You may remember learning that mitochondria are the powerhouse of the cells, but do you know the dangers that result if these cell superheroes get sick?
In addition to powering healthy cells, mitochondria are also important players in certain diseases. This is especially true in kidney disease. While kidney cells make up only 1% of our body weight, they consume over 10% of our daily energy supply. When either acute or chronic disease strikes these vital organs, the health of the mitochondria can literally be the difference between life and death. Specialized pores inside the mitochondria called mitochondrial permeability transition (MPT) pores regulate programmed cell death—or apoptosis. Under disease conditions, MPT pores are stuck open allowing fluid to rush in causing swelling which leads to the death of the cell.
What if you could close these pores? Would that restore the normal function of damaged mitochondria and possibly be a cure for disease? That’s just what a California-based biotechnology company specializing in diseases of the kidney is hoping to do.
Unicycive Therapeutics Inc. UNCY recently announced findings from a preclinical trial for their drug called UNI-494 that reduced a known biomarker of ischemic injury that is present in acute kidney injury (AKI), a result described as “very encouraging” by a leading expert in the field of AKI research. UNI-494 is a potent activator of the mitochondrial adenosine triphosphate-sensitive (ATP) potassium channel (mitoKATP). That’s important because when these channels are activated, they close the MPT pores and prevent the damage that leads to cell death.
Mitochondrial dysfunction is closely associated with kidney issues, including (AKI). Affecting over 13 million people worldwide each year, AKI increases the risk of mortality by 23.9% in adults and 13.8% in children. Companies like DaVita Inc. DVA and Akebia Therapeutics Inc. AKBA are among the companies seeking solutions to a variety of renal diseases. Unbelievably, however, there are still no Food and Drug Administration (FDA)-approved drugs for AKI.
Sadly, damage to mitochondria is seen in other diseases as well, including cardiovascular, liver and eye degeneration. While Unicycive is initially focused on producing a treatment for AKI and chronic kidney disease (CKD), it believes that the mechanism of action of UNI-494 may also be applicable for the treatment of other diseases where the mitochondria are affected.
The company is now beginning its next milestone, filing to begin Phase 1 human trials of the drug.
“We are excited to report this key finding,” Unicycive CEO Dr. Shalabh Gupta said. “We remain on track to file a regulatory submission by the end of 2022 that will allow us to initiate our Phase 1 study with UNI-494.”
If the FDA approves the drug, it would be the first drug for AKI available on the market.
Learn more about Unicycive on its website.
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