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HLX-1502 is an investigational oral small-molecule drug developed by Healx for the treatment of neurofibromatosis type 1 (NF1), particularly in patients with progressive and/or symptomatic plexiform neurofibroma. Discovered using Healx’s proprietary AI platform, HLX-1502 acts as a mitochondrial dysregulator, modulating mitochondrial function through various pathways to control tumor growth in NF1 cells. The drug demonstrates nerve-specific accumulation, resulting in a long half-life within nerve tissue and rapid clearance from plasma and other tissues, which may contribute to its favorable safety profile. It has received Fast Track, Orphan Drug, and Rare Pediatric Disease designations from the US FDA for NF1. Clinical development is ongoing with Phase II trials underway[5][6][7][8].
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