The Company has a fast-tracked Phase III registration study in the US, and a separate Phase III registration study in Europe. While both studies are evaluating Xilonix therapy in advanced colorectal cancer patients, the studies differ with respect to primary endpoints, size and anticipated duration. Xilonix is a True Human monoclonal antibody that targets and neutralizes interleukin-1 alpha (IL-1α). IL-1α is produced by cells in response to injury, where it acts to stimulate a cascade of events leading to inflammation, tissue breakdown, angiogenesis, swelling and pain. IL-1α also acts as a messenger, relaying a danger signal via central nervous system (CNS) pathways in the brain to mediate psychological and metabolic changes associated with injury, including fatigue, malaise and muscle wasting. Extensive clinical studies with the anti-il-1α monoclonal antibody therapy have been or are being conducted, including late stage cancer (colorectal cancer, non-small cell lung cancer and others), diabetes, vascular disease, acne, psoriasis and pyoderma gangrenosum. Picture shows vials of drug ready for shipment from the Company s manufacturing operations in Austin, Texas. Protein therapeutics from the Texas facility are manufactured, filled and finished, and shipped to clinics throughout the US and Europe. 3

4 KEY CLINICAL MILESTONES The five clinical trial programs the Company launched in 2011 in oncology, cardiovascular disease, type 2 diabetes, psoriasis and acne have all concluded with complete data analysis in Oncology data from the Phase I/II program at MD Anderson showed evidence of novel anti-tumor activity in several diseases. In total, 66 patients were treated at MD Anderson Cancer Center, with the largest cohorts studied being colorectal and non-small cell lung cancer. Responses in colorectal cancer were particularly dramatic, including the unprecedented observation of rapid physical recovery of patients during treatment. Based on these findings, X received fast track designation by the FDA. In 2013, a 656 patient Phase III pivotal study was launched to assess Xilonix monotherapy in colorectal cancer patients with associated cachexia. Overall survival of patients is the primary endpoint of the study. In 2013, X formed a partnership with McKesson/US Oncology, to gain access to their extensive network of cancer treatment centers. With McKesson on board, the Company now has access to cancer treatment centers across the United States, where colorectal cancer patients will be treated with Xilonix. X expanded its clinical partnerships in 2014, working with Sarah Cannon Institute to bring Xilonix to more cancer centers. endpoints in evaluating cancer therapy. Using radiographic techniques largely novel to oncology, the Company established a regulatory path in Europe that is faster and we believe may be more informative than other methods. The EMA regulatory path was established only in March 2014, while the first patient enrolled in the European study was in August While much of the Company s clinical focus is on its Phase III oncology programs, other important clinical programs are in development. The fast track designation received late in 2012 for a vascular disease therapy has, for example, provided a regulatory path for marketing approval for this major opportunity; and a definitive clinical study is being planned for this indication. Clinical and regulatory planning for other indications where proof of concept studies showed promising results are ongoing. Based on clinical efficacy seen with the anti-il-1α therapeutic antibody in dermatology, a Phase II study was recently started for a rare but debilitating disorder, pyoderma gangrenosum. Because of the small population of sufferers and the severity of the disease, this indication is expected to be classified as an orphan indication, providing important clinical development and marketing benefits to X. Based on the unique Phase I/II clinical findings, which included the first ever reported observations of lean body mass recovery in refractory cancer patients, X worked with the European Medicines Agency (EMA) to find a better way to evaluate Xilonix therapy in advanced stage cancer. The challenge was to use the unprecedented measure of recovery seen in patients treated with Xilonix to define new clinical SELECT HIGHLIGHTS X Germany/ Japan Vascular Disease Fast Track Launch of Phase II PG Study X formed a partnership with McKesson/US Oncology to gain access to their extensive network of cancer treatment centers to advance its Phase III Study. X has received approval from the EMA for a Phase III registration study in Europe for treatment of colorectal cancer with its lead product, Xilonix.. X established subsidiaries in both Frankfurt, Germany & Tokyo, Japan with X Germany GmbH and X Japan KK to facilitate clinical operations in these jurisdictions. The fast track designation received late in 2012 for vascular disease therapy has provided a regulatory path for marketing approval for this major indication. Based on results from previous dermatology studies, a Phase II Study was recently launched for a rare but debilitating disorder, Pyoderma Gangrenosum (PG). 4

