Investors appear disappointed that Protagonist Therapeutics (PTGX -25.5%) did not announce more definitive results from a Phase 2 clinical trial, TRANSCEND, evaluating PTG-300 in patients with transfusion-dependent beta thalassemia, a blood disorder characterized by chronic anemia and iron overload.
The company says preliminary data showed dose-related drug exposure and reductions in serum iron and transferrin saturation levels from baseline for the 40 mg and 80 mg weekly doses. There were some clinical responders as measured by reductions in transfusion burden, but “continued evaluation at higher doses will be required” to definitively assess the rate and durability of the effects. Additional data, including results from longer follow-up periods and additional dosing regimens, should be available in 2020.
On the safety front, the most frequent treatment-related adverse event was transient erythema (reddening of the skin) (12%, n=4/33).
PTG-300 is an injectable hepcidin mimetic peptide. Hepcidin is a peptide hormone that plays a key role in regulating iron absorption, recycling and utilization. It has Orphan Drug and Fast Track status in the U.S. for the indication.
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.