Trial Outcomes Support Progression to Advanced Development Stages, with the Program Anticipated to be Ready for Phase 2b/3 in the Second Half of 2026
BERLIN, GERMANY, January 29, 2026 – OMEICOS, a biopharmaceutical company in the clinical stage focused on developing pioneering small molecule therapies for mitochondrial and inflammatory conditions, announced the successful completion of its multi-center, open-label Phase 2a PMD-OPTION Study. This study evaluated its primary candidate, OMT-28, in individuals diagnosed with Primary Mitochondrial Disease (PMD). The findings from the study indicate OMT-28’s therapeutic capacity to enhance the physical condition in PMD patients, evidenced by a notable recovery of impaired mitochondrial fitness among those who responded to treatment. The study also reaffirmed OMT-28’s excellent safety and tolerability profile, which has now been assessed in over 220 individuals. OMEICOS is currently preparing for a potentially pivotal Phase 2b/3 study involving PMD patients experiencing myopathy and/or cardiomyopathy across sites in the EU and US. The company anticipates being prepared to commence this study later this year, contingent upon the finalization of partnership discussions.
PMD encompasses a diverse array of conditions, including the more common subtypes such as MELAS, non-MELAS, and MIDD. Patients with PMD endure debilitating and life-threatening health consequences, including severely restricted physical endurance and disease-related alterations in cardiac and skeletal muscles, alongside associated neurological disorders. OMT-28, an orally available biased modulator, targets the GPCR-receptor S1PR1 (Sphingosine-1-Phosphate Receptor 1), thereby initiating the downstream activation of mitochondrial sirtuin family members SIRT1 and SIRT3. By targeting S1PR1 and activating SIRT1/SIRT3, OMT-28 combines immunomodulation with mitochondrial protection—a dual mechanism designed to address both inflammation and energy deficiencies characteristic of primary mitochondrial diseases.
“Enhancing physical performance through improved mitochondrial metabolism and reduced oxidative stress holds significant promise for PMD. Our PMD-OPTION study results demonstrate a strong correlation between OMT-28 treatment, the observed positive impact on mitochondrial bioenergetics and fitness, and relevant clinical improvements in functional measures, which could translate into substantial patient benefit,” stated Dr. Robert Fischer, CEO/CSO of OMEICOS Therapeutics. “The profound effects on NAD⁺ and GSH levels, as well as the simultaneous improvement of the NAD⁺/NADH and GSH/GSSG ratios observed in the responder group, serve as integrated indicators of enhanced electron transport chain function and cellular redox homeostasis. Overall, these results provide a robust pathway for late-stage development.”
Overview of Study Design and Results
The PMD-OPTION study enrolled a total of 29 PMD patients diagnosed with mitochondrial tRNA point mutations or single mtDNA deletions. Recruitment took place across nine specialized centers in Germany, Italy, and The Netherlands. The study garnered considerable interest from both patients and key opinion leaders (KOLs), leading to prompt recruitment and a high level of adherence to the study protocol and follow-up appointments. The study incorporated a 12-week untreated run-in phase, serving as an integrated control to capture patients’ natural disease progression and baseline parameters for evaluating treatment outcomes. Following this, all patients received a 24 mg once-daily dose of OMT-28 for a treatment duration of up to 24 weeks. The study concluded after a subsequent four-week follow-up period. The concentration of GDF-15, a prospective biomarker reflecting cellular stress and inflammation, was utilized as a screening and inclusion criterion, while a reduction in GDF-15 was designated as a primary endpoint in addition to demonstrating safety and tolerability in PMD patients. The study’s outcome did not support the selection of GDF-15 in this context, suggesting that OMT-28 acts downstream of the GDF-15 release mechanism.
To evaluate clinically meaningful improvements within the study population, the PMD-OPTION study employed a combination of objective exercise endpoints and patient-reported outcomes. Utilizing these assessment tools, the study revealed a response rate exceeding 60%. In both the 12-Minute Walk Test (12 MWT) and the 5x Sit-to-Stand Test (5xSST), which are recognized endpoints for pivotal studies, the entire study population demonstrated improvements from baseline. Furthermore, OMT-28 responders exhibited profound and statistically significant (12 MWT) clinical improvements when compared to non-responders.
These findings showed a strong correlation with a highly significant increase in total NAD+ levels within the responder group compared to baseline, and a clear distinction between responders and non-responders in NAD+/NADH ratios throughout the study period. In patients who responded to OMT-28 treatment, mean NAD+ levels were approximately 30% higher than baseline, bringing this critical indicator of mitochondrial energy metabolism and redox status close to healthy ranges. Similarly, OMT-28 demonstrated a significant improvement in total GSH and GSH/GSSG ratios—key indicators of reduced oxidative stress in mitochondrial diseases—thereby restoring normal, healthy levels and even showing a trend toward further enhancement. Collectively, these results illustrate OMT-28’s capacity to normalize both NAD⁺/NADH and GSH/GSSG ratios, addressing the fundamental pathologies of PMD—energy deficiency and oxidative stress—thus distinguishing it from single-mechanism approaches and supporting its potential as a first-in-class therapy.
About OMEICOS
OMEICOS Therapeutics has identified a series of metabolically stable synthetic analogues of epoxyeicosanoids derived from omega-3 fatty acids. These compounds hold the potential to treat mitochondrial dysfunction, inflammatory, cardiovascular, and other diseases. Epoxyeicosanoids activate cell type-specific endogenous pathways that promote the protection of organs and tissues. OMEICOS’ small molecules are orally available and exhibit enhanced biological activity and pharmacokinetic properties compared to their naturally occurring counterparts. For more information, please visit:
Contact Information
| OMEICOS Therapeutics GmbH Dr. Robert Fischer, CEO, CSO Phone: +49 (0) 30 9489 4810 E-Mail: |
Media Inquiries Valency Communications Mario Brkulj Phone: +49 (0) 160 93529951 E-Mail: |
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