Compugen has reported new and promising results from studies on animal models of multiple sclerosis (MS) that support its lead drug candidate, CGEN-15001, as a potential treatment for a variety of autoimmune diseases, including MS.
Specifically, CGEN-15001 was shown to restore immune tolerance and balance in a durable and sustained manner in treated animals, a major unmet research goal for these diseases.
“Autoimmune diseases, such as multiple sclerosis, rheumatoid arthritis, type 1 diabetes and psoriasis, are conditions in which the immune system attacks the body’s healthy tissues due to loss of self-tolerance. Restoring this tolerance without impacting the immune system’s ability to fight other diseases is the Holy Grail of immunology,” Anat Cohen-Dayag, CEO and president of Compugen, said in a press release. “Currently, most drugs indicated for autoimmune diseases are general immuno-suppressants, which may lead to serious side effects including infections and an elevated risk of cancer.”
CGEN-15001 is an Fc fusion protein based on an immune checkpoint protein that company researchers discovered. It has shown the potential to restore immune tolerance and immune balance in a specific manner, so as to avoid systematic or global immune suppression. Together with previous test results, the new data demonstrate that CGEN-15001 has the potential to induce a sustained and safer therapeutic response in patients with autoimmune diseases.
Researchers, working with a team from Feinberg School of Medicine at Northwestern University led by Professor Stephen Miller, showed that treatment with CGEN-15001 in animal models of relapsing-remitting multiple sclerosis (RRMS) allowed for the transfer of immune tolerance from diseased mice to non-treated mice (through the exchange of immune T-cells). This result was shown to be specific, protecting the recipient mice from developing MS even upon disease stimulation.
Similar tests conducted using a different an immune checkpoint-based drug designed to treat autoimmunity, Orencia (CTLA4-Ig), failed to induce immune tolerance or protect the mice from developing the disease, the researchers reported.
Although both drugs, CGEN-15001 and Orencia, improved the clinical MS symptoms in the diseased donor mice, they had very different long-lasting effects on the recipient mice.
“This observation is of significance since CGEN-15001 appears to not only act as an immune tolerance inducing agent, but also to act in an antigen-specific manner, thus having the potential to benefit patients with a long-lived drug free remission while avoiding the global immune suppression induced by other treatments,” said Miller. “This data therefore supports a safety profile for CGEN-15001 with potentially low risk for infections and neo-malignancies which are unfortunately recognized side effects of many of the currently available drugs for autoimmunity.”