Mission Therapeutics has secured $13.3 million in financing from existing investors to advance its lead candidate MTX325 through Phase Ib clinical trials in patients with Parkinson's disease. The Cambridge-based clinical-stage biotech company announced the funding will support the full execution of a proof-of-mechanism study, marking a critical step toward validating the first-in-class therapeutic's potential as a disease-modifying treatment.
The financing comes after Mission successfully completed Phase Ia studies in healthy volunteers, including recent PET scans that confirmed MTX325 adequately penetrates functional brain tissues. The company has received regulatory approval from the UK's Medicines and Healthcare products Regulatory Agency (MHRA) for the Phase Ib clinical trial, which is scheduled to begin in the first half of 2026.
Novel Mechanism Targets Mitochondrial Dysfunction
MTX325 represents a novel therapeutic approach, working by inhibiting USP30, a mitochondrial de-ubiquitylating enzyme (DUB). This inhibition increases mitochondrial ubiquitylation and promotes mitophagy – the essential cellular process for removing dysfunctional mitochondria. When cells such as neurons cannot eliminate damaged mitochondria, they begin to malfunction and die, contributing to neurodegenerative diseases.
Dr. Anker Lundemose, Executive Director at Mission Therapeutics, stated: "Thanks to this additional $13.3m from our investors, we can now make the next vital steps progressing MTX325 into PD patients with this essential Phase 1b clinical trial. This will enable us to build upon the compelling preclinical data package for MTX325, published in Nature Communications in 2023, and the results from the Phase 1a studies that we have obtained."
Strong Preclinical Evidence Base
The therapeutic rationale for MTX325 is supported by robust preclinical data published in Nature Communications in November 2023. The research, conducted by scientists at Cambridge University, Harvard University, University of Dundee, and Mission Therapeutics, provided key experimental evidence that MTX325 can modify the course of Parkinson's disease by targeting USP30.
Using both USP30 knockout mouse models and pharmacological strategies with MTX325, researchers demonstrated that USP30 inhibition led to protection against loss of dopamine and dopaminergic neurons induced by alpha-synuclein in vivo. The treatment also reduced key biomarkers of Parkinson's disease, including phosphorylated alpha-synuclein and glial cell activation.
Clinical Development Strategy
The Phase Ib trial objectives focus on demonstrating robust clinical proof-of-mechanism in Parkinson's disease patients while gathering additional safety and tolerability data. Dr. Sarah J. Fritchley, Chief Development Officer at Mission Therapeutics, explained: "The overall objectives of this Phase Ib trial are to demonstrate robust clinical proof-of-mechanism (PoM) in patients with Parkinson's disease, and to gather further information on safety and tolerability. We look forward to progressing MTX325 rapidly through clinical testing and anticipate we will have PoM data in H2 2027."
The clinical development program is further supported by a $5.2 million grant from the Michael J. Fox Foundation and Parkinson's UK, demonstrating significant backing from leading organizations in Parkinson's disease research.
Broader Pipeline and Investment Support
Mission Therapeutics is developing two small molecule drugs targeting USP30: MTX325 for central nervous system applications and MTX652 for peripheral conditions. The company's investor base includes blue-chip pharmaceutical venture funds such as Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group, and Rosetta Capital.
Dr. James B. Summers, Chairman of Mission Therapeutics, noted: "This latest financing round is a sign of our investors' confidence in the Company and the enormous potential of MTX325 as a first-in-class, disease-modifying treatment for PD."
MTX325 has composition of matter intellectual property exclusivity extending through 2041, providing substantial commercial protection for the potential therapy. The drug candidate has demonstrated impact on multiple aspects of Parkinson's disease pathophysiology in preclinical studies, including mitochondrial dysfunction, alpha-synuclein accumulation, and loss of nigrostriatal dopaminergic neurons.
