MetrioPharm Investor-Newsletter QIV 2025
Dear shareholders,
As we approach the end of a turbulent 2025, we are pleased to report that MetrioPharm has achieved several key milestones in advancing MP1032 to the next stage of development.
A robust non-clinical data package is essential to prepare for clinical trials. In 2025, we therefore focused on further characterizing dosing, safety, how MP1032 acts in the body (ADME, PK/PD, mode of action), and continuing to optimize and de-risk manufacturing and supply. These activities are designed to build a solid scientific and regulatory foundation while maintaining flexibility to pursue different development pathways in the future.
Key progress in dose selection and safety
A core activity was a juvenile animal model dose-finding study designed to establish appropriate dose levels for a subsequent toxicology study. The data generated represent an important contribution to early regulatory interactions (e.g., in the context of FDA pre-IND discussions) as well as to the further strategic planning of future clinical studies. In this preliminary study, no indications of MP1032-related toxicity were observed.
EMA Orphan Drug Designation
On 18 July 2025, the European Medicines Agency (EMA) granted orphan drug designation (ODD) to MP1032 for the treatment of Duchenne muscular dystrophy (DMD), following the ODD previously received from the U.S. FDA. This status represents an important regulatory milestone in the context of a rare, severe disease with high unmet medical need. During the evaluation process, the EMA requested additional data, which we successfully generated using the established mdx mouse model of DMD. The results showed positive effects and contributed significantly to the EMA’s positive decision.
How MP1032 works in the body
In 2025, we conducted additional preclinical studies (ADME, PK/PD, and mode of action) to characterize MP1032 further:
- Mode of action: Initial investigations confirm that MP1032 can neutralize specific reactive oxygen species (ROS), such as superoxide, thereby reducing the excessive production of pro-inflammatory cytokines. These ROS act upstream in inflammatory signaling cascades that can lead to cytokine release.
- ADME: We gained additional insights into the absorption, distribution, metabolism, and excretion of MP1032, improving our understanding of its bioavailability following oral administration. The favorable safety profile of MP1032 was also reconfirmed in this study.
- PK/PD in mice (dose-exposure-effect relationship): We observed a linear, highly predictable relationship between orally administered MP1032 dose and systemic exposure (plasma levels). In addition, anti-inflammatory effects were confirmed, including reductions in cytokines such as IL-1β, IL-6, and TNF-α. These results provide an important basis for dose selection in future clinical trials.
Manufacturing and independence
On the manufacturing side, we initiated and implemented several key steps in 2025:
- Drug substance (API) supply: To reduce the risk of dependency on a single supplier, we completed technology transfer and established production with an additional drug substance manufacturer.
- Oral bioavailability: We launched a project to improve the bioavailability of MP1032 after oral dosing. Several promising formulation concepts have been developed and are currently being evaluated. The most promising approach(es) will advance to the next step.
- Sterile i.v. formulation: We have begun preliminary work on a sterile formulation to enable future intravenous administration of MP1032. Our goal is to have a GMP-compliant product available by 2026 to support potential studies, such as a sepsis study.
Acknowledgements
Throughout these steps, we were fortunate to have the valuable support of our Scientific Advisory Board. The board's members are recognized experts in their fields and have significantly strengthened our work through their experience and independent perspective. We would like to express our sincere thanks and look forward to continuing to work together to tackle the tasks ahead.
We would also like to thank our shareholders for their continued trust, as well as all MetrioPharm employees and the members of the Board of Directors for their tireless commitment.
We wish you and your families a restful holiday season and a healthy and successful 2026.
With best regards,
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Jörg Gruber |
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Thomas Christély |
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