MNV-201 / Minovia Therapeutics - LARVOL DELTA

Therapeutic potential of mitochondrial transplantation in the treatment of MDS (ASH 2025) - P1 | "This was a 69-year-old with transfusion-dependent MDS-LB (MDS with low blasts) refractory toerythropoiesis stimulating agents and luspatercept. The patient, transfusion dependent for 7 monthsprior to enrollment, achieved transfusion independence two months following MNV-201 treatment...Collectively, the accumulated mouse and human MDS studies suggest the potential for mitochondrialaugmentation of HSPCs to improve the differentiation defect of a subset of human MDS and postponetransformation to acute leukemia. These results support further evaluation of mitochondrialaugmentation of HSPCs as a novel autologous cell therapy approach to treat MDS patients."

Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Metabolic Disorders • Myelodysplastic Syndrome • Transplantation • CD34 • CD48 • GDF15 • GYPA • NUP98 • PTPRC • TFRC

Mitochondrial Augmentation of Hematopoietic Stem and Progenitor Cells As a New Therapeutic Approach for Myelodysplasia (ASH 2024) - "Recently, an allogeneic placental mitochondria source was developed (MNV-201) and is undergoing a phase I trial to treat Pearson Syndrome, a pediatric disease caused by single mitochondrial DNA deletions, resulting in sideroblastic anemia and high frequency of MDS...Taken together, the accumulated studies with mouse and human MDS models suggest the potential for mitochondrial augmentation of HSPCs to improve the differentiation defect of a subset of human MDS, enhance in vivo reconstitution potential, and postpone transformation to acute leukemia. These preclinical results support the potential of mitochondrial augmentation of HSPCs as a novel approach to treat MDS patients utilizing autologous cell therapy."

Anemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Metabolic Disorders • Myelodysplastic Syndrome • Oncology • Pediatrics • NUP98 • PTPRC • TFRC

Minovia Therapeutics Receives FDA Orphan Drug Designation for MNV-201 in Myelodysplastic Syndrome (GlobeNewswire) - "As such, Minovia is currently conducting a Phase Ib study of MNV-201 in low-risk MDS patients."

Orphan drug • Myelodysplastic Syndrome

Minovia Therapeutics Receives FDA Fast Track Designation for MNV-201 in Myelodysplastic Syndrome (GlobeNewswire) - "As such, Minovia is currently conducting a Phase Ib study of MNV-201 in low-risk MDS patients. Six out of the nine expected patients in the study have been dosed so far."

Fast track • Trial status • Myelodysplastic Syndrome

Minovia Therapeutics Announces FDA Fast Track and Rare Pediatric Disease Designations for MNV-201 in Pearson Syndrome (GlobeNewswire) - "Minovia Therapeutics Ltd...announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to the Company’s lead investigational compound, MNV-201. The FDA has also granted 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 finalize a pivotal trial design and expects to initiate registrational studies in 2026."

Fast track • FDA event • New trial • Rare Diseases

Evaluate the Safety and Therapeutic Effects of a Single Intravenous Infusion (IV) of Autologous CD34+ Cells Enriched With Allogenic Placenta-derived Mitochondria in Patients With a Diagnosis of Pearson Syndrome (PS) (clinicaltrials.gov) - P2 | N=6 | Recruiting | Sponsor: Minovia Therapeutics Ltd. | Phase classification: P1 ➔ P2

Phase classification • Metabolic Disorders

Minovia Therapeutics Announces FDA Clearance of Second IND Application, for a Phase II Clinical Trial of Lead Product MNV-201 in Pearson Syndrome (GlobeNewswire) - "Minovia Therapeutics Ltd...today announced that the U.S. Food and Drug Administration (FDA) has cleared its second Investigational New Drug (IND) application for MNV-201...The IND supports the initiation of a Phase II clinical trial of MNV-201 in pediatric patients with Pearson Syndrome, a primary mitochondrial disease."

IND • Trial status • Rare Diseases

Minovia Therapeutics Announces FDA Clearance of IND Application for a Phase Ib Clinical Trial of MNV-201 in Low Risk Myelodysplastic Syndrome (GlobeNewswire) - "Minovia Therapeutics Ltd...announced that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for MNV-201, an autologous hematopoietic stem cell product augmented with allogeneic mitochondria. The IND supports the initiation of a Phase Ib dose exploration clinical trial of MNV-201 in patients with Low Risk Myelodysplastic Syndrome (MDS)....The Phase Ib clinical trial is an open-label, dose exploration study to evaluate the safety and efficacy of MNV-201 in subjects with low risk MDS....The study is expected to enroll at least three patients each in the low, medium and high dose cohorts, and up to a total of 15 patients in total."

IND • Trial status • Myelodysplastic Syndrome

A Study to Evaluate the MNV-201 in Patients With Low Risk MDS (clinicaltrials.gov) - P1 | N=5 | Recruiting | Sponsor: Minovia Therapeutics Ltd.

New P1 trial • Hematological Malignancies • Myelodysplastic Syndrome • Oncology