Protagonist Therapeutics is facing a significant decision following the submission of a new drug application (NDA) for rusfertide, its rare blood cancer therapy, by partner Takeda at the end of December. The move has triggered a three-month window in which Protagonist must choose whether to continue co-developing the drug on equal terms or opt out.
If Protagonist decides to opt out, it stands to receive $400 million in payments, increased milestone payments, and tiered royalties ranging from 14% to 29% on global sales. This would also shift full responsibility for development and commercialization to Takeda.
“Now the clock is ticking,” said CEO Dinesh Patel during an interview with BioSpace at the J.P. Morgan Healthcare Conference in San Francisco.
Patel indicated that Protagonist is leaning toward opting out due to favorable economic terms. “What we have started expressing is we are strongly leaning towards opting out,” he said. “And the reason for that is like the economics around opting out are just amazing.”
Rusfertide’s strong performance in clinical trials supports this stance. The drug targets polycythemia vera, a rare cancer characterized by overproduction of red blood cells in bone marrow. Current treatments often involve removing blood from patients, which can lead to complications such as fatigue and visual disturbances.
In last year’s Phase III VERIFY trial, rusfertide reduced patients’ need for blood withdrawals and lowered hemocrit levels compared to placebo while meeting secondary endpoints related to symptom improvement and treatment efficacy.
“All of a sudden it’s like, oh, this could be a big drug,” Patel commented.
The success of these results means that Protagonist could receive up to 29% royalties if annual sales exceed $1.5 billion. Takeda would manage manufacturing, sales operations, and direct-to-consumer engagement efforts.
“Without lifting a finger, I’m getting such a big share,” Patel noted.
The U.S. Food and Drug Administration (FDA) has granted priority review status for rusfertide; Patel expects an agency decision within months or by August.
Beyond rusfertide, Protagonist maintains additional partnerships and product candidates before regulators. It collaborates with Johnson & Johnson on icotrokinra—an IL-23 receptor blocker intended for multiple immunology and inflammatory diseases—which showed promising Phase III results in plaque psoriasis last March. An NDA was filed last July with regulatory decisions anticipated mid-year; both J&J and Takeda are preparing launches should approvals come through.
“There’s a very nice setup,” said Patel regarding ongoing launch preparations by partners.
Although rumors surfaced about Johnson & Johnson considering acquiring Protagonist last fall, no agreement has been reached so far; Patel declined comment on any potential deal but stated that commercializing products directly is not part of Protagonist’s strategy going forward. Instead, he emphasized continued focus on developing new treatments—including oral obesity drugs designed to compete with established pharmaceutical companies Novo Nordisk and Eli Lilly—and leveraging existing partnership structures for financial stability throughout future research phases.
“We are laser-focused on the future,” Patel concluded. “All the nuggets we have in the next wave of R&D.”
