OMEICOS takes repurposed drug directly into Phase 3
Berlin-based OMEICOS Therapeutics can move OMT-28 directly into a pivotal Phase 3 trial in primary mitochondrial diseases after receiving supportive feedback from the U.S. Food and Drug Administration (FDA).
Why it matters: The regulatory agreement validates a strategic pivot that began after OMT-28 failed to show sufficient efficacy in atrial fibrillation and gives the small biotech a potentially shorter route toward registration in a group of rare diseases with few treatment options.
Zoom in: OMEICOS said the FDA endorsed its late-stage development plans during an End-of-Phase 2 meeting, removing the need for an intermediate Phase 2b study.
- The proposed adaptive Phase 3 trial will enroll up to 160 adults with common primary mitochondrial disease subtypes, including MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes), non-MELAS and maternally inherited diabetes and deafness, or MIDD.
- Participants will receive 24 mg of oral OMT-28 once daily for 24 weeks, with an extension lasting up to two years.
- The primary endpoint will combine two measures of physical function: a 12-minute walking test and a five-times sit-to-stand test.
Backstory: OMEICOS originally developed OMT-28 for cardiovascular diseases, particularly the prevention of atrial fibrillation.
- A placebo-controlled Phase 2 study did not produce enough evidence of efficacy to continue in that indication. However, the trial showed changes in inflammatory marker IL-6 and mitochondrial stress marker GDF-15, prompting the company to redirect the programme toward mitochondrial disorders in 2022.
- Existing investors, including Forbion, High-Tech Gründerfonds and Remiges Ventures, backed the change in strategy.
How it works: OMT-28 is a synthetic analogue of a metabolite derived from omega-3 fatty acids. The small molecule modulates the S1PR1 receptor and activates mitochondrial proteins SIRT1 and SIRT3. OMEICOS believes the combination can reduce inflammation and oxidative stress while improving cellular energy production.
What the data show: The open-label Phase 2a PMD-OPTION study enrolled 29 patients across Germany, Italy and the Netherlands.
- OMEICOS reported improvements in physical performance and markers of mitochondrial function among a subset of participants. The study did not include a placebo arm, instead comparing results with a 12-week untreated run-in period.
Yes, but: Primary mitochondrial diseases are genetically and clinically heterogeneous, and the Phase 2a efficacy findings came from a small, uncontrolled study and a responder subgroup. A larger randomized trial will need to show that the improvements translate across a clearly defined patient population.
What’s next: OMEICOS is now seeking financing and a development partner to launch the Phase 3 study.
