KDFX (Deferasirox)

KDFX is an enhanced KinetiSol® reformulation of the approved drug deferasirox (the active ingredient in Exjade® & Jadenu®) used for treating iron overload disorder. Iron overload disorder is an orphan disease characterized by excess iron in the blood. It can be hereditary or, more commonly, secondary to chronic blood transfusions. Patients with beta-thalassemia, sickle cell syndrome or myelodysplastic syndromes often receive red blood cell transfusions as part of their disease treatment. The extra iron from transfused red blood cells over time accumulates in patients and can become life threatening. To compensate, patients are given iron chelators that remove this excess iron from their bodies.

KDFX is an improved formulation of deferasirox designed to:

 

  • Work in the 30% of deferasirox patients who don’t absorb the current formulations well
  • Have reduced inter- and intra-patient variability
  • Be taken with or without food as the patient desires

 

Clinical development of KDFX

DST-0509 was tested in 2 phase I studies, Study 101 and 102. Study 101, a single dose study in healthy volunteers, demonstrated improvements in the bioavailability of DST-0509 compared to Jadenu®  at a dose of 14 mg/kg. Furthermore, DST-0509 is the first deferasirox formulation to show no differences in total absorption between high-fat fed and fasted states.

Study 102 was a randomized, single dose, 3-period, 3-sequence crossover, oral bioavailability study of DST-0509 compared to Exjade® and Jadenu® in fasted healthy volunteers at higher doses (25 – 33 mg/kg). The results showed less variability in Cmax and AUC with DST-0509 versus Exjade®, and meaningfully enhanced absorption versus Exjade® and Jadenu®.

KDFX phase II studies have just been completed and results are expected to be published shortly. The enhanced bioavailability seen in these studies translates into superior iron control in patients not achieving target iron control on current deferasirox treatments.