Minovia Therapeutics Ltd. (“Minovia” or the “Company”), a clinical-stage biotechnology company developing novel therapies to treat mitochondrial diseases and combat age-related decline, announces that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to the Company’s lead investigational compound, MNV-201 for Myelodysplastic Syndrome (MDS), a serious age-related hematopoietic disease. This designation is in addition to the existing FDA Fast Track Designation in MDS, as well as both Fast Track and Rare Pediatric Disease Designations for MNV-201 in the treatment of Pearson Syndrome.

“We continue to receive validation from the FDA for the potential of our lead product, MNV-201, this time in the form of Orphan Drug Designation in MDS. MNV-201 targets the mitochondria, a critical multi-functional organelle. FDA designations such as ODD underscore the urgency of drugs treating these diseases affecting smaller populations, while providing additional benefits across the FDA process that, we expect, will prove both medically and financially valuable,” said Minovia Co-founder and CEO, Natalie Yivgi-Ohana, Ph.D.

“Orphan drug designation for MNV-201 marks an important milestone in our mission to address critical challenges in mitochondrial health in both primary and acquired mitochondrial diseases. By leveraging our expertise in mitochondrial and hematopoietic science, and through the innovative mechanism of action of our drug product, we hope to bring forward a treatment option that could significantly improve outcomes for MDS patients,” added Minovia Chief Scientific Officer, Noa Sher, PhD.

Orphan drug designation is a status granted by the FDA to drugs developed to treat rare diseases affecting fewer than 200,000 people in the U.S. This designation provides various incentives, such as tax credits and market exclusivity, to encourage the development of treatments for conditions that may not be profitable otherwise.  

The Company also announced entry into a definitive business combination agreement (the “Business Combination Agreement”) with Launch One Acquisition Corp. (Nasdaq: LPAA, “Launch One”), a publicly traded special purpose acquisition company. Following the expected closing of the transaction contemplated by this Business Combination Agreement (the "Business Combination"), projected for late 2025, the combined company will operate as Minovia Therapeutics and trade on Nasdaq under a new ticker symbol.

MDS is defined by ineffective hematopoiesis resulting in blood cytopenia, and clonal instability with a risk of evolution to Acute Myeloid Leukemia (AML). Patients with MDS collectively have a high symptom burden and are also at risk of death from complications of cytopenia and AML. The goals of therapy for patients with MDS are to improve cytopenia, reduce disease-associated symptoms and the risk of disease progression and death, thereby improving both quality of life and lifespan. The median age at diagnosis of MDS is ~70 years, but surprisingly some of the Pearson Syndrome patients develop MDS in a much higher prevalence relative to the disease population. About 15% of the MDS patients will present with Sideroblastic Anemia, the most common symptom in Pearson Syndrome. Minovia developed novel blood biomarkers to measure mitochondrial health and has been able to demonstrate for the first time that MDS is presumably an age-related mitochondrial disease. 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.

MNV-201 is a first-in-class cell therapy that uses Minovia’s proprietary Mitochondrial Augmentation Technology (MAT) to add healthy, energy-producing mitochondria into a patient’s own stem cells — aiming to restore organ function and improve health. In early-stage clinical studies, MAT has demonstrated a strong safety profile and signs of multi-system benefit in patients with Pearson Syndrome, including improvements in growth, muscle function, hematologic stability, and improved quality of life.

Source: globenewswire.com