Protagonist Therapeutics(PTGX)2025-06-02 13:00Summary of Protagonist Therapeutics Inc (PTGX) Conference Call - June 02, 2025 Company Overview - Company: Protagonist Therapeutics Inc (PTGX) - Focus: Discovery and development of peptide therapeutics - Key Products: Ruspatercept (Respotec) and Iotrokinra, both nearing NDA filing Industry Context - Industry: Biotechnology, specifically focused on treatments for polycythemia vera (PV) - Collaboration: Partnership with Takeda Pharmaceuticals for co-development and commercialization of ruspatercept Key Findings from the VERIFI Study - Study Design: Phase 3 randomized double-blind placebo-controlled study of ruspatercept in patients with phlebotomy-dependent PV - Primary Endpoint: 77% of ruspatercept patients achieved clinical response compared to 33% in the placebo group, with a p-value of <0.0001 [14] - Secondary Endpoints: - Mean number of phlebotomies: 0.5 for ruspatercept vs. 1.8 for placebo, p-value <0.0001 [18] - 73% of ruspatercept patients had zero phlebotomies during the treatment period [19] - Hematocrit control: Ruspatercept maintained hematocrit below 45% significantly better than placebo [20] - Patient-Reported Outcomes (PROs): - Significant improvement in fatigue (PROMIS Fatigue Questionnaire) with a p-value of 0.03 [22] - Improvement in symptoms measured by MFSAF with a p-value of 0.24 [24] Safety Profile - Adverse Events: Generally well tolerated, with most adverse events being mild to moderate [26] - Cancer Incidence: No new safety signals; cancer rates were similar between treatment and placebo groups [28] Market Potential - Revenue Potential: Takeda estimates 2 billion market potential for ruspatercept in PV [32] - Adoption Strategy: Ruspatercept is positioned to potentially become a new standard of care in PV treatment [31] Regulatory and Commercial Strategy - NDA Filing: Planned for Q4 2025 [39] - Commercial Readiness: Takeda is preparing for the launch and marketing of ruspatercept [38] Additional Insights - Patient Demographics: The study included a diverse patient population, with a notable percentage having high-risk disease characteristics [11] - Future Studies: Ongoing analysis of long-term effects and potential combination therapies with ruspatercept [46][51] - Payer Considerations: The unique mechanism of action and patient experience improvements are expected to resonate well with payers [82] Conclusion - Overall Assessment: The VERIFI study results are seen as practice-changing, with ruspatercept showing significant efficacy, safety, and potential to improve patient quality of life in the treatment of PV [29][30]