U.S. FDA Grants Fast Track to Tisento's Zagociguat for MELAS Treatment

Tisento Therapeutics has recently announced that the U.S. Food and Drug Administration (FDA) has awarded Fast Track designation to their investigational drug, zagociguat, aimed at treating MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes). Zagociguat is an oral medication, administered once daily, that holds promise for addressing both the neurological and systemic symptoms associated with mitochondrial diseases. The FDA's Fast Track process is designed to speed up the development and review of drugs that address serious conditions and satisfy an unmet medical need, thus ensuring that important new therapies can reach patients more quickly.

MELAS is a rare mitochondrial disorder that impacts multiple organ systems, characterized by severe symptoms such as chronic fatigue, cognitive challenges, muscle weakness, seizures, and episodes resembling strokes. The disease imposes a significant burden on patients, often resulting in social isolation, diminished quality of life, and reduced life expectancy. Currently, no approved treatments exist for MELAS, and the available management strategies largely focus on symptom relief and lifestyle modifications, which fall short of effectively addressing the debilitating nature of the condition.

Peter Hecht, PhD, the CEO of Tisento, emphasized the urgent need for meaningful treatments to address the challenges faced by individuals living with MELAS and their families. He expressed the company's satisfaction with receiving the Fast Track designation, which highlights the pressing unmet needs of the MELAS community and underscores the potential of zagociguat to become an essential therapeutic option for managing both neurological and systemic manifestations of the disorder.

Tisento is currently conducting the global Phase 2b PRIZM study to assess how zagociguat affects fatigue, cognitive impairment, and other critical aspects of MELAS. The study's design and assessment strategy have been informed by interviews with individuals living with MELAS, who provided insights into the symptoms and disease impacts that matter most to them. The PRIZM trial is actively enrolling participants across North America, Europe, and Australia.

The PRIZM study is a randomized, placebo-controlled trial investigating the safety and effectiveness of zagociguat, administered at doses of 15 mg or 30 mg once daily for 12 weeks, compared to a placebo. The study employs a crossover design, with participants receiving the active drug during one 12-week period and a placebo during another, separated by a four-week washout period. Participants who complete the trial may be eligible for an open-label extension study to further explore the drug's potential benefits.

Zagociguat operates by stimulating soluble guanylate cyclase (sGC), an enzyme present in nearly every cell in the body, which plays a crucial role in regulating physiological functions such as neuronal activity and blood flow. The drug aims to rebalance dysregulated cellular pathways in MELAS, helping to restore cellular operations that support mitochondria, thereby potentially improving mitochondrial energy production and overall physiological function. Early studies have demonstrated favorable safety and improved neuronal and mitochondrial functions in patients with MELAS, prompting further investigation in the ongoing PRIZM trial.

Tisento Therapeutics is a biotech company dedicated to developing novel treatments for diseases with significant unmet needs, focusing initially on MELAS and other genetic mitochondrial disorders. The company's innovative approach is driven by insights gathered from patients, and it is supported by an experienced team of biopharma experts and collaborations with leading physicians, researchers, and patient advocacy groups.