Protagonist Therapeutics, Inc. · NASDAQ:PTGX
Protagonist Therapeutics — Technical Presentation (March 2025)
13 slides from Protagonist Therapeutics's technical presentation, Mar 3, 2025
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 This presentation and the accompanying oral presentation contain forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All statements other than statements of historical facts contained in this prese
Slide 3: A Peptide Therapeutics Company
A Peptide Therapeutics Company Two Late-Stage Assets with Potential for NDA Filings in 2025 • Biologically and commercially validated targets – Immunology & inflammation, hematology, and metabolic diseases • Protagonist peptides offer strong differentiation vs existing therapies Peptide Technology P
Slide 4: Rusfertide Clinical Development Program in Polycythemia Vera
Polycythemia Vera (PV) Rusfertide Clinical Development Program • PV is a rare myeloproliferative neoplasm characterized by excessive production of red blood cells1 – Elevated hematocrit (Hct) >45%2 – Primary treatment goal is to maintain Hct <45%3,4 DISC/Pre-Clinical Phase 1 Phase 2 Phase 3 Key Mile
Slide 5: Phase 3 VERIFY Study – Primary Efficacy and Safety Analysis
Phase 3 VERIFY Study – Primary Efficacy and Safety Analysis Positive study results: – Efficacy ✓ – Symptoms ✓ – Safety ✓ 5
Slide 6: Phase 3 VERIFY Study is a Global Trial
Phase 3 VERIFY Study is a Global Trial 293 patients randomized in VERIFY across 19 countries Europe Austria, Belgium, Czechia, France, Germany, Hungary, Italy, Netherlands, Poland, Spain, UK Canada United States Turkey Israel Hong Kong Mexico Chile Australia 6
Slide 7: Rusfertide Phase 3 VERIFY Study Clinical Study Design and Topline Results
Rusfertide Phase 3 VERIFY Study Clinical Study Design and Topline Results Primary endpoint:3 Wks 20-32 Part 1A: Double-Blind1,2 Part 1B: Open-Label1,2 1. Clinical Response rusfertide vs Durability of Response placebo Inclusion Criteria Dose titration 1° endpoint (Wks 32-52) Wks 0-20 Wks 20-32 ≥3 PHL
Slide 8: Primary Efficacy Endpoint—Response Rate
VERIFY: Primary Efficacy Endpoint—Response Rate Phlebotomy Eligibility (Week 20 through Week 32) The primary endpoint was met – Significantly higher proportion of clinical responders1 among rusfertide-treated PV patients (77%) compared to those who received placebo (33%) during weeks 20-32; p<0.0001
Slide 9: Key Secondary Endpoint #1—Phlebotomy Rate
VERIFY: Key Secondary Endpoint #1—Phlebotomy Rate Mean Number of Phlebotomies: Baseline through Week 32 • 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
Slide 10: Other Key Secondary Endpoints Also Achieved Statistical Significance
VERIFY: Other Key Secondary Endpoints Also Achieved Statistical Significance • Hematocrit control1 • Patient-reported outcome: PROMIS Fatigue SF-8a2 • Patient-reported outcome: MFSAF TSS-73 1 Proportion of patients with hematocrit <45% 2 Mean change from baseline to week 32 using PROMIS Fatigue SF-8
Slide 11: Overall Summary of Adverse Events (Weeks 0-32)
VERIFY: Overall Summary of Adverse Events (Weeks 0-32) • 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 events r
Slide 12: Primary Efficacy and Safety Analysis Conclusions
VERIFY: Primary Efficacy and Safety Analysis Conclusions • 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
Slide 13: Major Upcoming Catalysts in 2025
Major Upcoming Catalysts in 2025 Expected Clinical Trial Initiations, Data Readouts and Development Candidate Nominations 2024 Q1 2025 Q2 2025 2H 2025 ❖ New Engl J Med, 2024; ❖ PV day1 ✓ 390:723-35, REVIVE Ph2 Rusfertide ❖ Ph3 VERIFY enrollment completion ❖ Topline results2 ✓ ❖ NDA filing4 ❖ Medical
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