Protagonist Therapeutics Initiates Phase 2 Trial of Novel Hepcidin Mimetic PTG-300 for the Treatment of Patients with Beta Thalassemia
"We are encouraged to move forward with clinical development of PTG-300 in patients with beta thalassemia," commented
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
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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, including PTG-300, and the timing of the initiation and availability of results of our clinical trials. 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 ability to enroll and complete our planned clinical trials, our ability to develop and commercialize our product candidates, our ability to earn milestone payments under our collaboration agreement with Janssen, our ability to use and expand our programs to build a pipeline of product candidates, our ability to obtain and maintain regulatory approval of our product candidates, our ability to operate in a competitive industry and compete successfully against competitors that have greater resources than we do, 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 for the three months ended
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SOURCE
Solebury Trout, Rich Allan (media), Tel: +1 646-378-2958, Email: rallan@troutgroup.com, or Marcy Nanus (investors), Tel: +1 646-378-2927, Email: mnanus@soleburytrout.com