Mocetinostat Granted Orphan Drug for MDS

Mirati Therapeutics announced that the FDA has granted Orphan Drug designation to mocetinostat, a selective HDAC inhibitor for the treatment of myelodysplastic syndrome (MDS).

In addition to the ongoing Phase 2 clinical trials, 13 clinical trials have been completed, which enrolled over 400 patients with a variety of hematologic malignancies and solid tumors. Mocetinostat being developed in a Phase 2 dose confirmation study in combination with Vidaza as treatment for intermediate and high-risk MDS. Mirati also plans to initiate Phase 2 studies of mocetinostat as a single agent in patients with mutations in histone acetyl transferases in bladder cancer and diffuse large B-cell lymphoma (DLBCL).

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Initial data from the Phase 2 studies is expected by the end of 2014.

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