Minovia Therapeutics Ltd., an Israeli clinical-stage biotechnology company, announced on Wednesday that the US Food and Drug Administration has granted Orphan Drug Designation for its lead investigational compound MNV-201 for myelodysplastic syndrome (MDS).

Myelodysplastic syndrome is marked by ineffective haematopoiesis, blood cytopenia and risk of progression to acute myeloid leukaemia with substantial symptom burden and mortality risk.

MNV-201 is described as a first-in-class cell therapy using Minovia's Mitochondrial Augmentation Technology to add healthy mitochondria into a patient's own stem cells to restore function.

The Orphan Drug Designation adds to the existing FDA Fast Track Designation in MDS and prior Fast Track and Rare Paediatric Disease Designations for MNV-201 in Pearson Syndrome.

Minovia is currently conducting a Phase 1b study of MNV-201 in low-risk MDS patients and has dosed six of the nine expected patients so far.

The company also said it has entered into a definitive business combination agreement with Launch One Acquisition Corp. (NASDAQ: LPAA). Following the expected closing projected for late 2025, the combined company will operate as Minovia Therapeutics and trade on Nasdaq under a new ticker.