Protagonist Therapeutics Announces Preliminary Phase 2 Results with Hepcidin Mimetic PTG-300 in the Treatment of Transfusion Dependent Beta-Thalassemia
"The dose-related pharmacodynamic responses in serum iron and TSAT levels observed in this preliminary analysis provide the first evidence of the effects of PTG-300 in patients with beta-thalassemia, who have highly elevated levels of iron in the body," commented
"Treatment options for patients with beta-thalassemia are limited and the complications associated with transfusion are serious," commented
"The consistent and significant effect on iron levels observed in normal healthy volunteers in a previous study, and now in patients with beta-thalassemia, provides strong rationale for potential utility of PTG-300 in blood disorders directly dependent on disruption of normal iron homeostasis in the body," commented
In the study, PTG-300 was well-tolerated and systemic adverse events were mild to moderate in severity and were typical of patients with TD beta-thalassemia. These events were not dose-related and did not prevent dose escalation. There was one serious adverse event of vomiting and confusion, and the most frequent treatment emergent adverse event observed was transient erythema in 4 out of 33 patients (12 percent).
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About the Phase 2 TRANSCEND Study
The global Phase 2 study is a single-arm, open label, multiple-ascending dose design that will evaluate safety, proof-of-concept and dose finding in adolescent and adult patients with anemia associated with non-transfusion-dependent (NTD) or transfusion-dependent (TD) beta-thalassemia. NTD patients will receive 12 weeks treatment with PTG-300 in escalating dose cohorts. The primary efficacy endpoint in NTD patients will be change in hemoglobin from baseline. TD patients will receive 16 weeks treatment with PTG-300 in escalating dose cohorts. The primary efficacy endpoint in TD patients will be a change in transfusion burden from baseline. All patients completing the trial will have the opportunity to participate in an open-label extension for two years. Additional information on the PTG-300 beta-thalassemia study is available at https://clinicaltrials.gov/ct2/show/NCT03802201.
PTG-300 is an injectable hepcidin mimetic in clinical development for the potential treatment of beta thalassemia and polycythemia vera. Hepcidin is a natural peptide hormone that is a critical regulator 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 addressed with 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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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 of PTG-300 as a possible treatment for TD beta-thalassemia, the Company's success at finding appropriate doses of PTG-300 for the treatment of beta-thalassemia, the results of future studies for the treatment of beta-thalassemia, the potential utility of PTG-300 in blood disorders including beta-thalassemia, polycythemia vera, and hereditary hemochromatosis, as well as myelodysplastic syndromes, the Company's ability to fund its clinical trials, results of the Phase 2 TRANSCEND Study,, the potential of PTG-200 and PN-943 as possible treatments for inflammatory bowel disease, the initiation of and enrollment of patients in our clinical trials, the results of clinical trials and the outlook for our other programs. In some cases, you can identify these statements by forward-looking words such as "plan," "will," "expect," "potential," 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 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. Additional information concerning these and other risk factors affecting our business can be found in our periodic filings with the
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