What is Hematide?

Background: Hematide is an innovative, synthetic, pegylated, peptidic ESA that binds to and activates the erythropoietin (EPO) receptor. Although its amino acid sequence is unrelated to that of EPO, Hematide demonstrates in vitro and in vivo activities comparable to EPO and an extended serum half life in animal studies. This may allow less frequent dosing in patients with chronic anemia relative to EPO. Methods: A randomized double-blind placebo controlled dose escalation study was conducted to assess safety, PK and PD of Hematide administered as a single IV infusion to male NHV. Up to 7 cohorts of 7 subjects each (5 on Hematide and 2 on placebo) were planned with doses escalated unless or until stopping rules were met. Results: Three dose cohorts have been completed involving 21 NHV. As of this date, the study remains blinded and we assume increases in reticulocytes and hemoglobin (Hb) are due to Hematide. Injections were well tolerated with report of only a few Grade 1 adverse events and no pattern across cohorts. Reticulocyte increases were dose proportional; average reticulocyte peak concentrations (+/- SE) were 148 (+/- 19); 186 +/- (20); 230 (+/- 22) 10⁹/L among the NHV with a reticulocyte response in Cohort 1 (n=4), Cohort 2 (n=5), and Cohort 3 (n=5) respectively, compared to 59 (+/- 5) 10⁹/L among the 7 (pooled) non-responding subjects. In Cohort 3, an increase in Hb was seen in 5 subjects. The mean difference in changes from baseline Hb between these 5 responding and the 2 non-responding subjects was 1.0 g/dL at day 9 (p = 0.014) and remained detectable as of Day 35. Unblinded results, including PK data, will be presented. Conclusions: A single IV injection of Hematide administered to NHV was well tolerated and demonstrated potent dose dependent erythropoietic activity. These data will be used to plan Phase 2 studies of Hematide in patients with anemia related to cancer and renal failure.

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