Slide 1: Phase 3 VERIFY Study Topline Results
Phase 3 VERIFY Study Topline Results March 3, 2025 Dinesh V. Patel, Ph.D. Director, President, and CEO Arturo Molina, M.D., M.S. Chief Medical Officer Protagonist Therapeutics, Inc. Newark, CA 1
Slide 2: Forward-looking Statements
Forward - looking Statements 2 This presentation and the accompanying oral presentation contain forward - looking statements made pursuant to the safe harbor pro visions of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in thi
Slide 3: A Peptide Therapeutics Company
Potency Peptide Chemistry SAR, Leads Phage Libraries Hits Computational Vectrix, Clusters Vectrix ® Clusters 1 2 3 POTENCY Oral Stability Peptidomimetics GI Assays GI-Restricted Targeted GI absorption & delivery Systemic Availability Formulation SAR, Transport 4 5 6 STABILITY Computational Vectrix,
Slide 4: Polycythemia Vera (PV) Rusfertide Clinical Development Program
4 • PV is a rare myeloproliferative neoplasm characterized by excessive production of red blood cells 1 – Elevated hematocrit ( Hct ) >45% 2 – Primary treatment goal is to maintain Hct <45% 3,4 Polycythemia Vera (PV) Rusfertide Clinical Development Program Key Milestones Phase 3 Phase 2 Phase 1 DISC
Slide 5: Phase 3 VERIFY Study – Primary Efficacy and Safety Analysis
Phase 3 VERIFY Study – Primary Efficacy and Safety Analysis 5 Positive study results: – Efficacy ✓ – Symptoms ✓ – Safety ✓
Slide 6: Phase 3 VERIFY Study is a Global Trial
Phase 3 VERIFY Study is a Global Trial 6 Europe Austria, Belgium, Czechia, France, Germany, Hungary, Italy, Netherlands, Poland, Spain, UK Turkey Chil e Canada United States Mexico Hong Kong Australia Israel 293 patients randomized in VERIFY across 19 countries
Slide 7: Rusfertide Phase 3 VERIFY Study
7 Rusfertide Phase 3 VERIFY Study Primary endpoint: 3 Wks 20 - 32 1. Clinical Response rusfertide vs placebo Key 2 ƒ endpoints: Wks 0 - 32 1. Average number of PHLs 4 2. Proportion of patients with Hct <45% 3. Average PROMIS Fatigue SF - 8a Score 5 4. Average MFSAF Total Symptom Score 6 * Ongoing th
Slide 8: VERIFY: Primary Efficacy Endpoint — Response Rate
8 The primary endpoint was met – Significantly higher proportion of clinical responders 1 among rusfertide - treated PV patients ( 77% ) compared to those who received placebo ( 33% ) during weeks 20 - 32; p<0.0001 VERIFY : Primary Efficacy Endpoint — Response Rate 1 Definition of “clinical response
Slide 9: VERIFY: Key Secondary Endpoint #1 — Phlebotomy Rate
• The first key secondary endpoint (pre - specified primary endpoint for European regulatory agencies) was also met • Mean number of phlebotomies per patient in the rusfertide arm was 0.5, compared to 1.8 phlebotomies in the placebo arm during weeks 0 - 32; p<0.0001 VERIFY : Key Secondary Endpoint #
Slide 10: VERIFY: Other Key Secondary Endpoints Also Achieved Statistical Significance
VERIFY: Other Key Secondary Endpoints Also Achieved Statistical Significance 10 • Hematocrit control 1 • Patient - reported outcome: PROMIS Fatigue SF - 8a 2 • Patient - reported outcome: MFSAF TSS - 7 3 1 Proportion of patients with hematocrit <45% 2 Mean change from baseline to week 32 using PROMI
Slide 11: VERIFY: Overall Summary of Adverse Events (Weeks 0-32)
VERIFY: Overall Summary of Adverse Events (Weeks 0 - 32) 11 • Rusfertide was generally well tolerated • Safety data were in line with other rusfertide clinical studies; no new safety findings were observed • Majority of adverse events were grade 1 - 2 injection site reactions • All serious adverse e
Slide 12: VERIFY: Primary Efficacy and Safety Analysis Conclusions
12 • Study met primary endpoint with a significantly higher proportion of clinical responders on rusfertide compared to placebo during weeks 20 - 32 (p<0.0001) • Study met all four key secondary endpoints (weeks 0 - 32) – Phlebotomy rate - EU primary endpoint (p<0.0001) – Hematocrit control – Patien
Slide 13: Major Upcoming Catalysts in 2025
2H 2025 Q2 2025 Q1 2025 2024 □ NDA filing 4 □ PV day 1 □ Topline results 2 □ New Engl J Med, 2024; 390:723 - 35, REVIVE Ph2 □ Ph3 VERIFY enrollment completion Icotrokinra Expected Clinical Trial Initiations, Data Readouts and Development Candidate Nominations Major Upcoming Catalysts in 2025 13 Disc