Eyes return to the prize for Protagonist’s rusfertide
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|
|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.|