Mitochondrial Diseases
We are focused on developing novel medicines for diseases with significant unmet needs, beginning with MELAS and other genetic mitochondrial diseases.
Mitochondrial Disease Overview
Known as “the powerhouse of the cell,” mitochondria generate about 90% of the energy our bodies need for healthy living. They support cell and organ function and are essential for a wide array of biological processes.
Primary mitochondrial disease is a family of hundreds of genetic diseases that negatively impact mitochondrial function. Given the critical role of mitochondria across many different bodily systems and organs – particularly those with high energy demands such as skeletal and cardiac muscles and the central nervous system – mitochondrial diseases have broad and debilitating consequences.
MELAS
Our clinical development program is focused on MELAS (Mitochondrial Encephalomyopathy with Lactic Acidosis and Stroke-like episodes), a rare mitochondrial disease that affects multiple organ systems and is characterized by both neurological and systemic manifestations. MELAS frequently causes severe and burdensome symptoms including chronic fatigue, cognitive dysfunction, muscle weakness, seizures, and stroke-like episodes. The significant burden of disease often leads to social isolation and reduced quality of life. MELAS is also associated with shortened life expectancy.
There are no approved treatments for MELAS, and current disease management options are primarily limited to symptomatic treatments and lifestyle changes—all of which are inadequate to manage the disease. At Tisento, we are working on what could be the first treatment for this devastating disease.
MELAS Patient Voices
We’re proud to support the United Mitochondrial Disease Foundation’s “Our MELAS Story” video series, which is designed to increase understanding of MELAS and what matters most to those living with it.
MELAS Interview Study
At Tisento, patient perspectives are at the forefront of our decision making. We conducted an interview study with adults living with MELAS to gather their experiences and shape our clinical development program accordingly. The interview study ensures our clinical studies measure the most relevant and meaningful aspects of MELAS, as reported directly by people affected by the disease.
The results of the adult interview study informed the endpoints for the zagociguat Phase 2b PRIZM clinical study. In the interview study, the heterogeneity of MELAS presentation was apparent, with fatigue- and cognitive-related symptoms reported most frequently.
Using these data, we developed a comprehensive group of endpoints that includes both subjective and objective measures of fatigue, cognition, exercise intolerance, and muscle weakness.
Adolescent MELAS Interview Study
We want to hear from teens (ages 12-17 years) with MELAS to understand their experiences of living with MELAS.
Additional Resources
If you or a loved one have been diagnosed with a mitochondrial disease, we recommend connecting with one or more of the patient advocacy groups below for additional resources and support.
