- The European Medicines Agency acknowledges the potential of the primary compound, MP1032, to address a significant unmet medical need in Duchenne Muscular Dystrophy (DMD).
- This designation facilitates the development of a safer long-term treatment.
- MP1032 has shown promise in maintaining muscle function and decreasing inflammation without interfering with regular cellular activities.
Zurich, July 29, 2025 – A pharmaceutical firm specializing in treatments for inflammatory and infectious conditions, announced today that its primary compound, MP1032, has been granted Orphan Drug Designation (ODD) by the European Medicines Agency for treating children with Duchenne muscular dystrophy (DMD). This achievement comes after the U.S. FDA also granted ODD, highlighting MP1032’s promise as a novel therapy that could lessen the need for high-dose corticosteroids, known to cause serious adverse effects such as osteoporosis, hindered growth, and metabolic problems.
The EMA’s ODD offers significant benefits, including procedural guidance from the EMA, exemption from fees, and up to 12 years of market exclusivity within the EU (an increase from 10 years, due to DMD’s focus on pediatric patients) upon regulatory approval. This validation by authorities is supported by strong preclinical evidence indicating MP1032’s capacity to maintain muscle function and decrease inflammation without interfering with normal cellular processes.
“This dual Orphan Drug Designation from both the FDA and EMA, along with the existing Rare Pediatric Disease Designation from the FDA, represent transformative developments for MetrioPharm and the Duchenne Muscular Dystrophy community,” stated CEO Thomas Christély. “MP1032’s distinct action—regulating mitochondrial reactive oxygen species in excessively active immune cells—has the potential to revolutionize the long-term management of Duchenne Muscular Dystrophy by significantly reducing corticosteroid dosages and associated adverse effects. We are expediting MP1032 towards Phase II clinical trials, working with patient organizations, and investigating collaborations to make this available to children requiring safer alternatives. Beyond Duchenne Muscular Dystrophy, our platform’s host-directed methodology has demonstrated potential in autoimmune conditions and even pandemics, as shown by our HERA-funded COVID-19 Phase IIa study.”
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About MP1032
MetrioPharm’s primary investigational drug, MP1032, is a distinctive small molecule designed to target exaggerated responses of activated immune cells by modulating a core, natural metabolic process within them. When activated, these cells’ mitochondria increase their energy metabolism, leading to the excessive production of reactive oxygen species (ROS). MP1032 is an innovative anti-inflammatory medication that reduces elevated ROS without affecting the normal, essential physiological ROS levels in other cells, thereby ensuring an excellent safety profile. When used alone, MP1032 yields therapeutic outcomes comparable to corticosteroids (cortisone-based treatments) but without their severe adverse effects. Unlike conventional anti-inflammatory agents, it replicates the benefits of corticosteroids as a single therapy while avoiding their toxicities, as shown in preclinical studies for Duchenne Muscular Dystrophy, multiple sclerosis, arthritis, and other conditions. Its host-directed mechanism also makes it suitable for infectious diseases, with Phase IIa results from 131 hospitalized COVID-19 patients (published in ) confirming its safety and effectiveness against viral inflammation, irrespective of the specific pathogen.
About DMD
Duchenne Muscular Dystrophy (DMD) is the most prevalent type of muscular dystrophy. This genetic condition is marked by the gradual deterioration of muscles, with symptoms typically appearing in early childhood, often between two and three years of age. The disorder predominantly impacts boys, leading to severe muscle wasting and heart failure. DMD considerably reduces life expectancy. Current standard treatments involve long-term administration of high-dose corticosteroids, which, despite causing serious side effects, can substantially delay disease progression.
About MetrioPharm AG
MetrioPharm, a private biotechnology company, is headquartered in Zurich, Switzerland, and operates an R&D subsidiary in Berlin, Germany. It is at the forefront of developing small-molecule treatments for inflammatory, autoimmune, and infectious diseases. Its platform, which modulates reactive oxygen species (ROS), has created a strong pipeline, featuring disease-specific combinations for conditions such as multiple sclerosis, arthritis, sepsis, inflammatory bowel disease, psoriasis, and rare diseases like Duchenne Muscular Dystrophy, which is its primary focus. Regarding infectious diseases, MetrioPharm is enhancing pandemic readiness, supported by its successful COVID-19 Phase IIa study, which received €7.9 million in funding from the European Health Emergency Preparedness and Response Authority (HERA). MP1032’s host-directed mechanism addresses excessive immune responses rather than targeting specific viruses, providing a rapid intervention for new threats. With a consistent safety record across preclinical and clinical investigations, MetrioPharm is positioned for strategic partnerships and future achievements.
Forward-looking statements
This press release includes forward-looking statements that carry inherent risks and uncertainties, reflecting MetrioPharm AG’s evaluation as of the publication date. These statements are not assurances or guarantees but are subject to various risks and unpredictable factors. No responsibility or guarantee is assumed, nor any claim made, regarding the currency, precision, or comprehensiveness of such data and information, and no reliance should be placed on this data and information, whether explicitly or implicitly.
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