Newark, Calif.-based Protagonist Therapeutics' shares traded 70% higher today after the firm reported preliminary Phase 2 results for Hepcidin Mimetic PTG-300 in the treatment of transfusion dependent beta-thalassemia.
Clinical-stage biopharmaceutical company Protagonist Therapeutics Inc. (PTGX:NASDAQ)announced preliminary results from the company's ongoing Phase 2 TRANSCEND study of PTG-300 in patients with transfusion-dependent beta-thalassemia. The firm noted that in the trial, dose-related drug exposure and significant reductions from baseline serum iron and transferrin saturation levels were observed providing the first evidence of pharmacodynamic effects in patients.
Protagonist's Chief Medical Officer Samuel Saks, M.D., commented, "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...These early results suggest the potential of finding an appropriate dose of PTG-300 for continued development in the treatment of beta-thalassemia...We look forward to the results from further study with additional dose regimens and longer follow-up, with clinical efficacy results expected in 2020."
Ashutosh Lal, M.D., program director of the Comprehensive Thalassemia Center at the UCSF Benioff Children's Hospital, Oakland, and PTG-300 beta-thalassemia study investigator, remarked, "Treatment options for patients with beta-thalassemia are limited and the complications associated with transfusion are serious...The TRANSCEND trial is examining whether constraining iron availability improves endogenous hemoglobin synthesis in patients with beta-thalassemia, an endpoint for which there exists considerable preclinical evidence. These results demonstrating the pharmacodynamic activity of PTG-300 in reducing TSAT, though preliminary, warrant continued evaluation of PTG-300 for the potential treatment of beta-thalassemia."
The company's President and CEO Dinesh V. Patel, Ph.D., added "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...We are encouraged by these findings and are continuing with our original plans of conducting clinical proof-of-concept studies with PTG-300 in different blood disorders such as beta-thalassemia, polycythemia vera, hereditary hemochromatosis, and an investigator sponsored study in myelodysplastic syndromes. We are well financed to conduct these studies and our corporate objective is to make data-driven decisions in 2020, with the intent of choosing our first clinical indication for a potential pivotal study to begin in 2021."
The firm indicated that PTG-300 was well-tolerated in the study and systemic and non dose-related adverse events were only mild to moderate in severity and typical for patients with TD beta-thalassemia.
The company explains in the release that "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...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."
Protagonist Therapeutics, headquartered in Newark, Calif., describes its business as "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." The company's drug candidate pipeline includes PTG-300 used in the treatment of iron overload anemia and related rare blood diseases including beta-thalassemia and polycythemia vera; PTG-200, which is currently in Phase 2 clinical development for the potential treatment of inflammatory bowel disease, with Crohn's disease as the initial indication; and PN-943, which is in clinical development for the potential treatment of inflammatory bowel disease, with a Phase 2 ulcerative colitis study expected to commence in Q2/20.
Protagonist Therapeutics has a market capitalization of about $127.6 million with around 27.2 million shares outstanding and a short interest of approximately 3.74%. PTGX shares opened higher today at $5.50 (+$0.81, +17.27%) over yesterday's closing price of $4.69. The stock has traded today between $5.40 and $8.51/share and at present is trading at $8.06 (+$3.37, +71.43%).
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