Our Science

Delivering on the Untapped Potential of Peptides

Ra Pharmaceuticals utilizes a technology process called “mRNA display” as the foundation for our  Extreme Diversity™ platform to produce extremely large and diverse libraries of macrocyclic peptides from which to screen for potential product candidates.

The Extreme Diversity™ platform can identify macrocyclic peptides that combine the diversity and specificity of antibodies with the pharmacological properties of small, synthetic molecules. These highly specific and stable peptide-like molecules are much smaller than monoclonal antibodies and other biologics, enabling more convenient routes of administration and potentially improved tissue penetration, while offering the opportunity to target protein-protein interactions, a type of molecular interaction that historically has been difficult to address with other small molecules.

Zilucoplan is an investigational, synthetic, macrocyclic peptide discovered using this powerful proprietary drug discovery technology. The peptide is designed to bind complement component 5 (C5) with sub-nanomolar affinity and allosterically inhibit its cleavage into C5a and C5b upon activation of the classical, alternative, or lectin pathways. This, in turn, blocks the assembly of the membrane attack complex, a potent mediator of tissue damage in many autoimmune and inflammatory diseases.

The relatively small size of this peptide allows for distinct advantages compared to larger monoclonal antibodies, including enhanced tissue penetration and convenient, subcutaneous (SC) self-administration, an improved route for C5 inhibitor therapy as compared to intravenous infusion with monoclonal antibodies.

Ra Pharma is developing zilucoplan for generalized myasthenia gravis (gMG), immune-mediated necrotizing myopathy (IMNM), amyotrophic lateral sclerosis (ALS), and other tissue-based complement-mediated disorders.

Leveraging our scientific expertise in the field of peptides, Ra Pharma has also built libraries of passively permeable peptides that are capable of crossing cell membranes. Further, the ability of these peptides to cross the blood-brain barrier provides a unique opportunity to research and develop novel therapeutic options against diseases of the central nervous system (CNS).