**Takeda and Protagonist Therapeutics have shared positive top-line results from a phase 3 trial of rusfertide in the rare blood cancer polycythaemia vera (PV).**
The ongoing VERIFY study has been evaluating rusfertide against placebo as an add-on to standard of care treatment in phlebotomy-dependent PV patients.
As many as 160,000 people in the US are affected by PV, which causes the body to produce too many red blood cells and increases the risk of life-threatening cardiovascular and thrombotic events.
Many patients require treatment with cytoreductive therapies as well as regular phlebotomies, a process of removing blood to manage elevated haematocrit levels. Phlebotomies can, however, be burdensome and exacerbate symptoms such as severe fatigue and iron deficiency.
Rusfertide, which Takeda [gained rights to](https://pmlive.com/pharma_news/takeda_gains_rights_to_protagonists_blood_disorder_asset_in_deal_worth_over_300m_1507781/) last year, is thought to regulate iron homeostasis and control the absorption, storage and distribution of iron in the body.
VERIFY met its primary endpoint, with 77% of rusfertide-treated patients achieving a clinical response, defined as the absence of phlebotomy eligibility, compared to 33% of those who received placebo during weeks 20 to 32.
The trial met its first key secondary endpoint, which is the pre-specified primary endpoint for EU regulators, with a mean of 0.5 phlebotomies per patient in the rusfertide arm compared to 1.8 phlebotomies per patient in the placebo group during weeks zero to 32.
The remaining three key secondary endpoints of haematocrit control and patient-reported outcomes were also achieved, and rusfertide was generally well tolerated with no new safety findings observed.
Andy Plump, president of research and development at Takeda, 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.”
Also commenting on the readout, Protagonist’s president and chief executive officer, Dinesh Patel, said: “The totality of impressive clinical data to date shows that rusfertide has the potential for meaningful positive impact on the lives of patients with PV. We look forward to working with our partner, Takeda, to submit our findings to the regulatory agencies.”
Takeda and Protagonist announced a worldwide license and collaboration agreement for the development and commercialisation of rusfertide in January 2024.
Following these positive results, Protagonist will now receive a $25m milestone payment, which is payable following completion of the VERIFY clinical study report.