Protagonist Therapeutics (PTGX) investor relations material

Key clinical and regulatory updates

• Achieved breakthrough designation for rusfertide in polycythemia vera after strong phase 3 results, meeting primary and all secondary endpoints, including patient-reported outcomes.

• Phase 3 data presented at ASCO plenary, with independent discussant calling it potentially practice-changing and standard of care.

• NDA filing for rusfertide expected by year-end, with potential approval in about a year; priority review could shorten this timeline.

• ICO (oral IL-23 blocker) NDA filed for psoriasis; phase 3 data show head-to-head superiority over BMS’s Ducravacitinib and strong adolescent data.

• ICO also advancing in IBD, with phase 2 UC data showing 30% clinical remission at high dose, and phase 3 studies planned for UC and Crohn’s.

Market opportunity and strategic decisions

• Polycythemia vera market in the US estimated at 150,000+ patients, with about 50,000 as the target population for rusfertide.

• Revenue forecast for rusfertide is $1–2 billion, with expectations to reach the higher end.

• Opt-in/opt-out decision with Takeda due 4–7 months after NDA filing; opting out yields $425M in upfront and milestone payments plus 14–29% royalties.

• Opt-out strategy would enable significant reinvestment in internal R&D, external innovation, and potential share buybacks.

Pipeline innovation and differentiation

• Oral hepcidin program to be nominated by year-end, fully owned, with potential beyond PV, including other indications.

• 477 program in obesity focuses on differentiation, offering both oral and subcutaneous options, with preclinical data suggesting strong efficacy.

• 881 (oral IL-17 blocker) offers high potency and unique triple isoform blockade, with clinical studies initiated in Q4.

• Emphasis on platform validation, leveraging both internal and external innovation, and maintaining a differentiated approach across all assets.