- MTX325 is a first-in-class, highly potent, orally bioavailable, brain-penetrant USP30 inhibitor designed to modify the course of Parkinson’s by improving mitochondrial control
- Phase 1b ‘MTX325-101’ study – taking place in UK, Europe and Australia – will assess MTX325’s clinical proof-of-mechanism, safety and tolerability in 36 patients with mild to moderate Parkinson’s disease
- Comes after successful completion of Phase 1a study of MTX325 in healthy volunteers
CAMBRIDGE, United Kingdom, Oct. 15, 2026 (GLOBE NEWSWIRE) — Mission Therapeutics Limited (“Mission”), a clinical-stage biotechnology company discovering and developing novel therapeutics to restore cellular health and function by promoting the removal of dysfunctional mitochondria, today announced the first patient has been dosed in its Phase 1b proof-of-mechanism study of MTX325 in patients with Parkinson’s disease (PD). The patient was dosed at the Royal Liverpool University Hospital in the UK.
Dr Sarah Fritchley, Chief Development Officer of Mission Therapeutics, said: “Dosing the first Parkinson’s disease patient is a significant milestone in the development of MTX325, a potential disease-modifying treatment for this serious neurodegenerative disease, which affects almost one million people in the US and 1.2 million in Europe. Mission Therapeutics is fortunate to be working with an outstanding international team of investigators whose expertise and engagement will be invaluable as we rigorously assess MTX325’s proof-of-mechanism.”
Dr Suhail Nurbhai, Chief Medical Officer of Mission Therapeutics, said: “By improving mitochondrial quality control, MTX325 has the potential to change the course of treatment for patients with Parkinson’s disease – moving beyond management of symptoms offered by current therapies. The start of the Phase 1b study is a major step forward on our journey to making this a reality.”
MTX325 is a first-in-class, highly potent and selective, orally bioavailable and brain-penetrant inhibitor of the deubiquitinating enzyme (DUB) USP30. MTX325 has been designed to improve the clearance of dysfunctional mitochondria, support cell health and reduce inflammation — thus slowing or stopping processes thought to underpin the progression of PD. In preclinical studies, MTX325 demonstrated impact across multiple aspects of PD, including an increase in mitophagy, and beneficial effects on alpha-synuclein accumulation and nigrostriatal dopaminergic neurons. In a completed Phase 1a SAD/MAD study in healthy volunteers, MTX325 demonstrated high CNS penetration, while a PET study confirmed distribution into brain parenchyma.
The MTX325-101 Phase 1b study is designed to establish clinical proof-of-mechanism (PoM) and assess biological and pathophysiological responses using a wide range of biomarkers and imaging measures. Recruitment is ongoing, with 36 participants with mild to moderate idiopathic PD expected to enrol across the UK, Europe and Australia. A data readout is anticipated in late 2027.
For further information, please contact:
Optimum Strategic Communications:
Mary Clark | Stephen Adams | Vareen Outhonesack
Tel: +44 (0) 203 821 6420
E-mail: mission@optimumcomms.com
About Mission Therapeutics
Mission Therapeutics is a world leader in discovering and developing novel therapeutics which promote the removal of dysfunctional mitochondria, promoting cell health and function. Mission is currently developing small molecule drug, MTX325 (targeting the CNS) which, through inhibition of the mitochondrial DUB enzyme USP30, promotes clearance of dysfunctional mitochondria – consequently improving overall cellular health. Mission is backed by blue chip investors including Pfizer Venture Investments, Sofinnova Partners, Roche Venture Fund, SR One, IP Group and Rosetta Capital.
About MTX325 and USP30
MTX325 is a potent, selective, central nervous system-penetrant compound designed to improve mitochondrial quality and function by enhancing mitophagy. MTX325 inhibits USP30, a deubiquitylating enzyme localised to mitochondria that negatively regulates mitophagy. In a study titled “Knockout or inhibition of USP30 protects dopaminergic neurons in a Parkinson’s disease mouse model”, published in Nature Communications, findings demonstrated that the genetic deletion of USP30 protected dopaminergic neurons in an in vivo model of Parkinson’s, validating USP30 as a potential therapeutic target. MTX325 produced a similar in the same model, further supporting the therapeutic potential of USP30 inhibition in PD. MTX325 has composition of matter IP exclusivity through 2041.
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