New laboratory data shows that Stealth Biotherapeutics’ investigational drug SBT-589 has been found to be effective in reducing cellular energy deficiency in several preclinical models of Friedreich’s ataxia (FA), including a mouse model of significant heart disease. It showed protective effects against myocardial diseases.

SBT-589 is designed to increase the activity of mitochondria, known as the powerhouses of cells, whose function is impaired in FAs, particularly in cardiomyocytes. As a result, FA patients develop cardiomyopathy, or heart muscle disease, for which there is no approved treatment.

The data was presented at the Wellcome Trust Conference on Mitochondrial Medicine – Therapeutic Development, held March 18-20 in the UK, by Laura E. Cropp, Ph.D., Stealth’s senior manager of discovery biology.

“The new findings…support the continued development of SBT-589 as a potential disease-modifying therapy to address the unmet needs of Friedreich’s ataxia-associated cardiomyopathy,” said Stealth’s senior vice president of discovery. President David A. Brown said in an internal press release. release. This release calls SBT-589 a “promising new molecule.”

Myocardial disease in FA ranks as one of the main causes of death

FA is caused by mutations such as: FXN This gene causes a defective production of frataxin, a protein essential for normal mitochondrial function. When mitochondria are malfunctioning, they are unable to provide the energy needed by high-demand cells, such as cells in the nervous system and heart muscle.

“Mitochondrial dysfunction is a central cause of Friedreich’s ataxia, a disease in which heart failure and sudden cardiac events are among the main causes of death,” Dr. Brown said. Stated.

“Reducing heart disease in FA patients is central to Stealth’s continued efforts to improve the lives of FA patients,” added Brown.

A team of scientists evaluated the protective potential of SBT-589 in three different preclinical models. One was a model of his FA patient-derived cells grown in the lab, and the other was a model of isolated cardiac mitochondria. The third was a mouse model of FA-related heart disease. This mouse model exhibits symptoms of marked cardiac hypertrophy or cardiomegaly and dies early.

Reducing heart disease in FA is central to Stealth’s ongoing efforts to improve the lives of FA patients.

Results showed that SBT-589 prevented mitochondrial damage and promoted energy production in all models. Data showed that cardiac hypertrophy was significantly reduced and death was delayed in animals given SBT-589 once daily compared to mice given a sham solution.

According to Steals, further preclinical studies are underway to continue to evaluate the therapeutic potential of the therapeutic candidate, and “we will continue to build on these insights as SBT-589 progresses through the developmental gates.” It is hoped that it will be built upon.”

The results will be used to support the potential application of testing SBT-589 in human clinical trials, the company said.