5 FUTURE CLINICAL PROGRAMS In 2013 X delivered on its discovery efforts for novel therapies for infectious disease. Among these is the discovery of a True Human antibody therapy for treating methicilin resistant staphylococcus (S. aureus) infections. S. aureus causes devastating and lethal infections and is a global medical problem. A generation of vaccines against S. aureus have failed and antibiotic therapy to manage these infections are often incapable of saving life or, literally, limb. 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. Pre-clinical studies are ongoing. Meanwhile, manufacturing systems for this antibody are in development. An IND submission to allow testing of the antibody therapy in human infections is anticipated before year end MRSA A FDA submission for human treatment of this antibody is projected for Plate shows bacteria colonies being screened for MRSA at X s R&D laboratories in Austin, TX. Neupogen TM /Neulasta TM generated nearly $5Billion in revenue for Amgen in LEVERAGING OUR CAPABILITIES Significant opportunities are emerging with the patent expiration of currently marketed blockbuster biologics. X has made substantive progress in developing a next generation therapy based on Neupogen /Neulasta (Neulasta is a modified version of Neupogen ), products which generated nearly $5Billion in revenue in X has created a new version of this molecule dubbed NeupoX that, based on preclinical models to date, is expected to be highly competitive in what is becoming a crowded arena for biosimilars. NeupoX will be simpler and much cheaper to manufacture than the branded product marketed in the US. With numerous biosimilar products coming to market, X believes that only the highest quality products, with the lowest cost basis, will ultimately survive in the market place. The development of NeupoX, including a manufacturing process, is well advanced. X had planned to revolutionize the biosimilar industry with clinical launch of NeupoX late in The clinical launch of NeupoX has, however, been delayed in order to prioritize nearer term opportunities. 5

6 STREAMLINING DRUG DEVELOPMENT X is innovating with manufacturing processes to use the latest technologies to reduce needed infrastructure and go-forward operating costs for the production of biological drugs. Compared to existing commercial manufacturing programs, which use massively complex plant and equipment infrastructure, X s manufacturing technology is the epitome of elegant simplicity. At the heart of X s process is a single use container (essentially a large plastic bag) which is filled with nutrient media and used to grow the drug-producing cells. While X s novel manufacturing platform reduces infrastructure costs and operating complexity, it also minimizes lead times, simplifies our ability to comply with manufacturing regulations for drug makers, and improves production flexibility compared to the infrastructure-laden, complex bioreactor systems currently used in the industry. Forty-six thousand square feet of facilities have been builtout in Austin, Texas to house current manufacturing and R&D needs. To accommodate future commercial demand, X has purchased 48 acres of land and designed and engineered a commercial manufacturing facility. Ground breaking on this new facility occurred in September Comparison of Amgen s manufacturing equipment (top image) with those in operation at X (lower image). The single bag reactor depicted in the X operation has the same operating capacity and potential output as the row of incredibly complex, costly machines shown on the Amgen manufacturing floor. X believes that these manufacturing practices are relics of an old biotech industry. X is at the forefront of establishing vastly more cost effective and efficient operations. 6

7 PIONEERING TRUE HUMAN TM THERAPEUTIC ANTIBODIES X s True Human antibodies are cloned from a natural human immune response isolated from living donors. This is perhaps the most challenging way of discovering antibodies but ultimately the only approach to generate True Human therapeutic antibodies. Genes for therapeutically useful antibodies are not encoded in the human genome. Rather, antibody genes are encoded as segments in the germline and are shuffled and mutated to produce a mature antibody. The remarkable process of genetic engineering takes place for every antibody within specialized cells and compartments of the human body to produce billions of novel and effective antibodies. Crucial steps in this engineering process involve selection and deletion of desired and undesirable antibodies, respectively. This selection process is vital to the development of tolerant antibodies that exhibit good functionality and do not cause adverse reactions in the body. Only antibodies developed naturally in the body may be expected to not cause untoward responses when used as drugs. True Human antibodies are thus expected to exhibit superior safety, efficacy and commercial value compared to earlier generation antibody therapeutics. In all clinical studies to date after more than 600 treatments there has been not a single drug related serious adverse event. This is remarkable considering the therapy has been given alone, without any other treatment or drugs to reduce the side effects typically associated with antibody therapy. The safety-efficacy profile to date is simply unparalleled. A short list of clinical milestones is shown on a timeline from 2010 through milestones to date in

8 CURRENT PIPELINE STATUS X has clinical trial activities in a number of major indications. The Company will launch its first anti-infective therapy targeting methicilin resistant staphylococcus aureus (MRSA) in X is also leveraging its manufacturing platform to develop highly competitive biosimilar drugs. Contact 8201 E Riverside Dr. Bldg 4 Ste 100 Austin, TX (Ph) Ms. Ashley Otero aotero@xbiotech.com Please visit our website at 8

9 PARTNERSHIPS Howard Holdings, Inc. GAS Partners, L.P. Pantheon Total Fund, L.P. X is Partnered with Leading Clinical and Financial Institutions Pelota Ventures, LLC GMP Securities, L.P. Roytor & Co. Jacesa Investments Limited 9