Floatz Rating
CC43/100
Confidence
Indicative
Sponsor
Michio Hirano, MD
Modality
Small molecule
Development Phase
Phase 1
Status
Active
Evidence ledger · v0.2
Clinical Evidence
Clinical track record: trial progression, reported outcomes, safety signals, and endpoint quality.
28Indicative
Detailed axis rationale is planned and will be published soon.
| Trial | Phase | Status | N | Primary endpoint | Readout |
|---|---|---|---|---|---|
NCT05241262CT.gov Study of N-acetylcysteine in the Treatment of Patients With the m.3243A>G Mutation and Low Brain Glutathione Levels | — | Recruiting | — | — | — |
Competitive Position
Competitive setting: how crowded the indication is, class-level failures, and timing against rivals.
95Indicative
Detailed axis rationale is planned and will be published soon.
Same indication · Mitochondrial Disease
| Asset | Sponsor | Phase | Rating |
|---|---|---|---|
| Acetylcysteine (this asset) | Michio Hirano, MD | P1 | CC · 43 |
| Sonlicromanol | Khondrion BV | P3 | BB |
| Trimetazidine | Steen Hvitfeldt Poulsen | P4 | BB |
| Sodium Chloride | University Hospital, Strasbourg, France | P4 | BB |
| ARG | University Hospital, Strasbourg, France | P4 | BB |
| Efavirenz | The Cleveland Clinic | P4 | BB |
| NORVIR | The Cleveland Clinic | P4 | BB |
| Atazanavir | The Cleveland Clinic | P4 | B |
| Mitoquinol Mesilate | Mclean Hospital | P2/3 | B |
+8 more in the Mitochondrial Disease cohort
Other indications for Acetylcysteine
| Indication | Sponsor | Phase | Rating |
|---|---|---|---|
| Idiopathic Pulmonary Fibrosis | — | P4 | BBB · 74 |
| Schizophreniform Disorder | — | P4 | BBB · 74 |
| Pulmonary Fibrosis | — | P3 | BB · 70 |
Scientific Foundation
Strength of the underlying biology: target validation, tractability, modality fit, and how related mechanisms have fared.
NR
Planned for methodology v0.2.
Development Feasibility
How realistically the program can be executed, drawing on modality precedent, enrollment dynamics, and sponsor delivery.
NR
Planned for methodology v0.2.
Commercial Opportunity
Commercial prize: addressable population, unmet need, and the value case for the indication.
NR
Planned for methodology v0.2.
IP & Exclusivity
Exclusivity position, covering patent protection and freedom-to-operate runway.
NR
Planned for methodology v0.2.
Manufacturing & Supply
Manufacturing and supply readiness, driven by modality process and scale-up risk.
NR
Planned for methodology v0.2.
Related assets
Citation
Floatz Terminal. Acetylcysteine in Mitochondrial Disease. Methodology v0.2. Rated under v0.2 effective July 8, 2026. Last refreshed July 8, 2026. Accessed July 14, 2026. https://terminal.floatz.ai/assets/acetylcysteine-mitochondrial-disease
Are you the sponsor?
Submit your data room for a Verified Rating. The public-data rating remains visible alongside.
Contact →