FRANKFURT, Germany — Protagonist Therapeutics has run into some speed bumps as it’s developed its medicine for a rare blood cancer characterized by the overproduction of red blood cells.
In 2021, the Food and Drug Administration put studies of the drug, rusfertide, on hold after a mouse study raised concerns about skin malignancies (the hold was lifted quickly, after less than a month). Then last year, citing concerns about malignancies, the FDA yanked the therapy’s “breakthrough” designation, which can expedite a medicine’s review.
But the therapy received some primetime attention here at the European Hematology Association’s annual meeting over the weekend. In presentations at a press briefing and at a session for late-breaking studies, researchers outlined what they portrayed as promising Phase 2 results indicating rusfertide helped stabilize red blood cells at healthier levels and improved patients’ symptoms, including fatigue and brain fog. A Phase 3 trial of rusfertide is ongoing.
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