Clinical Data A total of six trials have been run with GALE-401, five Phase 1 trials in healthy volunteers (N=98), and one Phase 2 single arm, open label pilot study in MPN patients (N=18). The poster, entitled, “Anagrelide Controlled Release (GALE-401) Safety Profile Consistently Well Tolerated in Myeloproliferative Neoplasms Patients and Healthy Volunteers” was designed to characterize the safety profile of GALE-401 in all subjects treated to date. The results demonstrated that GALE-401 is well tolerated in MPN patients as well as in healthy volunteers. In the Phase 2 study, fewer moderate to severe (Grade 3/4) adverse events (AEs) and fewer AEs per patient (2.3 vs. 3.3) were observed with GALE-401 compared to what has been reported previously with the immediate release (IR) formulation.1 Additionally, for a small subset of subjects treated in the Phase 2 study who were intolerant to anagrelide IR (n=5), GALE-401 appears to offer a longer duration on therapy compared to previous administration of anagrelide IR. Therefore, a randomized trial comparing GALE-401 vs. anagrelide IR in anagrelide naïve subjects, alternatively or together with a trial evaluating anagrelide IR intolerant subjects is warranted.

In the healthy volunteer studies, single and multiple doses of GALE-401 were safe and well tolerated and there were no clinically relevant changes in vital signs, electrocardiograms, and safety laboratory parameters other than a reduction in platelet counts. The most frequent treatment emergent adverse events reported included headache, pain in extremities or back, palpitations and gastrointestinal disturbances. In the Phase 2 MPN trial, patients treated with GALE-401 exhibited fewer of the more common adverse events associated with anagrelide IR (cardiac; general; gastrointestinal; respiratory, thoracic, and mediastinal; skin and subcutaneous tissue; nervous system). Some of the less common AEs of anagrelide IR were comparatively more frequent for GALE-401 (vascular; hepatobiliary; blood and lymphatic).