Fusion Protein, CGEN-15001, Shows Ability to Restore Immune Balance in Mice with MS

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.”

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.”

The company is now searching to partners to help it bring the treatment into clinical testing in people.