FDA has granted orphan-drug designation to Arno Therapeutics' investigational compound, AR-42, for the treatment of meningioma and schwannoma of the central nervous system.
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AR-42 is a broad-spectrum deacetylation inhibitor of both histone and non-histone proteins, which help in regulation of gene expression, cell growth and survival.
The compound has shown greater potency and activity in solid tumors and hematological malignancies when compared to the approved HDAC inhibitor, vorinostat.
Meningioma and schwannoma are benign tumors that can present in different locations within the brain and the spinal cord and may cause substantial morbidity for those affected individuals.
Arno president and CEO Glenn Mattes said AR-42 has shown promising activity in preclinical studies, which have encouraged them to pursue its clinical development in a variety of tumor types.
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