Protagonist Therapeutics (PTGX) Update Summary Company Overview - Protagonist Therapeutics is focused on peptide therapeutics, with a history of 15-18 years in the field [4][5] - The company is advancing two late-stage assets towards NDA filing in 2025: Icotropinirap for psoriasis and Ruscotag for polycythemia vera (PV) [5][11] Industry Context - The presentation centers on the treatment of polycythemia vera (PV), a chronic myeloproliferative neoplasm characterized by the overproduction of red blood cells [25][26] - PV affects approximately 150,000-250,000 patients in the U.S., with a median age of diagnosis at 61 years [27][28] Key Points on Ruscotag (Ruspatercept) - Ruspatercept is being evaluated in multiple studies, with Phase III results expected in March 2025 [6][11] - The drug is a hepcidin mimetic aimed at reducing red blood cell production by blocking ferroportin, thus decreasing iron delivery to the bone marrow [52][53] - The PACIFIC Phase II study showed rapid hematocrit control, with 85% of patients achieving levels below 45% within eight weeks [56][58] - The REVIVE Phase II study demonstrated a significant response rate, with 69% of patients on ruspatercept not being phlebotomy eligible compared to 15% in the placebo group [73] Clinical Development and Pipeline - The clinical development plan includes three studies: PACIFIC, REVIVE, and the upcoming VERIFI Phase III study [20][21] - The VERIFI study is designed to evaluate the efficacy of ruspatercept against standard care, with a focus on maintaining hematocrit levels below 45% [91][92] Financial Position - Protagonist has a strong cash runway projected through the end of 2028, bolstered by partnerships with Johnson & Johnson and Takeda Pharmaceuticals [13][14] - Potential milestone earnings of $200 million to $600 million are anticipated, along with future royalties from the assets [14] Safety and Efficacy - The safety profile of ruspatercept is comparable to other studies, with injection site reactions being the most common adverse event [57][86] - Long-term safety data is being collected through the open-label extension of the REVIVE study, with a focus on maintaining patient adherence to therapy [88] Unmet Needs and Market Opportunity - There is a significant unmet need in the treatment of PV, with current therapies associated with risks of thrombosis and secondary malignancies [31][32][86] - Ruspatercept aims to address these needs by providing a new therapeutic option that can improve quality of life for patients [77][98] Conclusion - Protagonist Therapeutics is positioned to make a significant impact in the treatment of polycythemia vera with ruspatercept, supported by a robust clinical pipeline and strong financial backing [11][14]