Mgmt Thoughts On Upcoming PN-881 Data In Q3 & Ico/Rusfertide Launches

Research evidence excerpt

Mgmt Thoughts On Upcoming PN-881 Data In Q3 & Ico/Rusfertide Launches

ibutor to company financials. Ultimately the company

estimates that while the amount of royalties received may not be broken out in its own

earnings report in Q2, Q3 is a more realistic expectation for these figures.

Rusfertide Remains On Track For An August PDUFA, With Takeda Positioned To Drive

Commercial Uptake

Regarding rusfertide, recall that the NDA was submitted at the end of December, with

an approval decision expected in late August around the PDUFA date. Upon approval,

management expects to host an investor call similar to the one held following Ico's approval.

Management noted that Takeda's launch strategy and messaging will be the most important

determinants of commercial expectations. While Takeda has not yet disclosed pricing

assumptions, management highlighted that therapies in rare hematology indications have

generally commanded premium pricing (e.g., Besremi, which carries a WAC of ~$10,400

per dose, implying an annual treatment cost of ~$270K per patient at the labeled Q2W

maintenance schedule).

Management also highlighted encouraging updates from the recent EHA presentation,

including data showing that more than 40% of patients in the long-term extension either

eliminated or reduced their use of hydroxyurea after achieving the primary endpoint.

Management believes this finding is particularly meaningful given physicians' longstanding

desire to minimize hydroxyurea exposure when possible. Additional analyses also provided

greater detail on symptom improvement across individual symptom categories. These

incremental datasets are very meaningful to physicians when making prescribing decisions,

which underscores confidence in a strong launch upon approval.