Tisento Therapeutics Initiates Interview Study with Individuals with MELAS to Elucidate the Symptoms and Impact of Disease and Inform the Zagociguat Clinical Development Program

2023-10-25

临床2期临床结果

– Interviews Intended to Illuminate Experience of Living with MELAS, a Rare Mitochondrial Disease –

– Findings to Support Data-driven Endpoint Selection for Phase 2b Study of Zagociguat in MELAS –

CAMBRIDGE, Mass., Oct. 25, 2023 (GLOBE NEWSWIRE) -- Tisento Therapeutics, a privately held, clinical-stage biotechnology company developing novel medicines for diseases with significant unmet medical needs, today announced the initiation of a patient interview study to identify and describe the signs, symptoms, and health-related quality-of-life impacts of MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis and Stroke-like episodes) that are most burdensome to adults and adolescents living with this rare mitochondrial disease. The interviews will inform the selection of clinical outcomes assessments and endpoint strategy for the company’s planned Phase 2b study of its investigational medicine, zagociguat, in MELAS.

“At Tisento, patient perspectives are at the forefront of the decisions we make. This interview study is one way we will thoughtfully and intentionally listen to individuals with MELAS and take action in response,” said Peter Hecht, Ph.D., chief executive officer of Tisento. “The goal of the interview study is to ensure our Phase 2b study and all future studies are measuring the impact of zagociguat on the most meaningful aspects of the disease, as reported directly by people living with MELAS, and that our assessment tools are both comprehensive and relevant. We look forward to partnering with the patient community to finalize the endpoints for this clinical study of what could be the first treatment for this devastating disease.”

“MELAS is a complex and little understood disease characterized by both neurological and systemic symptoms that impact patients’ health and lives in a myriad of ways,” said Philip Yeske, science and alliance officer at United Mitochondrial Disease Foundation. “The first step in improving quality of life for this community is to better understand what individuals with MELAS face on a daily basis. Tisento’s first-of-its-kind MELAS interview study will add to the scientific and clinical body of knowledge about this disease and ensure that their clinical research has been informed by the voices of those who are living with MELAS.”

The interview study is expected to enroll approximately 20 adults and adolescents with MELAS and is designed to document the severity and frequency of MELAS symptoms, as well as gather descriptive information about the most salient aspects of these symptoms. Tisento plans to use the findings from the interviews to identify clinical outcomes assessments that may be used as clinical study endpoints to evaluate the efficacy of zagociguat for the treatment of MELAS.

About MELAS

MELAS (Mitochondrial Encephalomyopathy with Lactic Acidosis and Stroke-like episodes) is a rare mitochondrial disease that affects multiple organ systems, especially organs that require significant energy like the brain and muscles. MELAS frequently causes severe and burdensome symptoms including chronic fatigue, cognitive dysfunction, muscle weakness, seizures, and stroke-like episodes. MELAS is also associated with shortened life expectancy.

About Zagociguat

Zagociguat is a once-daily, oral, clinical-stage investigational medicine with the potential to positively impact both peripheral and central nervous system components of mitochondrial diseases. Zagociguat acts upon the central node of the nitric oxide (NO) – soluble guanylate cyclase (sGC) – cyclic GMP (cGMP) pathway, which plays an essential role in fundamental physiological processes, such as mitochondrial function, inflammatory processes, cerebrovascular hemodynamics, and neuronal function. As a first-in-class, brain-penetrant sGC stimulator, zagociguat is intended to restore deficient NO-sGC-cGMP signaling in diseases where it is dysregulated, such as MELAS.

In a Phase 2a study in patients with MELAS, zagociguat exhibited an excellent safety profile, exposure throughout the body including in the central nervous system, and improvements across key domains of MELAS disease pathophysiology including neuronal function, mitochondrial function, cerebrovascular hemodynamics, and inflammatory processes.

About Tisento Therapeutics

Tisento Therapeutics is developing novel medicines to treat diseases with significant unmet medical needs, beginning with MELAS and other genetic mitochondrial diseases. Ti sento means “I hear you” in Italian; our approach to innovation begins with listening first 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 we consider 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 (Tisento Therapeutics) or X (@tisentotx).

Media Contact:

Jessi Rennekamp, Astrior Communications

Email: jrennekamp@tisentotx.com