Zagociguat

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 P hase 2b R andomized, Placebo-Controlled Trial I nvestigating Z agociguat in M ELAS – 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 Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

CAMBRIDGE, Mass., Dec. 17, 2024 (GLOBE NEWSWIRE) -- Cyclerion Therapeutics, Inc. (Nasdaq: CYCN), today announced an update on its progress in catalyzing the Company’s next stage of growth. The Company is leveraging its legacy soluble guanylate cyclase (sGC) stimulator assets to generate near-term revenues which will be used to implement its strategic building plan without near-term dilution. “These agreements demonstrate Cyclerion’s progress in maximizing its legacy asset value while redirecting resources toward acquiring potential new assets,” said Regina Graul, Ph.D., President and Chief Executive Officer of Cyclerion. “These newly finalized agreements, combined with our significant reduction of operating expenses, enable the focused use of our capital to support our anticipated pipeline build in the central nervous system (CNS) space. Concurrently, we plan to raise capital, as needed, to fund our product plans to create value for shareholders and patients.” Graul continued, “Cyclerion’s diligence team, comprised of committed external experts in their respective fields, is currently in advanced stages of conducting promising asset evaluations, which we believe have the potential to be the new foundation for Cyclerion.” Cyclerion and Akebia have re-negotiated a mutually beneficial amendment to their exclusive license agreement for praliciguat, a systemic sGC stimulator. Under the new license amendment with Akebia, Cyclerion will receive $1.75 million in upfront and near-term payments. In addition, Akebia will assume responsibility for all intellectual property expenses associated with praliciguat after Q1 2025. In 2021, Akebia paid a $3.0 million upfront payment to the Company upon signing of the license agreement, and the Company is eligible to receive additional milestone cash payments of up to approximately $560 million in total potential future development, regulatory, and commercialization milestone payments for praliciguat. In exchange for a reduction in certain development milestone payments, Cyclerion is eligible to receive certain higher, tiered, sales-based royalties ranging from mid-single-digits to twenty percent. Cyclerion has also entered into an exclusive license option agreement for its vascular sGC stimulator, olinciguat, with a separate entity, wholly controlled by CVCO Therapeutics, Inc., a clinical stage company focused on microvascular dysfunction in cardiovascular, inflammatory and metabolic disease states. Under the terms of the agreement, the potential partner has exclusive rights to evaluate olinciguat during the option period. During the option period, the grantee has assumed responsibility for all ongoing intellectual property-related expenses associated with olinciguat. As part of its strategic initiatives, Cyclerion previously announced a definitive agreement for the sale of its CNS assets zagociguat and CY3018 to Tisento Therapeutics in May 2023 for an $8 million cash payment and a 10% equity stake in Tisento. The amended agreement for praliciguat, and if the option for olinciguat is exercised, would represent the likely final steps in the monetization of Cyclerion’s historical portfolio. Cyclerion believes it is well positioned for the next phase in its overall strategy, to bring in new CNS assets to rebuild the pipeline. Forward Looking Statement Certain matters discussed in this press release are “forward-looking statements”. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should”, “positive”, or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. These statements involve risks, uncertainties and other factors that may cause actual results, levels of activity, performance or achievements to be materially different from the information expressed or implied by these forward-looking statements. Although we believe that we have a reasonable basis for each forward-looking statement contained in this press release, we caution you that these statements are based on a combination of facts and factors currently known by us and our projections of the future, about which we cannot be certain. Forward-looking statements in this press release include, but are not limited to, statements about pursuing collaborations, licenses, mergers, acquisitions and/or other targeted investments aimed at enhancing shareholder value. We cannot assure you that the forward-looking statements in this press release will prove to be accurate. Furthermore, if the forward-looking statements prove to be inaccurate, the inaccuracy may be material. Actual performance and results may differ materially from those projected or suggested in the forward-looking statements due to various risks and uncertainties, including, those under the heading “Risk Factors” in our Annual Report on Form 10-K filed with the SEC on March 5, 2024 as well as other risks and uncertainties which may be described in any subsequent quarterly report on Form 10-Q filed by the Company and the other reports the Company files with the SEC. In light of the significant uncertainties in these forward-looking statements, you should not regard these statements as a representation or warranty by us or any other person that we will achieve our objectives and plans in any specified time frame, or at all. The forward-looking statements are made only as of the date of this press release and the Company undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances. Source: Cyclerion Therapeutics, Inc.