DelveInsight’s, “MELAS Syndrome Pipeline Insight, 2025” report provides comprehensive insights about 4+ companies and 4+ pipeline drugs in MELAS Syndrome pipeline landscape. It covers the MELAS Syndrome pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

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Key Takeaways from the MELAS Syndrome Pipeline Report

• In June 2025, Tisento Therapeutics announced a PRIZM study is evaluating 2 dose levels of zagociguat in a crossover design consisting of two 12-week treatment periods separated by a 4-week washout. Patients will be screened and if eligible, randomly assigned either to receive placebo in period 1 followed by active drug in period 2 OR to receive active drug in period 1 followed by placebo in period 2. Study medication is a once daily oral tablet and will be provided at the clinic and/or shipped to the participant’s home. Clinic visits will occur at screening and Week 1 and Week 12 of each treatment period.
• In May 2025, Thiogenesis Therapeutics Inc. announced a Study Exit visit will occur at Week 24, and subjects will be offered to continue on an open-label extension study of TTI-0102. If a subject does not complete the study, they will be asked to return for a Study Exit visit 4 weeks after last study drug dose.
• DelveInsight’s MELAS Syndrome pipeline report depicts a robust space with 4+ active players working to develop 4+ pipeline therapies for MELAS Syndrome treatment.
• The leading MELAS Syndrome Companies such as Cyclerion Therapeutics Inc., Khondrion BV, Abliva AB and others.
• Promising MELAS Syndrome Therapies such as TTI-0102, Idebenone, zagociguat 15mg, KH176, Vatiquinone, OMT-28 and others.

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MELAS Syndrome Emerging Drugs Profile

• Sonlicromanol: Khondrion

Sonlicromanol is Khondrion’s lead investigational drug, developed as a first-in-class, brain-penetrant redox modulator with anti-inflammatory properties. It is designed to treat primary mitochondrial diseases (PMD) by targeting disrupted cellular redox balance and mitochondrial dysfunction. The drug modulates reactive oxygen species (ROS) levels and supports mitochondrial function by influencing key metabolic and inflammatory signaling pathways, potentially modifying disease progression rather than just alleviating symptoms. Currently, the drug is in Phase III stage of its clinical trial for the treatment of MELAS Syndrome.

• CY-6463: Cyclerion Therapeutics

CY6463 is the first CNS-penetrant sGC stimulator to be developed as a symptomatic and potentially disease-modifying therapy for serious CNS diseases. The nitric oxide (NO)-soluble guanylate cyclase (sGC)-cyclic guanosine monophosphate (cGMP) signaling pathway is a fundamental mechanism that precisely controls key aspects of physiology throughout the body. In the CNS, the NO-sGC-cGMP pathway regulates diverse and critical biological functions including neuronal function, neuroinflammation, cellular bioenergetics, and vascular dynamics. Although it has been successfully targeted with several drugs in the periphery, this mechanism has yet to be fully leveraged therapeutically in the CNS, where impaired NO-sGC-cGMP signaling is believed to play an important role in the pathogenesis of many neurodegenerative and neuropsychiatric diseases and other disorders associated with cognitive impairment. As an sGC stimulator, CY6463 acts as a positive allosteric modulator to sensitize the sGC enzyme to NO, increase the production of cGMP, and thereby amplify endogenous NO signaling. Currently, the drug is in Phase II stage of its clinical trial for the treatment of MELAS Syndrome.

The MELAS Syndrome Pipeline Report Provides Insights into

• The report provides detailed insights about companies that are developing therapies for the treatment of MELAS Syndrome with aggregate therapies developed by each company for the same.
• It accesses the Different therapeutic candidates segmented into early-stage, mid-stage, and late-stage of development for MELAS Syndrome Treatment.
• MELAS Syndrome Companies are involved in targeted therapeutics development with respective active and inactive (dormant or discontinued) projects.
• MELAS Syndrome Drugs under development based on the stage of development, route of administration, target receptor, monotherapy or combination therapy, a different mechanism of action, and molecular type.
• Detailed analysis of collaborations (company-company collaborations and company-academia collaborations), licensing agreement and financing details for future advancement of the MELAS Syndrome market

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MELAS Syndrome Companies

Cyclerion Therapeutics Inc., Khondrion BV, Abliva AB and others.

MELAS Syndrome pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Intra-articular
• Intraocular
• Intrathecal
• Intravenous
• Oral
• Parenteral
• Subcutaneous
• Topical
• Transdermal

MELAS Syndrome Products have been categorized under various Molecule types such as

• Oligonucleotide
• Peptide
• Small molecule

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Scope of the MELAS Syndrome Pipeline Report

• Coverage- Global
• MELAS Syndrome Companies- Cyclerion Therapeutics Inc., Khondrion BV, Abliva AB and others.
• MELAS Syndrome Therapies- TTI-0102, Idebenone, zagociguat 15mg, KH176, Vatiquinone, OMT-28 and others.
• MELAS Syndrome Therapeutic Assessment by Product Type: Mono, Combination, Mono/Combination
• MELAS Syndrome Therapeutic Assessment by Clinical Stages: Discovery, Pre-clinical, Phase I, Phase II, Phase III

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Table of Contents

1. Introduction
2. Executive Summary
3. MELAS Syndrome : Overview
4. Pipeline Therapeutics
5. Therapeutic Assessment
6. MELAS Syndrome – DelveInsight’s Analytical Perspective
7. Late Stage Products (Phase III)
8. Sonlicromanol: Khondrion
9. Mid Stage Products (Phase II)
10. CY-6463: Cyclerion Therapeutics
11. Early Stage Products (Phase I)
12. Drug Name: Company Name
13. Preclinical Stage Products
14. Drug Name: Company Name
15. Inactive Products
16. MELAS Syndrome – Collaborations Assessment- Licensing / Partnering / Funding
17. MELAS Syndrome – Unmet Needs
18. MELAS Syndrome – Market Drivers and Barriers
19. Appendix

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