Eyes return to the prize for Protagonist’s rusfertide

Snippets

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.  

Rusfertide (PTG-300) clinical programme in disorders of iron overload/excessive red blood cell production
Setting  Trial Next steps 
Polycythemia vera (rare chronic blood disorder that affects about 160,000 US patients) Phase 2 in phlebotomy-requiring PV Latest cut of data could be at Ash; previous presentation at EHA
Phase 2 in PV patients with elevated haematocrit Data could come 2022
Hereditary haemochromatosis (inherited iron overload blood disorder affecting over a million US people)  Open label phase 2 study fully recruited Data to be presented in oral session at AASLD November 13th
Third indication To be announced by YE 2021
Source: Company statements & clinicaltrials.gov. 

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