Minovia reports positive interim results for rare disease treatment

Published 24/07/2025, 13:36
Minovia reports positive interim results for rare disease treatment

HAIFA, Israel - Minovia Therapeutics Ltd., soon to merge with Launch One Acquisition Corp (LPAA) - a company currently valued at $299 million with a Fair financial health rating according to InvestingPro - announced positive interim results from its ongoing Phase 2 trial of MNV-201 in Pearson Syndrome, a fatal pediatric mitochondrial disease characterized by sideroblastic anemia and pancreas dysfunction.

The interim analysis, presented at the United Mitochondrial Disease Foundation Annual Meeting on June 20, 2025, showed that MNV-201 has met the trial’s primary safety endpoint with no treatment-related severe adverse events reported.

According to the company, two of the first three patients experienced improved growth parameters after six months of treatment, as measured by height standard deviation score. Quality of life scores also showed improvement for two of these patients.

"These Phase 2 data show extremely encouraging results, even in such a small patient sample," said Natalie Yivgi-Ohana, Ph.D., Minovia’s Co-founder and CEO, in the press release.

The company reported that all adverse events were transient, with the majority resolving within four days. Most adverse events were associated with the apheresis procedure conducted prior to drug administration.

MNV-201 has received both Fast Track and Rare Pediatric Disease Designation from the FDA. The company expects to complete patient enrollment before the end of 2025 and plans to initiate registrational studies in 2026.

Minovia is also collecting data from two compassionate use patients with Kearns-Sayre syndrome, another mitochondrial deletion syndrome. Early data indicates improved quality of life scores for both patients.

The company recently announced a definitive business combination agreement with Launch One Acquisition Corp. Following the expected closing in late 2025, the combined company will operate as Minovia Therapeutics and trade on Nasdaq. LPAA currently trades near its 52-week high of $10.70, with a P/E ratio of 29.2. InvestingPro subscribers have access to over 30 additional financial metrics and insights about the company’s valuation.

MNV-201 is a cell therapy that uses Minovia’s proprietary Mitochondrial Augmentation Technology to add healthy mitochondria into a patient’s own stem cells, aiming to restore organ function in patients with Pearson Syndrome, for which there are currently no approved therapies. Investors following this development should note that LPAA’s next earnings report is scheduled for August 14, 2025, which may provide additional insights into the merger progress and clinical developments.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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