Announcement • May 29

Sharp Therapeutics Corp Presents New Preclinical Data Supporting Novel Small-Molecule Therapeutic Approach for Niemann-Pick Disease Type C

Sharp Therapeutics Corp. announced new preclinical data supporting the Company’s novel therapeutic approach for Niemann-Pick disease type C. Using Sharp’s proprietary Disco™ small-molecule discovery platform and chemical library, the Company identified a novel lead chemical series that reduced intracellular accumulation of free cholesterol in primary fibroblast cells derived from NPC patients across multiple genetic backgrounds at sub-micromolar concentrations. Additional data from the lead series demonstrated increased lysosomal exocytosis and reversal of disease-associated mTOR pathway hyperactivation in fibroblasts from NPC patients. These data support continued development of the series toward a CNS-penetrant, orally available small-molecule therapeutic candidate designed to address the underlying cellular pathology of NPC. Sharp screened its compound library for molecules capable of reducing unesterified cholesterol accumulation in NPC1-mutant cells and subsequently evaluated compounds from the lead chemical series in primary human fibroblasts derived from NPC patients. The lead series significantly reduced intracellular accumulation of free cholesterol at sub-micromolar concentrations across multiple genetic backgrounds. In additional secondary assays, compounds from the lead series reduced total unesterified cholesterol in a dose-dependent manner, increased lysosomal exocytosis, and reversed hyperactivation of the mTOR signaling pathway in NPC patient fibroblasts. These data provide further evidence that the lead series may address lysosomal dysfunction and disrupted cellular signaling associated with NPC. Sharp is continuing medicinal chemistry efforts to optimize the activity and pharmaceutical properties of the lead chemical series. Additional in vitro studies are underway to evaluate rescue of lysosomal dysfunction, further characterize the mechanism of action, and determine the direct impact of the series on the mutated NPC1 protein. The Company also plans to conduct in vivo pharmacodynamic and pharmacokinetic testing to support the selection of a CNS-penetrant, orally available small-molecule clinical candidate.

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