Applied Therapeutics to Present Data Highlighting AT-001 for the Treatment of Diabetic Cardiomyopathy at the Heart Failure Society of America (HFSA) 23rd Annual Scientific Meeting
“We are pleased to share additional data from our Phase 1/2 study of AT-001 in diabetic patients with the heart failure community,” said
Preclinical and Clinical Proof of Concept for Metabolic Intervention in Diabetic Cardiomyopathy
- AT-001 significantly reduced cardiac damage in a relevant mouse model of DbCM
- AT-001 potently inhibited AR within a favorable safe dosing range
- In humans, single and multiple ascending doses were well-tolerated with no safety concerns identified, and proof of biological activity was obtained in all patients dosed
About Diabetic Cardiomyopathy
Diabetic Cardiomyopathy (DbCM) is a rapidly progressing degenerative disorder of the heart in patients with both type 1 and type 2 diabetes. There are no approved therapies for this fatal condition, which affects 17 – 24 percent of people with diabetes, or approximately 77 million patients worldwide. Hyperglycemia, a symptom that characterizes diabetes, triggers the enzyme Aldose Reductase to convert excess glucose into sorbitol and fructose, both of which can lead to cell death in the heart muscle. When this happens, the heart fibroses, or “hardens,” such that the organ is unable to circulate blood through the body effectively. Approximately 25 percent of patients with DbCM progress to overt heart failure or death within 1.5 years of diagnosis.
AT-001 is an investigational oral, novel, potent Aldose Reductase inhibitor in Phase 3 clinical development for the treatment of Diabetic Cardiomyopathy. AT-001 has been studied in a Phase 1/2 study in approximately 120 patients with type 2 diabetes, a subset of which had DbCM.
Applied Therapeutics is a clinical-stage biopharmaceutical company developing a pipeline of novel drug candidates against validated molecular targets in indications of high unmet medical need. The company’s lead drug candidate, AT-001, is a novel aldose reductase inhibitor (ARI) that is being developed for the treatment of Diabetic Cardiomyopathy, or DbCM, a fatal fibrosis of the heart. The company initiated a Phase 3 registrational study in DbCM in September 2019. Applied Therapeutics is also developing AT-007, a central nervous system penetrant ARI, for the treatment of Galactosemia, a rare pediatric metabolic disease, and initiated a Phase 1/2 clinical trial in June 2019. The preclinical pipeline also includes AT-003, an ARI designed to cross through the back of the eye when dosed orally, for the treatment of diabetic retinopathy, expected to advance into a Phase 1 study in 2020.
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Source: Applied Therapeutics