Enabling Therapeutics

Enabling Innovation
Our product pipeline stems from three robust technology platforms. These platforms can generate potential therapies for a variety of conditions in gastroenterology, ophthalmology, metabolic and bone disorders and infectious diseases.
Enzo Therapeutics’ product pipeline reaches into a broad range of markets with therapeutic modalities for inflammatory bowel disease, uveitis, HIV infection, bone disorders , diabetes, and other immune mediated diseases.
Inflammatory Bowel Disease
Inflammatory Bowel Disease (IBD) refers to conditions such as ulcerative colitis and Crohn’s disease that are characterized by chronic inflammation of the intestine. Although the cause of IBD remains unknown, various features of the disease strongly suggest that autoimmunity is a major component of the pathogenesis. This suggests that Enzo’s immune regulation technology platform may offer an effective treatment for IBD
IBD patients are currently managed during short-term episodes through the use of anti-inflammatory medications, or immunosuppressants such as corticosteroids. These provide symptomatic relief over short periods of time, but do not deliver a cure. These non-specific drugs all act by a generalized suppression of the immune response, with the unfortunate side effect of increasing the patient’s susceptibility to infection. Enzo’s drug candidate, AlequelT, is designed as a therapeutic modality that specifically quiets the inflammatory responses associated with IBD but does not induce a generalized immune suppression.
Approximately one million people in the United States suffer from IBD. In recent years the total treatment market exceeded 1.5 billion dollars.
Uveitis
Uveitis, an inflammatory disease of the eye, is the third leading cause of blindness in the US, after diabetes and macular degeneration. Autoimmune uveitis is believed to result from an autoimmune reaction to antigens in the eye, specifically the S-antigen and the interphotoreceptor retinoid-binding protein (IRBP). OptiquelT our candidate drug based on Enzo’s immune regulation technology platform is a potential therapy for autoimmune uveitis.
There is no known cure for uveitis, a disease that, according to the American Uveitis Society, is newly diagnosed in approximately 38.000 people every year. A particular concern is that it is a major cause of blindness among the younger population, especially in the 25 to 44 age group. Recent research suggests that autoimmune uveitis is significantly under-diagnosed an under-recognized. While there are steps that can be taken to preserve sight and slow the progress of vision loss, individuals with uveitis are also at increased risks of developing cataracts, glaucoma, or retinal detachment. Current mainstays of therapy, including systemic steroids and immunosuppressive agents, are associated with significant systemic toxic side effects.
HIV
After infection, HIV-1 runs a slow course in which immune T-cells (CD4+ cells) progressively disappear from the circulation. In the absence of treatment an infected individual loses the ability to mount an immune response. This loss of immune responsiveness leads to an increased susceptibility to infection that results in a complex of diseases know as AIDS and ultimately to death.
According to the World Health Organization, during 2007 there were more than 60 million individuals worldwide living with HIV infection. Over 5 million new infections and 3 million deaths occurred during that same year. More than 20 million individuals have died since the first cases of AIDS were identified in 1981. At present, several classes of products have received FDA marketing approval for HIV-1 infection, including reverse transcriptase inhibitors, protease inhibitors, fusion inhibitors and binding inhibitors.
Bone Disorders
Osteoporosis
Osteoporosis, meaning “porous bones”, is a condition of weakened and brittle bones that become more prone to fracture. The 10-billion dollar osteoporosis market is crowded with bone-preserving drugs such as bisphosphonates and selective estrogen receptor modulators, commonly referred to as SERMS. Bone-building drugs such as Enzo’s candidate compounds offer the potential for promising alternatives.
Periodontitis
One of the most challenging problems chronicled throughout the history of clinical dentistry is the loss of alveolar bone. Alveolar bone loss is characterized by a reduction in the height and volume of the maxillary and mandibular bones that underlie and support the teeth. The primary causes of alveolar bone loss are periodontitis and tooth loss, although osteoporosis may also contribute.
According to the literature (see Albandar JM 1999 J Periodontal., 70:13-29) roughly 35% of US adults between the ages of 30 and 90 have periodontitis. The lack of an effective treatment for periodontal bone loss has encouraged the continued search for a successful therapeutic approach. We believe that Enzo’s candidate bone-building drugs may be such an approach.
Diabetes
Type 2 diabetes is a metabolic disorder in which the body cannot produce sufficient amounts of insulin and/or cannot process insulin adequately. If left untreated diabetes can damage many organs in the body, including the heart, kidneys and eyes.
Western lifestyle associated with obesity, lack of exercise and high fat diets contributes to what is now recognized as a diabetes “epidemic”. The global incidence of diabetes is rising astronomically as developing countries adopt western lifestyles. This has contributed to an exponential growth in childhood type 2 diabetes. In the year 2000, there were an estimated 171 million individuals afflicted with diabetes worldwide and 16 million in the US alone. There is an expectation that the number of diabetic individuals world wide will rise to 366 million (see Wild et al., 2000 Diabetes Care, 27:1047-1053).
Since the “cure for diabetes” has proven elusive, new therapies administered alone and in combination are an important focus for pharmaceutical companies, regulatory authorities and health-related organizations worldwide.
We believe that Enzo’s candidate drug compounds, that have been shown to lower blood glucose in animal model systems of diabetes, show considerable promise as a therapy for diabetes.
Our Technologies and Pipeline

Enabling Immune Regulation
Many pathological conditions stem from deregulation of the immune system. Enzo has developed proprietary technology in two areas of immune regulation: Oral tolerance and Immune modulation

Enabling Cell Signaling Modulation
Enzo’s therapeutic platform provides a basis for the development of pharmaceutical agents that modulate protein-protein interactions. Our scientists and collaborators have unlocked the secrets of a major cell signaling pathway and developed a means to modify biologic activity in several metabolic systems through the use of small molecule drug candidates.