Pipeline
The PMD-OPTION Study
Our lead program OMT-28 is a stable synthetic analog of the omega-3 fatty acid metabolite 17,18-EEQ, which has a structure optimized to provide high efficacy, safety and oral bioavailability.
OMT-28 reduces mitochondrial dysfunction and oxidative stress via activation of the AMPK/SIRT-1/PGC1-alpha axis leading to a significant reduction of mitochondrial stress marker GDF-15 and inflammation stress marker IL-6 in a Phase 2 study in patients with atrial fibrillation and in preclinical experiments. The excellent safety and tolerability of OMT-28 was confirmed already in 190 individuals.
OMT-28 has successfully been evaluated in the multi-centre, open-label Phase 2a PMD-OPTION Study evaluating in patients with Primary Mitochondrial Disease (PMD). The study results demonstrate OMT-28’s therapeutic potential to improve the physical condition in PMD based on significant recovery of the impaired mitochondrial fitness in the responding patients.
The PMD-OPTION study enrolled a total of 29 PMD patients with mitochondrial tRNA point mutations or single mtDNA deletions across nine expert sites in Germany, Italy, and The Netherlands. The study generated strong interest among patients and key opinion leaders (KOLs), resulting in timely recruitment and a high degree of compliance with the study protocol and follow-up appointments. The study included a 12-week untreated run-in phase as an integrated control, capturing the patients’ natural history and baseline parameters for evaluating treatment results. Subsequently, all patients received a 24 mg once-daily dose of OMT-28 for a treatment period of up to 24 weeks. The study ended after a subsequent four-week follow-up period. The level of GDF-15, a prospective biomarker for reflecting cellular stress and inflammation, was used as a screening and inclusion criteria, while reduction of GDF-15 was used as a primary endpoint next to demonstrating safety and tolerability in PMD patients. The study outcome did not support the choice of GDF-15 in this setting suggesting that OMT-28 is acting downstream of the release mechanism of GDF-15.
To assess clinically meaningful improvements in the study population, the PMD-OPTION study utilized a combination of objective exercise endpoints and patient-reported outcomes. Using these measures, the study demonstrated a response rate of more than 60%. In the 12-Minute Walk Test (12 MWT) and the 5x Sit-to-Stand Test (5xSST), both accepted endpoints for pivotal studies, the entire study population showed improvements over baseline, while OMT-28 responders exhibited profound and statistically significant (12 MWT) clinical improvements compared to non-responders.
These results strongly correlated with a highly significant increase in total NAD+ levels in the responder group compared to baseline, and a clear separation between responders and non-responders in NAD+/NADH ratios over the course of the study. In patients responding to OMT-28 treatment, mean NAD+ levels were approximately 30% higher compared to baseline, bringing this crucial 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 reestablishing normal, healthy levels and even showing a trend toward further enhancement. Together, these results demonstrate that OMT-28’s ability to normalize both NAD⁺/NADH and GSH/GSSG ratios addresses the core pathologies of PMD—energy deficiency and oxidative stress—differentiating it from single-mechanism approaches and supporting its potential as a first-in-class therapy.
Study Design
The PMD-OPTION study was divided into 3 parts:
- Screening / run-in phase w/o treatment: 12 weeks
- Treatment Phase: 24 weeks
- Safety follow-up: 4 weeks
The total duration of the study is 40 weeks.
6 visits at the clinical study centers are planned during the study.
Patients who completed the screening / run-in phase received the study medication OMT-28 in the treatment phase.
Recruitment has been concluded. A list of participating clinical centers is available here.
OMT-28, a Phase 2 Clinical Candidate, ready for evaluation in CV Inflammation
OMT-28 has completed a Phase 2 clinical study for the prevention of atrial fibrillation. While the impact on atrial fibrillation was not sufficient to progress this indication, OMT-28 demonstrated a significant positive impact on a number of inflammatory biomarkers such as IL-6 and mitochondrial dysfunction marker GDF-15.
OMT-28 targets inflammation plus mitochondrial dysfunction, a unique mode of action. The clinical data, together with pre-clinical anti-inflammatory and anti-atherosclerotic data support the application of OMT-28 in the treatment of cardiometabolic syndromes. We are actively planning a proof of concept clinical study in patients with chronic inflammation in coronary artery disease on biomarkers of inflammation. In independent outcome studies, a reduction of these biomarkers has been directly associated with positive clinical outcomes.
In a previously performed Phase 1 study, OMT-28 exhibited an excellent tolerability profile and showing no safety signals in vital signs or safety laboratory parameters up to the maximum dose tested of 60 mg. This excellent overall and also cardiovascular safety profile has been confirmed in our Phase 2 study with atrial fibrillation patients.
Broadening the Pipeline in Ophthalmology
In addition to the company’s development activities in the cardiovascular space, OMEICOS has begun branching out into additional indications, initially focusing on severe eye disorders.
Exploratory work with OMEICOS’ therapeutic candidates included the generation of preclinical data comparing our approach to the current standard-of-care, injections of anti-VEGF therapies directly into the eye.
In a widely used mouse model for wet AMD OMEICOS’ approach reduced the formation of abnormal blood vessels and the leakiness of these blood vessels typical for wet AMD.
The therapeutic effects of oral OMT-28 observed were comparable with the intravitreally applied anti-VEGF therapy, in this case Aflibercept. Considering that therapeutic compounds based on OMEICOS’ technology have the potential to be conveniently given to patients as a pill, avoiding the need for injections into the eye, these results provide a very solid basis for further development in these indications.
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Nature as the Blueprint for a Family of First-in-class Therapeutics.
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