Primary mitochondrial disease affects the ability of cells to convert energy. It can manifest itself very differently depending on the organs impacted and the number of dysfunctional mitochondria in that organ. Historically viewed as clinical syndromes, our knowledge about the various mutations underlying mitochondrial disease has increased, improving our ability to identify and treat these patients. It is estimated that 125 people per million have primary mitochondrial disease.

Primary mitochondrial disease often presents in early childhood and can lead to severe symptoms, such as stunted growth, muscle weakness, pronounced fatigue, heart failure and rhythm disturbances, diabetes, movement disorders, stroke-like episodes, deafness, blindness, limited mobility of the eyes and epileptic seizures.

Abliva's lead candidate KL1333 restores the levels of the coenzymes NAD+ and NADH, creating new mitochondria and improved energy levels. KL1333 has completed a number of key Phase 1 studies that enabled the start of a potentially registrational Phase 2 study in 2022 - the FALCON study.

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