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.
About NEI and NIH
The National Eye Institute (NEI) is part of the National Institutes of
Health (NIH) and is the Federal government's lead agency for vision
research that leads to sight-saving treatments and plays a key role in
reducing visual impairment and blindness. For more information, visit
the NEI Website at www.nei.nih.gov.
The National Institutes of Health (NIH) - The Nation's Medical Research
Agency - includes 27 Institutes and Centers and is a component of the U.
S. Department of Health and Human Services. It is the primary Federal
agency for conducting and supporting basic, clinical, and translational
medical research, and it investigates the causes, treatments, and cures
for both common and rare diseases. For more information about NIH and
its programs, visit www.nih.gov.
About Enzo
Enzo Biochem is engaged in the research, development, manufacture and
licensing of innovative health care products and technologies based on
molecular biology and genetic engineering techniques, and in providing
diagnostic services to the medical community. Enzo’s Life Sciences
division develops, produces and markets proprietary labeling and
detection products for gene sequencing, genetic analysis and
immunological research among others. Its catalog of over 30,000 products
serves the molecular biology, drug discovery and pathology research
markets. The Company's therapeutic division is in various stages of
clinical evaluation of its proprietary immune regulation medicines for
uveitis and Crohn's disease and conducts pre-clinical research on
several candidate compounds aimed at producing new mineral and organic
bone, including technology that could provide therapy for osteoporosis
and fractures, among other applications. Enzo’s Clinical Labs division
provides routine and esoteric reference laboratory services for
physicians in the New York Metropolitan and New Jersey areas.
Underpinning the Company’s technology and operations is an extensive
intellectual property estate in which Enzo owns or licenses over 240
patents worldwide, and has pending applications for over 200 more. For
more information visit our website www.enzo.com.
Except for historical information, the matters discussed in this news
release may be considered "forward-looking" statements within the
meaning of Section 27A of the Securities Act of 1933, as amended and
Section 21E of the Securities Exchange Act of 1934, as amended. Such
statements include declarations regarding the intent, belief or current
expectations of the Company and its management. Investors are cautioned
that any such forward-looking statements are not guarantees of future
performance and involve a number of risks and uncertainties that could
materially affect actual results. The Company disclaims any obligations
to update any forward-looking statement as a result of developments
occurring after the date of this press release.