hCDR1 is a novel compound, working via a novel mechanism of action, for the treatment of Systemic Lupus Erythematosus ("SLE"). Currently, treatment for SLE is based primarily on immune suppressing agents, such as corticosteroids, azathioprine, cyclosporine and others. Only one product has been approved by the FDA for the treatment of SLE in the last 50 years: Benlysta (belimumab), which was approved in 2011. Design of hCDR1 – a Targeted Peptide hCDR1 is poised to be a disease-specific treatment and was developed by Prof. Edna Mozes of the Department of Immunology, The Weizmann Institute of Science (Israel). hCDR1 ameliorates the SLE-related autoimmune process by specific upstream immunomodulation through the generation of regulatory T cells. The regulatory T cells then initiate a physiological cascade of events involving beneficial effects on auto reactive T and B cells, autoantibodies and various pathogenic cytokines and immunosuppressive molecules, which together resume immune balance.
Preclinical and Clinical Information A large body of preclinical and clinical research has been performed on hCDR1, including on the mechanism of action on the clinical as well as the molecular levels. hCDR1’s beneficial effects in SLE were demonstrated in a number of animal models and species in >200 experiments. Similar effects were also demonstrated on human peripheral blood lymphocytes (PBL) ex-vivo and in-vitro. The results of these studies have been published in more than 40 peer reviewed papers. There have been two placebo controlled Phase I trials and a placebo controlled Phase II trial (PRELUDE) conducted with hCDR1. The Phase I and Phase II studies consisted of over 400 patients, demonstrating that hCDR1 is well tolerated by patients and has a favorable safety profile. The PRELUDE trial did not achieve its primary efficacy endpoint based on the SLEDAI scale. However, the PRELUDE trial showed encouraging results in its secondary clinical endpoint, the BILAG index, and, in fact, the 0.5mg weekly dose showed a substantial effect. Multiple post-hoc analyses showed impressive results for this dose using the BILAG index. Such dose will be the focus of our clinical development plan moving forward. The FDA has since directed that the primary endpoint in future trials for Lupus therapies, including those for hCDR1, should be based on either the BILAG index or the SLE Responder Index (SRI). About SLE Lupus is a chronic autoimmune disease involving many systems in the human body, including joints, kidneys, central nervous system, heart, hematological system and others. The biologic basis of the disease is a defect in the immune (defense) system, leading to production of self (auto) antibodies, attacking the normal organs and causing irreversible damage. The clinical manifestations are numerous and variable, from a minimal and stable disease to a rapidly progressive and fatal one, especially if vital organs, such as the kidneys or CNS are affected. According to the Lupus Foundation of America, at least 1.5 million Americans have the disease (>5 million worldwide) with more than 16,000 new cases diagnosed each year. The vast majority of patients are women of childbearing years. For additional information about Lupus please visit the Lupus Foundation of America site, www.lupus.org.