GL-2045 is the lead recombinant Intravenous Immunoglobulin (IVIg) mimetic in the Gliknik portfolio, expected to advance through multiple subcutaneous dosing in Healthy Participants in 2023 towards a Phase 2 trial in Chronic Inflammatory Demyelinating Polyneuropathy with subcutaneous administration.

The FDA acknowledged a shortage of IVIg in 2019 as a result of growing IVIg demand and then the shortage worsened during the pandemic as plasma collections fell off. Every other country uses even less IVIg per capita than the United States because of product shortage, high costs, and avoidance of blood products. There is a clear need for a recombinant equivalent of IVIg

The U.S. Food and Drug Administration granted Orphan Drug designation to GL-2045 for the treatment of CIDP.

Researchers have increasingly realized that the active fraction of IVIg in the treatment of autoimmune diseases is the aggregates found in low abundance in IVIg and specifically in the Fc portion of aggregates in IVIg*. One implication of this finding is that the vast majority of IVIg protein infused is not active in the treatment of autoimmune diseases.

IVIg Fc aggregates bind many receptors and ligands simultaneously and have numerous simultaneous beneficial effects. For this reason, researchers have thought it might be impossible to make a recombinant equivalent of IVIg for the treatment of autoimmune diseases. Gliknik recognized that if we could make a product that structurally is similar to the Fc aggregates in IVIg then it would be expected to reproduce the multiple effects of IVIg in the treatment of autoimmune diseases.

There are numerous marketed brands of IVIg with some products approved for use in the autoimmune indications including Chronic Inflammatory Demyelinating Polyneuropathy (CIDP, the largest autoimmune IVIg indication), multifocal motor neuropathy (MMN), dermatomyositis, and Idiopathic Thrombocytopenic Purpura (ITP). While IVIg is produced by pooling and purifying plasma from tens of thousands of human blood donors, GL-2045 is recombinant, so it does not carry the risk of blood-borne pathogen transmission.

Patients want a medicine that has a safer and more convenient administration profile compared to that of IVIg. Because GL-2045 is designed to mimic only the active fraction of IVIg, it is expected to require significantly less drug than IVIg in the treatment of autoimmune diseases. Its increased potency may enable infusion of smaller volumes over shorter infusion periods or subcutaneous injections, thereby potentially improving ease of administration and quality of life for these patients. Our goal is to demonstrate that GL-2045 meets or exceeds the efficacy of the pooled human blood product IVIg in treating autoimmune diseases, beginning with CIDP, with greater patient convenience and without exposure to blood products.

IVIg is also approved for treating immunodeficiency disorders, acting primarily through the Fab portion of IgG1. GL-2045 does not contain an Fab and was not designed to treat these patients. Since GL-2045 is a recombinant product, it can be produced in limitless quantities. If successfully commercialized, GL-2045 might allow more of the IVIg products, currently used to treat both immunodeficiency disorders and autoimmune indications, to be available for individuals living with immunodeficiency disorders, thereby helping to address a chronic worldwide IVIg supply shortage.

* Augener, 1985; Bussel, 1991; Debré, 1993; Teeling, 2001; Kroez, 2003; Bazin, 2004; Siragam, 2005; Clynes, 2005; Bazin, 2006; Siragam, 2006