Takeda and Protagonist Therapeutics, Inc. Enter into Agreement for Rusfertide
The FDA gave Protagonist Therapeutics’ rusfertide breakthrough designation last year. Now, the agency wants to take it back “based on observed malignancies,” triggering a 30% drop in Protagonist’s stock as investors scrambled to figure out the implications of the news.
When the FDA halted all clinical work with Protagonist's rusfertide over cancer concerns, investors feared the worst. The lifting of that hold a mere three weeks later therefore prompted a similarly dramatic reaction: the stock surged 94% yesterday. But the shares still sit some 25% below levels seen before the clinical hold. Aside from requiring enhanced monitoring for skin cancer, the FDA seems comfortable for the programme to continue largely as before. Such a swift resolution is reassuring, though confidence in rusfertide’s potential has clearly taken a knock. More data will help, and two presentations before year end are now keenly awaited. The first is at the Liver Meeting, concerning a second indication for the project: hereditary haemochromatosis, a rare inherited disorder that causes iron build-up. Phlebotomy is the effective but burdensome standard of care. The abstract points to a dramatic reduction in phlebotomies as well as improvements in various pharmacological markers. The latest cut from an ongoing phase 2 trial in polycythemia vera is also due, possibly at Ash. Protagoinst confirmed yesterday that it still plans to start phase 3 in polycythemia vera early next year.
Protagonist’s experimental drug rusfertide is cleared to resume dosing in all studies, the biotech announced Monday morning, paving the way for researchers to launch a Phase III trial in polycythemia vera early next year. The move comes exactly 25 days after the FDA halted rusfertide studies due to skin tumors arising in mice treated with the drug.
The US Food and Drug Administration (FDA) has lifted the full clinical hold on Protagonist Therapeutics’ study of its therapy, rusfertide.