PRDセラピューティクス社がPRD001の最初のヒト臨床試験を開始

 PRD Therapeutics Inc. has announced a first-in-human clinical trial for PRD001. This is a new selective inhibitor of SOAT2. It targets homozygous familial hypercholesterolemia (HoFH) and metabolic steatosis-associated liver disease (MASH/MASLD). This is the first clinical evaluation of a drug that targets SOAT2. It stands out from earlier work that focused on dual SOAT1/2 or SOAT1 inhibitors.

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CEO and co-founder Dr. Kanji Hosoda highlighted this important milestone. He said that preclinical studies show targeting SOAT2 alone can ensure safety and effectiveness. In animal models of HoFH and diet-induced MASH, PRD001 reduced fat buildup in the blood and liver. It also slowed down fatty liver disease and atherosclerosis. No adverse effects were noted.
If PRD001 succeeds in human trials, it could be the first oral therapy for HoFH patients. This drug targets those with little or no LDL receptor activity. It aims to meet a significant need in treating cardiovascular and metabolic diseases.