Overview

Peptides as targeted therapies for blood disorders and GI disease

Protagonist is shaping the future of peptide-based therapeutics, unlocking their potential against a broad range of serious chronic diseases.

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Disorders of Red Blood Cells and Iron Regulation

Rusfertide (PTG-300) is currently being evaluated for various disorders associated with iron overload and/or erythrocytosis (excessive 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 Phase 2 studies 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 blood disorder causing abnormal iron levels in tissues and organs arising primarily from absence or deficiency of the hepcidin pathway in these patients.

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Ulcerative Colitis

PN-943 is an investigational, orally delivered, gut-restricted alpha-4-beta-7 (α4β7) integrin antagonist being developed for potential treatment of Inflammatory Bowel Disease (IBD). This target is validated through the marketed injectable antibody drug Entyvio® that works 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).

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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 Psoriasis and IBD. The current oral candidate being evaluated is PN-235.