Minovia Therapeutics reports positive Phase 2 trial results for MNV-201 in Pearson Syndrome.

Minovia Therapeutics has reported positive interim results for its Phase 2 trial of MNV-201 in Pearson Syndrome, a fatal pediatric mitochondrial disease. The trial has met its primary safety endpoint, and MNV-201 has shown a positive safety profile in the patient population. The results were presented at the United Mitochondrial Disease Foundation Annual Meeting on June 20, 2025.

Minovia Therapeutics (NASDAQ: LPAA) has announced positive interim results from its Phase 2 trial of MNV-201 for Pearson Syndrome, a fatal pediatric mitochondrial disease. The trial demonstrated no treatment-related serious adverse events and showed preliminary efficacy signals in growth parameters. Key findings include improved growth parameters in two out of three initial patients at six-month follow-up, and enhanced quality of life scores. The company also reported favorable safety data from two compassionate use patients with Kearns-Sayre syndrome [1].

MNV-201 has received both Fast Track and Rare Pediatric Disease Designation from the FDA, which significantly enhances the development pathway. Fast Track status enables increased FDA interactions and potential rolling BLA submission, while the Rare Pediatric Disease designation positions Minovia for a potential priority review voucher upon approval, historically valued at $100+ million [1].

Minovia plans to initiate registrational studies in 2026 and is merging with Launch One Acquisition Corp., with the combined entity expected to trade on Nasdaq by late 2025. The company aims to complete enrollment by the end of 2025 and is preparing for pivotal registrational studies in 2026. The parallel business combination with Launch One Acquisition Corp. should provide public market access and potentially additional capital to support these advanced clinical stages [1].

For rare mitochondrial disorders like Pearson Syndrome, which manifests with sideroblastic anemia, growth failure, and pancreatic dysfunction, even modest clinical improvements can represent meaningful patient benefit. The quality of life improvements noted in two of three trial patients and both compassionate use Kearns-Sayre syndrome patients suggest potential broader applicability across mitochondrial deletion syndromes [1].

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 [1].

References:
[1] https://www.stocktitan.net/news/LPAA/minovia-therapeutics-announces-interim-data-from-phase-2-trial-in-4i479rhtq6ij.html