Peptides as targeted therapies for blood disorders and GI disease
Protagonist is combining scientific rigor and a proprietary technology platform to innovate and develop well-differentiated assets to address unmet needs in various rare and chronic diseases.
Disorders of Red Blood Cells and Iron Regulation
Rusfertide (PTG-300) is currently being evaluated for various disorders associated with iron overload and/or excessive erythrocytosis (red blood cell production). Rusfertide is a synthetic mimetic of the natural hormone hepcidin that regulates iron homeostasis and controls the absorption, storage, and distribution of iron in the body. Discovered through our peptide technology platform, rusfertide is currently in a Phase 2 study in polycythemia vera (PV), a rare chronic blood disorder that affects about 160,000 patients in the U.S. In addition, it is also being evaluated in a small open-label Phase 2 study in hereditary hemochromatosis (HH), a broader population-based blood disorder affecting over a million people in the U.S. alone and arising primarily from absence or deficiency of the hepcidin pathway in these patients.
Inflammatory Bowel Disease (IBD)
PN-943 is an investigational, orally delivered, gut-restricted alpha-4-beta-7 (α4β7) integrin antagonist being developed for potential treatment of IBD. This target is validated through marketed injectable antibody drugs like Entyvio® that work by blocking the same target with the key differentiation being the oral, gut-restricted approach offered by PN-943. This candidate is currently in a placebo-controlled, randomized, double-blinded, 150-patient Phase 2 study for moderate-to-severe ulcerative colitis (UC).
Oral IL-23 Receptor Antagonists
Protagonist and Janssen Biotech, Inc. operate under a co-development and commercialization agreement for oral IL-23 receptor targeted therapeutics with applications in various disease areas already validated through approved and marketed injectable antibody drugs that target the IL-12/23 or IL-23 pathway. IL-23 is a protein that regulates inflammatory and immune function and plays a key role in the development of immunomodulatory diseases including but not limited to IBD. The current oral candidates being evaluated are PN-235 and PN-232.