OMEICOS Fast-Tracked to Phase 3, A Potential Game-Changer for PMD

BERLIN, GERMANY – July 21, 2026 – In a move that significantly de-risks its lead asset and accelerates its path to market, late-stage biopharmaceutical company OMEICOS Therapeutics announced today that it has received a strong endorsement from the U.S. Food and Drug Administration (FDA). Following a successful End-of-Phase 2 meeting, the company's first-in-class drug candidate, OMT-28, will advance directly into a pivotal Phase 3 study for the treatment of Primary Mitochondrial Diseases (PMD).

This decision allows OMEICOS to bypass a lengthier, more traditional development path, signaling the FDA's confidence in the existing data and the urgent need for new therapies in this devastating rare disease category. For investors and patients alike, the announcement marks a critical inflection point, transforming a promising clinical candidate into a late-stage asset with a clear regulatory pathway.

A Beacon of Hope in a Landscape of Unmet Need

Primary Mitochondrial Diseases are a group of complex, debilitating genetic disorders caused by dysfunctional mitochondria—the powerhouses of our cells. These conditions lead to a diverse array of severe symptoms, impacting organs with high energy demands like the heart, brain, and muscles. Patients often suffer from severely limited physical stamina, muscle weakness, neurological disorders, and life-threatening cardiomyopathy. For the vast majority, treatment has been limited to palliative care that manages symptoms without addressing the root cause.

The therapeutic landscape, while evolving, remains sparse. Recent FDA approvals, such as KYGEVVI for Thymidine Kinase 2 Deficiency (TK2D) and FORZINITY for Barth Syndrome, were landmark achievements but cater to very specific, small subsets of the PMD population. This leaves a significant treatment void for patients with more common subtypes, including MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes) and MIDD (Maternally Inherited Diabetes and Deafness). OMT-28 is being developed to address these broader groups, positioning it to serve a substantial portion of the PMD community currently without an approved therapeutic option. The convenience of a once-daily oral pill further distinguishes it from injectable alternatives, potentially improving long-term adherence and quality of life.

The Science Behind the FDA's Green Light

The FDA's decision to accelerate OMT-28 was not made in a vacuum; it was underpinned by compelling results from the Phase 2a PMD-OPTION study. This open-label trial provided robust evidence of OMT-28's unique mechanism of action and its potential to meaningfully impact the disease.

OMT-28 is a synthetic analogue of omega-3 fatty acid metabolites, engineered for stability and oral availability. It employs a novel dual mechanism that targets the core pathologies of PMD: energy deficiency and oxidative stress. By modulating the S1PR1 receptor, OMT-28 activates two crucial mitochondrial sirtuins, SIRT1 and SIRT3. These enzymes are vital regulators of mitochondrial health, promoting the creation of new mitochondria and enhancing the cell's antioxidant defenses.

Data presented at major mitochondrial disease conferences, Euromit 2026 and Mito MED 2026, revealed that OMT-28 delivered on this scientific promise. Key findings from the Phase 2a study showed:

• Biomarker Response: Over 60% of patients were classified as responders based on a significant reduction in GDF-15, a key biomarker of mitochondrial stress.
• Metabolic Restoration: Responding patients saw their NAD+ levels—a crucial indicator of cellular energy metabolism—increase by approximately 30%, approaching the range seen in healthy individuals.
• Functional Improvement: Crucially, the biochemical improvements translated into tangible physical benefits. Responders demonstrated profound and statistically significant improvements in the 12-Minute Walk Test (12MWT) compared to non-responders, a key functional endpoint accepted by regulators.

This combination of a strong safety profile, established across more than 190 individuals, and clear signals of both biological and functional efficacy gave the FDA the confidence to support a direct-to-Phase-3 strategy.

A Strategic Inflection Point for OMEICOS

For OMEICOS, a 2013 spin-off from Berlin's renowned Max Delbrück Center, this FDA guidance is a watershed moment. The company, backed by a syndicate of European venture firms including Forbion and Vesalius Biocapital, has strategically pivoted OMT-28's development towards its area of greatest impact. While initially explored for atrial fibrillation, the compound's profound effects on mitochondrial function and inflammation proved most promising in PMD.

"The strong endorsement from the FDA marks a significant milestone for OMEICOS as a company and for our mission to address Primary Mitochondrial Disease," said Dr. Robert Fischer, CEO and CSO of OMEICOS. "The meeting’s outcome even surpassed our expectations, as we are now able to advance directly from our concluded Phase 2a study into a pivotal Phase 3 study."

Dr. Fischer’s subsequent comments on evaluating the "best possible infrastructure, strategic partners, and resources" are a clear signal to the market. Executing a global Phase 3 trial is a capital-intensive endeavor. This positive regulatory feedback massively strengthens OMEICOS's negotiating position as it seeks a partner to co-fund or license OMT-28 for its final development stage and commercial launch. The company's 'pipeline-in-a-drug' strategy, which envisions expanding OMT-28 into other diseases, further enhances its value proposition for potential partners.

Charting the Course: The Pivotal Phase 3 Trial Design

With the FDA's guidance in hand, OMEICOS has a well-defined plan for its pivotal study. The Phase 3 trial is designed to enroll up to 160 adult patients, who will be stratified by the three most common PMD subgroups: MELAS, non-MELAS, and MIDD. Participants will receive a 24 mg once-daily dose of OMT-28 over 24 weeks, with an option to continue in a long-term extension study for up to two years.

The trial will employ an adaptive design, a modern and efficient approach particularly suited for rare disease research. This allows for pre-planned adjustments based on interim analyses, optimizing the trial's chances of success while managing resources effectively. The primary endpoint is a robust composite measure combining two well-established functional tests: the 12-Minute Walk Test (12MWT) and the 5-repetition Sit-to-Stand Test (5xSST). This dual endpoint is designed to capture a comprehensive picture of improvement in both endurance and strength, reflecting a more holistic impact on a patient's daily life. Secondary endpoints will further assess quality of life and key exploratory biomarkers, providing a deep data set to support a future regulatory submission.