Takeda and Protagonist Therapeutics have reported positive topline outcomes from their Phase III VERIFY trial of rusfertide as an add-on to standard of care treatment for polycythaemia vera (PV) in phlebotomy-dependent individuals.
The randomised trial met its primary endpoint, demonstrating a significant increase in clinical responders in the therapy arm, with 77% of these subjects achieving an absence of phlebotomy eligibility in weeks 20-32 against 33% of those given placebo.
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All the key secondary endpoints were also met, including the number of phlebotomies for each subject, which was the pre-specified primary endpoint for the regulators in the European Union (EU).
A mean result of 0.5 phlebotomies for each subject was recorded in the therapy arm for the first 32 weeks, compared with 1.8 phlebotomies for each subject in the placebo arm.
Rusfertide’s safety profile was consistent with previous studies, with most adverse events being mild injection site reactions.
Takeda research and development president Andy Plump said: “We are encouraged by these results and excited about the potential of rusfertide to help patients living with PV.
“These patients may experience a high treatment burden, and severe symptoms can impact their quality of life.
“We are deeply committed to bringing additional treatment options to those living with blood cancers, including myeloid cancers such as PV.”
Individuals with PV often face a high risk of cardiovascular and thrombotic events, with most of them requiring regular phlebotomy, a blood-removing process for managing increased haematocrit levels that occur due to an excess of red blood cells.
Rusfertide’s ability to decrease phlebotomy frequency could significantly improve the quality of life for these patients by relieving associated symptoms and treatment burdens.
Under the licensing agreement with Takeda, Protagonist is set to receive a $25m milestone payment following the positive trial outcomes and the completion of the VERIFY clinical study report.
