Why This Transfusion-Dependent Beta-Thalassemia Trial Is Taking Off

Health and Healthcare

Published: December 4, 2019 12:00 pm

Last Updated: January 6, 2020 12:46 am

Protagonist Therapeutics Inc. (NASDAQ: PTGX) shares jumped on Wednesday after the company announced preliminary results from its midstage study for patients with transfusion-dependent beta-thalassemia. Specifically, the results come from the firm’s Transcend Phase 2 study of PTG-300.

The most severe type of transfusion-dependent beta-thalassemia is characterized by severe anemia and a lifelong dependence on red blood cell transfusions that can lead to iron overload. Ultimately, the accumulation of iron can be toxic and lead to progressive multi-organ damage.

In the study, dose-related drug exposure and reductions from baseline serum iron and transferrin saturation levels were observed, with significant reductions at the 40 mg and 80 mg weekly doses.

These early results suggest the potential of finding an appropriate dose of PTG-300 for continued development in the treatment of beta-thalassemia.

The results from further study with additional dose regimens and longer follow-up, with clinical efficacy results are expected in 2020.

Dinesh V. Patel, Ph.D., Protagonist president and CEO, 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. 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.

Shares of Protagonist Therapeutics traded up about 53% on Wednesday to $7.18, in a 52-week range of $4.47 to $16.67. The consensus price target is $20.17.