Pipeline

We are a clinical-stage biopharmaceutical company with a proprietary technology platform focused on discovering and developing peptide-based new chemical entities (NCEs) to address significant unmet medical needs. Our primary focus is on developing first-in-class oral peptide drugs that specifically target biological pathways also targeted by currently marketed injectable antibody drugs. Compared to injectable antibody drugs, our oral peptides offer targeted delivery to the gastrointestinal (GI) tissue compartment, potential for improved safety due to minimal exposure in the blood, improved convenience and compliance due to oral delivery, and the opportunity for earlier introduction of targeted therapy for inflammatory bowel disease (IBD). Our initial lead product candidates, PTG-100 and PTG-200, are based on this approach, and we believe they have the potential to transform the existing treatment paradigm for IBD, a GI disease consisting primarily of ulcerative colitis (UC) and Crohn’s disease (CD).

IBD is a chronic inflammatory disease with significant unmet medical need, which has created a large and growing market with an estimated 1.6 million patients in the United States in 2013. As of 2008, annual direct treatment costs for patients with IBD in the United States were estimated to exceed $6.3 billion, with indirect costs estimated to be an additional $5.5 billion. In 2012, Global Data estimated that the UC and CD markets reached approximately $4.2 billion and $3.2 billion, respectively, across ten major markets, and Global Data estimates that these markets are expected to grow at a compound annual growth rate of approximately 3% to 5% through 2022. The current tumor necrosis factor-alpha (TNF-α) antibody drugs approved for moderate-to-severe IBD, Humira® and Remicade®, are both injectable. According to Global Data, the 2013 sales for Humira® and Remicade® for IBD were $3.4 billion in the United States. Approximately one third of IBD patients are nonresponders to TNF-α antibody drugs and approximately another 30% to 40% become refractory within the first year of treatment. Additionally, TNF-α antibody drugs may predispose patients to an increased risk of serious infection and the development of anti-drug antibodies (ADAs), which over time can cause loss of drug response. Thus, while available treatments exist for moderate-to-severe IBD, there continues to be a significant medical need for efficacious, safer, and convenient treatments.

R&D Pipeline: Oral Peptides Based Targeted Therapy for GI Diseases and Disorders

Protagonist leverages its proprietary peptide technology platform to discover and develop novel product candidates to treat diseases with significant unmet medical needs.

Program* Dosing Form Indication Preclinical Phase 1 Phase 2 Anticipated Milestones

PTG-100 (Oral)

α4β7 Antagonist

  • Initiate Phase 2b Clinical Trial by the end of Q4 2016

PTG-100

α4β7 Antagonist

Oral IBD (Ulcerative Colitis)
  • Initiate Phase 2b Clinical Trial by the end of Q4 2016

PTG-200 (Oral)

IL-23 Antagonist

  • Initiate Phase 1 Clinical Trial in 2017

PTG-200

IL-23 Antagonist

Oral IBD (Crohn’s Disease)
  • Initiate Phase 1 Clinical Trial in 2017

PTG-300 (Injectable Sub-Q)

Hepcidin Mimetic

  • Initiate Phase 1 Clinical Trial in 2017

PTG-300

Hepcidin Mimetic

Injectable (Sub-Q) Iron Overload Disorders
  • Initiate Phase 1 Clinical Trial in 2017

*All Programs include worldwide commercial rights.

PTG-100: A GI-Restricted α4β7 Integrin-Specific Antagonist for the Treatment of Moderate-to-Severe UC

PTG-100 is a potential first-in-class oral, alpha-4-beta-7 (α4β7) integrin-specific antagonist peptide product candidate which has now completed a Phase 1 clinical trial in normal healthy volunteers (NHVs), and is being developed initially for potential treatment of moderate-to-severe ulcerative colitis (UC). α4β7 integrin is considered to be one of the most GI-specific biological targets for IBD due to its binding to MAdCAM-1, an extracellular protein that resides mostly in the GI vasculature.

PTG-200: A Potential First-in-Class Oral IL-23R Antagonist for the Treatment of Moderate-to-Severe IBD

PTG-200 is a potential first-in-class oral Interleukin-23 receptor (IL-23R) antagonist being developed initially for moderate-to-severe CD. PTG-200 is currently in Investigational New Drug (IND) enabling studies, and we plan to initiate a Phase 1 clinical trial in 2017.

We believe PTG-100 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 potentially offer improved convenience, patient compliance, and safety and tolerability compared to currently approved injectable antibody drugs. We believe these potential advantages could allow our products to replace and expand the IBD market beyond the moderate-to-severe IBD patient population currently treated by injectable antibody drugs.

PTG-300: An Injectable Hepcidin Mimetic Being Developed to Treat Iron Overload Disorders

Our novel peptides have potential applicability in a wide range of therapeutic areas in addition to GI diseases. Our first product candidate beyond IBD is PTG-300, an injectable hepcidin mimetic, which is currently in pre-clinical development. PTG-300 has potential utility for the treatment of iron overload disorders, such as transfusion-dependent β-Thalassemia, hereditary hemochromatosis (HH) and sickle cell disease (SCD), each of which may qualify for orphan designation.

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