Clinical DataA Nstat monotherapy cohort, required by the FDA, showed limited efficacy with only a 10% overall response rate in treated patients.
Drug SafetyInvestor concerns arose due to a serious adverse event related to the drug that resulted in a patient's death from pancytopenia and sepsis.
Regulatory RisksRisks include potential inability to generate further favorable clinical data, failure to secure accelerated FDA approval, and possible poorer market uptake due to competition or other factors.