While the FDA mulls whether to approve Protagonist Therapeutics’ hematology asset, the biotech is thinking it will pocket $400 million rather than split U.S. profits with its partner Takeda.
The Japanese pharma paid $300 million back in 2024 to license rusfertide, an injectable hepcidin mimetic designed to treat a rare chronic blood disorder called polycythemia vera (PV). The deal terms included splitting U.S. profits for the drug equally between the two companies. Since then, rusfertide has sailed through a pivotal phase 3 study, and the companies submitted the drug for FDA approval at the start of this year.
Rusfertide mimics the action of hepcidin, a natural hormone that regulates iron homeostasis and red blood cell production. Protagonist has touted the drug as a way for PV patients to reduce or even eliminate the need for regular phlebotomy.
The approval submission Jan. 6 started the clock ticking on an 120-day period, after which Protagonist has a 90-day window to decide whether to stick with the 50/50 U.S. profit split set out in the original agreement or to opt out. If Protagonist goes down the second route, it will be eligible to receive opt-out payments of up to $400 million as well as “enhanced milestone payments” and 14%-19% tiered royalties on worldwide sales of rusfertide.
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The biotech had been weighing the two options, but, in its fourth-quarter earnings release yesterday, the company said it “currently expect[s] to exercise” its opt-out right in the second quarter of 2026.
Protagonist entered the year with $646 million in the bank. CEO Dinesh Patel, Ph.D., said in yesterday’s release that the company is “well equipped to fund all our internal wholly owned programs to clinical proof-of-concept with the cash on hand and potential revenue from the partnered assets.”
Protagonist is also awaiting an FDA decision on its Johnson & Johnson-partnered once-daily oral peptide icotrokinra for psoriasis.
