Pharmaceutical Technology

Tisento’s MELAS treatment receives fast track status from FDA

MELAS patients often endure severe symptoms such as chronic fatigue, muscle weakness and cognitive issues.

Tisento Therapeutics has received fast track designation from the US Food and Drug Administration (FDA) for its investigational oral medicine, zagociguat, for mitochondrial encephalomyopathy, lactic acidosis and stroke-like episodes (MELAS), a rare mitochondrial disease.

Zagociguat acts by stimulating soluble guanylate cyclase (sGC), an enzyme found across every cell in all tissues of the body.

Tisento Therapeutics CEO Peter Hecht stated: “People living with MELAS and their families face tremendous challenges and deserve to have treatments that address the aspects of the disease that matter most to them.

“We are pleased to receive fast track designation from the FDA in recognition of the MELAS community’s unmet medical needs and the potential of zagociguat to be a meaningful therapeutic option to address both neurological and systemic manifestations of the disease.”

Tisento is studying the influence of this brain-penetrant sGC stimulator on cognitive impairment, fatigue and other primary aspects of the disease in the global Phase IIb PRIZM trial, which is currently enrolling in Europe, Australia and North America.

The study's clinical outcome evaluations and endpoint strategy were shaped by insights from people with MELAS, who provided input on the disease's symptoms and impacts that matter most to them.

The study is assessing the safety and efficacy of 15 mg or 30 mg of the therapy against a placebo, administered once daily for 12 weeks to those with genetically and phenotypically defined MELAS.

All subjects will be given zagociguat in one of the 12-week durations and a placebo during the other.

Subjects who conclude the trial will be qualified for an open-label extension trial.

MELAS patients often display severe symptoms such as chronic fatigue, muscle weakness, cognitive issues, stroke-like episodes and seizures.

With no approved treatments available, present management strategies focus on symptomatic treatments and lifestyle modifications, which fall short of addressing the disease's profound and persistent effects.