Protagonist Therapeutics Initiates Phase 2 Trial of Novel Hepcidin Mimetic PTG-300 for the Treatment of Patients with Beta Thalassemia

-- Initial Phase 2 results expected in the second half of 2019 --

NEWARK, A, USA I January 9, 2019 I Protagonist Therapeutics, Inc. (Nasdaq: PTGX) today announced that the first patient has been dosed in the Phase 2 study of PTG-300, an injectable hepcidin mimetic peptide in development for the treatment of patients with beta thalassemia, a rare disease characterized by chronic anemia and iron overload. The study is designed to evaluate the safety and preliminary efficacy of PTG-300 in patients with transfusion-dependent or non-transfusion-dependent beta thalassemia. In a completed Phase 1 study in healthy volunteers, administration of PTG-300 was well tolerated and demonstrated a dose-related and sustained reduction in serum iron levels.

"We are encouraged to move forward with clinical development of PTG-300 in patients with beta thalassemia," commented Dinesh V. Patel, Ph.D., Protagonist President and Chief Executive Officer. "In addition to beta thalassemia, PTG-300 has broad potential in the treatment of other disorders, including hereditary hemochromatosis and the myeloproliferative neoplasms polycythemia vera and myelodysplastic syndrome. The Phase 2 trial incorporates an open label trial design and we expect to report initial results in the second half of 2019. We are actively evaluating additional disease indications for development of PTG-300 and plan to commence on a second indication in the second half of 2019."

The global Phase 2 study is a single-arm, open label, multiple-ascending dose design which will evaluate safety, proof-of-concept and dose finding in approximately 84 adolescent and adult patients with anemia associated with non-transfusion-dependent or transfusion-dependent beta thalassemia. Non-transfusion-dependent patients will receive 12 weeks treatment with PTG-300 in escalating dose cohorts. The primary efficacy endpoint in non-transfusion-dependent patients will be change in hemoglobin from baseline. Transfusion-dependent patients will receive 16 weeks treatment with PTG-300 in escalating dose cohorts. The primary efficacy endpoint in transfusion-dependent patients will be a change in transfusion burden from baseline.

About PTG-300

PTG-300 is an injectable hepcidin mimetic in clinical development for the potential treatment of beta thalassemia, a rare disease characterized by chronic anemia and iron overload. Hepcidin is a natural peptide hormone that is a critical regulatory hormone governing iron absorption, recycling and utilization by the body. Iron plays an essential role in various body functions, especially blood formation. Excess iron in the body is toxic, resulting in tissue and organ damage over time. Abnormally low hepcidin levels caused by genetic mutations or secondary pathology can be replaced by a hepcidin mimetic to restore iron homeostasis. PTG-300 has been granted Orphan Drug designation in the U.S. and EU and has received Fast Track designation by the FDA for development in the treatment of beta thalassemia. Treatment of patients with polycythemia vera, myelodysplastic syndromes and hereditary hemochromatosis represent additional opportunities for future development of PTG-300.

About Protagonist Therapeutics

Protagonist Therapeutics is a clinical stage biopharmaceutical company that utilizes a proprietary technology platform to discover and develop novel peptide-based drugs to transform existing treatment paradigms for patients with significant unmet medical needs. PTG-300 is an injectable hepcidin mimetic for the potential treatment of anemia and iron overload related to rare blood diseases with an initial focus on beta thalassemia. PTG-200 is an oral peptide interleukin-23 receptor antagonist in development for the treatment of Crohn's disease. The company has entered into a worldwide license and collaboration agreement with Janssen Biotech for the clinical development of PTG-200. PN-943 is an oral, gut-restricted alpha-4-beta-7 integrin antagonist peptide in development for the treatment of inflammatory bowel disease.

Protagonist is headquartered in Newark, California, with pre-clinical and clinical staff in California and discovery operations in both California and Brisbane, Queensland, Australia. For further information, please visit http://www.protagonist-inc.com.

SOURCE: Protagonist Therapeutics

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