Mirati Therapeutics Inc. (Nasdaq: MRTX) has announced that mocetinostat, the company's spectrum selective HDAC inhibitor, has been granted orphan drug designation by the U.S. Food & Drug Administration as a treatment for myelodysplastic syndrome (MDS).
Mocetinostat is being developed in phase 2 clinical studies in combination with Vidaza as a treatment for intermediate and high-risk MDS. A single agent treatment is being studied in patients with diffuse large B-cell lymphoma (DLBCL) and bladder cancer targeting specific genetic mutations in histone acetylation that increase the likelihood of response in tumor cells.
“Orphan designation is an important piece of the development plan for mocetinostat as we evaluate combination and single agent clinical development opportunities for the program,” said Dr. Charles Baum, president and CEO of Mirati. “We are excited about the opportunity to identify and select patients whose cancers may be especially sensitive to mocetinostat, and we expect to have initial data from phase 2 studies by the end of the year, which will allow us to move quickly into a registration path.”
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