Protagonist (PTGX) hits on all key endpoints in PV trial
Protagonist Therapeutics (NASDAQ:PTGX) said on March 3, 2025, that its phase III Verify trial testing rusfertide in phlebotomy-dependent polycythemia vera (PV) patients met its primary endpoint and all four key secondary endpoints.
For the primary endpoint of the proportion of patients achieving a response, was defined as the absence of phlebotomy eligibility, Protagonist said 77% of rusfertide-treated patients with PV achieved a response compared to those who received placebo (33%) during weeks 20-32 (p<0.0001). The trial also satisfied the key secondary endpoint with a mean of 0.5 phlebotomies per patient in the rusfertide arm compared to 1.8 phlebotomies per patient in the placebo arm during weeks 0-32 (p<0.0001).
PV is a rare blood cancer that results from excess production of red blood cells (RBCs); the disease afflicts 160,000 people in the US. Many PV patients are diagnosed after age 60, but PV also develops in younger people. Without treatment, PV can be life threatening as it can induce unexpected blood clots, leading to a heart attack or stroke. The disease also may progress into myelofibrosis or leukemia. The most common PV treatment is to receive regular blood withdrawals via a needle inserted into a vein (phlebotomy).
In January 2024, Takeda Pharmaceutical Company Limited (NYSE:TAK), made an upfront cash payment of $300 million for rights to rusfertide outside of the US. Takeda and Protagonist share profits and losses on US sales; Protagonist will receive royalties on sales outside the US.
Because of the March 3 rusfertide results, Takeda will make a $25 million milestone payment to Protagonist.
We issued a report on the phase III rusfertide trial in October 2024. At the time of the report, Protagonist’s stock traded around $45 per share.
While Protagonist’s shares initially climbed ~8.5% on the rusfertide data, the market slide of the past two days resulted in Protagonist’s stock closing at $38.66 per share on March 4.
Disclosures:
Discontinuing coverage and exiting part of positions in PTGX.