Tisento Therapeutics Receives U.S. FDA Fast Track Designation for Zagociguat for the Treatment of MELAS
Global Phase 2b PRIZM Study of Zagociguat is Currently Enrolling Participants with MELAS
CAMBRIDGE, Mass., June 17, 2025 (GLOBE NEWSWIRE) -- Tisento Therapeutics today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to zagociguat for the treatment of MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes). Zagociguat is a once-daily, oral, clinical-stage, investigational medicine with potential to positively impact both peripheral and central nervous system manifestations of mitochondrial diseases. Fast Track is a process designed to facilitate the development and potentially expedite the review of medicines to treat serious conditions and fill an unmet medical need, with the goal of getting important new drugs to patients earlier.
MELAS is 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, reduced quality of life, and 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 debilitating and lasting consequences of the disease.
“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,” said Peter Hecht, PhD, chief executive officer of Tisento. “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.”
In the global Phase 2b PRIZM study, Tisento is evaluating the impact of zagociguat treatment on fatigue, cognitive impairment, and other key aspects of MELAS. The clinical outcome assessments and endpoint strategy for the PRIZM study were informed by Tisento’s interview study in which individuals living with MELAS described the symptoms and impacts of the disease that are most important to them. PRIZM is now enrolling in North America, Europe, and Australia. Patients and families interested in learning more can visit Tisento’s PRIZM page, ClinicalTrials.gov (NCT06402123), or discuss the PRIZM trial with their physicians.
About the PRIZM Study
PRIZM – a Phase 2b Randomized, Placebo-Controlled Trial Investigating Zagociguat in MELAS – is evaluating the efficacy and safety of oral zagociguat 15 mg or 30 mg compared to placebo when administered once-daily for 12 weeks in participants with genetically and phenotypically defined MELAS. The PRIZM study has a crossover design, with two 12-week treatment periods separated by a 4-week washout period. All participants will receive zagociguat during one of the 12-week periods and placebo during the other. Participants who complete the study may be eligible for an open-label extension study.
PRIZM is a global study that will enroll approximately 44 participants at mitochondrial disease centers of excellence in the U.S., Italy, Germany, United Kingdom, Australia, and Canada. For more information, please visit www.tisentotx.com/prizm or ClinicalTrials.gov (NCT06402123). Interested individuals can also reach out to their physicians for participation details.
About Zagociguat
Zagociguat is a once-daily, oral, clinical-stage investigational medicine with potential to positively impact both peripheral and central nervous system manifestations of mitochondrial diseases. Zagociguat stimulates soluble guanylate cyclase (sGC), an enzyme that is found in virtually every cell in every tissue of the body and is part of a system of cellular mechanisms that control critical physiological functions including neuronal function and blood flow.
A first-in-class, brain-penetrant sGC stimulator, zagociguat is hypothesized to rebalance dysregulated cellular pathways in MELAS. By restoring cellular functions that support mitochondria, zagociguat may help restore mitochondrial energy production and physiological function.
In a Phase 2a study in patients with MELAS, zagociguat exhibited a favorable safety profile, exposure throughout the body including in the central nervous system, and improvements in neuronal function, mitochondrial function, and blood flow in the brain. Zagociguat is currently being evaluated as a treatment for MELAS in the Phase 2b PRIZM study.
For more information, visit www.tisentotx.com/our-science.
About Tisento Therapeutics
Tisento Therapeutics, a privately held biotech company, is developing novel medicines to treat diseases with significant unmet need, beginning with MELAS and other genetic mitochondrial diseases. Ti sento means “I hear you” in Italian; our approach to innovation begins with listening to patients and then channeling what we learn into decisive actions that shape our research and clinical programs.
Tisento is guided by a high-caliber internal team of biopharma veterans and an extensive external network of expert physicians, patient advocacy groups, researchers, industry-leading vendors, and other close collaborators who are partners in our mission to develop meaningful treatments for mitochondrial diseases.
Learn more at our website, www.tisentotx.com, or connect with us on LinkedIn, Facebook, X (@tisentotx), or Bluesky.
Contact
Tisento Media Relations
Jessi Rennekamp, Astrior Communications
Email: jessi@astriorcomms.com
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