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

Novel lead small molecules demonstrate substantial reduction of cholesterol accumulation in NPC patient-derived fibroblasts, with additional data supporting lysosomal health and cellular signaling

NPC represents a significant new pipeline opportunity for Sharp, expanding the application of its Disco™ small-molecule discovery platform

PITTSBURGH and TORONTO, May 28, 2026 (GLOBE NEWSWIRE) -- Sharp Therapeutics Corp. (“Sharp” or the “Company”) (TSX-V: SHRX) (OTCQB: SHRXF), a biotechnology company developing small-molecule therapeutics for genetic diseases, today announced new preclinical data supporting the Company’s novel therapeutic approach for Niemann-Pick disease type C (“NPC”). The data are being presented in a poster at the 2026 Michael, Marcia, and Christa Parseghian Scientific Conference for Niemann-Pick Type C Research NNPDF Family Support & Medical Conference, being held May 30-June 2, 2026, in Tucson, Arizona.

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.

“NPC represents a significant new opportunity for Sharp and expands the reach of our platform into a devastating genetic disease with serious neurological and visceral manifestations,” said Scott Sneddon, Ph.D., J.D., Chief Executive Officer of Sharp Therapeutics. “Our lead chemical series significantly reduced the cholesterol accumulation central to NPC pathology, while additional lysosomal exocytosis and mTOR signaling data support a potentially differentiated approach targeting key aspects of the underlying disease biology. We are continuing preclinical development and IND-enabling activities to optimize this series and advance a candidate toward clinical development.”

Preclinical Data and Next Steps

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.

About Niemann-Pick Disease Type C

Niemann-Pick disease type C is a rare, progressive autosomal recessive lysosomal storage disorder primarily caused by mutations in the NPC1 gene. The NPC1 protein plays a critical role in transporting cholesterol out of late endosomes and lysosomes. Mutations in NPC1 result in intracellular accumulation of cholesterol and sphingolipids, leading to lysosomal dysfunction, impaired cellular homeostasis, and progressive central nervous system and visceral organ manifestations. While recently approved therapies provide important new treatment options for the neurological manifestations of NPC, there remains a need for therapeutic approaches designed to address the underlying disease pathology across both neurological and visceral manifestations. NPC is estimated to occur in approximately 1 in 100,000 to 120,000 live births globally.

About Sharp Therapeutics Corp.

First-Choice Therapies for Genetic Diseases

Sharp Therapeutics is a preclinical-stage company developing first-choice small-molecule therapeutics for genetic diseases. The Company’s small molecule discovery platform combines novel high-throughput screening technologies with compound libraries computationally optimized based on the physics and biology of cellular trafficking defects and allosteric activation of proteins. The platform produces small-molecule compounds that restore activity in mutated proteins, providing the potential to treat genetic disorders with conventional pill-based medicines.

Caution Regarding Forward-Looking Information

Certain statements contained in this press release constitute "forward-looking information" as such term is defined in applicable Canadian securities legislation. The words "may", "would", "could", "should", "potential", "will", "seek", "intend", "plan", "anticipate", "believe", "estimate", "expect" and similar expressions are intended to identify forward-looking information. All statements other than statements of historical fact may be forward-looking information. Such statements reflect Sharp's current views and intentions with respect to future events, and current information available to Sharp, and are subject to certain risks, uncertainties and assumptions. Many factors could cause the actual results, performance or achievements that may be expressed or implied by such forward-looking information to vary from those described herein should one or more of these risks or uncertainties materialize. Should any factor affect Sharp in an unexpected manner, or should assumptions underlying the forward-looking information prove incorrect, the actual results or events may differ materially from the results or events predicted. Any such forward-looking information is expressly qualified in its entirety by this cautionary statement. Moreover, Sharp does not assume responsibility for the accuracy or completeness of such forward-looking information. The forward-looking information included in this press release is made as of the date of this press release and Sharp undertakes no obligation to publicly update or revise any forward-looking information, other than as required by applicable law.

Neither TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

For additional information on Sharp, please visit: www.sharptx.com.

Sharp Therapeutics Corp.
Scott Sneddon, PhD, JD
CEO/CSO
Email: scott@sharptx.com

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