Tisento to Present at the “Primary Mitochondrial Diseases” Therapeutic Development Workshop Hosted by the Reagan-Udall Foundation for the FDA on May 22, 2025

Tisento to Sponsor the First Externally Led Patient-Focused Drug Development Meeting Dedicated to MELAS on February 10, 2026; MitoAction to Lead

CAMBRIDGE, Mass., May 14, 2025 (GLOBE NEWSWIRE) -- Tisento Therapeutics today announced upcoming opportunities to collaborate with patient advocates, leading clinicians, and regulators to optimize therapeutic development for mitochondrial diseases, including the rare mitochondrial disease MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like Episodes).

“Incorporating the insights gleaned from listening to patients and integrating the perspectives of all critical stakeholders are foundational to our drug development approach,” said Peter Hecht, Ph.D., chief executive officer of Tisento. “We look forward to advancing patient-focused therapeutic development for mitochondrial diseases at two important events. We will share key learnings related to the design of our Phase 2b PRIZM study in MELAS at the primary mitochondrial diseases public workshop, hosted by the Reagan-Udall Foundation for the FDA. We also appreciate the opportunity to hear more from patients and advocates at the first externally led patient-focused drug development meeting dedicated to MELAS and are proud to support MitoAction’s efforts to shed light on the unmet needs in this rare condition.”

Primary Mitochondrial Diseases Public Workshop

Chad Glasser, Pharm.D., senior director of clinical research at Tisento, is presenting Tisento’s approach to developing zagociguat for the treatment of MELAS, a primary mitochondrial disease, at a public workshop. Dr. Glasser will share key insights on overcoming challenges associated with developing medicines for rare, heterogeneous diseases, including how learnings from Tisento’s MELAS patient interview study informed selection of assessments and endpoints in the company’s ongoing Phase 2b PRIZM study in adults with MELAS.

Hosted by the Reagan-Udall Foundation for the FDA in collaboration with the U.S. Food and Drug Administration, the workshop brings together patient advocates, academics, healthcare practitioners, and drug developers to explore opportunities to optimize the development of therapies to treat primary mitochondrial diseases and best practices in designing and interpreting clinical studies to accelerate drug development. The Reagan-Udall Foundation for the FDA is an independent 501(c)(3) organization created by Congress to modernize product development, accelerate innovation, and enhance product safety. The Foundation serves as a crucial conduit between the FDA and the public, providing a means for the FDA to interact directly with stakeholders, including industry and consumers.

Virtual Event Details
Date: Thursday, May 22, 2025
Time: 10am-4pm ET
Registration: Free; register here

MitoAction-Led Patient-Focused Drug Development Meeting

Tisento is sponsoring the first externally led patient-focused drug development (EL-PFDD) meeting dedicated to systematically obtaining patient perspectives on living with MELAS and on the most important symptoms to address when developing drug candidates. Led by MitoAction, the EL-PFDD is intended to provide an opportunity for the FDA, pharmaceutical industry, and other key stakeholders to hear directly from patients, their families, caregivers, and patient advocates about the symptoms that matter most to them, the impact MELAS has on patients’ daily lives, and patients’ experiences with currently available disease management approaches. There are no FDA-approved treatments specifically for MELAS. MitoAction is planning and hosting the meeting; Tisento is providing financial support and is not involved in planning or content development.

Virtual Event Details
Date: Tuesday, February 10, 2026 
Registration: Free (registration details to be announced); reach out to MitoAction at info@mitoaction.org for updates

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