Pittsburgh, Pennsylvania and Toronto, Ontario--(Newsfile Corp. - May 13, 2025) - Sharp Therapeutics Corp. (TSXV: SHRX) ("Sharp" or the "Company"), a pre-clinical-stage biotechnology company developing small molecule therapies to treat genetic diseases, announces that it has nominated a small molecule compound from its GBA program for clinical development in Gaucher's disease.
Sharp has launched its clinical development program for Gaucher's disease by nominating a compound from the SEL-148,721 series of GBA1-restoring small molecules to enter IND-enabling studies. The company plans to begin compound scale-up and formal safety studies during the second half of 2025. If successful, the Company expects to file an IND application with the Food and Drug Administration and enter Phase I clinical trials in 2026.
About the SHRX Gaucher's Program.
Sharp discovered the SEL-148,172 series by applying its Disco™ discovery platform to identify compounds that enhance mutant GBA functional activity. Gaucher's disease is caused by mutation(s) in the GBA enzyme that reduce enzymatic function leading to disease. The candidate compound restores enzymatic activity, which has been shown to be an effective means of treating Gaucher's. Current Gaucher's treatments include recombinant replacement enzyme therapies, which require regular infusions with some patients developing allergic resistance to therapy. Sharp's candidate compound is an orally-available small molecule making it much more convenient for patients, and much more efficient to produce and distribute reliably.
"This is the first program from our platform to enter FDA-reportable studies and is a milestone for Sharp as it transitions to a clinical stage company," said Scott Sneddon, CEO/CSO of Sharp. "The compounds show robust activity in animal models, and more importantly, in cells taken from Gaucher's patients containing several of the most common GBA mutations," he added. "These compounds are also brain penetrant, leaving the prospect for treating CNS manifestations of Gaucher's, a market not effectively treated by existing therapies."
The scientific data supporting the compounds will be presented at the GBA1 Conference in Montreal starting June 5, 2025 of which Sharp is also a meeting sponsor.
About Sharp Therapeutics Corp.
- First-Choice Therapies for Genetic Diseases
Sharp Therapeutics is a pre-clinical stage company developing first-choice small-molecule therapeutics for hereditary disorders. The Company's discovery platform combines novel high throughput screening technologies, with compound libraries computational 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 giving the potential to treat genetic disorders with conventional pill-based medicines.
For additional information on Sharp, please visit: www.sharptx.com.
Sharp Therapeutics Corp.
Scott Sneddon, PhD, JD
CEO/CSO
Email: scott@sharptx.com
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.
To view the source version of this press release, please visit https://www.newsfilecorp.com/release/252004
Sharp has launched its clinical development program for Gaucher's disease by nominating a compound from the SEL-148,721 series of GBA1-restoring small molecules to enter IND-enabling studies. The company plans to begin compound scale-up and formal safety studies during the second half of 2025. If successful, the Company expects to file an IND application with the Food and Drug Administration and enter Phase I clinical trials in 2026.
About the SHRX Gaucher's Program.
Sharp discovered the SEL-148,172 series by applying its Disco™ discovery platform to identify compounds that enhance mutant GBA functional activity. Gaucher's disease is caused by mutation(s) in the GBA enzyme that reduce enzymatic function leading to disease. The candidate compound restores enzymatic activity, which has been shown to be an effective means of treating Gaucher's. Current Gaucher's treatments include recombinant replacement enzyme therapies, which require regular infusions with some patients developing allergic resistance to therapy. Sharp's candidate compound is an orally-available small molecule making it much more convenient for patients, and much more efficient to produce and distribute reliably.
"This is the first program from our platform to enter FDA-reportable studies and is a milestone for Sharp as it transitions to a clinical stage company," said Scott Sneddon, CEO/CSO of Sharp. "The compounds show robust activity in animal models, and more importantly, in cells taken from Gaucher's patients containing several of the most common GBA mutations," he added. "These compounds are also brain penetrant, leaving the prospect for treating CNS manifestations of Gaucher's, a market not effectively treated by existing therapies."
The scientific data supporting the compounds will be presented at the GBA1 Conference in Montreal starting June 5, 2025 of which Sharp is also a meeting sponsor.
About Sharp Therapeutics Corp.
- First-Choice Therapies for Genetic Diseases
Sharp Therapeutics is a pre-clinical stage company developing first-choice small-molecule therapeutics for hereditary disorders. The Company's discovery platform combines novel high throughput screening technologies, with compound libraries computational 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 giving the potential to treat genetic disorders with conventional pill-based medicines.
For additional information on Sharp, please visit: www.sharptx.com.
Sharp Therapeutics Corp.
Scott Sneddon, PhD, JD
CEO/CSO
Email: scott@sharptx.com
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.
To view the source version of this press release, please visit https://www.newsfilecorp.com/release/252004
