Therapeutic Areas
RXi will use its expertise in RNAi compound design and delivery to identify lead therapeutic candidates for inflammatory and metabolic diseases. The RXi technology platform allows us to discover many drug candidates that can be developed as therapeutics. To move as many of these therapeutic candidates forward into clinical trials as possible, RXi’s is seeking to work with partners in the discovery and development process.
Inflammatory Disease
Inflammatory diseases now affect roughly 8million people in the United States and, with a continued need for safe and effective alternatives to existing therapies, the market is experiencing tremendous growth. Ongoing research, including the area of RNAi therapeutics, is focused on targeting specific biological modifiers that might avoid generalized damage to the immune system. These new approaches have the potential to generate improved therapies for a number of high profile inflammatory disorders, including rheumatoid arthritis, atherosclerosis, diabetes, and inflammatory bowel diseases such as Crohn’s disease and ulcerative colitis.
RXi is working with a new mode of RNAi therapy that takes advantage of an oral route of administration. This non-invasive approach may lead to therapies that control many severe inflammatory diseases while negating the need for injected drugs which are the current standard of care. The use of RNAi oral administration in animal models was recently published in the journal Nature (Aouadi, et al, Nature, Vol. 458 (7242), pp. 1180-1184) in which a reduction in a systemic inflammatory response was clearly demonstrated. This new technology provides an important scientific and commercial opportunity for the company, and RXi is focusing its resources on this exciting therapeutic area.
Metabolic Disease:
High cholesterol, obesity and type 2 diabetes are major health problems and affect hundreds of millions of people worldwide. The World Health Organization estimates that, on a worldwide basis, there are more than 300 million cases of obesity and 159 million cases of type 2 diabetes.
RXi has two primary efforts in metabolic disease. First, we are targeting a gene thought to be responsible for elevated cholesterol. Second, RXi has in-licensed intellectual property developed by Dr. Michael Czech, an RXi co-founder and Professor and Chair of Molecular Medicine and Professor of Biochemistry and Molecular Pharmacology at the University of Massachusetts Medical School (UMMS), on genes that appear to be important regulators of metabolism. Studies conducted in Dr. Czech’s laboratory at UMMS and by others at Imperial College of London demonstrated that inactivation of one of these genes, called RIP140, can cause fat cells to metabolize rather than store fat. Mice in these studies that did not express RIP140 remained lean and non-diabetic even when maintained on a high-fat diet. RXi is currently designing rxRNA™ compounds targeting RIP140 as a potential treatment for obesity and obesity-related type 2 diabetes.
OTHER THERAPEUTIC AREAS:
Many well-studied targets exist for numerous diseases that RXi believes will be difficult to treat with conventional medicinal chemistry. With that in mind, RXi may also pursue pre-clinical studies in several additional disease areas, with the goal of creating multiple clinical development programs. RXi intends to focus on combining its expertise in RNAi with existing disease models through collaborative interactions with academic, biotech and pharmaceutical industry scientists.
Alliance Partners in Therapeutic Areas:
RXi is actively seeking to leverage its technology platform by working with larger pharmaceutical and biotechnology partners in the partners' fields of interest. Furthermore, the RXi team has experience targeting genes in virtually every major therapeutic area and RXi believes it will discover many more drug candidates than can be advanced in-house. RXi will seek to work with partners in the discovery and development process in additional fields of interest.
In addition, the oral delivery technology offers a unique opportunity to treat the inflammatory component of numerous additional diseases. These conditions include, for example, ischemia, type 2 diabetes, and the inflammatory and angiogenic components of cancer progression. These diseases may be good examples of diseases with target genes that are not currently funded internally at RXi, but may be a good fit for potential partners.
