Peptides as targeted therapies for GI diseases and blood disorders

Protagonist leverages its proprietary peptide technology platform to discover and develop novel drug candidates that can transform the existing treatment paradigms in diseases with significant unmet medical needs.

We are developing first-in-class oral peptide drugs in GI diseases that specifically target biological pathways that are currently targeted by marketed injectable antibody drugs. Compared to injectable antibody drugs, oral GI-restricted peptides can potentially offer:

  • Improved safety due to minimal exposure in the blood compartment
  • Improved convenience and compliance due to oral delivery
  • The opportunity for earlier introduction of targeted therapy for inflammatory bowel disease (IBD)

Our product candidates, PN-943 and PTG-200, are based on this approach, with the potential to transform the existing treatment paradigm for IBD, a GI disease consisting primarily of ulcerative colitis (UC) and Crohn’s disease (CD).

In blood disorders, our most advanced clinical product candidate, PTG-300, is under development for the treatment of certain disorders characterized by ineffective erythropoiesis, excessive red blood cells or iron overload. PTG-300 is an injectable compound that mimics the effect of the natural hormone hepcidin, but with greater potency, solubility and stability. Hepcidin is a key hormone in regulating iron equilibrium and is critical to the proper development of red blood cells. We are currently developing PTG-300 for beta-thalassemia (non-transfusion dependent and transfusion dependent), Polycythemia Vera, Hereditary Hemochromatosis and Myelodysplastic Syndromes. PTG-300 has received an orphan drug designation from the U.S. Food and Drug Administration (“FDA”) and European Union (“EU”) regulatory authorities for the treatment of beta-thalassemia. The FDA has also granted Fast Track designation to PTG-300 for the treatment of beta-thalassemia.

Protagonist product candidate pipeline

Potential advantages over current options

We believe PN-943 and PTG-200 have the potential to transform the existing IBD treatment paradigm because they offer significant advantages over injectable antibody drugs. These complementary assets target different biological pathways, and compared to currently approved injectable antibody drugs, offer potential improvements in:

  • Convenience
  • Patient compliance
  • Safety
  • Tolerability

There is also potential to use PN-943 and PTG-200 as monotherapies as well as in combination with each other or with other targeted therapies to increase the remission rate in the moderate-to-severe patients. We believe that all of these advantages could also allow our products to expand the IBD market beyond the moderate-to-severe IBD patient population currently treated by injectable antibody drugs. Additionally, PTG-300 has the potential to offer a novel approach for the treatment of anemia in patients with rare blood disorders such as β-Thalassemia.