Enzo Biochem, Inc. (NYSE: ENZ), announced today that it had entered into a Cooperative Research and Development Agreement (CRADA) with the National Eye Institute, part of the National Institute of Health, to conduct a human clinical trial of Optiquel™, Enzo’s oral, proprietary therapeutic for chronic autoimmune uveitis.
Under the terms of the CRADA, Enzo will supply its proprietary compound, Optiquel™, and will fund a portion of the studies. The clinical trial will be conducted to assess the safety and efficacy of Optiquel™ in a proof-of-concept clinical trial designed as a randomized, double-masked, placebo-controlled study with a long-term follow-up. The agreement additionally includes non clinical research focusing on the use of various compounds that may serve to enhance the immune mediated oral tolerance response to specific antigens. Such research may be applicable across the entire spectrum of the Company’s immune regulation platform, including Alequel™, and EGS21™, its therapeutic candidates for the treatment of Crohn’s Disease and NASH, respectively.
Robert Nussenblatt, MD, Chief of NEI’s Laboratory of Immunology, will serve as the Principal Investigator on this project. Dr Nussenblatt, a world-renowned ocular immunologist and expert in clinical research design and conduct, has authored or co-authored over 400 publications and a number of books, including a standard text on uveitis.
An autoimmune disorder is a malfunction of the immune system which causes it to attack itself. In autoimmune uveitis, the body’s own immune system attacks the uvea of the eye, the portion of the eye that lies between the outermost and innermost layers of the eye. Uveitis occurs most frequently in people ages 20 to 50. A recent study estimated that more than 280,000 people in the United States are affected by uveitis each year, and the disease is associated with 30,000 cases of blindness annually.
Patients with chronic autoimmune uveitis are currently treated with general immunosuppressive agents such as steroids. The aim of immunosuppression is to control inflammation and to preserve visual acuity. However, immunosuppression therapy is frequently associated with significant adverse effects, including ocular toxicity (glaucoma and cataract), that limit the duration and intensity of treatment. Therefore, there exists an urgent need for new, non-general and specific therapeutic approaches with less material side effects.
Optiquel™ has been designed as such a therapeutic approach as it specifically targets the HLA proteins. A Phase I study of Optiquel ™ showed no treatment related side effects. Optiquel™ therapy is based on oral tolerance, an immunologic mechanism that down-regulates immune responses in an antigen-specific manner, rather than a non-specific immunosuppressive approach. A Phase I study of Optiquel™ demonstrated a therapeutic effect with no treatment-related side effects. Optiquel™ has been awarded orphan drug status in the European Union and it is anticipated that a similar designation will be applied for in the US. Enzo has exclusive rights to both US and international patents claiming the use of this compound for the treatment of uveitis.
Enzo will sponsor an investigational new drug (IND) application and submit it to the FDA. Additionally, Enzo has completed all of the bulk manufacturing, stability studies, pharmacokinetics evaluations, and formulation in capsules, as well as prepared the necessary documentation to treat patients. Toxicity studies have all proven negative.
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