Astex Pharmaceuticals announced that the Food and Drug Administration (FDA) has granted Orphan Drug designation to guadecitabine (SGI-110) for the potential treatment of myelodysplastic syndromes (MDS) or acute myeloid leukemia (AML).

Guadecitabine has been evaluated in multiple Phase 1 and Phase 2 trials, including the recently completed Phase 1/2 study which investigated over 400 patients with MDS or AML in four different patient populations: treatment naïve and relapsed/refractory AML and MDS. The trial demonstrated that guadecitabine was clinically active and well tolerated in all four patient groups.

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Currently, the ASTRAL-1 study, a large, global, randomized trial, is evaluating guadecitabine in 800 treatment-naïve AML patients who are unsuitable for intensive induction chemotherapy. The trial compares guadecitabine with physician’s choice of low-dose cytarabine, decitabine or azacitidine.

Guadecitabine is a novel next-generation, small molecule DNA hypomethylating agent formulated as a single, small volume, subcutaneous injection designed to deliver longer exposure to the active moiety, decitabine.

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