Protagonist Therapeutics (PTGX) Investor Update summary

Key clinical results

• The phase III VERIFY study of Rusfertide in polycythemia vera met its primary and all four key secondary endpoints, demonstrating significant efficacy and symptom improvement based on patient-reported outcomes.

• 77% of Rusfertide-treated patients achieved clinical response versus 33% for placebo during weeks 20-32 (p<0.0001).

• Secondary endpoints included reduced phlebotomy rates, improved hematocrit control, and statistically significant improvements in fatigue and symptom scores.

• 73% of Rusfertide patients required zero phlebotomies over 32 weeks, compared to 22% in the placebo group; mean phlebotomy rate was 0.5 for Rusfertide vs 1.8 for placebo.

• Rusfertide is the first therapy to prospectively show significant improvement in patient-reported outcomes for PV.

Safety and tolerability

• Rusfertide was generally well tolerated, with most adverse events being mild injection site reactions.

• No new safety signals or serious adverse events attributed to Rusfertide were observed.

• No evidence of increased cancer risk in Rusfertide-treated patients compared to placebo.

Regulatory and commercial outlook

• The positive results support regulatory filings in both the US and Europe, with NDA submissions expected in 2025 and plans to work closely with Takeda on submissions.

• The study qualifies for a $25 million milestone payment from the partner; financial impact on Takeda for FY2024 is immaterial.

• Fast Track and Orphan Drug designations are in place, and rolling NDA submissions are being considered.

• Market potential is estimated at $1–$2 billion, with no downward revision to guidance expected.

• Collaboration agreement includes co-development and co-commercialization of Rusfertide.