Miramoon Pharma Breakthrough: Two Lead Candidates Show Promise in Prestigious Publications
Miramoon Pharma has hit a major milestone with the publication of preclinical data for its two lead candidates in top-tier peer-reviewed journals.
MP-004: Orphan Drug Designation & Clinical Trial on the Horizon for Ophtalmological indications. Miramoon’s lead candidate, MP-004, has already secured Orphan Drug Designation from both the FDA and EMA for the treatment of retinitis pigmentosa, a degenerative retinal disease affecting over 2 million people worldwide without effective global treatment. Published in Investigative Ophthalmology & Visual Science (IOVS), the preclinical studies demonstrate how MP-004 enhances retinal function and preserves photoreceptors in the rd10 mouse model, significantly slowing disease progression. These results highlight the company’s potential to transform the treatment landscape for neurodegenerative diseases.
Miramoon Pharma is now completing pre-IND studies and plans to launch a clinical trial next year—a crucial step toward significantly enhancing patients' quality of life by preserving vision for longer, promoting independence, easing adaptation to vision loss, and increasing opportunities for future curative treatments.
MP-010: A New Hope for ALS. Meanwhile, MP-010, targeting amyotrophic lateral sclerosis (ALS), has shown remarkable neuroprotective benefits in the gold-standard SOD1G93A mouse model. Published in the British Journal of Pharmacology, the study reveals how MP-010 improves motor coordination, enhances neuromuscular junction innervation, and preserves spinal motor neurons, hallmarks of slowing disease progression. These promising results position MP-010 as a potential breakthrough therapy for ALS, where treatment options remain limited.
Innovation Rooted in Science.
Founded as a spin-off of the Biogipuzkoa Health Research Institute and the University of the Basque Country (UPV/EHU), Miramoon Pharma has pioneered a unique class of FKBP12-modulating small molecules that address calcium dysregulation, a key driver of neurodegenerative diseases. With a strong IP portfolio, an experienced leadership team, and now compelling preclinical evidence, Miramoon is well-positioned to advance its pipeline into clinical development.
Stay tuned—2025 promises to be a defining year as Miramoon takes its first steps into the clinic, bringing new hope to patients battling currently untreatable conditions.