Mr. Scott Sneddon reports
SHARP THERAPEUTICS UPDATES ON '901 FOR GAUCHER AND GBA1 PARKINSON'S AND PLATFORM PROGRAMS
Following longer-term non-GLP testing, Sharp Therapeutics Corp. has decided to advance an alternative compound for its Gaucher and GBA1 Parkinson's programs. The company continues to advance all platform programs including programs for genetic and sporadic forms of neurodegeneration (Progranulin modulators) and a rare neurodegenerative lysosomal disease (Niemann Pick Type C).
"Sharp has a high standard for advancing any therapeutic candidate into clinical development, and, while the '901-series compounds continue to perform quite well, based on the suboptimal dose proportionality of '901 in longer-term non-GLP studies, we have made the decision to evaluate replacement candidates. Since nominating '901, our team has maintained an active backup program, which has produced multiple compounds with superior physical properties and metabolic behaviour (key drivers of dose proportionality). This provides an opportunity to quickly elevate one of these compounds to candidate status for the Gaucher and GBA1 Parkinson's programs," said Scott Sneddon, PhD, JD, Sharp's chief executive officer.
Dr. Sneddon continued: "The Gaucher/PD program has yielded compounds that restore GBA1 function through a novel mechanism, with potent activity in patient cells and in vivo, and good oral availability and CNS penetrance. Advancing a compound with superior pharmaceutical properties provides the opportunity to bring the highest-quality compound into phase 1 development and eventually to patients. We continue to believe that our unique approach to addressing serious disease caused by GBA1 deficiency (Gaucher and GBA1-Parkinson's) could result in significant therapeutic and commercial opportunities, and we continue to emphasize moving this program forward."
In the company's view, the greatest value in Sharp is the platform that produces the assets it takes forward to development. Therefore, Sharp continues to advance small-molecule therapies for Nieman Pick Type C as well, having discovered small-molecule lead compounds that directly lower accumulated cholesterol in cells from patients, which would provide a unique therapeutic opportunity in that growing market.
Sharp is also advancing it other platform project: its small-molecule Progranulin modulation program for broad potential application to neurodegenerative disease, including genetic and sporadic forms of frontotemporal dementia, Alzheimer's and Parkinson's.
About Sharp Therapeutics Corp.
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 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.
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