First mitophagy drugs: MTX325, ABBV-1088

The research record

This is the project's file for this landmark, as written and checked. Sources are listed at the end; anything not yet confirmed is marked "Not yet verified".

Who

Mission Therapeutics (UK; MTX325), funded for the patient study by the Michael J. Fox Foundation and Parkinson's UK. AbbVie (ABBV-1088), which bought Mitokinin in October 2023 for $110 million upfront.

What happened

• Idea. In landmark 15, PINK1 and parkin mark failing mitochondria for removal (mitophagy). USP30 is an enzyme that undoes that marking, so blocking it should help clearance. A PINK1 activator pushes the same pathway from the other end.
• MTX325 (NCT07630545). Multi-part Phase 1, started 30 Nov 2023, about 106 planned. Registry (updated 15 Sep 2026): recruiting; Parts 1–4 (single and multiple doses, older volunteers, PET biodistribution; 1.5–100 mg) are complete, and Part 5 (20 mg or placebo in people with Parkinson's) is in progress. Part 5 lasts 28 days with 36 people planned, per Parkinson's UK. Primary completion estimated June 2027. Mouse work showed USP30 knockout or inhibition protected dopamine neurons (Fang et al., Nat Commun 2023, DOI 10.1038/s41467-023-42876-1).
• ABBV-1088. Phase 1 in healthy adults (NCT06414798, 60 people, single dose), completed February 2025. A multiple-dose, drug-interaction and Asian pharmacokinetics study in healthy adults (NCT06579300, 72 enrolled) was terminated in September 2025 for "strategic considerations". I found no Parkinson's patient trial.

Why it moved us toward a cure

These are the first attempts to treat the mitochondrial quality-control failure behind the genes in landmark 15 with drugs in people.

Outcome

Open. Early safety stage only; the first patient data will be about safety and biomarkers.

Nix gene therapy for PINK1/PARKIN disease is being developed at NeuRA (Carolyn Sue) with Shake It Up Australia and Michael J. Fox Foundation funding from December 2022; no results or trial registration are public as of 20 Oct 2026.

Sources

• Shake It Up Australia project page (funding and status facts only)
• NCT07630545 (MTX325) · Parkinson's UK trial page · Clinical Trials Arena
• NCT06414798 (ABBV-1088) · NCT06579300 · Fang et al. 2023 · AbbVie/Mitokinin deal coverage
• Not yet verified: that ABBV-1088 is the Mitokinin PINK1 activator (the registry records do not name the mechanism; a drug database lists it as an inhibitor); Part 5 dosing progress; any other PINK1 or USP30 programmes.