Minovia announces interim data from Phase 2 trial in Pearson Syndrome

Minovia Therapeutics announces interim results of the ongoing Phase 2 trial of the Company’s lead investigational compound MNV-201 in Pearson Syndrome, a fatal pediatric mitochondrial disease characterized by sideroblastic anemia, failure to thrive and exocrine pancreas dysfunction. The interim analysis was presented at the United Mitochondrial Disease Foundation Annual Meeting on June 20, 2025. The analysis showed that MNV-201 has a positive safety profile even in this fragile patient population, currently meeting the Phase 2 trial’s primary safety endpoint. In the presentation, Minovia’s CSO Noa Sher reported no treatment related severe adverse events and no anti-mitochondrial antibodies were detected. In addition, all adverse events were transient in nature and the majority resolved within four days. Most adverse events were associated with the apheresis procedure necessary to procure patient-derived cells and conducted prior to the drug’s administration. In addition to safety data, Minovia is collecting preliminary efficacy data based on a novel endpoint identified through a natural history study performed at the Children’s Hospital of Philadelphia and supported by Minovia funding. Results indicate that at the six-month follow-up, two of the first three patients experienced improved growth parameters relative to those prior to treatment, as measured by height standard deviation score. Quality of life scores also showed improvement for two of the first three patients. The Company continues to enroll patients in the trial, which is expected to conclude before the end of 2025.In addition to the three patients in the trial, the Company gathered data from two compassionate use patients affected by Kearns-Sayre syndrome, another mitochondrial deletion syndrome. In addition to a favorable safety profile, early data indicates improved quality of life scores for both patients. The FDA has granted both Fast Track and Rare Pediatric Disease Designation to MNV-201, which is in Phase 2 clinical trials for the treatment of Pearson Syndrome, an ultra-rare and life-threatening mitochondrial disorder affecting children. The Company is advancing interactions with the FDA to align on a pivotal trial design and expects to initiate registrational studies in 2026.