FDA Grants Orphan Drug Designation for Protagonist Therapeutics' PTG-300 for the Treatment of Beta-Thalassemia

NEWARK, Calif., March 6, 2018 /PRNewswire/ -- Protagonist Therapeutics, Inc. (NASDAQ: PTGX) today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PTG-300, the company's sub-cutaneous injectable hepcidin mimetic for the treatment of beta-thalassemia.

"Beta-thalassemia is a rare genetic blood disorder that is characterized by impaired red blood cell production that can result in life-threatening chronic anemia, usually requiring regular and life-long blood transfusions for survival. Over time, these transfusions can lead to excessive iron levels in the body which can be toxic and consequently lead to end-stage damage to vital organs such as the liver and the heart," said David Y. Liu, Ph.D., Chief Scientific Officer and Head of Research and Development of Protagonist Therapeutics. "As a hepcidin mimetic, PTG-300 is designed to help reduce these excessive iron levels and thereby it may lead to improvements in anemia and decreased need for blood transfusions and chelation therapy."

Protagonist recently completed a Phase 1 study of PTG-300 that established pharmacodynamic-based clinical proof-of-concept by achieving dose-related and sustained reductions in serum iron levels in normal healthy volunteers. It was well tolerated with no serious adverse events or dose-limiting toxicities. The company intends to initiate a global clinical trial with PTG-300 in patients with beta-thalassemia following our upcoming meetings with the U.S. and European regulatory agencies.

About PTG-300 and Hepcidin

PTG-300, an injectable hepcidin mimetic, is currently in clinical development for the potential treatment of beta-thalassemia, a rare disease characterized by chronic anemia and iron overload. PTG-300 therapy may also be potentially beneficial in other diseases such as myelodysplastic syndrome (MDS), hereditary hemochromatosis (HH), polycythemia vera (PCV), siderophilic infections, and liver fibrosis which provide additional opportunities for future development. Hepcidin is a natural peptide hormone that is the main regulatory hormone governing iron absorption, recycling and utilization by the body. Iron plays an essential role in various body functions, especially blood formation, but too much iron is toxic and causes organ damage over time. Abnormally low hepcidin levels, caused by genetic mutations or secondary pathology, can result in the body absorbing and storing more iron than is needed, leading to iron overload.

About Orphan Drug Designation

The FDA grants Orphan Drug Designation to novel drugs or biologics that treat rare diseases or conditions affecting fewer than 200,000 patients in the United States. The designation allows the sponsor of the drug to be eligible for a seven-year period of U.S. marketing exclusivity on approval of the drug, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical trial design assistance, and the waiver of Prescription Drug User Fee Act (PDUFA) filing fees.

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. Two of its clinical stage product candidates, PTG-100 and PTG-200, are oral targeted therapy drugs being developed for inflammatory bowel diseases (IBD). The alpha-4-beta-7 integrin antagonist peptide PTG-100 is currently in a Phase 2b clinical trial for moderate-to-severe ulcerative colitis, and the company's interleukin-23 receptor antagonist peptide PTG-200 is currently being studied in a Phase 1 clinical trial in healthy volunteers to support further development in Crohn's disease.  Both alpha-4-beta-7 integrin and IL-12/23 pathway blockade are approaches that have been validated through FDA-approved injectable antibody drugs. The company has entered into a worldwide license and collaboration agreement with Janssen Biotech for the clinical development of PTG-200.  Protagonist has also applied its versatile platform outside of the GI disease areas and is developing an injectable hepcidin mimetic, PTG-300, for the treatment of anemia related to rare blood diseases with an initial focus on beta-thalassemia. PTG-300 recently completed a Phase 1 clinical trial which established pharmacodynamic-based clinical proof-of-concept in normal healthy volunteers. The U.S. Food and Drug Administration has granted Orphan Drug Designation to PTG-300 for beta-thalassemia.

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

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements regarding our intentions or current expectations concerning, among other things, the potential for our programs, the initiation and availability of results of our clinical trials and research and development. In some cases, you can identify these statements by forward-looking words such as "anticipate," "believe," "may," "will," "expect," or the negative or plural of these words or similar expressions.  Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, our history of net operating losses, our reliance on third parties and uncertainty regarding our ability to achieve profitability, our ability to develop and commercialize our product candidates, our ability to obtain and maintain regulatory approval of our product candidates, and our ability to obtain and adequately protect intellectual property rights for our product candidates.  We discuss many of these risks in greater detail under the heading "Risk Factors" contained in our quarterly report on Form 10-Q filed with the Securities and Exchange Commission. Forward-looking statements are not guarantees of future performance, and our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate, may differ materially from the forward-looking statements contained in this press release. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements, whether as a result of new information, future events or otherwise.

SOURCE Protagonist Therapeutics, Inc.

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