Berlin, Germany | July 21, 2026
OMEICOS announced that the U.S. Food and Drug Administration (FDA) has provided positive feedback during the End-of-Phase 2 (EOP2) meeting, supporting the company’s plan to advance its lead investigational therapy OMT-28 directly into a pivotal Phase 3 clinical trial for Primary Mitochondrial Diseases (PMD). The FDA’s constructive guidance enables OMEICOS to accelerate late-stage clinical development of its first-in-class oral small-molecule therapy, addressing a significant unmet medical need for patients with PMD, where the vast majority currently have no FDA-approved treatment options. The regulatory milestone was supported by encouraging findings from the completed Phase 2a PMD-OPTION study, which demonstrated improvements in patients’ physical function through restoration of impaired mitochondrial activity while maintaining an excellent safety and tolerability profile across more than 190 treated individuals. The advancement positions OMT-28 as one of the most promising late-stage therapeutic candidates in the rapidly evolving mitochondrial disease landscape.
OMT-28 Targets Core Mitochondrial Dysfunction Through a Novel Dual Mechanism
OMT-28 is designed as a once-daily oral therapy that differentiates itself from competing approaches by simultaneously targeting redox balance and mitochondrial restoration, addressing the underlying biology of mitochondrial disorders rather than a single disease pathway. The investigational therapy works by activating mitochondrial sirtuin proteins SIRT1 and SIRT3 through biased modulation of the Sphingosine-1-Phosphate Receptor 1 (S1PR1) signaling pathway, supporting improved mitochondrial function and cellular energy production. Compared with injectable therapies or twice-daily treatment regimens currently under investigation, OMT-28 offers the potential for greater patient convenience, improved treatment adherence, and broader clinical applicability. Importantly, the program also includes patients with cardiomyopathy, a subgroup frequently excluded from other clinical development programs, expanding the potential patient population and strengthening OMT-28’s profile as both a first-in-class and best-in-class therapeutic candidate.
Adaptive Phase 3 Trial Designed to Accelerate Rare Disease Development
Following FDA guidance, OMEICOS plans to initiate a pivotal adaptive Phase 3 study enrolling up to 160 adult patients across three common PMD subtypes, including MELAS, non-MELAS, and MIDD. Participants will receive OMT-28 at a 24 mg once-daily dose over 24 weeks, with an option to continue treatment for up to 104 weeks. The adaptive trial design will allow adjustments to sample size and treatment duration based on interim analyses, providing greater flexibility while maintaining scientific rigor in this rare disease setting. The primary efficacy endpoint combines improvements in the 12-Minute Walk Test (12MWT) and the 5x Sit-to-Stand Test (5xSST), while secondary endpoints will evaluate quality of life measures alongside exploratory biomarkers including NAD⁺, glutathione (GSH), and related metabolic ratios. This comprehensive clinical strategy reflects FDA recommendations and is intended to efficiently generate robust evidence supporting potential regulatory approval.
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FDA Milestone Strengthens OMEICOS’ Pipeline-in-a-Drug Strategy
According to Dr. Robert Fischer, Chief Executive Officer and Chief Scientific Officer of OMEICOS, the positive outcome of the FDA meeting exceeded expectations by allowing the company to move directly from Phase 2a into a pivotal Phase 3 study without additional intermediate clinical development. The company is now evaluating strategic partnerships, infrastructure, and operational resources needed to rapidly advance the program toward commercialization. Beyond Primary Mitochondrial Diseases, OMEICOS views OMT-28 as the foundation of a broader “pipeline-in-a-drug” strategy, with potential applications across additional mitochondrial, inflammatory, cardiovascular, and metabolic disorders. With encouraging Phase 2 clinical results, regulatory alignment from the FDA, and a differentiated mechanism of action targeting fundamental mitochondrial dysfunction, OMT-28 has the potential to become a transformational therapy for patients with rare mitochondrial diseases while significantly expanding OMEICOS’ position in the global biopharmaceutical innovation landscape.
Source: OMEICOS press release
