Momenta Pharmaceuticals has dosed the first subject in a Phase I/II clinical trial of M254 for the treatment of Immune Thrombocytopenic Purpura (ITP), a rare autoimmune disease that results in excessive bruising and bleeding.
M254 is a hypersialylated human immunoglobulin G that contains improved tetra-sialylation.
The four-part trial features a placebo-controlled, randomised double-blinded cross-over design and is expected to enrol 65 healthy subjects and patients with ITP.
The trial is designed to examine the safety, efficacy, pharmacokinetics, and pharmacodynamics of intravenous (IV) M254 in comparison with intravenous immunoglobulin G(IVIg).
Parts A and B of the trial will feature double-blind, single ascending dose cohort design and will include healthy subjects and ITP patients respectively.
Part C of the trial involves the administration of M254 or IVIg in a cross-over dosing design, while Part D will see patients with ITP treated with multiple doses of M254.
The trial’s primary efficacy objective is an assessment of platelet response.
Momenta Pharmaceuticals Development senior vice-president and chief medical officer Santiago Arroyo said: “We believe M254 has the potential to be a significantly better option for patients than conventional IVIg.
“Our aim for this study is to show clinically what we have observed in extensive preclinical models, which is that hypersialylated IgG is substantially more potent than intravenous immunoglobulin G (IVIg) in ITP and other inflammatory disorders.
“We look forward to obtaining initial clinical data in the first half of 2020.”
Results from preclinical studies of ITP and other inflammatory diseases showed that M254 increased the potency of patients up to ten times more than those treated with conventional IVIg.