Minovia Therapeutics Gets FDA OK for MNV-201 Phase Ib Trial in Low Risk Myelodysplastic Syndrome

Minovia Therapeutics Ltd., a clinical-stage biopharmaceutical company focusing on mitochondrial cell therapies, recently announced that the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for MNV-201. This innovative therapy, an autologous hematopoietic stem cell product enhanced with allogeneic mitochondria, is set to begin Phase Ib clinical trials in patients with Low Risk Myelodysplastic Syndrome (MDS).

Anemia is a prevalent and critical issue in patients suffering from low-risk myelodysplastic syndrome (LR-MDS), affecting nearly 90% of these patients. This condition significantly impacts their quality of life and is often associated with reduced progression-free and overall survival rates.

"The FDA’s approval of our IND is a crucial milestone for Minovia, enabling the clinical evaluation of our allogeneic mitochondrial cell therapy. We are set to progress with the Phase Ib clinical trial for this pioneering treatment for low-risk MDS patients," stated Natalie Yivgi Ohana, PhD, CEO of Minovia. "We have already safely dosed two MDS patients in an ongoing study under Israel's Ministry of Health. We are eager to treat more patients under this IND and to explore the potential of MAT in improving anemia among these individuals."

The Phase Ib clinical trial is designed as an open-label, dose exploration study to assess MNV-201's safety and efficacy in individuals with low-risk MDS. This trial will investigate different dose levels to determine the most effective and safe regimen for patients. It will also evaluate the therapy's effectiveness in improving anemia and the durability of the response. The study plans to enroll at least three patients in each of the low, medium, and high dose cohorts, with a total enrollment of up to 15 patients.

MNV-201 is a mitochondrial cell therapy product composed of autologous hematopoietic stem cells enriched with allogeneic mitochondria. It aims to restore mitochondrial function in the hematopoietic stem cells of low-risk MDS patients, enhancing their ability to differentiate into erythroid cells and potentially improving anemia. Preclinical studies have shown that MNV-201 can be safely administered with a low risk of immunogenicity. The therapy's scalable manufacturing process addresses the needs of a significant number of patients who might benefit from MNV-201.

Minovia Therapeutics Ltd. is focused on developing mitochondrial cell therapies for both primary-genetic and age-related mitochondrial diseases. Their leading product candidate, MNV-201, is created from mobilized peripheral blood and autologous CD34+ cells enriched with allogeneic, cryopreserved, placental-derived mitochondria. This is achieved through Minovia’s proprietary Mitochondrial Augmentation Technology (MAT). The objective is to restore the function of hematopoietic stem cells in patients experiencing mitochondrial dysfunction due to mtDNA mutations or deletions, seen in both pediatric patients with primary mitochondrial diseases and adults with age-related bone marrow failure disorders. MNV-201 is currently undergoing clinical trials for pediatric patients with single-large scale mtDNA deletion syndromes, such as Pearson Syndrome and Kearns-Sayre Syndrome, and for low-risk Myelodysplastic Syndrome.

Myelodysplastic syndromes (MDS) represent a cluster of bone marrow failure diseases where the blood-forming cells in the bone marrow become abnormal, resulting in defective differentiation and production of blood cells. Patients with MDS endure a significant symptom burden and face risks of death due to complications from cytopenias or progression to acute myeloid leukemia (AML). Typically, MDS is an age-related disease, with the median age of diagnosis around 70 years.

Scientific literature suggests a link between mitochondrial dysfunction and the progression of MDS. Ineffective hematopoiesis in MDS is attributed to the increased susceptibility of clonal myeloid progenitors to apoptosis, potentially triggered by factors like mitochondrial polarization due to iron retention in ringed sideroblasts. Some MDS patients exhibit sideroblastic anemia, similar to conditions seen in Pearson Syndrome, implicating mitochondrial dysfunction in hematopoietic stem progenitor cells as a factor in MDS pathology.